I am in my fifties and I have the gene that causes Huntington’s disease. This disease lays waste to entire families for generations. I am not a medical person, but I always knew I was at risk. In 1977 I started reading all the previous year’s research on this disease. For many years, I would go to a medical library and it would take less than an afternoon to read every journal article and realize nothing from the previous year would offer any hope. At the time, I felt this disease was simply too powerful for any medical intervention.
In 1993, against long odds, the gene responsible for Huntington’s disease was discovered. Then it was possible for fruit flies and mice to be engineered with it, allowing researchers to conduct studies towards a treatment or cure. Huntington’s disease was suddenly a strong candidate for research because a single, known gene causing something so serious was an attractive research problem. As the years went by and the Internet evolved, dozens, and then hundreds of studies using flies and mice were developed. However, I gradually realized that our medical system was predicated on testing only one thing at a time. Even without experience in the field, I suspected nothing by itself would affect this disease. Eventually, researchers developed a treatment for the involuntary movements characteristic of Huntington’s, but there was nothing available to treat the disease itself.
By the time of that discovery, I was a high-ranking police official at a major research university. Desperate to treat my Huntington’s disease, I started creating my own cocktail of medicines. If a substance slowed the disease in fruit flies or mice; if it was proven safe; and if it could be obtained at reasonable cost, I would consider it. I started taking melatonin, CoQ-10, Trehalose, Tumeric, citalopram, NAC, montelukast, fish oil, and a few other things many years before some of these became widely known to the public as containing things that crossed the blood-brain barrier as anti-oxidant or anti-inflammatory.
I volunteered for many years of medical studies at the University of Iowa and visited my neurologist every six months for occasional baseline Neuropsychological and MRI testing. With a CAG count of 42, at age 57, I remain mostly pre-symptomatic. My neurologist showed me my MRI from earlier this year and said, “It’s nice to look at a healthy brain now and then.”
Thanks to years of invaluable animal research, there is an RNA interference treatment that will treat the actual disease for the first time. I’ve been following this treatment for many years and I’m confident it will finish its way through the Canadian approval process, make its way to the United States, and go on the market in time to help me and future generations of my family.
Animal research and FBR’s efforts have made a definitive difference in my life. With their help, I can spend nearly all my time with my work and my adopted daughter, instead of obsessing over research. I can be secure in knowing that the studies I participate in, the medications I take, or treatments I get are safe and steps closer to finding a cure. To paraphrase Mark Twain, “’tis the difference between the lightning-bug and the lightning.”