Chronic Fatigue Syndrome Patients Need an Effective Therapeutic
Released: 1/24/2013 4:00 PM EST
Source Newsroom: Nova Southeastern University
(more news from this source)
Newswise — Ampligen, the first drug ever seeking approval to treat
chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME), recently
hit another roadblock with the U.S. Food and Drug Administration
(FDA). In its long quest to treat 1 million Americans suffering from
this debilitating illness, the FDA advisory panel did not recommend
the drug to be sold on the market, largely because CFS/ME doesn't have
clear biomarkers such as blood tests to define patients who most
likely to respond to the drug. Data from clinical trials of Ampligen
has not convinced the FDA so far.
The real loser is not Ampligen, but CFS/ME patients whose daily suffering continues to be unabated. CFS/ME feels like you've been run
over by a truck – pain, inflammation, utter exhaustion and trouble
I have been caring for patients with CFS/ME for 26 years now. It's
heartbreaking seeing them struggle and suffer from this serious
illness that has been trivialized by science and society. One of the
early controversies quickly disproven suggested that CFS/ME is a form
of depression. This led to enduring public policies that allowed
insurance companies to limit coverage to CFS/ME to either mental
health or exercise therapy, neither get to the root cause of CFS/ME.
CFS/ME researchers, including myself, have seen major advances in our
understanding of the biology of CFS/ME. It seems to resemble an
illness we know how to treat like multiple sclerosis (MS), chronic
viral diseases and autoimmune diseases.
Around since the late 1980s, this drug is not new to science and
medicine. Two phase 3 clinical studies have been completed. The data
shows that a subgroup of CFS/ME patients showed marked improvement,
even recovery on the drug.
Yet, that's not enough evidence for the FDA advisory committee to
approve because they would like to see a conclusive biomarker. As a
physician, I could live with this decision if I had other effective
therapies to treat my CFS/ME patients. But I do not.
Moreover, it defies common logic in used in drug approval for other
complex immune mediated diseases.
Take for example, MS: Its earliest approved treatments had opposite
immune effects. One interferon increased immune activity and a second
interferon quieted immune activity. In the studies that led to
approval, MS drugs, like Ampligen, had about a 40 percent success
Clinical research for these early MS drugs produced no biomarkers
other than a patient's successful response to therapy, such as the
case of Ampligen. The biomarker the FDA relied on for approval of MS
--- seeing if the lesions in a patient's brain decreased -- had no
correlation to the patient's improvement.
Why would the FDA approve MS drugs before there were concrete biomarkers to determine success? The answer is simple. The advisory panel saw MS as a serious disease that required interventions ASAP, and were willing to accept that clinicians would better understand where to use the first drugs with more experience using them. Now there are seven approved drugs for MS that have significantly improved quality of life for patients. But they are not willing to use the same logic for Ampligen.
Because CFS/ME patients are stilling waiting for their first
therapeutic, Nova Southeastern University (NSU) in Fort Lauderdale,
Fla. has brought on board a nationally renowned group of experts to
form the NSU College of Osteopathic Medicine Institute for Neuro
Immune Medicine, which will open in February. These doctors and
scientists will conduct basic research using genomics to help further
develop drugs to treat this disease, while they treat patients. They
are also doing clinical testing for Ampligen.
With or without a biomarker, the FDA should recognize the seriousness
of CFS/ME and approve Ampligen, and open the door for other targeted
Nancy Klimas, M.D., one of the world's leading researchers and
clinicians in chronic fatigue syndrome/myalgic encepahalomyelitis
(CFS/ME), is the director of the Nova Southeastern University's
College of Osteopathic Medicine Institute for Neuro Immune Medicine