Global Stem Cell Therapy Market Analysis

The recent report published by TMRR on the global Stem Cell Therapy market is an in-depth analysis of the overall prospects of the Stem Cell Therapy market in the upcoming years. The data collected from credible primary and secondary sources is accurately represented in the report backed up by relevant figures, graphs, and tables. The report includes a quantitative and qualitative analysis of the various aspects of the market by collecting data from the key participants in the Stem Cell Therapy market value chain.

The report reveals that the global Stem Cell Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and surpass the value of ~US$XX by the end of 2029. The presented study also includes a thorough analysis of the micro and macroeconomic factors, regulatory framework, and current trends that are expected to influence the growth of the Stem Cell Therapy market during the assessment period.

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Stem Cell Therapy Market Segmentation

The market study put forward by TMRR segments the global Stem Cell Therapy market to offer a microscopic understanding of the various aspects of the Stem Cell Therapy market. The Stem Cell Therapy market is segmented on the basis of region, product type, end-user, and more.

The study offers a Y-o-Y growth projection of each market segment and sub-segment over the stipulated timeframe of the study.

Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Size, Share, Trends, and Opportunity Analysis by 2017 - 2025 - The Market Expedition

London, UK, 4 December 2019:The first UK clinical trial site for the treatment of diabetic patients with chronic limb-threatening ischemia (CLI) using a novel patient-specific regenerative therapy has opened for patient recruitment at the University Hospital of Wales in Cardiff. The site will be evaluating Rexgeneros REX-001 in two Phase III trials, codenamed the SALAMANDER trials. The trials are being led by Mr Ian Williams, a Consultant Vascular Surgeon and the Principal Investigator at the site.

The University Hospital of Wales is participating in the trials through a consortium, the Midlands-Wales Advanced Therapy Treatment Centre (MW-ATTC), part of the Advanced Therapy Treatment Centre Network (ATTC) which aims to bring pioneering advanced therapy medicinal products (ATMPs) to patients. THE MW-ATTC has been working in collaboration with the Cardiff & Vale University Health Board to progress the initiation of the two SALAMANDER trials and is planning to activate new clinical trial sites in the Midlands in England shortly.

CLI is a chronic disease and the most serious form of peripheral arterial disease (PAD), a common condition in which a build-up of fatty deposits in the arteries reduces the blood flow to the legs and feet. CLI is characterized by chronic ischemic at-rest pain, ulcers or gangrene in one or both legs. CLI is a common condition in Europe and the United States affecting 1-1.5% of the population aged over 401. It represents an area of high unmet medical need as there are currently no approved therapies that successfully treat the CLI patient population. Patients with CLI have a very negative prognosis. A year after initial diagnosis, around 12% of patients have had an amputation. Five years after diagnosis the situation is even worse with mortality at 50%, rising to 70% after ten years2.

REX-001 represents a new class of regenerative medicines. It is an autologous cell therapy manufactured using the patients own bone marrow and consists of immune cells (lymphocytes, monocytes and granulocytes) and progenitor cells involved in immune modulation and tissue regeneration. It is administered as a single dose within 4 days after collection of bone marrow cells.

Ian Williams, Consultant Vascular Surgeon and Principal Investigator commented,Chronic limb-threatening ischemia is a serious disease with severe consequences and limited treatment options. There is a high unmet need for novel and innovative therapiessuch as REX-001that have the potential to be a highly effective treatment and to reduce amputation and mortality rates amongst the patient population.

Chris Fegan, Consultant Haematologist, Cardiff and Vale University Health Board said, We have brought together many highly specialized teams from diabetes, surgery, radiology and stem cell transplantation to participate in the pioneering SALAMANDER study here at Cardiff and Vale, which we hope will revolutionize treatment options for patients with chronic limb-threatening ischemia.

Rexgenero, the company pioneering the development of REX-001, says that the experimental product has already demonstrated efficacy in Phase I/II studies. In the Phase II clinical trial, 82% of patients with non-healing ischemic ulcers were healed within the first 12 months after a single administration dose of REX-001.

Joe Dupere, CEO of Rexgenero added, Treating our first patient with REX-001 in the UK will be an important milestone for our Phase III program in diabetic patients with chronic-limb threatening ischemia, a severe condition with high unmet need. With clinical trial sites and manufacturing bases now open across multiple countries in Europe, we are one step closer to completion of the Phase III studies and potential regulatory and market approval for an innovative and much-needed product.

Rexgenero is planning to treat a total of 60 patients with CLI and rest pain and 78 patients with CLI and non-healing ischemic ulcers in two independent Phase III SALAMANDER trials in approximately 25 hospitals across Europe.In addition to the trial sites in the UK, Rexgenero is also recruiting patients for both trials at sites inSpain, Austria, Portugal, Poland, Hungary, the Netherlands and the Czech Republic.

For more information about the REX-001 Phase III SALAMANDER trials, and how to participate, please visit theclinical trial website.

References

ENDS

For further information, please contact:

At Rexgenero

For media enquiries (Rexgenero)

Joe Dupere, CEO+44 (0)20 3700 7480info@rexgenero.com

Instinctif PartnersAshley Tapp+44 (0)20 7866 7923Rexgenero@instinctif.com

At the University Hospital of Wales

Cardiff and University Health BoardCommunications Team+44 (0)29 2074 6381news@wales.nhs.uk

About Rexgenero

Rexgenero is a clinical-stage regenerative medicine company developing innovative cell-based therapies targeting serious diseases with unmet medical needs.

The Companys lead candidate, REX-001, is a highly innovative autologous cell therapy that is being studied in a Phase III clinical programme in patients with chronic limb-threatening ischemia (CLI) with diabetes, a poorly treated disease with a high risk of amputation and death. REX-001 has been shown to be effective in Phase I/II and Phase II trials, alleviating CLI in the majority of patients, offering the potential to increase the quality of life of CLI patients by reducing pain, alleviating ulcers, increasing mobility, improving sleep and reducing the need for amputation. Rexgenero is developing REX-001 in a range of indications and, pending approval, intends to launch and market this specialty product in major territories.

Rexgenero is a privately-owned company, which draws on an exceptional understanding of the fundamental science of cell therapies developed by the Andalusian Health Authority (Servicio Andaluz de Salud) and Andalusian Initiative of Advanced Therapies.

The Company was founded in 2015 and is headquartered in London (UK) with R&D and manufacturing operations in Seville (Spain) and Frankfurt (Germany).

For more information, please visit:www.rexgenero.com

Connect with us: Twitter:@_Rexgenero; LinkedIn:https://www.linkedin.com/company/rexgenero-limited/

About the REX-001 Phase III SALAMANDER Trials

REX-001 has shown efficacy in 70% of patients in Phase I and I/II studies and is currently progressing through two Phase III SALAMANDER trials in Europe being conducted at approximately 30 sites, with plans to enrol a total of 138 patients. The trials are given the name SALAMANDER in reference to the amphibians ability to regenerate its tail and limbs.

