Advisory Body Backs MesoBlast Therapy for Transplant Complication – Xconomy
By daniellenierenberg
XconomyNew York
An FDA advisory committee Thursday voted 9-1 to recommend that the agency approve a stem cell therapy developed by MesoBlast as a treatment for acute graft-versus-host disease (aGVHD) in children. The panel weighed the need for a new way to address the potentially fatal condition against shortcomings of the clinical trial the Australian biotech conducted to evaluate the investigational cell therapy.
Shares in (NASDAQ: MESO), which fell more than 30 percent earlier in the week after the FDA released briefing documents ahead of the committees meeting, closed up 51 percent Friday at $17.88 apiece compared to $11.81 at market close Wednesday. (Trading was halted on Thursday.)
The MesoBlast cell therapy, remestemcel-L (Ryoncil), is made from mesenchymal cells taken from healthy donors. The properties of these cells, which dont prompt an immune reaction, allow them to be used as an off-the-shelf treatment without accompanying immunosuppressants that put patients at greater risk for infection, among other side effects.
Some patients with blood cancer are treated with a stem cell transplant, in which cells from a healthy donor are infused into their bloodstream with the intent that those cells will travel to the bone marrow and form new healthy blood cells. Frequently, however, when donors are unrelated, their cells identify the recipients as foreign, prompting them to attack organs and tissues. Treatment with systemic corticosteroids can help control the severity of the condition. But in up to 90 percent of aGVHD patients who dont respond to steroid treatment, the condition can prove fatal.
MesoBlasts submission was based on a clinical trial that enrolled 55 children age 2 months to 17 years who had received a transplant of bone marrow, peripheral blood stem cells, or cord blood, were diagnosed with aGVDH and werent responding to steroid therapy. The study tracked their responses to remestemcel on day 28. Overall, 70 percent of the patients responded, including 76 percent of the 25 patients whose condition was graded as most severe. On day 100, 74 percent of patients remained alive; on day 180, 69 percent.
However, the trial was neither randomized nor controlled, raising concerns of confounding factors and potential bias. MesoBlast said investigators werent willing to enroll children in such a trial. But the company said randomization and controlled design would be incorporated into a planned post-marketing study in adults.
In previous clinical trials in wider patient populations, the treatment missed the primary goal. Analyses of results from those earlier trials prompted MesoBlasts decision to focus the drugs further development to steroid-refractory pediatric patients. In its presentation to the advisory panel the company said the remestemcel manufacturing process has been improved since those trials in ways that have made the treatment more potent.
The panel voted on whether MesoBlast provided enough clinical data to show that its therapy was effective in treating aGVHD in this narrower group. Panelist Christian Hinrichs, a clinical researcher at the National Cancer Institute and physician by training, was the sole no vote. Nine panelists felt the available data did indicate efficacy. (The committee recorded the tally as 8-2, but a MesoBlast representative said one no vote was made in error.)
I do think that the two prior randomized trials convincingly show that the [earlier version of remestemcel], at least in the population that was being studied, which is similar but not the same, clearly did not have meaningful activity, Hinrichs said. So, you know, do we think that these tweaks to the manufacturing have suddenly made it highly effective, and the change in patient population has suddenly made it highly effective?
Jorge Garcia, division chief of solid tumor oncology at University Hospitals Seidman Cancer Center in Cleveland, however, said while it isnt clear how the treatment compares to other drugs used to treat patients with the condition, the data indicate it is safe and has shown some efficacy.
In May 2019 an Incyte (NASDAQ: INCY) treatment, ruxolitinib (Jakafi), became the first FDA-approved treatment for patients with aGVHD who didnt responded to steroid therapy. Ruxolitinib was OKd for patients starting at age 12. But no treatment is approved for those younger.
Although the FDA considers advisory panel recommendations during drug reviews, committee recommendations are not binding, and the agency doesnt always follow them. Its decision on remestemcel is anticipated by Septembers end.
Image: iStock/Yarygin
Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.
