A new year and fresh hope for Huntington’s disease treatments
How much closer are research, trials, and science to finding a cure?
My brother, Gavin, was diagnosed with Huntington’s disease in January 2022, at the age of 42. The disease took our dad’s life in May 2020, when he was 68. So far, it has affected four generations of our family.
As we enter a new year, I’m reflecting on how this time of year can mean many different things to different people. It might involve making a New Year’s resolution, starting a new job, entering a new relationship, or simply carrying on with life as usual.
For those of us in the Huntington’s community, every day is spent hoping for a cure. For us, the start of a new year raises the question of how much closer science, research, and clinical trials are to bringing us closer to that goal.
Developments on many fronts
Pharmaceutical companies are currently sponsoring the development of drugs to treat the symptoms of Huntington’s and, eventually, cure the disease. Some clinical trial results are expected this year, and hopefully, we will hear encouraging updates on others.
One type of treatment being investigated for Huntington’s is gene therapy, which involves “silencing” the mutated HTT gene that causes the disease. I’m hopeful about it because gene therapy has proven helpful for people with other medical conditions.
Some children in China who were born deaf, for instance, have appeared to benefit from this kind of treatment and are reported to be able to hear again. A gene-editing therapy has been authorized here in the U.K. to treat sickle cell disease. Last year, a toddler with a rare genetic disease became the first child to receive gene therapy from the U.K.’s National Health Service, and not long before that, a gene-silencing drug was reported to relieve severe pain and symptoms by 74% in people with acute porphyria in a clinical trial, according to the BBC.
Other types of studies are underway as well. I am enrolled as a gene-negative blood relative in Enroll-HD, an observational study with a site here in Exeter, England. Enroll-HD links Huntington’s families, clinicians, and researchers in order to monitor how the disease affects people and ultimately to make progress in developing effective treatments. Visit the website to learn more about how you can participate in research and support clinical trials.
Coming together in 2024
When I think of all the amazing people I’ve met through online support groups and my contact with the Huntington’s Disease Association, I reflect on a particular line from the Bible: “And now these three remain: faith, hope, and love. But the greatest of these is love.”
These are some of the kindest, bravest, and most caring souls I have ever encountered. The groups are filled with people who want to support one another through shared experiences with the disease. They share helpful information about research, trials, medication, managing symptoms like chorea, dealing with cognitive and behavioral problems, and diet. Together, they keep faith, hope, and love alive for one another, often in times of great difficulty.
My hope for the new year is that more people will come together within the Huntington’s community, and that they’ll continue to grow in number to provide support and raise awareness. We can all play a part by completing online surveys, joining research trials, or donating to Huntington’s charities. Doing what we can will help those managing the disease now, as well as future generations who remain at risk for it.
Note: Huntington’s Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Huntington’s Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Huntington’s disease.
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Comments
Peter tornaben
My daughter is married to a man whose father is struggling with the end of life stage of Huntington’s disease and my son in law does not want to be tested . He is 35 and so is my daughter
They want to have a baby but they want to protect it from the possibility of having this disease
They are exploring ivf
When will my son in law show signs if in fact he has it ???
Becky Field
Dear Peter, do you know the CAG repeat size of your son in law's father? Are there any hospitals with genetic counselling clinics near you? Or do you have any local support groups who specialize in Huntington's disease? They may have specialist care advisors? IVF would be a way of having a child, free of Huntington's disease. Here are 2 links, one web page giving information about the testing process and one web page giving information about the average age of onset, based on your CAG repeat size. Please let me know if I can be of any further help. https://www.hda.org.uk/information-and-support/getting-help/genetic-testing/
https://hopes.stanford.edu/the-inheritance-of-huntingtons-disease-text-and-audio/