I Want to Be Hopeful About New Research but I’m Still Wishing for a Cure
This week, I read two articles that were sent to me describing scientific breakthroughs regarding possible treatments for Huntington’s.
The first described a case study recently published in the journal Psychiatry and Clinical Neurosciences. It details how Rexulti (brexpiprazole), an antipsychotic medicine indicated for schizophrenia and in some cases, depression, was shown to greatly decrease one person’s symptoms caused by Huntington’s.
While Rexulti is not currently indicated as a treatment for Huntington’s, I am not surprised that therapies already on the market for other diseases could benefit Huntington’s patients. It’s commonplace for some therapies to be used in multiple indications, such as cancer treatments used to treat multiple sclerosis.
However, this case study described only one person, and it’s possible that a larger study might not obtain the same results. On the other hand, if a larger study did produce positive results, it would be a great discovery for treating symptoms and giving us a bit of our functional time back.
Nevertheless, that’s not what I’m holding out hope for. I am hoping for a cure. I want to live a longer life without Huntington’s looming over me.
The other article describes a step toward that goal. Researchers at Massachusetts Institute of Technology have discovered new genes to potentially target with therapy, including a family of genes that could help break down the Huntington’s protein. That would be monumental. If we could prevent the Huntington’s protein from damaging the brain, there wouldn’t be symptoms of the disease to treat.
While this is an interesting development, the research has only been conducted with mice. And the mouse-to-human conversion in studies is far from perfect. One review noted that only 8 percent of cancer treatment studies translated successfully from animals to humans.
I really wish I could look at both of these articles and feel hopeful, but my hope is tempered by all of the things I’ve learned over the years. For example, the chance of an investigational therapy in rare diseases moving from Phase 2 to Phase 3 in clinical trials is only about 50 percent, which is considered a high figure by the pharmaceutical industry. Plus, successful therapies take an average of 10 years to reach the market.
None of this is to say that there’s no hope — there always is. I would rather be surprised than disappointed, but it’s important to take in the entire picture. To conduct clinical studies, participants are required, giving patients access to medication long before it hits the market. Additionally, countless treatments have started in animal trials, so there is at least some success there. In the worst case, every failure is one step closer to success.
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Is the effort to find a cure going to have a good outlook? My daughter has HD and I don’t feel that there is enough being done! Nothing seems to being done at UNMC in Omaha for my daughter Heather Ciboron. I would like to know if she is going to be able to go through any trials