Friday, September 12, 2008

Harvard researchers find protein link to cystic fibrosis early death

By Mass High Tech Staff

Researchers at Harvard Medical School have revealed new information about a key protein, missing or faulty in cystic fibrosis patients, that could lead to new treatment, according to published reports.

The protein CFTR exists in healthy people for the purpose of recognizing a lung-damaging bacteria and removing it. In patients with cystic fibrosis, this protein is either missing or faulty, causing them to develop chronic lung infection and almost always leading to early death before the age of 35 years, ScienceDaily reports.

The researchers intend ultimately to find points where cystic fibrosis patients can benefit from drugs to develop stronger resistance to infection.

In July, a report revealed that the U.S. market for the treatment of pulmonary symptoms of cystic fibrosis grew more than 15 percent annually to $397 million, from 2000 to 2007. New England companies have helped lead that growth.

Lexington-based Epix Pharmaceuticals Inc. grabbed a deal in April worth up to $37.7 million with the Cystic Fibrosis Foundation Therapeutics Inc. to develop a drug for the disease. And Cambridge-based Vertex Pharmaceuticals Inc. reported positive results in March from an ongoing Phase 2a clinical trial of a drug that targets a protein linked to cystic fibrosis.

Lexington-based Predix Pharmaceuticals Inc. also has a an agreement with the Cystic Fibrosis Foundation Therapeutics Inc., worth up to $16 million, for research into small-molecule therapeutics and initiation of screening efforts.

And Cambridge-based FoldRx Pharmaceuticals Inc., a company focused on protein misfolding diseases, signed a deal with the Cystic Fibrosis Foundation to develop new drugs for cystic fibrosis worth $22 million over five years.