FAST Receives $250,000 Anonymous Donation – Ambitious Research Project Underway

FAST Receives $250,000 Anonymous Donation – Ambitious Research Project Underway

In 2008, the Foundation for Angelman Syndrome Therapeutics (FAST) was formed and invigorated the community with our fresh energy, our aggressive approach to Angelman Syndrome (AS) research, and our concise mission to bring practical treatment into current medical practice as quickly as possible.

In April 2012, FAST was thrilled to receive an anonymous donation of $250,000. The donor was drawn to the FAST organization for two reasons: our aggressive approach to AS research and our work within the Angelman community.

Vivint Gives Back Project Winners

The anonymous donor was most impressed by FAST’s approach to exclusively funding research that will identify, or help to identify, safe and effective treatments that will address the symptoms of AS as quickly as possible, while continuing to aggressively pursue the path towards a cure. In addition to being drawn to the FAST Mission, the anonymous donor was very impressed by the community’s involvement in the Vivint Gives Back Project and the unity and solidarity shown during the Annual FAST Gala. The annual FAST Gala fundraiser unites the AS community in ways never seen before and solidifies us in our common goal, as was demonstrated by our monumental win of $250,000 in the Vivint Gives Back Project.

Clinical Trials in Tampa

In January 2011, FAST funded research that identified a Food and DrugAdministration (FDA) approved compound that restored cognition and motor function in the AS mouse model. With the help of the Angelman community and the $250,000 won by the community for FAST in the Vivint Gives Back Project, FAST was able to take this finding from the laboratory to a human clinical trial within one year with Institutional Review Board (IRB) and FDA approval. FAST was able to do this quickly and with relatively little funding, demonstrating the organization’s ability to spend raised funds efficiently to produce results that will have the most impact on our children and their families.

Stephanie Manning & Colin Farrell at the 2011 FAST Gala

The recent $250,000 donation will be combined with additional FAST funds to initiate the most aggressive, comprehensive and ambitious AS research project to date. The project will bring together an impressive group of leading AS experts to work collaboratively on identifying treatments and a cure. With experts in the fields of neurophysiology, pediatric neurology, genomic

2011 FAST Gala

therapeutics, learning and memory, synaptic plasticity, epigenetics, and genomic imprinting working together, the project’s novel approach will accelerate AS research in ways never imagined. This FAST-funded project is a five-year project that will expand in terms of scope and number of researchers over the next several years. The goal of this project is simple: to bring effective treatments into current medical practice and to cure AS as quickly as possible. Our goal at FAST is to raise $5 million to provide the funding needed to allow these scientists to hire additional researchers, purchase reagents and equipment, and move research forward faster than currently possible.

FAST is enormously grateful to this donor for assisting us in making this innovative, ambitious project a reality as this donation will allow these researchers to get started now. Look for more news about this exciting project in the coming months. If you or someone you know is interested in supporting this project and the work FAST does on behalf of our children, please contact FAST at info@CureAngelman.org.

FAST funds 3rd Postdoctoral Fellowship Grant

FAST funds 3rd Postdoctoral Fellowship Grant

The path to running a research group is a long one.  Scientists are first trained in graduate school to obtain a doctorate degree in their chosen field.  In the US this can typically take four to seven years.  After graduation, scientists typically do an additional three to six years of training beyond their graduate work, called a “postdoc,” to expand their skills.  Most often, the work conducted during the postdoctoral training time determines the work that scientist will focus on in their own research group.  To be competitive for a position running a research group, the postdoctoral researcher must focus intensely on their project and produce publishable results.  To help increase the number of talented and dedicated researchers focusing on Angelman Syndrome research in their careers, FAST is providing fellowship awards that will cover the salary and experimental costs associated with postdoctoral research.  These fellowships also provide travel funds to allow the fellows to attend scientific meetings to present their work and learn about recent advances in the field.  Researchers who obtain one of these fellowships can focus exclusively on their work and not spend time they could be using for research on writing grants to obtain funding. This will allow these researchers to get a jump on the competition and make them more competitive for research positions in the next few years.

In January of 2012, FAST awarded our third postdoctoral fellowship to Dr. Sarah Black at the University of Texas A&M. Our scientific advisory board was impressed with the potential of her project to identify therapeutic targets that regulate paternal UBE3a expression and unanimously recommended funding. Please help us welcome our newest Angelman Syndrome researcher!

