Gene therapy fights immune disorder

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Gene therapy fights immune disorder

BY PEGGY O'FARRELL | ENQUIRER STAFF WRITER

Cincinnati,OH

http://news.enquirer.com/apps/pbcs.dll/article?

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An international team of researchers has used gene therapy to

correct an immune deficiency disease in two men.

The two men, diagnosed with a genetic immune disorder, were able to

overcome life-threatening fungal infections after receiving the gene

therapy, according to the study in Nature Medicine, a biomedical

research journal.

The infections had not responded to standard therapies. The disease

is caused by the lack of an enzyme that leaves the immune system

unable to fight off certain infections, including those caused by

fungi and some bacteria. The findings " show how to cure a whole

category of diseases, " said Christof von Kalle, an adjunct associate

professor at Cincinnati Children's and a co-author of the study.

Kalle is director of the National Center for Tumor Diseases at the

German Cancer Research Center. Researchers in Germany, Switzerland,

England and Cincinnati Children's Hospital Medical Center cooperated

in the analysis of the study.

" It's too early to call the results a cure, " Kalle said. " We know

too little. But it's a great improvement. "

Scientists used genetically altered viruses, called vectors, to

deliver genetic coding that corrected the enzyme deficiency in the

two men. The coding " switched on " the men's ability to produce the

missing enzyme and fight infections.

A key finding of the study, published in Nature Medicine, showed

that placing the vectors next to certain genes increased the body's

ability to produce infection-fighting blood cells.

In this case, Kalle said, the patients' ability to fight infection

increased about 60 percent after the therapy.

The patients received their therapy about 18 months ago at a

hospital in furt, Germany. Doctors will continue to follow them

to see if their immune response remains strong and to check for

potential side effects from the therapy.

Children treated with gene therapy in France in 2002 developed

diseases similar to leukemia.

Follow-ups to the French study showed the virus vector carrying the

therapeutic gene was inserted close to a gene that causes cancer to

develop and the insertion triggered the development of the cancer.

Because gene therapy makes cells grow rapidly, there is concern that

it could cause malignancies to develop, Kalle said, but in the case

of the furt study on X-linked chronic granulomatous disease,

early indications show cells grow rapidly, then die off before

becoming malignant.

, director of experimental hematology at Cincinnati

Children's, said the new study shows gene therapy can be used to

successfully treat genetic disorders in children. The men's response

to the therapy was " extraordinary, " said.

E-mail pofarrell@...