Foundation for Angelman Syndrome Therapeutics

Our goal is to bring practical treatment into current medical practice as quickly as possible. It is our hope that grants we fund will lead to additional research support from government agencies, other funding sources and organizations around the globe. FAST is served by two boards: the board of directors and the scientific advisory board. Together, we are working hard to bring practical treatment into current medical practice as quickly as possible.

A true research collaboration, and the infrastructure required to advance a cure for Angelman syndrome, was nonexistent. FAST addressed this challenge by creating the first-of-its-kind FIRE Initiative (FAST Integrative Research Environment), which funds more than two-dozen scientists from top universities to work collaboratively to identify, characterize and implement new therapeutics for the treatment and ultimate cure of Angelman syndrome. The FIRE Initiative is the most aggressive research program to date dedicated to finding a cure for AS.

For decades, small, non-profit disease research organizations like FAST have modeled their funding philosophies after the National Institutes of Health (NIH) where they budget their research dollars, put out a once-a-year call for applications, wait for scientists to come to them with ideas, select the most promising applications and hope they actually see results. FAST is not at all interested in this slow, linear approach to funding research and instead adopted the innovative model of venture philanthropy, recruiting a stellar in-house team to work in partnership with leading scientists on ambitious, high-risk/high-reward study designs that will ensure promising therapeutics make it from the laboratory bench to the patient’s bedside as quickly as possible.

Adopting a business model where we have input and control over the direction of the research, FAST assembled an in-house team of experts in science, medicine, business management, finance, law, accounting and technology. In addition to funding research grants, including over $360,000.00 in Postdoctoral Fellowships, FAST has also entered into contracted research on targeted projects with well defined milestones and deliverables.

Angelman Syndrome is currently one of the most promising fields of scientific research; relying solely on investigator-initiated research to identify and implement patient treatments is inefficient and short sighted. Additionally, true collaboration of researchers working cooperatively is the only successful approach to treatment science.

In January 2011, FAST contracted research with Dr. Edwin Weeber to test four FDA approved compounds in our mouse model. One of those compounds, Minocycline, showed promise in treating some of the symptoms of AS. With additional funding and testing, Minocycline was identified as a candidate for human clinical trial. FAST provided the funding for the trial in 2012 and we anticipate results to be published soon.

In May of 2013, FAST launched the most aggressive Angelman research initiative in history, bringing 24 researchers from 4 universities together in true collaboration to identify additional treatments and a cure for Angelman Syndrome. The FAST Integrative Research Environment (FIRE) Initiative has already identified several possible treatment candidates for human clinical trial. FAST will continue to either fund small, proof of concept trials that will better position the Angelman community for larger FDA funding and/or partner with pharmaceutical companies to bridge the so-called “Valley of Death” in translational research.

FAST has proven the success of our funding philosophy in a very short amount of time and with relatively little funding. It is not about how much you spend, but rather how you spend it. FAST is funding smarter, faster science and with proper funding, a cure is now just within our reach.

The FAST track: from bench to bedside: FAST is the largest non-governmental funder of Angelman syndrome research and the only organization with a detailed plan towards a cure. While others said a cure was not possible, FAST spent the past seven years strategically investing millions of dollars in research to lay the foundation for scientific breakthroughs. Recent advancements in the understanding of Angelman syndrome, made by FAST scientists, suggest finding a cure for this disorder in the near future is not only possible, it’s inevitable. Brightest minds partner in race to cure Angelman syndrome FAST brought together a multi-disciplined team of more than two-dozen scientists from top research universities and pharmaceutical companies to join forces on a focused path to a cure. Known as the “FIRE” Consortium: FAST Integrative Research Environment, FAST’s scientists know exactly what causes Angelman syndrome. They have cured the symptoms of the disorder in mice using several strategies.

Now is the time for action. FAST’s Roadmap to a Cure for Angelman syndrome is bold, ambitious, and, with your help, achievable. The Roadmap costs an estimated $20 million dollars, and AS families have been challenged to raise $2 million of that investment. We are calling on everyone connected to a loved one with Angelman syndrome to support this research. Please join us by making a financial gift, spreading the word with friends and family members, and fundraising to help us cross the finish line. Your efforts will take us one step closer to making therapeutics available to those affected with AS and, ultimately, finding a cure that will free our children.