Cystic fibrosis trial shows promise

[Guest guest]

Cystic fibrosis trial shows promise

04/30/03

Harlan Spector

Plain Dealer Reporter

The first human experiments using a gene therapy technique pioneered in

Cleveland appear to have succeeded in correcting the faulty cellular process

responsible for cystic fibrosis. Researchers at University Hospitals and Case

Western Reserve University began the clinical trial one year ago with 12

patients who received a corrected gene in a saline solution dripped into

their nasal passages. Cystic fibrosis patients have a genetic defect that

causes the buildup of thick mucus in the lungs and digestive system.

Scientists using a gene-transfer system developed by Cleveland-based

Copernicus Therapeutics Inc. found that the patients suffered no ill effects

and that the therapy appeared to normalize the nasal cells. The experiment

did not attempt to cure the 12 volunteers because the nasal passages were

merely a test site. Doctors don't know yet whether the gene transfer will

take hold in the lungs or how frequently therapy would be necessary as mature

cells die off and new, defective cells take their place. But in eight of the

12 patients, the cells produced enough protein to improve the transport of

salt and water, a critical balance that is upset in cystic fibrosis patients.

The gene controls production of the protein responsible for maintaining the

balance. Doctors are far from fixing the genetic cause of cystic fibrosis,

which affects 30,000 Americans. " Data is encouraging. We're continuing

clinical development, but it's not a cure yet, " said Dr. Moen,

president and CEO of Copernicus. The eight-employee biotechnology firm has a

$1 million state grant to develop an aerosol technology that will enable the

gene to be pumped into the lungs. Human trials are to begin next year. The

company licensed technology that compacts DNA - the structure that carries

the genetic code - so the corrected gene would be small enough to enter the

cell nucleus. The compacting technology is critical because viruses that are

commonly used to introduce corrected genes into cells are fraught with

problems for cystic fibrosis patients. Early gene therapy trials caused a

devastating inflammatory response. The 12 patients will have first crack at

enrolling in the clinical trial using aerosol technology for the lungs, said

Dr. Konstan, director of the Cystic Fibrosis Center at UH's Rainbow

Babies & Children's Hospital. " We've got to get it into the lungs, " said

Beall, president and CEO of the Cystic Fibrosis Foundation. " Then I'll

call it a breakthrough, if we see improvement in lung function. "

Stoutenberg, a Cleveland native who lives in Boulder, Colo., has been closely

following the clinical trial. Her 20-month-old son, Jack, has cystic

fibrosis, and she contacted Moen at Copernicus. " It seems so promising, " she

said. " If therapy works . . . Jack can have a healthy adulthood. "

© 2003 The Plain Dealer.

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