John Davy of Hancock marks two birthdays. The first isNov. 16, the day he was born in 1941. The second is Jan. 6,the day in 2014 when he received a lifesaving stem-cell transplantthanks to a complete stranger.

Now, John and his wife Sandhy Kale have become advocates for Be the Match, the national stem cell registry that found John his rare genetic match.

How often in the world do you get to save someones life? Davy asked. You fantasize about it sometimes. Heres an opportunity for anyone between 18 and 44 to do just that.

Davy said he began feeling abnormally tired sometime in 2013. One day, he walked to the mailbox, only a few hundred feet away from his front door, and had to stop several times on his way back to his house.

I said, Thats not me. Theres something off here, Davy said.

Davy went in to the hospital for some testing, and after a few false starts looking at his heart and running stress tests, doctors performed a Complete Blood Count, or CBC.

My blood count was so low, it wouldnt support life, he said.

Thats when Davy received his diagnosis. Myelodysplastic syndrome, or MDS, a form of blood cancer.

My first thought was, OK, what are we going to do about this? Davy said. Thats when the doctor told me there was no cure.

MDS cannot be cured through usual chemotherapy or radiation treatments. However, it can be treated with bone marrow or, as with Davy, the transplant of stem cells.

After receiving a second opinion, and speaking with a doctor experienced with stem cell transplants, Davy went on the national stem cell registry, known as Be the Match.

He was told he might have to wait upwards of a year before finding his match. But Davy got lucky in only three months, a viable donor joined the registry.

Davy knows little about the man who saved his life. He was 30 years old at the time, and a member of the United States military. Be the Match allows donors and patients to connect, if both sides are interested, but while John said he would love to shake the mans hand, his donor has wished to remain anonymous.

If I could speak to him, I would thank him profusely. For someone to be that generous, to donate to someone that hes never met, is astounding, Davy said.

Joining the Be the Match registry is as simple as swabbing a cheek.

Your genetic profile goes into the system, and, if donors are found to be a match to any patients waiting for transplants, only then are they called to go through the donation process.

There are two ways to donate stem cells. In either case, the donor will first undergo two injections to increase the production of their stem cells. In the first type of donation procedure, liquid bone marrow is extracted using a needle while the donor is under anesthesia. But the much more common way to donate used about 80 percent of the time is through a blood donation.

Similar to the process for donating plasma, the donor has blood drawn, it is cycled by a machine to remove only the stem cells, and the remaining blood is returned to the donor.

The recipient of the stem cells has to undergo a process to suppress their immune system, and the donated stem cells are given to the patient.

Because the immune system has to be repressed to accept the new cells, there is danger in the procedure, and even those who successfully accept the new stem cells can experience side effects of graft-verses-host reactions.

There is no guarantee, Kale said. This is a chance. You can take it if you want. Even if it buys you four or five years, you might get to see your kids graduating, your grandkids grow up. It was worth it to us.

And for Davy, they said, there was no other option. He accepted the risk, and said hes one of the lucky ones he had one minor reaction resulting in a rash across his chest, but overall, since his transplant, he has been able to resume a normal life. Today, seven years later, he is on no medications, and has no restrictions for how he can live his life.

It is that new lease on life that Davy said convinced himself and Sandhy that they had to become involved with Be the Match on a level besides being a recipient of their services. The two are now advocates for the system, traveling to drives to tell their story, and Davy acts as a support person for patients who may be recipients of transplants, telling them about what to expect in the process.

Its crucial, Davy said, to get as many people on the registry as possible. Because matches work on how genetically compatible two people are, people of similar ethnic backgrounds are more likely to match, and your ethnicity greatly impacts the likelihood of finding a good match.

White patients are the most likely to find a match within the system, at a rate of 79 percent. Native Americans have a 60 percent chance, Hispanic people a 48 percent, Asian 47 percent, and Black people only 29 percent.

Thats why Sandhy and I try to get as many people involved as we can, Davy said. The more people in the registry, the better chance you have.

Be the Match currently has a donor drive scheduled for Aug. 14 from 10 a.m. to 2 p.m. at E. Paul Community Center at 61 South Street in Troy. To join the registry you must be between the ages of 18 and 44 and be in good general health, and committed to donating to anyone in need. If you cannot attend the physical drive, a free cheek swab kit will be mailed to you. If you are interested in a kit, text TroyFD to 61474.

Excerpt from:
Hancock man advocates for national bone marrow and stem cell registry that saved his life - Monadnock Ledger Transcript

Bone Marrow Stem Cells Comments Off on Hancock man advocates for national bone marrow and stem cell registry that saved his life – Monadnock Ledger Transcript | August 6th, 2021

DUBLIN--(BUSINESS WIRE)--The "Hematopoietic Stem Cell Transplantation (HSCT) Market - Size, Share, Outlook, and Opportunity Analysis, 2019 - 2027" report has been added to ResearchAndMarkets.com's offering.

The global hematopoietic stem cell transplantation market is expected to witness significant growth during the forecast period owing to the increasing prevalence of leukemia and lymphoma. According to Center for Disease Control and Prevention (CDC), in the U.S., around 45,360 people were diagnosed with leukemia in 2013, leading to 23,549 fatalities (13,625 men and 9,924 women). According to the same source the condition is more prevalent among men than women. Leukemia accounts for around 3% of all new cancer cases.

Hematopoietic stem cell transplantation is a procedure in which multipotent hematopoietic stem cells sourced from peripheral blood cells, bone marrow, or umbilical cord blood are transplanted into the patient. Hematopoietic stem cell transplantation is commonly used in the treatment of lymphoma (Hodgkin, Non-Hodgkin), leukemia, multiple myeloma, thalassemia, sickle cell anemia, and osteoporosis. It includes two transplantation sources; 1) autologous, that uses stem cells from the patient's own body, 2) and allogeneic that sources stem cells from a donor's body. According to World Health Organization (WHO), over 50,000 hematopoietic stem cell transplantation procedures are carried out globally, every year and this number is expected to increase over the years.

Company Profiles

Key features of the study:

Key Topics Covered:

1. Research Objectives and Assumptions

2. Market Overview

3. Market Dynamics, Regulations, and Trends Analysis

4. Impact Analysis of COVID-19

5. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Transplant Type, 2016 - 2027, (US$ Million)

6. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Indication, 2016 - 2027, (US$ Million)

7. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Application, 2016 - 2027, (US$ Million)

8. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Region, 2016 - 2027, (US$ Million)

9. Competitive Landscape

10. Section

For more information about this report visit https://www.researchandmarkets.com/r/jtneqg

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Global Hematopoietic Stem Cell Transplantation Market to 2027 - Size, Share, Outlook, and Opportunity Analysis - ResearchAndMarkets.com - Business...

Bone Marrow Stem Cells Comments Off on Global Hematopoietic Stem Cell Transplantation Market to 2027 – Size, Share, Outlook, and Opportunity Analysis – ResearchAndMarkets.com – Business… | August 6th, 2021

A study released inSTEM CELLS Translational Medicinehas confirmed the safety of a novel type of cellular therapy for knee pain caused by osteoarthritis. Conducted by a multi-institutional team of researchers in Japan who had developed the new therapy, the study was designed to confirm that their treatment which involves transplanting the patients own mesenchymal stem cells (MSCs) into the affected knee did not cause tumors.

The results showed that five years after transplantation, osteoarthritis-related tears to the knee meniscus had healed and, just as importantly, none of the patients experienced any serious side effects from the treatment. The meniscus is a crescent-shaped cartilage in the knee joint that plays a role in shock absorption. Age-related damage to the meniscus often leads to the progression of osteoarthritis of the knee.

Chronic knee pain is a major issue for the aging, affecting approximately 25 percent of all adults, according to the Centers for Disease Control and Prevention (CDC). Osteoarthritis is the most common cause of this condition in people aged 50 and older. Along with pain, which can be debilitating, knee problems can significantly affect the persons mobility and quality of life.

Knee replacement surgery is the gold standard of treatment, with the majority of people experiencing a dramatic reduction in pain and, thus, improvement in their ability to live a normal life. However, though rare, such surgery does come with risks such as the possibility of infection.

Lead investigator Mitsuru Mizuno, DVM, Ph.D. and corresponding author Ichiro Sekiya, M.D., Ph.D. Credit: AlphaMed Press

Cellular therapies are showing great potential as a less invasive way to treat difficult-to-heal knee injuries. The team behind the current study, which included researchers from Tokyo Medical and Dental University, Kyoto University and Kazusa DNA Research Institute, recently developed a therapy involving the transplantation of MSCs derived from the knees soft tissue (the synovium) into the injured meniscus. MSCs are multipotent adult stem cells present in the umbilical cord, bone marrow, fat, dental and other body tissues. Their ability to secrete biologically active molecules that exert beneficial effects on injured tissues makes them a promising target in regenerative medicine.

But some stem cell treatments have been known to cause tumors, which is why the team wanted to ensure that their therapy was free of any negative side effects. In particular, they wanted to investigate the safety of any MSCs that might show a type of chromosomal disorder called trisomy 7.

Trisomy 7 occurs frequently in patients with severe knee disease such as osteoarthritis. The detection of trisomy 7 in epithelial cells has been associated with tumor formation. However, the safety of these cells after transplantation has not been investigated. Thats what we wanted to learn from this study, said corresponding author Ichiro Sekiya, M.D., Ph. D., director and professor of the Center for Stem Cell and Regenerative Medicine (CSCRM) at Tokyo Medical and Dental University.

Mitsuru Mizuno, DVM, Ph.D., assistant professor with CSCRM, served as the studys lead investigator. He reported on the results. We recruited 10 patients for the study and transplanted their own stem cells into the affected knee joints, then followed up with MRIs over the next five years. The images revealed that tears in the patients knee meniscus were obscured three years after transplantation. We also identified trisomy 7 in three of the patients, yet no serious adverse events including tumor formation were observed in any of them.

Dr. Sekiya added, Keep in mind that these were autologous MSCs used in our study, which means that the transplanted MSCs came from the patients themselves. Any problems that might arise in the case of allogeneic cells, which are donated by someone other than the patient, still need to be determined.

Nevertheless, we believe that these data suggest that MSCs with trisomy 7 are safe for transplantation into human knees and show much promise in treating osteoarthritis.

This study highlights the ability of a patients own stem cells to potentially heal torn cartilage in the knee, said Anthony Atala, M.D., Editor-in-Chief ofSTEM CELLS Translational Medicineand director of the Wake Forest Institute for Regenerative Medicine. These outcomes suggest a potential approach that could change the overall physical health of patients who suffer from osteoarthritis and experience debilitating joint pain. We look forward to the continuation of this research to further document clinical efficacy.

Reference: Transplantation of human autologous synovial mesenchymal stem cells with trisomy 7 into the knee joint and 5 years of follow-up by Mitsuru Mizuno, Kentaro Endo, Hisako Katano, Naoki Amano, Masaki Nomura, Yoshinori Hasegawa, Nobutake Ozeki, Hideyuki Koga, Naoko Takasu, Osamu Ohara, Tomohiro Morio and Ichiro Sekiya, 3 August 2021, STEM CELLS Translational Medicine.DOI: 10.1002/sctm.20-0491

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Safety of Stem Cell Therapy for Chronic Knee Pain Confirmed in New Study - SciTechDaily

Bone Marrow Stem Cells Comments Off on Safety of Stem Cell Therapy for Chronic Knee Pain Confirmed in New Study – SciTechDaily | August 6th, 2021

Dublin, Aug. 03, 2021 (GLOBE NEWSWIRE) -- The "Hematopoietic Stem Cell Transplantation (HSCT) Market - Size, Share, Outlook, and Opportunity Analysis, 2019 - 2027" report has been added to ResearchAndMarkets.com's offering.

