FDA approves gene therapy for sickle cell disease: 'One incredible journey of success.'

Originally from USA Today by Karen Weintraub

Federal officials have granted approval for two gene therapy treatments for sickle cell disease, offering hope to tens of thousands afflicted by the painful and life-threatening blood disorder. Sickle cell, the most common disease to receive gene therapy approval, will initially see limited availability for patients aged 12 and older with the most severe form, estimated to be around 20,000 of the 100,000 affected Americans.

The therapies, although expensive, mark a significant breakthrough, providing a viable option for those living with the unpredictable pain crises of sickle cell disease. Individuals like Jennelle Stephenson, who underwent gene therapy six years ago, report a life-changing reduction in symptoms. While not declared cured, 95% of recipients, including Stephenson, experience freedom from symptoms.

Sickle cell disease results from a genetic disorder affecting the oxygen-carrying ability of red blood cells, causing pain crises triggered by routine activities. The approved gene therapies involve a complex process of modifying bone marrow cells, with patients undergoing chemotherapy to replace unhealthy cells with modified, healthy ones.

Despite the transformative potential, gene therapy remains an ordeal, requiring careful monitoring after the procedure. The FDA's approval opens new possibilities for patients previously limited to treatments with varying efficacy.

The approved gene therapies, CASGEVY and LYFGENIA, priced at $2.2 million and $3.1 million, respectively, employ different technologies but share the goal of addressing the genetic roots of sickle cell disease. The potential impact on patients' lives is immense, with gene therapy offering prevention of future damage.

While uncertainties remain about the broader adoption of gene therapy, researchers aim to refine the approach for wider application. Dr. Stuart Orkin envisions developing less challenging and expensive treatments, exploring alternatives to chemotherapy and streamlining the genetic modification process.

The approval of gene therapy for sickle cell disease not only brings hope to those affected but also raises awareness about the underfunded and often mistreated condition. Patients like Jennelle Stephenson and Victoria Gray, who have experienced life-changing results, highlight the potential for gene therapy to transform the lives of those grappling with sickle cell disease.

Citations:
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FDA approves gene therapy for sickle cell disease: “One incredible journey of success.” (n.d.). USA TODAY. https://www.usatoday.com/story/news/health/2023/12/08/fda-approves-sickle-cell-gene-therapy/71672233007/

Photo by Fayette Reynolds M.S.: https://www.pexels.com/photo/cell-seen-under-microscope-11198507/