Posted by Michael Wonder on 07 Jun 2018
The ‘right to try’ law says yes, the drug company says no
6 June 2018 - Vibhav Rangarajan and Sonal Patel, doctors in Chicago, learned in April that their 2-year-old daughter, Radha, has a rare genetic disease called late-infantile metachromatic leukodystrophy that is expected to cause rapid deterioration of her nervous system.
Like most 2-year-olds, Radha speaks in two-to-three-word sentences. (“Go outside!” “Mama sit.”) She gives kisses to anyone who asks.
There are no available treatments designed for the disease, though several are in development. In May, Radha’s doctor applied to Shire, an Ireland-based biopharmaceutical company focused on rare diseases. The request: for “compassionate use” of an enzyme replacement therapy that has been tested on a small number of children with MLD in an early clinical trial. Shire denied the request a day later, and again on appeal.
