The antiviral drug Ampligen has supposedly been the big breakthrough in chronic fatigue syndrome (CFS) treatment for 20 years. The long wait for patients looks set to continue and the reason ironically seems to be that clinical trial data submitted by Hemispherx Biopharma Inc., the drugs maker, is too old and doesn't meet new criteria.
Hemispherx submitted a New Drug Application (
NDA) with the US Food and Drug Administration (FDA) back in early October asking for the drug to be approved as a treatment for CFS. The company this week revealed that the FDA had found 11 problems in the application's clinical section and three in its pre-clinical section.
A spokesperson for Hemispherx said it was believed part of the problem with the application may be that clinical data reporting guidelines have changed since the initial studies on Ampligen were executed in the late 1980s and early 1990s. The company says it will respond in writing to the FDA as a matter of urgency and seek a meeting to clarify the situation.
On reading this news I couldn't help but think "how can a pharmaceutical company such as this get an application so wrong, surely they would have filed other NDAs over the past 10 years and be familiar with current guidelines?" After visiting the company's website however it turns out they are a small firm with only a handful of products on the market or currently under development, one of which is Ampligen itself.
Still, it will be hard for many people suffering from CFS to understand why a company such as Hemispharx couldn't aprise themselves of the current FDA guidelines prior to submitting their application for Ampligen and get things right first time.
With initial clinical trials of Ampligen in CFS patients taking place 20 years ago this must be one of the slowest drug development programs in history. Of course I would never suggest a drug be rushed to market until its safety and efficacy had been clearly established but surely things could be sped up if silly mistakes were avoided.
As regular visitors to this site are no doubt aware I, as a sufferer of CFS, multiple chemical sensitivity and other chronic health issues, try to avoid drugs unless absolutely necessary. It has been my personal exprience that most are at best useless and at worst harmful. Antidepressant drugs have been particularly ineffective for me and I have tried quite a few from various different classes, including the newer combined serotonin and noradrenaline reuptake inhibitors (SNRIs) such as Venlafaxine.
The reason I am writing about Ampligen here is that so many people I respect believe it holds a great deal of promise. In addition it targets the underlying immune dysfunction which is believed to be at the heart of CFS. It is thought that the inability of the immune system to deal with infections properly, particularly viruses, in some people starts a cascade that leads to the disabling condition we know as CFS.
Dr. Paul Cheney is a leading light in the CFS community and someone I greatly respect as he really seems to "get" the illness and patient's experiences of it on every level. In the early 90s Dr. Cheney said:
"Ampligen can down-regulate an anti-viral enzyme pathway, which is excessively turned on in most patients with Chronic Fatigue Syndrome. It also down-regulates the production of tumor-necrosis factor alpha. Besides its immune modulating effects, Ampligen also has direct anti-viral effects. Ampligen in our experience and in others, causes a significant improvement in cognitive dysfunction and a general lessening of all symptoms seen in Chronic Fatigue Syndrome."
Let's hope that the NDA for Ampligen gets back on track soon and that the drug is true to its promise so that the suffering of millions with CFS can be avoided or at least greatly reduced.