Hematopoietic stem cell transplantation is a procedure in which multipotent hematopoietic stem cells sourced from peripheral blood cells, bone marrow, or umbilical cord blood are transplanted into the patient. Hematopoietic stem cell transplantation is commonly used in the treatment of lymphoma (Hodgkin, Non-Hodgkin), leukemia, multiple myeloma, thalassemia, sickle cell anemia, and osteoporosis. It includes two transplantation sources; 1) autologous, that uses stem cells from the patient's own body, 2) and allogeneic that sources stem cells from a donor's body. According to World Health Organization (WHO), over 50,000 hematopoietic stem cell transplantation procedures are carried out globally, every year and this number is expected to increase over the years.

Market Dynamics

The global hematopoietic stem cell transplantation market is expected to witness significant growth during the forecast period owing to the increasing prevalence of leukemia and lymphoma. According to Center for Disease Control and Prevention (CDC), in the U.S., around 45,360 people were diagnosed with leukemia in 2013, leading to 23,549 fatalities (13,625 men and 9,924 women). According to the same source the condition is more prevalent among men than women. Leukemia accounts for around 3% of all new cancer cases.

Key features of the study:

This report provides in-depth analysis of the global hematopoietic stem cell transplantation market, market size (US$ Mn), and compound annual growth rate (CAGR %) for the forecast period 2020-2027, considering 2019 as the base year

It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market

This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by leading players

It profiles key players in the global hematopoietic stem cell transplantation market based on the following parameters - company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments, and strategies

Key players covered as a part of this study are Pluristem Therapeutics Inc., CellGenix GmbH, Regen Biopharma Inc., Lonza Group, Kiadis Pharma, Taiga Biotechnologies, Inc., Takeda Pharmaceutical Company Limited, Escape Therapeutics, Inc., Bluebird Bio, Talaris Therapeutics, Inc., Marker Therapeutics Inc., and Stempeutics Research Pvt Ltd.

Insights from this report would allow marketers and management authorities of companies to make informed decision with respect to future product launches, government initiatives, technological upgradation, market expansion, and marketing tactics

The global hematopoietic stem cell transplantation market report caters to various stakeholders in this industry, including investors, product manufacturers, distributors, and suppliers in the hematopoietic stem cell transplantation market, research and consulting firms, new entrants, and financial analysts.

Key Topics Covered:

1. Research Objectives and Assumptions

Story continues

2. Market Overview

3. Market Dynamics, Regulations, and Trends Analysis

Market Dynamics

Drivers

Restraints

Market Opportunities

Impact Analysis

Key Developments

Pipeline Analysis

PEST Analysis

Reimbursement Scenario

Regulatory Scenario

Epidemiology

Government Initiatives

Treatment Algorithm

4. Impact Analysis of COVID-19

5. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Transplant Type, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Autologous

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Allogeneic

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

6. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Indication, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Acute Myeloid Leukemia (AML)

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Acute Lymphoblastic Leukemia (ALL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Hodgkin lymphoma (HL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Non-Hodgkin Lymphoma (NHL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Multiple Myeloma (MM)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Other Non-malignant Disorders

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

7. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Application, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Bone Marrow Transplant (BMT)

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Peripheral Blood Stem Cells Transplant (PBSCT)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Cord Blood Transplant (CBT)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

8. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Region, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, By Region, 2020 and 2027 (%)

Y-o-Y Growth Analysis, By Region, 2017 - 2027

Regional Trends

North America

Market Size and Forecast, By Transplant Type, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Indication, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Application, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Country, 2016 - 2027, (US$ Million)

U.S.

Canada

Europe

Market Size and Forecast, By Transplant Type, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Indication, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Application, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Country, 2016 - 2027, (US$ Million)

U.K.

Germany

Italy

France

Link:
Insights on the Hematopoietic Stem Cell Transplantation Global Market to 2027 - Key Drivers, Restraints and Opportunities - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Insights on the Hematopoietic Stem Cell Transplantation Global Market to 2027 – Key Drivers, Restraints and Opportunities – Yahoo Finance | August 6th, 2021

Abstract: Global Stem Cell Banking Market to Reach US$11. 3 Billion by the Year 2027. Amid the COVID-19 crisis, the global market for Stem Cell Banking estimated at US$7.

New York, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell Banking Industry" - https://www.reportlinker.com/p05799719/?utm_source=GNW 1 Billion in the year 2020, is projected to reach a revised size of US$11.3 Billion by 2027, growing at a CAGR of 6.8% over the analysis period 2020-2027.Placental and Cord Blood Stem Cells, one of the segments analyzed in the report, is projected to grow at a 7.4% CAGR to reach US$7.3 Billion by the end of the analysis period.After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the Adipose Tissue-Derived Stem Cells (ADSCS) segment is readjusted to a revised 6.3% CAGR for the next 7-year period. This segment currently accounts for a 6.6% share of the global Stem Cell Banking market.

The U.S. Accounts for Over 29.5% of Global Market Size in 2020, While China is Forecast to Grow at a 6.4% CAGR for the Period of 2020-2027

The Stem Cell Banking market in the U.S. is estimated at US$2.1 Billion in the year 2020. The country currently accounts for a 29.55% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$2 Billion in the year 2027 trailing a CAGR of 6.4% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 6.4% and 5.5% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 5.5% CAGR while Rest of European market (as defined in the study) will reach US$2 Billion by the year 2027.

Bone Marrow-Derived Stem Cells (BMSCS) Segment Corners a 10.3% Share in 2020

In the global Bone Marrow-Derived Stem Cells (BMSCS) segment, USA, Canada, Japan, China and Europe will drive the 5.4% CAGR estimated for this segment. These regional markets accounting for a combined market size of US$592.8 Million in the year 2020 will reach a projected size of US$858.7 Million by the close of the analysis period. China will remain among the fastest growing in this cluster of regional markets. Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$1.3 Billion by the year 2027.

Select Competitors (Total 78 Featured)

Story continues

Cord Blood Registry

CordLife Group Ltd.

Cryo-Cell International, Inc.

Cryo-Save AG

Global Cord Blood Corporation

LifeCell International Pvt., Ltd.

Smart Cells International Ltd.

StemCyte Inc.

ViaCord

Vita 34 AG

Read the full report: https://www.reportlinker.com/p05799719/?utm_source=GNW

I. METHODOLOGY

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Influencer Market Insights World Market Trajectories Impact of Covid-19 and a Looming Global Recession Stem Cells, Application Areas, and the Different Types - A Prelude Applications of Stem Cells Types of Stem Cells Cord Blood Umbilical Cord Tissue Bone Marrow Stem Cells Adipose-Derived Stem Cells (ADSCs) Number of Clinical Trials Using Adipose Stem Cells: 2007-2018 Number of Adipose Stem Cell Trials by Phase: 2007 to 2018 Human Embryo-Derived Stem Cells (HESCS) Global Stem Cell Banking Market Poised for a Rapid Growth Developed Regions Lead, Emerging Economies to Spearhead Future Growth List of Family Cord Blood Banks in the US Placental and Cord Blood Banks Dominate the Cord Blood Banking Market Global Number of Annual Newborns and Private Cord Blood Banks Global Select Leading Cord Blood Banks Based on Inventory A Peek into China?s Cord Blood Banking Industry Evolving Landscape of Cord Blood Banking Industry Placental Stem Cells and Potential Clinical Applications EXHIBIT 1: Global Cord Blood Banking Market Share Breakdown (%) by Bank Type: 2019 EXHIBIT 2: US Cord Blood Banking Market by Bank Type (in %) for 2019 Changing Business Models for Stem Cell Banking

2. FOCUS ON SELECT PLAYERS Cord Blood Registry (CBR) Systems, Inc. (USA) Cordlife Group Limited (Singapore) Cryo Stemcell Private Limited (India) Cryo-Cell International, Inc. (USA) Cryoviva Biotech Private Limited (India) Global Cord Blood Corporation (China) LifeCell International Pvt. Ltd (India) Smart Cells International Ltd. (UK) StemCyte (USA) Takara Bio Europe AB (Europe) ViaCord (US) Vita34 AG (Germany)

3. MARKET TRENDS & DRIVERS Increasing Investments in Stem Cell-Based Research Widen Prospects for Stem Cell Banking Market EXHIBIT 3: Stem Cell Research Funding in the US (in US$ Million) for the Years 2011 through 2017 Stem Cell Research Policies Impact Funding Volumes Adult Stem Cell Research Gains Traction, Accelerating Research Funding Adult Stem Cells Vs. Embryonic Stem Cells: A Comparison Embryonic Stem Cell Research Bogged Down by Ethical Issues & Technical Hurdles Induced Pluripotent Stem Cell (iPSC) Research: The Latest Vertical Sustained Emphasis on Mesenchymal Stem Cell Research Emergence of Advanced Technologies for Stem Cell Preservation, Storage and Processing Augurs Well for Market Growth Growing Incidence of Major Diseases to Boost the Demand for Stem Cells, Driving Stem Cell Banking EXHIBIT 4: Worldwide Incidence of Cancer (2012, 2018 & 2040): Number of New Cases Diagnosed Table 8: World Cancer Incidence by Cancer Type (2018): Number of New Cancer Cases Reported (in Thousands) for Breast, Cervix uteri, Colorectum, Liver, Lung, Oesophagus, Prostate, Stomach and Others

Table 9: Fatalities by Heart Conditions - Estimated Percentage Breakdown for Cardiovascular Disease, Ischemic Heart Disease, Stroke, and Others

Table 10: Global Annual Medical Cost of CVD in US$ Billion (2010-2030) Ageing Demographics to Drive Demand for Stem Cell Banking Global Aging Population Statistics - Opportunity Indicators Table 3: Elderly Population (60+ Years) as a Percentage of Total Population (2017 & 2050)

Table 4: Global Aging Population (2017 & 2050): Population of 60+ Individuals in ?000s and as a Percentage of Total Population

Table 5: Life Expectancy for Select Countries in Number of Years: 2018 Bone Marrow Stem Cells Market on a Rapid Growth Path, Spurring the Need for Stem Cell Banking Development of Regenerative Medicine Accelerates Demand for Mesenchymal Stem Cell Banking Table 2: Global Regenerative Medicines Market by Category (2019): Percentage Breakdown for Biomaterials, Stem Cell Therapies and Tissue Engineering Rise in Volume of Orthopedic Procedures Boosts Prospects for Stem Cell Banking Table 1: Global Orthopedic Surgical Procedure Volume (2010-2020) (in Million) Increasing Demand for Stem Cell Based Bone Grafts: Promising Growth Ahead for Stem Cell Banking Rise in the Number of Hematopoietic Stem Cell Transplantation Procedures Propels Market Expansion Hematopoietic Stem Cell Storage Dental Mesenchymal Stem Cells: An Evolving Niche Therapeutic Potential of Dental Pulp Stem Cells (DPSCs) in Various Diseases High Operational Costs of Stem Cell Banking - A Key Market Restraint