Dr Sarah Black

Sarah G. Black, Ph.D.
Texas A&M University
Mentor: Dr. Scott Dindot
Title: Epigenetic repression of the paternal Ube3a allele in neurons

  1. Where did you do your graduate training and what did you work on? I received my PhD in genetics from Texas A&M University in 2010, under the mentorship of Tom Spencer. My thesis involved understanding the role of endogenous retroviruses in placental and fetal development.  My project demonstrated that retroviral sequences are involved in the establishment of pregnancy.
  2. What interests you about research on Angelman Syndrome? I am intrigued by the many unanswered questions regarding the genetic and epigenetic regulation of the Ube3a gene in the brain. While there has been great progress made in Angelman Syndrome research, the mechanisms regulating genomic imprinting of Ube3a in the brain are still not understood.  With this fellowship, I hope to better understand the factors involved in repressing the paternal Ube3a allele in the brain.  Results from this fellowship may lead to the identification of those factors.  They may then be explored as therapeutic targets.
  3. How will this funding help you to focus on your research? I am very grateful to be awarded this postdoctoral fellowship by FAST.  The funding from this fellowship will allow me to focus on my research in AS, without having to concentrate on grant writing or other projects.  As a member of a lab driven by a young, promising principal investigator that is dedicated to studying Ube3a and AS, I will use this money to pursue more ambitious projects related to understanding the mechanisms of imprinting of Ube3a and finding therapeutic targets.
  4. How will your funded project help FAST advance towards potential therapeutics for Angelman Syndrome? The mechanisms regulating genomic imprinting of Ube3a remain poorly understood.  My project will utilize RNA interference technology to identify epigenetic modifiers or other factors that initiate and/or maintain repression of the paternal Ube3a allele in neurons. Results from this study will provide valuable insight into the fundamental mechanisms regulating genomic imprinting of Ube3a in the brain and may provide the foundation for therapeutic strategies aimed at reactivating the paternal UBE3A allele in AS patients.
Funds donated to FAST in the memory of Colby Goldstein were utilized in support of this award.
Firing research in Australia

Firing research in Australia

Miles for Smiles Great Wheelbarrow Race team

With a relatively small Angelman syndrome (AS) population (statistically around 1500 cases) spread across our wide brown land, the Foundation for Angelman Syndrome Therapeutics (FAST) Australia has had an amazing first two years. A number of fantastic events organised by dedicated families and friends; a Night at the Races, the glamorous Night for Angels in Sydney and the Take Flight for Angels Gala Ball in Launceston have contributed to our success  In addition to these events, family and friends who care for individuals with Angelman syndrome  have put their bodies on the line entering events; marathons, half marathons, mud runs, fun runs – even a wheelbarrow race, and of course our regular monthly donors! FAST Australia was formed to both compliment and join forces with FAST United States, however with research taking off at breakneck speed in the US there has been no laboratory research into Angelman Syndrome in Australia to date – that is all set to change!

The launch of the FAST Integrative Research Environment (FIRE) brings together four world class research laboratories, creating the perfect opportunity for Australia to enter the fold. Thanks to monumental fundraising efforts, including a very impressive eighty thousand dollar profit from A Night for the Angels Gala Ball in Sydney, we are able to offer our first Post Doctoral Fellowship Grant for an Australian researcher to learn from a team with extensive Angelman syndrome research targeted specifically at our goals. The initiative creates a unique opportunity where potential candidates can match their skills with one of the four US labs; Weeber Lab in the University of South Florida, Texas A&M University,  University of California Davis or Baylor College of Medicine. After a two year appointment in the United States, where the candidate will be actively working on joint FAST goals under the umbrella of the FIRE project, he/she will receive seed funding to continue Angelman syndrome research in Australia. FAST Australia has committed $150,000 to the FIRE initiative in the form of at least one research fellow.

One of the visiting researchers, Dr David Segal

On February 1st 2013, FAST will host it’s first Angelman Syndrome Symposium at the Royal Children’s Hospital in Melbourne, Australia; uniting Australian researchers (including the Australian scientific Advisory board) with three of the four FIRE laboratory heads; Dr Edwin Weeber, David Segal and Scott Dindot. US researchers will discuss the state worldwide AS research including developments, challenges, updates from the minocycline clinical trials and the FIRE initiative. The day will include an afternoon brainstorming session for researchers and medical professions to ensure the post-doctoral vacancy reaches the widest range of best possible candidates, future research collaborations and opportunities for Australian participation. and potential venues for the candidates return.

Families and friends will get the opportunity to meet the researchers, hear up to date information on research and connect with others in an informal setting on the evening of February 1st. (7 – 10.30 pm – details to follow). Funds raised from the Take Flight with Angels Gala Ball in Launceston (approximately twenty five thousand dollars) will directly fund an Australian Research Assistant to work with Dr Honey Heussler and her team at the Mater Hospital and Mater Medical Research Institute to further develop, collect and collate data for the much anticipated Angelman Syndrome Research Register. The register will provide the perfect tool for future clinical trials in Australia.

FAST Australia is enormously grateful for the support and efforts that have brought us to this pivotal point in Australian research, driving us towards safe and effective treatments and ultimately a cure for Angelman syndrome.  This is a very exciting time for our loved ones and for Australians as we take an active role in creating a brighter future for individuals with Angelman Syndrome, in our country and abroad.  As we are poised to make a global impact on this rare but potentially treatable disorder, we ask all of our supporters to commit to ensuring our success as this is just the beginning of our exciting and promising journey.

Enquire about the symposium or parent evening – info@cureangelman.es.au

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