Gene therapy provides hope for Huntington’s Disease patients 

When you live with an incurable neurological disease that will shorten your life or have a genetic predisposition to becoming afflicted with that disease, the emotional roller coaster looping between hope and despair is inevitable. May is Huntington’s Disease Awareness Month and an opportunity to observe that hope is currently trending well, particularly if Congress takes action to make possible therapies accessible for those who need them. 

Huntington’s is an inherited genetic disorder that causes nerve cells in the brain to break down over time. Those who have the disease can find their physical movements being affected, including difficulty maintaining balance. It can impair speech and the ability to swallow. The disease can cause psychiatric disorders. There are medications to help manage symptoms, but there is no treatment to prevent the inevitable physical and mental decline. I lost my father to Huntington’s two years ago, and I have the gene for it. 

As a result, I have been living on that emotional roller coaster. There have been clinical trials for new gene therapies that have been halted, leaving me with that punched-in-the-gut feeling. It’s easy to feel a sense of grief when you see time that you may not have slipping away without scientific progress being made to fight this awful disease. 

Now, though, is a time for hope and, yes, even optimism. Today, more than 20 companies are working on treatments for Huntington’s Disease. Many of these are gene therapies which target the cause of a disease at the genetic level, often with one dose as opposed to medicines you must keep taking over the course of a lifetime. I’m a member of the Institute for Gene Therapy Patient Advocacy Advisory Council and the discussions I’ve had with pharmaceutical companies and the Food and Drug Administration have convinced me that gene therapy will be a critical component in what I hope, for myself and others in the same situation, will be a healthy future. 

There are however significant questions about how accessible these treatments will be, especially given current Centers for Medicare & Medicaid Services CMS coverage restrictions for Alzheimer’s treatments, and even the recent prior authorization decision by United Healthcare for colonoscopies. Gene therapies require enormous investments in research and, because they are life-saving treatments that may only require one dose, they present unique challenges in valuation and reimbursement. These challenges not only impact patient access to treatments once they’re FDA approved but also the research and development environment for additional new treatments, especially treatments that aim to address an unmet medical need. 

Fortunately, there are many stakeholders in this effort, and that’s where Congress comes in. Bipartisan legislation called the Medicaid VBPs for Patients Act, or the MVP Act (H.R. 2666) has been introduced in the U.S. House of Representatives. This legislation would position value-based purchasing approaches as one solution to put these therapies within the reach of many patients. Under a value-based purchasing structure, payment for a new gene therapy would be tied to the effectiveness of the treatment and would help mitigate the considerable cost involved for health care payers, creating greater access for patients. 

The MVP Act builds also on the work already taking place by the CMS, which has established clear pathways to value-based payment arrangements for manufacturers, payers, and providers where outcome-based approaches can be implemented. 

After those periods of darkness when I felt that progress toward an effective treatment for Huntington’s wasn’t keeping pace with my sense of urgency, there is a far brighter outlook today because of the advances being made in genetic research. Breakthrough science, combined with public policymaking and a regulatory framework that support access to novel treatments, can make this roller coaster ride a little easier in the future. 

Lauren Holder is Huntington’s Disease Patient Advocate and member of the Institute for Gene Therapy Patient Advocacy Advisory Council.