Dying for lifesaving drugs: will desperate patients destroy the pharmaceutical system that produces tomorrow's treatments?

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Dying for lifesaving drugs: will desperate patients destroy the pharmaceutical system that produces tomorrow's treatments?

TEN YEARS AGO, doctors drilled a hole into John Gotschall's skull, inserted two catheters, and pumped a poison into his brain. Using a child's morphine pump, the team of neurosurgeons pushed diphtheria toxin into Gotschall's temporal parietal lobe over a period of four days. Eight weeks later, they did it again, reusing the same cavity and pumping in a slow stream of tissue-killing fluid.

Gotschall was not well. Shortly before he invited a team of surgeons to experiment with his cerebral tissue, the 44-year-old municipal worker had plowed his car into a snow bank on a Baltimore street. When he woke up at the hospital, doctors told him he'd had a seizure at the wheel. An MRI revealed the cause of that seizure: a tumor deeply embedded in his brain tissue. There are different grades of brain tumor, many of them slow-growing. Gotschall had glioblastula multiforma (GBM), brain cancer in its most aggressive and deadly form, and he likely had only months to live.

Gotschall's physicians initially ordered chemotherapy and radiation, the same weapons with which doctors have fought cancers for decades. Neither worked. After he exhausted the standard options, he started searching for nonstandard ones. His neuro-oncologist pointed him to a group of doctors at Johns Hopkins and a drug in development called TransMID. TransMID had survived Phase I testing, in which researchers evaluate a medication's safety and appropriate dosage, and was then in Phase II, in which researchers begin to evaluate efficacy. Gotschall finally caught a break: He qualified for entrance into the trial. Along with 43 other patients, Gotschall would have a chance at a radical new treatment that might add years to his life.

TransMID is a ...

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