4. GLOBAL MARKET PERSPECTIVE Table 1: World Current & Future Analysis for Stem Cell Banking by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 2: World 7-Year Perspective for Stem Cell Banking by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2020 & 2027

Table 3: World Current & Future Analysis for Placental and Cord Blood Stem Cells by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 4: World 7-Year Perspective for Placental and Cord Blood Stem Cells by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 5: World Current & Future Analysis for Adipose Tissue-Derived Stem Cells (ADSCS) by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 6: World 7-Year Perspective for Adipose Tissue-Derived Stem Cells (ADSCS) by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 7: World Current & Future Analysis for Bone Marrow-Derived Stem Cells (BMSCS) by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 8: World 7-Year Perspective for Bone Marrow-Derived Stem Cells (BMSCS) by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 9: World Current & Future Analysis for Human Embryo-Derived Stem Cells (HESCS) by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 10: World 7-Year Perspective for Human Embryo-Derived Stem Cells (HESCS) by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 11: World Current & Future Analysis for Dental Pulp-Derived Stem Cells (DPSCS) by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 12: World 7-Year Perspective for Dental Pulp-Derived Stem Cells (DPSCS) by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 13: World Current & Future Analysis for Other Stem Cell Sources by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 14: World 7-Year Perspective for Other Stem Cell Sources by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 15: World Current & Future Analysis for Sample Preservation and Storage by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 16: World 7-Year Perspective for Sample Preservation and Storage by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 17: World Current & Future Analysis for Sample Analysis by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 18: World 7-Year Perspective for Sample Analysis by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 19: World Current & Future Analysis for Sample Processing by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 20: World 7-Year Perspective for Sample Processing by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 21: World Current & Future Analysis for Sample Collection and Transportation by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 22: World 7-Year Perspective for Sample Collection and Transportation by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 23: World Current & Future Analysis for Personalized Banking Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 24: World 7-Year Perspective for Personalized Banking Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 25: World Current & Future Analysis for Research Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 26: World 7-Year Perspective for Research Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

Table 27: World Current & Future Analysis for Clinical Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 28: World 7-Year Perspective for Clinical Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2020 & 2027

III. MARKET ANALYSIS

UNITED STATES Table 29: USA Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 30: USA 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 31: USA Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 32: USA 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 33: USA Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 34: USA 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

CANADA Table 35: Canada Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 36: Canada 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 37: Canada Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 38: Canada 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 39: Canada Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 40: Canada 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

JAPAN Table 41: Japan Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 42: Japan 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 43: Japan Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 44: Japan 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 45: Japan Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 46: Japan 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

CHINA Table 47: China Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 48: China 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 49: China Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 50: China 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 51: China Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 52: China 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

EUROPE Table 53: Europe Current & Future Analysis for Stem Cell Banking by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Million for Years 2020 through 2027 and % CAGR

Table 54: Europe 7-Year Perspective for Stem Cell Banking by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2020 & 2027

Table 55: Europe Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 56: Europe 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 57: Europe Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 58: Europe 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 59: Europe Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 60: Europe 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

FRANCE Table 61: France Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 62: France 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 63: France Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 64: France 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 65: France Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 66: France 7-Year Perspective for Stem Cell Banking by Application - Percentage Breakdown of Value Sales for Personalized Banking Applications , Research Applications and Clinical Applications for the Years 2020 & 2027

GERMANY Table 67: Germany Current & Future Analysis for Stem Cell Banking by Source - Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 68: Germany 7-Year Perspective for Stem Cell Banking by Source - Percentage Breakdown of Value Sales for Placental and Cord Blood Stem Cells, Adipose Tissue-Derived Stem Cells (ADSCS), Bone Marrow-Derived Stem Cells (BMSCS), Human Embryo-Derived Stem Cells (HESCS), Dental Pulp-Derived Stem Cells (DPSCS) and Other Stem Cell Sources for the Years 2020 & 2027

Table 69: Germany Current & Future Analysis for Stem Cell Banking by Service Type - Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Table 70: Germany 7-Year Perspective for Stem Cell Banking by Service Type - Percentage Breakdown of Value Sales for Sample Preservation and Storage, Sample Analysis, Sample Processing and Sample Collection and Transportation for the Years 2020 & 2027

Table 71: Germany Current & Future Analysis for Stem Cell Banking by Application - Personalized Banking Applications , Research Applications and Clinical Applications - Independent Analysis of Annual Sales in US$ Million for the Years 2020 through 2027 and % CAGR

Continued here:
Global Stem Cell Banking Market to Reach US$11.3 Billion by the Year 2027 - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Global Stem Cell Banking Market to Reach US$11.3 Billion by the Year 2027 – Yahoo Finance | August 6th, 2021

A mum-of-three and the hospital chaplain she befriended when she received a bone marrow transplant two years ago have enjoyed a tearful reunion after discovering he was the donor who saved her life.

Clergyman Mario Sant, 39, is one of two hospital chaplains stationed in London as part of a joint venture by the Maltese government and the Catholic Church to support patients sent to the UK for procedures which cannot be performed on the tiny island.

Taking the position six years ago, Maltese national Mario was moved by the plight of a five-year-old boy who needed the same transplant at the famous Great Ormond Street Hospital in 2018 to become a bone marrow donor.

A chaplain based in the UK, Mario was moved by a brave boy to donate stem cells (Collect/PA Real Life).

Sadly, he was too late to help the boy, who died a few months later, but he joined the international database DKMS as a bone marrow donor in December 2018.

In the meantime, he met leukaemia patient Agnes Vella, 59, a mum of three from Malta, in March 2019, who needed stem cells from bone marrow to stop her cancer from returning and they bonded, according to Mario, who said: We got on immediately.

She was at the Royal Marsden in London, and we joked that I could be her donor, as I was called to donate as she arrived.

He added: But her records said the donor was English and I was born in Malta, so we didnt think it was me.

Plus, I donated at a different hospital, so it just didnt fit. I think we both hoped and joked about it, but we thought it wasnt possible.

Neither Mario nor Agnes, a housewife, whose husband Francis, 65, is a retired freight worker, gave it a second thought although their friendship blossomed, and they stayed in touch.

Mario has been living in London for six years (Collect/PA Real Life).

Then, in May 2020, when she felt compelled to thank the donor who had saved her life emailing the DKMS asking if she could contact the stranger that helped her she and Mario were in for a gigantic surprise.

Records revealed that Agnes guardian angel was in fact the hospital chaplain who had become her friend.

It was amazing to discover that I was Agnes donor, said Mario.

Story continues

He added: The work I do is very special. We are there for people during their joy and sadness.

When Agnes called saying she had asked for the details to be released and I got the email instantly asking if I wanted to give the woman I donated to my details, it all added up.

It was amazing, from that moment we knew I was her donor. We just couldnt believe that we had unwittingly shared such a special journey together.

Mario with Agnes on the day of her transplant in March 2018 (Collect/PA Real Life).

Meanwhile, Mario, who has been living in London and supporting Maltese patients for six years, says the amazing news is a poignant reminder of the little boy who inspired him to donate.

He said: Sadly, he had leukaemia and he didnt make it, but its all thanks to this child that I donated.

I spent a lot of time with him and his family at Great Ormond Street.

He added: He was so brave. He needed another bone marrow transplant, but they were struggling to find a match.

I just thought to myself, Why dont I donate? So, I registered, but I didnt realise it can take nine to 10 months to become a bone marrow donor.

I couldnt help the child, but I could help others.

Agnes Vella with her husband Francis, and three children (Collect/PA Real Life).

And, three months after signing up to DKMS, an international non-profit bone marrow donor centre, he was called to donate.

He said: Over that time, I continued working and helping other patients and thats how I met Agnes.

I love my job. London is one of the nicest cities Ive lived in and the work I do is very special.

90% of stem cell cases are taken from the bloodstream

At any one time there are around 2,000 people in the UK in need of a blood stem cell transplant

By January 2021 two million people had registered to be blood stem cell donors in the UK

He added: Maltese patients come over for treatment and so, as a religious state, the government provides two chaplains to help them through their treatment.

For a lot of patients were the only family they have during some dark times, so its really special to be part of it.

For Agnes, who was in remission from leukaemia after previously surviving two bouts of breast cancer, the bone marrow transplant was essential to stop her disease from returning within a year.

Agnes and Francis Vella (Collect/PA Real Life).

And Mario, who donated his own stem cells at Londons Kings College Hospital the day before her op, was there to hold her hand as she was prepared for the procedure at the Royal Marsden.

The transplant was a success and four months later, Agnes returned home to Malta.

But the friends stayed in touch, chatting by phone every week.

And when they discovered he was her donor, their thoughts immediately went to arranging a reunion as soon as Covid travel restrictions allowed it.

So, on July 31, they finally got to have the hug of a lifetime, when they met for the first time since her transplant, at Agnes home in Malta.

I was so excited to see Agnes, said Mario.

We hadnt been able to meet since we found out because of the pandemic. We chat all the time, but we wanted to meet in person.

We laugh that we must be related now, because I was a match. Its crazy to think that in an international database two people from such a small island could be a match.

Im just so grateful shes healthy.

While Agnes was ecstatic to meet up with not just her friend, but the donor who saved her life.

She said: I met Mario when I went to London for treatment. We met every day. He would even come by at weekends and we would have dinner together or a little party with the other patients.

It was really special. We became like a little family.

Mario and Agnes reunited August 2021 (Collect/PA Real Life).

She added: The staff at the Royal Marsden and at the Sir Anthony Mamo oncology centre, where I was on the haematology ward, in Malta took such good care of me, too Im so grateful to them.

When I arrived in London, Mario told me he had been called to donate. But we never thought it would be for me. I was told my donor was English, and though Mario lives in England, hes Maltese, so we felt sure it couldnt be him.

And she is keen for the lifesaving DKMS register, which also operates in Germany, India, Chile, Poland, Africa and the United States to operate in Malta too.

She said: My family wanted to register but they cant as they dont have UK addresses that was why Mario was able to donate.

He saved my life, Im so thankful to him.

MUST PAR: Taking the first steps to register as a potential blood stem cell donor can be done from the comfort of your own home. If you are aged between 17-55 and in good health you can sign up for a home swab kit online at https://www.dkms.org.uk/register-now. Your swabs can then be returned with the enclosed pre-paid envelope to DKMS in order to ensure that your details are added to the UKs aligned stem cell register. [END]

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Mums tearful reunion with hospital chaplain she befriended during a bone marrow transplant in London after surprise discovery that he was her donor -...

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August2, 20215 min read

Opinions expressed by Entrepreneur contributors are their own.

A healthy spine must be strong enough to support your entire body, yet flexible enough to allow you to move your limbs. Thats why your spine isnt just one bone, but an intricately designed set of smaller bones called vertebrae each separated by a disc of cartilage.

These discs cushion each vertebra, so they dont grind against each other and cause pain. When degenerative disc disorders set in, however, these cushions wear out. But patients can now take advantage of advanced stem-cell therapy that can heal disc tissue and reduce inflammation, alleviate chronic pain and restore flexibility and range of motion.

While surgery and medications may be treatment options, surgical procedures can be risky and many patients cannot tolerate the side effects of many medications.

