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Genocea Initiates Restructuring and Announces Plan to Explore Strategic Alternatives

By Dr. Matthew Watson

CAMBRIDGE, Mass., April 28, 2022 (GLOBE NEWSWIRE) -- Genocea Biosciences, Inc. (Nasdaq: GNCA), a biopharmaceutical company developing next-generation neoantigen immunotherapies, today announced that it has initiated a process to explore a range of strategic alternatives to maximize shareholder value and has engaged professional advisors, including an investment bank to act as a strategic advisor for this process. Strategic alternatives that will be evaluated include the sale of all or part of the Company, merger or reverse merger.

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Travere Therapeutics to Report First Quarter 2022 Financial Results

By Dr. Matthew Watson

SAN DIEGO, April 28, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced it will report first quarter 2022 financial results on Thursday, May 5, 2022, after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 4:30 p.m. ET.

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Adaptimmune to Report Q1 2022 Financial Results and Business Updates on Monday, May 9, 2022

By Dr. Matthew Watson

PHILADELPHIA and OXFORDSHIRE, United Kingdom, April 28, 2022 (GLOBE NEWSWIRE) -- Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, will report financial results and provide business updates for the first quarter ended March 31, 2022, before the US markets open on Monday, May 9, 2022. Following the announcement, the Company will host a live teleconference and webcast at 8:00 a.m. EDT (1:00 p.m. BST) that same day.

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Eagle Pharmaceuticals to Host First Quarter 2022 Financial Results on May 9, 2022

By Dr. Matthew Watson

WOODCLIFF LAKE, N.J., April 28, 2022 (GLOBE NEWSWIRE) -- Eagle Pharmaceuticals, Inc. (“Eagle” or the “Company”) (Nasdaq: EGRX) today announced that the Company will release its 2022 first quarter financial results on Monday, May 9, 2022, before the market opens.

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Dyadic to Report First Quarter 2022 Financial Results on Thursday, May 12, 2022

By Dr. Matthew Watson

JUPITER, Fla., April 28, 2022 (GLOBE NEWSWIRE) -- Dyadic International, Inc. (“Dyadic”, “we”, “us”, “our”, or the “Company”) (NASDAQ: DYAI), a global biotechnology company focused on further improving, applying and deploying its proprietary C1-cell protein production platform to accelerate development, lower production costs and improve the performance of biologic vaccines and drugs at flexible commercial scales, today announced that it will report its financial results for the first quarter 2022 and host a corporate update conference call on Thursday, May 12, 2022.

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Oncternal Therapeutics to Provide Business Update and Report First Quarter 2022 Financial Results

By Dr. Matthew Watson

SAN DIEGO, April 28, 2022 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that it will report first quarter 2022 financial results after the U.S. financial markets close on Thursday, May 5, 2022. Oncternal’s management will host a webcast at 2:00 p.m. PT (5:00 p.m. ET) to provide a comprehensive business update and discuss the Company’s financial results.

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Aadi Bioscience to Announce First Quarter 2022 Financial Results on May 12, 2022

By Dr. Matthew Watson

Company to hold conference call on May 12th at 8:30 am EDT Company to hold conference call on May 12th at 8:30 am EDT

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Finch Therapeutics Announces Removal of FDA Clinical Hold on CP101 IND

By Dr. Matthew Watson

SOMERVILLE, Mass., April 28, 2022 (GLOBE NEWSWIRE) -- Finch Therapeutics Group, Inc. (“Finch” or “Finch Therapeutics”) (Nasdaq: FNCH), a clinical-stage microbiome therapeutics company leveraging its Human-First Discovery® platform to develop a novel class of orally administered biological drugs, today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on Finch’s investigational new drug (IND) application for CP101. CP101 is the Company’s investigational orally administered microbiome therapeutic which is in late-stage clinical development for the prevention of recurrent C. difficile infection (CDI). The FDA lifted the clinical hold following a review of information Finch provided related to its SARS-CoV-2 screening procedures and associated informed consent language.

