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Experimental Drug Helps Fight Some Childhood Cancers, Study Finds
Date:5/17/2012

By Serena Gordon
HealthDay Reporter

WEDNESDAY, May 16 (HealthDay News) -- A new targeted drug therapy may help treat certain advanced cancers in children, a new preliminary study indicates.

In some cases, the oral medication even made tumors disappear after regular cancer treatments had failed, the researchers reported.

"This is an exciting first step, and it looks very promising for kids who have had very few options," said study author Dr. Yael Mosse, an assistant professor of pediatrics in the division of oncology at Children's Hospital of Philadelphia.

Mosse is scheduled to present the findings on June 2 at the American Society of Clinical Oncology (ASCO) meeting in Chicago. However, ASCO released the results during a news conference Wednesday. Funding for the study was provided by the drug's manufacturer, Pfizer Inc.

The new drug is called crizotinib (Xalkori), and it targets abnormalities in a gene called ALK that's present in certain cancers. The drug was recently approved by the U.S. Food and Drug Administration for treating adult lung cancers caused by an ALK defect.

ALK is not "expressed" -- or produced -- in healthy individuals. Only cancer cells express ALK, according to Mosse. "ALK is there to promote growth of cancer cells," she explained. That means any treatment that blocks ALK will only affect cancer cells and not healthy ones.

"Healthy cells don't recognize this drug. It's much different than chemotherapy," Mosse said.

The three types of pediatric cancers currently targeted for this drug therapy include anaplastic large cell lymphoma (ALCL), a type of blood cancer; neuroblastoma, a nerve cancer that usually affects the adrenal gland or the chest; and inflammatory myofibroblastic tumors (IMT), which often causes tumors to develop in muscle.

ALK genetic abnormalities are found in between 80 percent and 95 percent of anaplastic large cell lymphoma cancers, about hal
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