Behind the scenes enabler: Almost two years ago, CHDI was added to the drug development team with the goal of helping to bring together the scientific "know how" of academic scientists and the experience of the pharmaceutical industry. CHDI is a drug company for Huntington's that has no laboratories of its own but expertly solicits and provides incentives to industry partners to work with scientists to meet the needs of the drug development process. The goal is to facilitate the most rapid development of drugs for Huntington's.
CHDI work in progress: As collaborative partner, CHDI has funded academic labs and pharmaceutical companies, providing more than ten million dollars each year for 11 academic labs, 10 biotech and pharmaceutical companies, and 20 vendors (product suppliers). Funded projects include large scale screening for effective drug candidates and drug combinations. There are others that modify and improve molecules selected from screens. These steps maximize potency, decrease toxicity, and improve brain delivery; turning the molecule into the best drug. Best molecules are then tested in the equivalent of a mouse model factory. Several companies are working on specialized types of drugs. Still others are working on biomarkers that can speed the clinical trial process.
Families on stage and in the audience: There are also very important Huntington's people on the symphony stage. These are clinical trial volunteers. There can be no music of a drug brought forward without families who give of themselves to volunteer for clinical trials. At this stage in drug development for HD, volunteers for clinical trials are essential.
But whether we are on stage, or are spectators in the audience, all HD families wait for the music. And though we'd dearly like to be hearing the music of a successful drug, we have lived long enough with this disease to be realistic. Drug development takes time.