Tuesday, April 12, 2011

Biotech and cystic fibrosis: A kid's view

By James M. Connolly

The star of the show was an 8-year-old Red Sox fan from Dedham, not the 200 globe-trotting investors and accomplished life sciences executives who were both the attendees and speakers at the Boston Biotech Conference.

Fresh-faced Bobby Coughlin, son of MassBio president and CEO Robert Coughlin, warmed the audience at the Mandarin Oriental Hotel in Boston when he shared his life experiences with cystic fibrosis. He sat with his father on a panel discussion focused on development of therapies for rare diseases.

In five minutes of questioning by his father and moderator Rich Aldrich of Longwood Founders Fund, Bobby explained how he takes 50 pills per day, sometimes four at a swallow, with some of those pills designed just to help him digest his food. When asked about the worst part of having CF, Bobby said, “The nebs (nebulizer). I have to do it while the other kids are outside playing.” He said that besides that misting treatment, he also misses play for chest physical therapy.

With the panel discussing topics such as the work of disease foundations, the U.S. Food and Drug Administration, funding models and research, Aldrich asked the 8-year-old if he understood what the panel and audience were talking about, and he honestly smiled, “Not really.”

After Bobby’s talk, it was his father’s turn at the microphone. Emphasizing that he was speaking as a parent and not as the president of a 650-company association, Coughlin focused initially on the FDA’s decision to send Eli Lilly’s liprotamase treatment for CF back to the lab for more research. Coughlin said of liprotamase, “It works. The FDA is asinine. The regulatory process does not make sense.”

Coughlin noted that he had been active in the CF Foundation even before Bobby was born and before he himself knew he was a carrier. He said that while drugs get bogged down in the approval process, parents of children with CF “hear a clock ticking.”

Coughlin called for a new approach to lobbying efforts by rare drug associations and pharmaceutical companies. “Don’t use the CEOs to lobby. Bring in the people who have the disease. Wheel them in there so people can see what they are dealing with.”

Another panelist, CEO Alex Margolin of Alnara, which helped to develop liprotamase, said that one issue with rare diseases is the lack of independent experts who can serve on FDA review panels. Most experts are tied to a company and ineligible to serve on a panel, so experts are drawn from unrelated disease fields. “It’s the blind leading the blind,” said Margolin.