Perspectives on the Rett Syndrome Research Trust and Rett Syndrome research

Thomas R. Insel, M.D.
Director, National Institutes of Mental Health, NIH

"While the NIH, with a budget of over $28 billion, supports a broad range of science, most of our funding mechanisms are not designed to move quickly to support highly innovative opportunities. This is where private foundations play an important role.  With more nimble, proactive approaches to funding science, these smaller efforts can be critical for rapid progress in areas that are evolving quickly.  I view Rett Syndrome research as such an area. The recent rescue experiments are truly remarkable, as they suggest that therapeutics could be important in reversing the symptoms of Rett and other neurodevelopmental diseases. Monica Coenraads has been a tireless and effective advocate for moving Rett research forward.  I wish her well in the new venture, which is timely and potentially transformative."

Marc Tessier-Lavigne, Ph.D.
Senior Vice President, Research Drug Discovery, Genentech

"Rett Syndrome is in a unique and enviable position: the genetic cause is identified, excellent animal models exist, and recent data suggests the syndrome is reversible.  At this pivotal juncture the Rett Syndrome Research Trust has assembled a group of projects to carry the science toward the next milestone to clinical trials.  This ambitious and essential work is being spearheaded by an elite group of researchers and involves meaningful international collaboration.  Monica Coenraads, the Executive Director of the Trust, has been highly effective and influential in organizing and driving Rett research for nearly a decade.  With her experience, passion, and ability to engage high level scientific minds with the problem of solving Rett Syndrome, I believe the Trust is well positioned to lead the "de-risking" of early drug discovery, which will be necessary for the pharmaceutical and biotech industries to become interested in investing in this area.  This de-risking phase cannot be accomplished without significant financial support from private entities." 

Fred H. Gage, Ph.D.
Professor, Salk Institute, UCSD
Co-founder, Brain Cells, Inc.

"When developing therapies for very specific diseases, availability of experimental models that are tractable and productive is crucial. Additionally, it is important that a molecular underpinning of the disease has been discovered, on which new and deep knowledge can be built. Finally, links to other serious disorders which may benefit from the discovery of therapies developed for the disease justifies a strong incentive to put significant resources and energy toward this effort.

Rett Syndrome fits all these criteria and more. There are good experimental models.  Molecular and genetic causal factors are identified, and Rett research bears on the autism spectrum disorders, as well as other diseases. Add to this the remarkable finding that well after the disease has taken hold in the experimental model, the symptoms can be reversed by the reactivation of a single gene. With public support through NIH collapsing, the generosity of private philanthropy is critical at this juncture, at the brink of what may become a transformational treatment paradigm."          
     

Relevant Links

Entrepreneurs for Cures: The Critical Need for Innovative Approaches to Disease Research

Buying a Cure - What business know-how can do for disease.
New Yorker Magazine - January 28, 2007

Who's In Charge of Finding Cures - Commentary by Michael J. Fox
Forbes - October 25, 2007

Silicon Valley venture capitalist backs offbeat cancer cure ideas
San Francisco Chronicle - January 15, 2008

Venture Philanthropy on the Rise
The CenterWatch Monthly - August 2007

Sowing Seeds of Cures
Chemical & Engineering News - May 7, 2007