In a world where some research findings take a decade or more to surface in the world of patients, the National Institutes of Health (NIH) is trying to whittle that time of translation down to a single year. That includes several rounds of preclinical studies and then a phase one clinical trial to determine efficacy by the end of the twelve months.
Jordan Miller, Ph.D., a Mayo Clinic researcher in surgery and physiology, says, “This is highly significant, not only because of the speed of the process, but the impact that it may have on prolonging lives. We have a year to determine if there’s a positive change in experimental models and in patients. If so, we move on with more studies.” Mayo Clinic is one of only nine awardees of a new type of research grant, one that takes an existing but unused drug from a participating pharmaceutical company and studies it for a different disease or condition.
In this case the drug is supplied by Sanofi, and Mayo’s team led by Dr. Miller will see if it will work to slow the advance of aortic valve stenosis, in which calcification keeps the heart valve to the aorta from opening fully.
While only three percent of Americans of retirement age develop aortic valve stenosis, calcium can be seen (using x-ray or CT scanning) on the valves of approximately half of the population over 65. As baby boomers continue to live longer, the actual numbers of patients expected to progress to aortic valve stenosis are expected to grow dramatically, and less than 40 percent survive beyond five years from diagnosis. Right now there is no effective treatment for advanced stenosis, other than replacing the heart valve – a surgical procedure that carries its own risk.
Dr. Miller says this new application of the drug may not remove existing calcification, but it may slow the progression or stop it from getting worse. “Even slowing progression of valve calcification by half will greatly reduce or delay the need for surgery in these patients.” The first steps of the study are already underway.
An interview with Dr. Miller will be posted later this week.