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Appendix 4C Quarterly Activity Report

By Dr. Matthew Watson

Mesoblast Operational and Financial Highlights for Quarter Ended March 31, 2022 Mesoblast Operational and Financial Highlights for Quarter Ended March 31, 2022

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Bone Therapeutics announces 2021 full year results

By Dr. Matthew Watson

REGULATED INFORMATION

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New three-year data for Roche’s Evrysdi (risdiplam) show long-term improvements in survival and motor milestones in babies with Type 1 spinal…

By Dr. Matthew Watson

Basel, 29 April 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new three-year data from the FIREFISH study, including one-year data from the open label extension, reinforcing the long-term efficacy and safety of Evrysdi® (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed an estimated 91% of infants (n=58) treated with Evrysdi were alive after three years of treatment. The Evrysdi-treated infants continued to improve or maintain motor functions, including the ability to swallow, sit without support, stand with support and walk while holding on, between two and three years of treatment. Without treatment, children with Type 1 SMA are never able to sit without support. The study also showed overall continued reductions in serious adverse events (SAEs) and hospitalisations over time.

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New three-year data for Roche’s Evrysdi (risdiplam) show long-term improvements in survival and motor milestones in babies with Type 1 spinal...

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Santhera will Publish its 2021 Annual Report by June 3, 2022, and Hold its Annual General Meeting on June 29, 2022

By Dr. Matthew Watson

Ad hoc announcement pursuant to Art. 53 LR

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Santhera will Publish its 2021 Annual Report by June 3, 2022, and Hold its Annual General Meeting on June 29, 2022

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Letter to shareholders from CLINUVEL’s CEO

By Dr. Matthew Watson

LONDON, April 29, 2022 (GLOBE NEWSWIRE) --      Dear shareholders, friends,

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Versant-backed startup launches with plans to broaden cell therapy’s reach – BioPharma Dive

By daniellenierenberg

Stem cell transplants can effectively cure a wide range of diseases, from blood cancers to rare genetic disorders. They've been used for decades and are considered standard treatment for certain conditions.

But for a good number of patients, stem cell transplants are out of reach. Drug regimens used to prepare the body for a transplant are toxic and can cause serious side effects. The transplanted cells don't always "engraft," or take root in the bone marrow. Even when they do, patients' disease may linger or recur.

A biotech startup launching Wednesday with $50 million in funding hopes that, by combining cell, antibody and gene editing technologies, at least some of these problems can be overcome. Called Cimeio Therapeutics, the new company is led by a team of pharmaceutical industry veterans and an advisory board filled with scientific luminaries, including immunologist Jeffrey Bluestone and gene editing pioneer Fyodor Urnov.

Cimeio's approach involves "shielding" transplanted cells by genetically editing them in ways that allows paired immunotherapies to be safely used both before and after a transplant.

Thomas Fuchs

Courtesy of Cimeio Therapeutics

"We think that this can really unleash the power of hematopoietic stem cell transplant and make a lot more patients eligible for it," said Thomas Fuchs, Cimeio's CEO and a former Genentech executive.

The "shielding" technology used by Cimeio was developed in Switzerland at the laboratory of Lukas Jeker, a physician-scientist from Basel University Hospital who will join Cimeio as head of gene editing.

Jeker's lab discovered that protein receptors on the surface of cells could be genetically edited in such a way that prevented antibodies from binding to them, while leaving their function intact. In preclinical testing, these edits could cloak, or "shield," the cells from being depleted by antibody drugs and T cell therapies.

The work could have powerful implications for improving stem cell transplant and adoptive cell therapy, according to Fuchs.

Once a stem cell or T cell is shielded, a complementary immunotherapy could be used to either help ready patients for a transplant or to further treat disease afterwards, he said. "Maybe you could give a cycle or two of the paired immunotherapy, implant the shielded cells and then continue to administer the immunotherapy," he added.

