WASHINGTON (NewsNation) — A bipartisan group of senators is proposing a plan to give patients with terminal illnesses access to therapies still in development before they obtain full Food and Drug Administration approval.
Dubbed the Promising Pathway Act, it could go a long way for people such as 8-year-old Ben from Katonah, N.Y., who suffers from a rare condition known as Salla disease, which affects the nervous system, his mother said.
Ben recently visited the nation's Capitol with his mom — to press those in power for help in coping with a terminal disease. They may not be able to save his life, but they could at least help him live longer.
“Ben has a disease that he's going to succumb to as a young adult,” said his mom, Jessica Klein-Foglio. “It causes muscle deterioration and he'll lose ability to swallow. Most patients pass away of cardiac arrest or inability to eat."
Klein-Foglio, who now runs a foundation dedicated to researching the disease, has spent years searching for options to try to extend her son's life.
She has become interested in a drug already used to treat other conditions that may be able to slow the progression of his disease. It’s still under research for Salla disease.
Under FDA rules, it's not an option for Ben.
“It's sad because isn't his life worth it, too?” Klein-Foglio said. “If you have cancer or Parkinson's, you have research options and viable treatment options. But when it comes to a rare [pediatric disease], is it because there's not enough money? There's not enough patients?
“It's infuriating,” she continued. “Let us try."
The goal is to give patients a chance before it's too late, said U.S. Sen. Mike Braun, R-Ind., one of the lawmakers leading the effort.
“You could get it, instead of 15 years, after three or four years,” he said.
Braun says he has become passionate about the cause since losing a cousin to ALS, commonly known as Lou Gehrig's disease.
Under his proposal, drugs awaiting final approval must reach certain benchmarks to be considered safe and effective for temporary use, and their provisional status would be up for renewal every two years.
“If data would come in that there are side effects that you didn't see early, then it slows the process down,” Braun said.
Though the legislation has bipartisan support, some have raised concerns.
Holly Fernandez-Lynch, an assistant professor of medical ethics and law at the University of Pennsylvania, testified to Coness in October that she is worried the bill would "set the bar too low" for granting provisional approval.
“Weakening FDA's approval standards risks conflating the essential goal of getting patients access to more drugs that work with the deflated goal of simply getting them more drugs,” Fernandez-Lynch told lawmakers.
Braun released an amended version of the bill in late February, which he believes addresses those concerns by further narrowing eligibility for provisional approval and ensuring the FDA has the oversight it needs.
Still, the proposal gives hope to patient Michael Hugo, who has battled glioblastoma — an aggressive brain cancer — for nearly two years.
“If you don't do something and you are diagnosed with glioblastoma, you're going to die,” he said.
As a father of two young girls, Hugo has been searching for a treatment that may give him more time.
"What it means is I get more daddy-daughter dances with my girls,” he said. “There's a chance that I make it to their graduation."
Any chance adds a little hope for those whose futures are dim.
“I know it wouldn't be able to reverse what's happened,” Klein-Foglio said of a potential treatment for her son. “But it would mean that Ben's life and my purpose with Ben has a bigger, more universal meaning and his life is ultimately paving the way for future kids.”
The bill is awaiting approval from the Senate Committee on Aging before it goes to the full Senate for approval.