REDWOOD CITY, Calif., Sept. 26, 2022 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that data from the companys investigator-sponsored study of JSP191 as a conditioning agent in the treatment of Fanconi Anemia were presented at the annual conference of the Inborn Errors Working Party (IEWP), a research group of the European Society of Blood and Marrow Transplantation, held on September 23-25, 2022, in Paris, France.

The study is a Phase 1/2 clinical trial (NCT04784052) utilizing JSP191 to treat Fanconi Anemia patients in bone marrow failure requiring allogeneic transplant with non-sibling donors. The objective of the study is to develop cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting TCR ab+ T-cell/CD19+ B-cell depleted stem cells from a donor, after using JSP191 as a part of conditioning. Primary outcome measures include the number of patients without treatment-emergent adverse events following the administration of JSP191.

In the data series presented, 100% complete donor chimerism was achieved through six months for the first patient and at one month for the second patient. Neutrophil engraftment was reached on day 11 for both patients and platelet engraftment was achieved on days 9 and 14. JSP191 was cleared by day 9 after dosing and no treatment-related adverse events or toxicities were observed.

Patients affected by Fanconi anemia have increased sensitivity to conventional conditioning regimens and radiation due to innate defects in DNA repair, said Ronald Martell, President, and CEO of Jasper Therapeutics. JSP191 offers a targeted conditioning strategy that eliminates the need for radiation or alkylating agents like busulfan. Initial data from this study suggest that a conditioning regimen that includes JSP191 has the potential to achieve successful donor transplant with no JSP191-related adverse events or toxicities reported to date. The positive update presented gives us increased confidence in JSP191, which has now shown promise as a conditioning agent in four indications including acute myeloid leukemia, myelodysplastic syndromes, severe combined immunodeficiency, and Fanconi anemia. We look forward to continuing support for Stanfords investigation of JSP191 and advancing our broader pipeline for JSP191 to the next phase of development.

The details of the oral presentation are as follows:

Title: JSP 191 clinical trial updateSession Name: Conditioning for HSCT in IEIPresenter: Rajni Agarwal-Hashmi, M.D., Professor of Pediatrics and Stem Cell Transplantation, the Stanford University School of MedicineDate/Time: Saturday, September 24, 2022, 2 pm CESTLocation: The Imagine Institute in Paris, France

About Fanconi AnemiaFanconi Anemia (FA) is a rare but serious blood disorder that prevents the bone marrow from making sufficient new red blood cells. The disorder can also cause the bone marrow to make abnormal blood cells. FA typically presents at birth or early in childhood between five and ten years of age. Ultimately it can lead to serious complications, including bone marrow failure and severe aplastic anemia. Cancers such as AML and MDS are other possible complications. Treatment may include blood transfusions or medicine to create more red blood cells, but a hematopoietic stem cell transplant (HSCT) is the only cure.

About JSP191

JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or genetically modified transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 100 healthy volunteers and patients. Four clinical trials for acute myeloid leukemia (AML)/ myelodysplastic syndromes (MDS), severe combined immunodeficiency (SCID), sickle cell disease (SCD) and Fanconi anemia are currently ongoing. The Company plans a new study of JSP191 as a second-line therapeutic in lower-risk MDS patients in 2022 as well as to a pivotal study in AML/MDS transplant in early 2023. Enrollment in additional studies are planned in patients with chronic granulomatous disease and GATA2 MDS who are undergoing hematopoietic cell transplantation as well as a study of JSP191 as a chronic therapeutic for low to intermediate risk MDS patients.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA Stem Cell Graft Platform which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases, and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the potential long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) and Jaspers ability to potentially deliver a targeted non-genotoxic conditioning agent to patients with SCID. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jaspers product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jaspers product candidates may not be beneficial to patients or successfully commercialized; patients willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jaspers business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jaspers business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jaspers filings with the SEC. If any of these risks materialize or Jaspers assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts:John Mullaly (investors)LifeSci Advisors617-429-3548jmullaly@lifesciadvisors.com

Jeet Mahal (investors)Jasper Therapeutics650-549-1403jmahal@jaspertherapeutics.com

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Jasper Therapeutics Announces Positive Clinical Data from Investigator Sponsored Study of JSP191 Conditioning in Fanconi Anemia Patients at IEWP...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Announces Positive Clinical Data from Investigator Sponsored Study of JSP191 Conditioning in Fanconi Anemia Patients at IEWP… | September 27th, 2022

The family of a Chilliwack man who was diagnosed with leukemia raised more than $10,000 during a golf fundraiser to help fellow patients battling the disease.

Dave Corke was diagnosed with leukemia just two days before Christmas 2020.

Earlier this year, the Corke family hosted a fundraiser golf tournament The Corke Classic to raise money for a B.C. hospital foundation.

He is now a year since his stem cell transplant and doing well, said wife Vanessa Corke. We wanted to help other families battling this cruel disease by organizing a fundraising golf tournament (with) all proceeds benefiting the VGH & UBC Hospital Foundation Leukemia/Bone Marrow Transplant Program of BC.

In April, friends and family came out to the Cheam Mountain Golf Course for the event and raised a total of $11,337.70.

Dave and Vanessa Corke hold a cheque which was presented to Chelsea Wallace, community partnerships officer at VGH Foundation. (Submitted by Vanessa Corke)

Vanessa said the money will significantly impact patients and their families.

She added that she felt it was an important time to share the news of the successful fundraiser as September is Leukemia Awareness Month, plus Daves birthday is Sept. 24.

They plan to make the golf fundraiser an annual event. Next years is set for April 22, 2023.

Back in 2021, the Corke family had financial help while Dave underwent treatment at Vancouver General Hospital.

READ MORE: Maple Ridge woman runs more kms each day to raise funds for Chilliwack friend with leukemia

Friends rallied to raise funds to help pay for hospital appointments, medication and to ease their financial stresses due to Dave being unable to work.

April Migneault was one of those friends who stepped up, doing a month-long fundraiser run. She started out on April 1, 2021 running one kilometre and gradually increased her distance by a kilometre every day, ending with a 30-kilometre run on April 30.

Earlier that same month another friend, Sharon Reeder, organized a golf fundraiser which brought in more than $10,000. And Debbie Channing set up a GoFundMe where more than $15,000 was raised.

Patients who undergo leukemia/bone marrow transplants have their diseased bone marrow destroyed by high-dose chemotherapy and are then transplanted by infusing healthy bone marrow stem cells. Through the course of their treatment, patients need to visit the hospital regularly until they can return back to their community.

Donations received through VGH & UBC Hospital Foundation support temporary relief to patients and families in need by providing them with travel vouchers, grocery cards, medication and accommodation support so they can take the time to heal without worrying about living essentials.

Do you have something else we should report on? Email: jenna.hauck@theprogress.comTwitter: @PhotoJennalism

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Dr. Zachary Prudowsky has never met the woman whose life he helped save by being a bone marrow donor, but he feels a special connection to her.

Prudowsky, a pediatric hematologist/oncologist, was preparing to move to Houston when the National Marrow Donor Program reached out to him in 2018. The nonprofit, which receives funding from Congress to operate its Be The Match registry of volunteer donors in the U.S., told Prudowsky he was a preferred donor for a woman with leukemia.

It wasnt until one year after his donation that he learned the woman lived in Katy and had her bone marrow transplant at M.D. Anderson Cancer Center.

MORE HOUSTON GIVES: After pandemic isolation, Ronald McDonald House volunteers eager to bring back human touch

Absolute serendipity, said Prudowsky, who is now 33. This saved a Texan, which is really, really cool.

She also sent him a note, saying his donation allowed her to meet her newborn grandchild. She referred to herself as granny, which tugged at Prudowskys heartstrings. That's what he calls his own grandmother.

That child gets to grow up with a grandmother because of Be The Match, he said. Thats where its really special for me.

Donate bone marrow: If you are between the ages of 18 and 40 and meet health guidelines, you can sign up to join the Be The Match Registry at bethematch.org. You can sign up online or find a local Be The Match Registry event.

Make a financial donation: You can also sign up to make one-time or monthly donations to the Be The Match Registry, or arrange your own fundraiser, at bethematch.org.

Prudowsky now serves as an advocate for Be The Match and the NMDP, which oversees a registry that includes more than 39 million potential donors. The NMDP helps facilitate more than 7,500 bone marrow transplants each year, Chief Policy Officer Brian Lindberg said.

Prudowsky and Lindberg are now advocating for Congress to pass H.R. 7770, or the Life-Saving Leave Act. The bill, introduced in May by Democratic Rep. Dean Phillips of Minnesota, would amend the Family Medical Leave Act of 1993 to provide up to 40 hours of unpaid, non-consecutive leave for bone marrow or blood stem cell donors.

The bill essentially seeks to guarantee that a donor wont lose his or her job while taking time off to help save a life, Lindberg said.

The thing that we cant do is promise that person that after theyre done with this process that their job will be waiting for them in the end, he said. Thats what this bill is intended to solve.

Prudowsky enrolled as a donor in 2011, during his first year of medical school in South Carolina. He knew he planned to treat cancer and blood disorders for a career, so he felt it made sense for him to sign up.

He didnt hear anything until 2018, near the end of his residency in Ohio. The NMDPreached out to inform him that he was the preferred donor for the Texas woman. Coincidentally, Prudowsky was preparing to move to Texas for a fellowship at Baylor College of Medicine.

He underwent several tests to confirm he was the best match. Less than three months after the call, he made his donation at the Gulf Coast Regional Blood Center in Houston.

Prudowsky donated via a process called peripheral blood stem cell collection (PBSC). Prior to the donation, donors get five days of injections of a drug called filgrastim to increase the number of blood-forming cells in their bloodstream. Those same blood-forming cells are found in bone marrow. The injections are given in outpatient clinics, and there are many instances where donors can return to work immediately afterward, Lindberg said.

On the day of the donation, blood was taken from Prudowskys arm and routed through a machine that collects those blood-forming cells. The rest of his blood was then returned to him through a needle in his other arm. The whole procedure took roughly four hours.

It was pretty uncomplicated, Prudowsky said. I kind of knew what I was getting myself into, but it was not a difficult process by any means.

Approximately 70 to 80 percent of all marrow donations occur via PBSC, Lindberg said.

The rest occur via traditional bone marrow donation, where a donor is put under anesthesia so a physician can collect cells from their hip. After that procedure, donors may experience soreness for the next few days, Lindberg said.

