Stem Cell Breakthrough Offers New Hope to Cancer Patients

By Becky Upham
Researchers have discovered a new treatment that greatly expands the universe of people who can be successful stem cell donors.

Key Takeaways
• Until now, people who needed a bone marrow or stem cell transplant had to find a 100-percent matched donor.
• An old chemotherapy drug has now made it possible for people to successfully receive transplants from partially matched donors.
• The new technique will expand access to stem cell transplants, especially among minority communities.

For some people with blood cancers who need a stem cell transplant, finding a donor who is an excellent match can mean the difference between life and death.

Unfortunately, even though there are more than 40 million potential donors in the national registry, finding a perfect match isn’t always possible, especially in underrepresented racial and ethnic groups.

But a new approach using an old chemotherapy drug, cyclophosphamide, is is opening up new possibilities for people with cancers like leukemialymphoma, and multiple myeloma.

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Researchers have found that by administering the drug several days after transplantation, people receiving blood stem cells from unrelated, partially matched donors can have survival rates comparable with those who received exactly matched cells.

The interim results from the ACCESS trial will be presented as an oral abstract on May 31 at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.

“This innovative approach can greatly expand patient access to safe and effective stem cell transplant, regardless of matching degree with the donor,” says lead coauthor Monzr M. Al Malki, MD, a hematologist and oncologist and director of the Unrelated Donor BMT program at City of Hope, a cancer research and treatment organization with locations across the United States.

That’s exciting because it means more patients will be able to receive this potentially life-extending therapy, says Dr. Al Malki.

Many Communities With Cancer Can’t Find a Stem Cell Donor Match

Donor compatibility is determined by a set of protein markers on blood cells called HLAs (human leukocyte antigens), says David Miklos, MD, a professor of medicine and chief of Stanford BMT and Cell Therapy Program at Stanford Medicine in California. Stanford was one of the medical sites of the trial, though Dr. Miklos is not a coauthor of the research.
Since stem cell transplants began over 50 years ago, the gold standard has been finding a close relative, usually a sibling. But even then, the chance that a sibling is a fully matched donor is 25 percent, and the chance of a partial sibling match is 50 percent — leaving many people without a suitable donor.
While close to 4 in 5 white people could find a registry match through the National Bone Marrow Registry, fewer than 3 in 10 Black individuals and fewer than half of Latino and Hispanic people could.
Why was an exact match needed? Anything less increased the likelihood of a graft failure, as well as graft-versus-host disease — meaning the transplanted cells attack the patient’s own, which can cause serious or even fatal complications, explains Miklos.

The Drug That Transformed Stem Cell Transplants

About a decade ago, researchers started using cyclophosphamide to destroy the parts of a person’s immune system that would reject the transplant. That breakthrough allowed researchers to not only have better outcomes in fully matched donors, it also opened the door for success.

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