If the shielding works as intended, Cimeio could develop treatments for conditioning that are more tolerable than the chemotherapy or radiation-based regimens currently in use. Shielding might also allow existing drugs that target cell proteins on healthy as well as diseased cells to be used more flexibly with transplants, such as to treat residual disease that lingers afterwards.

For example, Cimeio could engineer stem cells that are protected against binding via a protein called CD19 that's often the target for CAR-T therapies that treat lymphoma, but is also found on healthy B cells that help the immune system fight off threats.

"One benefit could be that you could prevent a lifetime of B cell depletion, which happens when you give a CAR-T," said Fuchs.

Alex Mayweg

Courtesy of Cimeio Therapeutics

Cimeio was built from Jeker's lab by Versant Ventures at the company's "Ridgeline" incubator in Basel, which has previously produced companies like Monte Rosa Therapeutics and Black Diamond Therapeutics. The initial $50 million Versant provided will fund Cimeio through next year, said Alex Mayweg, a managing director at the venture firm and a Cimeio board member. Additional investors will be brought on later this year or early next, Mayweg said.

Cimeio will need the money, as its research and development plans are expansive. The company has identified four drug candidates already and envisions a dozen more behind those, said Fuchs. Its research spans blood cancers, rare genetic diseases and autoimmune disorders.

In some cases, Cimeio will develop paired immunotherapies to go with the shielded cells. In others, it will use existing treatments. Three of the first four candidates involve protecting hematopoietic stem cells, while the fourth involves T cells. The company hopes to begin human testing next year.

Cimeio plans to choose gene editing technologies based on the type of alteration it needs to make to shield cells. "Rather than building up an internal editing capability," Mayweg said, "we wanted to stay as flexible as possible."

That might mean partnerships or alliances with other companies, some of which have reached out to Cimeio already, according to Mayweg.

Cimeio is aided by a group of scientific advisers notable for their work in areas the company is focusing on. Urnov, of the University of California, Berkeley, is well known for his research in gene editing using zinc finger nucleases and CRISPR. Bluestone previously led the Parker Institute for Cancer Immunotherapy and is CEO of the cell therapy-focused biotech Sonoma Biotherapeutics.

Suneet Agarwal, a co-program leader of the stem cell transplant center at Boston Children's Cancer and Blood Disorders Center, is also on the advisory board, while Cimeio has a research collaboration in place with Matthew Porteus, a gene editing specialist at Stanford University.

About 20 people currently work at Cimeio directly, a number Fuchs expects will grow as the company's research advances. Another 15 are currently supporting Cimeio from Versant's Ridgeline group.

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Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why – WATN – Local 24

By daniellenierenberg

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about a lawsuit against one company and what consumers need to watch out for.

MEMPHIS, Tenn. The Federal Trade Commission and Georgia Attorney General have sued the founders of a company they claim has made unsubstantiated claims its stem cell therapy can treat arthritis, joint pain, and other orthopedic ailments.

The company is called Stem Cell Institute of America. It claimed its treatments are comparable to or better than surgery, steroid injections, and painkillers. The FTC said the company charged up to $5,000 per injection. It said a related company taught chiropractors and other healthcare professionals how to offer stem cell therapy.

ABC24 talked with Randy Hutchinson from the Better Business Bureau of the Mid-South about the claims and what consumers need to watch out for.

So what are the facts about stem cells?

They're sometimes called the body's "master cells" because they develop into blood, brain, bones, and other organs.

Stem cells from bone marrow or blood are used to treat certain kinds of cancer and disorders of the blood and immune system. But other uses have not been properly studied and approved.

The FDA cites these potential risks from unproven treatments:

There could be safety risks even using a persons own stem cells.

Other claims by some companies

The FTC has also looked into companies claiming their stem cell therapies can treat Parkinson's, multiple sclerosis, COVID, and a host of other ailments. They're sometimes referred to as "regenerative medicine."

So what do consumers need to do?

Take miracle health care claims with a grain of salt.

Check out a company and treatment online using terms like "complaints," "scam" and "reviews."

Consult your own health care provider before using any product or treatment.

