After their twin daughters were diagnosed with Niemann-Pick Type C, a fatal genetic disease, Hugh and Chris Hempel sought experimental treatments to save their daughters’ lives. They went public with their findings and kept detailed medical records to share with researchers, assuming the role of “citizen-scientists”:

Ms. Hempel began giving cyclodextrin to the girls in early 2008. “I am posting this message to the entire world to let everyone know that Hugh and I will not sue any doctor, scientist, researcher, hospital or non-profit if anything happens to Addi and Cassi as we embark on trying experimental treatments to save them from Niemann-Pick Type C disease,” she wrote in her blog.

From the beginning, scientists said drinking cyclodextrin wasn’t likely to help because not enough of the drug could reach the brain and other organs.

In December 2008, the Hempels’ doctor applied to the Food and Drug Administration for permission to give the girls intravenous infusions. The scientists were alarmed. No one knew how cyclodextrin worked. No one knew effective dose levels. And no one was sure if it was safe.

The planned collaboration of parents and scientists was moving forward. But Chris and Hugh Hempel wanted to set the pace.