FDA approves drug tested at Children's

BUFFALO, N.Y. (WIVB) - Buffalo's medical community played a major role in developing and testing a newly-approved treatment for a devastating disease.

When Reed Lesmeister was 2-weeks-old, his parents learned that he has cystic fibrosis.

Dad Todd Lesmeister said, "We were terrified. I mean, we had a beautiful young baby and two weeks later we found out he had a disease that - we didn't even know what it was. So we were very scared."

Cystic fibrosis is an inherited disease that causes breathing problems, recurrent lung infections, difficulty with digestion and poor growth. Reed got excellent care, and when he was three, he was present when Women and Children's Hospital announced that they would be the first hospital in the country to test a new drug that might change the course of the disease.

The drug worked extremely well. Results of the international study were published last November, and the FDA has already approved the drug.

Dr. Drucy Borowitz said, "We're proud that we're the trailblazers. This really is the first drug developed in this way that targets a particular mutation. It's the first drug that is personalized medicine."

Reed was too young to try the new drug back then, but he'll be six this month, and he's eligible to take it. Up to now, he's been taking medicine to control the symptoms, and getting regular check-ups and he's doing pretty well.

The team at Women and Children's Hospital is also working on additional drugs that may extend the benefits to many more cystic fibrosis patients.

"It is a devastating disease, and to be able to change the course of that disease is really - it's really a wonderful thing," Dr. Borowitz said.

The drug that's been approved targets one of many mutations that may cause cystic fibrosis. But the strategy that was used to develop it can be applied to many other cystic fibrosis mutations - and many other diseases as well. This really is part of a new generation of personalized medicine.