THURSDAY, Dec. 20 (HealthDay News) -- A U.S. Food and Drug
Administration advisory committee will meet Thursday to consider
approval of the first drug to treat chronic fatigue syndrome.
The experts will discuss the risks and benefits associated with
the intravenous drug rintatolimod (proposed brand name Ampligen).
The drug's maker, Hemispherx Biopharma of Philadelphia, failed to
win the FDA's OK in 2009 because of concerns about study
Experts said they would welcome a treatment for this disabling
condition that affects as many as 4 million Americans, mostly
women. There is no cure, but the drug appears to reduce symptoms
for some patients.
"It does seem to help at least a subset of patients significantly. For others, there isn't a significant response," said K. Kimberly McCleary, president of the Chronic Fatigue and Immune Dysfunction Syndrome Association of America.
"This drug has been studied in chronic fatigue syndrome since the late 1980s, so it's been around for a while," McCleary added.
Dr. Nancy Klimas, professor of medicine at Nova Southeastern
University College of Osteopathic Medicine in Fort Lauderdale,
Fla., who is part of an ongoing trial of the drug, said some of her
patients have benefitted from the drug. Now there needs to be a way
to identify which patients will do well on the drug, she added.
Chronic fatigue syndrome is little understood, and Ampligen's
approval would give patients some standing with their insurers,
"Even a single approved therapy, even if it were one I choose not to use, would be very helpful when I am arguing with insurance companies to legitimize the condition and that it is serious enough to require an intervention," Klimas said.
According to the drug company, Ampligen is a new type of drug
called a nucleic acid compound, which uses specially made RNA to
target a variety of diseases. Hemispherx believes the drug has the
potential to fight HIV, kidney cancer and melanoma in addition to
chronic fatigue syndrome.
The drug is said to work by modulating the immune and antiviral
functions in diseased cells.
One drawback of the treatment is that it needs to be infused
twice a week, Klimas said. It also is very expensive, she said.
The maker of the drug couldn't estimate the retail cost but said
the manufacturing cost is about $1,000 a month per patient.
The FDA denied approval for Ampligen in November 2009 because of
concerns about the way two studies were conducted -- too few
patients, a protocol change and an early end to one study. This
time around, the FDA will review a new analysis of one trial result
submitted in 2009, but not a new study.
It's possible that a lack of supporting data could again hold up
approval. "Multiple conduct issues with the trial suggest that
results should be interpreted with caution," the agency said in a
letter to the committee. "The confirmatory trial failed to
replicate" the results of the first trial.
In September, the FDA conducted a teleconference with people
with chronic fatigue syndrome who had used the drug. These
anecdotal reports were largely positive. What weight, if any, these
patient experiences will have on the drug's approval isn't
Some experts think chronic fatigue syndrome is a virus; others
believe it is a bacteria. It can begin after an illness from which
a patient doesn't quite recover, or the symptoms can appear almost
overnight, McCleary said.
Symptoms often include flu-like weakness. The one common thread
is the inability to do almost anything without becoming totally
exhausted, McCleary said. Even simple tasks like reading a magazine
can set off a cascade of symptoms that last for days or weeks, she
"It's a bone-crushing exhaustion," McCleary said. "There is pain in the muscles and joints that can move from one body part to another, sore throat, headaches like migraines, and trouble falling asleep or staying asleep."
There are mental symptoms as well, including difficulty
processing information and a "big problem" with short-term memory,
The FDA's decision is expected early next year. The agency isn't
bound to follow the recommendations of its advisory panels but it
usually does so.
For more information on chronic fatigue syndrome, visit the
U.S. National Library of Medicine.
Please be aware that this information is provided to supplement the care provided by your physician. It is neither intended nor implied to be a substitute for professional medical advice. CALL YOUR HEALTHCARE PROVIDER IMMEDIATELY IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY. Always seek the advice of your physician or other qualified health provider prior to starting any new treatment or with any questions you may have regarding a medical condition.
Copyright © EBSCO Publishing. All rights reserved.