ThePhase III studyin patients with Rutherford stage 4 CLI will assess the efficacy and safety of REX-001 with a primary endpoint of complete relief of ischemic rest pain.

ThePhase III studyin patients with Rutherford stage 5 CLI will assess the efficacy and safety with a primary endpoint of complete ulcer healing.

Amputation-free survival is included as a secondary endpoint in both studies. The trials are expected to produce interim analysis in early 2021 with full results expected later that year; all dependent on the speed of patient recruitment.

For more information about the REX-001 Phase III SALAMANDER trials, please visit:https://www.cli-treatment.com

About the Midlands and Wales Advanced Therapy Treatment Centre (MW-ATTC)

The Midlands and Wales Advanced Therapy Treatment Centre (MW-ATTC) consists of a large regional network with the necessary commercial and NHS infrastructure required to facilitate the delivery of advanced therapy treatments to patients. The centre includes a wide range of specialists in advanced therapy manufacturing including academic and commercial partners, logistics companies, specialists in clinical trial delivery and teams focussed on IT solutions and health economics.

For more information, please visit:https://www.theattcnetwork.co.uk/centres/midlands-wales

The ATTC Network Programme is a world-first, UK system of Advanced Therapy Treatment Centres (ATTC) operating within the NHS framework and coordinated by the Cell and Gene Therapy Catapult to address the unique and complex challenges of bringing pioneering advanced therapy medicinal products (ATMPs) to patients. The centres include Innovate Manchester Advanced Therapy Centre Hub (iMATCH), Midlands-Wales Advanced Therapy Treatment Centre (MW-ATTC, comprising Birmingham, Wales and Nottingham) and Northern Alliance Advanced Therapies Treatment Centre (NA-ATTC, comprising Scotland, Newcastle and Leeds).

The network is initially supported by the Industrial Challenge Strategy Fund with the aim to develop first-of-a-kind technologies for the manufacture of innovative medicines across areas including blindness, cancer, heart failure, liver disease, neurological conditions and rare paediatric diseases.

For more information, please visit:https://www.theattcnetwork.co.uk/

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First UK Clinical Trial Site Open for Recruitment of Diabetic Patients with Chronic Limb-Threatening Ischemia Using Novel Patient-Specific...

I have been reading a book, The Man Who Wouldn't Die, that satirizes Silicon Valley and the venture-capital start-up culture. It's reasonably funny. I was reminded, however, that sometimes culture is crazy.

At one point a character casually describes how stem-cell therapy is obviously going to make 50-year-olds feel like 20-year-olds. Grow a new liver and you're good to go, so to speak! The author clearly meant the idea as a joke. However, that idea is out there and taken seriously. Stem cells made national headlines in the late '90s and early 2000s because of some scientific breakthroughs and their promise for future medical treatments.

Just about all of our body is made up of specialized cells. These are cells that have grown to perform some specific function. Some examples are heart muscle cells, say, or red blood cells.

Stem cells are different; they have not yet specialized. That is, they have not developed many of the specific properties that are unique to the different parts of our bodies.

Given the right environment the right chemicals, for examplethey can grow into specialized cells.

There are, of course, all sorts of important details for how stem cells grow, when they specialize and how much flexibility they have in specializing. Somehow, however, there is now an industry that has skipped all that work and is marketing stem cells as a general cure. Alzheimer's disease and joint pain are frequently mentioned. If that makes you suspicious that these clinics are targeting the elderly, you would be right.

A typical treatment involves taking stem cells from a patient's bone marrow or fat and injecting it back into a sore knee or hip or whatever needs fixing. The idea, somewhat vaguely, is that the stem cells will grow to replace whatever is worn down by age.

As is true of all good scams, this one has a good story, one that can pass as proven medicine for many patients. The treatment also benefits by treating a problem with symptoms that can come and go irregularly, so a patient can honestly claim that they recovered in, say,six months.

The body is a complicated machine. It is hard to predict what will cure or not cure any specific problem. Just because a story involving stem cells sounds reasonable is not a sufficient reason to try something.

For a typical patient, we should have good, strong, positive evidence that a treatment will help. A good story is not strong, positive evidence. In the language of science, it is a hypothesis. Potentially true.

It is worth remembering that many, many things are potentially true. We don't try them all out on ourselves when we are sick.

Many of these ideas have in fact been tested in rigorous, peer-reviewed studies. So far, the evidence for positive effects is weak.

One reason these clinics have managed to skirt the rules is that extracting material from a patient then reinjecting requires less oversight for safety. Depending on the details, this treatment is considered, for Food and Drug Administration regulations, something like plasma donation (where blood is extracted, platelets removed, then reinjected into the body).

Just because a process may be safe does not make it good medicine. Medicine is not and should not be a free market. Patients will almost never have enough knowledge to reasonably choose between treatment options.

The FDA is the federal agency in charge of ensuring companies do not offer useless or harmful procedures. Unfortunately, in 2017 the FDA decided to allow stem cell clinics a three-year grace period to describe their procedures for the FDA's evaluation. As a result of that open window, hundreds of clinics have opened nationwide, offering services for which there is no strong evidence.

In the past year the FDA has realized where the industry has gone with this treatment and begun trying to crack down. Some clinics have unsafe procedures; others are misleading patients about treatments. Many clinics are still operating, unfortunately, and it can be difficult for typical patients to recognize the difference between these treatments and proven remedies. Furthermore, these bad actors could potentially make people skeptical of stem cell-based technology entirely.

That would be a shame because there is still great promise for stem cells. That may be cold comfort for those who want a treatment immediately, but that desire is the exact motivation that has led to terrible medicine for centuries.

Christer Watson, of Fort Wayne, is a professor of physics at Manchester University. Opinions expressed are his own. He wrote this column for The Journal Gazette, where his columns normally appear the first and third Tuesday of each month.

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Stem of the problem | Columns | Journal Gazette - Fort Wayne Journal Gazette

This fall, 21-year-old Jurnee Farrell, a Howard University senior and a member of the universitys volleyball team donated stem cells to a complete stranger.

This fall, 21-year-old Jurnee Farrell, a Howard University senior and a member of the universitys volleyball team, was set to play in the Mid-Eastern Athletic Conference Tournament.

But instead, she was sidelined by a decision she made herself.

Two years ago, she signed up with Be the Match, a nonprofit that registers potential bone marrow and stem cell donors. When she got the call that she was a match for a 57-year-old woman with a form of leukemia, she was surprised.

At first I was like, This isnt real, Farrell recalled, but then said her decision was clear. She would follow through on the commitment she made two years ago when she signed up.

That meant undergoing a series of shots five days before the outpatient procedure, and then undergoing apheresis, a process in which the donor has blood removed through a needle in one arm, blood-forming cells are collected, and then the blood is returned through a needle in the other arm. The session can take up to eight hours.

Farrell said that the actual donation wasnt bad, but that the shots given in a series five days prior proved a little uncomfortable.

Nevertheless, she urges potential donors to sign up.

