Lighting a fire; FAST Integrative Research Environment (FIRE)
The Foundation for Angelman Syndrome Therapeutics (FAST) is proud to announce the launch of the most aggressive, novel and innovative research program to date into finding treatments and a cure for Angelman Syndrome (AS). The FAST Integrative Research Environment (FIRE) is a coordinated consortium of leading researchers working together to identify, characterize and implement new therapeutics for the treatment and ultimate cure of AS. Evidence strongly suggests that therapeutic intervention can ameliorate many, if not all, of the symptoms associated with AS, which include seizures, severe verbal impairment, cognitive and motor challenges. The underlying cause of AS is the loss of function of the maternal copy of the UBE3A gene. In the brain, the paternal copy of UBE3A is intact, but is epigenetically silenced in most regions. The FIRE team will aim to identify new therapeutics to treat AS and associated symptoms while simultaneously identifying ways to safely reactivate the paternal UBE3A allele, which may provide a true cure for this disorder. FIRE launches with four prominent Angelman Syndrome researchers and an initial commitment from FAST US of $648,000 and FAST Australia of $150,000, totaling $798,000 for the first year of the project. FAST Australia’s investment of $150,000 to the FIRE initiative will provide funding for an Australian postdoctoral fellowship, allowing for a qualified researcher to work in a US laboratory conducting Angelman Syndrome research. The expertise gained by the fellow will pave the way for expansion of research to Australia upon his/her return. Additional collaborations with other international organizations and researchers will help extend and expand the reach of FIRE worldwide. FAST intends to generate a minimum of ten million dollars to allow additional researchers and their teams to enter the initiative as funds are raised. The four inaugural researchers bring unique expertise and perspectives to this endeavor. The combination of their abilities ensures research is not duplicated in multiple laboratories, but rather endorsed by fellow investigators, bringing results from bench to bedside in the most efficient and cost effective timeframe possible. The FIRE model allows researchers to focus on identifying, validating and implementing novel treatments for AS while simultaneously evaluating existing FDA approved drugs, novel compounds, specific interventions, and new animal models. Samples, resources and data can be shared almost instantaneously to increase efficiency and reduce overall costs. FIRE will exponentially escalate the journey towards our ultimate goal – a cure for Angelman Syndrome - by bringing together prominent AS researchers to not only collaborate but also exchange information in real-time. The Inaugural Projects: FIRE will focus on short and long-term strategies for therapeutic development. Short-term research will involve identifying pharmaceuticals that are, or are in the process of being, FDA approved. This category of drugs represents those that have passed rigorous testing for safety and tolerability and most have known mechanisms of action. Several potential drugs will be tested to determine their effectiveness in the AS mouse model to treat the 5 major areas of AS symptomology:
- Seizure control (in collaboration with Dr. Anderson);
- Cognitive ability;
- Motor coordination;
- Sleep patterns; and
- Synaptic function and neuronal morphology.