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Be wary of companies offering stem cell therapy for arthritis, joint pain, COVID, and more. Heres why - WATN - Local 24

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Columbus man to be one of first to receive transplant via new OhioHealth program – 10TV

By daniellenierenberg

Marc Howard was diagnosed with multiple myeloma. He will receive a transplant using his own cells later this year.

COLUMBUS, Ohio It all started with back pain that seemed to be progressively getting worse.

My back, the structure of my body, was like starting to deteriorate, and I could tell, he said. I'm tall, so when I started to lean over, and the pain and things of that nature, I'm like, yo, something's going on.

His longtime love Sonia Grant noticed, too. And she was right there to encourage him to get it checked.

When he did, doctors found holes in his spine where his bone had deteriorated. He had a vertebroplasty procedure to have those holes filled with bone cement. But that was not the end of his journey. In fact, it was really only the beginning.

After the surgery, he was okay for about a month, then I saw him (leaning) over again, and he couldnt get off the bed one day, Grant said. I said, uh uh, were going back up there (to the hospital). Theres something wrong.

And something was. Howard was diagnosed with multiple myeloma, a cancer that forms in the plasma cells.

I dont want to be the woe is me, Howard said. I want to be the success story for somebody, for the world to look at, like, that man went through a situation, and he made it.

And hes making it so far, with the help of Grant. Hes been doing weekly chemotherapy treatments and taking daily medication. Meanwhile, Grant is making sure hes eating his fruits and veggies and drinking plenty of water, too.

If youre not up to the challenge, I will help you get there, I will, Grant said. Because failure is just not a thing when it comes to fighting something like cancer. You gotta fight, you just gotta fight.

This fight will culminate with a major procedure later this fall via OhioHealths new Blood and Marrow Transplant Program. Howard will be one of the first patients to receive an autologous stem cell transplant, meaning the procedure will use his own cells.

Dr. Yvonne Efebera, the medical director for the program, explains this process is different than a procedure using donor cells.

BMT, blood and marrow transplant, is a process where, certain diseases require this, where non-functioning, deficient bone marrow or cancer cells are eliminated by giving high-dose chemotherapy, with or without radiation, and then replaced by new, healthy cells, Dr. Efebera said.

Shes been treating Howard throughout this process and points out that this is one of the benefits of the new program. Before, patients who needed transplants would have to be sent to other healthcare systems. Now, they can go from start to finish with the same clinical team.

Marc always wanted to be the first, she joked. Hes anxious to have his stem cells to be the first collected and the first admitted.

Both Howard and Grant are up to the challenge.

Its a battle, Grant said. Were halfway through the battle, and so, were going to get all the way to the end of the battle. Bruised, not broken. But were in the battle. But were going to get through it.

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Molecular Characteristics of Resistance to CD19-Directed CAR T Cells Revealed in Pediatric ALL – Cancer Network

By daniellenierenberg

Investigators uncovered potential mechanisms of resistance to CD19-directed CAR T-cell therapy in patients with pediatric acute lymphoblastic leukemia.

Bone marrow samples from a trial examining CD19-directed CAR T-cell therapy helped investigators identify epigenetic biomarkers predicting resistance to therapy in pediatric acute lymphoblastic leukemia (ALL), according to a presentation from the 2022 American Association for Cancer Research (AACR) Annual Meeting.1

Investigators observed 3 key features of leukemia cells that do not respond to CAR T-cell therapy: hypermethylation of DNA, a stem cell-like phenotype and inherent plasticity, and decreased antigen presentation. These are independent of CD19 status and leukemia subtype, indicating a new predictive biomarker.

Whats most important about this is we can detect it prior to therapy in patient samples, so this highlights its potential as a biomarker for response, Katherine E. Masih, BS, an NIH-Cambridge scholar in the Genetics Branch, Center for Cancer Research at the National Cancer Institute, said in a press conference. We hope that eventually this can improve patient selection and eligibility for CD19 CAR T-cell therapy.

