As we end the year 2001, in addition to being busy with holiday plans, we at the ALS Center are busy with new clinical trials. We have started the clinical trial of Indinavir (a protease inhibitor), with a small number of patients (pilot study). It is a double-blind study involving a placebo, to test the medication’s feasibility and potential benefits in ALS. Hopefully the outcome of this trial will lead to further investigation with a larger population of patients.

We are also busy with a new CoQ10 study, which is led by Dr. Petra Kaufmann. In this study we are looking at the short-term effects of CoQ10 on chemical and metabolic properties of motor neurons in the brain. If it is found that CoQ10 has such effects, we will be more convinced that CoQ10 must have an important impact on diseased motor neurons.

Celebrex is yet another clinical trial we are about to begin. All the paper work (business contracts and human subject research approval) have neared completion, so we hope to begin enrolling our first set of patients by January.

Another important project is to identify chemical and physiological changes in upper and lower motor neurons from the very early stages of ALS (Vol. 13 No. 2). Until we understand the disease process and mechanisms, it will continue to be very hard to assess the efficacy of potential medications and determine how the drugs work. This kind of study is imperative. We have begun testing the machine for multi-slice, multi-voxel magnetic spectroscopy (different from the currently available technology) and diffusion tensor imaging which are novel, state-of-the-art technology. We can measure the chemical and structural integrity of upper motor neurons. We desperately need healthy volunteers, ideally gentlemen who are older than 50 years old. This is an important preliminary stage of investigation. In the spring of 2002, we will start a large-scale investigation in patients with ALS. The National Institute of Neurological Disorders and Stroke have funded this research. The investigators are now preparing to tackle this big project in the New Year.

We have some extremely important news at Columbia University Health Science Center here in Washington Heights. We are about to have the first meeting for a new research initiative for ALS here at this medical research center. As you already know, Dr. Serge Przedborski, who gave us his research update in the last newsletter, and I are taking the initiative to assemble a group of extraordinary investigators at our campus.

Thomas Jessell, PhD is a world leader of motor neuron development and its circuit development, has been an instrumental

component in developing this new ALS research initiative. Our Dean, Dr. Gerald D. Fischbach, who joined us last year after serving as the Director of National Institute of Neurological Disorders and Stroke, and himself is the world’s top neuroscientist in the field of neurodegeneration, is most supportive of this gathering. Other world-class investigators include Dr. Lorna Role, whose expertise is in the connection of muscle and motor neurons; Dr. Peter Scheiffele a rare world expert of cell structure of motor neurons; Dr. Ronal Liem another world expert of cytoskeletons (bony structure of the cell); Dr. Arthur Hays, ALS neuropathologist; Dr. Truman Brown who brings to us novel neuroimaging technology; and, Dr. Lewis Rowland who will provide the clinician’s view to this basic research assembly.

Personally I feel so fortunate to have incredibly sophisticated world-class neuroscientists and neurobiologists at our campus. Our department Chairman, Dr. Timothy A. Pedley, is putting all these efforts together and is most supportive of developing research breakthroughs and a cure of ALS.

I know everyone at the Center is already so busy with new clinical trials, but I hope we also have a wonderful time during the next years being even busier with research and clinical activities. All of us wish everyone who reads this newsletter a joyful holiday season and peaceful and rejuvenating New Year.