Its really not that bad, and the person whose life youre saving is probably going through so much more than you are, she said.

And while Farrell missed the tournament last month, shes back on the court already. We are going to the NCAA tournament this weekend, actually, and we play Pitt on Friday, she said. I just started practicing last week!

Farrells decision to register as a donor is one for which the staff at Be the Match is especially grateful.

Lauren Mueller, a public relations specialist with Be the Match, explained that for Caucasian patients waiting for a bone marrow or stem cell donation, the odds of finding a match are roughly 70%. For African-American patients, the odds are much lower, at 23%.

As a result, we are always looking to increase our diversity on the registry, said Mueller, who encourages people to consider registering.

It starts with a cheek swab, and Mueller said its not uncommon for years to go by before a potential donor hears that they might be a match, just as it was in Farrells case.

Your selfless action can help save a life, Mueller said.

Farrell said that shes hopeful her donation will prove successful, and that she would love to meet the recipient one day.

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Howard University athlete makes the call to donate stem cells - WTOP

The latest market report published by Credence Research, Inc.GlobalBlood and Bone Marrow Cancer Treatment Market Growth, Future Prospects, Competitive Analysis, 2019- 2027,the global market for the treatment of blood and bone marrow cancer was valued at US$ 38.8 billion in 2018 and is expected to reach US$ 74.9 billion by 2027, increasing to CAGR by 7.7% from 2019 to 2027.

Market Insights

Blood cancer begins in the bone marrow, an integral source of stem cells that are later differentiated between different types of blood cells in the human body. Researchers at Bristol Myers Squibb Company have indicated that around 1.85 million new cases of blood cancer will be diagnosed worldwide by 2040.

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Lymphoma is the largest indication in the blood and bone marrow cancer treatment market. It is prevalent across the globe in two types of Hodgkin lymphoma and non-Hodgkin lymphoma. The main variables responsible for increasing prevalence worldwide are increasing the prescription of immunosuppressive drugs for the treatment of chronic infections and genetic mutations. Leukemia occurs when the DNA of immature white blood cells is damaged by exposure to ionizing radiation, hazardous chemicals, smoking, etc. The prevalence rate of leukemia is highly variable across different ethnic groups with a male-to-female ratio of 1.4.

Chemotherapy is part of the blood and bone marrow cancer therapy market. The key factor responsible for increasing demand is the availability of its generic version at an affordable cost, which significantly reduces the health burden on sick patients. Oncologists prefer to use them in combination therapy with either radiotherapy or immunotherapy to treat patients with resistance to first-line drug therapy. Immunotherapy will be the fastest growing segment in the forecast period due to its promising drug line for the treatment of blood cancer.

North America representing a market share of 34.6% is dominating the regional segment for blood and bone marrow cancer treatment market. The chief contributing factor for its market supremacy is growing incidence of blood cancer. As per the research citings of the Leukemia and Lymphoma Society (CDC) figures after every 3 minutes 1 person in the U.S. is diagnosed with blood cancer. In 2019, approximately 176,200 people in the U.S. are diagnosed with blood cancer in the United States. Existence of key players such as F.Hoffman La-Roche Ltd., Pfizer, Inc., Novartis AG, Merck & Co., Inc., etc further strengthens the market growth. Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer. Asia Pacific accounts for 18.4% market share on account of rising public health awareness related to blood cancer & its treatment and developing healthcare infrastructure.

Pharmaceutical companies spearheading the blood and bone marrow cancer treatment market are Bristol Myers Squibb & Company, AstraZeneca, Plc., Eli Lilly & Company, Johnson and Johnson Company, F.Hoffman La-Roche Ltd., Celgene, Inc., Merck & Co., Inc., Novartis AG, Varian Medical Systems, Inc. and Pfizer, Inc.

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By Geography Segment

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Blood and Bone Marrow Cancer Treatment Market Is Expected To Reach US$ 74.9 Bn by 2027 - Credence Turbine

WASHINGTON WhenHoward University student athlete Jurnee Farrellsigned up for the Be The Match registry during a Get In The Game campus drive, she didnt know if she would ever be called upon. However, when her phone rang two years later and she discovered she was a match for a 57-year-old woman suffering from acute lymphoblastic leukemia, she did not hesitate to participate.

The football team was hosting a Be The Match booth after Coach London had donated bone marrow to his daughter, recalls Farrell. I filled out a little form and turned it in. When I got the call a few months ago, they asked if I was still interested in donating. I didnt hesitate to say yes because this is somebodys life and there was no way I could say no to that.

Farrell is a senior criminology major from Denver, Colorado, and a member of the Howard University MEAC Championship Volleyball team. Wearing jersey No. 5 as a defensive specialist, Farrell is known for her bubbly personality off the court and her intensity on the court. At senior night, the day before her donation, the entire team rallied behind her in support of her decision. Unfortunately, the timing of the donation process meant that Farrell was not able to participate in this years MEAC playoffs with her teammates, who brought home their fifth MEAC championship on Nov. 24.

Of course, we were sad to miss out on having Jurnee play in the playoffs, but this is such a worthy cause and we were happy to support her all the way, saysHead Volleyball Coach Shaun Kupferberg.What she is doing speaks directly to Howard Universitys mission of truth and service, that each student comes here not only to learn, but to make a difference in the global community. Im extremely proud of her decision.

Thanks to several medical advances, the process to donate stem cells has drastically changed over the years. In addition to bone marrow donations, doctors can also use a stem cell procedure called Peripheral Blood Stem Cell (PBSC) donation through a short 4-hour out-patient procedure where blood is circulated from one arm, into a machine and then back into the donor.

Beth Carrion, account manager for Registry Growth and Development, says individuals like Farrell help to demystify the giving process and raise awareness of the need for a more diverse donor registry. The chances of finding a match for a stem cell transplant is dependent on a persons genetic markers. Outside of a family member, finding a donor within in ones ethnicity is the next viable option. According to Be The Match, each year approximately 14,000 patients are waiting for a transplant from someone outside of their family. The current odds to match a patient with a donor in one out of 430.

Be the Match is truly thankful for our partnership with Howard University because it plays a vital role in helping the African American community have a higher rate of finding a match, says Carrion. A white person in the registry has a 78 percent chance of finding a match. For Hispanics, its 46 percent, but for African Americans, its only a 23 percent chance. We look forward to hosting more events with Howard this spring.

One week after her procedure, Farrell is back at volleyball practice with her fellow teammates, preparing for the Tournament. As she looks to finish out her senior year, she says shes also hopeful that shell get to meet the woman she helped one day.

After the donation, I can have anonymous communication, but I cannot tell my identity. After a year, they will deem the transplant successful and then we can communicate, says Farrell. I for sure want to meet her.

To join the Howard University registry, text Howard to 61474, follow the prompts and a kit will be mailed to you. You may also register online atjoin.bethematch.org/howard.