CD19 is a common target of CAR T cells, and resistance to treatment can occur even if patients continue to show CD19 expression. The investigators explored primary non-response (PNR) to CAR T-cell therapy, which occurs in 10% to 20% of patients and whose causes are not fully understood. Known reasons for non-response to CAR T cells include collection of dysfunctional T cells and decreased death cell receptors on the cell surface.2,3

The investigators used bone marrow samples from 14 participants in the PLAT-02 trial (NCT02028455) of CD19-directed CAR T-cell therapy for relapsed/refractory pediatric ALL.1 These samples included those of 7 patients who had a complete response to therapy and 7 who had no response. Non-response was defined as not achieving and maintaining minimal residual disease negativity at 63 days.

A multiomic analysis of the bone marrow included whole-exome sequencing, RNA sequencing of the bulk cells, single-cell RNA sequencing, array-based methylation analysis, and ATAC-seq (Assay for Transposase-Accessible Chromatin using sequencing).

In patients who went on to have PNR, investigators discovered epigenetic markers including 238 regions of hypermethylated DNA, which is associated with inactivated genes (P = 8.15 10-25). These regions are known to be activated in stem cells.

The ATAC-seq analysis showed increased accessibility of chromatin at regions linked to stem cell proliferation (normalized enrichment score [NES] = 2.31; P < .0001) and cell cycling (NES = 2.27; P < .0001), indicating inherent plasticity that may allow leukemia cells to adapt to resist CAR T-cell therapy.

Investigators found that the epigenetic differences did not lead to differences in phenotype of B cells between primary sensitive and PNR patients. However, PNR cells did show an increase in regions associated with hematopoietic stem cells and myeloid and lymphoid progenitors. In addition, investigators observed decreased antigen presentation and processing that could lead to lack of response in cells that still express CD19 (P = .0001).

These factors characterized a potential novel biomarker associated with PNR that investigators named Stem-Cell Epigenome with Multi-Lineage Potential (SCE-MLP). Masih acknowledged that the sample size of 14 patients was small and more research on SCE-MLPs link to PNR is needed. We would love to see this validated in a larger cohort with more cases of PNR that exist around the country, she said.

Another potential use of SCE-MLP could be to find ways to overcome resistance to CAR T-cell therapy by combining it with targeted therapies that disrupt these epigenetic factors for resistance.

Currently, the investigators hope that this research will be used to shape patient selection for CAR T-cell therapy and alternative therapies.

If we can reliably identify responders, perhaps through screening for SCE-MLP, we can prioritize less toxic targeted therapies for our patients and overall improve outcomes for children with this devastating disease, Masih said.

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Penticton woman donated bone marrow to save stranger’s life, urges others to do the same – Penticton News – Castanet.net

By daniellenierenberg

Photo: Contributed

Salny Ehman, left, and Tom Ellison, the man whose life she saved through a bone marrow donation.

Penticton resident Salny Ehman saved a complete stranger's life, and hopes to inspire others to do the same.

Ehman, now 35, signed up to be a bone marrow donor at the age of 19, after watching her grandmother's sibling go through leukemia.

"She has a whole bunch of siblings, and one of them had blood cancer, and only she was a match. And the math of that blew me away, how likely is it to have a close relative that can help?" Ehman said.

"If it's that unlikely to have a match when you have that many siblings, then people need to sign up."

Ehman did just that. She registered with Canadian Blood Services, and a few months later, got a call that she was a match with an anonymous recipient signed up through an American service, fighting cancer and in need of bone marrow stem cells.

Despite not knowing who she was donating to, Ehman underwent the donation procedure, and then one year later, did the same thing again after learning the recipient's body had been rejecting the donated cells.

"One year after the second donation, we were both allowed to say yes, we would like to know the other person. And we both said yes, but I was living in Nova Scotia at the time," Ehman said, having learned her stem cell recipient, Tom Allison, lived in Seattle.

"The only information given to me about [my donor] was it was a 19 year old girl from Nova Scotia. And I thought, what's a 19 year old girl doing on a bone marrow registry for?" Allison said.

