2010  DIRECTOR'S MESSAGE AND PROGRESS REPORT

Since the founding of CNS more than 30 years ago, there has been a remarkable change in health care and an almost unbelievable expansion in our knowledge of diseases that involve the nervous system. And since CNS has played a part in this we feel justified in celebrating our accomplishments.  From our inception we had three main ideas: support patients and their families, conduct  basic research and develop therapies for currently untreatable neurodegenerative diseases. 

It is my belief that CNS has enjoyed considerable success in fulfilling these commitments. We were among the first to initiate support groups that put patients and scientist in the same room on a regular basis and our online site was among the first on the WEB to disseminate information.

With the move to the Scripps campus, we have expanded our clinical research effort. As a result, we are now conducting studies in the area of Alzheimers disease, epilepsy, neuropathy and pain, in addition, to our longstanding research on the treatment of ALS and MS.  Dr Jonathan Licht is primarily in charge of these activities under the auspices of Coordinated Clinical Research which is affilitated with CNS.

One exciting outgrowth of CNS's effort has been the issuance of World wide  patents based on discoveries made at CNS which led to the development of Zenvia which has been licensed to Avanir Pharmaceutical Corporation. Three clincial trials involving patients with ALS and multiple sclerosis have shown that Zenvia is highly effective form of symptomatic treatment.  Avanir has announced that FDA will complete its approval process by October 2010.  If approved this will be a milestone and we earnestly believe that the full medical value for the product has not been fully demonstrated. We are working to remedy this. 

While DMQ is a powerful symptomatic treatment work initiated by CNS could lead to the first meaningful treatment for the familial form of amyotrophic lateral sclerosis, Huntingtons disease and the like. Like most good ideas this one is simple. It turns out that many neurodenerative diseases, such as ALS, are likely caused by the accumulation of proteins that interfer with the normal health of a cell. In 1993 it was discovered that mutations in the gene coding for superoxide dismutase are responsible for about 20% of the familial cases. But we still don't know how this and many other such mutations actually cause disease. And it may be a long time before we understand what is going on. But we now know, thanks to work spearheaded by CNS in collaboration with Don Cleveland(UCSD) and Tim Miller(Washington University), and Frank Bennett and others at Isis Pharmaceutical corporation that lowering the amount of  mutant protein in the nervous system increases survival in an animal model of ALS. Earlier the first patients were enrolled in the phase 1 study to test whether the therapy is safe and to determine the pharacokinetic profile of the drug.  A total of 36 patients will be studied at 6 centers throughout the United States. Harvard(Merit Cudkowicz), John Hopkins (Jeff Rothstein),  Carolinas Health Care System (Benjamin Brooks), Methodist Hosptial in Houston(Stan Appel), Scripps Hospital in San Diego(Isaac Bakst) and Washington Univ. 

None of these accomplishments would have been possible without the support of the ALS and Muscular Dystrophy associations and contibutors to CNS. In this regard I particularly wish to thank the Thagard Foundation, and the families and friends of Tom Shea and Murry Sandler, particularly Basil Witt. In difficult economic times your help is particularly appreciated.

With best wishes for the year ahead.

Richard A. Smith, M.D.