The fast fail initiative is considering many potential autism drugs including Isis Pharmaceutical’s pending drug to treat myotonic dystrophy. This “FAST FAIL” initiative funded by NIH looks at promising drugs to treat Autism and several other conditions. It enables the drug to go to human trials earlier and see if the drug will pan out. The Isis drug by far is the most probable drug to treat and reverse the course of the Autism disease.
I recently became aware of a new and exciting company working on a potential drug therapy in the Myotonic Dystrophy space. Valentia Biopharma S.L. is a biotechnology company born in August 2006 as a University of Valencia spin-off.
NIH officials announced yesterday that a new contract has been let with UCLA to form a network of researchers at various academic institutions to identify promising new and older drug compounds to treat Autism Spectrum Disorder (ASD) and to see if they merit additional investments.
The program is part of a new initiative called “Fast Fail” intends to vastly speed up the drug development process and reduce the costs of this drug development. Instead of taking years of work to see if a drug works this Fast Fail process could see results within weeks. As ASD is a huge issue for the USa and other countries this fast testing with myotonic dystrophy drugs could lead to treatments in a much faster time frame.
Dr. James McCraken who is leading the effort at UCLA states “The Whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe and thereby speeding the development of effective new therapies and reducing the overall cost”
This is a copy of the article from J of Pediatrics MArch 16, 2013
The myotonic dystrophies have been called the most “diverse diseases known in medicine,”1 and in many respects, the broad spectrum of manifestations observed in myotonic dystrophy type 1 (DM1) are most evident when an undiagnosed and mildly affected mother gives birth to a severely affected child. DM1 results from an unstable trinucleotide repeat expansion (CTG) in the dystrophia myotonica-protein kinase gene (DMPK), located on chromosome 19q13.3.2, 3, 4
Congenital myotonic dystrophy (CDM) often presents as the index case in a family affected by DM1, and the diagnosis is made within the first few hours and days after birth. Neonatal symptoms can be life- threatening and include respiratory failure, feeding difficulties, hypotonia, and muscle weakness.5, 6, 7 The childhood years may bring marked dysarthria, intellectual impairment, and features of autistic spectrum disorders.8, 9, 10 Mothers of patients with CDM often have a diagnosis of DM1 established when their child is diagnosed. These mothers often have mild facial or distal muscle weakness, mild myotonia, and other multisystem manifestations, such as cataracts. Both diagnoses (CDM and adult DM1) can usher in a whirlwind of short- and long-term medical, psychosocial, and economic complications and decisions. Unfortunately, to date, limited empirical information is available to inform care providers, patients, and their family members about the prevalence and natural history of CDM.
A highly promising Compound has formed the basis for a new pharmaceutical company. This company is Called Atricode and is based on compounds that have been researched at University of Southern California to treat and cure Myotonic Dystrophy. Sita Reddy’s Lab has been instrumental in moving these potential treatments (MDI16) for Myotonic Dystrophy. The lab and company has recently been awarded a $90,000 grant through a competitive process. More about the grant:
2012 USC Ideas Empowered Program Grant: $90,000
Atricode is developing treatments for rare diseases. The lead indication, Myotonic Dystrophy Type 1 (DM1,) is a devastating genetic multisystem disorder with no available treatment or cure. This team identified highly potent and selective small molecule leads that rescue DM1 pathology in patient myoblasts and in DM1 mouse models. The team joined forces with experienced entrepreneurs to form a start-up company that moves the drug candidates towards the clinic. The drug candidate could be the first therapy to treat this devastating disorder and the Ideas Empowered funds are critical for the selection of the lead candidate for clinical development and to support fundraising efforts.
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