A British consortium wins the joint Call for Proposals launched by three Wolfram-syndrome organizations.
Three British and one Australian research teams, coordinated by Professor Timothy Barrett, have
been awarded the funding of 200,000 € by the Association syndrome de Wolfram, the Eye Hope
Foundation and the Snow Foundation. Their project aims at “developing biomarkers that will show
early evidence of benefit of a treatment; and patient self-report outcome measures that will help
with regulatory approval. These two initiatives will speed up the delivery of treatments to patients in
On April 25 th 2017, the Association syndrome de Wolfram (France), the Eye Hope Foundation
(Belgium) and the Snow Foundation (USA) have decided to join forces to more efficiently fight the
disease. Their objective: fund a large-scale project aimed at accelerating the development of new
treatments against Wolfram syndrome.
They organized a call for proposals, which was a great success. Six research teams, located in six
different countries (USA, Belgium, France, United-Kingdom, Spain and Israel), have sent a proposal.
These proposals were then evaluated by a pool of independent world-renowned experts in the field
of Wolfram syndrome and drug development. Based on their recommendations, the Presidents of
the three organizations have elected the most promising project.
The project coordinated by Professor Timothy Barrett is entitled “Accelerating clinical trials in
Wolfram syndrome: development of efficacy biomarkers and patient relevant outcome measures”. It
will start on October 1 st 2017 and will terminate by September 2019. It is funded through equal
contributions from the Association syndrome de Wolfram (France), the Eye Hope Foundation
(Belgium) and the Snow Foundation (USA).
A note from Dr. Timothy Barrett
“The international Wolfram community stands out from other rare disease communities in that it is
highly collaborative: research scientists and patient groups work closely together across academic
institution and national boundaries. The initiative by Association Syndrome de Wolfram, Eye Hope
Foundation, and The Snow Foundation, is an outstanding example of this. Our teams are highly
honored to be awarded the first joint funding, and we will work hard to ensure our research leads to
benefits for patients. We will address one of the blocks to treatments, by finding ways to measure
their effects on outcomes important for patients. At the end of our studies, we will have a toolbox of
markers to show when treatments work, and to help convince health regulators to license
treatments for patients to use in the clinic.”