Weve heard it described many times as the kind of soreness that would be involved if you accidentally bumped into the corner of your dining room table.

The 40 hours outlined in the Life Saving Leave Act could be applied to recovery time, Lindberg said.

Prudowskys decision to be a donor while working as a doctor is not unique; both he and Lindberg said they know of others who work in medicine and are also signed up to be potential donors. But actually being matched can be tricky. Genetics has a lot to do with it: roughly 30 percent of patients have a family member who will be a fully-matched donor; the other 70 percent will need an anonymous donor from a registry.

Even then, the odds of finding a match vary greatly by ethnicity. While 79 percent of white patients will find an anonymous match, that drops to just under 50 percent for Asian and Hispanic patients, and to just 29 percent for Black patients, according to the NMDP.

The NMDPdoes help facilitate bone marrow transplants involving international donors and patients to increase the odds of finding a match, Lindberg said.

MORE HEALTH NEWS: A Katy man opted for surgery to treat prostate cancer, and a novel procedure limited side effects

However, its critical to keep recruiting a diverse pool of potential donors, Lindberg said. The greater the number, the greater the chance a patient will find a match.

Signing up to be a donor is straightforward, Prudowsky said. Anyone who is interested can sign up online on the Be The Match website. Theyll then submit a swab of the inside of their cheek, he said.

But Lindberg and Prudowsky also feel the Life Saving Leave Act could make the decision to be a donor even more straightforward. The NMDP can help donors by covering lost wages, as well as travel costs and other expenses during the process. But it cant guarantee theyll have a job if they take time off to donate.

There is simply no reason for anyone to be at risk of losing their job when it comes to potentially saving a life, Phillips said in a news release announcing the introduction of the bill.

Thirty-eight states already have laws that offer some level of paid or unpaid leave for bone marrow donors, but they vary widely, Lindberg said. Texas, for example, offers up to five days of leave without a reduction in salary, but the law only applies to state employees. The Life Saving Leave Act would create a uniform, federal standard, Lindberg said.

Lindberg is optimistic that the bill will become law. It has bipartisan support among seven cosponsors. Lindberg also said it has little to no economic impact because the leave is unpaid.

In the meantime, Prudowsky hopes more people will sign up for the Be The Match registry. He's seen his own patients learn they had a match for a bone marrow transplant, so he knows what a difference it can make.

"In my time in medicine, one of the most fulfilling things, if not the most fulfilling thing I've ever done, is this," he said. "And I didn't even have to be a doctor to do it."

evan.macdonald@chron.com

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--Two-Year Follow-Up Results Confirm Significant Efficacy and No Serious Adverse Effects in Patients who Underwent the StemSpine Procedure for Treating Chronic Lower Back Pain --

PHOENIX, Sept. 26, 2022 /PRNewswire/ -- Creative Medical Technology Holdings, Inc. ("Creative Medical Technology" or the "Company") (NASDAQ: CELZ), a leading biotechnology company focused on a regenerative approach to immunotherapy, endocrinology, urology, neurology, and orthopedics, today announced the peer reviewed publication of positive, two-year follow-up data for the Company's StemSpine study, showing significant efficacy of the StemSpine procedure for treating chronic lower back pain without any serious adverse effects reported.

Creative Medical Technology Holdings, Inc. Logo (PRNewsfoto/Creative Medical Technology Hol)

The StemSpine publication demonstrates the clinical use of the patented procedure that utilizes a patient's own bone marrow aspirate for the treatment of chronic lower back pain. There were no safety related concerns at up to two years follow-up. The StemSpine procedure resulted in an efficacy rate of 87% in the treated patients in terms of decreased pain and increased mobility.

"The positive two-year data from our StemSpine study is very encouraging and may help alleviate the current opioid crisis related to prescription medication abuse for chronic lower back pain," said Timothy Warbington, President and CEO of the Company. "To our knowledge, this is the first demonstration of the clinical efficacy of injecting bone marrow aspirate into areas surrounding the disc, which may repair, remodel and improve the blood supply around the disc and lower back area. We believe StemSpine represents an attractive non-surgical option for many of the millions of Americans who suffer from chronic lower back pain and look forward to further translation."

The StemSpine patent also covers the use of off the shelf adult donor stem cells (allogeneic) for this indication.

The publication may be found at http://www.creativemedicaltechnology.com.

Story continues

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a biotechnology company specializing in regenerative medicine in the fields of immunotherapy, endocrinology, urology, neurology, and orthopedics. For further information about the Company, please visit http://www.creativemedicaltechnology.com.

Forward Looking Statements

This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website at http://www.sec.gov.

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Predicting the risk of acute kidney injury after hematopoietic stem cell transplantation: development of a new predictive nomogram | Scientific...

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ThisBone Marrow MarketReport provides details on Recent New Developments, Trade Regulations, Import-Export Analysis, Production Analysis, Value Chain Optimization, Market Share, Impact of Domestic and Localized Market Players, Analyzes opportunities in terms of emerging revenue pockets, changing market regulations, strategic market growth analysis, market size, market category growth, niche and application dominance, product endorsements, product launches, geographic expansions , technological innovations in the market.For more information on the bone marrow market, please contact Data Bridge Market Research for a summary of theanalyst, our team will help you make an informed market decision to achieve market growth.

Bone Marrow Market is expected to experience market growth during the forecast period of 2021 to 2028. Data Bridge Market Research analyzes that the market is growing with a CAGR of 5.22% during the forecast period of 2021 to 2028 and it is projected to reach USD 13,899.60 Million by 2028. The increasing number of bone marrow diseases will help accelerate the growth of the bone marrow market.Bone marrow transplant also called hematopoietic stem cell.It is a soft vascular tissue present inside the long bones.It includes two types of stem cells, namely hematopoietic and mesenchymal stem cells.The bone marrow is primarily responsible for hematopoiesis (blood cell formation), lymphocyte production, and fat storage.

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The main factors driving the growth of the bone marrow market during the forecast period are the growth in the incidence of non-Hodgkins and Hodgkins lymphoma, thalassemia, and leukemia, as well as common bone marrow diseases worldwide, developments in technology and improvements.in health infrastructure.In addition, advanced signs of bone marrow transplantation for cardiac and neural disorders, increased funding for logistics services, and rising health care spending per capita are some of the other factors expected to further drive growth. growth of the bone marrow market in the coming years.years.However, the high costs of treatment,

Key Players Covered in the Bone Marrow Market Report are AGendia, Agilent Technologies, Inc., Ambrilia Biopharma Inc., Astellas Pharma Inc., diaDexus, Illumina, Inc., QIAGEN, F Hoffmann-La Roche Ltd, Sanofi, Stryker Corporation, PromoCell GmbH, STEMCELL Technologies Inc., Lonza, ReachBio LLC, AllCells, ATCC, Lifeline Cell Technology, Conversant bio, HemaCare, Mesoblast Ltd., Merck KGaA, Discovery Life Sciences, ReeLabs Pvt. Ltd., Gamida Cell, among others national and global players.Market share data is available separately for Global, North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA), and South America.DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

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Bone MarrowMarket Scope and Market Size

The bone marrow market is segmented based on transplant type, disease indication, and end user.Growth between these segments will help you analyze weak growth segments in industries and provide users with valuable market overview and market insights to help them make strategic decisions to identify leading market applications.

Country-level analysis of thebone marrow market

The bone marrow market is analyzed and information is provided on market size and trends by country, transplant type, disease indication, and end user, as mentioned above.Countries Covered in Bone Marrow Market Report are USA, Canada, and Mexico, North America, Germany, France, UK, Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, the Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific region (APAC), Saudi Arabia, United Arab Emirates , South Africa, Egypt, Israel, Rest of the Middle East and Africa (MEA) under Middle East and Africa (MEA), Brazil,

Europe dominates the bone marrow market due to the proliferation of innovative health centers.Furthermore, the health systems have introduced bone marrow transplantation in their contributions and state-of-the-art public facilities that will further drive the growth of the bone marrow market in the region during the forecast period.North America is expected to witness significant growth in the bone marrow market due to increasing cases of chronic diseases such as blood cancer.In addition, the increase in the geriatric population is one of the factors that is expected to drive the growth of the bone marrow market in the region in the coming years.

Explore Full TOC At:- https://www.databridgemarketresearch.com/toc/?dbmr=global-bone-marrow-market

The country section of the Bone Marrow market report also provides individual market impact factors and regulatory changes in the country market that affect current and future market trends.Data points such as consumption volumes, production sites and volumes, import and export analysis, price trend analysis, raw material cost, Downstream and Upstream value chain analysis are some of the main indicators used to forecast the scenario. of the market for each country.Additionally, the presence and availability of global brands and the challenges they face due to significant or rare competition from local and national brands,

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Bone Marrow market estimated to reach US$13899.60 Million during the forecast period - Digital Journal

Bone Marrow Stem Cells Comments Off on Bone Marrow market estimated to reach US$13899.60 Million during the forecast period – Digital Journal | September 19th, 2022

Our work on an enzyme that is mutated in leukemia patients has led to the discovery of an entirely new way of regulating this enzyme, as well as new molecules that are more effective and less toxic to human cells, said Norbert Reich, a distinguished professor at the University of California, Santa Barbara, and the corresponding author of the study.

A cells epigenome is copied and maintained by an enzyme called DNMT1. For instance, this enzyme ensures that a dividing liver cell produces two liver cells rather than a brain cell.

However, some cells need to undergo differentiation to become new types of cells. For instance, bone marrow stem cells can developall the various blood cell types, which are incapable of self-replication. DNMT3A, another enzyme, manages this.

This is not a problem until a dysfunction of DNMT3A results in the production of abnormal blood cells from bone marrow. This is a prominent factor in the development of several types of leukemia as well as other cancers.

Most cancer medications are intended to attack cancer cells while only leaving healthy cells. But this is quite a challenging process; therefore, most have severe side effects.

Current leukemia medications, such as Decitabine, bind to DNMT3A in a way that disables it. So that they slow the progression of the disease by obstructing the enzyme's active site, preventing it from continuing its function.

Unfortunately, the active site of DNMT3A is virtually identical to that of DNMT1, therefore, the medication blocks epigenetic regulation in patients' 30 to 40 trillion cells. This leads to off-target toxicity- one of the drug industry's largest bottlenecks.

Link:
Researchers discover a new class of medications that offer a safer treatment for leukemia - Interesting Engineering

Bone Marrow Stem Cells Comments Off on Researchers discover a new class of medications that offer a safer treatment for leukemia – Interesting Engineering | September 19th, 2022

Louise Herbert flew more than 8,000 miles in January for a risky procedure to slow her MS and says its the best thing shes ever done.

The 41-year-old mum, originally from Shetland, had been a keen runner and netball player before she was diagnosed with the condition at 26.

It caused her immune system to attack itself, at times leaving her so exhausted she could barely spend time with her nine-year-old daughter.

At the start of the year Louisetravelled from her home in Newmachar, Aberdeenshire to Puebla, Mexico for 47,000 haematopoietic stem cell transplantation (HSCT).

Some patients find it does more harm than good causing hair loss, nausea and even infertility and around 3% die from the treatment.

But Louise felt it was a risk worth taking.

During the treatment, doctors took stem cells from Louises bone marrow.

Then used intense chemotherapy to wipe out her immune system, before reintroducing the cells to try and reset her body.

Although Louise did suffer excruciating back pain during her treatment, shes relieved she dodged many of the other side effects.

To be honest, I think I was really lucky because the chemotherapy never made me feel sick, I never felt nauseous or lost my appetite, she explained.

The only thing that really bothered me was these injections we got every morning and every night for a week.

I got horrendous back pain one night, it was so bad I pressed the SOS button on the phones we had to speak to a doctor, she recalled.

Though the back pain was an unpleasant experience, from a medical point of view it was actually a positive.

She said: It was showing there was plenty of stem cells there and they were ready to come out, I think they came out two days later.

One effect of the chemo was that Louises hair fell out but its started to grow back with wild and frizzy curls.

I dont think Ill grow it much beyond my chin, she added.

Though its still too early to be certain, Louise believes there have already been some encouraging signs.

She said: Its still early days, they say its 12 to 18 months before any improvements are seen. Im at seven-and-a-half.

I dont think I can walk any further than before, but I have a bit more confidence in my walking.

I went on the same walk seven times last week, I used to come home and the first thing I wanted to do was sit down in the chair, I wasnt like that last week.

Tasks such as putting on trousers also seems to be less strenuous for Louise than before.

She explained: It was a case I had to lift my left leg up because it couldnt lift itself, its been like that for about five years.

But for the past week Ive noticed I could lift it myself, Ive been going to Pilates which should help improve my balance.

I dont know if its that or the HSCT, but somethings done it.

Im not saying I can lift my leg every day, but if I can do it even three out of seven, thats great.

Upon reflection, Newmachar mum Louise is happy she made the decision to travel to Mexico for this MS treatment.

Im so glad I did it, its the best thing I ever did, youve got to try, she added.

Aberdeen woman with MS to spend 46,000 for stem cell treatment

Aberdeenshire mum says 8,000 mile Mexico trip is only option for her incurable condition

I wanted to see if I still could: MS patient raises charity cash with gruelling challenge

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'Best thing I ever did': Newmachar mum showing promising signs after 47000 MS treatment in Mexico - The Press & Journal

Bone Marrow Stem Cells Comments Off on ‘Best thing I ever did’: Newmachar mum showing promising signs after 47000 MS treatment in Mexico – The Press & Journal | September 19th, 2022

Five people with the autoimmune condition lupus are now in remission after receiving a version of CAR-T therapy, which was originally developed for cancer

By Clare Wilson

Illustration of a CAR-T cell

CHRISTOPH BURGSTEDT/SCIENCE PHOTO LIBRARY

A high-tech cell therapy used to treat cancer has been repurposed as a treatment for lupus, an autoimmune condition that can cause joint, kidney and heart damage.

CAR T-cell therapy has put all five people with lupus treated so far into remission. The participants have been followed up for an average of 8 months, with the first person treated 17 months ago. Thats kind of unheard of, says Chris Wincup at Kings College London, who wasnt involved in the study. This is incredibly exciting.

But it is too soon to know how long the remissions will last, says Georg Schett at the University of Erlangen-Nuremberg in Germany, who was part of the study team.

CAR T-cells were developed to treat blood cancers that arise when B cells, a type of immune cell that normally makes antibodies, start multiplying out of control.

The approach requires taking a sample of immune cells from a persons blood, genetically altering them in the lab so they attack B cells and then infusing them back into the individuals blood. It seems to put 4 out of 10 people with these kinds of cancers into remission.

Lupus, also called systemic lupus erythematosus, is caused by the immune system mistakenly reacting against peoples own DNA. This is driven by B cells making antibodies against DNA released from dying cells.

It is currently treated with medicines that suppress the immune system or, in more severe cases, with drugs that kill B cells. But the treatments cant kill all the B cells, and if the disease flares up badly, some people develop kidney failure and inflammation of their heart and brain.

Schett and his team wondered whether using CAR T-cells to hunt down all the B cells would be more effective. Within three months of receiving the treatment, all five participants were in remission, without needing to take any other medicines to control their symptoms.

The CAR T-cells were barely detectable after one month, and after three and a half months, the volunteers B cells started to return, having been produced by stem cells in bone marrow. These new B cells didnt react against the DNA.

We dont know what normally causes B cells to start reacting against DNA in people with lupus, so it is possible that some kind of trigger may start the process happening again, says Wincup.

The achievement means CAR T-cells may also be useful against other autoimmune diseases that are driven by antibodies, such as multiple sclerosis (MS), in which the immune system attacks nerves, says Schett.

Another radical treatment for MS involves rebooting the immune system by destroying it with chemotherapy. By comparison, CAR T-cells would be less invasive and more tolerable, he says.

But it is too soon to know how effective CAR T-cells will be for autoimmune conditions, says Wincup. This is a small number of patients, so we dont know if this is going to be the result for everyone.

When used in cancer, CAR T-cells are expensive to create for each person, so they may only be used for autoimmune conditions in people with severe disease when no other treatments are available, he says.

Journal reference: Nature Medicine , DOI: 10.1038/s41591-022-02017-5

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Radical lupus treatment uses CAR T-cell therapy developed for cancer - New Scientist

Bone Marrow Stem Cells Comments Off on Radical lupus treatment uses CAR T-cell therapy developed for cancer – New Scientist | September 19th, 2022

Introduction:Aplastic anemia (AA)is characterized by pancytopenia and hypocellular marrow in the absence of an abnormal infiltrate or increase in reticulin fibrosis. The diagnosis of AA is challenging at times due to decreased cellularity and overlapping morphological features with other bone marrow failure syndromes. Hepatitis-associated aplastic anemia (HAAA) is a rare variant in which patients typically present with jaundice and hepatitis followed by pancytopenia almost within 6 months. Post-hepatitis AA accounts for approximately 1-5%of cases, and invariably such cases are negative for the known hepatitis virus as well. There is limited literature available to understand the correlation of AA with hepatitis with none reported at the national level in our region. As AA is relatively more prevalent in Southeast Asia as compared to the western world and hepatitis is a prevalent disease in our population, the main purpose of this study was to assess the hepatic profile and determine the association of hepatitis in AA at the time of diagnosis.

Materials and methods:A cross-sectional study was carried out at the National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, from November 2019 to December 2020 after the informed consent from patients. The study included all treatment-nave patients of acquired AA with no prior history of taking steroids, immunosuppressive treatment, or chemoradiation therapy. Liver function tests, complete blood count, prothrombin time (PT), and activated partial thromboplastin time were performed, along with viral profiles (HAV, Hep B, Hep C, and HIV). SPSS version 23 (IBM Corp., Armonk, NY) was used for statistical analysis. Mean and standard deviations were computed for quantitative variables while percentages and frequencies were reported for qualitative variables. T-test was used to observe the main difference between groups and a p-value <0.05 was considered to be significant.

Results:Out of a total of 351 patients, 29 (8.2%) patients with AA tested positive for viral hepatitis. Hepatitis A was the most prevalent hepatitis (4.0%), followed by hepatitis C (3.7%). The comparison of platelet counts in patients with and without hepatitis was reported to be of statistical significance (p-value < 0.05). A significant statistical difference (p-value< 0.0001) was found in platelet count and PTin patients of AA with and without hepatitis.

Conclusion:Overall, this study revealed that <10% of patients of AA had a positive screening for hepatitis A, B, and C and low platelet count, and PT was statistically significant when compared between the patients with and without hepatitis. Hepatitis being prevalent in our part of the world might have an important causal association with AA. Patients with AA should be screened for liver functions and viral hepatitis at the time of diagnosis. In addition to hepatitis A, B, and C and HIV, other causes of hepatitis should also be screened such as parvovirus B19, human herpes virus 16, and adenovirus which are not included in routine diagnostic viral testing panel.

The distinctive manifestation(s) of aplastic anemia (AA) are pancytopenia and hypocellular bone marrow without evidence of infiltration, dysplasia, and fibrosis. It is caused by several risk factors including infections, toxins, chemotherapeutic drugs, and radiation but the precise cause remains unclear [1]. An uncommon form of AA known as hepatitis-associated aplastic anemia (HAAA) occurs when pancytopenia develops simultaneously with or within six months after an elevated serum alanine aminotransferase (ALT) level. Significantly, these ALT levels are five times higher than the upper limit of normal. Post-hepatitic AA accounts for approximately 1-5% of cases invariably, and such cases are negative for the known hepatitis virus as well [2].When compared to patients with non-hepatitis-associated AA in the pediatric population, those with HAAA have considerably lower survival and prognosis [3]. Hepatitis symptoms linked to HAAA can be self-limiting, but sometimes showmoderate to severe or acute to the chronic clinical course[4]. Necrosis of the portal region and fibrosis that extends up to the centrilobular area can be found in the transjugular liver biopsy of HAAA patients and chronic severe hepatic inflammation quickly progresses to liver fibrosis [5]. First-line therapies include stem cell transplantation from a sibling donor who matches the patient's human leukocyte antigen (HLA) profile or immunosuppressive therapies like cyclosporine and/or antithymocyte or antilymphocyte globulin [6,7].

