Akshay Sharma, MBBS, on the Rapidly Expanding Landscape of Care for Sickle Cell Disease
The attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital discussed recently approved and still-upcoming gene therapy options for SCD.
“To have a one-time, potentially curative therapy is certainly very, very attractive—and now we don't have just 1, but we have 2 potentially curative therapies.”
Until relatively recently, hydroxyurea was the only drug on the market for patients with sickle cell disease (SCD). Over the past 10 years, however, and especially over just the past year or so, the landscape of care for SCD has rapidly expanded, with the approval of both new small molecule drugs for symptom management and the approval of 2 gene therapy products in December 2023: Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia). Notably, the gene therapy products have curative potential: they may be able to provide a durable and transformative impact on patients’ disease by addressing the root cause, and come in the form of a one-time treatment. Beyond these 2 approved gene therapy products, multiple other gene therapy approaches to treating SCD are now in clinical development, as well. In addition, allogeneic hematopoietic stem cell transplantation, another potentially curative treatment option for SCD, may soon become more accessible with transplants from haploidentical donors recently having shown success in clinical trials.
Akshay Sharma, MBBS, the attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital, spoke at a session focused on this growing landscape of care at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. After the close of the conference, CGTLive® reached out to Sharma, who spoke about autologous gene therapy options in the session, to get his insight on their place in the evolving range of treatment options for SCD. Sharma spoke about the slight, but important difference in indication between lovo-cel and exa-cel and the different ways in which these 2 therapies address the root cause of SCD. He also gave his perspective on the black box warning issued by the FDA for lovo-cel and briefly touched on 2 notable investigational gene therapies that are currently in clinical trials for SCD.
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REFERENCES
1. Sharma A, Sheehan V, Shenoy S. Tandem Thursday breakfast symposium: the evolving paradigm of sickle cell disease management. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas. Session #S-B1.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
