Written by Dr. Taimur Mirza
The recent BBC report describes what may be the first successful treatment of Huntington’s disease using gene therapy. For decades, families and clinicians have lived with the knowledge that Huntington’s is a relentlessly progressive and incurable illness. To see data suggesting that progression can be slowed by as much as seventy-five percent over three years is a profound moment. It is a story of hope, but it is also a reminder of the responsibilities we carry as caregivers, advocates, and leaders in health care.
The therapy highlighted in the report uses viral vectors to deliver corrective DNA into brain cells. This approach aims to lessen the toxic effects of the mutant Huntingtin gene. Early trial results are encouraging, yet they are still only the beginning. We must be honest that much remains unknown. Questions about safety, durability, and long-term outcomes will take years to answer.
For patients and families, the promise is enormous. A treatment that slows decline could mean more years of walking, talking, and making decisions with clarity. It could also ease the heavy weight carried by caregivers who watch loved ones lose independence. At the same time, expectations must be managed carefully. Slowing a disease is not the same as reversing it, and advanced cases may not see the same benefit.
For ArchCare and for health systems more broadly, this development calls us to prepare. Gene therapies are not delivered like standard medications. They require surgical expertise, advanced imaging, neurorehabilitation, and close monitoring afterward. This one in particular requires open brain surgery. We must also ensure that our clinicians are trained, our protocols are updated, and our facilities are ready.
There is also the matter of equity. These treatments are often introduced to younger and healthier patients in trials, yet many of those we serve are older, poorer, or burdened by other illnesses. We cannot allow a future where only a privileged few benefit from medical breakthroughs. That means working with regulators, payers, and policymakers to build pathways that guarantee access for all.
Ethics is central to this effort. Gene therapy carries permanent consequences. Patients and families need full and clear explanations of both risks and uncertainties before making decisions. We must be sure that consent is informed, that questions are answered, and that trust is honored.
The financial realities cannot be ignored. Gene therapies are among the most expensive treatments in medicine today. The only known FDA approved treatment for gene therapy is for Duchenne’s Muscular Dystrophy and costs 2 million dollars per infusion. This treatment may cost more given the need for surgery. Sustaining access without bankrupting patients or health systems will require creative approaches to reimbursement and contracting. These conversations need to begin now, not later. CMS and insurance providers need to review the research and results and commit to paying the cost for the treatment so we can focus on rehabilitation otherwise, sadly, even if this is a cure, it will only be one for those who can afford it.
ArchCare will continue to follow this research closely. Our role is to celebrate progress while also protecting patients from unrealistic expectations. Our duty is to prepare for the future so that when breakthroughs arrive, we are ready to deliver them safely, fairly, and wisely.
Hope is powerful, but preparation gives hope its strength. This is the road ahead for us and for those we serve.
Source
BBC. Huntington’s Disease Successfully Treated for the First Time.