Despite an embrace of greater patient populations by the FDA, cardiovascular research into stem cell therapy has been slow and burdened.
A new study presented this week at the Heart Failure Society of America (HFSA) 2019 Scientific Sessions in Philadelphia, PA, shows just about 55% of heart failure-based clinical trials have published findings in the past decade. Investigators expressed frustrations with the lack of fulfilled promise—and worthwhile data—from these studies, which would benefit clinicians and patients equally.
There’s similar frustrating being felt in the field of caridiology stem cell therapy, currently. Despite an embrace of greater and faster regulatory pathways to US Food and Drug Administration (FDA) indication, stem cell therapy options have been mostly limited to oncologic indications.
In an interview with MD Magazine® while at HFSA 2019, Joshua Hare, MD, director of the Interdisciplinary Stem Cell Institute at the University of Miami, diagnosed the challenges facing cardiologic stem cell therapy, and what needs to be done for indications including heart failure care to be reached.
MD Mag: Have changes to FDA regulation and pathway indications improved the case for stem cell therapy?
Hare: Unfortunately, it hasn't. The funding for cell therapy in heart disease has remained relatively lackluster, and I would say inadequate over the years. It's taken a very long time to get these trials done and to get to the point where we can have the data we're going to have next year.
It's taken so long that Congress actually passed a law, called the 21st Century Cures Act, that gave the FDA powers to give accelerated approval pathways for cell-based therapies. Those accelerated pathways have been very useful for investigators working in the field of cancer or gene therapy, but we've yet to see a cardiac cell-based therapy approved by the FDA.
We're very hopeful that’s going to come in the next 1-2 years with this new data that I talked about. It's been a bit slow, but it's coming. It's around the corner.
MD Mag: What drives the difficulty to complete pivotal cardiology trials for stem cell therapy?
Hare: It's always difficult. It’s very, very expensive to develop new therapies, and big pharmaceutical companies spend the billions and billions of dollars on these development pathways—and they don't always come up with a successful target, a successful drug.
So it's a very risky business, but fortunately, there's a tremendous amount of scientific innovation in the United States. The United States is a leader in developing new therapies, and it requires innovators and investors to get a drug all the way down the down the field to get an approved drug.
We haven't seen the kinds of dollars going to cell-based therapies that have typically gone into, say, developing new cancer drugs. And these are just decisions that are being made by pharmaceutical companies about what seems to be most promising.
I think we're not yet certain that we have developed the winning formula for cell-based therapy, and it's another reason why the studies I've talked about are so important to be completed—so we can get the data and then go on to the next stop.