New bone marrow transplant method may cure sickle cell disease

A new bone marrow transplant method can help cure sickle cell disease and is more accessible than highly expensive gene therapies for the disease, researchers say.

Sickle cell disease is a genetic blood disorder, primarily occurring in Black individuals, that changes the shape and function of red blood cells, leading to severe pain, organ damage, reduced quality of life, and increased mortality.

The new transplant procedure, already available at multiple medical centers, is less costly than recently approved gene therapies for sickle cell disease, researchers reported in two separate papers.

Among 42 young adults with severe sickle cell disease who underwent the procedure, called reduced intensity haploidentical bone marrow transplantation, 95% were alive two years later and 88% were considered cured and experienced no disease-related events, according to a report published in The New England Journal of Medicine Evidence.

“Our results... are every bit as good or better than what you see with gene therapy,” study coauthor Dr. Richard Jones of Johns Hopkins University School of Medicine said in a statement.

Unlike traditional bone marrow transplants, the new method does not require marrow from a specifically matched donor to avoid triggering an immune response.

That difference makes it much easier to find an appropriate marrow donor.

“A common misconception in the medical field is that transplantation for sickle cell disease requires a perfect matched donor... which this trial and other studies have shown aren’t true,” coauthor Dr. Robert Brodsky, also of Johns Hopkins, said in a statement.

Before the transplant, patients received low doses of chemotherapy and total body irradiation. Afterward, they were treated for one year with chemotherapy to prevent the donor’s immune cells from attacking the recipient's body.

In a separate paper published in Blood Advances, the research team estimated that new gene therapies for sickle cell disease cost $2 million to $3 million, compared to less than $500,000 for a bone marrow transplant.

Furthermore, hospitalizations for bone marrow transplants average about eight days, as opposed to six-to-eight weeks for gene therapy, Brodsky said.

While many with sickle cell disease have organ damage that would make them ineligible for the high-dose chemotherapy required with gene therapy, most would be eligible for the transplant, Jones said. — Reuters