Related:This Is HowStem-CellTherapy Treats Serious Brain Injuries

Thats why more patients choose stem-cell therapy a procedure that takes advantage of the bodys natural healing processes. Discover how stem-cell therapy can help you heal more quickly and enjoy a more active lifestylewith less pain.

Advanced stem-cell treatments can help a range of issues:

Another remarkable aspect of the human body is that it actually knows how to heal itself, which is why the latest advancements in stem-cell therapy offerhope to more patients to relieve pain without the need for surgery or medications that can lead to serious side effects.

Because stem cells that come from your bone marrow have the potential to become any type of cell, the body turns those stem cells into specific cells needed to heal various tissues. If you burn your skin, for example, stem cells are turned into new skin cells. If youve injured a muscle, your body uses stem cells to regenerate muscle tissue. And as discs deteriorate, your body can use stem cells to create new disc tissue to rehydrate those discs and return them to a normal shape easing pain and inflammation.

Unfortunately, stem-cell production begins to decline as we age. But with an infusion of millions of fresh new stem cells, the body can use those cells to quickly stimulate healing without the need to go under the knife or risk serious side effects from steroids or the consequences of using addictive pain killers.

Related:Former Quarterback Jim McMahon Calls AdvancedStem-CellTreatment 'Truly Miraculous'

At BioXcellerator, we treat many patients for conditions like these with exceptional results often within days and even more ongoing improvement in the months and years following treatment.

For example, we treated Superbowl champion Mark May, who told us that he noticed improvement in just one day. I feel better. My neck feels a lot better and thats only after 24 hours, May said. Im shockingly surprised about how well its gone so far.

He also said that the first night after his treatment was the first he'd slept in once place in many years.

And army veteran and WWF Hall of Famer Kevin Nash let us know that his stem-cell therapy was a life-changing experience. He said that hes suffered from chronic pain for many years, but the very day after treatment said that when he was walking, I probably passed 300 people. Its the fastest Ive probably walked since I was 30 and that was 30 years ago.

Not only that, butafter two months of stem-cell therapy, he also reported the alleviation of his 24/7 pain.

These are only a few examples of the exceptional results stem cells can offer patients with disorders and injuries in the back and the neck. But its important to realize that the stem cells that various clinics offer can vary widely in quantity and potency. Stem cells derived from the placenta or umbilical cord are considered the gold standard and are rarely available in clinics located in the United States.

Our research team has developed a proprietary protocol for harvesting and reproducing only the most potent stem cells possible. Starting with a specific type of stem cell mesenchymal stem cells (MSCs) from donated umbilical cordswe then test these cells for specific proteins and genes that indicate the highest potential to reduce inflammation and stimulate healing. Then, those cells are reproduced into formulations of millions of high-potency stem cells called Golden Cells for infusion into patients during treatment.

Related:High-Potency 'Golden Cells' Offer Hope to Those With Severe Brain Injuries

In addition to promoting healing of damaged discs, stem-cell therapy can also be an effective treatment for other spinal injuries and diseases, brain injuriesand many other conditions. And one common treatment benefit is that because stem cells help the body better modulate the immune system and have powerful anti-inflammatory properties, stem-cell therapy helps improve immunity, performance and longevity.

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High-Potency 'Golden Cells' Offer Hope to Those With Severe Chronic Back and Neck Pain - Entrepreneur

Bone Marrow Stem Cells Comments Off on High-Potency ‘Golden Cells’ Offer Hope to Those With Severe Chronic Back and Neck Pain – Entrepreneur | August 6th, 2021

ELK CITY, Idaho, Aug. 5, 2021 /PRNewswire/ -- Therapeutic Solutions International, Inc., (OTC Markets: TSOI), announced today clearance from the Food and Drug Administration (FDA) to initiate a Phase III pivotal trial for registration of the Company's JadiCell universal donor stem cell as a treatment for COVID-19 associated lung failure.

In previous studies the Company has demonstrated the superior activity of JadiCell to other types of stem cells including bone marrow, adipose, cord blood, and placenta. Furthermore, the JadiCell was shown to be 100% effective in saving the lives of COVID-19 patients under the age of 85 in a double-blind placebo controlled clinical trial with patients in the ICU on a ventilator. In patients over the age of 85 the survival rate was 91%1.

"We are thankful for the strong regulatory, basic research and translational team that has worked in successfully obtaining this FDA clearance," said Dr. Thomas E. Ichim, Director of the Company. "FDA clearance to initiate a Phase III clinical trial means we are at the last phase of development before commercially selling the product. This positions us in a highly exclusive place in that to our knowledge no other cells have this potent ability to concurrently suppress inflammation while restoring function of tissue damaged by SAR-CoV-2."

"Having personally seen the effects of JadiCells on patients, I have seen their clinical potential firsthand" said Dr. James Veltmeyer, Chief Medical Officer of the Company. "I am very excited to enter the final step of clinical development before being able to provide these cells to the general population."

"Despite the initial promise of vaccine approaches, there exists a significant portion of the population refusing them and there are also patients in whom vaccines have failed to induce appropriate immunity.Once COVID-19 initiates its pathological cascade leading to lung failure, no therapies exist until now to address this population" said Famela Ramos, Vice President of Business Development. "We are looking forward to leveraging the current clearance not only for obtaining market registration but also for expanding into other COVID-19 related pathologies."

"Today marks a significant milestone in the growth of our Company as we have received the final regulatory clearance before final marketing approval," stated Timothy Dixon, President and CEO of Therapeutic Solutions International. "Successful completion of the agreed upon trial with the FDA will position the Company as a significant force in the global battle against this unseen enemy that to date has caused over 4.25 million deaths. We are extremely proud of our progress and vow to accelerate our work for humanity and for our shareholders."

About Therapeutic Solutions International, Inc.Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com,and our public forum is https://board.therapeuticsolutionsint.com/

1Umbilical cord mesenchymal stem cells for COVID19 acute respiratory distress syndrome: A doubleblind, phase 1/2a, randomized controlled trial - Lanzoni - 2021 - STEM CELLS Translational Medicine - Wiley Online Library

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Therapeutic Solutions International Receives FDA Clearance to Initiate Phase III Pivotal Registration Trial for JadiCell Universal Donor COVID-19...

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Myelodysplastic syndromes, known commonly as MDS, are a group of bone marrow diseases characterized by bone marrow failure, or an inadequate production of blood counts called cytopenia.

In a presentation at the CURE Educated Patient Leukemia Summit, Dr. Rami Komrokji, section head for Leukemia and MDS and Vice Chair of the Department of Malignant Hematology at Moffit Cancer Center, gave a run down on the diagnosis and staging process for MDS.

Diagnosing MDS

Komrokji explained that the myelo- prefix means bone marrow, and -dysplasia means abnormal-looking cells. When a patient has cytopenia, they may experience certain symptoms.

If patient is anemic, they will have shortness of breath, fatigue, palpitations, said Komrokji in an interview with CURE. If they have low platelets, they will have bleeding tendency, bruising. If they have low white blood cell counts, they will have maybe infections. So usually, either some of those symptoms will prompt blood testing, or on routine physical exam, the patients are found to have low blood counts. So that's usually the initial step.

Doctors will usually look into nutritional deficiencies such as B12, folate and ferritin, said Komrokji. Eventually, the patients will get a bone marrow aspirate and biopsy to diagnose their disease, which includes several parts.

There is the morphologic part, which means the pathologists are looking at the cells under a microscope, explained Komrokji. And then there is also some genetic testing. We look at cytogenetics nowadays, we look at gene mutations. So we put all of this information together to make the diagnosis.

The hematopathologist must see dysplasia, increased myeloblasts (immature cells known as blasts within the bone marrow) or certain cytogenetic abnormalities to make their diagnosis, Komrokji said.

Sometimes the diagnosis is straightforward, but sometimes it could be challenging, he added. It truly depends on an experienced hematopathologist to make the diagnosis.

Staging and Risk Stratification

Once a patient receives an MDS diagnosis, their doctor will go over risk stratification, or understanding what the risk of their disease is, which is what they consider staging, Komrokji said.

Now in MDS, its not like a lung cancer or colon cancer, he said. The disease does not spread around. The staging is based on the blood counts, on the percentage of those myeloblasts or immature cells (and) the chromosomal makeup of the cells. And nowadays, we sometimes also incorporate the presence of gene mis-happenings as well. So we get a lump score to estimate the risk.

Doctors typically use the International Prognostic Scoring System (IPSS) to categorize patients into one of five categories very low, low, intermediate, high and very high. The risk is the impact on survival and whether the disease will transform to leukemia, Komrokji explained. The disease risk must be known in order to tailor the patients treatment to them.

I always advise patients to see a specialized center in MDS, because obviously, those are not that common diseases, he said. A community oncologist could see a few (cases) per year, while an experienced center like in our place, we see like 15 to 20 per week.

Gene Mutations

Komrokji said that understanding gene mutations is an evolving field that is slowly becoming routine.

I advise all patients to inquire if theyve gotten genetic testing or not, he said. This sort of testing will help them understand any abnormalities. Doctors can look at a patients individual gene levels and detect for mutations, of which at least one was identified in 90% of patients with MDS.

Understanding the patients mutation(s) helps them tell whether there is a clonal hematopoiesis or mis-happening that occurred. It can also impact prognosis and allow them to further understand the disease risk.

And finally, some of them are targetable or important to follow through the treatment, said Komrokji. So patients should probably definitely have a genetic testing done. And sometimes after a treatment failure, we repeat it because we see other mutations that we could target with new drugs.

What Causes MDS?

In most cases, the cause of a patients MDS diagnosis is unknown.

We think it's phenomena of senescence or aging of those stem cells in the bone marrow that produces the blood, said Komrokji. Obviously, the process is very complicated. We have billions and billions of cells divide billions of times a day. So you know, as those cells age, mistakes can happen in them.

In most cases, he said, the mis-happenings which lead to the disease are random and at no fault of the patient. It is extremely rare for MDS to be inherited through familial genes.

There are, however, several known risk factors of MDS. If someone has history of another form of cancer and has received chemotherapy or radiation therapy, they may have possible stem cell damage and can develop MDS this is called therapy-related MDS. There has also been association of the disease with benzene exposure, chemical exposure and radiation exposure. Patients who have connective tissue diseases such as rheumatoid arthritis and lupus are at a slightly higher risk of getting MDS due to inflammation in the body and certain medications used to treat those diseases.

I would say there's a lot of better understanding of the disease in the past several years, of genetic mutation testing and incorporating them in practice, Komrokji said. And I think, you know, there are a lot of new treatments on the horizon for patients; there are several clinical trials in advanced phase.

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Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. The two most common fungal infections that affect people with AML are aspergillosis and candidiasis.

Invasive fungal infection is a leading cause of illness and mortality in people with acute leukemia. According to a 2019 study, about 12 to 24 percent of people with AML develop invasive fungal infections. But there are medications to prevent and treat these fungal infections.

Read on to find out how AML lowers resistance to fungal infection, as well as prevention and treatment strategies.

AML is a type of blood cancer. It starts in the bone marrow but usually moves into the blood fairly quickly. It tends to develop from white blood cells that dont function as they should.

White blood cells are a vital part of the immune system. When a foreign invader like bacteria, virus, or fungus enters your body, white blood cells spring into action. Their job is to attack the invader and prevent illness.