"Neither one of my boys were matches, and neither one of my brothers. So this seemed just a million to one chance for somebody out there with a match that's no relation to me."

Allison and Ehman ultimately got in touch and kept in touch for more than a decade.

"We would write each other letters over the years, and little postcards," Ehman said.

"And then I moved here [to Penticton] two years ago. So as soon as I moved here, I was like, 'Wow, I'm so close.' Just a quick drive, like three and a half hours away."

She and Allison, who is now in his 60s and healthy post-cancer, met up in person recently, and Ehman was thrilled to see him thriving thanks to her donation.

"Second to my daughters, [donating] was the best, best experience in my entire life. And to know that he was out there, and spending time with his family, his grandchildren, it really meant a lot. And it showed me what I was capable of," Ehman said.

She and Allison remain good friends, and she urges anyone who can help to either sign up to be a donor, or if they can't participate in that way, give financially to Canadian Blood Services.

"There's lots of opportunities to help that cause," she said.

Find out more about Canadian Blood Services and how you can help here, and learn how you can be the match that saves someone's life here.

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microRNA-148a in Exosomes Derived from Bone Marrow Mesenchymal Stem Cells Alleviates Cardiomyocyte Apoptosis in Atrial Fibrillation by Inhibiting…

By daniellenierenberg

This article was originally published here

Mol Biotechnol. 2022 Apr 9. doi: 10.1007/s12033-022-00487-z. Online ahead of print.

ABSTRACT

Exosomes-related microRNAs (miRNAs) have been considered to be the significant biomarkers contributing to the development of atrial fibrillation (AF). We observed the implicit mechanism of exosomes-miR-148a derived from bone marrow mesenchymal stem cells (BMSCs) in AF. The AF cell and mice models were established firstly. QRT-PCR and Western blot analysis were applied to detect the expression of miR-148a, SPARC-associated modular calcium-binding protein 2 (SMOC2), Bcl-2, Bax, and caspase-3. BMSCs were separated from healthy mice and exosomes were obtained from BMSCs. BMSCs were transfected with mimics and inhibitor, and HL-1 cells were treated with mimics and pcDNA3.1. MTT assay were used to detect cell viability of cells. Flow cytometric analysis and TUNEL analysis were used for detecting cell apoptosis of cells. In our study, exosomes derived from BMSCs inhibited the development of AF, and miR-148a acted a vital role in this segment. SMOC2 was a target gene of miR-148a and promoted apoptosis of HL-1 cells. Additionally, miR-148a mimics decreased cellular apoptosis, eliminated SMOC2 expression, and elevated Bcl-2 expression in AF-treated cells. Collectively, miR-148a overexpressed in BMSC-exosomes restrained cardiomyocytes apoptosis by inhibiting SMOC2.

PMID:35397056 | DOI:10.1007/s12033-022-00487-z

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microRNA-148a in Exosomes Derived from Bone Marrow Mesenchymal Stem Cells Alleviates Cardiomyocyte Apoptosis in Atrial Fibrillation by Inhibiting...

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Donors needed to save young lives – Benoni City Times

By daniellenierenberg

Three children are fighting for their lives and require stem cell transplants to cure their blood cancers and disorders.

Rayaan (four months), Emily (five months) and Neo (7) all share one commonality: being diagnosed with life-threatening blood diseases. They are waiting on blood stem cell transplantations from unrelated donors.

Between 800 and 1 000 children in South Africa are diagnosed with cancer annually.

However, this number does not account for the almost 50% of cases of childhood cancer that are never diagnosed, due to a lack of knowledge regarding the disease and how it presents in children.

Because children still experience growth spurts within a short space of time, this may cause blood cancer and disorders to spread quicker and more aggressively. Therefore, diseases affecting young children are those most often occurring in the developing cells, such as bone marrow, blood, kidneys and nervous system tissues.

Rayaan, Emily and Neos families lives have been turned upside down by blood cancer and disorders.

