Obstacles to Translation (2006)

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:

  • There are many brilliant and dedicated minds working on rare inherited skin disorders.
  • Researchers are making progress in knowledge of these diseases and are sharing that knowledge with each other.
  • The Federal government, specifically the NIH (National Institutes of Health) and the Office of Orphan Drug Development within the Food and Drug Administration, has several initiatives in place to help academic scientists move their research efforts forward.
  • Many people are thinking about translational research.

The bad news is:

  • It takes a great deal of time and money to for an academic scientist to reach the point where he or she can identify a promising drug or targeted therapy.
  • It takes significant time and money to move a promising drug or targeted therapy through the drug development process.
  • Biotech and pharmaceutical companies and venture capitalists are not interested in helping unless the drug or therapy has a large potential market value.

So where do the Foundation and other patient advocacy groups go from here?

The consensus is that we need to:

  • Keep raising money for research, because there is no promise that anyone else will step in.
  • Continue and refine lobbying efforts to encourage expansion of the research in rare diseases, with a possibility of focusing on expanding the FDA Office of Orphan Drug Development.
  • Be prepared to collaborate with other groups to identify promising "demonstration" projects (projects that have promise for more than one disease type) and pool money and resources to promote those projects.
  • Be prepared to step in to reduce regulatory or financial barriers by advocating for creative solutions, such as a national Institutional Review Board (IRB) for rare disease research. (Currently researchers have to get their own institutions' IRB approval for new or continuing projects, which makes collaboration with other scientists in other institutions and settings difficult.

    Read more about the conference here.

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