Groundbreaking Trial Seeks to Enhance Blood Cancer Treatment Efficacy and Safety

MINNEAPOLIS, Feb. 20, 2024 (GLOBE NEWSWIRE) — NMDPSM (formerly known as the National Marrow Donor Program), home to the most diverse blood stem cell and marrow donor registry in the world, announced the enrollment of the first patient in the groundbreaking OPTIMIZE clinical trial today. The trial is sponsored by NMDP through the Center for International Blood and Marrow Transplant Research (CIBMTR®).

The OPTIMIZE trial aims to evaluate the efficacy of a reduced dose of post-transplant cyclophosphamide (PTCy) in patients with blood cancers who have received peripheral blood stem cells from partially matched unrelated donors (MMUD). The study seeks to determine whether this lower dose of PTCy can decrease the incidence of infections in the first 100 days following a transplant while maintaining effective prevention against graft-versus-host disease (GvHD), a serious complication where the donor’s immune cells attack the recipient’s body.

PTCy has been crucial in the success of hematopoietic stem cell transplants (HCT) using mismatched donors by preventing acute and chronic GvHD. However, standard dosage PTCy is linked to significant risks of bacterial and viral infections, among other severe side effects. The OPTIMIZE trial hypothesizes that a reduced PTCy dosage could reduce these risks while still effectively guarding against GvHD.

The OPTIMIZE trial’s goals include:

  • Improve patient survival and quality of life by decreasing acute and long-term toxicities associated with standard dose PTCy.
  • Decrease relapse by enabling increased use of myeloablative conditioning (MAC) in the MMUD setting​.
  • Permit future clinical trials incorporating reduced dose PTCy in combination with other novel immune therapies to prevent GVHD and malignant disease relapse.

“We are encouraged by the OPTIMIZE trial’s potential to transform patient care in blood cancer treatment,” said Dr. Jeffery J. Auletta, Senior Vice President, Health Equity at NMDP and Chief Scientific Director, CIBMTR. “By exploring a reduced dosage of post-transplant cyclophosphamide, we aim to enhance patient survival and quality of life by minimizing the toxicity typically associated with standard dosing. This trial represents a pivotal step forward, laying the groundwork for future research using innovative strategies for preventing and treating not only graft versus host disease, but also malignant disease relapse as well as applying MMUD transplant to cure non-malignant diseases like sickle cell disease. Our commitment to improving outcomes for transplant patients drives our enthusiasm for the groundbreaking work in the OPTIMIZE trial.”

The OPTIMIZE trial is the third trial conducted by NMDP within the Donor For All initiative, which aims to close the access-to-HCT gap for patients who do not have a fully matched donor. The first two trials included:

  • 15 MMUD was a prospective, Phase II multi-center clinical trial, which assessed the safety and efficacy of MMUD (4/8-7/8) HCT using the PTCy GvHD prophylaxis backbone. The trial demonstrated the feasibility and effectiveness of HCT with an MMUD in the setting of PTCy. Remarkably, nearly half of the study participants belonged to an ethnic minority population, suggesting this approach may significantly expand access to HCT.
  • ACCESS (NCT04904588) is an NMDP-sponsored multicenter Phase II study of HCT using MMUD for peripheral blood stem cell transplant in adults and bone marrow stem cell transplant in children. This trial recently completed enrollment in its adult arms ahead of schedule and is continuing to enroll within its pediatric arm.

“I am thrilled about the initiation of the OPTIMIZE clinical trial, which represents a significant advance in blood cancer treatment by exploring the potential of reduced dose PTCy to mitigate adverse effects and ensure broader treatment inclusivity,” said Rachel J. Cook, MD, network chair and principal investigator at OHSU where the first OPTIMIZE patient was enrolled. “This trial is a step towards significantly improving patient outcomes by aiming for more personalized and less toxic treatment protocols.”

The OPTIMIZE trial is currently recruiting patients with plans to open at up to 50 centers across the U.S. For more information on enrollment, please visit

About NMDP

At NMDP, we believe each of us holds the key to curing blood cancers and disorders. As a global nonprofit leader in cell therapy, NMDP creates essential connections between researchers and supporters to inspire action and accelerate innovation to find life-saving cures. With the help of blood stem cell donors from the world’s most diverse registry and our extensive network of transplant partners, physicians, and caregivers, we’re expanding access to treatment so that every patient can receive their life-saving cell therapy. NMDP. Find cures. Save lives. 

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