Robin Alderman’s son Camden suffers from a rare, inherited immune deficiency called Wiskott-Aldrich syndrome, for which gene therapy could potentially provide a cure. However, in 2022, the London-based Orchard Therapeutics halted investments in the experimental treatment for this condition, leaving families like the Aldermans struggling to find help. With 350 million people worldwide affected by rare diseases, most of which are genetic, the lack of commercial incentives for developing one-time therapies to fix faulty genes poses a significant challenge for families seeking cures for these rare disorders.
The absence of financial incentives in the field of gene therapy for rare diseases hinders the progress of potential treatments. Companies prioritize profitability, leading to a lack of development and accessibility for these innovative therapies. However, charitable organizations and patient groups are stepping in to fill the gap left by pharmaceutical companies. For example, an Italian charity has taken over the Wiskott-Aldrich treatment previously pursued by Orchard, and a foundation has helped launch a company to advance gene therapy research for various rare diseases.
For families like the Aldermans, who have been dealing with the challenges of Wiskott-Aldrich syndrome for years, the hope for gene therapy as a potential cure has been met with disappointment due to the lack of available treatments. Camden Alderman relies on daily medications and immunoglobulin infusions to manage his condition, but the promise of gene therapy could significantly improve his quality of life. Despite successful clinical trials in other countries, the lack of access to these treatments for American residents highlights the disparities in healthcare access based on geographical location.
Financial disincentives, including the high costs associated with developing and gaining approval for gene therapies for rare diseases, pose a significant barrier to advancing these treatments. The investment required for research and development, coupled with the limited number of patients affected by each rare genetic condition, makes it challenging for pharmaceutical companies to prioritize these therapies. Ultimately, the focus on shareholder profit in the biotechnology industry creates a misalignment of incentives between financial gain and potentially life-changing treatments for rare diseases.
Efforts are underway to address the challenges surrounding gene therapy for rare diseases, including the establishment of organizations and consortia dedicated to supporting the development and accessibility of these treatments. Scientists are exploring innovative approaches to gene therapy that could address multiple rare diseases simultaneously, offering new possibilities for patients and families affected by these conditions. While progress is being made in specific cases, the broader financial quandary surrounding gene therapy for rare diseases remains a significant obstacle to ensuring equitable access to potentially life-saving treatments.
