Center for International Blood and Marrow Transplant Research

Sickle Cell Warrior Program from The National Marrow Donor Program® (NMDP)/Be The Match® Expands Access to Bone Marrow Transplant as a Curative Treatment

Retrieved on: 
Thursday, August 31, 2023

MINNEAPOLIS, Aug. 31, 2023 (GLOBE NEWSWIRE) -- Committed to accelerating progress and expanding access to curative treatment, the National Marrow Donor Program® (NMDP)/Be The Match® is encouraging sickle cell warriors and caregivers to turn to Sickle Cell Warrior Program for free resources, support and for others to join the Be The Match® Registry to potentially save a life.

Key Points: 
  • “Sickle cell is the most common inherited blood disorder in the U.S. affecting over 100,000 people of all ages.
  • While a bone marrow transplant is a curative treatment, not enough of patients have awareness of this option, access to resources or a donor to receive a transplant,” said Amy Ronneberg, Chief Executive Officer, NMDP/Be The Match.
  • Every individual who signs up increases the potential matches for sickle cell warriors and other blood disorders for which a bone marrow transplant can be a curative treatment.
  • The free Sickle Cell Warrior Program aims to increase access to treatment through patient education, navigation, financial assistance, peer connections, and research.

Mesoblast Receives Complete Response From U.S. Food and Drug Administration for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in Children

Retrieved on: 
Friday, August 4, 2023

To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality.

Key Points: 
  • To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality.
  • This adult study is in line with our overall commercial strategy, which envisioned a sequenced progression from pediatric to adult SR-aGVHD indications.
  • Mesoblast has successfully met the pre-specified primary endpoint, prospectively agreed with FDA, of a single-arm Phase 3 trial in 54 children with SR-aGvHD.
  • It can be accessed via: https://webcast.openbriefing.com/msb-mu-2023/
    The archived webcast will be available on the Investor page of the Company’s website: www.mesoblast.com

National Marrow Donor Program® (NMDP)/Be The Match® Names Christina Melief, Ph.D., Senior Scientific Advisor

Retrieved on: 
Tuesday, July 25, 2023

MINNEAPOLIS, July 25, 2023 (GLOBE NEWSWIRE) -- Underscoring its commitment to advance the cell therapy ecosystem, providing cures so all patients thrive, the National Marrow Donor Program® (NMDP)/Be The Match® announced that Christina Melief, PhD, joined the organization as Senior Scientific Advisor.

Key Points: 
  • MINNEAPOLIS, July 25, 2023 (GLOBE NEWSWIRE) -- Underscoring its commitment to advance the cell therapy ecosystem, providing cures so all patients thrive, the National Marrow Donor Program® (NMDP)/Be The Match® announced that Christina Melief, PhD, joined the organization as Senior Scientific Advisor.
  • Prior to NMDP/Be The Match, Dr. Melief worked at Bloodworks Bio, a cord blood and cellular material provider at Bloodworks Northwest.
  • Find more about cord blood unit sourcing for cell and gene therapies on the Be The Match BioTherapies website.
  • The National Marrow Donor Program® (NMDP)/Be The Match® is the leading global partner working to save lives through cellular therapy.

Ossium Health Secures Series C to Expand Bone Marrow Bank, Advance Clinical Trials to Treat Life-Threatening Blood and Immune Diseases

Retrieved on: 
Tuesday, July 25, 2023

Ossium Health , a bioengineering company developing the world's first bank of on-demand bone marrow, today announced a $52 million Series C funding round.

Key Points: 
  • Ossium Health , a bioengineering company developing the world's first bank of on-demand bone marrow, today announced a $52 million Series C funding round.
  • For decades, bone marrow transplants have been used to successfully treat diseases such as leukemias, lymphomas, aplastic anemia, and immune deficiency disorders.
  • Nearly half of the 18,000 patients per year who search for a bone marrow donor do not receive a transplant.
  • Ossium has unlocked a new source of stem cells by processing bone marrow from organ donors at scale.

Akari Therapeutics Provides Updates on Development of Nomacopan in Pediatric and Adult HSCT-TMA

Retrieved on: 
Thursday, July 13, 2023

NEW YORK and LONDON, July 13, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced updates in the development programs investigating nomacopan in pediatric and adult hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).

Key Points: 
  • NEW YORK and LONDON, July 13, 2023 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, today announced updates in the development programs investigating nomacopan in pediatric and adult hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).
  • Akari also is planning to move forward into a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA and to open enrollment in 2024.
  • Akari is advancing into Phase 3 open-label single-arm study of nomacopan in pediatric HSCT-TMA and is planning to move into a Phase 3 double-blind placebo-controlled study of nomacopan in adult HSCT-TMA in 2023 and 2024, respectively.
  • Orphan Drug, Fast Track, and Rare Pediatric Disease designations have been granted by the FDA for nomacopan in pediatric HSCT-TMA.

Kite’s Tecartus® CAR T-cell Therapy Demonstrates 78% Complete Response Rate and 90% Overall Response Rate in Largest Real-World Evidence Analysis for Relapsed/Refractory Mantle Cell Lymphoma

Retrieved on: 
Tuesday, June 6, 2023

Higher CR was seen when Tecartus was given as second-and-third-line compared to later lines of treatment.