When you have AML, leukemia cells crowd out healthy white blood cells. The production of new white blood cells becomes impaired.

In addition, treatment for AML involves intense chemotherapy, which can also lower your white blood cell count. As a result, the immune system is suppressed and youre more vulnerable to infection and illness.

Other treatments that can weaken the immune system include:

Other health problems and nutritional deficiencies can also contribute to the suppression of the immune system.

During cancer treatment, your doctor will monitor your white blood cell count, particularly a type of white blood cell called neutrophils. Theyre an important line of defense against infection. If your neutrophil count is low, you have a condition called neutropenia, which increases the risk of infection.

Aspergillus molds and Candida yeasts are the most common fungi to affect people with AML.

Aspergillosis is an infection triggered by Aspergillus. Its a common mold that can be found indoors or outside. Most of us breathe it in every day without cause for concern. But if you have a weakened immune system, you have an increased likelihood of developing the illness.

There are different types of aspergillosis, each causing a different set of symptoms:

While its possible to develop any of these types if you have a weakened immune system, about 10 percent of people with AML develop invasive aspergillosis. This infection most commonly affects the lung.

Candidiasis is an infection caused by Candida. We all have this yeast on our bodies. It only causes problems when it grows out of control or enters the bloodstream or internal organs.

Different types of candidiasis cause different symptoms:

Invasive candidiasis is a serious infection that can affect many parts of the body. In addition to causing fever and chills, invasive candidiasis can affect the:

Some less common types of fungi that can also affect people with AML are:

Fungi are everywhere, so its difficult to completely avoid them. Here are a few things you can do to help lower your risk of infection:

Prevention and treatment for fungal infections in people with AML require an individualized approach. Even if you show no sign of infection, your doctor may prescribe a prophylactic medication designed to prevent a fungal infection. They include:

If you do develop a fungal infection, some drugs above can help treat it. Additional medications used to treat fungal infection are:

Fungal infections can recur. Thats why you may need both antifungal therapy and prophylactic therapy until your blood counts improve.

Medications to prevent or treat fungal infections each have potential benefits and risks. The treatment that is best for you depends on a number of factors, such as:

Symptoms of fungal infections are similar to those of other health conditions. Its a good idea to get in touch with your doctor whenever you have new or worsening symptoms. While some fungal infections are minor, others can be life threatening.

Untreated, fungal infections can spread to other parts of the body. Getting a quick diagnosis means you can start treatment that may prevent illness. Some signs of fungal infection include:

Fungal infections are not uncommon in people with AML. Both AML and chemotherapy can significantly weaken the immune system, increasing risk for infection. Fungal infections can affect a single organ, such as the lungs or sinuses, or they can affect the bloodstream and multiple organs.

Aspergillosis and candidiasis are the most common fungal infections affecting people with AML.

Fortunately, there are medications to help prevent and treat fungal infections. If you have AML, speak with your doctor about your risk factors and how you can prevent fungal infection.

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Acute Myeloid Leukemia Fungal Infections: Types and Treatment - Healthline

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Regulatory feedback obtained on OTL-200 (MLD) and OTL-203 (MPS-IH) programs

New HAE collaboration with Pharming Group highlights broad potential for HSC gene therapy

Multiple presentations from neurometabolic programs at MPS Symposia including additional follow-up in MPS-IH

Cash and Investments of Approximately $270M Provide Runway into First Half 2023

BOSTONandLONDON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended June 30, 2021, as well as recent business updates and upcoming milestones.

This past quarter Orchard has shown great progress against multiple core strategic objectives across the portfolio, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Obtaining regulatory clarity from the FDA on our investigational OTL-200 program in early-onset MLD represents a tremendous step toward making a treatment option available for young patients in the U.S. A second neurodegenerative program in MPS-IH is also advancing toward a pivotal trial, incorporating recent feedback from both the U.S. and EU regulatory agencies. In our earlier stage pipeline, were very excited for our new collaboration with Pharming exploring the potential of HSC gene therapy in hereditary angioedema.

Summary of Recent Publication and Business Updates

Data presentations at MPS 2021

Presentations from investigational hematopoietic stem cell (HSC) gene therapy programs in mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and mucopolysaccharidosis type IIIB (MPS-IIIB) were featured at the 16th International Symposium on MPS and Related Diseases on July 23-25, 2021.

OTL-203 for MPS-IH: Updated data for OTL-203 showing positive clinical results in multiple disease manifestations of MPS-IH were highlighted in an oral presentation. With follow-up in five of eight patients now out to two years, all patients treated with OTL-203 continue to show stable cognitive and motor function and growth within the normal range throughout the follow-up period. Treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.OTL-202 for MPS-IIIB: Long-term results following HSC gene therapy in a mouse model of MPS-IIIB were also presented. Significant -N-acetylglucosaminidase (NAGLU) enzyme expression was seen in the bone marrow, blood plasma and other somatic tissues following gene therapy. Importantly, at six months post-treatment, sufficient expression of NAGLU enzyme was observed in the brain of mice treated with gene therapy, which led to a normalization of heparin sulfate levels and neurological corrections, which was not observed in mice treated with hematopoietic stem-cell transplantation (HSCT).

Collaboration with Pharming Group for hereditary angioedema (HAE)

On July 1, 2021 Orchard Therapeutics and Pharming Group N.V. announced a strategic collaboration to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivo autologous HSC gene therapy for the treatment of HAE. OTL-105 is designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks. In preclinical studies, to date, OTL-105 demonstrated high levels of SERPING1 gene expression via lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs. A link to the full announcement can be found here.

Under the terms of the collaboration, Pharming has been granted worldwide rights to OTL-105 and will be responsible for clinical development, regulatory filings, and commercialization of the investigational gene therapy, including associated costs. Orchard will lead the completion of IND-enabling activities and oversee manufacturing of OTL-105 during pre-clinical and clinical development, which will be funded by Pharming. Orchard received an upfront payment of $17.5 million in the form of cash and an equity investment and is also eligible to receive up to $189.5 million in development, regulatory and sales milestones as well as mid-single to low double-digit royalty payments on future worldwide sales.

Clinical and Regulatory Updates

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), MPS-IH and Wiskott-Aldrich syndrome (WAS). A link to the full announcement can be found here.

OTL-200 for MLD: Orchard held a productive meeting with the U.S. Food and Drug Administration (FDA) and has received written feedback concerning the clinical package expected to support a Biologics License Application (BLA) for OTL-200 in MLD. Based on the feedback from this meeting and previous interactions, the company is preparing for a BLA filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing patients. This approach and timeline are subject to the successful completion of the remaining regulatory activities in advance of an expected pre-BLA meeting with FDA, including CMC interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard received feedback on the design of a global registrational trial for OTL-203 following a parallel scientific advice meeting with FDA and the European Medicines Agency (EMA). The interaction offered guidance on the proposed clinical trial protocol from each of the regulatory agencies, including elements of the trial design, comparator arm and recommended endpoints. Orchard will be incorporating this feedback into a revised global clinical study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: The proof-of-concept trial for OTL-201 has met its recruitment goal with the enrollment of a fifth patient. Interim data from this study is expected to be presented at medical meetings in the second half of 2021 and 2022.OTL-103 for WAS: Orchard updated its guidance regarding the Marketing Authorization Application (MAA) and BLA submissions for the OTL-103 program in WAS to take into account work remaining on potency assay development and validation. The company now expects a MAA submission in 2022, subject to further dialogue with EMA, and will provide updated guidance for a BLA submission following additional FDA regulatory interactions.

Research Programs

Orchard plans to announce new preclinical data from research programs in frontotemporal dementia with progranulin mutations (GRN-FTD) and Crohns disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2-CD) in the second half of 2021.

Second Quarter 2021 Financial Results

Research and development expenses were $21.8 million for the second quarter of 2021, compared to $31.6 million in the same period in 2020. The decline is primarily due to non-cash impairment charges of $5.7 million taken in the second quarter of 2020 and other savings associated with our corporate restructuring. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative expenses were $14.3 million for the second quarter of 2021, compared to $15.7 million in the same period in 2020. The decrease was primarily due to savings associated with personnel and related changes.

Net loss was $36.6 million for the second quarter of 2021, compared to $47.5 million in the same period in 2020. The decline in net loss as compared to the prior year was primarily due to savings realized in our operating expenses as a result of the companys May 2020 updated strategy and corporate restructuring. The company had approximately 124 million ordinary shares outstanding as of June 30, 2021.

Cash, cash equivalents and investments as of June 30, 2021, were $269.3 million compared to $191.9 million as of December 31, 2020 and excludes the $17.5 million in upfront payments from the collaboration with Pharming Group N.V. entered into on July 1, 2021. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement, offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of June 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes the additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

Orchard Therapeuticsis a global gene therapy leader dedicated to transforming the lives of people affected by severe diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy inMilan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedJune 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Condensed Consolidated Statements of Operations Data (In thousands, except share and per share data) (Unaudited)

Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited)

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Second Quarter 2021 Financial Results and Highlights Recent ... - The Bakersfield Californian

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DetailsCategory: DNA RNA and CellsPublished on Tuesday, 03 August 2021 10:03Hits: 951

Off-the-Shelf CAR T-cell Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

Phase 1 Clinical Study will Evaluate Three Dosing Regimens of FT819 for Patients with Advanced B-cell Leukemias and Lymphomas

SAN DIEGO, CA, USA I August 02, 2021 I Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, announced today that the first patient has been treated with FT819, an off-the-shelf chimeric antigen receptor (CAR) T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master induced pluripotent stem cell (iPSC) line, a renewable cell source that enables mass production of high quality, allogeneic CAR T cells with greater product consistency, off-the-shelf availability, and broader patient accessibility. FT819 is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy.

Remarkable clinical outcomes have been achieved through treatment with patient-derived CAR T-cell therapy, however, next-generation approaches are necessary to reach more patients who are in need of these highly-effective therapies, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Treatment of the first-ever patient with FT819 ushers in a new era for off-the-shelf CAR T-cell therapy, with the potential to overcome the real-world limitations of existing patient- and donor-derived therapeutic approaches and unlock the full potential of CAR T-cell therapy. We would like to thank our collaborators at Memorial Sloan Kettering Cancer Center, whose partnership over the past five years has profoundly contributed to this landmark achievement.

FT819 was designed to specifically address several limitations associated with the current generation of patient- and donor-derived CAR T-cell therapies. Under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering and Head, Gene Expression and Gene Transfer Laboratory, the Company incorporated several first-of-kind features into FT819 including:

The multi-center Phase 1 clinical trial of FT819 is designed to determine the recommended Phase 2 dose and schedule of FT819 and assess its safety and clinical activity in adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), and B-cell lymphomas (BCL). Three treatment regimens will be independently evaluated for each type of malignancy in dose escalation: Regimen A as a single dose of FT819; Regimen B as a single dose of FT819 with IL-2 cytokine support; and Regimen C as three fractionated doses of FT819. For each indication and regimen, dose-expansion cohorts may be enrolled to further evaluate the clinical activity of FT819. The first patient with relapsed / refractory ALL was enrolled in Regimen A and received a dose of 90 million cells.