The exact etiology of hepatitis causing AA is nearly unknown and the pathogenesis of HAAA has been associated with activated T1 cells [8]. Hepatitis B and C virus, parvovirus B19, human herpes virus 16, and transfusion-transmitted virus (hepatitis B, C, HIV) can all be causal associations. The traits include CD8+-predominant lobular necroinflammatory and endothelial damage associated with sinusoidal obstruction syndrome, conjugated hyperbilirubinemia, elevated antinuclear antibody titers, and elevated transaminases [9]. Dietary or nutrition supplements at times may also result in toxin-induced hepatitis [10].It is typical for intrabiliary cholestasis and liver toxicity to result in the apoptotic killing of hematopoietic cells by CD8 lymphocytes and T cell-induced gamma interferon, which leads to hepatitis. Clinical symptoms include pallor, exhaustion, petechial rash, and infections due to pancytopenia [11]. When severe thrombocytopenia and anemia appear at the same time as HAAA, it is important to distinguish this condition from infantile giant cell hepatitis with autoimmune hemolytic anemia [12].

Allogeneic bone marrow transplantation (allo BMT) is the conventional curative treatment for HAAA from an HLA-matched donor. Since HAAA has a poor prognosis, allo BMT has been the curative form of treatment [13-15]. On the other hand, immunosuppressive medication results in a response rate of 70% and a survival rate of 85% for patients not receiving hematopoietic cell transplantation, respectively [16]. Children respond to BMT better than adults do, and the survival rates of patients receiving bone marrow from HLA-matched donors are found to be comparable to that of the patients with AA not caused by hepatitis [17]. There is no recognized antiviral medication for hepatitis B-related HAAA. Lamivudine, a nucleoside analog, has been studied for use in treating hepatitis B-related HAAA, and it has been shown to be effective in causing remission in cases of severe AA and hepatitis B virus infection. Because of its myelosuppressive effects, interferon, an acclaimed antiviral agent in the treatment arsenal of HBV- and HCV-mediated infections, cannot be used as a treatment option for the HAAA [18]. Patients with HAAA exhibit severe pancytopenia following an episode of acute hepatitis, and if left untreated, the marrow suppression is frequently swift and severe.

In Pakistan, cases of hepatitis have been increasing over time, and given the current policies and practices, eradicating hepatitis from Pakistan by 2030 seems unreasonable[19]. We have also noted a rising trend in the incidence of AA than reported internationally. The association of hepatitis with AA is underreported and limited literature is available regarding the determinants of AA. Hepatitis might be one of the causes of AA and it must be considered an alarming situation.The only curative option for AA is BMT, which is an unaffordable treatment option for many patients in Pakistan. Therefore, the aim to conduct this study was to evaluate the hepatic status in relation to hepatitis in treatment-nave AA patients.

This was a cross-sectional study carried out at the National Institute of Blood Diseases and Bone Marrow Transplantation (NIBD & BMT). Patients were recruited between November 2019 and December 2021. Prior to the initiation of the study, approval was taken from the NIBD ethics committee bearing NIBD/RD-188/11-2019 and informed consent was taken from the participants prior to enrollment in the study. The study included all treatment-nave patients of acquired AA with no prior history of taking steroids, immunosuppressive treatment, or chemoradiation therapy. Patients with positive chromosomal breaks (Fanconis anemia) or inherited bone marrow failure were excluded. WHO sample size calculator was used to calculate the sample size, and by taking the percentage reported in the literature (5%) [14] it was found to be at least 73, and hence we recruited 351 patients who visited during the study duration.Non-probability purposive sampling method was used for patient selection. Demographicand laboratory parameters were recorded through a structured questionnaire and the diagnosis of AA was confirmed on bone marrow biopsy. Liver function tests (LFTs), complete blood count (CBC), prothrombin time (PT), and activated partial thromboplastin time (APTT) were performed. PT and APTT were run on STAGO and Sysmex CA-1500, and CBC was performed using a Sysmex XN-1000 analyzer from the Sysmex Corporation in Kobe, Japan. LFTs were performed on a Cobas C 111 analyzer machine that runs on a spectrophotometer principle.Within 2 hours of the blood sample collection, an aseptic setting was used to take a 4 ml blood sample from each patient, which was then tested for the presence of hepatitis A, HBV, HCV, and HIV. Detection of hepatitis B surface antigen and anti-hepatitis C antibodies was done using the chemiluminescence technique. To confirm chronic HCV and HBV infection in individuals with positive antibody tests, a qualitative nucleic acid test was employed as the initial diagnosis of a suspected acute infection. Ultrasound was done to complement the diagnosis of hepatitis. The data were entered in MS Excel and analysis was done by using Statistical Package for Social Sciences (SPSS) version 23.0 (IBM Corp., Armonk, NY). Shapiro-Wilk test was performed for normality and data were found normally distributed. Mean and standard deviations were computed for quantitative variables and percentages and frequencies were reported for qualitative variables. T-test was used to observe the mean difference between the two groups and a p-value <0.05 was found to be significant statistically.

A total of 351 patients were enrolled, out of which 222 (63%) were males, 265 (76%) were unmarried, 11 (3.1%)had hypertension, and 9 (2.5%) had diabetes. The mean age of participants was 30.9 27.5. Twenty-nine (8.2%) patients with AA had hepatitis. The most common hepatitis was hepatitis A, 14 (4.0%), followed by hepatitis C, 13 (3.7%). The most common form of hepatitis C was acute 9 (69.2%). Two patients had compensated cirrhosis and two patients had decompensated cirrhosis (Table 1).

The association of CBC parameters such as hemoglobin, red blood cell count, packed cell volume, mean corpuscular volume, mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, total leukocyte count, neutrophils, lymphocytes, monocytes, and platelets with the patients with and without hepatitis was assessed and it was found out that platelet counts were statistically significant (p-value: 0.0020) as shown in Table 2.

Table 3 depicts the association of liver profile such as serum glutamate pyruvate transaminase, gamma glutamyl transferase (GGT), serum glutamic oxaloacetic acid transaminase, PT, APTT, and international normalized ratio with the patients with and without hepatitis, and it was found out that PT (p-value< 0.0001) was statistically significant.

In our study, 8.2% of AA patients had hepatitis. This is comparatively high as compared to theprevalence of hepatitis in the general population in Pakistan, which is reported to be around 4% [20]. A retrospective study carried out in Europe from 1997 to 2007 demonstrated that HAAA patients had a slight male predominance with a value of 68% [21], which is close to the prevalence of male participants in our study, i.e. 63.2%. This European study also demonstrated that there was no causative virus for hepatitis in 94% of HAAA patients. However, 15 patients (6%) had hepatitis with nine having hepatitis B virus and six having hepatitis A virus [21]. Similar results have been observed in our study with 91.4% HAAA serologically negative and not having any signs or symptoms of hepatitis. Twenty-nine patients (8.3%) had evidence of hepatitis: hepatitis A virus in 14 patients, hepatitis B virus in 2, and hepatitis C virus in 13 patients. Assessing the association of hepatitis C virus with HAAA, a study conducted in France showed that 15.8% of HAAA patients had hepatitis C virus [22], which was approximately three-fold higher compared to our study suggesting a prevalence of 3.7% of HAAA patients with hepatitis C virus. On the contrary, slightly similar figures were recreated in another comparative study done in Thailand which showed that 5.7% of the patients who were never transfusedwere found to have hepatitis C virus [23].

During the initial course of hepatitis, cytotoxic T lymphocytes (CTLs) occupy the same receptor antigen between liver and bone marrow cells. These CTLs replicate and extend to destroy bone marrow hematopoietic stem cells and result in AA [24,25].However, another interesting finding of our study was that AA patients with hepatitis had considerably high platelet counts compared to those without hepatitis (29 28 vs 4 3). This finding is also in concordance with a study conducted by Wang WH et al. [3] in which the HAAA group had a considerably higher platelet count compared to the non-hepatitis-associated AA (50 109/L vs 12 109/L).

Literature reveals that amongthe most frequently assessed values in HAAA, there is a significant rise in ALT, GGT, and serum alkaline phosphatase [26]. The elevated levels are also shown in our results (Table 3). Considerable discrepancies were observed in PT in AA patients with and without hepatitis. This could be on account of viral hepatitis causing deranged LFTs [27].However, studies comparing the levels of liver enzymes between hepatitis-associated and non-hepatitis-associated AA are not extensively reported in the literature.

To the best of our knowledge, this is the first study at the national level to determine the association of hepatitis in patients with AA. However, the limitations of our study were that it was of cross-sectional nature and included non-probability sampling. Prospective cohort studies with stringent inclusion and exclusion criteria, a large sample size, and a controlled environment are needed to validate the findings.

AA is a heterogeneous disease with a limited approach to curative treatment options like allogeneic stem cell transplant in our region. Hepatitis being prevalent in our part of the world might have an important causal association with AA. Patients should be screened for viral hepatitis at the time of diagnosis. Moreover, other causes of hepatitis should also be screened at the time of diagnosis such as parvovirus B19, human herpes virus 16, and adenovirus. Based on the rapidly advancing research methodologies, it is necessary to comprehensively analyze the underlying mechanisms of HAAA.

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Assessment of Hepatic Profile in Acquired Aplastic Anemia: An Experience From Pakistan - Cureus

Bone Marrow Stem Cells Comments Off on Assessment of Hepatic Profile in Acquired Aplastic Anemia: An Experience From Pakistan – Cureus | September 19th, 2022

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Epidemiology of early infections and predictors of mortality after autologous hematopoietic stem-cell transplantation among multiple myeloma, Hodgkin,...

Bone Marrow Stem Cells Comments Off on Epidemiology of early infections and predictors of mortality after autologous hematopoietic stem-cell transplantation among multiple myeloma, Hodgkin,… | September 11th, 2022

September is observed as Blood Cancer Awareness month all over the world. During this month, activists and stakeholders work to raise awareness about the disease and the efforts being taken to fight blood cancers including leukemia, lymphoma, myeloma and Hodgkins disease.

The term blood cancer is a general description of various hematopoietic cancers. Our blood flows through blood vessels to supply all tissues in the body with nutrients. In the approximately 5 litres of blood circulating in our body there are billions of blood cells that carry out various vital functions. All blood cells originate from hematopoietic stem cells.

Haematopoietic stem cells are known as mother cells and do not yet have a specific function. They are able to renew and differentiate into cells with a specific function, thus replacing cells that die. In bone marrow, blood stem cells divide and develop into progenitor cells. Through further division, the progenitor cells mature and transform into different types of blood cells and then enter the bloodstream, says Dr Nitin Agarwal, HOD, Donor Request Management, DKMS BMST Foundation India.

