New And Effective Treatment For Patients With Huntington Disease
Researchers from University of Alberta discovered a new therapy which can restore motor skills that are affected by Huntington disease. This promising therapy may delay or even stop the progression of this debilitating disease.
“We didn’t expect to see such dramatic changes after administering this therapy. We expected to see improvement, but not complete restoration of motor skills. When we saw this, we were jumping with excitement in the lab. This is very promising and should give hope to those with Huntington disease. I think it’s a treatment that deserves to go to clinical trials because it could have huge potential.”, said Simonetta Sipione, the leader of this study.
Huntington diseases is a genetic condition in which is synthesized an abnormal protein that can trigger brain cell death, causing impairment of motor and cognitive functions and eventually death. Patients diagnosed with this disease present lower levels of GM1, a brain molecule. The scientists were able to normalize the level of this molecule in lab models with Huntington disease and after this procedure they also observed that the motor skills of lab models normalized within days. This study was published in the journal Proceedings of the National Academy of Sciences.
GM1 molecule which was used by the scientists in this research was produced both naturally and synthetically. The molecule which was also used in clinical trials for Parkinson’s disease treatment and for the treatment of others neurodegenerative conditions, represents a hope for patients with Huntington disease. The clinical trial for the treatment of this debilitating disease will probably begin within a year or two.
In this research lab models with Huntington disease received GM1 molecule based therapy for a period of four weeks. In the first two weeks after finishing the treatment, the lab models presented normal or improved motor skills. The bad news is that after this period the motor skills declined and after another four weeks, the motor skills returned to the same level of impairment as before treatment. The scientists hope that in near future they will be able to develop a treatment with this molecule which can be administrated at certain intervals of time.
Researchers were also curious if restored levels of GM1 molecule can improve cognitive function which is affected by Huntington disease. They observed that therapy with GM1 improves neurons activity and decrease the toxicity of the mutant protein which is a result of this genetic disease.
“Because of the way it works, we think it will work on cognitive symptoms of the disease too,” says Sipione.
The scientists highlight that it is very important for the medical world to understand that some of the treatments are working in lab models and may no effect on human models. For this reason they hope that the clinical trials for this new therapy for Huntington disease will begin as soon as possible. Researchers are very optimistic that in the near future, a working treatment for this debilitating disease will be established in practice, and this particular study results are definitely a huge step forward.