CHERRY HILL, N.J. (CBS) — A big focus on a congressional hearing is set for Thursday, where the U.S. Food and Drug Administration is expected to provide an update on access to treatments for ALS, also known as Lou Gehrig’s Disease. The treatments currently available for ALS are limited. Patients want legislation for better access to experimental drugs, along with more help from the FDA.
Stacy Lewin was living her dream as an obstetric anesthesiologist and now, she’s fighting to stay in the world after being diagnosed with ALS.
She’s traded in her scrubs for a walker and breathing devices at her Cherry Hill home.
“I love taking care of the moms and babies and bringing life into the world,” she said. “It’s horrifying and I’m terrified about what possibly lays ahead.”
ALS is a progressive neurodegenerative disease that leads to paralysis.
“It robs you of your ability to function,” Lewin said.
For her husband and two sons, it’s emotional, but they’re focused on making each moment county.
“I need the ability to live another three months, another six months to be with my family, to watch my boys grow up, to be with my husband,” Lewin said.
But Lewin and other patients with ALS say approved treatments are limited and FDA regulations, designed to assure safety, prevent them from getting experimental drugs.
“Even if the drugs don’t work, I’m willing to try them. I have nothing to lose, there’s nothing else for me, nothing else available,” Lewin said.
ALS advocates want the FDA to come through on a commitment to provide better access, an issue being addressed by a congressional committee on Thursday.
“All we need is for congress and the FDA to make drugs accessible,” Lewin’s husband said.
That could come with the passage of the ALS Act, legislation that would fund early access to clinical trials.
“I want a chance to live more,” Lewin said.
She says her first symptom was muscle weakness.
It’s unknown what causes ALS.