BOSTON (CBS) – There is new hope for ALS patients like Pete Frates. The FDA just approved a new drug for treatment and as Dr. Mallika Marshall explains, while it is not a cure, it could help put the brakes on this fatal illness.
In 2012, Boston College baseball star Pete Frates was diagnosed with ALS. He was only 27 years old.
“It’s the cruelest of the cruel,” says Nancy Frates, Pete’s mother. “It just continues to ravage your body until there’s nothing else to be taken away.”
ALS attacks nerve cells, causing people to lose muscle function over time. Most patients die from respiratory failure within three to five years.
Since 1995, there has been only one drug specifically approved to treat ALS. Pete has been on it for five years but continues to deteriorate.
Now, for the first time in 22 years, the FDA has approved a new drug, Radicava, which is already available overseas.
A recent study out of Japan found that after six months of therapy, patients with ALS who received the drug experienced less decline in daily function.
Experts say those in the earliest stages of disease are most likely to benefit and it is a tough regimen with cycles of daily infusions, but Pete plans to take it.
“We’re going from no options to an option,” says Nancy.
It is unusual for the FDA to approve a drug this quickly without confirmatory study results in the U.S., but Nancy is thrilled.
“We are applauding the FDA for doing this,” she says, “And listening to our community that has been advocating urgency every time we meet with them, and they must have heard of us.”
Nancy says the Ice Bucket Challenge has certainly brought much needed attention, research dollars and optimism.
“This community has more hope than it has had in the 150 years that this disease has been around,” she says.
Radicava should be available in the U.S. by August. It is expected to cost about $145,000 a year.