More than 1.7 million Americans will be diagnosed with cancer this year. Almost one in three of those patients will eventually succumb to it.
Knowing these odds, patients are eager to try anything to impro
That's why so many Americans applauded passage of the "Right To Try Act," which was signed by President Trump in May. The measure creates a legal pathway for terminally ill patients to access new medications that have only been through one phase of Food and Drug Administration approval.
Consider the case of BrainStorm Cell Therapeutics, a drug company with a new experimental stem cell treatment called NurOwn. The treatment is for ALS, the terrifying disease to which baseball superstar Lou Gehrig succumbed. Those suffering from the neurogenerative disorder gradually but inexorably lose all ability to control muscle movements. Eventually, patients are completely isolated in their immobile bodies—typically until their breathing stops.
NurOwn is harvested from the stem cells of each patient. It's currently under development, and results to date have been inconclusive.
RELATED: Trump signs Right to Try into law
A midstage study involving 48 patients found those given the drug did appear to respond, though the benefit didn't last for most. It's highly uncertain whether NurOwn will eventually pass the FDA's scientifically rigorous safety and efficacy standards.
Nevertheless, NurOwn's develop
Insurance doesn't generally cover treatments that haven't been approved by regulators or proven to work in clinical trials. That means patients would have to pay for the therapies out of their own pocket. Some could be desperate enough to sell the house to come up with the money.
The right-to-try legislation isn't designed for commercial profit. Yet after Trump signed right-to-try, requests for NurOwn skyrocketed, and BrainStorm's shares closed up 2.8%.
No one actually knows if NurOwn works. But desperately ill patients may gamble everything to try it anyway. Such are the unintended consequences of right-to-try.
No wonder the American Society of Clinical Oncology opposes right-to-try legislati
"ASCO supports access to investigational drugs outside of clinical trials, when adequate patient protections are in place," ASCO chief medical officer Richard Schilsky, MD, said. "We don't support right-to-try legislation, however, because these laws ignore key patient protections without actually improving patient access to in
Let's hope BrainStorm—and other likeminded companies—have learned that cashing in on the uncertain hopes of desperate patients is an unacceptable industry practice. And let's make sure the FDA sends that message in no uncertain terms.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.