At the 24th American Society of Gene & Cell Therapy Annual Meeting held in May 2021, the Company presented preclinical data demonstrating that FT819 exhibits uniform 1XX CAR expression with complete elimination of endogenous TCR expression. The product candidate was shown to contain a stem- and central-memory T-cell phenotype, and had high-level expression of the activation marker CD25 and the trafficking marker CXCR4 and very low-level expression of the checkpoint proteins PD1, TIM3, CTLA4 and LAG3. Additionally, data from functional assessments showed that FT819 had potent antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines comparable to that of healthy donor-derived CAR T cells, and persisted and maintained tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia.

Pursuant to a license agreement with MSK, Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452, which covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

Fate Therapeutics haslicensedintellectual propertyfrom MSK on which Dr. Sadelain is aninventor.As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT819FT819 is an investigational, universal, off-the-shelf, T-cell receptor (TCR)-less CD19 chimeric antigen receptor (CAR) T-cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line, which is engineered with the following features designed to improve the safety and efficacy of CAR19 T-cell therapy: a novel 1XX CAR signaling domain, which has been shown to extend T-cell effector function without eliciting exhaustion; integration of the CAR19 transgene directly into the T-cell receptor alpha constant (TRAC) locus, which has been shown to promote uniform CAR19 expression and enhanced T-cell potency; and complete bi-allelic disruption of TCR expression for the prevention of graft-versus-host disease (GvHD). FT819 demonstrated antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines comparable to that of primary CAR T cells, and persisted and maintained tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia (Valamehr et al. 2020). FT819 is being investigated in a multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell malignancies, including B-cell lymphoma, chronic lymphocytic leukemia, and acute lymphoblastic leukemia (NCT04629729).

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

SOURCE: Fate Therapeutics

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Fate Therapeutics Announces Treatment of First Patient in Landmark Phase 1 Clinical Trial of FT819, the First-ever iPSC-derived CAR T-Cell Therapy |...

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LIFESAVING After being on the Be the Match registry for eight months, Lacey Jones donated stem cells to a 75-year-old woman battling myeloid leukemia.

A Kokomo teacher has spent part of her summer possibly saving the life of a 75-year-old woman.

Late last month, Lacey Jones, a veterinary careers instructor at the Kokomo Area Career Center, traveled to the Hoxworth Blood Center at the University of Cincinnati to donate stem cells to a woman she was genetically matched with who was battling myeloid leukemia. Now, all Jones hopes is that her stem cells were enough.

I really want nothing more than to hear that she is in remission. That was the emotional part of the process. Once it was all over, I just went to my hotel room, and I could not help but to just kind of cry and cry and cry and pray and hope because you want nothing more than for your cells to work for the patient, Jones said.

But the opportunity to help someone was a chance that some dont ever get. Jones registered as a donor through Be the Match, which operates the national bone marrow registry, late last year after the story of her friends daughter, who was diagnosed with infant leukemia, inspired her to want to help someone.

According to Be the Match, only one of 430 people who register as a donor is selected as a match, and some of those who are selected wait years before theyre matched. For Jones, she became a genetic match for someone in just eight months.

At the end of May, she received a call from Be the Match, letting her know that there was a woman who had been diagnosed with myeloid leukemia, and Jones was a secondary match for her. Jones was told that there was someone who was the primary match who matched just a little better, but in the chance that that donor fell through, Jones would be at the plate.

I just kind of waited around, didnt really think anything of it, and then they called me at the beginning of June. She explained that the original person who matched ended up not being who they really needed, and of course with HIPPA and all that they cant really tell you any of that information as to why, Jones said.

As far as the patient, all Jones was told was that it was a 75-year-old woman who was battling myeloid leukemia somewhere in the world. Jones was asked if shed be available to donate during certain dates, but at that point, there were no details as to where she would be donating or exactly when. What Jones did know, though, was that she would do it.

The process then became a whirlwind of scheduling, logistics, and injections. She went to Indianapolis for lab work to ensure she was healthy enough to donate. Then, for a week leading up to her donation, she was required to take injections of a drug called filgrastim to increase the number of white blood cells in her bloodstream so they could be collected more easily.

Theyre actual injections that are given to people who have cancer to increase the white blood cell count, but theyre given to donors because it increases your bone marrow production. Then what happens is your body naturally expels the extra bone marrow into the bloodstream, she said.

Jones was a bit hesitant because shed never given herself injections before, and she was warned of the side effects that could occur. The most common side effect of filgrastim, she said, was soreness due to the overproduction of bone marrow.

They explained some of the side effects of the filgrastim, and that makes you feel a little intimidated. But because of my passion and my empathy for the patient and hearing about there not being donors and just knowing that I could potentially help save her life, it overruled any type of fear I had, she said.

At the end of June, Jones traveled to Hoxworth Blood Center at the University of Cincinnati to begin the donation.

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There are two donation methods, either marrow or peripheral blood stem cells. The marrow donation is a surgical procedure done under anesthesia. Doctors use needles to retrieve liquid marrow from both sides of the back of a patients pelvic bone. The hospital stay is usually from early morning to late afternoon, according to Be the Match, while some donors are kept overnight for observation.

The peripheral stem cell donation, on the other hand, is a non-surgical procedure that takes place at a blood center or outpatient hospital. Blood is removed through a needle in one arm and passed through a machine that collects only the blood-forming cells and returns the blood through a needle in the donors other arm.

As soon as Jones got to the blood center, nurses took a blood sample to see where her white blood cell count was. A normal count, she said, was between four and 10. Hers was at 42. Because the filgrastim injections were so effective for Jones, she was able to do the peripheral stem cell method.

So began a five-hour process of Jones sitting very still while a machine filtered out blood-forming cells from her left arm and put the blood back in her right arm. The nurses took her blood pressure every 15 minutes to ensure she wasnt having any kind of reaction, and the staff knew down to the minute when her donation would be complete, she said, and had the transfer staff ready.

At the end of the donation, Jones said it was like a movie when the person came in to take her stem cells that would be delivered to the patient.

They actually had somebody come up with the cooler because they flew my sample to the patient. Its almost like a movie. You watch someone come through the big steel door with the cooler and watch them package your sample, she said.

And while she didnt know where the sample was going, she was told the recipient was nowhere close to where they were.

When the process was over, Jones was thankful. She said the nurses told her it was a textbook donation, and the only side effect Jones experienced from the injections was mild soreness.

Three months after the donation, the recipient will have the option to find out who her donor was and to contact Jones if she chooses.

Jones said shed love to one day hear from the recipient.

I want nothing more than to one day get that phone call that says, OK, the person you donated to wants to get in contact with you. I dont know what Ill do. Ill probably cry again, she said.

Jones will remain on the Be the Match registry, and in the event she ever receives a call that shes a match again, she will be ready to go, she said.

To join the bone marrow donor registry, visit join.bethematch.org. The process requires a cheek swab, which can be done with a kit thats mailed to potential donors. Afterward, the person will be added to the registry and have the chance to get matched.

Be the Match encourages those who are contacted to donate to go forward with the donation as theyre the patients best genetic match from the entire registry.

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Donating a chance at life: Kokomo teacher donates stem cells to 75-year-old woman through Be the Match - Kokomo Perspective

Bone Marrow Stem Cells Comments Off on Donating a chance at life: Kokomo teacher donates stem cells to 75-year-old woman through Be the Match – Kokomo Perspective | July 22nd, 2021

58% relapse free survival at 1 year post-transplant79% overall survival at 1 year post-transplant

BOSTON, July 21, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, today announced positive results from its Phase 2 trial evaluating eprenetapopt with azacitidine for post-transplant maintenance therapy in patients with TP53 mutant MDS and AML.

In 33 patients enrolled in the trial, the relapse free survival (RFS) at 1 year post-transplant was 58% and the median RFS was 12.1 months. The overall survival (OS) at 1 year post-transplant was 79%, with a median OS of 19.3 months. Prior clinical trials evaluating post-transplant outcomes in TP53 mutant MDS and AML patients have reported a 1-year post-transplant RFS of ~30% and a median OS of ~5-8 months. In addition, the post- transplant regimen of eprenetapopt and azacitidine was well tolerated among patients in the clinical trial. The Company plans to discuss the data from this Phase 2 clinical trial with the U.S. Food and Drug Agency (FDA) in the second half of 2021 and expects to present data at a future scientific or medical conference.

The post-transplant RFS and OS data with eprenetapopt and azacitidine maintenance therapy in these very difficult-to-treat TP53 mutant MDS and AML patients are incredibly exciting, said trial principal investigator Asmita Mishra, M.D., of the H. Lee Moffitt Cancer Center and Research Institute. Although transplant is currently the only potentially curative treatment for patients with TP53 mutant MDS and AML, the risk of relapse with current standard of care remains unacceptably high and the median OS post-transplant is very limited at 8 months or less. Post-transplant maintenance therapy with eprenetapopt and azacitidine could, if approved, represent a new treatment paradigm that meaningfully improves outcomes for these patients with limited treatment options.

About Aprea Therapeutics, Inc.

Aprea Therapeutics, Inc. is a biopharmaceutical company headquartered in Boston, Massachusetts with research facilities in Stockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53. The Companys lead product candidate is eprenetapopt (APR-246), a small molecule in clinical development for hematologic malignancies and solid tumors. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for myelodysplastic syndromes (MDS), Orphan Drug and Fast Track designations from the FDA for acute myeloid leukemia (AML), and Orphan Drug designation from the European Commission for MDS and AML. APR-548, a next generation small molecule reactivator of mutant p53, is being developed for oral administration. For more information, please visit the company website at http://www.aprea.com.

The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.

About p53, eprenetapopt and APR-548

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

Eprenetapopt (APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with eprenetapopt in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with eprenetapopt has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

A pivotal Phase 3 clinical trial of eprenetapopt and azacitidine for frontline treatment of TP53 mutant MDS has been completed and failed to meet the primary statistical endpoint of complete remission. A Phase 1/2 clinical trial of eprenetapopt with venetoclax and azacitidine for the frontline treatment of TP53 mutant AML met the primary efficacy endpoint of complete remission. Additional clinical trials in hematologic malignancies and solid tumors are ongoing. Eprenetapopt has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Orphan Drug and Fast Track designations from the FDA for AML, and Orphan Drug designation from the European Medicines Agency for MDS and AML.

APR-548 is a next-generation small molecule p53 reactivator. APR-548 has demonstrated high oral bioavailability, enhanced potency relative to eprenetapopt in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice.

About MDS

Myelodysplastic syndromes (MDS) represent a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis. There are no currently approved therapies specifically for TP53 mutant MDS or AML patients.

About AML

AML is the most common form of adult leukemia, with the highest incidence in patients aged 60 years and older. AML is characterized by proliferation of abnormal immature white blood cells that impairs production of normal blood cells. AML can develop de novo or may arise secondary to progression of other hematologic disorders or from chemotherapy or radiation treatment for a different, prior malignancy; secondary AML carries a worse prognosis than de novo AML. Mutations in TP53, which are associated with poor overall prognosis, occur in approximately 20% of patients with newly diagnosed AML, more than 30% of patients with therapy-related AML and approximately 70-80% of patients with complex karyotype.

Forward-Looking Statement

Certain information contained in this press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our study analyses, clinical trials, regulatory submissions, and projected cash position. We may, in some cases use terms such as future, predicts, believes, potential, continue, anticipates, estimates, expects, plans, intends, targeting, confidence, may, could, might, likely, will, should or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward-looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with the U.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

Source: Aprea Therapeutics, Inc.

