From the Desk of Hiroshi Mitsumoto, MD Dear friends, the spring is quickly changing into summer. We have had another busy spring, which means some of you also have been quite busy in helping us with our studies by participating in some of our research. We are actively recruiting patients for the minocycline clinical trial, a large National Institute of Health (NIH)-funded clinical trial for which Dr. Paul Gordon, Associate Director of the Center, explained the scientific rationale in a previous newsletter. We are also engaged in a clinical trial with TCH346, which is expected to suppress the cell death process of degenerating motor neurons. We have completed enrollment for this large international study. Additionally, we have several patients who began a TCH 346 open-label study as a follow up to participating and completing an earlier phase II study many months ago. We are actively recruiting patients for the study of high frequency chest oscillation treatment for investigating potential benefits of this airway clearance system (called VEST®). The NIH-funded “Markers of ALS Study” has only one more year to go. We reported the preliminary results at the annual Neurology meeting in San Francisco in April. We found the novel technology of multivoxel (brain tissue is divided and sliced into many small sections for special biochemical measurement) magnetic resonance spectroscopy to be highly reliable in detecting upper motor neuron dysfunction, shown by chemical abnormalities, in motor neurons of the brain. Furthermore, transcranial magnetic stimulation, which is able to measure the speed and sensitivity of an impulse from the brain to lower motor neurons residing in the cervical and lumbosacral spinal cord, has also demonstrated a high reliability of objectively detecting upper motor neuron dysfunction. These results are only preliminary and more data needs to be accumulated. In all of these clinical studies, mentioned above, the patients who kindly participate must come every month or every three months for the evaluations. We are deeply grateful for the commitment that these patients and their families have extended to the study and us. I have to say that we are making a big dent in this terrible disease with good teamwork. We are working very hard to finalize a new questionnaire for the genetic environmental epidemiology study. This study, which is funded by the MDA and Wings Over Wall Street, will study the interaction between our genetic make-up and environmental exposures. There may be some weakness or condition that makes a person prone to have ALS that we can identify. Certain environmental exposures or certain specific personal behaviors may negatively impact upon weak motor neurons. This will be a long-term future study, but making the right questionnaire is the critical first step. When the questionnaire is established, we are going to test the reliability of the questionnaire both by personal interview and telephone interview. Only after this stage is completed can we launch the main project of a genetic environmental epidemiology study. In the next few months, we will be ready to begin the first stage. We are preparing for our next large multicenter study with Coenzyme Q10 (CoQ10). CoQ10 is a normal enzyme present in the mitochondrial (power house generating cell energy) membrane and functions as a potent anti-oxidant. Dr. Petra Kaufmann, who has received a large NIH research grant, will be in charge of this important study. Colleagues from the Biostatistics Department of the Columbia School of Public Health (Drs. John P. Thompson and Bruce Levin) will function as the Biostatistical and Data Coordinating Center. In one of the next newsletters, Dr. Kaufmann will bring you up to date with detailed information. We have been working with Dr. Edward Kasarskis from the University of Kentucky closely for the past few years. Dr. Kasarskis has just been awarded a large NIH-funded grant to study ALS management. He has been most persistent and finally received this groundbreaking grant, as this is the first of its kind investigating the benefits of early gastrostomy or early BiPAP use for patients with ALS. We routinely use nutritional care and respiratory assistive devices, but we do not know when is the optimal time to start, or what are the benefits of those treatments. This is a prospective study, that is, research based on a hypothesis, which is carefully planned in advance and executed based on a detailed plan. If these treatments are really proven to be helpful, there will be enormous implications, including insurance coverage and scientific approach to ALS management. As one of eleven sites, we will start this project this winter. Again, we will keep you informed. Actually we have a few more exciting projects to tell you, but I will share them on the next occasion. As you see, we are really working hard. I personally appreciate our research staff: Jackie Montes, our physical therapist, who is on her way to receiving a MA degree, is coordinating our clinical trials. Vanessa Battista is our research assistant, working for the Marker studies and TCH program. Carolyn Doorish is our new research assistant, rapidly expanding her capability in the minocycline study. Nayra Gad, MA, is a national multicenter coordinator for the minocycline study and new staff, Alexandra Barsdorf, MA, has just begun her work as a national multicenter coordinator for CoQ10. Grace Hillyer, MPH, is a project scientist and is finalizing the epidemiology questionnaire in ALS. Nancy Hirshan just joined us as our new Divisional Administrator so we can function more efficiently. We have administrative assistants, Anisara Thankachan, Catherine Amar, Katherine Narbonne, and Arthurine Hall, who help us enormously to make our system work effectively. Maura Del Bene, MS, RN, NP-P, has been instrumental for all of our clinical and research activities but has decided to move on to a different career path. Our Center owes her greatly. It is personally sad news for all of us, but we all understand and respect her decision. Everyone who worked for this Center will miss her. We wish her the very best in her career. I am certain, whatever career she chooses, she will do an outstanding job as she did for ALS. She set the bar very high as an ALS nurse clinician. We are in the midst of actively recruiting her replacement. We are certainly determined to continue to provide the highest care for those who have this difficult disease. Until then, I wish you all the best. Hiroshi Mitsumoto, MD