Continued here:
Advisory Body Backs MesoBlast Therapy for Transplant Complication - Xconomy
- 5 year old boy from Iraq undergoes world's first of its kind bone marrow transplantation in India - The Times of India - March 31st, 2025
- Superior outcomes of haploidentical hematopoietic cell transplantation over chemotherapy in AML patients 55 years or older - Nature.com - March 31st, 2025
- Blood and Marrow Transplantation and Cellular Therapy Center - NYU Langone Health - March 22nd, 2025
- Bone Marrow Transplantation Market Poised to Achieve USD 14,336 Billion by 2031 - Persistence Market Research - openPR - March 22nd, 2025
- BioRestorative Therapies to Report 2024 Financial Results and Host Conference Call on March 27, 2025 - The Manila Times - March 22nd, 2025
- Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy - Forbes - March 22nd, 2025
- Boost in cancer treatment: PGI working on lab for stem cell, gene therapies - The Times of India - March 22nd, 2025
- Clonal dynamics and somatic evolution of haematopoiesis in mouse - Nature.com - March 11th, 2025
- Mitochondria-enriched hematopoietic stem cells exhibit elevated self-renewal capabilities, thriving within the context of aged bone marrow -... - March 11th, 2025
- TET2 deficiency increases the competitive advantage of hematopoietic stem and progenitor cells through upregulation of thrombopoietin receptor... - March 11th, 2025
- Garuda Therapeutics Closes $50 Million Series A-1 Financing to Advance Off-the-Shelf Blood Stem Cell Therapies - GlobeNewswire - March 11th, 2025
- Abu Dhabi Stem Cells Center and Yas Clinic first in UAE to receive AABB accreditation for haematopoietic progenitor cell collection - Abu Dhabi Media... - March 11th, 2025
- Discovery of lung-based blood stem cells may transform transplant therapies - Medical Xpress - March 1st, 2025
- VUMC part of new study validating curative therapy for sickle cell disease - VUMC Reporter - March 1st, 2025
- What Is the Role of MRD Testing Before HSCT in MDS/MPN? - DocWire News - March 1st, 2025
- Melphalan-based conditioning with post-transplant cyclophosphamide for peripheral blood stem cell transplantation: donor effect - Nature.com - March 1st, 2025
- Mesenchymal Stem Cells Market Projected to Reach USD 11.26 Billion by 2034, Growing at a CAGR of 12.9% - openPR - March 1st, 2025
- See snazzy slugs in all their luminous glory Februarys best science images - Nature.com - March 1st, 2025
- On the origin of neutrophils - Nature.com - March 1st, 2025
- Four-year-old donates stem cells to save her baby sister from blood cancer in Odisha - The Hindu - February 20th, 2025
- Effect of pre-transplant cytoreductive therapy on the outcomes of patients with MDS or secondary AML evolving from MDS undergoing allo-HSCT: a... - February 20th, 2025
- A heart disease trigger that lurks inside bone marrow - Harvard Health - February 20th, 2025
- 4-year-old donates stem cells to save sister as SCB performs first-of-a-kind bone marrow transplant in Odisha - OTV News - February 20th, 2025
- KU Cancer Center recognized for transplant that saved 1-year-olds life - WDAF FOX4 Kansas City - February 20th, 2025
- Orca-T With RIC Is Safe in Advanced Hematologic Malignancies - OncLive - February 20th, 2025
- SCB conducts Odisha's first bone marrow transplant on two-year-old - The New Indian Express - February 20th, 2025
- Bahrain's pioneering use of sickle cell disease treatment hailed by medical experts - The National - February 20th, 2025
- Cancer survivor is the first monumental bone marrow transplant patient in Baton Rouge in 8 years - NOLA.com - February 11th, 2025
- Autologous Cell Therapy Market to Hit Valuation of US$ 44.55 Billion By 2033 | Astute Analytica - GlobeNewswire - February 11th, 2025
- Nanoparticle that cuts middlemen could improve stem cell therapy - Futurity: Research News - January 31st, 2025
- GATA2 mutated allele specific expression is associated with a hyporesponsive state of HSC in GATA2 deficiency syndrome - Nature.com - January 31st, 2025
- Coordinated immune networks in leukemia bone marrow microenvironments distinguish response to cellular therapy - Science - January 31st, 2025
- How the bone marrow microbiome responds to immunotherapy - Chemical & Engineering News - January 31st, 2025
- My Experience With Stem Cell Therapy: Snake Oil or Silver Bullet? - GearJunkie - January 31st, 2025
- Hematopoietic Stem Cell Transplantation - StatPearls - NCBI ... - January 22nd, 2025
- Doctors retrieve stem cells from 20-month-old to treat thalassaemic sister - The Times of India - January 22nd, 2025
- YolTech Therapeutics to Initiate a Clinical Trial for YOLT-204, a First-in-Class Bone Marrow-Targeted In Vivo Gene Editing Therapy for -Thalassemia -... - January 22nd, 2025
- School of Medicine professor receives grant to study improved cancer treatments - Mercer University - January 14th, 2025
