Co-Enzyme Q10 in ALS: New Research Study at Columbia ’s ALS Center By Vanessa Battista, BA The MDA sponsored ALS Educational Support Group offers both patients and caregivers the unique opportunity to not only share in comfort and support, but also to take part in an educational experience. Each month, the group begins with a speaker from various disciplines ranging from physical therapy, to medical equipment, financial information, and periodic research updates, just to name a few. After the presentation, there is a chance to ask questions before splitting into two groups to continue to dialogue and to offer each other support. This past fall, several people attended the support group to hear Petra Kaufmann, MD, MSc, present on “Clinical Research in ALS with Co-enzyme Q10”. “Medications tested in ALS clinical trials are targeting one of the mechanisms thought to cause the disease”, Dr. Kaufmann, began. She continued by explaining that impaired mitochondrial function is thought to play a role in causing ALS. Mitochondria serve as the “powerhouses” of cells and provide cells with the energy needed to function properly. Free radical damage is another factor thought to contribute to the disease. Coenzyme Q10, commonly referred to as CoQ10, is a naturally occurring cofactor that is considered to potentially remedy both mitochondrial impairment as well as free radical damage. In animal experiments, scientists were able to show that CoQ10 is absorbed by the nervous system. More importantly, in so-called “transgenic SOD1 mice”, the animal model for ALS that were given CoQ10 fared better than those that were not. Dr. Kaufmann also noted that CoQ10 has shown good success in patients with Parkinson’s disease. Based on the promising results in Parkinson’s disease and in the transgenic ALS mice, the next step was to conduct a Pilot Study that would determine the safety of CoQ10 in humans with ALS. In this pilot study, 20 patients received either CoQ10 or placebo for 4 weeks. The medication was very well tolerated overall, but some patients had gastrointestinal symptoms such as stomach upset. The pilot study was not designed to evaluate whether the drug is effective. The next step is to find out whether there is evidence that CoQ10 is effective in ALS and to find out what is the best dose that should be taken. Pursuant to that, Dr. Kaufmann has received a National Institutes of Health (NIH) grant for a multi-center clinical trial to answer these important questions. The trial will have two stages: a first stage in which two doses are compared and a second stage in which the efficacy is evaluated. Given the seemingly positive results of CoQ10 trials for ALS thus far, Dr. Kaufmann mentioned that people should still be cautious and that not everyone with ALS should necessarily take CoQ10. She continued that, “there are side effects and it is not known whether it really helps or not. If everyone is on it, there will be no way to determine whether it is helpful or not”. Because the benefits of CoQ10 have not yet been properly established, it is safer for people to take it under controlled circumstances, such as in a clinical trial, where their health can be closely monitored. Also, in clinical trials, safety monitoring boards are involved that will ensure that the use of the drug or supplement being tested, (in this case, CoQ10) is safe for everyone involved. Also, it is important to mention that high dose CoQ10 in the well-absorbed preparation used in the trial is currently not available outside of the clinical trial. Lastly, CoQ10 in high dosages is very costly and is not presently paid for by insurance companies. Dr. Kaufmann concluded by reiterating that, as with any trial, it is important to remember that participants will contribute to the greater good of research, which has the ultimate goal of finding a cure for ALS. Her final comments touched on the ethics of clinical research trials when she said, “If we knew it was helpful, it would not be ethical to test it, and if we don’t know if it is helpful or not, it remains unethical to prescribe it.” For these reasons, more research must be conducted to further explore the implications and effects that CoQ10 has on ALS. Dr. Kaufmann’s presentation on “Clinical Research with Coenzyme Q10” was well received by everyone present at the ALS Educational Support Group. We hope that you will join us, the 4 th Monday of every month at the All Souls Church (1157 Lexington at 80 th Street), from 6-8 p.m. for other exciting topics such as this. For more information regarding the group, please kindly contact Vanessa Battista, Support Group Facilitator, at 212-305-1677, or Leah Bailin, ALS Health Care Services Coordinator, at 212-689-9040.