Tag Archives: cure

New Approach Cures some RNA toxicity in Fly’s and Mice with Myotonic Dystrophy

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Another strategy for a cure of for myotonic dystrophy treatment

Researchers at the University of Valencia in Spain led by Amparo Garcia-Lopez recently identified a new way to block the genetic change that causes myotonic dystrophy type 1. They used a fruit fly model to screen for potential drugs and then tested the most promising one in mice. The muscles of the treated mice appeared healthier under the microscope. This new research is encouraging and if replicated in humans it would be expected to be an effective treatment.

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Prosensa recieves 5 million Euros for Myotonic Dystrophy Research

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The USA is not the only location where drug development for myotonic Dystrophy is taking place. Here is a one year old report (October 2011) on the research in the Netherlands for Myotonic Dystrophy treatment. Prosena also receive an Emerging Star award this year for the most innovative biotech company in Europe. They also announced a 23 million euro financing round in January. Prosensa has about 4-5 potential drugs for Duchene Muscular Dystrophy in development and we are very happy that they are looking at Myotonic Dystrophy as a therapy as well.

 

Prosensa Awarded up to €5m Innovation Credit from Dutch Government for research in Myotonic Dystrophy

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October 25, 2011

For immediate release

Leiden, The Netherlands, October 25, 2011 – Prosensa, the Dutch company focusing on RNA modulating therapeutics for rare diseases with unmet medical needs, announced today that it has received an innovation credit of up to €5m from AgentschapNL, a subsidiary of the Dutch Ministry of Economic Affairs, Agriculture and Innovation, to support further development of a series of preclinical compounds for the treatment of Myotonic Dystrophy type 1 (DM1).

According to the terms of the agreement, Prosensa will receive €940,000 to support the initial stage of the project, with the potential to receive additional funding of up to €5m after successful completion of specific milestones and an initial review in 2012.

The genetic mutation underlying DM1 is characterized by expansion of so-called triplet repeats in the DMPK gene leading to transcripts with toxic effects. Prosensa applies its RNA modulation platform to reduce the number of these expanded repeat, toxic transcripts. One compound, PRO135, reversed the transcript toxic effects after in vivo intramuscular administration in preclinical DM1 models. Prosensa works in close collaboration with the department of Cell Biology, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, on this project, one of the leading centres in DM1 research in the world.

Judith van Deutekom, Vice President of Discovery at Prosensa commented: “We are very pleased with this grant by the Dutch government, which allows us to accelerate our research in this important therapeutic area and helps us grow into a specialist rare disease company. The innovation credit from the Dutch government is intended to stimulate development of technologically innovative products. In addition to Duchenne muscular dystrophy, we are also working on a potential therapy for patients affected by Myotonic Dystrophy, yet another rare disease for which no treatment currently exists.”

The Laboratories….. Researchers working on Myotonic Dystrophy

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Here are a few of the labs working on Myotonic Dystrophy

Dr. Puymirat Quebec, Canada
The major aim of the Puymirat lab is to develop a genetic therapy for Steinert dystrophy. During the last few years, the Puymirat lab developed a genetic approach capable of restoring normal functions of the affected human muscle cell. Indeed, Steinert dystrophy is caused by an abnormal accumulation of RNA in the nucleus of muscle cells. The therapy developed by the Puymirat lab is based on the specific destruction of RNA using antisense RNA and ribozymes. The research group showed in vitro that specific destruction of mRNA restored normal functions of the cell. In vivo, intramuscular injection of vectors producing antisense RNA or ribozymes reduced the levels of mutated RNA by 80%. The team is currently studying the effectiveness of this therapy in mice carrying the pathology.

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New Novel Drug – Testing in Myotonic Dystrophy – Isis

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Recently Isis Pharmaceuticals gave a talk at the myotonic dystrophy foundation conference in San Fransisco, CA. This talk including just information that had been publicly given out previously. However, one of the lead researchers in the Myotonic Dystrophy field commented that this new drug that Isis is pursuing in collaboration with Biogen could be the “Fountain of Youth Drug”. The basic mechanism of antisense technology, if aging is caused by cell clogging with these proteins that are not being released to proper area. This could be a hug huge blockbuster drug that would exceed everyone potential.

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DM1 Muscle Issues Cured in Mouse Model

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Exciting news. This summer researchers at ISIS Pharmaceuticals were able to treat and Cure DM1 in a mouse model (A mouse model is a mouse that has been genetically altered to have the symptoms of DM1) This is really exciting news. The treatment seemed to work well and was by injection. The injection seemed to work long term as well. Next they will most likely use a Monkey and then if that is successful with Humans! This is 5-8 years off and will need millions of dollars of research.

The payoff for these companies will be huge though. With over 42,000 people in North America alone the market size is roughly $1 Billion for the USA. If the injections prove successful a once a year or 2x per year injection would lead to a low-cost of goods sold and high profitability. Worldwide at $20,000 per dose this is about a $17Billion dollar market.

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