Key Points: 
  • Higher CR was seen when Tecartus was given as second-and-third-line compared to later lines of treatment.
  • The data are being presented orally today at the 2023 American Society of Clinical Oncology Annual Meeting (ASCO) (Abstract #7507).
  • Patients had a median of four lines of prior therapy; prior to infusion, 87% were BTKi-exposed, 56% received bendamustine, and 30% received autoHCT.
  • Median time from leukapheresis to infusion (also referred to as vein-to-vein time) was 28 days, during which time 46% received bridging therapy.

Results of Study on COVID-19 Vaccine Response in Hematopoietic Cell Transplant Patients Published in eClinicalMedicine

Retrieved on: 
Wednesday, May 24, 2023

“Nonetheless, vaccine responses remained lower than observed in non-immunocompromised patients, underscoring the need for other prevention and treatment strategies.”

Key Points: 
  • “Nonetheless, vaccine responses remained lower than observed in non-immunocompromised patients, underscoring the need for other prevention and treatment strategies.”
    “Vaccination continues to be important following hematopoietic stem cell transplantation (HCT) due to the higher risk of severe COVID-19 infection in these vulnerable patients.
  • “Study results confirm recommendations from the Centers for Disease Prevention and Control for initiating SARS-CoV-2 vaccination early after allogeneic HCT.
  • The results show similar effectiveness in those vaccinated less than four months versus at or after four months post-treatment.
  • The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) conducts rigorous multi-institutional clinical trials of high scientific merit, focused on improving survival for patients undergoing hematopoietic cell transplantation and/or receiving cellular therapies.

Data from Across Incyte’s Oncology Portfolio Accepted for Presentation at the 2023 ASCO Annual Meeting and EHA2023 Hybrid Congress

Retrieved on: 
Thursday, May 25, 2023

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Key Points: 
  • ET)
    Phase 1b, Open-Label Study of Add-On Therapy with CK0804 in Participants with Myelofibrosis, with Suboptimal Response to Ruxolitinib (Abstract #TPS7087.
  • ET)1
    A Phase 1/2 Study of Retifanlimab (INCMGA00012, Anti–PD-1), INCAGN02385 (Anti–LAG-3), and INCAGN02390 (Anti–TIM-3) Combination Therapy in Patients (Pts) with Advanced Solid Tumors (Abstract #2599.
  • ET)
    Abstracts are available on the EHA2023 Congress platform and accessible for on-demand viewing until August 15, 2023.
  • ET)5
    For full session details and data presentation listings, please see the ASCO ( https://conferences.asco.org ) and EHA2023 ( https://ehaweb.org/congress ) online programs.

The National Marrow Donor Program® (NMDP)/Be The Match® and Be The Match BioTherapies® Mark Clinical Trials Day With New Resources and Services for Patients, Researchers, and Industry Sponsors

Retrieved on: 
Wednesday, May 17, 2023

The new resources are designed to complement and raise awareness about their breadth of services for patients, researchers, and trial sponsors.

Key Points: 
  • The new resources are designed to complement and raise awareness about their breadth of services for patients, researchers, and trial sponsors.
  • Finally, by supporting expanded clinical trial access, the NMDP/Be The Match, is working to ensure that enrollment in clinical trials reflects the diversity of the whole patient population.
  • The NMDP/Be The Match simplifies access to clinical trials for patients, their families, and care teams through navigational and financial support.
  • The National Marrow Donor Program® (NMDP)/Be The Match® is the leading global partner working to save lives through cellular therapy.

Results of Study on Cryopreserved Hematopoietic Stem Cell Grafts By CIBMTR Published in Blood Advances

Retrieved on: 
Tuesday, April 18, 2023

MINNEAPOLIS, April 18, 2023 (GLOBE NEWSWIRE) -- CIBMTR® ( Center for International Blood and Marrow Transplant Research® ) announced that the results of a multi-center observational study, around the impact of cryopreserved hematopoietic stem cell (HSC) grafts on patient survival rates were published in Blood Advances , a peer-reviewed open access medical journal published by the American Society of Hematology.

Key Points: 
  • MINNEAPOLIS, April 18, 2023 (GLOBE NEWSWIRE) -- CIBMTR® ( Center for International Blood and Marrow Transplant Research® ) announced that the results of a multi-center observational study, around the impact of cryopreserved hematopoietic stem cell (HSC) grafts on patient survival rates were published in Blood Advances , a peer-reviewed open access medical journal published by the American Society of Hematology.
  • The study showed that the shift in clinical practice to cryopreserved products necessitated during the pandemic did not adversely impact one-year overall survival.
  • The study subjects were US patients receiving fresh (March-August 2019) or cryopreserved (March-August 2020) bone marrow or peripheral blood stem cell transplants from matched related or unrelated donors.
  • One advantage observed with cryopreserved grafts was a decreased risk of chronic GVHD consistent with results previously described in a single center study published by Dana Farber Cancer Institute.