Corporate Contacts:

Scott M. Coiante Sr. Vice President and Chief Financial Officer 617-463-9385

Gregory A. Korbel Sr. Vice President and Chief Business Officer 617-463-9385

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Aprea Therapeutics Announces Positive Results from Phase 2 Trial of Eprenetapopt + Azacitidine ... - The Bakersfield Californian

Bone Marrow Stem Cells Comments Off on Aprea Therapeutics Announces Positive Results from Phase 2 Trial of Eprenetapopt + Azacitidine … – The Bakersfield Californian | July 22nd, 2021

Dr. Bill Cimikoski, Medical Director of Utah Stem Cellsjoined Surae on The Daily Dish to discuss the BodyTite and Facetite procedures. He tells Surae that these procedures are excellent for getting rid of unwanted fat, while at the same time shrink wrapping the skin so that any loose skin is simultaneously tightened at the same time.

For some individuals, there may only be minimal (or none at all) fat to extract and it might be only necessary to tighten the skin. Depending on the area Utah Stem Cells are treating, they often see that in some individuals, there isnt really any fat to speak of and their patients are just looking for skin tightening and this is an excellent way to achieve that goal!

Unfortunately, on the other hand, some patients do have a large amount of fat in certain areas and then this device is also accompanied by liposuction. This is where they can suck the fat in addition to tightening the loose skin at the same time.This procedure is called Radio Frequency assisted Liposuction. At Utah Stem Cells they also offer High Definition Radio Frequency assisted liposuction to sculpt abs.

They offer many different treatments for different areas of the body, including the following:

All procedures are in-office and with only small holes or needle punctures, which heal completely without scarring. There is no need for general anesthesia and all are completed with lidocaine fluid although they do offer nitrous oxide, ketamine, and other methods to keep people comfortable and less anxious.

As a special gift, anyone who calls in after viewing The Daily Dish today will be entitled to $200 off any procedure.

To find out more about how Dr. Bill Cimikoski and Utah Stem Cells can help you, visit their website or you can give them a call at Phone number: (801) 999-4860

This article contains sponsored content.

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Learn how to remove unwanted fat with procedures beyond liposuction - ABC 4

Skin Stem Cells Comments Off on Learn how to remove unwanted fat with procedures beyond liposuction – ABC 4 | July 22nd, 2021

SAN DIEGO--(BUSINESS WIRE)--Stemson Therapeutics announced today the closing of a DCVC Bio-led $15 million Series A financing to advance development of Stemsons proprietary therapeutic solution to cure hair loss. Genoa Ventures, AbbVie Ventures and other investors join in supporting Stemsons efforts to restore human hair growth with a novel cell regeneration technology using the patients own cells to generate new hair follicles.

In addition, Kiersten Stead, Ph.D., Co-Managing Partner at DCVC Bio and Jenny Rooke, Ph.D., Managing Director at Genoa Ventures will join Stemsons Executive Chairman Matt Posard and Chief Executive Officer and co-founder Geoff Hamilton on the board of directors. Dr. Stead invests in early-stage companies that build novel deep tech businesses in the life sciences. Stead received a Ph.D. in Molecular Biology & Genetics and an MBA in finance from the University of Alberta. Dr. Rooke is founder and Managing Director at Genoa Ventures where she specializes in early-stage companies innovating at the convergence of technology and biology. Rooke received a Ph.D. in Genetics from Yale University and a degree in physics from the Georgia Institute of Technology.

We are excited and honored to welcome DCVC Bio and a fantastic syndicate of investors to the Stemson team. The Series A funding will help us optimize our solution for human skin structure and environment so we can go into our first human clinical trial with high confidence for a positive outcome. We have the technical and biological building blocks to successfully address hair loss that overcomes failures of past therapies, said Hamilton. The addition of key venture capital investors DCVC Bio, Genoa Ventures and AbbVie Ventures broadens and strengthens our investor base. DCVC Bio and Genoa Ventures are successful early-stage development investors, and I am pleased to welcome Dr. Stead and Dr. Rooke, our newest board members, to the team. In addition, the AbbVie Venture investment comes on the heels of an initial seed investment from Allergan Aesthetics in 2020, and the continued industry interest in our technology is encouraging.

Globally, hundreds of millions of men and women suffer from various forms of hair loss. Though there are many possible causes of hair loss, including chemotherapy, autoimmune disease, scarring, and genetics, all can result in a loss of self-esteem and cause depression, anxiety and other mental health disruption for those affected. The hair restoration market is expected to exceed $13.6 billion by 2028, and no solution today is capable of generating an unlimited new supply of healthy follicles for patients in need.

Almost 30 years have passed since the last FDA-approved hair loss treatment, yet millions still suffer the physical and mental impact of losing their hair each year, stated Dr. Stead. Stemsons novel stem cell engineering platform has the potential to cure hair loss once and for all, treating not only the physical symptoms of this complex problem, but the mental burden as well.

"The team at Genoa is impressed with Stemsons vision to blend biology and technology and apply it beyond traditional biotech," added Dr. Rooke. "By combining exciting advancements in iPSCs with novel technologies in materials and data sciences, Stemson exemplifies the kind of chimeric teams Genoa seeks to support on their journey to become a category-defining company."

The Series A financing brings the total funding raised to date to $22.5 million and allows Stemson to further the next stage of research and development of its cell engineering platform, where is it being combined with bioengineered material and robotic delivery as a novel solution for natural hair replacement. Currently, Stemsons research and development efforts are focused on developing an optimized solution for human skin structure environment in larger animal models. Stemsons Induced Pluripotent Stem Cell (iPSC) based technology is capable of producing the cell types required to initiate hair follicle growth and have been successfully tested in small animal models.

About Cell Regeneration Technology

Human Induced Pluripotent Stem Cells (iPSC) have the unique capability to replicate indefinitely and give rise to all cell types of the human body, including the cell types required for repair. iPSC-based technology is capable of producing the cell types required to initiate hair follicle growth. As a new therapeutic platform, iPSCs represent an emerging area of regenerative cell therapy. Stemson is one of a growing number of companies at the forefront in developing iPSC-based treatments.

About DCVC Bio

For over twenty years, DCVC and its principals have backed brilliant entrepreneurs applying Deep Tech, from the earliest stage and beyond, to pragmatically and cost-effectively tackle previously unsolvable problems in nearly every industry. DCVC Bio specializes in supporting life sciences platform companies at the intersections of engineering and therapeutics, industrial biotechnology and agriculture. For more information, please visit https://www.dcvc.com/companies.html#dcvc-bio

About Genoa Ventures

Genoa Ventures invests in early-stage companies working at the convergence of biology & technology to accelerate the pace of innovation, transform industries, and solve some of the most fundamental challenges to life. Genoa, identifies opportunities early and focuses its investments and expertise to empower the next great category-defining companies. The Genoa team has a unique chimeric blend of experience from scientific research and discovery to executive management in the life sciences and technologies sectors. The team applies this diverse experience to provide expert guidance to its companies and stellar returns to its investors.

About AbbVie Ventures

AbbVie Ventures is the corporate venture capital group of AbbVie. We are a strategic investor, investing exclusively in novel, potentially transformational science aligned with AbbVie's core R&D interests. We measure success primarily by the extent to which our investments foster innovation with potential to transform the lives of patients that AbbVie serves. AbbVie Ventures enables its portfolio companies with both funding as well as access to AbbVie's internal network of experts across all phases of drug development, from drug discovery through commercialization. For more information, please visit http://www.abbvie.com/ventures

About Stemson Therapeutics

Stemson Therapeutics is a pre-clinical stage cell therapy company founded in 2018 with a mission to cure hair loss by leveraging the regenerative power of Induced Pluripotent Stem Cells. Based on the breakthrough innovation by Stemson Therapeutics co-founder, Dr. Alexey Terskikh, Stemson uses iPSC to regenerate the critical cells required to grow hair and which are damaged or depleted in patients suffering from hair loss. The iPSC-derived cells are used to grow de novo hair follicles, offering a new supply of hair to treat people suffering from various forms of Alopecia. Today, there are no available treatments capable of growing new hair follicles. Stemsons world class team of scientists, advisors and collaborators are passionate about delivering a scientifically based, clinically tested cure for hair loss to the millions of hair loss sufferers who seek help for their hair loss condition. Stemson Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.stemson.com.

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Stemson Therapeutics Secures $15M Series A Funding to Cure Hair Loss - Business Wire

Skin Stem Cells Comments Off on Stemson Therapeutics Secures $15M Series A Funding to Cure Hair Loss – Business Wire | July 22nd, 2021

Blood cancers are not like other cancers. Unlike a cancer that affects one region of the body, such as lung cancer afflicting the lungs, blood cancers affect the entirety of the body because blood flows throughout it. Blood cancers do not form a lump or tumor in a specific organ, potentially making blood cancers more difficult to detect.

Blood contains various components. Red blood cells, white blood cells, plasma, and platelets all combine to make blood. Blood cancers, which include lymphoma, leukemia and myeloma, affect different components of the blood and interfere in different ways with the normal function of blood in the body.

Lymphoma affects the lymphatic system, which includes white blood cells called lymphocytes. These cells help protect the body against infection, advises the health site OnHealth. Leukemia originates inside of the bone marrow. Production of an overabundance of white blood cells may impede the marrows ability to make sufficient red blood cells and plasma, states the Leukemia & Lymphoma Society. Myeloma affects plasma cells, a type of white blood cell that fights off infection. When they become cancerous, the affected white blood cells can manufacture an abnormal protein that may damage organs and bodily systems.

Individuals affected with lymphoma, leukemia or myeloma may not feel a tumor, but they can look out for other symptoms. These include:

Frequent infections;

Coughing or chest pain;

Fever or chills;

Ongoing weakness or fatigue;

Shortness of breath;

Night sweats; and/or

Itchy skin or rash.

Even though blood cancers differ from other cancers in the way they present and what parts of the body they affect, they often are initially treated in the same way. SurvivorNet, a cancer survivor resource, says chemotherapy and radiation can target these cancers. Stem cell transplant, also known as bone marrow transplant, is a much more common treatment for blood cancers. Stem cells may be extracted from the patient or received from a donor.

Those who suspect the presence of blood cancers should consult with a doctor who can order blood tests to form a diagnosis.

We are making critical coverage of the coronavirus available for free. Please consider subscribing so we can continue to bring you the latest news and information on this developing story.

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What sets blood cancers apart from other cancers? | Lifestyles | washtimesherald.com - Washington Times Herald

Skin Stem Cells Comments Off on What sets blood cancers apart from other cancers? | Lifestyles | washtimesherald.com – Washington Times Herald | July 22nd, 2021

Scientists say that base editing proved itself efficient in correcting a mutation in patient cells with the monogenic disease Alpha-1 antitrypsin deficiency (AATD). The disorder is a common inherited disease that affects the liver and the lungs.

Base editing is different from other forms of editing, including CRISPR, because the base editors do not induce a break in the DNA, which helps prevent double strand breaks, potential off-target editing, and unwanted mutations during cell repair.

