InterMune Inc. (ITMN)

[someconcerns]

Tiny Germ-Fighting Molecules Mean Survival From Rare Immunity Deficiency
By Nicole Ostrow -

A U.S. study of a rare illness that wipes out the immune system may help doctors assess which patients should receive bone-marrow transplants.

The severity of chronic granulomatous disease, a genetic malady affecting about 1 of every 200,000 people, reflects the number of germ-fighting molecules called superoxide rather than the way the condition is inherited, researchers said today in the New England Journal of Medicine. Gene tests can predict superoxide production, helping determine whether a patient needs a transplant, the most aggressive treatment, the authors said.

The study “gives the physicians a tool, a marker, of who is going to do well and who is not going to do well,” said John Gallin, the senior study author, in a telephone interview on Dec. 27. Gallin is the director of the National Institutes of Health’s Clinical Center in Bethesda, Maryland. The NIH funded the research.

The estimated 1,200 people in the U.S. with the immune deficiency have an increased risk of bacterial and fungal infections, according to the NIH. Treatments include lifelong antibiotics and antifungal medicines, injections of InterMune Inc.’s Actimmune, and bone-marrow transplants for those with the disease’s most-severe forms. Even trace amounts of superoxide can help patients survive the disease, the researchers said.

Chronic granulomatous disease affects about 25,000 people worldwide, according to the NIH. Patients’ white blood cells don’t make enough hydrogen peroxide and bleach to fight off infections, Gallin said. The patients are at risk of long-term and repeated infections that may lead to tissue masses’ obstructing the bowel or urinary tract.

Blood Samples

The researchers measured superoxide in blood samples taken from 287 people with the disease and compared the amounts with those in people without the deficiency. Patients ranged in age from 1 year to 64 years.

Those with the highest levels of superoxide had the best survival and were less sick, the researchers said. Patients with the lowest levels were about 5.5 times more likely to die from the disease than those with the highest levels, Douglas Kuhns, the lead study author and a scientist at SAIC-Frederick Inc., an NIH contractor in Frederick, Maryland, said yesterday in a telephone interview. The company is a unit of McLean, Virginia- based SAIC Inc.

Before the study, the severity of the illness had been thought to depend on how patients inherited the genetic mutation that triggers the condition, the researchers wrote. Patients who got it from their mothers on the X chromosome tended to have a more-severe form than those who got it from both parents, Gallin said.

Now the researchers have shown that the severity of the disease depends on the production of the germ-killing molecules, he said.

‘Tiny Bit’

“If they make just a tiny, tiny bit, they will do better than those who make none,” Gallin said.

Intermune’s drug, which helps stimulate the immune system, is expensive, he said, so researchers are trying to determine which patients are most helped by the Brisbane, California-based company’s treatment. Actimmune generated $25.4 million in 2009 sales, according to data compiled by Bloomberg.

“We basically have now provided a new tool to clinicians that allows them to assess the severity of the disease and what the survival is in patients and identifies those patients we need to be more aggressive with in therapy,” Kuhns said.

The findings may help doctors who treat patients with other immune system illnesses better understand those conditions, he said.

To contact the reporter on this story: Nicole Ostrow in New York at [e-mail].

To contact the editor responsible for this story: Reg Gale at [e-mail]

http://www.bloomberg.com/news/2010-12-29/tiny-germ-fighting-molecules-mean-survival-from-rare-immunity-deficiency.html?cmpid=yhoo