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Transplant Drug Shrinks Tumors in Women With Rare Lung Disease
Date:1/10/2008

he division of nephrology and hypertension at Cincinnati Children's Hospital, said in a prepared statement. "Our data suggest that mTOR inhibition with sirolimus may hold promise for treating these and other disease manifestations in patients with TSC and LAM."

Bissler and his colleagues treated 20 patients who had angiomyolipomata tumors with sirolimus for 12 months. They reported that, at the end of the year, the tumors shrank by about half. Eleven of the patients who had LAM and took sirolimus for the year also showed a 10 percent to 15 percent improvement in expiratory air flow, a standard measure of lung function.

The researchers followed up with 18 of the original patients after a year without the sirolimus treatment. They found that tumor size had increased to about 85 percent of original pre-treatment size.

Tumors in five of the original patients, though, were 30 percent smaller than their original size after a year without the medication. The study authors theorized that this could be a result of apoptosis, a kind of programmed cell death.

After a year without treatment, the patients' lung function declined but was still better than what could be expected from similar patients after two years without any intervention, the researchers said. The improved lung function could be because of the lessening of obstructions and trapped gas in the lungs, the researchers noted.

Patients reported side effects from the sirolimus treatment that included mouth sores, diarrhea, upper respiratory infections and joint pain.

The researchers acknowledged that the small study needs further follow-up. A phase III clinical trial of the drug as a treatment for LAM patients is planned, they said.

More information

To learn more about LAM, visit the American Lung Association.


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