From the Desk of Dr. Kaufmann I am very pleased to report the progress of our trial entitled "Clinical Trial of High Dose Coenzyme Q10 (CoQ10) in ALS". Thanks to your help and support, we were able to surpass our goals in terms of patient participation here at the Eleanor and Lou Gehrig MDA/ALS Center as well as at the other sites that are participating throughout the US. Clinical trials are a collaborative effort between researchers, clinicians and patients. Without patient and caregiver support and their efforts in taking the study medication and coming to the center for visits, clinical trials would not be possible. Clinical trials are an important step in bringing new treatments to patients. Patients and clinicians cannot be sure if a new medication is truly beneficial if it is not tested in a trial. Also, the FDA will not allow the use of a new medication for ALS without controlled trials. Many patients have asked us why this trial is called a "Phase II" trial. Clinical trials are divided into three phases: Phase I trials typically have less than 20 participants, often healthy volunteers, who first take a new medication at different doses to make sure that it is safe. Phase II trials are the next step, typically have less than 200 participants and are there to find out if there is some evidence that the new medication is beneficial. Phase II trials cannot definitely answer the question if a new drug is truly effective. However, they can tell us if a new drug is worthwhile pursuing in a Phase III trial. A Phase III trial is a definite trial to test a new drug’s efficacy. It typically has several hundred participants and takes several years. The FDA usually requires two positive Phase III trials to approve a new drug. The clinical trial of CoQ10 in ALS was based on encouraging results from a pilot study conducted by Sheila Hays at the Eleanor and Lou Gehrig MDA/ALS Center and Dr. DiMauro, an expert in mitochondrial disease and CoQ10. Dr. Mitsumoto and I, working with statisticians here at Columbia University and ALS centers around the country, have followed up on the pilot study with a Phase II clinical trial, funded by the National Institute for Neurological Disorders and Stroke. CoQ10 is a promising treatment for ALS because it is a mitochondrial co-factor and powerful antioxidant. Mitochondria are the "powerhouses" of the cell, and they are thought to have impaired function in ALS. Free radicals that can be neutralized with antioxidants are also thought to play a role in ALS. The trial will be completed in late spring of this year. With the results, we will be able to answer the question if it is worthwhile pursuing CoQ10 as a treatment for ALS in Phase III. The trial will not definitely answer the question if CoQ10 is actually beneficial in ALS. Only a Phase III trial can show this. You may ask why we did not start a Phase III trial right away. The reason is that Phase III trials are very large, expensive and take a long time. When there are several new drugs on the horizon, it is an efficient strategy to test some of them in Phase II so that we can have at least preliminary answers more quickly and without using up all the resources. Some patients who have already completed their follow-up time in the CoQ10 trial have asked us why we cannot tell them right now what type of medication they have been taking. The reason is that in a controlled trial, not only the patients but also the investigators are "blinded", meaning that they don't know if the patients was taking CoQ10 or placebo. We can share the treatment assignment with patients once the analyses are completed which we anticipate to occur in mid- April. We want to again thank everyone for their support and their contributions to this trial. Most of all, we want to thank patients and caregivers who have participated and generously given their time and effort. Clinical trials can not only benefit those who directly participate, but they can help others with ALS. Clinical trials allow us to rationally test treatments, eliminate harmful or ineffective treatments rapidly, and, hopefully, find an effective and safe treatment for ALS soon.