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Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD).

The therapy, nonmyeloablative haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy), is proving to have equivalent efficacy and one-fifth the cost as recently FDA-approved myeloablative gene therapy options, according to Michael DeBaun, MD, MPH, director of the Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease.

Results from the 10-year trial, published June 20 in the American Society of Hematology journal Blood, show a two-year overall survival of 94.1% with no difference between children and adults.

DeBaun and Adetola Kassim, MD, professor of Medicine and clinical director of the Adult Stem Cell Transplant Program at VUMC, led the international trial that included 32 children and 38 adults from five countries.

The team developed the protocol for the newest treatment.

"We are introducing another curative option for SCD treatment that is just as safe and more affordable than the recently FDA-approved myeloablative gene therapy and gene editing trials," said DeBaun, vice chair of Clinical Research and professor of Pediatrics at Monroe Carell Jr. Children's Hospital at Vanderbilt. "It's imperative that physicians and families are aware of our findings so that they can make decisions about the best personalized curative therapy option.

"One of the most significant advantages of our curative therapy approach -; most transplant centers in the United States and the world can provide this life-sustaining treatment tomorrow because they are very familiar with the now routine haploidentical transplant protocol with PTCy."

Until now, there were few curative options presented to adults with SCD because of the few full-matched donors and the ability of most adults with SCD to tolerate myeloablative treatment before transplant.

The haploidentical transplant protocol only requires a half match, which can be from the mother, father, siblings, and even children, and is nonmyeloablative, an approach that typically preserves fertility.

In the recently completed trial, 90% of the potential participants had an available donor, according to study investigators, greatly improving access to lifesaving therapy