SILVER SPRING, Md., Dec. 7, 2017 /PRNewswire-USNewswire/ — Chairman Alexander, Ranking Member Murray, and members of the committee, thank you for the invitation to testify at this hearing to discuss the implementation of the 21st Century Cures Act.
“Cures” set FDA on a transformative path. It set out to optimize our investments in science by modernizing how FDA oversees breakthrough technologies. You asked us to advance innovations more efficiently, while maintaining our gold standard for protecting patients.
This focus on innovation couldn’t come at a better time. Across multiple fields of science, we stand at an inflection point in medicine – where new technology is creating foundational opportunities to treat and cure disease in ways that weren’t possible just a short time ago.
Take, for example, our recent experience with gene therapy.
We’ve seen two recent approvals of CAR-T therapies for cancer, where a patient’s own immune cells are re-engineered – using the tools of gene therapy – to target a patient’s individual cancer. This form of gene therapy represents a whole new paradigm in treating cancer. And the early results are changing the way we treat serious tumors.
This experience shows how a single, fundamental breakthrough in science can open up a whole new way of combatting disease.
In gene therapy, that breakthrough has been the development of vehicles that can deliver genes more efficiently to their target inside the body. These often are referred to as vectors. And they’ve taken the form of viruses that are specially engineered for this purpose.
In particular, the advent of a specific kind of largely inert adeno-associated virus – or “AAV” vector – was an inflection point in this field.
I liken the advent of AAV vectors to the development of processes for developing antibody drugs and making these medicines nearly identical to the fully human cells that they were mimicking.
Monoclonal antibodies represented a promising field of potentially breakthrough medicines in the 1990s. But for a long time, these therapeutic drugs fell short of their promise.
That was because these drugs were made with antibodies from mice, and the antibody drugs themselves were soon rejected by patients’ immune systems. Then came the science for humanizing these antibodies, so they’d more fully mimic their normal human counterparts. And pretty soon, we saw many breakthrough drugs result. A whole new field of medicine grew up very fast.