# # #

Media Contact: Alonda Thomas,Alonda.Thomas@Howard.edu

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Howard University Student Gives a Stranger the Greatest Gift for the Holidays: Life - Howard Newsroom

Hes done it - Stephen Armstrong has scooped a prestigious award for his huge fund raising efforts as his son fought a rare blood disorder.

The doting dad took home the award for Individual Fundraiser of the Year at the Anthony Nolan Supporter Awards 2019 ceremony held at the Tower of London.

The top awards were back for their seventh year to recognise the outstanding achievements of the volunteers, fundraisers and campaigners who help the pioneering blood cancer charity save lives.

And the award is in recognition of Stephens incredible fundraising efforts - leading a group of 19 friends and family in a series of physical challenges, all while his son Jacob was undergoing treatment.

When Jacob was diagnosed in 2017 at two-years-old, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob is now four-years-old and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, 33, of Wallsend,North Tyneside.

On winning the award, Stephen said: I feel very proud- I really didnt expect it. You dont do it for recognition, but to get more people to join the register.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen, who has raised a further 8,000 for other smaller charities, added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Henny Braund, Chief Executive at Anthony Nolan, said: Stephen is a hugely deserving winner of this award; his incredible support and passion for our work is a fantastic example of our charity, which is built on making lifesaving connections. It was lovely to meet Stephen and I continue to find myself inspired and humbled by the dedication and strength of supporters like him.

By raising vital funds and much needed awareness, we are curing blood cancer together. We can give families hope, and give more people a future. But without supporters like Stephen, lives cant be saved. Without him, there is no cure.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

To see the full shortlist, and find out more about the charity visit the website here.

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Wallsend dad who called on the public for stem cells for his son scoops top Anthony Nolan Award - Chronicle Live

Cancer | One of the most common disease in the world

In what may prove as a breakthrough in the treatment of leukaemia and other blood diseases, scientists at UCLA have discovered a protein produced by a gene known as MLLT3 and its connection to the multiplication of human blood stem cells.

The discovery which was published in a study is very much significant as cancers such as leukaemia can be effectively treated using blood stem cells, also known as Hematopoietic stem cells (HSCs), produced outside the human body and could serve as an alternative to existing treatment options such as bone marrow transplants.

Self-renewal is the process by which stem cells divide to create more cells. The study focused on a specific type of the kind: Hematopoietic stem cells (HSCs) which are present within the bone marrow where along with self-renewal, they also produce different types of blood cells such as red and white by transforming into them.

Placing HSCs in laboratory dishes after their removal from the bone marrow causes then to lose their ability to self-renew, and they either transform into other blood types or perish. It is this process that the scientists studied. Through a series of steps, the researchers studied the genes that shutdown as the cells lost their capacity to self-renew.

They discovered that the HSCs' ability to self-renew corresponded with the expression of a gene called MLLT3. They also found that MLLT3 generated a protein that instructed HSCs to retain their capacity to self-renew. As the cells divide, the protein works along with other regulatory proteins to keep vital components of the HSCs' functioning.

Employing a viral vector the researchers tried to ascertain if maintaining the MLLT3 protein levels in lab dishes would help improve the self-renewing abilities of HSCs. A viral vector is a specially designed virus that transfers genetic information to the nucleus of a cell without giving rise to a disease. Using the vector, the scientists introduced an active MLLT3 gene into HSCs. They found that there was nearly a twelvefold multiplication of working HSCs in lab dishes.

"If we think about the amount of blood stem cells needed to treat a patient, that's a significant number," said Dr. Hanna Mikkola, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and senior author of the study, to the UCLA Newsroom.

The UCLA scientists observed that the use of 'small molecules' organic compounds that help in the multiplication of human HSCs, improved self-renewal in general. However, the cells were unable to maintain stable MLLT3 levels and did not perform well when implanted into mice.

"Our method, which exposes blood stem cells to the small molecules and also inserts an active MLLT3 gene, created blood stem cells that integrated well into mouse bone marrow, efficiently produced all blood cell types and maintained their self-renewing ability," said Vincenzo Calvanese, a UCLA project scientist and the study's co-corresponding author, to the UCLA Newsroom.

The team noted that the self-renewal of HSCs caused by MLLT3 was at a safe rate. This means that they did not acquire potent characteristics such as mutation or excessive multiplication, or the production aberrant cells that can cause leukaemia.

Determining which proteins and constituents within the DNA of the HSCs affect the activation and deactivation of MLLT3 is the next step for the team. Also, understanding the regulation of the process using components in the lab dishes is another step. The information gathered may help find ways in which MLLT3 could be switched on and off without employing a viral vector.

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Protein that can help treatment of leukemia identified, UCLA scientists upbeat - International Business Times, Singapore Edition

MAGICAL Nature is Rebecca Bennett's first exhibition in four years following her stem cell transplant.

Running throughout December at Rydal Hall Old School Room Teashop at Ambleside, the exhibition features various media from acrylics and watercolours to photography.

Rebecca loves capturing the magic of colour and pattern within nature in her artwork. She grew up surrounded by the beautiful fells and lakes of Cumbria. Coming from an artistic family, painting and drawing from an early age was natural.

From photographs of lakes seen through the prism of a crystal ball to an enigmatic acrylic painting of a grey heron, Magical Nature includes striking images of the wildlife and countryside of Rebeccas native Lake District.

With a BA(hons) degree in Contemporary Applied Arts from Cumbria Institute of the Arts, Rebecca furthered her art practice and skills to create porcelain ceramic pieces and textile mixed media artworks.

Following university Rebecca completed a variety of successful art projects and workshops alongside her exhibitions. These included art workshops with young people at the Coniston Water Festival and Blencathra Field Studies Centre.

In 2015 Rebecca had a stem cell transplant at London's Kings College Hospital. An unrelated donor provided the stem cells to treat failing bone marrow caused by rare Gata2 deficiency and Myelodysplastic Syndrome. Painting and photography have, she says, been a great therapy throughout her transplant journey.

"When you feel up to it having something to immerse yourself in such as art and photography can be a fantastic way to help you forget about your pain and problems. For those moments, you are focused on what you are creating and can escape for a little while."

Alongside exhibiting her work for the first time since being diagnosed with her illnesses, Rebecca hopes to raise awareness of MDS and blood disorders. Donations to the charity MDS UK patient support will be made from sales of Rebeccas greeting cards and prints during the exhibition.

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Artist's first exhibition following stem cell transplant - The Westmorland Gazette

Emi receives kisses from her adoptive parents, Jason and Katie Ballard, at the NIH Clinical Center as she gets ready to receive a lifesaving hematopoietic stem cell transplant, the only way to cure her fatal immune deficiency, using cells donated by her birth mom.

Emi smiles at The Children's Inn at NIH prior to undergoing a hematopoietic stem cell transplant, the only potential cure for her rare and deadly immune deficiency.

Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) -- One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow transplantthe only chance for a cure for her rare and deadly disease. But Emis story is not only a story about the triumph of medical research that is making her cure possibleits also a story about extraordinary parental love and sacrifices by her birth mom and her adoptive family that are giving this very ill girl the best chance at life. Emi's birth mom donated her stem cells to make the lifesaving transplant possible.

