FDA approval of gene therapy for MLD

🧬 Exciting news! #FDA approved a gene therapy for metachromatic leukodystrophy (MLD). MLD is a genetic disorder that affects the metabolism of certain fats, leading to the progressive deterioration of the central and peripheral nervous systems in children.

👧Those following the news may remember this treatment’s approval in the UK last year, and the touching story of how life changing it was for a family’s younger child, but how sadly the treatment couldn’t be given to the kid’s older sibling, as the treatment has to be given very early on.

📊 A few data-related questions come to mind for me:

• Demonstrating value: these treatments are expensive, but seemingly durable. How do we get to a pricing model that is sustainable for health systems, but also values, well, saving a life!
• Ensuring safety and efficacy: these children (and probably anyone who receives cell or gene therapy) will need to be followed for 15+ years. I think passive data collection is a must here. As we expand cell/gene therapy use, these patients and their families should feel free to continue living their daily lives without much undue burden to provide active information. I hope these kids move around a lot — to go to college, to travel, to switch jobs — and don’t feel obligated to always travel back to their treatment centers. Every country should have a way to passively. review healthcare data, wherever these patients choose to go.
• Providing timely treatment: for a disease like MLD, early diagnosis and early treatment access are critical. The window to symptomatic events is very small. Early testing, and expansion of access opportunities are potentially costly, but needed to have an impact.
• Generalization of findings: In the -omics and AI age we live in, every rare metabolic or genetic disorder should be identified, with gene therapy candidates defined rapidly in silico. We have the data, we have the technology. There should be vested interest in getting more treatment options out there faster.

What’s still a problem is the cost. I’m not an economist, so I’ll let those who know better to do the risk-benefit analyses. But from a scientific perspective, we’re in a position to do so much good, and kind of in the holy grail stages of molecular biology. I am excited to witness more developments in this space. 🔬

DALL-E’s imagination of what “gene therapy” and “FDA approval” look like 🙃