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A groundbreaking gene therapy has demonstrated a 100% survival rate for children suffering from adenosine deaminase-severe combined immunodeficiency (ADA-SCID), a rare genetic disorder that compromises the immune system and can be fatal. The treatment, detailed in a recent study published in *The New England Journal of Medicine*, has successfully saved the lives of 62 children diagnosed with this condition, offering new hope for families facing this life-threatening diagnosis.
ADA-SCID affects the body’s ability to fight infections, leaving many afflicted children vulnerable to everyday germs. Without intervention, the prognosis is dire, with many children not surviving past the age of two. The innovative gene therapy works by extracting blood stem cells from the patient, inserting a healthy copy of the faulty ADA gene using a lentivirus, and reinfusing the modified cells back into the body. This single-dose treatment has shown remarkable results, with patients followed for over a decade remaining healthy and thriving.
Among the successful recipients is Eliana Nachem, who received the therapy ten years ago. Prior to treatment, her family was forced into isolation to protect her from infections. Now, Eliana actively participates in school activities, including playing basketball and singing in the choir.
The therapy represents a significant advancement over previous treatments, such as weekly enzyme injections and bone marrow transplants, which carry higher risks and require ongoing care. The new approach also allows for the freezing and shipping of modified cells, providing a pathway for global access to this potentially life-saving treatment.
However, the cost of the therapy poses a significant challenge, with estimates reaching up to one million euros per patient. Although the price is steep, advocates point out that traditional enzyme therapies are also costly and require long-term administration. In contrast, the gene therapy offers a “one and done” solution that could be more economical over time.
Despite the promising results, concerns surrounding the commercialization and accessibility of gene therapies for rare diseases persist. Previous gene therapies, such as Strimvelis, faced financial challenges leading to their discontinuation. Safety remains a top priority, and while earlier therapies have encountered complications, the new lentivirus-based method has not shown similar adverse effects so far.
To address these challenges, researchers are working to develop scalable production methods and sustainable pricing models. A recent $14.7 million grant from the California Institute for Regenerative Medicine aims to enhance the accessibility of this innovative therapy.
As families and researchers continue to navigate the complexities of rare disease treatments, the success of this gene therapy serves as a beacon of hope, illustrating the potential for scientific advancement to transform lives. With ongoing research and commitment, the future holds promise for many more children facing similar health challenges.
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