BIOGEN IDEC UPDATE
We are very hopeful that January, 2013 will bring the long-awaited results of Dexpramipexole (R+Pramipexole) to the forefront following the analysis by Biogen Idec of its Phase III Clinical Trial that ended October, 2012. If the unblinded results are as impressive as we feel they will be, there should be FDA approval shortly thereafter and commercialization of Dexpramipexole (availability through pharmacies by prescription and insurance coverage) in September, 2013. This view is an amalgam of communications with many sources and our own perspectives. With a high level of dosage dependency in its prior trials, a very positive sign, Dexpramipexole appears to both slow progression in ALS and improve survival time, perhaps by increasing the efficiency of mitochondria, the energy producing organelle in the body's cells. Mitochondria in the motor neurons undergo significant stress in ALS patients, and Dexpramipexole improves energetic functions of stressed mitochondria. A complete explanation of how Dexpramipexole affects the mitochondria is available at Brain Research 2012.
Biogen Idec Dexpramipexole Identifier Number NCTO1281189*
Earlier trials of Dexpramipexole suggest that it improves mitochondrial function and offers significant protection to neurons under stress. Its effect is to notably ameleorate the symptoms of ALS even to a point of some reversal. Phase I and II trials demonstrated relief of both limb and bulbar symptoms. The benefits of the drug appear to be dose-dependent as the higher dosage of 300 mg daily was significantly better than the lower dosage of 150 mg daily. At this time, participants are being recruited into the Phase III trial. Specific centers throughout the US, Canada and Europe have been identified and that information is now on the Biogen-Idec website. It is a double blind, placebo controlled, multi-center trial which means that participants may or may not receive the medication. Patients must be able to visit the clinic every other month.
Why this trial is notable
Dr. James Bennett, esteemed neuroscientist formerly of the University of Virginia and currently Director of Neurology at Virginia Commonwealth University, believed that by reversing the molecule through a complex process, the mirror image of Pramipexole miight provide better results for those with ALS. Phase I and Phase II trials were completed in 2009 and Phase III has begun to demonstrate efficacy. Dr. Merit Cudkowiicz is the International Primary Investigator of this trial as well as Ceftriaxone. Biogen Idec, A Weston, Massachusetts based biotechnology company has joined Knopp Biosciences of Pittsburgh to provide expertise and financial resources in what they believe will improve mitochondrial function, sigificant protection to neurons under stress and minimize or even improve the symptoms of ALS.
The first Phase II Clinical Trial detail appeared in the November 20 issue of Nature Magazine. It is written by Dr. Merit Cudkowicz and colleagues from Knopp Biosciences and details treatment, dosage and mortality effects. The paper is titled Effects of Dexpramipexole and can be read in its entirety.
Issues of Concern
Patients cannot have experienced any symtoms prior to 24 months pre-trial entry.
Patients must have an FVC value of 65% or greater.
Patients may receive the placebo in a 1:1 ratio of medication/placebo.
The Dexpramipexole Phase III trial is perhaps the most important ALS trial thus far. It represents the first great chance for a successful, measureable, helpful and perhaps even life-saving pharmaceutical product to come along thus far. Those who qualify by not having symptoms prior to 24 months should contact the site closest to their home and request to be included in the trial. Even if you receive the placebo, you will be first in line to receive the medication once it is given FDA approval.
Here are the specific site locations both in the US and internationally for the Phase III Dexpramipexole Clinical Trial as of April 20, 2011. Not all sites are currently enrolling, but they can be contacted by patients wishing to participate in this important clinical trial
Please read the fascinating story of Dr. Bennett's efforts.