- 1st stem cell therapy, new HIV drug approved - ecns - January 5th, 2025
- Suppression of thrombospondin-1mediated inflammaging prolongs hematopoietic health span - Science - January 5th, 2025
- A pilot raced through the airport to surprise an old friend: the woman who saved his life - CNN - December 27th, 2024
- Types of Stem Cell and Bone Marrow Transplants - December 27th, 2024
- Explained: What is mesenchymal stem cell therapy? - Drug Discovery News - December 18th, 2024
- Stem Cell Transplants Offer New Hope for Saving the Worlds Corals - Technology Networks - December 18th, 2024
- Scientists Present Research on Novel Cancer Therapies at ASH - City of Hope - December 18th, 2024
- Navigating CAR-T cell therapy long-term complications - Nature.com - December 18th, 2024
- High-dose chemotherapy followed by autologous stem cell transplant ineffective for patients with mantle cell lymphoma - News-Medical.Net - December 18th, 2024
- Stem Cell Therapy Market Is Expected To Reach Revenue Of - GlobeNewswire - December 18th, 2024
- The Importance of Cellular Therapy in the Clinical Case of a Young Man With a Challenging Precursor B-cell Lymphoblastic Leukemia - Cureus - December 18th, 2024
- A search for the perfect match, Apex six year old in need of donor - CBS17.com - December 18th, 2024
- New insights into survival of breast cancer cells in the bone marrow - News-Medical.Net - December 9th, 2024
- Cellular trafficking and fate mapping of cells within the nervous system after in utero hematopoietic cell transplantation - Nature.com - December 9th, 2024
- Saving lives, one stem cell at a time - Texas A&M The Battalion - December 9th, 2024
- Turn Biotechnologies Announces Landmark Study to Assess Effectiveness of ERA Therapy in Restoring Bone Marrow - PR Newswire UK - December 9th, 2024
- Orca Bio Presents Three-Year Survival Data with Orca-T in Patients with Hematological Malignancies at the 66th ASH Annual Meeting - Yahoo Finance - December 9th, 2024
- You are the match. How UNC student honored her late grandfather with life-saving effort - Raleigh News & Observer - November 29th, 2024
- scRNA-seq revealed transcriptional signatures of human umbilical cord primitive stem cells and their germ lineage origin regulated by imprinted genes... - November 29th, 2024
- Atlanta pilot with an aggressive cancer finds lifesaving help from a stranger and a simple test - The Atlanta Journal Constitution - November 29th, 2024
- Researchers have brought the promise of stem cell therapies closer to reality - The Week - November 29th, 2024
- Bone Marrow Donors Can Be Hard to Find. One Company Is Turning to ... - November 15th, 2024
- Hematopoietic Stem Cells and Their Niche in Bone Marrow - November 15th, 2024
- Bone Marrow Transplant Program - Overview - Mayo Clinic - November 15th, 2024
- Bone Marrow Donors Can Be Hard to Find. One Company Is Turning to Cadavers - WIRED - November 15th, 2024
- More stem cells for sickle cell gene therapy readied with motixafortide - Sickle Cell Disease News - November 15th, 2024
- Skull bone marrow expands throughout life and remains healthy during aging, researchers discover - Medical Xpress - November 15th, 2024
- Adult skull bone marrow is an expanding and resilient haematopoietic reservoir - Nature.com - November 15th, 2024
- Evaluation of standard fludarabine dosing and corresponding exposures in infants and young children undergoing hematopoietic cell transplantation -... - November 15th, 2024
- Stem cells grown in space show super powers but theres a catch - Study Finds - November 15th, 2024
- Getting a Stem Cell or Bone Marrow Transplant - October 21st, 2024
- Acquisition of durable insulin-producing cells from human adipose tissue-derived mesenchymal stem cells as a foundation for cell- based therapy of... - October 21st, 2024
- 1.5 Lakh Indians Register To Save Lives: Join the Mission To Fight Blood Cancer - The Better India - October 21st, 2024
- How Stem Cell and Bone Marrow Transplants Are Used to Treat Cancer - October 13th, 2024
- Stem Cell (Bone Marrow) Transplants - MD Anderson Cancer Center - October 13th, 2024
- Donating Bone Marrow and Stem Cells: The Process and What To Expect - October 13th, 2024
- What to expect as a stem cell or bone marrow donor - October 13th, 2024
- Structural organization of the bone marrow and its role in ... - October 13th, 2024
- Stem cell donor from down the road saved my life after global search - BBC.com - September 23rd, 2024
- Awaiting the call: family hopes to find blood stem cell donor - Claremont Courier - September 23rd, 2024
- Michigan woman one of first in world to successfully receive bone marrow from deceased donor - WDIV ClickOnDetroit - September 23rd, 2024
- Next-generation stem cell transplant: Revolutionizing a lifesaving cancer therapy - The Business Journals - September 23rd, 2024
