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Lehigh Valley doctors hopeful FDA maneuvers will fix pediatric cancer drug shortage

In the short life of Sam Grady, dates are divided into two categories: days that came before the 3-year-old's July 1 diagnosis of leukemia and the days that followed. Before July 1, the Lehigh Township boy loved tearing through the house with his older brother Eli and watching "SpongeBob SquarePants." Since then, he has lost his hair and grown nauseous from his chemotherapy; it took weeks for his parents to adapt to their new reality. His mother, Pauline Grady, said she feared a third time period for Sam: the dates after her son missed taking methotrexate, the medicine essential to his recovery. Doctors at Lehigh Valley Hospital-Muhlenberg in Bethlehem administered the drug to him Monday via spinal tap, but national reserves of the drug were reduced to a two-week supply at the time. "It's worth its weight in gold," Pauline Grady said of the drug. She took a photo of it Monday, not knowing if it was the last time her son would receive a dose of the medicine. Dr. Leslie Simpson, one of Sam's physicians, described methotrexate as a miracle drug for battling leukemia in children. Without it, children face a stronger risk of the cancer spreading to the brain, which would require radiation treatment. Children receiving radiation face strong risks of brain tumors, delayed growth and a drop in IQ, Simpson said. Without drug, brain at risk As supplies dwindled, the hospital began to reschedule cancer patients to receive care at the same time so they could use medicine from the same vial, Simpson said. Her clinic treats 20 children in the Lehigh Valley for leukemia, and she estimated another 10 to 15 families in the area travel to Hershey or Philadelphia for similar treatments. "We really need this drug to keep the leukemia from going to the brain," she said. Concerns of a shortage have dropped in the last week, and local hospital officials are hopeful rationing won't be needed. The Food and Drug Administration approved foreign imports of the drug Tuesday as a short-term solution. The FDA also cleared two American companies to begin producing it. It could take weeks for production to meet the demand, however. "We're cautiously optimistic that this will be a cork in the damn," Simpson said. Patrick Ferguson, network director of pharmacy for St. Luke's hospitals throughout the region, said the drug fell into short supply after Ben Venue Laboratories, one of the nation's largest suppliers of methotrexate, shut down its production in November over quality concerns. Without the company's supplies, stocks of the drug dropped to dangerous lows. "This is one of the worst times I can think of in pharmacy of trying to get a hold of a drug," Ferguson said. He added that St. Luke's had an adequate supply of the drug, which is also used to treat rheumatoid arthritis, but St. Luke's treats fewer patients needing the drug than Lehigh Valley Hospital. Seeking a production incentive Simpson and Ferguson said the near shortage was created in part because of economics. Medicine for children with cancer is a niche market that doesn't receive much funding, so pharmaceutical companies move on to more profitable ventures. Pauline Grady plans to head to Washington, D.C., to attend Childhood Cancer Action Day. On June 4 and 5, she and other advocates will meet with members of Congress in a push to get more funding for pediatric cancer research. One of the measures supporters are pressing for is the Creating Hope Act, which would put profitable pharmaceuticals on the fast track for approval if the companies making them increase production of less profitable, but needed, medications. The scare families received last week proved the need for such a law, Grady said. "Crisis averted for four weeks. But what's it going to be in May?" Grady said. U.S. Sen. Bob Casey, D-Pa., is one of the bill's supporters. He and a group of bipartisan senators called on the president of Ben Venue Laboratories to explain what forced the company to shut down production of methotrexate. In the meantime, Simpson said Sam Grady has responded fabulously to the treatment. Pauline Grady said there have been days she's felt like quitting. But then she sees Sam laughing and smiling with his brother, and it gives her the strength to continue. "For a 3-year-old with leukemia, he's doing great. He's the definition of resilience," Grady said.

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