Bioethics Blogs

When unapproved drugs are the only help: A case for compassionate use

Nathalie Traller with her mother, Vicki, outside the NIH Clinical Center in Bethesda, Maryland, in October 2012.[CNBC] wo years ago, Nathalie Traller spent her days like any seventh-grader might. She played soccer and swam and studied for classes. Then Nathalie started getting bad headaches, the kind that made it hard for her to concentrate.

A trip to the doctor revealed the unthinkable: a mass the size of a baseball in her chest. Cancer. It had already spread to her brain.

Nathalie was diagnosed with ASPS, alveolar soft part sarcoma, a cancer so rare that her oncologist, a sarcoma specialist, said Nathalie is her only patient with the disease. It makes up less than 1 percent of all sarcomas, which themselves make up just 1 percent of all cancers.

Nathalie, now 15, has since been through 10 surgeries, four medicines and several bouts of radiation to shrink the tumors in her brain and elsewhere. Still the cancer persists. Her doctors at the Knight Cancer Institute at Oregon Health and Science University in Portland have identified a new class of drugs they think could help her, but Nathalie hasn’t been able to access them. They’re still experimental, and Nathalie doesn’t qualify for the clinical trials: they start at age 18.

“She met the criteria in every way for a number of trials except for her age,” Nathan Traller, Nathalie’s dad, said in an interview in Portland. “So we were hoping maybe there could be an age exception made or some sort of provision so that she could try some of medicine’s best ideas. And we just kept finding that the door was closed.”

The Trallers are in a position countless others have been before: they’re out of options among approved drugs or those available through clinical trials.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.