Rédaction Africa Links 24 with Megan Molteni
Published on 2024-02-05 15:41:41
Afghan Family’s Struggle Pays Off as Texas Approves Life-Saving Gene Therapy
A long and arduous battle with Texas Medicaid has finally come to an end for an Afghan family who fought for coverage of a life-saving gene therapy. After an agonizing process, the family received the news they had been praying for: Texas officials have agreed to pay for the costly treatment, providing hope for their infant son. The family learned late last week that doctors at M Health Fairview, a research hospital in Minneapolis, are preparing to attempt a groundbreaking experimental gene therapy to save their 4-and-a-half-month-old son, Sufyan.
The family’s journey began with a devastating diagnosis. Mohammad, 3 years old, was diagnosed with metachromatic leukodystrophy (MLD), an ultra-rare genetic disorder that attacks the central nervous system. Subsequently, a DNA test revealed that his younger brother, Sufyan, also carried the same disease-causing mutation. The family was then presented with a glimmer of hope when Orchard Therapeutics offered to provide its multimillion-dollar MLD treatment, Libmeldy, free of charge. Specialists in Minnesota believed that Sufyan was a viable candidate for the gene therapy due to the absence of symptoms. However, the family faced a daunting challenge: securing funding for the associated procedures, which could amount to around $300,000.
The family and the medical team at M Health Fairview encountered numerous obstacles as they navigated the complex process of seeking coverage from Texas Medicaid. A prior denial to pay for an initial consultation was overturned after a story on the family’s appeal was published. However, further negotiations led to delays as Texas Medicaid hesitated to recognize the gene therapy and its associated procedures as a medical necessity for Sufyan.
The long-awaited breakthrough finally arrived when an agreement was reached between Paul Orchard, a pediatric hematologist at M Health Fairview, and Texas Medicaid on January 30. The family is now making preparations to travel to Minneapolis for Sufyan to undergo the necessary assessments and procedures. Now, Sufyan’s path to recovery will involve a multi-day procedure to collect his blood stem cells, which will undergo genetic engineering to express a functional copy of the gene necessary to clear toxins from his central nervous system. The family has even set up a GoFundMe to help with travel costs, reflecting the tremendous support and solidarity they have received.
While the family celebrates this milestone, the exorbitant cost of gene therapy poses broader concerns for the healthcare industry. The treatment’s price, ranging from $2.6 to $3.5 million in Europe, could strain the market structure, creating potential equity issues that could impact access to life-saving treatments. The overall affordability and accessibility of gene therapy for patients with public insurance are among the pressing concerns for medical professionals like Orchard.
As the family eagerly looks ahead to Sufyan’s treatment in Minneapolis, there is newfound optimism for his recovery and a sense of relief after a prolonged battle. The Pashais hope that Sufyan’s successful treatment will pave the way for a brighter future for other families facing similar challenges. This heartwarming victory highlights the resilience and tenacity needed to overcome obstacles and secure life-saving treatments for those in need.