We are most thankful for an answer to years of prayers, Emis adoptive mom says. Emi got a new start at life, a rebirth day. Every holiday this year will be like the first. Were so grateful to the doctors, nurses and The Childrens Inn.

Emi and her family will be celebrating Thanksgiving at The Childrens Inn at NIH, a nonprofit hospitality house that provides free lodging and a wide variety of support services to families of children with rare and serious diseases whose best chance for a treatment is a clinical research study at the National Institutes of Health. Emi and her mom have spent several months at The Childrens Inn so far and bonded with other families. On Thanksgiving Day, families staying at The Childrens Inn who cannot go home for the holiday will be served a traditional Thanksgiving meal prepared by a group of dedicated volunteers.

It took two moms who love this little nugget to fight for her right to life, Emis adoptive mom says. We finally are getting to see that beautiful part of the story that we always knew was there.

Read Emis full story.

See photos of Emi and her family.

About The Childrens Inn at NIH:

The Childrens Inn at NIH provides free lodging and a wide range of supportive services to more than 1,500 children and their families every year whose best chance for a treatment is a clinical trial at the National Institutes of Health. Opened in 1990 and located across from the NIH Clinical Center, the worlds largest hospital dedicated entirely to medical research, The Childrens Inn has welcomed children from all 50 states and 94 countries. Children staying at The Childrens Inn are making important contributions to rare disease and cancer research, including the successful treatment of childhood leukemia, as well as treatments for HIV/AIDS, childhood asthma, bone and growth diseases, childhood onset schizophrenia and other mental health issues, neurofibromatosis type 1 and a wide variety of genetic and rare diseases. For more information, visit http://www.childrensinn.org. To support The Childrens Inn, make a donation at http://www.childrensinn.org/donate.

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The Best Thanksgiving - GlobeNewswire

ANI | Updated: Nov 29, 2019 20:58 IST

Washington D.C. [USA], Nov 29 (ANI): Researchers have discovered a stem cell therapy that might help the heart recuperate from an attack.This study published in the journal Nature reported that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after a heart attack, according to the principal investigator Jeffery Molkentin, PhD, director of Molecular Cardiovascular Microbiology a Cincinnati Children's Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI)."The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties," Molkentin said.The findings build on a 2014 study published by the same research team. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesn't work.The findings prompted Molkentin and his colleagues to conclude that there is a need to "re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work."Researchers worked with two types of heart stem cells currently used in the clinical trials -- bone marrow mononuclear cells and cardiac progenitor cells.As they went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response.They reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extracellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself.Researchers also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury."Most of the current trials were also incorrectly designed because they infuse cells into the vasculature. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic," Molkentin explained. (ANI)

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Researchers discover a stem cell therapy that can help heal injured heart - ANI News

Hematopoietic stem cells (HSCs) can be used to better understand and treat blood-based diseases. Stem cell research in the model organism zebrafish is well-studied in the developmental stage, but is limited in the adult stage because HSCs are difficult to purify in this species. Researchers at Kanazawa University and their collaborators have developed a new purification scheme that allows HSCs to be purified from adult zebrafish kidneys, potentially opening new possibilities for stem cell research.

Kanazawa, Japan Hematopoietic stem cells (HSCs) are multipotent cells that can develop into every type of blood cell in the body. They can also be used in medical research to understand and treat blood-based diseases. Zebrafish (Danio rerio) are used to study HSCs, particularly in the field of developmental biology, but the research in the adult animal is often limited because stem cells are difficult to purify in this species. Researchers at Kanazawa University and their collaborators now describe a purification scheme that allows these elusive zebrafish HSCs to be collected.

Zebrafish are a great system to study how hematopoietic cells function in normal development and their role in disease, says lead researcher Isao Kobayashi. Much of their biology mirrors what we see in humans, and with zebrafish theres the added benefit of having quite a few experimental tools at our fingertips, including live cell imaging and comparative analysis among vertebrates. Unfortunately, its proven challenging to effectively isolate HSCs from this species, and this has been a major impediment to the field.

HSCs are highly abundant in the kidneys of adult zebrafish (unlike in humans, where HSCs are found in bone marrow). The challenge is separating them from other cells found in kidneys. Cell separation usually involves a purification technique called flow cytometry, where cells are sent in single file through a tube and hit with a laser beam. The machine (a flow cytometer) then sorts the cells based on how they reflect or scatter light.

In the study, published in Scientific Reports, the researchers created a strain of zebrafish that makes two light-emitting proteins, one green (Green Fluorescent Protein, GFP) and one red (mCherry), that can be sensed and sorted by a flow cytometer. Each fluorescent protein in this zebrafish strain was regulated by the genes related with blood cells, but the cells having both fluorescent proteins were limited in HSCs. By color coding the cells with two distinct blood cell markers, the team was able to purify cells that show hallmark signs stemness like the ability to self-propagate and differentiate into other types of blood cells.

So, what might the successful isolation of HSCs in zebrafish mean for the field of stem cell research?

When HSCs were finally purified in mice, the research community learned an enormous amount about how and where stem cells self-renew and differentiate to form blood cells, says co-author Mao Kondo. Were very hopeful that this might spur a similar proliferation of research in zebrafish. In addition to some experimental advantages in zebrafish, we found that zebrafish HSCs share many key genes in common with HSCs in mammals. This suggests that mechanistic discoveries in zebrafish could have direct implications for understanding blood diseases in humans and for developing new medical treatments.

Figure.

Hematopoietic stem cells can be isolated as gata2a:GFP+ runx1:mCherry+ (gata2a+ runx1+) cells in the zebrafish kidney by flow cytometry (left panels). Transplantation assays confirmed the hematopoietic potential of gata2a+ runx1+cells (right panels).

Article

Enrichment of hematopoietic stem/progenitor cells in the zebrafish kidney

Journal: Scientific Reports

Authors: Isao Kobayashi, Mao Kondo, Shiori Yamamori, Jingjing Kobayashi-Sun, Makoto Taniguchi, Kaori Kanemaru, Fumihiko Katakura & David Traver

DOI: 10.1038/s41598-019-50672-5

Funder

This work was supported in part by Grant-in-Aid for Young Scientists (B) from the Japan Society for the Promotion of Science (17K15393).

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Researchers Find a Way to Collect Elusive Blood Stem Cells from Zebrafish - Mirage News

A TODDLER born with two types of leukaemia will spend Christmas at home after recovering from a second stem cell transplant.

Amelia Topa, who turned two on Wednesday, was donated cells harvested from a newborn babys umbilical cord blood flown in from the US.

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The brave youngster is now in remission and is preparing to enjoy the festive season with relieved parents Kerri Paton, 23, and Igor Topa, 24, in Turriff, Aberdeenshire.

Recalling Junes life-saving op, mum Kerri said: Amelia soared through the transplant and shes doing really well. I couldnt be prouder.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Worried medics alerted Amelias parents to raised purple spots on her body shortly after she was born at Dr Grays Hospital, Elgin.

