A drug previously tested to treat Parkinson's disease and related neurodegenerative maladies has been used in preclinical testing to successfully restored motor skills damaged by Huntington's disease, at least for a few weeks after the dosing stopped. The scientists at the University of Alberta say the data is promising enough that human clinical trials need to happen next. And because the drug is being tested in clinical trials already, they expect it could pass regulatory hurdles to begin testing in Huntington's patients within two years.
"When we saw this, we were jumping with excitement in the lab," principal investigator Simonetta Sipione said in an announcement touting the news.
Interesting. Huntington's disease is inherited and deadly, as patients' neurons degenerate and they're left with increasingly uncontrollable movements, mood swings and problems remembering things. Huntington's sufferers eventually struggle to feed themselves or even swallow, and then die from the disease, according to the National Institute of Neurological Disorders and Stroke. So a viable treatment is invaluable. Many are trying to get there, through different mechanisms of action. The University of Alberta data, published in the Proceedings of the National Academy of Sciences, and covered by Bloomberg, among others, deserves a closer look. But the research is certainly early-stage and the benefits, even in the study, only lasted a short time.
Further testing of the drug on an ongoing basis in both animals and then people will be necessary to see if this route is even viable, said The Huntington Society of Canada, which funded the research, also in the same announcement. But the group is still hopeful. Huntington Society CEO Bev Heim-Myers noted in a statement that the research "for the first time, has demonstrated that in a Huntington disease laboratory model, the treatment reverts the lab model back to normal, not just slightly better."
The researchers said the drug they used, a GM1 molecule therapy, has previously been tested in Parkinson's and in other related diseases. For the trial, they gave "lab models" the drug for four weeks. The treatment restored motor function and GM1 molecule levels, apparently by making the neurons work better, and the toxic huntingtin protein become, well, less toxic. About two weeks after the treatment, the positive effects began diminishing, taking another two weeks to return to pre-treatment levels.
In the future, subsequent testing would look at long-term treatment with the drug. And an ongoing study continues to see, in lab models, if restoring GM1 molecule levels can also repair Huntington's-related cognitive damage.