CMT Research Update: The Drug Discovery Process

by Keri Calandro : 1/20/2012 8:52:02 AM : Category: research

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A recent, informative article from the MDA outlined the process for bringing a drug to market.  What’s striking is the length of this process, particularly within the context of the search for a cure for Charcot-Marie-Tooth.

Once a drug appears to work effectively in mice, it must undergo further testing before it can be used on humans in clinical trials.   Over an approximate two year period, researchers work to determine if that drug has the best chance of having the desired effect on the disease with the lowest possible harm to the patient. Then toxicology studies must be done to show the U.S. Food and Drug Administration (FDA) that the drug is safe and likely to be effective.  Further they must determine how to administer the drug and at what dosage.  This preclinical research takes five to seven years and costs millions of dollars.

Once the preclinical research is satisfied, researchers can petition the FDA for approval to start clinical trials.  This is when the drug is used on humans. Clinical trials can take another five to seven years to complete.  Only when the clinical trials have finished with positive results can researchers request approval (from the FDA) to market a drug.   Simply put, “on average it takes a drug about 12 years to get from discovery to market and it costs about $1.8 billion per drug that works,” according to the article.

It is because research is such a lengthy process that HNF’s TRIAD (Therapeutic Research in Accelerated Discovery) is so important.   Our three-prong approach includes

1) Clinical Therapeutics: research that will lead to improved therapies and a better quality of life for people with CMT right now, including studies on bracing, exercise, surgery, and mental health strategies;

2) Basic Science: research that will lead to a greater understanding of CMT,  including translational research not yet applied to CMT; and

3) Drug Discovery: research that has considerable potential to produce a cure for CMT.  

We believe that this multi-tiered approach will best support people with CMT while we work to develop effective therapies, treatments and a cure for CMT.

What can we do right now while we are waiting?  The single most important thing for everyone is of course to keep moving, both mind and body!  Whatever level you’re at, keep exercising, stay tuned in, read all the latest news at HNF and the National CMT Resource Center.



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