Given the unprecedented interest from biopharmaceutical firms and broadening range of approaches to attacking the disease, the need for the Huntington’s disease community to participate in research studies and clinical trials is “exploding,” CHDI’s head scientist said at the organization’s 14th Annual HD Therapeutics Conference yesterday.
“This is not a time to rest on our laurels,” said Robert Pacifici, Ph.D., the chief scientific officer for CHDI Foundation, Inc., the nonprofit virtual biotech dedicated to finding HD treatments. “Trials won’t go forward without people willing to take the risk of taking a drug never taken before.”
Dr. Pacifici’s comments came in an interview with me at the conference venue, the Parker Palm Springs hotel in Palm Springs, CA. The gathering of scientists, pharma executives, and advocates ends today.
Critical new research studies
The conference comes just after news of the start of Roche’s historic Phase 3 gene-silencing clinical trial and other clinical trial programs (click here to read more).
“It’s exciting times,” Dr. Pacifici observed. “Now more than ever we need to make sure that these channels of communication between patients and caregivers and researchers remain open and clear so that everybody knows what’s going on.”
With the advent of more trials, both the pace and number of research projects requiring volunteers is growing rapidly, he explained. One involves a forthcoming brain imaging study (using a PET-ligand).
To help understand the disease and measure the effect of potential drugs in clinical trials, scientists need to find more biomarkers (signals of the disease) in humans. For years, they have collected samples of blood. More recently, they started examining the cerebrospinal fluid and saliva.
Now CHDI wants to collect semen, Dr. Pacifici said. “There are certain things that happen only in those cell types that are going tell us what happens to Huntington’s DNA, and that’s going to be really critical for our efforts as well,” he explained.
CHDI will also establish a “Brains for HD” initiative to request donations of that organ from deceased individuals, Dr. Pacifici added. “Invaluable,” he said of the importance of brains in HD research. “Especially the rare young brain, if somebody happens to pass away from something else, and we can harvest the brain before it experiences the ravages of Huntington’s disease.”
You can watch my interview with Dr. Pacifici in the video below.
Dr. Pacifici outlined the conference’s three major areas of focus.
He described the first as “Huntington’s homework.”
Source: At Risk for Huntington’s Disease