Rayaan was diagnosed with life-threatening acute myeloid leukaemia when he was just eight weeks old. AML usually requires immediate treatment and for Rayaan, bone marrow or blood stem cell transplant is his only chance of a cure.

Without a successful transplant, Rayaan will have to endure continued chemotherapy and isolation, which will expose him to the terminal effects of infection. Rayaan is now in search of an unrelated matching donor, but the low representation of diverse stem cell donors across the country and in the global registry hampers the chances of saving this courageous baby.

Arlene said watching her son endure this pain is heartbreaking and there have been some very dark days. At one point, he had to be resuscitated after a spinal lumbar puncture, but their courageous little fighter battled on and still wakes up every day with a smile on his face.

Please help my baby to live. He is just too little to suffer like this. Dont delay, you could be his perfect match, Arlene added.

Meanwhile, five-month-old Emily from Johannesburg has been battling a blood disorder following her diagnosis in November at only three months. She was diagnosed with juvenile myelomonocytic leukaemia (JMML) and is undergoing treatment, hoping a stem cell transplant will be performed soon.

As in Rayaans case, Emilys best chance at survival is a blood stem cell transplant. Dr Theo Gerdener, a clinical haematologist at Albert Alberts Stem Cell Transplant Centre and medical director at DKMS Africa, said:

JMML, which is especially prevalent in young children, is a rare cancer of the blood and occurs when white blood cells, known as monocytes and myelocytes, mature abnormally. This cancer can occur spontaneously or, sometimes, is linked to other genetic disorders.

Leukaemia affects white blood cells and bone marrow, and alarmingly, childhood leukaemia accounts for around 25% of all cancer in children. With proper diagnosis and management, including stem cell transplantation, childhood leukaemia can be cured in 85% to 90% of patients.

According to Natalie, Emilys mother, her daughter has already endured multiple blood and platelet transfusions, frequent injections and other medication, lengthy hospital stays, including isolation and ICU admission, as well as surgery to insert a port in her chest for intravenous administration.

Her parents desperately hope to find a stem cell donor match through DKMS global stem cell registry and donor centre to provide them with this one in 100 000 chance.

Were keeping positive and are hoping a match will be found for Emily. We hope she grows up, has a normal childhood and becomes a beautiful, bright young lady, said Natalie.

Neo was diagnosed with Fanconi anaemia in April 2019 at only four years old. A couple of years earlier, his older sister, who was also diagnosed with the same blood disorder, received a stem cell donor transplant, giving her a second chance at life.

Their dad, Phoebus, recalls the late-night rushes to the hospital, overnight stays and time away from work, as both parents grappled with the unusual, but persistent symptoms in their child, as Neo endured uncontrollable nosebleeds, debilitating fatigue, prominent bruising and innumerable fevers and infections owing to his compromised immune system.

Neo is transfusion-dependent and receiving steroid treatment, among other things. He is also searching for his second chance at life through an unrelated donor match to provide him with a life-saving blood stem cell transplant.

A donor with the same ethnic background as a patient may be a better match than one who comes from an entirely different background.

Globally, there are a low number of registered donors among the black, Indian and mixed-ethnic populations, meaning the pool of prospective matches is significantly lower.

For patients like Neo, a substantial increase in the registration of black donors will directly impact his chances of a successful transplant from a matched donor.

Neos family has thrown its support behind DKMS Africa to champion the cause of education and awareness around blood diseases.

Also Read:Young couple says I do with the help of generous donors

People need to be aware of these medical conditions and empower themselves with the knowledge. Some people and organisations are there to help. People should not be afraid to reach out, said Phoebus.

If you are in good health and between the ages of 18 and 55 and considering joining the registry, visit http://www.dkms-africa.org or call 0800 12 10 82, weekdays between 08:30 and 16:30.

Once you have registered online, a swab kit is sent to you via courier and then collected when you have completed the process (at no cost to you). Take action, save a life!

Also Read:Become an organ donor and help save a life

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Donors needed to save young lives - Benoni City Times

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