FRIDAY, Dec. 21 (HealthDay News) -- A U.S. Food and Drug
Administration advisory committee on Thursday recommended against
approval of the first proposed drug to treat chronic fatigue
The advisors weighed the risks and benefits associated with the
intravenous drug rintatolimod (suggested brand name Ampligen). The
drug's maker, Hemispherx Biopharma of Philadelphia, also failed to
win FDA approval in 2009 because of concerns about the methodology
of clinical trials used to study the drug.
In Thursday's 8-to-5 vote, the advisory panel said the company
hadn't provided enough data to support the approval of Ampligen.
Although the FDA isn't bound to follow the recommendations of its
advisory committees it usually does so.
The central issue Thursday was whether Ampligen works and is
safe. The FDA said there wasn't sufficient data in clinical trials
to determine the drug's safety. Possible concerns include
infections and liver problems,
The Wall Street Journalreported.
Several panel members said they struggled with their decisions
because it seems the drug works in some patients, the newspaper
Experts have said they would welcome a treatment for chronic
fatigue syndrome, a disabling condition that affects as many as 4
million Americans, mostly women. There is no cure, but Ampligen
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 would do well on the drug, she
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.
Some experts think chronic fatigue syndrome is caused by a
virus; others believe it is linked to 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 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,
For more on chronic fatigue syndrome, visit the
U.S. National Library of Medicine.
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