Rudiger Poe’s documentary focuses on Hugh and Chris Hempel, a California couple whose twin eight-year-old daughters are slowly dying from Niemann-Pick Type C, a rare neurological disease that is often referred to as childhood Alzheimer’s. There is no cure for their disease – or, for that matter, for thousands of rare diseases that impact nearly one in 10 Americans. Indeed, of the 7,000 rare diseases known to doctors today, only 200 have drug treatments approved by the U.S. Food and Drug Administration.
This film details how the Hempels aggressively researched potential treatments for their children – Chris Hempel determined that a drug called cyclodextrin would offer the best possible course of action – and then pushed doctors and pharmaceutical companies for help.
Running concurrent to this compelling real-life story is an investigation on why medical research appears to move so slowly in finding breakthroughs for rare diseases. This production claims that the laborious tenure process for medical researchers bears a lot of blame because it detours time and energy away from laboratory work in favor of raising one’s academic standing. Perhaps not surprisingly, given the input of pharmaceutical executives and representatives from the National Institutes of Health, there is no overt criticism of the drug companies or the federal government for dragging their respective feet (the initial lethargic response to the AIDS/HIV crisis in the 1980s was the most egregious example of their lack of help).
Nonetheless, this film offers a compelling wake-up call for more action to help disenfranchised people in dire need of medical treatment.