FDA approves new drug to fight ALS

WASHINGTON — A new medicine is joining the battle to help people suffering from amyotrophic lateral sclerosis, which is also known as Lou Gehrig’s disease.

The U.S. Food and Drug Administration announced the approval of edaravone, which will be sold as Radicava. It is the first new drug on the market in 22 years that aims to slow the progression of ALS.

While it is a new option for people suffering from the potentially fatal disease, Dr. Justin Kwan, assistant professor of neurology at the University of Maryland, said a study of the drug doesn’t include head to head testing against the current treatment, Riluzole.

“It’s difficult to say whether or not it is more effective than the current best treatment,” Kwan said.

Mitsubishi Tanabe Pharma Corp., the company that makes the dug, described it as a free radical scavenger that can relieve the effects of oxidative stress, which is believed to be a factor in the onset of the disease.

Kwan said he sees the drug being used two ways: as an additional drug for an existing regimen, or as an option for someone who cannot take Riluzole.

Since neither drug is a cure for the disease, the hope of the drugs, according to Kwan, is to slow the progression of the disease.

Each dose of Riluzole is expected to cost $1,000 with a patient spending $145,000 for a year’s worth of treatment.

What is ALS?

It’s a rare disease that attacks and kills nerve cells that control voluntary muscles. The FDA estimates that 12,000 to 15,000 Americans suffer from the illness.

The disease can lead to paralysis, and many diagnosed with it only live three to five years past the onset of the disease. One out of 10 cases is caused by a genetic defect, according to the University of Maryland Medical Center.

What are the symptoms?

Kwan said most people with ALS experience weakness in their arms and legs, and may have trouble speaking or swallowing.

“A lot of our patients will have slurred speech or they may choke when they drink liquids or swallow food,” he said.

It is important to note that these symptoms may also be caused by several other neurological diseases, so Kwan said going to a doctor with experience diagnosing ALS is key.

How is it diagnosed?

There is no one test to confirm whether someone has ALS.

Kwan said doctors trained in spotting the disease will focus on tests like brain and spine MRIs, blood testing, and EMG nerve conduction studies to look at nerve and muscle function.

This content was republished with permission from CNN.

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