From NBC:
“New treatment may help slow
progression of ALS, research shows”
An experimental medication may
slow the progression of amyotrophic lateral sclerosis, or ALS, researchers
reported Wednesday. The research was supported in part by donations from the
Ice Bucket Challenge, the social media sensation that raised more than $200
million worldwide. The drug is not a cure, but it may help slow the inexorable
disability caused by ALS, which rapidly destroys the nerve cells that control
the muscles that allow us to move, speak, eat and even breathe. "Patients
keep telling me their No. 1 goal is to be able to retain physical function for
as long as possible," said the study's lead author, Dr. Sabrina Paganoni,
a neuromuscular specialist at Massachusetts General Hospital's Sean M. Healey
& AMG Center for ALS. "They want to be able to continue to walk and to
use their hands."
About 20,000 people in the U.S.
have ALS at any given time, according to the ALS Association. It usually
strikes between the ages of 40 and 70. Once symptoms set in, life expectancy is
two to six years, on average. The treatment studied by Paganoni and her
colleagues targets two cellular structures damaged by the disease: the
mitochondria, which are the cells' power plants, and the endoplasmic reticulum,
the cellular dump trucks that cart away waste that can gunk up the cells'
machinery. The multicenter, randomized, double-blind study is the second step —
a phase 2 trial — in a three-step process required by the Food and Drug
Administration for drug approval. In a double-blind study, neither the patients
nor the researchers know who is receiving the drug. If a phase 2 study
generates positive results, the FDA typically requires a larger and longer
phase 3 trial. To test the effectiveness of the two-drug combination, the
researchers recruited 137 ALS patients who had become symptomatic within the
previous 18 months. About two-thirds of the patients (89) received the drug,
while the remaining third were given a placebo. Participants were evaluated on
a scale of 0 to 48, measuring the disabilities caused by the disease. "By
the time they entered the trial, on average, patients had already lost 12
points. Their baseline score was about 36, on average," Paganoni said.
"Each question addresses a specific domain of function and is scored on a
scale from zero to four."
For example, for walking: 4 =
Normal 3 = Early ambulation difficulties 2 = Walks with assistance 1 =
Nonambulatory functional movement only 0 = No purposeful leg movement
During the six months of the
study, patients taking the medication lost an average of 2.32 points less than
those receiving placebos, a 25 percent better functional outcome.
"A 2- to 3-point change can
mean the difference between being able to do an activity independently or with
an assistance device," Paganoni said.
The trial did not show a
difference between medication and placebo in outcomes such as time to death,
tracheostomy, or permanent intubation, or hospitalization. But that may be
because it ran for just six months. Paganoni suspects that, if it is approved,
the new drug would be just one part of a cocktail of medications that would
help to keep ALS at bay. Because the trial showed that the medication might
make a difference, all participants were offered the opportunity to stay on it
or, in the cases of those who were given placebos, to start on it. The Mass
General researchers will track how the patients taking the medication do in the
long term. Paganoni credited the Ice Bucket Challenge for getting her study and
others going. "The Ice Bucket Challenge was an important turning point in
the fight against ALS," she said. "It put ALS on the map and raised
awareness of the disease and attracted more investigators and investment to the
research."
With the good news from the
trial, the ALS Association hopes to persuade the FDA to allow other patients to
have access to the drug, even before phase 3 trial results are available. "It's
very unusual for an ALS clinical trial to hit its primary endpoint, so we're
very excited about it," said Neil Thakur, chief mission officer for the
ALS Association. "It's the difference between being able to feed oneself
versus being fed or needing versus not needing a wheelchair." ALS experts
cautioned against rushing ahead without more data. "The current data are
definitely positive, but they need to be replicated," said Dr. Martina
Wiedau-Pazos, a neurologist who is director of the ALS Clinic and Research
Center at UCLA. "This study has limitations, such as being small and
lasting just six months. I think a phase 3 trial is needed, because, in the
past, positive outcomes from phase 2 trials were not confirmed in phase 3
trials." Another issue is that the study included subjects who had more
rapid disease progression than normal, said Dr. David Lacomis, chief of the
neuromuscular division at UPMC in Pittsburgh. "So it's unclear what the
effects would be in the broader ALS population," Lacomis said via email. While
the new findings are promising, they are not "earth shattering," said
Dr. Erik Pioro, director of the section of amyotrophic lateral sclerosis and
related disorders at the Cleveland Clinic. "But it does add credence to
the idea that other pathways are playing significant roles in ALS
pathogenesis." Results of the trial were published in the New England
Journal of Medicine.
^ This is such great news. I only
hope these findings and tests continue so we can see if it helps even more than
we already know. ^
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.