Huntington’s Disease Experimental Drug Slows Loss of Muscle Control

Huntington’s Disease

Raptor Pharmaceutical Corp. has made an exciting and potential quality of life changing announcement today for people afflicted with the hereditary brain disorder Huntington’s disease.  After the second phase of a three-phase trial of their experimental drug, RP103 (delayed-release cysteamine) the drug has been shown to be effective in slowing down the loss of muscle control and muscle spasms commonly experienced by people suffering from Huntington’s disease.

Participants in the study also evidenced a slower rate of deterioration in their hand and eye coordination than those who were administered a placebo during the trial.  Overall, during an 18-month course of treatment with the experimental drug RP103, the Total Motor Score (TPM) of the Huntington’s disease participants who received active doses showed a significant 32 percent slowing of the disease progression as it related to muscle control and other physical coordination deficits.

This is promising news for those who suffer from Huntington’s disease, as the loss of motor control is a distressing and difficult symptom that greatly affects their quality of life. In fact, the spasmodic muscle movements that are the most visible symptoms of the disease were described by Dr. George Huntington as a “hereditary chorea” in 1872 from the Greek word for dance. In the case of Huntington’s disease, it is an involuntary dance caused by the progressive death of nerve cells in the brain.

Less visible are the other symptoms that also present a great challenge to those afflicted with the disease. These can include depression, dementia, diminished cognitive functioning and in some cases the development of impulsive behaviors, which can make the caretaking of those with advanced Huntington’s disease a challenge as well.

Raptor Pharmaceutical Corp. is a global pharmaceutical company that has long focused on “life-altering” pharmaceutical applications for the treatment of degenerative and often fatal diseases. Dr. Christopher Verny who is the lead researcher for the current experimental trials of RP103 and the chief of neurology at the French hospital CHU d’Angers, is very encouraged by the trials. Verny is to be commended for his stated goal of not only continuing the cutting edge research he has been working on with Raptor but also his commitment to making sure that those who participated in the trials will continue to receive RP103 even after the current study has come to its conclusion. This will allow those experimental drug trial participants to continue to experience significant relief from their Huntington’s disease symptoms, specifically in the slowing down of the progression of their currently inevitable loss of muscle control.

In the United States alone, Huntington’s disease occurs in about 1 in 10,000 people and at this time, approximately 30,000 Americans are afflicted with the malady. Further, it is estimated that some 200,000 more are genetically at risk for developing the disease. Symptoms of the disease can first appear in the young and very elderly but most commonly begin between the ages of 30 and 50 and the progression of the disease is relatively gradual. However, with a life expectancy for those with Huntington’s disease of only 15-20 years following diagnosis, an experimental drug that can slow down the progressive loss of muscle control and coordination could considerably improve their quality of life. Today’s announcement by Raptor Pharmaceutical Corp. on the success of the experimental RP103 drug offers hope to people with Huntington’s disease and lends support to the need for continued cutting edge medical research.

By Alana Marie Burke
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The Wall Street Journal
Drug Discovery and Development
Huntington’s Disease Society of America
Counsel and Heal

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