For years, Christopher P. Austin has been trying to develop a way for science to build a better partnership with patients.
Dr. Austin is director of the National Center for Advancing Translational Sciences (NCATS), part of the NIH. NCATS is trying to find ways to get new treatments and cures for diseases delivered to patients faster.
At NCATS, Dr. Austin heads a state-of-the-art lab that uses robots to search for treatments.
In recent years, families of patients with rare diseases have made their way to the NIH to watch the robots at work in the $20 million, 30,000 square-foot system that includes refrigerators, automated incubators and computers. Machines work around-the-clock, testing hundreds of thousands of compounds against a variety of mostly rare diseases.
One of the first parents to visit was a woman whose college-age son had a rare cancer. During a tour in 2006, Dr. Austin told her that instead of 17 years to develop a new drug, he hoped that the lab could cut the time to 10 years.
“ ‘I love your technology,’ ’’ Dr. Austin recalled the woman saying. “ ‘I love your robots. I love this fancy stuff. But for my child and this disease, 10 years, 15 years, isn’t going to work. Isn’t there something else we can do?’ ’’
It is a question he hears again and again. There are roughly 7,000 known diseases; only about 500 have a treatment. Even with robots working day and night, Dr. Austin said, the arithmetic is discouraging.
Dr. Austin and the Journal’s Amy Dockser Marcus will answer readers’ questions about rare diseases and the research process. They are hosting an AMA (short for “Ask Me Anything). Ask your questions now.