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At the Eleanor and Lou Gehrig MDA/ALS Research Center - The Neurological Institute of New York Columbia University - a number of research projects are underway including: an ALS Cohort Study of Multicenter Oxidative Stress that is examining how genetic profiles, environmental and lifestyle factors, diet and psychological stress relate to an ALS patient's level of free radicals (oxidative stress). This study is funded by the National Institute of Health with a total 3.3 million dollars funded over a 5 year period; Non-Invasive Ventilation Research to identify the most effective ways that ALS patients can use non-invasive ventilators; An OEP study to investigate novel equipment called optoelectric plethysmography (OEP) to measure lung/breathing capacity without putting on a mouth piece. Only a dozen are available in the country. We are also conducting several drug trials (Neuraltus phase II and Knopp phase III studies) to identify effective treatments for ALS. Furthermore, we are working to develop a new scale that would tell us if medications tested in future clinical trials have clinically meaningful changes, not just statistically meaningful changes, for patients and their caregivers. We have collaborated with investigators at the New York State Psychiatric Institute to investigate the different rate of use of tracheostomy with invasive ventilation in ALS patients between the United States and Japan. We are working with a team at Cornell Medical center to compare brain neurochemicals in ALS patients and healthy controls using the Magnetic Resonance Spectroscopy technique. The Motor Neuron Center’s basic research colleagues are working on a study involving using a patient's skin cells for genetic engineering to be regrown into motor neurons, the nerve cells that become damaged in ALS. We are also currently preparing a grant for the first multidisciplinary study of early PLS that may have a strong therapeutic implication for treating ALS. At the ALS Center of Columbia University, we are attacking this devastating disease from all angles. We receive major funding from NIH, MDA and MDA Wings Over Wall Street as well as other philanthropic donations.

The Eleanor and Lou Gehrig MDA/ALS Research Center hosted the International ALS Conference from September 7th to September 9th at the Tarrytown House Estate and Conference Center. This revolutionary conference brought together 150 individuals that included national and international physicians, scientists, government agency representatives, pharmaceutical company representatives, etc. in an effort to promote clinically-oriented research to find the pathogeneses and cause of ALS. In fact, Outreach ALS Foundation of Westchester supported this most successful conference. This Conference, which became a platform for critical discussion, was desperately needed for the future of ALS. It was crucial for these key players and stakeholders of ALS to come together to review our current status of knowledge, as well as to plan for ways to move forward in the realm of research. We are thrilled to report that the Conference was highly successful and will hopefully inspire a new wave of research on both a national and international level. We are also in the process of organizing and publishing a supplement of key points and discussion that took place during the Conference as well.

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Although the cause of ALS is not completely understood, the recent years have brought a wealth of new scientific understanding regarding the physiology of this disease. However it is likely that ALS is a complex multi-system disease with several mechanisms that cause the death of motor neurons. There is, however, only one form where the cause is unequivocally known: the inherited form of ALS caused by a mutation in the copper/zinc superoxide dismutase enzyme (SOD1). Giving this mutated molecule to a normal mouse causes an ALS like disease. Completely inhibiting production prevents the disease from developing. Therefore, there are three studies underway now that are aimed at inhibiting the production of this enzyme. One is being conducted at Weill Cornell Medical Center using a drug called pyrimethamine that has been shown in preliminary studies to lower SOD1 levels in the spinal fluid. The two other studies use a drug called arimoclomal (Emory University) and antisense therapy (Washington University).

At Weill Cornell, three other studies are being conducted. One is investigating the role of pyrimethamine in patients with sporadic (i.e. non-familial) ALS because the two types of the disease are so similar. The second is investigating the role of nutrition (using the ketogenic diet as a means to strengthen the cell) and the third is a phase 3 nationwide study being conducted to determine the effects that ceftriaxone has on the rate ALS is progressing.

In addition, the Department of Neurology at The Hospital for Special Surgery is participating in the international multicenter trial of NOGO.

To date, there is only one FDA approved drug, riluzole, that has been repeatedly shown to slow the progression of ALS. We all hope that there will be more therapeutic agents to help us slow or reverse the weakness caused by this relentlessly progressive disease.