Blood cancer is an abnormal proliferation (abnormal growth) of cells in the bone marrow especially white blood cells (WBCs). Cancer cells flood the bloodstream and drive out healthy cells. As a result, the blood can no longer perform its basic tasks, such as transporting oxygen and protecting the body from infection.

LeukemiaThis cancer is found in the bone marrow and the bloodstream. It is caused by abnormal rapid production of WBCs and high number of abnormal WBCs which cannot fight against infection, and they impair the bone marrows ability to produce red blood cells and platelets, says Dr Jimmy Mirani, Consultant Onco Surgeon, Wockhardt Hospital, Mumbai Central.

LymphomaA type of blood cancer which affects the lymphatic system, which removes the risk excess fluids from body and generates immune cells. Lymphocytes are blood cells which are used to fight against infections. These abnormal lymphocytes become lymphoma cells which multiply and get collected in the tissues, adds Dr Mirani.

There are two types of lymphoma, namely, Hodgkins lymphoma and non-Hodgkins lymphoma.

Non-Hodgkins lymphoma:It mainly impacts the B-cell or T-cell. This type of lymphoma occurs more commonly than Hodgkins lymphoma. Can vary clinically and diagnostically into slow-growing ones to very aggressive types, notes Dr. Amrita Chakrabarti, Consultant, Haemato-Oncology & Bone Marrow Transplant, Max Hospital, Shalimar Bagh.

Hodgkins lymphoma This type of lymphoma affects the B cells. Broadly divided into classical Hodgkins and nodular lymphocyte predominant types. Occurs in the adolescence or elderly age group.

MyelomaIt is the cancer of plasma cells; WBCs which produce disease and infection fighting anti-bodies. Myeloma cells prevent the functions and productions of these antibodies leaving a week immune system.

Multiple myelomaThis starts in the bone marrow when plasma cells begin to grow uncontrollably. As the cells grow, they compromise the immune system and impair the production and function of white and red blood cells causing bone disease, organ damage and anemia among other conditions, adds Dr Agarwal.

In most cases of blood cancer, the patient feels tired and weak. This happens because the number of red blood cells in the blood starts decreasing due to which there is a lack of blood in the person. Someof the commonsymptoms of blood cancers are fever, severe fatigue, bleeding from gums or skin, back ache, or bone pains, says Dr Pravas Mishra, Head Haematology/ Medical Oncology and BMT, Amrita Hospital, Faridabad.

Patients with myeloma might first present to an orthopaedical with a fracture originating from trivial trauma or to a nephrologist with a kidney dysfunction.Pain in bones and joints can be a symptom of not only arthritis but also blood cancer. Blood cancer is a disease in the bone marrow that is found in large amounts around the bones and joints.

Patientsmight present with nodes in the neck or axilla or groin or swelling in any part of the body. However most often a patientwith blood cancermight present with just a low haemoglobin. It is strongly advised not to ignore any anaemia, warns Dr Mishra.

A person suffering from blood cancer is prone to repeated infections. When leukemia cells develop in the body, then complaints of infection can be seen in the patients mouth, throat, skin, lungs, etc.

People who have cancer tend to have an abnormally low weight. If the body weight is reduced without any obvious cause, then it can be seen as the primary symptom of cancer.

The abnormal formation of leukemia cells in the body prevents the bone marrow from forming healthy blood cells such as platelets. Due to its deficiency, more bleeding problems can be seen from the nose of the patient, during menstruation and gums.

Blood cancer is diagnosed with the help of a wide range of diagnostic methods along clinical evaluation, such as blood tests, bone marrow tests, cytogenetic/karyotyping and molecular analysis, flow cytometry.

Myth: Blood cancer cannot be treated?

Fact: Once a patient is diagnosed with blood cancer, the first concern that comes to ones mind Is blood cancer curable?

Blood cancer is one type of cancer that has a high curability rate especially due to the advancement in the medical field, availability of newer, improved chemotherapy regimens, targeted therapy, and improved infection control measures. Timely diagnosis, especially early diagnosis, increases the chances of cure from blood cancer.Some of the other factors that impact the cure of blood cancer include the age of the patient, physical condition, presence of other comorbidities, stage of the disease, subtype of cancer, molecular factors, whether low grade/high grade, acute or chronic, the body parts that are affected and whether the disease is new onset or has come back after a previous cure.

You must understand that the cure or recovery from cancer is unpredictable, adds Dr. Chakrabarti.

There are cases when the patient has recovered even in the later stages of blood cancer. On the other hand, there are recorded cases where the patient couldnt recover even in the initial stages of blood cancer. So, its important to have realistic expectations and focus on following a healthy lifestyle with the advised treatment and measures. Early diagnosis and treatment play an important role in attaining cure.

Myth: All blood cancer patients need a bone marrow transplant

Fact: No, majority of patients suffering from blood cancers are treated without bone marrow transplant. A combination of chemotherapy, targeted therapy and immunotherapy is the best line of treatment.

Myth: Blood cancer occurs only in children?

Fact: No, blood cancers can occur in all age groups. All have a higher incidence in young children whereas Myeloid Leukaemia (MLL) is more frequently seen in senior citizens.

India is reeling under pressure of many misconceptions that exist amongst people about blood stem cell donation, its process and even its after-effects.

Myth: Once you donate blood stem cells, you will lose them forever.

Fact: Only a fraction of total stem cells is extracted during the process. Also, all the cells are naturally replenished within a few weeks

Myth: Donating stem cells is a really invasive and painful process

Fact: Blood stem cells are collected through peripheral blood stem cell collection (PBSC) which is completely safe and a non-surgical procedure. The process is similar to blood platelet donation that takes approximately three to four hours to complete and the donor can leave the collection center the same day.

Myth: Blood donation and a blood stem cell donation are same

Fact: Unlike blood collection for transfusion, blood stem cells are collected only when there is a match between the donor and patients human leukocyte antigen (HLA) combination (tissue type). So, you could be potentially the only match and life saver for a person with blood cancer in need of a transplant, adds Dr Nitin Agarwal. Blood stem cell donors donate only blood stem cells and the process is similar to a platelet donation.

Myth: Pregnant women cant register

Fact: This is untrue, a woman can register even during her pregnancy.

Myth: Stem cell donation leaves prolonged side-effects

Fact: No, there are no major side effects post blood stem cell donation. A person may only experience minor flu like symptoms because of the GCSF injections given to him/her before the donation, to mobilize blood stem cells in your blood stream.

Myth: Piercing and/or tattoo is a restricting factor

Fact: Piercing or a tattoo doesnt stop you from registering yourself to be a potential donor.

Myth: My blood stem cells can be stored

Fact: Your blood stem cells will not be stored. They last for around 72 hours and are delivered for the recipient straight to the hospital by a special courier. If the recipients body accepts them, the stem cells will start making healthy blood cells.

Myth: Joining a blood stem cell registry is no use. Most patients can find a stem cell donor within their own families

Fact: Per statistics, only 30% of blood disorder patients in need of a stem cell transplant are able to find a sibling match. About 70% of patients need an unrelated donor.

A registry like DKMS BMST Foundation India is a data bank of potential blood stem cell donors that houses details on thousands of committed blood stem cell donors. Any patient can benefit from this registry provided an HLA match.

Some of the blood cancer treatments include the following

Chemotherapy

This is the most important aspect of blood cancer treatment and involves using certain chemicals to kill the cancer-causing cells in the patients body. The prescribed drugs are given in a particular timeframe for the best possible improvement in the patients health. In some patients, a stem cell transplant is provided along with high dose chemotherapy.

Radiation therapyRadiation therapy helps to destroy cancer cells with the help of specific high-energy beams to kill cancer cells in precise areas of the body. This treatment is much beneficial for patients with lymphoma

Bone marrow transplantIn this procedure, healthy stem cells are utilized to replace the cells affected by cancer. This helps the patients recover in the best possible manner. Can be autologous (where stem cells are taken from the patients own body) or allogenic (when a healthy donor gives stem cells to the patient.)

Targeted Therapy

Usually in the form of oral medications or pills. They are given alongside chemotherapy/ or radiotherapy and affect specific cancer cells and help in destroying them.

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September is Blood Cancer Awareness Month: All You Need to Know - News18

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Brave Aubrey Austin, four, donated her own stem cells and saved her baby brother Carey's life on the day he turned one, after he was diagnosed with a rare type of blood cancer aged just eight months

Image: Supplied via Lucy Laing)

A brave little girl saved the life of her baby brother on his first birthday.

Carey Austin was diagnosed with a rare type of blood cancer when he was just eight months old.

His only hope of survival was a stem-cell transplant.

Against all odds, his sister Aubrey, four, was a perfect match.

Surgeons operated on Careys first birthday and six months later he is cancer-free thanks to his big sister.

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Their mum Naomi said: She absolutely adores Carey and when we explained to her about the transplant she wanted to do everything she could to save him.

Shes only four years old, yet she was only thinking of how she could help him. We felt so guilty putting her through an operation too, but it was Careys only chance of survival.

"She was so brave about it. She knew that her blood was going to save him.

During a two-hour procedure at Great Ormond Street Hospital, London, surgeons took out Aubreys stem cells and they were put into Careys body via a drip.

Naomi said: The fact that the transplant took place on Careys birthday was so significant that she was giving him a second chance at life on that special day.

The doctors and nurses said they had never seen anyone have a stem cell transplant on their birthday before.

Aubrey was very groggy and woozy when she came around from the operation, and she had puncture wounds on her back from where the stem cells had been taken out.

But she was still smiling through it all. She was so brave. She never complained about being in pain and she was just pleased to see how her little brother was afterwards.

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When the brother and sister saw each other for the first time after the operation, there was not a dry eye in the room.

Naomi said: It was so sweet when they were reunited.

We took Aubrey to see Carey and she gave him a cuddle. They were thrilled to see each other again.

After a two-day hospital stay for Aubrey and seven weeks for Carey, the family were able to settle back into life back home in Brighton, East Sussex.

Carey is now in remission, with no signs of the cancer cells in his body.

But his parents have been warned that the disease is so aggressive that until March next year there is a 40% chance of it returning. After that, the likelihood falls to just 5%.

Naomi added: Two other children lost their lives on the cancer ward while we were there, so we know how lucky Carey has been.

He and Aubrey have always been close but now their bond is stronger than ever.

"Shes a superstar and he couldnt have wanted anything more from a big sister. Hes doing so well now. He loves playing with his cars and hes just learning to walk too.

