Effective therapy needed for chronic fatigue syndrome patients

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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 concentrating.

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 rate.

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 therapies now.

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.


  1. Hannah Goodfellow Hannah Goodfellow United Kingdom says:

    As a CFS sufferer diagnosed in April 2011 the lack of treatment for my disease is disgusting.  Only treatment offered is CBT, being told by my therapist to fill out a activity log, use a step-o-meter and log how many steps I take a day.  Also being told that maybe CFS WAS a virus but its NOT now its my brain thats giving incorrect signals.  So I need to increase my activity.   I feel this is discrimination -  I'm in my house virtually housebound day after day after day.  Cant even walk upstairs, make a meal, feel like I've got flu, swollen glands, terrible memory, loss of my independence and job and the pain is unbearable.  PLEASE PLEASE give us something !!!!

  2. Jill Gamble Jill Gamble United States says:

    I tried Ampligen years ago and it did not work for me.

  3. Tracee Hemann Tracee Hemann United States says:

    Please see that this is our only chance at recovery right now there are no other approved drugs for cfs/me.  Don't we deserve the same chance the MS patients got when they first started research on treatments for them.

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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