She was diagnosed with acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas 2017 in hospital.

The tot was given a bone marrow transplant six months later using stem cells donated by a man aged between 16 and 30.

The treatment worked but tests showed Amelias cancer had returned in February.

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She endured gruelling chemo before her second transplant in the summer.

Kerri added: I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

Amelia has now been chosen to receive Cancer Research UKs first Children & Young People Star Award.

The prize, backed by TK Maxx and stars including Dame Emma Thompson, is open to under-18s battling cancer or who have been treated in the last five years.

The charitys Lisa Adams said: Were calling on families to nominate inspirational youngsters so we can recognise their courage.

VISIT cruk.org/childrenand youngpeople to nominate.

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This Mesenchymal Stem Cells research study consists of the historical data from and forecasts till 2026. The report is created keeping in mind to make it a valuable source of information for market specialists in readily accessible documents. The documents are curated with clearly presented graphs and figures.

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The global mesenchymal stem cells market size to reach USD 2,518.5 Million by 2026, growing at a CAGR of 7.0% during forecast period, according to a new research report published by Alexa Reports Research. The report Mesenchymal Stem Cells Market, [By Source (Bone Marrow, Umbilical Cord Blood, Peripheral Blood, Lung Tissue, Synovial Tissues, Amniotic Fluids, Adipose Tissues); By Application (Injuries, Drug Discovery, Cardiovascular Infraction, Others); By Region]: Market Size & Forecast, 2018 2026 provides an extensive analysis of present market dynamics and predicted future trends. The market was valued at USD 1,335.1 million in 2017. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

are adult stem cells, which are traditionally found in the bone marrow. However, they can also be parted from other available tissues including peripheral blood, cord blood, fallopian tube. These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The significant aspects that are increasing the development in market for mesenchymal stem cells consist of enhancing need for these stem cells as an efficient therapy option for knee replacement. Raising senior populace throughout the world, as well as increasing frequency of numerous persistent conditions consisting of cancer cells, autoimmune illness, bone and cartilage diseases are elements anticipated to enhance the market development throughout the forecast period. The mesenchymal stem cells market is obtaining favorable assistance by the reliable federal government policies, as well as funding for R&D activities which is anticipated to influence the market growth over coming years. According to the reports released by world health organization (WHO), by 2050 individuals aged over 60 will certainly make up greater than 20% of the globes population. Of that 20%, a traditional quote of 15% is estimated to have symptomatic OA, as well as one-third of these individuals are expected to be influenced by extreme specials needs. Taking into consideration all these aspects, the market for mesenchymal stem cells will certainly witness a substantial development in the future.

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Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

Alexa Reports Research has segmented the global mesenchymal stem cells market on the basis of source type, application and region:

Mesenchymal Stem Cells Source Type Outlook (Revenue, USD Million, 2015 2026)

Bone MarrowUmbilical Cord BloodPeripheral BloodLung TissueSynovial TissuesAmniotic FluidsAdipose Tissues

Mesenchymal Stem Cells Application Outlook (Revenue, USD Million, 2015 2026)

InjuriesDrug DiscoveryCardiovascular InfractionOthers

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About Us:Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data. Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data.

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Angie Grice is a such huge Clemson football fan that in 20 years shes rarely missed a home game or any of the Tigers-Gamecocks match-ups.

At her tailgate parties, the plates, the tablecloth and even a rug are orange.

Angie Grice gets a visit from the Clemson Tigers mascot during her three months in the hospital.(Photo: Bon Secours St. Francis Health System)

But for this years annual Thanksgiving weekend face-off between Clemson and USC, shell be watching from home.

Diagnosed with aplastic anemia in May, the Simpsonville woman spent three months in the hospital and is still too sick to cheer her beloved Tigersfromthe stadium. Instead, she hopes to have a few friends over to catch the gameon TV.

Ive liked Clemson forever," she told The Greenville News.

"Im missing the game this yearand Im sad about that, she said.But its OK. At least Im able to watch it.

Grice, 52, first realized something was wrong in August 2018 when she suddenly had trouble crossing the parking lot from her car to her job as a physical therapy assistant.

I was very short of breath, she recalls. It would take me a long time to do anything. I just couldnt breathe.

She saw her family doctor, who sent her to Bon Secours St. Francis Health System when her blood work wassuspicious.

Angie Grice at Clemson University(Photo: Angie Grice)

An initial bone marrow biopsy was negative.But a second revealedaplastic anemia,which prevents the bone marrow frommakingenough new blood cells for the body to function normally, according to the National Institutes of Health.

The condition is so rare it strikes only 600 to 900 Americansa year, according to the The Aplastic Anemia and MDS International Foundation.

Symptoms include fatigue, weakness, dizziness, shortness of breath, infections, and easy bruising or bleeding,the NIHreports.The cause can bethe bodys own immune system attackingthe bone marrow, heredity, some drugs, and certain toxins likepesticides and benzene.

When St. Francis hematologist Dr. Fahd Quddus first saw her, Grices platelet level was 8,000 compared to a normal of 150,000.

Whenever you drop below 20,000, youre at risk of significant, life-threatening bleeding, he said. She also had significant anemia. And her white cells were also very low.

She was started on immunosuppressive medication and other drugs in combination with blood transfusions. But sadly, he said, she suffered multiple infections, fevers and a mild stroke, requiring her to stay in the hospital.

Dr. Fahd Quddus(Photo: Bon Secours St. Francis Health System)

For a few weeks, it was touch and go, Quddus said. She was very sick.

Grice'sblood counts eventually rebounded and though shes now out of the hospital, shestill needsregulartransfusions.

She's wellenough to begin a new treatment, he said, butnot yet strong enoughfor a stem celltransplant.

Theresstill a long road to recovery, Quddussaid. But she always looks at it half full. And thats a good thing because people who stay positive can do better.

No longer able to work because of the weakness and danger of infection, Gricesays shes doing OK thanks tofamily and friends.

Angie Grice at a Clemson game(Photo: Angie Grice)

My mom and dad and sister help, she says. And I am truly blessed with a lot of friends who help.

In years past, Grice and her friends arrived at the stadiumseveral hours before kick-off, spending 10 to 12 hours thereon game days.

Inside their orange tent, they set up a coupleTVs to watch other games before and after the Clemson game. There was always plenty ofgood food,smack talk and Tigersmerchandise.

Were a little over the top, she says. But its fun.

During her grueling three-monthhospital stay, it was a visit from the Clemson Tigers mascot that lifted her spirits.

One of Angie Grice's many Clemson decorations(Photo: Angie Grice)

While watching from home wont be as exciting, Grice says shes going to make the best of it. And when asked whos going to win this years game, sheexclaims, Clemson, of course!

If you ask Carolina, they will say they are, she adds with a chuckle. But theyre delusional. Were going to win this year.

