#Drive Toward a Cure…#Cocktails for a Cause…#Madison Reprom…#Knoxville Yogafest…#Hoop-a-thon…#CHDI HD Therapeutics Conference…what do all of these have in common? All are events aimed towards raising money and/or sharing research to help people who have Huntington’s disease.
Huntington’s disease is a genetic disease with symptoms that begin to present between 30 and 50 years of age. The symptoms, which may include uncontrolled movements, loss of intellectual faculties and emotional disturbance, worsen over a 10 to 25 year period. This disease is devastating both to those experiencing the disease, and also to family members, both as caretakers and eventual patients, if they carry the expanded gene.
Considering the gravity of Huntington’s disease, what is going on in Huntington’s disease research to help these people?
Data from Pharmaprojects (Fig. 1) shows a promising number of industry-sponsored pipeline therapies for Huntington’s, including 4 compounds that have reached the final stages of clinical testing. There are also over 32 compounds in preclinical development, which suggests that more clinical activity could occur in the near future.
Data from Trialtrove (Fig. 2) shows a lot of current activity, as shown by the number of open or planned trials.
Fig. 3 provides a view of the companies that are most active in the Huntington’s space, as evidenced by having more than a single drug project for this disease. It is interesting to note that private foundations have been important in promoting research, as is the case for some other rare diseases, such as, Parkinson’s disease, and cystic fibrosis. For example, Pharmaprojects data illustrates how the CHDI Foundation is currently collaborating with industry partners on the development of multiple compounds for Huntington’s disease.
The activity in Huntington’s disease research means potential promise for those with this disease. Day-by-day, hand-in-hand, a difference can truly be made. Let me know how you are you involved in making a difference in Huntington’s disease. February 28th is #RareDiseaseDay, please let yourself be heard!