- About 30,000 people in the United States are living with amyotrophic lateral sclerosis (ALS).
- There are five drugs approved by federal regulators to treat the disease.
- A new drug, edaravone, has joined the list. It’s an oral form of the drug Radicava.
- Experts say it will make treatment easier for people with ALS because it doesn’t require an intravenous infusion.
Some help may be on the way for people living with amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease.
The U.S. Food and Drug Administration (FDA) has
Why is that important?
“It makes it much easier for patients to be compliant and reduces the travel burden on many of these very disabled patients with ALS,” said Dr. Santosh Kesari, a neurologist at Providence Saint John’s Health Center in Santa Monica, California. He is also the regional medical director for the Research Clinical Institute of Providence Southern California.
“A weekly infusion means you usually have to either go to a hospital setting or an infusion center setting. But an oral solution makes it easy to administer at home,” Kesari told Healthline.
Dr. Jinsy Andrews is the director of neuromuscular clinical trials at Columbia University in New York as well as an international expert on ALS research and clinical trials. She also serves on the board of trustees for the ALS Association.
She says just maintaining the IV route to administer the drug is challenging.
“The disease creates difficulty with speaking and swallowing, breathing, walking and using their arms. They don’t need to have the additional burden of maintaining an IV access and all the complications that come with that,” Andrews told Healthline.
“The other benefit is that it is something that can be given through the feeding tube as well if people living with ALS don’t have the ability to swallow,” she added.
ALS affects as many as 30,000 people in the United States. About 5,000 new cases are diagnosed each year.
It’s estimated that ALS may be responsible for 5 out of every 100,000 deaths in people 20 years or older.
There are currently five drugs with FDA approval to treat people living with ALS and its symptoms. They are Radicava, Rilutek, Tiglutik, Exservan, and Nuedexta. Radicava was the first new treatment specifically for ALS in 22 years.
“It can improve symptoms or function outcomes for longer periods of time. It’s certainly not a cure, but it does improve and slow down disease progression,” said Kesari.
Experts say there are more treatments to come.
“There definitely has been an increased pace of drug development in ALS, pushed by the fact that we have things that have shown some modest benefit,” said Andrews. “I think we will make incremental steps to finding a therapy that will eventually have a bigger impact.”
The pharmaceutical company Amylyx has announced that the FDA is expected to make a decision on approval for its oral ALS drug, AMX0035, by the end of next month. That authorization is not guaranteed after an FDA advisory committee voted 6-4 in late March to report that they felt the research data wasn’t substantial enough to support the efficacy of AMX0035 for the treatment of ALS.
In June, the drug did receive approval from Canadian officials for use in that country.
There are multiple other research studies and clinical trials as well.
“Our center, Pacific Science Institute, is working on stem cell therapy for a variety of disorders, including ALS,” said Kesari.
“We are also looking at how to regenerate neurons and brain cells using drugs that can stimulate neurogenesis, the formation of new cells, and we’re looking at anti-inflammatory drugs” he explained.
In December, President Biden signed the “Accelerating Access to Critical Therapies for ALS Act.” It provides $100 million a year in federal funding over the next four years to research cures and the prevention and treatment of ALS.
Andrews says that move may help pave the way for more research on the drug development landscape. She is also hoping to get more people living with ALS into treatment earlier.
“A lot of clinical trials right now are looking for people with ALS in the early stage of disease,” she said. “That means we have to make sure that ALS is recognized quickly and they get to a multidisciplinary ALS specialty center sooner.”
“There’s a diagnostic delay because we don’t have a definitive diagnostic test for ALS. It’s still a clinical diagnosis,” she added.
Andrews says what’s needed is education and more awareness of the symptoms. She adds that improved technology, such as a shared database, could help.
“I hope one day we will have a matchmaking tool so that as soon as a person is diagnosed, they get matched into a clinical trial,” she said.