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Despite illness, this Clemson fan will be tuned in for the Tigers-Gamecocks game - Greenville News

It may finally be a happy Christmas for a brave toddler recovering from a rare combination of cancers after pioneering stem cell treatment.

Amelia Topa, who celebrated her second birthday yesterday, is looking forward to spending the festive period with her family at home in Turriff.

Her parents Kerri Paton, 23, and Igor Topa, 24, were told that raised purple spots across Amelias body could be a sign of something seriously wrong when she was born at Dr Grays Hospital in Elgin.

Amelia was soon after diagnosed with a mix of two types of leukemia acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas in hospital.

Miss Paton said: Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Doctors gave Amelia a bone marrow transplant using stem cells donated by a managed between 16 and 30.

The treatment worked and, by the following autumn, she was home and awaiting the arrival of her baby brother.

But tragedy struck when Amelias grandmother, Angela McNabb, died suddenly from heart failure aged 48 the day before Amelias birthday.

Miss Paton said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her.

My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

Things went from bad to worse for the family in February, when tests showed that Amelias cancer had returned.

After intense chemotherapy she was given a second transplant using stem cells from umbilical cord blood flown specially from America at the end of June.

That procedure was a success and the toddler has entered remission.

Having spent Christmas in 2017 in hospital, and in mourning last year, Amelias parents are now looking forward to a happy festive season.

Miss Paton said: Amelia soared through the transplant and shes doing really well now,I couldnt be prouder.

I hope Amelias story will help other families going through cancer there is a light at the end of the tunnel.

Amelia has now been selected to receive the first Cancer Research UK children and young people star award in recognition of the courage she showed since being diagnosed.

The award, supported by TK Maxx, is open to all people under 18s who currently have cancer or who have been treated in the last five years with every child being awarded a trophy, TK Maxx gift card, t-shirt and certificate signed by the likes of Nanny McPhee star Dame Emma Thompson.

Spokeswoman for the charity, Lisa Adams, said: We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families.

Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

Nominations can be made online at cruk.org/childrenandyoungpeople

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North-east toddler overcomes cancer after pioneering stem cell treatment - Press and Journal

(MENAFN - The Conversation) The global AIDS response has made significant progress in reducing HIV infections and AIDS-related deaths. New HIV infections dropped by16%from 1.9 million 2010 to 1.6 million in 2017. And the number of AIDS-related deaths decreased from 1.4 million to 940 000 in the same period.

But HIV/AIDS has not been brought under control and new infections continue to drive the epidemic. AIDS remains a leadingcause of deathin Africa.

Even if new infections are prevented,36.9 millionpeople with HIV around the world must take antiretroviral treatment to live a healthy life. While treatment is now as simple as taking a single pill a day, there are still many challenges to daily adherence, including ongoing stigma.

An ultimate solution would be a workable cure. At the recent Conference on Retroviruses and Opportunistic Infections researchersconfirmedthe second ever case of HIV remission or 'cure'. Known as the 'London patient', the person went into remission after a stem cell transplant as part of his treatment for cancer. He emerged from the procedure free of both his life-threatening Lymphoma and need for anti-HIV therapy.

The'Berlin patient' , Timothy Brown, made global headlines in 2008 when scientists announced that he had been cured of HIV. It's been 12 years since Brown was cured, after undergoing chemotherapy, total body irradiation and two stem cell transplants. Brown has been off treatment since the transplant and, after multiple tissue sampling procedures, has no remaining evidence of HIV reservoirs. The London patient is now the longest adult HIV remission after stem cell transplantation since the 'Berlin patient'.

This development is a triumph for medical science as well as for the London patient. But, as exciting as it is, stem cell transplant is a gruelling and dangerous procedure and isn't the magic bullet that will end HIV/AIDS. This is because it's unfortunately not a scalable, feasible cure for the 39 million people currently living with HIV.

The 'London patient' was HIV positive, but it was his Hodgkin's lymphoma that led to the need for a stem cell transplant.

The HI virus must link to a human host T cell in the blood or lymph nodes to replicate and infect the body. The virus attaches itself to a set of special links on the human T cell. If one of those links isn't available due to genetic mutations, the virus may find it harder to get an infection foothold.

One such genetic mutation occurs in a link called the 'CCR5 receptor'. Some people have this mutation naturally. The 'London patient', while on antiretroviral therapy and virally suppressed, had a bone marrow transplant as part of his lymphoma treatment. The bone marrow donor had the genetic mutation and passed it on to the 'London patient' through the procedure, making it more difficult for HIV to replicate.

The 'London patient' stopped taking antiretroviral therapy 16 months after the transplant. And 18 months later the virus remains undetectable. Usually, when a person with HIV stops treatment, the virus rebounds within the first month.

The achievement of remission in a second patient has provided further critical information to inform our understanding of how HIV infection occurs and the interaction between human cells and the virus.

As important as this work is, there's no scalable cure yet and it's also vital that researchers and countries keep putting effort into prevention. Important work continues to be done in this area.

As HIV cure research goes on, so does research into HIV prevention tools, such asPre-exposure prophylaxis(a daily pill that protects you from HIV infection) and the development of apreventative vaccine .

Two late stage vaccinetrialsare underway in sub-Saharan Africa. Results will be available in 2022. A preventative vaccine would also greatly enhance efforts to being the HIV epidemic under control.

A working cure, together with a preventative vaccine would be the ingredients for HIV eradication. Until then we need to get effective, accessible treatment for all who need it, while deploying the many prevention tools at our disposal.

MENAFN2811201901990000ID1099339725

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Why ending HIV still rests on a working cure -- as well as prevention - MENAFN.COM

Eileen Booker sat in her Southern California home last Sunday night, watching the Packers game like she does every week.

She grew up in Green Bay, and her sister still lives there. Her parents bought season tickets in 1957 and her father never missed a home game. She remembers sticking to frigid metal bleachers as a kid until the clock showed zeroes in the fourth quarter, win or lose, and always burning her lips with hot chocolate.

Still a die-hard fan today, Eileen was glued to her television for a prime-time game between the Packers and 49ers, even as her favorite team trailed, 10-0, early in the second quarter.

She had no idea her name was about to be plastered on TV screens across America.

After Packers outside linebacker ZaDarius Smith sacked 49ers quarterback Jimmy Garoppolo deep in 49ers territory, he immediately found the nearest camera and lifted his jersey, revealing a white undershirt that read, WE ...

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Newswise UCLA scientists have discovered a link between a protein and the ability of human blood stem cells to self-renew. In a study published today in the journal Nature, the team reports that activating the protein causes blood stem cells to self-renew at least twelvefold in laboratory conditions.

Multiplying blood stem cells in conditions outside the human body could greatly improve treatment options for blood cancers like leukemia and for many inherited blood diseases.

Dr. Hanna Mikkola, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAand senior author of the study, has studied blood stem cells for more than 20 years.

Although weve learned a lot about the biology of these cells over the years, one key challenge has remained: making human blood stem cells self-renew in the lab, she said. We have to overcome this obstacle to move the field forward.