Aubrey is with him all the time she just adores him. She knows that she has saved his life and she loves being a big sister to him. They play cars together and hes learning to walk, so she stands with him encouraging him to take his steps.

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Carey fell ill last November but Naomi, a paediatric audiologist, and her husband Simon, a CPS lawyer, both 43, thought it was bronchitis because his sister had recently had the same thing.

A GP agreed but two days later he was rushed to hospital by ambulance with breathing difficulties.

Doctors at Great Ormond Street diagnosed juvenile myelomonocytic leukaemia, or JMML, which cannot be treated with chemotherapy. There are only 1.2 cases per million children in the UK each year.

Naomi said: I was hysterical. I kept trying to tell them that it wasnt cancer, it was bronchilitis. I couldnt accept what was happening.

Because parents are not suitable donors, Aubreys bone marrow was tested, a process that involves drawing a sample out using a needle.

Naomi said: There is only a 25% chance of any sibling being a match, so even with Aubrey we knew that the odds werent in our favour.

"If she hadnt been a match then we would have had to wait until doctors found an anonymous donor, but that may not have happened in time for Carey.

When the results came back to say that she was a perfect match for him, we couldnt believe it. We had been praying that she would save him, so to get the news that she was a match for him was just incredible.

When we heard I couldnt stop crying, it was so emotional. To think that Carey was going to have a chance of survival thanks to his big sister was the answer to our prayers.

The mum added: We did feel guilty about putting her through the procedure, but when we spoke to her about it, all she wanted to do was help. We were so proud of her.

The transplant was made even more special as it took place on March 15, which was Careys first birthday, giving the family a double celebration.

They are keen to raise awareness of the cancer symptoms and the charity Childhood Cancer and Leukaemia Group, which has helped them throughout their ordeal.

Naomi said: Having a child with cancer is one of the worst things that can happen to you. We didnt realise that it was leukaemia so we are thankful that it was spotted in time.

We received amazing support throughout from the hospital and from the CCLG.

We feel so lucky that Carey has come through it and it feels like a miracle to have him with us now.

Geoff Shenton, a childrens cancer specialist at Newcastle Upon Tyne Hospitals NHS Foundation, said: In a very small proportion of cases JMML can disappear on its own, but this is rare.

Most children will need a bone-marrow or stem-cell transplant. There is still a significant chance that the disease can relapse. There may be a possibility of a second transplant if this happens, but despite our best efforts, children still die from JMML.

For more information and support visit cclg.org.uk

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This months top grants in regenerative medicine, sourced from Dimensions, includes projects on: a novel platform to enhance single cell interrogation of nervous system development, human endothelial cell regulation of ossification and the development of a dynamic double network hydrogel for generating pancreatic organoids from induced pluripotent stem cells.

This project aims to investigate a strategy, which utilizes novel spatial transcriptomics approaches, integrated multiplexed RNA/protein detection and visualization and computational algorithms to identify and map molecular markers of the preganglionic neurons in the ventral spinal cord and progenitor cell populations of the sympathetic ganglia. If successful, the approach could provide a foundation for basic research of peripheral nervous system birth defects and repair using stem cell-based therapies, as well as future studies of neuroblastoma initiation.

Funding amount:US$206,000

Funding period: 8 August 2022 31 July 2024

Funder:Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Research organization:Stowers Institute for Medical Research (MO, USA)

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Over one million patients undergo bone repair procedures in the USA annually, with autologous bone grafting remaining the preferred treatment for bone defects. The development of therapies that exploit the osteogenic potential of bone marrow-derived mesenchymal stem cells (bm-MSCs) has been limited due to limited understanding of the regulatory mechanisms of in vivo bm-MSC osteogenesis. Previous research from the group showed that the osteogenic potential of bm-MSCs is dependent on sustained proximity to endothelial cells. The goal of the present study is to elucidate the cellular and molecular mechanisms by which endothelial cells regulate the osteogenic differentiation of bm-MSCs and develop a foundation of knowledge upon which to build therapeutic strategies for bone regeneration utilizing autologous bm-MSCs.

Funding amount:US$442,000

Funding period: 10 August 2022 31 May 2027

Funder:National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Research organization:Boston Childrens Hospital (MA, USA)

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Human induced pluripotent stem cells provide a valuable source of cells for basic research and translational applications. While there have been advances in lineage-specific differentiation of human induced pluripotent stem cells, there remains limited understanding on the impact of matrix stiffness, viscoelasticity and integrin ligand presentation on the multi-stage development of exocrine pancreatic organoids. This research aims to define the influence of matrix properties on the generation of exocrine pancreatic organoids by developing a viscoelastic dynamic double network hydrogel platform with controllable matrix mechanical properties and biochemical motifs. This will advance the application of chemically defined matrices as xeno-free artificial stem cell niches for organoid growth and tissue regeneration applications.

Funding amount:US$468,000

Funding period: 1 August 2022 31 July 2026

Funder:National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Research organization: Indiana University Purdue University Indianapolis (IA, USA)

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Fast-track status is granted for frontotemporal dementia, next to the existing designation for traumatic spinal cord injury

GELEEN, Netherlands, Sept. 8, 2022 /PRNewswire/ -- The European Union has grantedstem cell biotech Neuroplastan orphan medicinal product designation for the applicability of its stem cell technology platform to frontotemporal dementia (FTD), following a positive opinion from The European Medicines Agency (EMA). With the existing orphan disease designation (ODD) for traumatic spinal cord injury (TSCI), Neuro-Cells is now approved for a fast-track development pathway with market exclusivity for both a trauma-induced and a chronic degenerative central nervous system disorder. This marks an important milestone in the development roadmap of Neuroplast's Neuro-Cells platform, as a stepping stone to other chronic neurodegenerative diseases such as Alzheimer's, ALS and Parkinson's Disease. The potential width in therapeutic applicability of the Neuroplast technology gives perspective to millions of people suffering from neurodegenerative diseases that currently have no outlook on effective treatment.

One technology addresses underlying mechanisms of multiple acute and chronic neurological disorders

Several conditions of the central nervous system, even when they seem unrelated at first and may have distinctive causes, have similar underlying disease mechanisms in common. These include unprogrammed cell death boosted by inflammation. Neuro-Cells, an autologous, bone-marrow derived Advanced Therapy Medicinal Product, addresses that disease mechanism by moderating inflammation of damaged cells in the central nervous system, to limit further impairment. The treatment objective in acute disorders is to limit impact of sudden injury, where the treatment objective in chronic disorders is to limit progression of the disease.

Neuroplast is already running a fast-track development pathway for traumatic spinal cord injury (TSCI), with a Phase II clinical trial in progress. This designation for frontotemporal dementia illustrates the broader applicability of the same technology for acute as well as chronic neurodegenerative disorders, paving the way to explore further applicability to conditions such as ALS, Alzheimer's disease, traumatic brain injury, subarachnoid stroke and Parkinson's Disease.

Orphan disease designation for FTD awarded based on pre-clinical evidence

Orphan disease designations are restricted to products for rare conditions for which there are no satisfactory methods of treatment authorized. It allows for a faster market authorization pathway and ten-year market exclusivity.

Frontotemporal dementia (FTD) is a degenerative condition in the brain that affect approximately 3.8 people in 10,000 persons in the EU. Typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

For this approval, the European Union followed the positive opinion from the EMA after the EMA followed positive recommendations from the Committee for Orphan Medicinal Products (COMP). COMP partly based their conclusions on the availability of pre-clinical evidence in mice, that showed decrease in neuroinflammation markers and rescue of cognitive and social behavioral deficits. Examples include reduction of anxiety, depressive-like behavior and abnormal social behavior.

Neuroplast CEO Johannes de Munter states:

"This designation for frontotemporal dementia is an important milestone in expanding the Neuro-Cells development to a wider range of therapeutic areas. Using the same technology platform for traumatic spinal cord injury and frontotemporal dementia, illustrates an unusual range of acute and chronic neurological disorders that could potentially benefit from this."

Neuroplast is open to discuss investor opportunities to effectuate the clinical pathways to a wider scope of neurological conditions.

About Frontotemporal dementia

Frontotemporal dementia (FTD) is a degenerative condition in the brain that is characterized by behavioral and language impairments. Depending on the variant, patients experience changes in personality, emotion, speech or motor functions. Patients may first become indifferent or careless and have difficulty understanding sentences. While the condition progresses, patients may become language impaired, lack initiative and lose executive functions. The typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

FTD affects approximately 3.8 people in 10,000 persons in the EU, for whom there are no effective treatments available. Patients typically receive antipsychotics to limit behavioral symptoms.

About Neuro-Cells

Neuro-Cells is a transformative treatment under GMP. It contains non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells, manufactured from a patient's own bone marrow (donor and receiver are the same person). Inflammatory inducing components and pathogens are removed during this process.

About Neuroplast

Neuroplast is a Dutch stem cell technology company focusing on fast-track development programs using autologous cell products for treatment of neurodegenerative diseases, with the aim of giving back perspective to people who suffer from those conditions.

The company was founded in August 2014 by physician Johannes de Munter and neurologist Erik Wolters. Current funders are Lumana Invest, Brightlands Venture Partners, LIOF and the Netherlands Enterprise Agency. Neuroplast is located at Brightlands Chemelot Campus in The Netherlands.

For more information, please visite http://www.neuroplast.com

About Lumana Invest

Investment company Lumana was established by entrepreneurs and unique due to not having a predetermined investment horizon. The Lumana founders showcase strong commitment to their portfolio companies by actively supporting management in strategic decision making.

About Brightlands Venture Partners

Brightlands Venture Partners (BVP) is the fund manager of Chemelot Ventures and is a so-called ecosystem investor. BVP invests in companies benefiting from and contributing to the Brightlands campuses in the south of The Netherlands. Other funds under management are BVP Fund IV, Brightlands Agrifood Fund and Limburg Ventures. The funds of BVP focus on sustainability and health; together the funds have made over 40 investments.

About LIOF

LIOF is the regional development agency for Limburg and supports innovative entrepreneurs with advice, network and financing. Together with entrepreneurs and partners, LIOF is working towards a smarter, more sustainable and healthier Limburg by focusing on the transitions of energy, circularity, health and digitalization.

About The Netherlands Enterprise Agency

The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy. It facilitates entrepreneurship, improves collaborations, strengthens positions and helps realize national and international ambitions with funding, networking, know-how and compliance with laws and regulations.

