Interview with CCSA’s Director of Scientific Affairs Howard Higley, PhD, DABT®
Q: What are some of the most promising agents you are currently working with?
A: Blueprint Neuroscience Network’s (BPN) small-molecule Alzheimer’s disease (AD) drug has great potential as new mechanism to control the disease. Confirmation that the amyloid hypothesis is the basis for disease pathology and control by intervention has been difficult to come by. The BPN drug holds promise in being able to specifically target amyloid build-up and also has fewer toxicities associated with other drugs in its class.
Q: What is amyloid hypothesis?
A: Certain normal and injury proteins that accumulate pathologically in neurons and impair their function. Ultimately impacting memory and other neurological functions.
Q: Do you know how this drug targets amyloid build-up?
A: It improves clearance or prevents accumulation of amyloid by inhibiting enzymes that enhance its deposition.
Q: What is its phase of development?
A: Phase I first in human trial. Most AD drugs are tested initially in normal human volunteers for safety and dose finding and then in minimally cognitively impaired (MCI) patients to get some assessment of efficacy.
Q: To what types of patients are they giving this drug?
A: First proof of concept will be in MCI patients (early AD), then they will assess the value for reversing or retarding progression in more advanced disease.
Q: In your opinion, where do you think this research is headed for this drug or Alzheimer’s research in general?
A: Ideally an oral small molecule drug that’s easy to take and has few side effects can revolutionize therapy for patients that at present have just a few limited options with drugs that only modestly slow the decline of memory and function. The AD field also needs to advance on other fronts, like earlier diagnosis driven by better indicators of disease and treatment response like central nervous system imaging or genetic and serologic biomarkers.