A path forward for experimental drug treatments


MATTHEW BELLINA, a retired Navy pilot, knows time is running out. Now 34, Bellina was diagnosed in 2014 with Lou Gehrig’s disease, a fatal disorder that attacks the central nervous system and gradually steals life and livelihood: the ability to speak, eat, and breathe.

Bellina wants to try NurOwn, an experimental adult stem cell therapy, because he wants to stick around awhile longer to see his three young sons thrive. He is pinning his hopes on a bill in Congress that would give terminally ill patients access to investigational drugs that have passed a Phase I clinical trial but have not yet gone through the full multiyear approval process required by the federal Food and Drug Administration. That bill, which cleared the US Senate last summer and is now before the House, is an important step forward in galvanizing a crucial national discussion — despite the off-putting “right to try” nomenclature, which evokes comparisons to the “right to die” movement and seems to position it narrowly as a libertarian battle cry.


That does a disservice to a broader question: Is the FDA able to keep up with the rapid pace of research and discovery ushered in by the genomic era? Certainly, the agency plays a critical role in public safety and, as important, in “using science for regulatory decision-making,” as former commissioner Margaret Hamburg once put it.

As things stand now, Right To Try laws have passed in 38 states, and state Representative Nicholas Boldyga, a Republican from Southwick, has introduced a measure in Massachusetts. But a patchwork of state laws serves no one well, particularly when terminal patients might be desperate to try drugs manufactured across state lines or overseas. The FDA has taken steps to speed access before, during the height of the AIDS epidemic. Launched in 1987, the agency’s “compassionate use” program allows access to drugs in the testing phase, provided a physician gets FDA approval. But that program is cumbersome and little-used, according to Starlee Coleman of the Goldwater Institute, a libertarian think tank pushing for Right To Try.


The federal bill isn’t perfect — doctors worry about issues like patient safety and consent, as well as diverting resources away from authorized clinical trials that generate important data. But regulatory agencies like the FDA need periodic rebooting to stay abreast of the fast pace of scientific discovery. Japan offers a valuable case study: In 2014, new laws were passed to accelerate approval of stem cell therapies, giving companies conditional approval while clinical trials continued. The laws are credited with accelerating Japan’s global role as a center for regenerative medicine. With the right bipartisan spirit, such a model might work in the United States. And Matthew Bellina might see his wish fulfilled.