Forward looking statements

All statements other than statements of historical facts, including the statements about the clinical and therapeutic potential and future clinical milestones of Neuro-Cells, the indications we intend to pursue and our possible clinical or other business strategies, and the timing of these events, are forward-looking statements. Forward-looking statements can be identified by terms such as "believes", "expects", "plans", "potential", "would" or similar expressions and the negative of those terms. These forward-looking statements are based on our management's current beliefs and assumptions about future events and on information currently available to management. Neuroplast B.V. does not make any representation or warranty, express or implied, as to the improper use of this article, accuracy, completeness or updated status of above-mentioned statements. Therefore, in no case whatsoever will Neuroplast B.V. be legally liable or liable to anyone for any decision made or action taken in conjunction with the information and/or statements in this press release or for any related damages.

In case of any further questions, please contact:

Neuroplast

Johannes de Munter, CEOT: +31 (0)85 076 1000E: [emailprotected]

LifeSpring LifeSciences Communication, Amsterdam

Leon MelensT: +31 6 538 16 427E: [emailprotected]

Logo: https://mma.prnewswire.com/media/1666795/Neuroplast_Logo.jpg

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Neuroplast receives second orphan medicinal product designation for Neuro-Cells, paving the way for application to both chronic and trauma-induced...

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How do bone marrow transplants work, and what conditions do they treat?

A bone marrow transplant is actually a misnomer, as these procedures transplant stem cells, not the actual bones. Specifically, these procedures use hematopoietic stem cells (HSC), also known as blood-forming stem cells, to potentially cure an ever-expanding number of diseases.

There are three main cell types found inside a persons blood based on their function:

red blood cells: these cells carry oxygen throughout the body

platelets: these cells help form clots to stop bleeding

white blood cells: these cells lead the charge in fighting infections (also known as the immune system)

Each of these cell types, despite their different functions, shapes, sizes and lifespan, arise from the same source the hematopoietic stem cell, which constantly replenish each cell type. HSCs reside almost exclusively deep inside our bones in the center of the hard, protective shelter of calcium and other minerals. So, the marrow (soft, middle portion of our bones) can be thought of as the factory that supply each person with the blood cells needed to overcome infections, trauma, and to live a healthy, long life.

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When we perform a transplant, we are actually either using a patients own stem cells (autologous transplant) or stem cells from another human (allogeneic transplant), leading to the more appropriate name of hematopoietic stem cell transplant (HSCT). These transplants are most commonly used to treat and cure cancer.

Autologous transplants are used in the treatment of many types of solid tumors (such as brain tumors, germ cell tumors, neuroblastoma), where the tumor can only be effectively destroyed by giving very high doses of chemotherapy that also damage the patients own HSCs. Before giving a patient those high doses of chemotherapy, we collect his or her own HSCs with a process very similar to dialysis (we remove stem cells from their blood), and then freeze and store them in a specialized lab.

After the patient receives that high dose of chemotherapy, the treatment team then thaws the stem cells and infuses them back into the patient via a specialized catheter placed in his or her veins. The stem cells quickly return home and find the bone marrow space, and within 10 to 21 days, they will start making new white blood cells and platelets, followed by red blood cells.

Allogeneic transplants are performed for many types of leukemias or bone marrow failure syndromes (such as aplastic anemia or Fanconi anemia) where the patients own stem cells are broken and need to be replaced by a healthy humans stem cells. However, many other non-malignant conditions (not cancer) can be effectively cured with this procedure, as conditions that result from defects of different blood cell types (red blood cells, white blood cells or platelets) are corrected when the factory is replaced with a healthy donors stem cells.

This is an exciting time in the field of transplant, as we are now able to offer cures for many childhood diseases that historically are chronic and/or life-threatening. HSCT is now being offered to patients with sickle cell disease/thalassemia (red blood cells are defective), along with many conditions that are now called inborn errors of immunity (white blood cells are defective).

Among the more than 500 different genetic conditions that damage white blood cells include severe combined immunodeficiency (SCID), hemophagocytic lymphohistiocytosis (HLH), chronic granulomatous disease (CGD), and severe congenital neutropenia (SCN). Not only are the numbers of conditions potentially cured with HSCT rapidly growing, but the success rates and ability to prevent and treat complications of this procedure are improving exponentially as well. We are looking forward to offering these procedures to more children here at UVa Childrens.

To learn more about Dr. Roehrs and the care he provides, visit uvahealth.com/findadoctor/profile/philip-a-roehrs.

Dr. Philip Roehrs is the clinical director for pediatric stem cell transplant and cellular therapy at UVa Childrens and UVa Health.

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Ask the Expert: How do bone marrow transplants work, and what conditions do they treat? - The Daily Progress

Bone Marrow Stem Cells Comments Off on Ask the Expert: How do bone marrow transplants work, and what conditions do they treat? – The Daily Progress | September 3rd, 2022

City of Hope recently shared significant news at the 24th Annual AIDS Conference about a patient treated in 2019 whose HIV has been in remission. The man had been living with HIV for 31 years before coming to City of Hope with another grave diagnosisacute myeloid leukemia.One of the best hopes for long-term remission of acute myeloid leukemia (AML) is a stem cell transplant, and City of Hope has one of the nations leading transplant programs, having performed more than 17,000 transplants since 1976. In addition, the institution is at the forefront of using transplants to treat older adults with blood cancers, including increasing efficacy and safety in those over 60 and those with comorbidities, like the then 63-year-old City of Hope patient with HIV. The research was presented by Jana K. Dickter, M.D., City of Hope associate clinical professor in the Division of Infectious Diseases.

City of Hope hematologist Ahmed Aribi, M.D., assistant professor in the Division of Leukemia, prepared the patient for an allogeneic blood stem cell transplant with a chemotherapy-based, reduced-intensity regimen developed for treatment of older patients with blood cancers. Reduced-intensity chemotherapy makes the transplant more tolerable for older patients and reduces the potential for transplant-related complications from the procedure.

Aribi and his team worked with City of Hopes Unrelated Donor BMT Program directed by Monzr M. Al Malki, M.D. to find a donor who was a perfect match for the patient and had the rare genetic mutation, homozygous CCR5 Delta 32, which is found in just 1 to 2% of the general population.

People who have this mutation have a resistance to acquiring HIV. CCR5 is a receptor on CD4+ immune cells, and most strains of HIV use that receptor to enter and attack the immune system. But the CCR5 mutation blocks that pathway, which stops HIV from replicating.

After this successful transplant for both AML and HIV, the patient has been in remission for HIV since stopping ART in March 2021. While this outcome has happened in three other patients, the City of Hope patient was both the oldest to undergo a transplant with HIV and leukemia and go into remission for both. He had also lived with HIV the longest 31 years.

The City of Hope patient is another major advancement. It demonstrates that research and clinical care developed and led at City of Hope are changing the meaning of an HIV diagnosis for patients across the United States and the world, said John Zaia, M.D., director of City of Hopes Center for Gene Therapy, Aaron D. Miller and Edith Miller Chair for Gene Therapy and a leader in HIV research. City of Hope remains at the forefront of clinical research that changes peoples lives for the better.

When I was diagnosed with HIV in 1988, like many others, I thought it was a death sentence. I never thought I would live to see the day that I no longer have HIV. City of Hope made that possible, and I am beyond grateful. The City of Hope patient

The story above is one significant example of several important advances being made at City of Hope in the care of people with HIV. When many centers still treated patients with low-intensity, noncurative treatment approaches for HIV-related lymphoma, City of Hope challenged that paradigm by demonstrating that autologous transplantation could be used to cure patients who would otherwise die.

More recently, City of Hope is leveraging its leadership in CAR T cell therapya groundbreaking treatment currently used to rally the bodys natural defenses against cancer and exploring its potential in tandem with another advance, City of Hopes vaccine for cytomegalovirus (CMV).

In a proof-of-concept study, funded by theCalifornia Institute for Regenerative Medicine, lab models demonstrated that the combination therapy could recognize and eliminate HIV without serious toxicity to cells in the virus host. In cultured human cells, the CAR T cells killed cells tagged with the gp120 protein, and kept killing them, without significant signs of risking damage to healthy cells. In a mouse model for HIV/AIDS, high doses of the dual-action CAR T cells followed by the CMV vaccine were successful in controlling HIV, and even nestled into the bone marrow, indicating potential for treatment to keep working over the long term.

In addition to achieving breakthrough outcomes in cancer and HIV, City of Hope has been recognized as the seventh "Best Hospital" for cancer in the nation according to U.S. News & World Report's 2022-23 Best Hospitals: Specialty Ranking. This marks the first time the cancer treatment center has cracked the top 10 of the U.S. News & World Report annual rankings and the 16th consecutive year it has been distinguished as one of the nation's elite cancer hospitals. It was also rated as high performing in four cancer surgery specialties: lung, colon, prostate and ovarian cancers.

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From optimized stem cell transplants to CAR T cell therapy: Advancing options for cancer, HIV and more - City of Hope

Bone Marrow Stem Cells Comments Off on From optimized stem cell transplants to CAR T cell therapy: Advancing options for cancer, HIV and more – City of Hope | September 3rd, 2022

North America Market Comprises Of 53.1% Market Share Due To Rising Number Of Spine-Related Disorders

Fact.MR A Market Research and Competitive Intelligence Provider: Theglobal bone grafts and substitutes marketreached a valuation ofUS$ 3.06 Bnin 2020. Moreover, sales of bone grafts and substitutes are slated to rise at a CAGR of4.9%to reachUS$ 4.44 Bnby the end of 2028.

Bone grafts and substitutes (BGS) are rapidly used common materials used mainly to replace missing bones or mend fractures. Moreover, it is commonly being used in the hip, foot, and ankle surgeries, as well as fractures and musculoskeletal injuries. Moreover, the primary goal of using bone grafts and substitutes is to aid in the healing of fractures and bone injuries, as well as to replace natural bone.

Moreover, surge in demand for synthetics and xenografts, rise in usage of bone graft substitutes in regenerative medicines, and the surge in the number of illnesses that necessitate their usage would propel the market for bone grafts and substitutes forward.

In addition to this, continuous R&D initiatives to upgrade product offerings are one of the most common trends in the market. This rise in R&D initiatives is driven by surge in need for bone graft substitutes for bone-related occurrences fractures and trauma. Researchers from across the globe are putting in efforts to find new ways to use bone grafts in regenerative medicines.

