Original published date: 8/29/2024

When Amelia was just 8 years old, her mom, Salome, noticed bruises and bleeding on Amelia’s body. As a doctor, Salome immediately knew something was wrong. After extensive evaluation, doctors told Amelia’s family she had myelodysplastic syndromes (MDS) along with high-risk acute myeloid leukemia (AML). Both blood cancers can be fatal.

To save her life, Amelia needed a blood stem cell transplant using cells from a donor. But Amelia is half Indian and half African American. Finding a fully matched donor would be next to impossible. That’s because people are most likely to match a donor of their own ethnic background. And donors with ethnically diverse ancestries are underrepresented on the NMDP Registry^SM.

In the past, not having a fully matched donor meant Amelia likely couldn’t have a transplant, or, if she did, her outcome could be poor.

That’s all changed because of new, innovative NMDP^SM-led research that gives patients like Amelia a chance to receive a potentially life-saving cure.

When searching for a blood stem cell donor for a patient, doctors use markers people inherit from their parents—called human leukocyte antigens (HLA)—to match the patient with a suitable donor. For decades, having a full HLA-matched donor was critical for a patient to have the best chance to survive after transplant.

That’s a problem because many patients don’t have fully matched donors on international registries like the NMDP Registry. This is especially true for patients who have ethnically diverse ancestries. For patients like Amelia who are multiracial, finding a fully matched donor is even harder because they have uncommon HLA types.

More potential donors are always needed for the NMDP Registry to improve all patients’ odds of finding a suitable match. But registry recruitment alone won’t solve the problem. Science will.

Amelia’s doctors enrolled her in a clinical trial called ACCESS. That trial is part of our NMDP Donor for All initiative, which aims to unlock access to treatment for more patients by using partially matched donors—all while providing good quality of life and outcomes comparable to transplants using a fully matched donor.

And it’s working.

The ACCESS trial builds upon a smaller study known as 15-MMUD. In that study, adults with blood cancer received bone marrow transplants from partially matched donors and a drug called post-transplant cyclophosphamide (PTCy).

PTCy has become a cornerstone in preventing a serious complication after transplant called graft-versus-host disease (GVHD). When a patient has GVHD, the donor’s cells (graft) attack the patient’s healthy cells (host). It can last for years and can be life-threatening for some patients.

The results from the 15-MMUD clinical trial exceeded expectations. Outcomes up to three years after transplant were similar to historical outcomes for fully matched transplants. Nearly 50% of the patients enrolled had ethnically diverse ancestries. Typical enrollment in transplant clinical trials has been less than 20% for those with ethnically diverse ancestry.

The ACCESS clinical trial includes both adults and pediatric patients who receive partially matched blood stem cells from a donor and PTCy. Adult patients enrolled in the trial receive blood stem cells collected through the more common peripheral blood stem cells (PBSC) method. Pediatric patients like Amelia receive bone marrow.

The adult groups of the clinical trial have completed enrollment while the pediatric group continues to enroll patients. More than 50% of patients enrolled in the ACCESS clinical trial have ethnically diverse ancestries.

Early results from a portion of adult patients presented at the 2024 American Society of Clinical Oncology (ASCO) annual meeting show excellent survival rates; very good GVHD-free, relapse-free survival; and low rates of life-threatening GVHD.

"Our research findings advance our ability to offer more options to patients without a fully matched donor, many of whom are ethnically diverse and have been underserved in receiving potentially life-saving cell therapy," said Steven Devine, MD, chief medical officer at NMDP and senior scientific director at CIBMTR® (Center for International Blood and Marrow Transplant Research®). CIBMTR a is research collaboration between the Medical College of Wisconsin® and NMDP.

While the results are encouraging, our work is ongoing. The OPTIMIZE clinical trial began enrolling patients in late 2023. The clinical trial is studying whether a reduced dose of PTCy will improve infection-free survival while still preventing GVHD. All three clinical trials have been sponsored by NMDP and conducted by CIBMTR.

For Amelia and her family, the ACCESS clinical trial was a lifeline. In June 2022, she received her life-saving transplant using blood stem cells from a partially matched donor.

While there were some bumps in the road on the way to recovery, including GVHD, Amelia is doing well today. She’s back in school, loves playing outside with friends, and enjoys drawing and painting. Her future—once uncertain—is bright again thanks to advances in science.

Through the Donor for All initiative and clinical trials like ACCESS, Dr. Devine says, “We’re showing that science can solve the gap in equitable access to transplant, giving new hope to patients worldwide.”