CENTER FOR AMYLOID BRAIN RESEARCH AND THERAPY
Director, Blas Frangione, M.D., Ph.D.

The primary focus of the Center for Amyloid Brain Research is to study the genetics and biochemistry of cerebrovascular amyloidosis (disease resulting from a build up of an abnormal protein, amyloid) leading to dementia. Two decades ago, Dr. Frangione developed a method to identify, extract and analyze proteins from brain tissue that cause neurodegeneration and to analyze genetic defect in familial types of dementia and Alzheimer's disease.

In early studies focusing on a Dutch family with a history of dementia and stroke, Dr. Frangione, was the first to identify a gene mutation associated with dementia and Alzheimer's disease. Since that time, in collaboration with several international research laboratories, Dr. Frangione's group has studied families from the United States, Holland, Iceland, Finland, Poland, England and Denmark in order to understand gene mutations and how they effect neuronal damage causing familial and other forms of neurodegeneration.

In later work, Dr. Frangione and Dr. Thomas Wisniewski, director of the Institute's Neurodegenerative Conformational Disorders Laboratory, identified the protein, apolipoprotein E, which appears to play a role in brain amyloid formation. In the late 1990's Dr. Frangione and associates were able for the first time ever to block the formation of amyloid b in the brains of rats with a synthetic protein, which they called a "beta-sheet breaker". The beta-sheet breaker interferes with the folding of amyloid b into pleated sheets within the brain. In 2001, Dr. Frangione together with Drs. Thomas Wisniewski and Einar Sigurdsson announced the development of a "vaccine", which shows promise as a treatment for Alzheimer's disease. The vaccine reduced the amount of amyloid b in the brains of mice genetically engineered for Alzheimer's disease and is non-toxic - a difficulty found in a previous vaccine introduced by other laboratories approximately two years ago. Clinical trials of this new vaccine are expected to begin within one year.

Dr. Frangione's current projects include the exploration of the genetic and biochemical relationship between apolipoprotein E and Alzheimer's disease, the continued exploration of amyloid gene mutations in familial forms of dementia and their clinical presentation, as well as the development of new therapeutic approaches.