More sickle cell patients may benefit from bone marrow transplants

SAN DIEGO — For the past few days, the sickle cell community has been abuzz with news of the first-ever approved gene therapy for this devastating disease. Meanwhile, at Tuesday’s meeting of the American Society of Hematology, researchers reported on advances in bone marrow transplantation, a cheaper and more established strategy that has been proven to cure patients.

Although this approach has been around for decades, the need for patients to obtain a compatible donor and endure high doses of chemotherapy rules out transplantation as an option for the majority of patients. I did. However, in mid-term trials, sickle cell patients who received milder chemotherapy and infusions of semi-compatible cells were treated better and with less pain, with 95% of participants surviving two years post-transplant. Only 7% of recipients experienced severe reactions caused by immune cells attacking their new home.

The news comes as researchers are still processing the Food and Drug Administration’s landmark approval of two cutting-edge sickle cell therapies, one manufactured by Vertex Pharmaceuticals and CRISPR Therapeutics and one manufactured by Bluebird Bio. It was announced during the. These drugs cost $2.2 million and $3.1 million, respectively. By comparison, bone marrow transplants cost about $200,000 to $400,000 and can be performed on a wider range of patients, the study authors argue.