Blood stem cells, also known as hematopoietic stem cells, are found in the bone marrow, where they self-renew as well as differentiate to create all types of blood cells.Bone marrow transplants have been used for decades to treat people with some diseases of the blood or immune system. However, bone marrow transplants have significant limitations: Finding a compatible bone marrow donor is not always possible, the patients immune system may reject the foreign cells, and the number of transplanted stem cells may not be enough to successfully treat the disease.

When blood stem cells are removed from the bone marrow and placed in laboratory dishes, they quickly lose their ability to self-renew, and they either die or differentiate into other blood cell types. Mikkolas goal, making blood stem cells self-renew in controlled laboratory conditions, would open up a host of new possibilities for treating many blood disorders among them safer genetic engineering of patients own blood stem cells. It could also enable scientists to produce blood stem cells from pluripotent stem cells, which have the potential to create any cell type in the body.

To uncover what makes blood stem cells self-renew in a lab, the researchers analyzed the genes that turn off as human blood stem cells lose their ability to self-renew, noting which genes turned off when blood stem cells differentiate into specific blood cells such as white or red cells. They then put the blood stem cells into laboratory dishes and observed which genes shut down. Using pluripotent stem cells, they made blood stem cell-like cells that lacked the ability to self-renew and monitored which genes were not activated.

They found that the expression of a gene called MLLT3 was closely correlated with blood stem cells potential to self-renew and that the protein generated by the MLLT3 gene provides blood stem cells with the instructions necessary to maintain its ability to self-renew. It does this by working with other regulatory proteins to keep important parts of the blood stem cells machinery operational as the cells divide.

The researchers wondered if maintaining the level of the MLLT3 protein in blood stem cells in lab dishes would be sufficient to improve their self-renewing abilities. Using a viral vector a specially modified virus that can carry genetic information to a cells nucleus without causing a disease the team inserted an active MLLT3 gene into blood stem cells and observed that functional blood stem cells were able to multiply in number at least twelvefold in lab dishes.

If we think about the amount of blood stem cells needed to treat a patient, thats a significant number, said Mikkola, who is also a professor of molecular, cell and developmental biology in the UCLA College and a member of theUCLA Jonsson Comprehensive Cancer Center. But were not just focusing on quantity; we also need to ensure that the lab-created blood stem cells can continue to function properly by making all blood cell types when transplanted.

Other recent studies have identified small molecules organic compounds that are often used to create pharmaceutical drugs that help to multiply human blood stem cells in the laboratory. When Mikkolas team used the small molecules, they observed that blood stem cell self-renewal improved in general, but the cells could not maintain proper MLLT3 levels, and they also did not function as well when transplanted into mice.

The previous discoveries with the small molecules are very important, and were building on them, said Vincenzo Calvanese, a UCLA project scientist and the studys co-corresponding author. Our method, which exposes blood stem cells to the small molecules and also inserts an active MLLT3 gene, created blood stem cells that integrated well into mouse bone marrow, efficiently produced all blood cell types and maintained their self-renewing ability."

Importantly, MLLT3 made the blood stem cells self-renew at a safe rate; they didnt acquire any dangerous characteristics such as multiplying too much or mutating and producing abnormal cells that could lead to leukemia.

The next steps for the researchers include determining what proteins and elements within blood stem cell DNA influence the on-off switch for MLLT3, and how this could be controlled using ingredients in the lab dishes. With that information, they could potentially find ways to switch MLLT3 on and off without the use of a viral vector, which would be safer for use in a clinical setting.

The research was supported by the National Institutes of Health, the Leukemia and Lymphoma Society, the California Institute for Regenerative Medicine, the American Association for Cancer Research, the Arnold and Mabel Beckman Foundation, the Jonsson Comprehensive Cancer Center Foundation, and the UCLA Broad Stem Cell Research Center, including support from The Rose Hills Foundation Innovator Grant, as well as the centers training program.

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Researchers identify protein that governs human blood stem cell self-renewal - Newswise

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Newswise CINCINNATI -- Stem cell therapy helps hearts recover from a heart attack, although not for the biological reasons originally proposed two decades ago that today are the basis of ongoing clinical trials. This is the conclusion of a Nov. 27 study in Nature that shows an entirely different way that heart stem cells help the injured heart not by replacing damaged or dead heart cells as initially proposed.

The study reports that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.

Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after heart attack, according to Jeffery Molkentin, PhD, principal investigator, director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI).

The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties, Molkentin said. The implications of our study are very straight forward and present important new evidence about an unsettled debate in the field of cardiovascular medicine.

The new paper builds on a 2014 study published by the same research team, also in Nature. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesnt work. The findings prompted Molkentin and his colleagues to conclude that there is a need to re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work.

An Unexpected Discovery

The study worked with two types of heart stem cells currently used in the clinical trialsbone marrow mononuclear cells and cardiac progenitor cells. As the researchers went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response

Researchers reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extra cellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself. The authors concluded: injected hearts produced a significantly greater change in passive force over increasing stretch, a profile that was more like uninjured hearts.

Molkentin and his colleagues also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury. This is in contrast to most past human clinical trials that for patient safety reasons simply injected stem cells into the circulatory system.

Most of the current trials were also incorrectly designed because they infuse cells into the vasculature, Molkentin explained. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic.

The researchers also noted an interesting finding involving zymosan, a chemical compound that binds with select pattern recognition receptors to cause an acute innate immune response. Using zymosan to treat injured hearts in mice resulted in a slightly greater and longer-lasting benefit on injured tissues than injecting stem cells or dead cell debris.

Looking to the Future

Molkentin said he and other collaborating scientists will follow up the findings by looking for ways to leverage the healing properties of the stem cells and compounds they tested.

For example, considering how heart stem cells, cell debris and zymosan all triggered an acute innate immune response involving macrophages in the current paper, Molkentin explained they will test a theory that harnesses the selective healing properties of macrophages. This includes polarizing or biologically queuing macrophages to only have healing-like properties.

Further testing of this, he said, could therapeutically be very important for developing future treatment strategies.

The studys first author was Ronald Vagnozzi, PhD, a fellow and investigator in the Molkentin laboratory. Key collaboration also came from scientists in the Cincinnati Childrens Heart Institute, the University of Cincinnati Department Of Pediatrics and the Center for Systems Biology (Department of Imaging) and the Cardiovascular Research Center at Massachusetts General Hospital and Harvard Medical School in Boston.

Funding support for the study came in part by grants from the National Institutes of Health (R01 HL132391) and an NIH Research Service Award via the National Heart Blood and Lung Institute (F32 HL128083), the Howard Hughes Medical Institute, and a Career Development Award from the American Heart Association (19CDA34670044). Flow cytometric data were acquired using equipment maintained by the Research Flow Cytometry Core in the Division of Rheumatology at Cincinnati Childrens.

Post Embargo Study Link: https://www.nature.com/articles/s41586-019-1802-2

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Stem Cell Therapy Helps Broken Hearts Heal in Unexpected Way - Newswise