Cancer medications geared to fighting genetic mutations are becoming more available.
Allan Brandt, a 63-year-old history of medicine professor at Harvard, seems taken aback to have become part of medical history himself. In 2012, he was diagnosed with acute myeloid leukemia, a deadly cancer that had a potential cure: a bone marrow transplant. His sister was a match, and he went through the two-month-long ordeal twice, only to have the cancer return. “No one is going to do well with a third transplant,” he says grimly.
He was saved by a hot biotech trend: medicines that are targeted to a protein made by a particular gene, often discovered by sequencing the cancer’s DNA. Sometimes they work in only a small number of people, requiring diagnostic testing. In Brandt’s case, Agios Pharmaceuticals, just a 15-minute ride from his Harvard office, had a drug that targeted a genetic mutation he was lucky enough to have. Only a few thousand of the 21,000 people diagnosed with AML each year do.
“This drug wasn’t available at all when I got sick,” Brandt says. “In the period of my illness, I’ve been able to get access to what I consider to be a lifesaving medication, and I’ve returned to health. As somebody who starts with a basic skepticism about medical technology and therapeutics, I have to say there are real possibilities for patients in these new developments.”
What drives drug companies to invest in markets of just a few thousand people? Speed is one reason–big benefits for very sick people mean smaller, shorter studies. One Agios drug, Idhifa, was approved by the Food & Drug Administration just 4 years after clinical trials started–a process that typically takes 12 years. Another reason to invest is price. Agios licensed Idhifa to Celgene, which charges $25,000 a month for it. (Brandt gets his medicine for free, because he’s in a clinical trial.)
Read full article: Fighting Cancer One Patient At A Time
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