From the Desk of Hiroshi Mitsumoto, MD

While we have been working very hard at the Eleanor and Lou Gehrig MDA/ALS Research Center, I realize it quickly comes time to write our next Newsletter. I am so delighted to share a lot of things that are happening at our Center and a first of its kind Motor Neuron Center at Columbia University.

We finally completed the NIH study investigating the utility of objective markers in ALS and related diseases. In total, more than 90 individuals (64 patients) participated and many of them came every 3 months having repeated testing with magnetic resonance spectroscopy, transcranial magnetic stimulation, motor unit number estimate, and clinical examination. We are deeply grateful to all of the individuals and caregivers, who helped our research, for their time and commitment. We know this study will provide a definitive study in objective markers, currently we are in midst of all analyses.

In clinical trials, we are the coordinating center of two major multicenter clinical trials supported by the NIH. Dr. Petra Kaufmann is in charge of CoQ10 study, and our statistical colleagues headed by Dr. John LP Thompson are managing a model web-based data entering system. The study is ahead of schedule. Another study, headed by Dr. Paul Gordon, is the minocycline clinical trial which is rapidly winding down in the enrollment. While we are still looking for eligible patients who can participate, to those who are already in the study we are deeply grateful for your participation. As you know, we are committed not to slow down, even a bit, our maximum effort to find the second and better medication for the treatment of this disease. Manganoporphyrin (Aeolus 10150), a potent anti-oxidant, which markedly slows down the disease in ALS mice, is now under investigation. We participated in the first stage of the study, a single injection, with increasing doses used at different study sites. Now we will study the effects of repeated injections for a week. In the late fall, we are supposed to participate in a number of novel medications, including a medication which is said to improve protein aggregate in the motor neuron, a drug which decreases effects of toxic amount of glutamate, and another anti-oxidant. Those medications have strong theoretical background and have worked in the ALS mouse model. As you can see, we will continue to test all potential medications.

Another project is an MDA-funded genetic and environmental epidemiology study. This is to find the cause of the disease, since this disease is highly likely due to an unusual combination of our genetic makeup and environmental and behavioral factors. It has been slow finalizing the interview structure, however we are now moving to the second phase. We are looking for candidates who are newly diagnosed and have relatives who can help us with the study. Participation involves one blood sample for DNA and cell-line and an extensive (in person or telephone) interview. This study is closely linked to our effort to get a new bill for a New York State ALS Registry. The bill passed the NYS Senate in May 2005, and now it will be presented at the Assembly. In the summer, we met State Assemblyman Adam Bradley, who is most supportive of sponsoring this important bill. If you would like to help, please write your Assemblyman to support the bill for NYS ALS Registry. The ALS Registry seems to be moving forward even at the Federal level. Unless we study all patients for genetic and environmental factors, we may not crack down this mysterious disease. We need the help and support of your letters.

Last but not least, I wanted to introduce to you the Columbia University Center for Motor Neuron Biology and Disease. To our knowledge, this is the first such center dedicated to motor neurons in the world. This sort of Center can only happen at Columbia because we already have tremendous expertise and talent existing here at our Campus. Our Dean, Dr. Gerald Fischbach, and Chair of Columbia’s Neuroscience Center, Dr. Thomas Jessell, who are both top leaders of motor neuron biology, are instrumental in developing this Motor Neuron Center (MNC). Dr. Serge Przedborski, who is the Science Director of our Center, is a world famous expert in translational research in ALS and neurodegenerative diseases. Dr. Daryl DeVivo is another world expert of pediatric neurology, particularly at the forefront of spinal muscular atrophy (SMA) research at Columbia. SMA is the child counterpart of adult ALS, both diseases, ALS and SMA, attack motor neurons. We strongly believe these killer diseases have common neurodegeneration and cell death processes. If we understand the mechanisms in one disease, ALS or SMA, that knowledge will surely help the other. The same principles apply for discovering novel medications. In May of this year, Dr. Chris Henderson joined this group from France to formally establish The Tow Center. Dr. Henderson is another world renowned expert in translations research in ALS and SMA. These three investigators, Drs. Henderson, Przedborki, and DeVivo are co-directing the MNC. The Founder of our Eleanor and Lou Gehrig MDA/ALS Research Center, Dr. Lewis P. Rowland, who has accepted to be an Honorary Director of the Motor Neuron Center, put this development as, “This is a major development in the history of the Medical Center, for neuroscience, and for patients with terrible diseases.” The first inaugural faculty meeting took place on November 2 and will be followed by monthly grand rounds, with research activity on the way. I must say, I am so thrilled and excited. I moved here 6 years ago to direct our ALS Center, and our efforts working with patients are now finally matched with enormous effort at the basic and translational science level.

I have asked Dr. Petra Kaufmann to tell you about a DNA Banking project organized by the North American ALS Research Group (see page 3). Now I have to get back to my work.

With all best wishes,
HM