(Ontario, CA) – Mary Brown, President and CEO of the Sickle Cell Disease Foundation issued the following statement in response to the U.S. Food and Drug Administration’s approval of the first two gene therapies to treat sickle cell disease in patients 12 years or older:
“The Sickle Cell Disease Foundation is elated to witness the recent U.S. Food and Drug Administration approval of Casgevy and Lyfgenia, two pioneering gene therapies. These therapies hold the potential to transform lives and mark a significant milestone in the journey toward finding better and more effective treatments for sickle cell disease. As we extend our heartfelt congratulations to the scientists and medical professionals behind these innovations, we also acknowledge the countless sickle cell warriors who have participated in clinical trials and contributed to the development of these groundbreaking treatments.
“The prospect of gene therapy to address the root cause of sickle cell disease provides a glimpse into a future where those living with the debilitating blood disorder can experience a significantly improved quality of life. This approval not only provides hope for patients, their families, and the entire sickle cell community, but also marks the advent of a new era in treatment possibilities.
“As we celebrate this medical milestone, it is essential to recognize that not all patients will qualify for these potential cures. Therefore, the health care system must redouble its efforts to provide quality, specialized comprehensive care to ensure that those who may not benefit directly from gene therapies still enjoy a high quality of life. In addition, it is crucial for the health care system to acknowledge its responsibility to eliminate barriers to care and build trust, the cornerstone of any successful doctor-patient relationship, within the sickle cell community, which has endured a long history of systemic challenges and provider mistreatment. Only through such efforts can the integration and benefits of these gene therapies prove truly effective.
“The Sickle Cell Disease Foundation remains committed to supporting and advocating for the best interest of our community through this transformative period. We also look forward to collaborating with the medical community, policymakers, stakeholders, and – most importantly – patients to ensure that advancements in gene therapy translate into meaningful improvements in the lives of those living with sickle cell disease.”
About the Sickle Cell Disease Foundation
The Sickle Cell Disease Foundation (SCDF) was incorporated in 1957 and is the first and oldest Sickle Cell Disease community-based organization of its kind in the nation. As the only organization in California approved to provide Hemoglobin-Trait Counseling Services and direct the Sickle Cell Educator/Counselor Certification Training Course, SCDF provides life-enhancing education, services and programs for individuals living with Sickle Cell Disease. SCDF broadens public awareness, delivers effective advocacy initiatives, and promotes innovative therapies to ultimately find a cure. Learn more by visiting www.scdfc.org.