Sick Patients Need Cutting-Edge Drugs
Anna Tomalis was a bright, pretty, 13-year-old girl who liked
horseback riding and soccer. During the last few years, she rarely had
a chance to think about those things. Since September 2005, Anna
battled cancer. And, instead of wringing all she could out of
childhood, this courageous teenager tried to get members of Congress to
act like adults.
Anna had embryonal sarcoma, a rare form of
liver cancer. Surgery and chemotherapy seemed to work at first, but the
tumors came back. In March of this year, doctors told her there was
nothing more they could do.
She and her parents didn’t give
up, though. With a little research, they discovered a number of
experimental drugs that show promise in treating soft-tissue sarcomas
like Anna’s, including one called Deforolimus, developed jointly by the
drug companies ARIAD and Merck.
Unfortunately, Anna was too young and too sick to be admitted to the
clinical trials in which she might have gained access to one of those
drugs. And the Food and Drug Administration (FDA) very rarely grants
so-called compassionate-use exemptions to administer unapproved
medicines outside the clinical testing process.