DR. MADHAVARAO PRESENTS RESEARCH AT CANAVAN SUMMIT

Among the researchers who presented at the Canavan Research Summit 2008 was Dr. Chikkathur Madhavarao, now in the third year of research funded in part by a grant from the Canavan Foundation. Dr. Madhavarao's goal is to advance understanding of the connection between the gene mutation that causes Canavan disease and the associated deterioration of the brain that the disease brings to its victims.

Canavan patients, Dr. Madhavarao and his mentor Dr. Namboodiri discovered, have a deficiency in the supply of ‘acetate’ in their brain since they are unable to break down an important brain chemical, N-acetylaspartate (NAA). The brain utilizes the acetate coming from NAA to make components that are used to insulate nerve fibers.

Dr . Madhavarao tested a treatment strategy in Dr. Namboodiri’s lab of supplementing acetate in the diet of ‘tremor rats’. These rats mimic the Canavan disease condition, since, like Canavan patients, they do not have a functional gene for breaking down NAA. Initially he was encouraged by signs that lab rats, especially females, responded to treatment. Last year he reported that treated rats, females most of all, showed improved motor skills and increased levels of some myelin lipids, which are used as nerve-insulation material.

At the Summit, Dr. Madhavarao reported that the oral acetate treatment unfortunately did not have the desired effect on one of the most severe symptoms of Canavan Disease, the formation of vacuoles or tiny pockets inside nerve cells. Although the number of vacuoles decreased, they still persisted. Perhaps, he believes, the ASPA enzyme (the gene for which is mutated in Canavan disease) performs additional roles that are as yet poorly understood.

Dr. Madhavarao plans to continue investigating how the brain creates and uses myelin proteins, and exploring the functions and mechanisms of the ASPA enzyme. He hopes this research will lead to further insights into the workings, causes, and potential treatment of Canavan disease.

Dr. Madhavarao is supported by the Rosalind Poss Rosen Clinical Research Training Fellowship, co-sponsored by the American Academy of Neurology Foundation and the Canavan Foundation.

-end-

FOUNDATIONS JOIN FORCES TO FINANCE RESEARCH FOR CANAVAN DISEASE

ST. PAUL, MN (July 14, 2003) - The Canavan Foundation and the American Academy of Neurology Foundation today announced the sponsorship of a new $100,000 Clinical Research Training Fellowship to support research towards the cause, treatment or cure of Canavan disease. Fellowship applications are due October 1, 2003.

This joint investment in research offers new hope for the patients and families of those affected by Canavan disease, a rare and fatal genetic disorder that affects the myelin sheath that insulates and protects nerves and the brain. Children with Canavan disease cannot crawl or walk, may suffer from seizures or mental retardation and cannot perform activities of daily living. A relentlessly progressive disease, Canavan disease generally results in death by 10 years of age. It is believed that research advances may lead to treatments or even a cure.

The new joint Clinical Research Training Fellowship provides a fresh opportunity to recruit the brightest young investigators in the neurosciences today to join the team dedicated to solving this puzzle. Under the auspices of the AAN Foundation, proposals are being solicited from across the country and around the world. Preference will be given to clinicians with an interest in a long-term career in clinical research who are able to demonstrate the support of a mentor and sponsor institution to conduct the proposed investigation. PhD candidates conducting clinically-oriented research will also be considered.

"We believe this is a prime opportunity to make research dollars do double or even triple duty," said AAN Foundation Chair Kenneth Viste, MD. "We hope that this fellowship makes a significant contribution to our understanding of Canavan disease and at the same time, supports and nurtures a promising young investigator who will continue to work in this area for years to come."

More information about the fellowship and application forms can be obtained from the AAN Foundation at www.neurofoundation.org or by calling 651-695-2712. Information about Canavan disease can be found at www.canavanfoundation.org or by calling 877-422-6282.

The AAN Foundation is a 501c3 nonprofit organization dedicated to advancing support for public education and research in neurology. The Canavan Foundation is also a 501c3 organization dedicated to education, prevention and research in Canavan disease.

-end-