Mayo Clinic is among the handful of centers launching one-year fast-track studies of existing drugs to determine their value for other conditions. In Mayo's case, Jordan Miller, Ph.D., a Mayo Clinic researcher in the Departments of Surgery and Physiology, and two colleagues were awarded the grant to study the drug from pre-clinical tests through the first clinical trial, all in one year. The targeted condition is aortic valve stenosis -- a type of cardiac stenosis in which the valve area calcifies, leading to serious heart problems.
In a world where some research findings take a decade or more to surface in the world of patients, the National Institutes of Health 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.
“This is highly significant, not only because of the speed of the process, but the impact that it may have on prolonging lives,” says Dr. Miller. “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 50 percent will greatly reduce or delay the need for surgical intervention in these patients.” The first steps of the study are already underway.