Sometimes, cures can even surprise the doctors who’ve delivered them.
In the early 1970s, Dr. Larry Einhorn was a young doctor treating cancer patients. But he noticed that the standard drug used to treat testicular cancer only succeeded five to 10 percent of the time. So, he tried adding a second drug to the treatment regimen. It worked. “I was amazed that the introduction of an experimental drug literally had a one-logarithmic increase in the cure rate, going from five percent in a contemporaneous actinomycin-D to 64 percent in this original phase II clinical trial,” he later said.
Needless to say, he didn’t stop at two-thirds success. By the 1980s, he had developed an even more effective treatment. “In a mere 12 years,” he points out, “we demonstrated that we no longer need two years of maintenance therapy, that we can cure the patients with just 12 weeks and four courses, that etoposide is better than vinblastine and also less toxic.”
Those doctors only solved these problems because they tried to. Yes, they designed clinical trials. But there are also times when a patient is too sick to wait. In those cases, they also need doctors to “try,” which is exactly why New Hampshire recently passed the most expansive “right to try” law in American history.
President Donald Trump wants to do the same for the country. During his first term, he signed right-to-try legislation to make it easier for seriously ill patients to use drugs on an experimental basis. If standard treatments have failed, they can go beyond the standards set by the Food and Drug Administration (FDA) and request access to experimental therapies that are not yet approved for public use. But the FDA hasn’t been willing to give up its control. It has been dragging its feet on approving treatments.
The Wall Street Journal last month wrote that, “In recent weeks, the FDA has rejected three therapies for debilitating diseases that have shown promise in clinical trials. The agency has also forced off the market a gene therapy that can slow the degenerative loss of muscular function in young boys with certain genetic mutations.”
The newspaper expressed particular concerns about Dr. Vinay Prasad, a vaccine expert at the FDA.
Pharmexec reported on July 30, 2025, that “during Prasad’s tenure, the FDA placed restrictions on Sarepta Therapeutics’ gene therapy, Elevidys, due to multiple deaths in patients taking the drug.” That ban was rolled back when parents of boys suffering from a fatal muscle-wasting disorder complained.
It doesn’t need to be like this. “Prasad’s predecessor at FDA, Dr. Peter Marks, oversaw a dramatic rise in approvals for new gene therapies, which aim to treat or prevent disease by replacing or modifying a portion of patients’ genetic code,” the Associated Press wrote. “Prasad has been an outspoken critic of Marks’ leadership at FDA, which included overseeing the approval of the first COVID vaccines and therapies.”
“Prasad, the head of the biologics division, has long criticized such single-arm studies that have no placebo groups,” the WSJ wrote. “He believes medicines should undergo randomized controlled trials that track patients over longer periods to measure overall survival. Never mind if patients die in the interim.”
To be sure, clinical trials are fine, but some patients don’t have any time to wait. They need treatment now. If it ends up helping them, medicine will have moved forward, and they’ll get more time with friends and loved ones.
Dr. Prasad resigned to spend more time with his family, but the FDA quickly convinced him to come back. That’s fine, as long as he changes his attitude enough to satisfy Trump, who demands that patients be allowed to experiment. The FDA should support that mission wholeheartedly.
