Founded in 1981Educate, inspire, and connect those touched by ichthyosis and related disorders through emotional support, information, advocacy, and research funding for better treatments and eventual cures.
Dr. Ervin Epstein, Jr., UCSF, San Francisco, CA
Dr. Leonard Milstone, Yale University, New Haven, CT
Dr. Barbara Gilchrest, Boston University, Boston, MA
There are many brilliant and dedicated minds working on rare inherited skin disorders.
The Foundation for Ichthyosis and other skin disease patient advocacy groups supported and attended an Obstacles to Translation Conference in early March of 2006. The Obstacles to Translation Conference, addressed the obstacles that have prevented the skin disease community from translating known information about the genetic basis and physiological pathways of inherited skin diseases into successful therapies.
The conference, organized by Dr. Ervin Epstein, Jr., UCSF, Dr. Leonard Milstone, Yale University, and Dr. Barbara Gilchrest, Boston University, brought together representatives from academic science, biotech and pharmaceutical companies, venture capital groups, the government, and patient advocacy groups to identify the barriers to translation and to emphasize strategies to surmount them. The program was divided into eight sessions, each focused around major topics important to the development of molecularly targeted drugs for inherited skin diseases. The topics included; the drug development process, financial issues, targeting DNA, and the regulatory process, among others.
Two days of detailed presentations and intensive discussion ended with all the participants dividing into breakout groups. The breakout groups were each given a specific obstacle to discuss and were charged with returning to the group at large with a short list of possible solutions to the specific problem. The obstacles identified for discussion were; obstacles to drug discovery, obstacles to translation of promising therapies, obstacles to various institutional cultures (academic, corporate, health delivery, government institutions, etc.), and obstacles to raising money.
Several interesting and varied suggestions came out of the discussion groups. The outcome of this conference offered good news and bad news for the Foundation and other patient advocacy groups.
The good news is:
The bad news is:
So where do the Foundation and other patient advocacy groups go from here?
The consensus is that we need to: