By Akshat Jain MD MPH, International Hematologist and Gene therapy specialist, California
In a significant leap forward for medical science, recent breakthroughs in gene therapy are ushering in a new era of treatment for various human diseases. The field of gene therapy, which involves manipulating or introducing genetic material into a person’s cells to treat or prevent disease, has seen remarkable advancements, with several gene therapy drugs receiving approval from the Food and Drug Administration (FDA). With global integration of health care and adaptation of skills through academic East-West partnerships, many such ground breaking therapies should see themselves reach the global south, if tapped into the potential of the diaspora intelligently.
Approved gene therapies by FDA
- Casgevy™ gene therapy for sickle cell disease
- LyfGenia™ gene therapy for sickle cell disease
- Elevidys™ gene therapy for Duchenne muscular dystrophy
- Leukemia and lymphoma CAR T-cell therapy (KYMRIAH™)
- Luxturna™ gene therapy for inherited retinal disorder
- Roctavian™ gene therapy for hemophilia A
- Hemgenix™ gene therapy for hemophilia B
- SKYSONA® gene therapy for cerebral adrenoleukodystrophy (CALD)
- Zolgensma® gene therapy for spinal muscular atrophy (SMA)
- Zynteglo® gene therapy for beta thalassemia
Gene therapy has emerged as a groundbreaking frontier in the treatment of genetic disorders, offering hope and transformative possibilities for individuals grappling with conditions such as sickle cell disease, thalassemia, and hemophilia. In 2024, the landscape of healthcare is witnessing remarkable advancements with FDA-approved gene therapy treatments that hold the potential to revolutionize the lives of patients around the world.
Global Accessibility: One of the significant advantages of these FDA-approved gene therapy treatments is their potential for global accessibility. Pharmaceutical companies and healthcare organizations are actively working to ensure that these groundbreaking therapies reach patients in diverse regions, transcending geographical boundaries. Collaborative efforts are underway to make these treatments available in countries where these genetic disorders are prevalent, thereby extending the benefits of gene therapy to a broader population.
Challenges and Considerations: While the strides in gene therapy are undeniably promising, challenges persist, including the need for rigorous long-term safety monitoring, addressing potential side effects, and ensuring equitable access to these innovative treatments. Additionally, the evolving landscape of gene therapy research underscores the importance of ongoing scientific inquiry and ethical considerations to optimize patient outcomes. The monumental costs associated with these approved therapies are out of reach for most patients in sub Saharan Africa and India where the bulk of the suffering patients reside.
These transformative therapies signify a shift towards personalized, targeted approaches to genetic disorders, offering not just treatment but the potential for a substantially improved quality of life for those who have long battled these challenging conditions. As the global healthcare community continues to embrace these advancements, the future holds promise for further breakthroughs and increased accessibility to gene therapy treatments for patients around the world. With global integration of health care and adaptation of skills through academic East-West partnerships many such ground breaking therapies should see themselves reach the global south.
