Genervon (GM604)

Genervon is an American pharmaceutical company that is behind a treatment called GM604. This drug is currently under investigation as a treatment for MND/ALS.

According to Genervon's press releases, 'very promising results' have been achieved from clinical trials conducted to date on 12 people over a 12-week period. The results of the trial were presented to the Food and Drug Administration (FDA), who license drugs in the USA, in February 2015.

Genervon requested an 'accelerated approval' for GM604, to make it available for people with MND NOW. The FDA has not published their decision yet.

Importance of rigorous trials

One of the possible reasons why the treatment has not been approved yet is because clinical and research communities are generally cautious about results from small, short-term studies, such as tests on GM604 to date.

In 2008 a clinical trial of lithium on 44 patients in Italy appeared to show a significant reduction in disease progression. Unfortunately, when larger trials were conducted they found no beneficial effect of taking lithium.

Publishing the results

Genervon has followed an unusual route for publishing its research. It has not published research in a peer-reviewed journal* that we are aware of, which makes it very difficult for us and others to assess its claims about GM604. Instead, they published their research article on the F1000 online platform**, where it is still waiting for peer-review.

If the company really has developed an effective treatment this would be of huge significance, but until its claims are rigorously tested we remain cautious.

The MND Association's Research Development team continues to follow the progress of GM604 and if its research is published in a peer-reviewed journal, or if the FDA (Food and Drugs Administration) in the USA gives approval for GM604 to be made available for patients, we will report back immediately.

*A peer-reviewed journal is one where published work has been evaluated by respected people qualified to comment on the subject matter. As a result, peer-reviewed journals are held in greater respect. Generally, scientists would only be confident in work that was published in a journal of this type.

**F1000 is an online open research publishing platform, where anyone can submit a research article to be read by the wide public and reviewed by invited or suggested experts. 

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