Wednesday, July 15, 2015

Precision Medicine Champions of Change

On Wednesday, July 8, 2015, I attended an event at the White House honoring 9 Champions of Change in healthcare. I've been thinking for a whole week about how I can share what I experienced there that day, and the best I could come up with was a storify. This captures the essence of the event - patients, parents, dedicated comrades, fighting together for equality of access, freedom of our data, and precision treatment options. This is Precision Medicine. This is what is going to save Drew's life. 

Thursday, July 2, 2015


We moved into our house in 2009. We had one daughter who had just turned one year old. Nine months later we welcomed twins, and our son Drew was born with cystic fibrosis. It was at that time that we learned that our neighbor had a 50yr old sister who had cystic fibrosis, the doctor across the street had a business partner whose granddaughter had cystic fibrosis, and another neighbor worked with a man whose grandson had cystic fibrosis. With only 30,000 people in the country with CF, the chances of us living among three other families affected by this disease was incredibly rare. This is the way the cystic fibrosis community is though. We celebrate each others victories and we mourn each others losses.
Today, a new drug called Orkambi was approved for use in patients ages 12+ with two copies the F508del mutation. It works to correct the genetic mutation that causes cystic fibrosis. No only will this add years to the lives of those living with cystic fibrosis, but it has the potential to dramatically improve the quality of their lives.
This is a remarkable day for us, for all of us; for those who will benefit from this medication and for those of us who will not. Our drugs will come, so for now we will celebrate with them because we know that when our drugs come they will be there to celebrate with us.