ASCO Annual Clinical Advances Report 2013

Mention of biliary cancer – but not addressed in their comments. The never ending dilemma we continue to face in respect to research development.

At FDA Rare Disease Day I spoke with Stephen P. Spielberg, M.D., Ph.D., then Deputy Commissioner for Medical Products and Tobacco, about the difficulties patients such as our encounter. He acknowledged that foundations such as ours are the major vehicle in driving disease recognition and subsequent research forward. In his presentation he referred to the success of the Cystic Fibrosis Foundation, which was instrumental in bringing a drug to the market for those (I believe 5% of patients) found to have a specific gene mutation. This development took 20 years.

Fast forward to the Myelomas Foundation which, in my opinion has made enormous strides and sets an example for disease specific foundation. With their input and collaboration with specific researchers and biotech companies (large and small) their disease has been addressed in incremental steps. It still is considered a fatal disease however; life expectancy has greatly increased due to the development of specific life extending drugs.

Just thought to share.
Hugs,
Marion