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Bone grafts manufacturers are constantly investing in the development of new products with improved bioactivity, biocompatibility, and mechanical qualities. Companies have a varied product portfolio that is technologically advanced, as well as a larger global presence. Key players in the market are putting emphasis on innovative products in various orthopedic application areas.

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Bone Marrow Processing Systems Market - A bone marrow processing system is a functionally closed, sterile system designed for automatically isolating and concentrating stem cells derived from donated bone marrow aspirate.

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Antibiotic-loaded Bone Cement Market - Infections are among the major issues encountered during various orthopedic surgeries, and antibiotic-loaded bone cement is commonly used to avoid any sorts of medical predicaments. To ensure the safety of patients undergoing orthopedic surgeries, the demand for antibiotic-loaded bone cement is increasing across the healthcare industry.

Injectable Bone Graft Substitutes Market - Growing instances of bone defects among individuals has fuelled demand for the bone grafting techniques in the healthcare industry. As the need to conduct trauma and orthopedic surgeries persist, manufacturers are developing a range of bone grafts or bone graft substitutes to stimulate insufficient or impaired bone regeneration.

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Bone Grafts And Substitutes Market Is Expected To Witness An Impressive CAGR Of 4.9% Due To Rise In Usage Of Bone Grafts And Substitutes For Healing...

Bone Marrow Stem Cells Comments Off on Bone Grafts And Substitutes Market Is Expected To Witness An Impressive CAGR Of 4.9% Due To Rise In Usage Of Bone Grafts And Substitutes For Healing… | September 3rd, 2022

So far, Bcells havent gotten the same attentionindeed, genetically engineered versions have never been tested in a human. Thats partly because engineering B cells is not that easy, says Xin Luo, a professor at Virginia Tech who in 2009 demonstrated how to generate B cells that have an added gene.

That early work, carried out at Caltech, explored whether the cells could be directed to make antibodies against HIV, perhaps becoming a new form of vaccination.

While that idea didnt pan out, now biotech companies like Immusoft, Be Biopharma, and Walking Fish Therapeutics want to harness the cells as molecular factories to treat serious rare diseases. These cells are powerhouses for secreting protein, so thats something they want to take advantage of, says Luo.

Immusoft licensed the Caltech technology and got an early investment from Peter Thiels biotech fund, Breakout Labs. Company founder Matthew Scholz, a software developer, boldly predicted in 2015 that a trial could start immediately. However, the technology the company terms immune-system programming didnt turn out to be as straightforward as coding a computer.

Ainsworth says Immusoft had to first spend several years working out reliable ways to add genes to B cells. Instead of using viruses or gene editing to make genetic changes, the company now employs a transposona molecule that likes to cut and paste DNA segments.

It also took time to convince the FDA to allow the trial. Thats because its known that if added DNA ends up near cancer-promoting genes, it can sometimes turn them on.

The FDA is concerned if you are doing this in a B cell, could you develop a leukemia situation? That is something that they are going to watch pretty closely, says Paul Orchard, the doctor at the University of Minnesota who will be recruiting patients and carrying out the study.

The first human test could resolve some open questions about the technology. One is whether the enhanced cells will take up long-term residence inside peoples bone marrow, where B cells typically live. In theory, the cells could survive decadeseven the entire life of the patient. Another question is whether theyll make enough of the missing enzyme to help stall MPS, which is a progressive disease.

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A new gene therapy based on antibody cells is about to be tested in humans - MIT Technology Review

Bone Marrow Stem Cells Comments Off on A new gene therapy based on antibody cells is about to be tested in humans – MIT Technology Review | September 3rd, 2022

By Giles Campion, EVP, head of R&D and chief medical officer, Silence Therapeutics

siRNA is a gene-silencing technology with great potential for treating a wide range of rare diseases, as I discussed in my previous article, but its promise doesnt end there. In this last article in the series, I examine siRNAs potential for treating not-so-rare and even quite common diseases.

Unlike rare diseases, which are often caused by pathological genetic mutations, common diseases may be associated with genetic variants that are not pathological and therefore do not dysregulate a biological process. For example, variants of the LPA or PCSK9 gene can increase a persons risk of cardiovascular disease by affecting cholesterol levels, but these variants do not directly cause cardiovascular disease by disrupting a fundamental biological process. This contrasts with, for example, mutations in the HBB gene that cause beta thalassemia and disrupt the mechanisms that protect the body from toxic iron buildup.

Nevertheless, the approach to treating rare and common diseases with siRNA therapies is similar: silence a gene that has little or no effect on phenotypes outside the disease, thereby maximizing safety. This is an important factor in rare diseases, which often begin early in life and require lifelong treatment. But it is equally important in common chronic diseases, such as hyperlipidemia, in which a patient has abnormally high levels of fats in the blood, where patients may live for decades before they experience any overt symptoms from their condition and are not likely to tolerate a therapy with even minor side effects that interfere with their quality of life.

At the forefront of common conditions being targeted by gene silencing is elevated lipoprotein (a), or Lp(a), a cholesterol-rich particle closely related to the well-known cardiovascular risk factor LDL. High levels of Lp(a) are associated with high risk of cardiovascular events, such as heart attacks and strokes; low levels of Lp(a) are associated with a low risk of these events.

Unlike other types of cholesterol-carrying particles, Lp(a) levels are not significantly modifiable by lifestyle factors; levels are genetically determined by the variant of the LPA gene, which encodes apolipoprotein (a) a major protein component of Lp(a) that a person has. Because these variants are not pathological mutations, the person may not experience disease symptoms for years and may even be unaware of their elevated Lp(a) levels. Yet the condition is common: One in five people have high levels of Lp(a), defined as 50 mg/dl or 120 nmol/L. Other cholesterol-reducing medicines, such as statins, have no effect on Lp(a) and can even increase levels; currently there are no approved Lp(a)-reducing therapies.

However, assessments of human genetic databases, such as the UK Biobank, have revealed that cardiovascular risk is the only phenotype associated with Lp(a) levels. Some individuals have zero levels of Lp(a), and the only known phenotype in them is a much-reduced incidence of cardiovascular events. This indicates that silencing LPA with a properly designed siRNA therapy, such as Silences clinical-stage asset SLN360, could reduce the risk of cardiovascular disease in people with elevated Lp(a) while minimizing the risk of any unwanted or unexpected side effects.

The PCSK9 gene is another example of an siRNA target for the common condition of hyperlipidemia. The PCSK9 protein negatively regulates the cellular uptake of low-density lipoprotein-cholesterol (LDL-C) in the bloodstream by reducing the number of LDL receptors on the surface of cells. This means that high levels of PCSK9 decrease cellular uptake of LDL-C, leaving more of it in circulation.

High LDL-C levels in blood are associated with coronary artery disease (CAD). While not entirely determined by genetics, as Lp(a) levels are, some variants of the PCSK9 gene are associated with low levels of LDL-C and a reduced incidence of cardiovascular disease. Similar to the LPA gene, this suggests that silencing PCSK9 with an siRNA could reduce LDL-C levels in the blood to treat hyperlipidemia and reduce the risk of CAD. Indeed, the siRNA therapy inclisiran, which silences PCSK9, was approved by the European Union in December 2020 and in the United States in December 2021 for use in people with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk equivalents, and heterozygous familial hypercholesterolemia (HeFH), in conjunction with lifestyle changes and other cholesterol-lowering medicines.

An important feature of siRNA therapies in the treatment of common chronic conditions such as elevated Lp(a) and elevated LDL-C is that they have long-lasting effects, and thus they require less frequent dosing than statins and other small molecule drugs, which must be taken daily. This in turn should increase patients compliance with the therapeutic regimen and thereby improve outcomes. In fact, a 2018 retrospective study found that hyperlipidemia patients who were prescribed the right intensity (level) of statin treatment and complied 100% with their therapy had a 40% lower risk of cardiovascular events than patients who received low-intensity statin treatment and had 5% compliance.1The study concluded that an optimal therapy could reduce the risk of cardiovascular events by 30% in three years.

Though published before any siRNA therapy was approved for hyperlipidemia, the studys implications are clear: Therapeutic intensity and patient compliance are important factors in saving peoples lives. With siRNA therapies, the intensity is known, and the compliance issues are likely to be less of an issue compared with oral drugs. This is just one aspect of siRNA that makes it as well-suited for treating common diseases as rare diseases.

siRNA also has the potential to improve outcomes in hematopoietic stem cell transplantation (HSCT). Though not a disease per se, HCST is a procedure commonly used to treat a range of blood cancers and, with increasing frequency, certain autoimmune disorders.

HCST involves ablating the existing bone marrow to make way for a healthy stem cell graft to repopulate the marrow. This ablation shifts an enormous load of dead iron-laden blood cells into the circulation. Retrospective studies suggest this acute release of toxic iron from ablated cells can adversely affect the survival of the stem cell graft and increase the risk of potentially lethal infections in HSCT patients.

As in the rare disease examples I mentioned previously, silencing TMPRSS6 with an siRNA could increase hepcidin to reduce iron levels in HSCT patients, potentially improving their survival and engraftment outcomes.

I am passionate about RNA technology and the benefits that targeted, precision siRNA medicines can bring to patients with rare diseases and not-so-rare diseases who need new therapeutic options. As both a physician and drug developer, I find it rewarding and exciting to witness this technology finally coming into its own, with the promise of delivering even greater benefits in the coming years.

Reference

About The Author:

Giles Campion, MD, joined Silence Therapeutics as head of R&D and chief medical officer in 2019 and was appointed as an executive director in 2020. He is an expert in translational medicine and an experienced biotech and pharmaceutical professional across many therapeutic areas, most recently in orphan neuromuscular disorders. He has held senior global R&D roles in several large pharma, diagnostics, and biotech companies, including as group vice president of the neuromuscular franchise at BioMarin Pharmaceutical Inc., and chief medical officer and senior vice president of R&D at Prosensa. He is also a co-founder of PepGen Ltd. He earned his bachelors and doctorate degrees in medicine from the University of Bristol and is listed on the General Medical Council (UK) Specialist Register (Rheumatology).

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The Promise Of Gene Silencing To Treat Not-So-Rare Diseases - BioProcess Online

Bone Marrow Stem Cells Comments Off on The Promise Of Gene Silencing To Treat Not-So-Rare Diseases – BioProcess Online | September 3rd, 2022