Whitehead, Hafler hope effort will open doors to drug development
July 19, 2002
Allison Connolly Journal Staff
CAMBRIDGE -- The Whitehead Institute and a well-known Boston neurologist are teaming up to draft the first-ever genome of multiple sclerosis, a complex neurological disease that has long baffled scientists but is now believed to be caused by a number of genes.
If successful, the project could do for the disease what the Human Genome Project has done to dramatically advance drug development. It is expected to yield new information about what causes the disease and may alter how biotechnology and pharmaceutical companies discover drugs to treat it.
"I do believe this is real -- it's not a flash in the pan," said Dr. David Hafler, director of the molecular immunology lab at Brigham and Women's Hospital, who is taking a sabbatical from the hospital in order to work on the project. "It will fundamentally change how we look at disease. But we have many years of work ahead of us."
Hafler and a team of researchers from Whitehead plan to raise "tens of millions of dollars" to support their research. They have established the MS International Consortium as well as three centers of research: at Whitehead, the University of California at San Francisco and Cambridge College in England. Each center is beginning to collect data on MS patients and to map haplotypes (common sequences between genes) associated with the disease.
"We hope to get at the genetic cause of the disease," said Eric Lander, director of the Whitehead Institute Center for Genome Research in Cambridge.
Symptoms of the often-debilitating disease are caused by the inflammation and breakdown of myelin, a molecular substance that coats nerve fibers in the brain and spinal cord. The myelin is destroyed, leaving lesions, or "plaque," which interfere with the transmission of signals along the nerve pathways. However, the severity of the disease varies from person to person, with some having few symptoms from time to time to others who are unable to walk.
Hafler began work on the project in January, when he left Brigham and Women's Hospital for a sabbatical. He sought the tutelage of Lander and his team, and Hafler now splits his time between an office at the Whitehead building and his office at Harvard.
Lander and the Whitehead are well known for their work on helping the public effort to map the human genome, which was published in February 2001. The Whitehead has been using genetic information learned from mapping the genome to relate it to the cause of disease, so drugs can be made more effective. And in some cases, by mapping new genes, scientists have learned that some diseases thought to be environmental are in fact genetically linked. For example, last week, Waltham-based Genome Therapeutics Inc. published its discovery of the first ever gene to be linked to asthma, which was thought to develop over time due to environmental factors like dirty air. The company found the gene by comparing the genetic history of families in Iceland. It is similar to the approach that Lander and Hafler plan on taking in studying the genetic data of MS patients.
"I think it's a very exciting project," said Steve Sookikian, a spokesman for the central New England Chapter of the National Multiple Sclerosis Society. "It offers hope for people with MS for better treatments."
Currently, there are five MS drugs on the market: Avonex, made by Cambridge-based Biogen Inc.; Copaxone, made by Israeli-owned Teva Pharmaceutical Industries Inc.; Betaseron, made by Montville, N.J.-based Berlex Laboratories Inc.; Novantrone, made by Seattle-based Immunex Corp.; and Rebif, which is made by Geneva-based Serono SA and was approved for market recently.
According to the MS society, nearly $32 million will be spent this year
on more than 300 clinical investigations into the disease. About $10 billion
is spent each year in the United States on health care costs related to
Copyright 2002 American City Business Journals Inc.