This webinar series addresses a wide range of current topics in Fragile X research. Hosted by FRAXA and organized by Michael Tranfaglia, MD and Patricia Cogram, PhD, sessions feature outstanding speakers from universities and the biotech and pharmaceutical industries. All are welcome; preregistration is required and free.
----- Speakers and Presentation Abstracts -----
David Brown, PhD
Co-founder and Chair of the Board, Healx
Healx’ Methods Used to Select Drugs for Preclinical Testing in Models of Fragile X
Healx has a range of methods, and two of these proved particularly successful in identifying drugs that could be repurposed for use in Fragile X. Single drugs were selected initially, then combinations were explored. Some combinations of two drugs exhibited remarkable synergy of action, boosting their effectiveness in the mouse model of Fragile X. These methods will be described in the presentation.
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Ivan Angulo-Herrera, PhD
Associate Director of Pharmacology, Healx
Preclinical Results from the Fmr1 KO Mouse Model
Fragile X syndrome (FXS) is a common inherited form of intellectual disability and one of the leading genetic causes for autism spectrum disorder. FXS is caused by the silencing of the FMR1 gene. The Fmr1 knockout (KO) mouse model presents several behavioral characteristics that recapitulate the human condition. Healx’ drug repurposing approach uses computational biology methods to rank drug predictions, pharmacology analysis to propose a rationale to support the in silico predictions, and preclinical validation to demonstrate efficacy. This methodology has been successful in identifying several drug candidates that benefit multiple behavioral phenotypes of the Fmr1 knockout (KO) mouse. Additionally, when these drugs are used in combination at ineffective low doses they also rescued several behaviors that suggest a synergistic or additive effect of the two drugs toward the impaired behaviors. This data will be presented along with complementary biochemical readouts as part of the preclinical package to support Healx clinical studies.
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Anthony Hall
Chief Medical Officer, Healx
The Challenges in Designing a Clinical Trial Programme for Fragile X
There have been many clinical trials in Fragile X, yet there are no authorised treatments available for patients. There are many possible reasons for the lack of success in Fragile X clinical programmes; it may be that the drugs under study were ineffective, or that the studies as designed were unable to detect the effects, even though they existed. This talk will discuss some of the considerations we had at Healx when designing our proof of concept clinical trial, in order to maximise the chances of seeing an effect if one exists.
----- TIMESTAMPS -----
0:00 Intro with Dr. Michael Tranfaglia
1:09 Healx’ Methods Used to Select Drugs for Preclinical Testing in Models of Fragile X
12:35 Preclinical Results from the Fmr1 KO Mouse Model
32:03 The Challenges in Designing a Clinical Trial Programme for Fragile X
54:10 Questions and Answers
1:22:45 Closing remarks
----- About FRAXA -----
FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments.
Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders.
To learn more about FRAXA's Fragile X syndrome research, to get involved with Fragile X awareness and events or to donate to FRAXA research, visit fraxa.org
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Website: fraxa.org
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