Researchers at Boston Medical Center and Boston University used patient-derived liver cells (iHeps) that mimic the biology of liver hepatocytes, the main producers of alpha-1 antitrypsin protein in the body. The base editing technology corrected the Z mutation responsible for AATD and reduced the effects of the disease in the hepatocytes, demonstrating successful base editing in human cells.

The study (Adenine Base Editing Reduces Misfolded Protein Accumulation and Toxicity in Alpha-1 Antitrypsin Deficient Patient iPSC-Hepatocytes), published inMolecular Therapy,can help pave the way for future human trials, according to the research team.

AATD is most commonly caused by the Z mutation, a single base substitution that leads to AAT protein misfolding and associated liver and lung disease. In this study, we apply adenine base editors to correct the Z mutation in patient-induced pluripotent stem cells (iPSCs) and iPSC-derived hepatocytes (iHeps), wrote the investigators.

We demonstrate that correction of the Z mutation in patient iPSCs reduces aberrant AAT accumulation and increases its secretion. Adenine base editing (ABE) of differentiated iHeps decreases ER stress in edited cells as demonstrated by single-cell RNA sequencing. We find ABE to be highly efficient in iPSCs and do not identify off-target genomic mutations by whole genome sequencing.

These results reveal the feasibility and utility of base-editing to correct the Z mutation in AATD patient cells.

This study shows the successful application of base editing technology to correct the mutation responsible for AATD in liver cells derived from patients with this disease, said Andrew Wilson, MD, a pulmonologist at Boston Medical Center and an associate professor of medicine at the Boston University School of Medicine, who served as the studys corresponding author. I am hopeful that these results will create a pathway to use this technology to help patients with AATD and other monogenic diseases.

Base editors created by Beam Therapeutics were applied to induced pluripotent stem cells (iPS cells) from patients with AATD, and then again in hepatocytes that were derived from iPS cells. This was done to study the correction of the Z mutation of the gene responsible for AATD in human cells.

The Z mutation in the SERPINA1 gene is responsible for causing chronic, progressive lung and liver disease in AATD. In patients with AATD, the mutant AAT proteins misfold and form aggregates of protein that build up inside the hepatocytes and cause damage.

For this study, researchers started with mutant (ZZ) iPSCs created from a patient with AATD. After the base editing process was completed, the DNA from the edited cells was sequenced to determine if the SERPINA1 gene had been corrected. Clonal populations of cells with either one (MZ) or both copies (MM) of the corrected gene were expanded and then differentiated over the course of 25 days to generate hepatocytes.

After sequencing the entire genome of the edited cells, there was no evidence of inadvertent mutations in the genome from the base editors, and the misfolding and associated protein buildup was partially corrected in MZ cells and completely in MM normal cells.

The process was repeated using hepatocytes derived from the mutant iPSCs. Two base editors were used in different conditions to test the efficiency of this process. In the best conditions, about 50% of the mutant genes were successfully edited. The cells were then analyzed to see if they still appeared hepatic and if there were fewer signs of the disease in the edited cells, compared to mutant ZZ cells.

Findings showed the base editing did not alter the hepatic program, and the liver cells still expressed hepatic genes and proteins at normal levels. In addition, there was less accumulation of aggregated misfolded Z AAT protein, showing less evidence of disease in the edited cells.

While augmentation therapy has been shown to slow the progression of lung disease in AATD patients, there are currently no treatments available for AATD-associated liver disease. Emerging treatment strategies have focused on the correction of the Z mutation.

Base editing is being evaluated as a treatment modality for a variety of monogenic diseases, according to the scientists. Alpha-1 antitrypsin deficiency is a prime target for base editing, likely to be one of the earlier diseases in which base editors are tried in human studies. Additional disease targets include retinal disease, hereditary tyrosinemia, sickle cell anemia, progeria, cystic fibrosis, and others.

Findings of this study suggest that future research may explore the usefulness of base-editors in editing other quiescent cell populations. Additionally, it has recently been shown that base-editors can edit RNA in addition to DNA in immortalized cell lines and warrants further investigation.

By quiescent, we are referring to differentiated cells (in this case hepatocytes) that are not stem cells or cells that are actively dividing. Basically, [we are talking about] any differentiated cell type, Wilson toldGEN. This is relevant because many of the cell types in the body that you would want to target are already differentiated cells. It is in many cases easier to edit an actively dividing cell, which is why we mention this. There are many examples of a differentiated cell type in the body, such as cardiac cells, lung cells, skin cells, etc., that you might want to target.

One of the major things researchers worry about in the field of gene editing is the possibility of off-target effectsunintended consequences of applying the editing machinery.

The most likely off-target effect, in this case, would be editing of DNA somewhere in the genome other than what we intended to edit, continued Wilson. When we looked by whole genome sequencing, we didnt see evidence of this in iPS cells. However, in addition to editing DNA, it has been reported that base editors can also edit RNA. This could have unintended consequences even if the DNA sequence isnt changed.

We didnt look in this study to see if this occurred, which is why we mentioned itjust to be up front about possible unintended consequences/toxicities that could be present and that we didnt exclude. It isnt something specific to our study or gene of interest but generalizable to the entire field of base editing.

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Base Editing as Therapy for Common Inherited Lung and Liver Disease Shows Promise - Clinical OMICs News

IPS Cell Therapy Comments Off on Base Editing as Therapy for Common Inherited Lung and Liver Disease Shows Promise – Clinical OMICs News | July 22nd, 2021

Q: Im considering having my own stem cells injected into me to improve physical and mental problems that I am having post-COVID-19 infection. What do you think?

James D., Huntington, N.Y.

A: Theres been a lot of talk about using what are called autologous stem cells (your own) to fight off COVID-19 long-haul symptoms, as well as to treat everything from torn ligaments to Alzheimers disease. None is approved by the Food and Drug Administration. The only stem-cell-based products that are FDA-approved come from blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood and theyre for treating disorders involving production of blood (the hematopoietic system). A list is at fda.gov; search for Approved Cellular and Gene Therapy Products.

In fact, stem cell/regenerative medicine treatment scams are so prevalent that this spring the FDA finally told manufacturers and marketers that they had to comply with regulations on human cell and tissue products. Unfortunately, a June report from Pew Trust found compliance by the companies and enforcement from the FDA to be anemic.

What the report did find was that more than 700 clinics in the U.S. offer unapproved stem-cell and regenerative medicine interventions for conditions such as Alzheimers, muscular dystrophy, autism, spinal cord injuries and, most recently, COVID-19. They also found post-injection infection happens frequently and is likely because of sloppily manufactured products and failure to properly screen for diseases such as HIV and hepatitis B and C.

If youre considering stem-cell treatment, the FDA urges you to ask the clinic for the following info before getting it even if the stem cells are your own:

Proof the FDA has reviewed and approved the treatment. Have your primary care doc confirm the information.

If the clinic is claiming it has an FDA-issued Investigational New Drug application number, ask for it and ask to review the FDA communication acknowledging the IND.

Stem-cell treatment has great potential, but when used for unapproved therapies outside a clinical trial, its risky (and expensive). To search for a trial, go to clinicaltrials.gov.

Q: My doctor says my high blood pressure puts me at increased risk for dementia. I think hes just trying to get me on one more med. Is there really a connection?

Lacie R., Sacramento, Calif.

A: Dementia means that you have cognition problems that cause trouble with memory, thought and everyday tasks. That could result from mini- or regular strokes, and we know that high blood pressure increases your stroke risk. In fact, one Harvard study found that high blood pressure increases a mans risk of stroke by 220 percent; another found that each 10 mmHg rise in systolic pressure (the top number) boosts your risk of ischemic stroke by 28 percent and of hemorrhagic stroke by 38 percent.

Even if your high blood pressure doesnt trigger a stroke, it can lead to impaired cognition and dementia. The 2018 SPRINT-MIND trial found that intensive control of high blood pressure (getting the top number below 120) lowered the risk of mild cognitive impairment by 19 percent compared with standard blood pressure control. Now, a new study in the journal Hypertension indicates that certain antihypertensive medications ACE inhibitors and ARBs (and angiotensin II receptor blockers) can cross the blood-brain barrier and lower dementia risk. Tracking almost 13,000 people for three years, the researchers found that folks taking those meds showed less memory loss than folks taking other sorts of antihypertensive medications.

You dont indicate how high your blood pressure is, but if it is only slightly elevated you may be able to bring it down through changing your diet, losing weight if you need to and exercising for 30 to 60 minutes five days a week. If it is above 125 (top number) or above 85 (bottom number), a combo of those self-care techniques and medication may be the safest choice. But either way, bringing your blood pressure to around 115/75 will protect your brain, as well as your heart, kidneys and eyes.

Contact Drs. Oz and Roizen at sharecare.com.

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Stemming the tide of stem-cell treatment scams - Houston Chronicle

Spinal Cord Stem Cells Comments Off on Stemming the tide of stem-cell treatment scams – Houston Chronicle | July 22nd, 2021

Wei Wu and Xiao-Ming Xu (Credit: IU School of Medicine)

Researchers at Indiana University School of Medicine (Indianapolis, USA) have announced the successful reprogramming of a glial cell type in the central nervous system into new neurons in order to promote recovery after spinal cord injuryrevealing an untapped potential to leverage the cell for regenerative medicine.

This is the first time that scientists have reported modifying a NG2 gliaa type of supporting cell in the central nervous systeminto functional neurons after spinal cord injury, saidWei Wu, research associate in neurological surgery at IU School of Medicine and co-first author of the paper, which was published in the Cell Stem Cell journal.

Wu andXiao-Ming Xu, the Mari Hulman George professor of Neuroscience Research at IU School of Medicine, worked on the study with a team of scientists from the University of Texas Southwestern Medical Center.

Spinal cord injuries affect hundreds of thousands of people in the United States, with thousands more diagnosed each year. Neurons in the spinal cord dont regenerate after injury, which typically causes a person to experience permanent physical and neurological ailments.

Unfortunately, effective treatments for significant recovery remain to be developed, Xu said. We hope that this new discovery will be translated to a clinically relevant repair strategy that benefits those who suffer from a spinal cord injury.

When the spinal cord is injured, glial cells, of which there are three typesastrocyte, ependymal and NG2respond to form glial scar tissue.

Wu added: Only NG2 glial cells were found to exhibit neurogenic potential in the spinal cord following injury in adult mice, but they failed to generate mature neurons. Interestingly, by elevating the critical transcription factor SOX2, the glia-to-neuron conversion is successfully achieved and accompanied with a reduced glial scar formation and increased functional recovery following spinal cord injury.

The researchers reprogrammed the NG2 cells from the mouse model using elevated levels of SOX2a transcription factor found inside the cell thats essential for neurogenesisto neurons. This conversion has two purposes, Xu said: to generate neurons to replace those lost due to a spinal cord injury and reduce the size of the glial scars in the lesion area of the damaged tissue.

This discovery, serves as an important target in the future for potential therapeutic treatments of spinal cord injury, adds Wu, who goes on to note that such a collaboration will be continued between the two laboratories to address neuronal remodelling and functional recovery after successful conversion of glial cells into functional neurons in future.

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IU School of Medicine researchers discover new potential for functional recovery after spinal cord injury - Spinal News International

Spinal Cord Stem Cells Comments Off on IU School of Medicine researchers discover new potential for functional recovery after spinal cord injury – Spinal News International | July 22nd, 2021