MDA AWARDS $368K TO AUSTRALIAN RESEARCHER ADVANCING EXON SKIPPING STUDIES(MOE)

TUCSON, Ariz., Aug. 18, 2010 – Fourteen years ago, Professor Steve Wilton had a revolutionary idea:  Why not use “molecular scalpels” to excise the flawed parts of a gene message, the way a surgeon removes diseased tissue from the body?  At the time, the idea sounded far-fetched, even to many scientists.  “I heard the words ‘party trick’ used,” recalled Wilton, head of the Molecular Genetic Therapies Group at the University of Western Australia. 

However, Muscular Dystrophy Association scientific advisors embraced the novel idea and today, Wilton’s technology, termed exon-skipping, is one of the best hopes for a therapy for Duchenne muscular dystrophy and several other genetic-based diseases.

Coming on the heels of more than a decade of almost continual funding from MDA, Wilton has been awarded a new three-year $368,100 MDA grant to further improve exon-skipping technology. The new project also will test the suitability of a “next generation” form of Wilton’s genetic band-aids, known as methoxyethyl oligomers (MOE).

 

http://www.mda.org/research/perth-uwa-368k.html

 

Hopefully, Prof Steve Wilton work will help all other Duchenne kids.

 

Raktim

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Good news is always welcome, thanks Raktim!! Surprised MDA could separate themselves from such a large grant.
They funded a number of projects. Steve's was one of them. Not too bad.


Becker and Duchenne muscular dystrophies (BMD and DMD)

Zolt Arany, assistant professor in medicine at Beth Israel Deaconess Medical Center, part of Harvard Medical School in Boston, received $352,188 for research into the role of blood vessels in skeletal muscle metabolism.

MDA awarded $408,915 to Carmen Bertoni, assistant professor of neurology at the University of California, Los Angeles, to continue research into DNA repair strategies using small molecules called single-stranded oligonucleotides, or ssODNs.

Michele Calos, a professor in the department of genetics at Stanford University School of Medicine in Stanford, Calif., received $200,000 to develop a new stem-cell-based therapy.

MDA awarded $179,327 to researcher Sean Forbes at the University of Florida in Gainesville for research into the effects of impaired blood flow to muscles lacking the dystrophin protein.

Bernard Jasmin, vice dean of research at the University of Ottawa in Canada, received $360,000 for research into using small molecules called "exercise mimetics" to increase levels of the structural protein utrophin in DMD.

MDA awarded Ashok Kumar of the University of Louisville School of Medicine in Kentucky $349,206 for continued study of the molecular mechanisms that underlie disease onset and progression.

MDA awarded $375,000 to Louis Kunkel, director of the program in genomics at Children's Hospital Boston, for research into compounds already approved for human use that may alter disease progression. Kunkel, a longtime MDA adviser and chairman of MDA's Scientific Advisory Committee, was on the MDA-funded scientific team that in 1986 first identified the gene mutation underlying DMD.

Gordon Lynch, professor of physiology at the University of Melbourne, Australia, received $375,000 for research into strategies aimed at using proteins called "growth factors" to improve muscle function.

Josephine Nalbantoglu, associate professor in the department of neurology and neurosurgery at McGill University in Montreal, Canada, received $313,170 for research into increasing levels of the muscle protein utrophin as a therapeutic strategy in DMD.

Vihang Narkar, assistant professor at the University of Texas Health Science Center at Houston, was awarded $302,326 to study the potential therapeutic value of increasing the overall amount of a specific type of muscle called "aerobic muscle" in DMD.

Professor Steve Wilton at the University of Western Australia in Perth received $368,100 for continued research into a strategy called "exon skipping," which bypasses mutations in the dystrophin gene responsible for DMD.
Two types of exon-skipping drugs that have reached the clinical trial stage are called 2Omethyl and morpholino. A third drug, 2 methoxyethyl, or "MOE," appears to be well-suited for exon skipping but was not made available for study until recently.
I understand that Prof Steve Wilton is working on 2 methoxyethyl.(MOE)
In their new work, Wilton and his team will conduct detailed preclinical studies in the mdx research mouse model of DMD to assess MOE's suitability for exon skipping.

In addition, the group has developed a number of other drugs designed to bypass a variety of different dystrophin mutations that should be responsive to exon skipping. The investigators will study the effectiveness and side-effect profiles of the new drugs.
This research is necessary and good but the money to bring any kind of exon skipping to market is astronomical. My take is that exon skipping has many more scientific issues and is further away from being a treatment than I wish it was (or than some seem to think). The systemic delivery issues remain. The inability to solve the cardiac problem remain. And the economics is not there. We are taking the small, total population of duchenne, and breaking it into even smaller subgroups. This makes the economics even more problematic. Even if drugs can be developed (lots of time and money), financing will be quite an issue. Once again, a very important area and one that is moving forward, but not something likely to come to fruition in the next several years. I hope I am wrong, but I don't think so.
Great news. Steve is a fantastic person, glad to see him get some financial support for his work.
Jeff,

Lets give exon skipping a chance since most other treatments in the works are failing one by one



Jeff Sobel said:
This research is necessary and good but the money to bring any kind of exon skipping to market is astronomical. My take is that exon skipping has many more scientific issues and is further away from being a treatment than I wish it was (or than some seem to think). The systemic delivery issues remain. The inability to solve the cardiac problem remain. And the economics is not there. We are taking the small, total population of duchenne, and breaking it into even smaller subgroups. This makes the economics even more problematic. Even if drugs can be developed (lots of time and money), financing will be quite an issue. Once again, a very important area and one that is moving forward, but not something likely to come to fruition in the next several years. I hope I am wrong, but I don't think so.
Forgive me but I have to disagree with your assessment Jeff. Even though, right now, it looks like the economics of exon skipping won't work I am seeing more and more large pharma's signing up to work specifically on rare, serious diseases. Many have scaled back traditional R & D, layed off hundreds internationally in order to change profiles to incorporate under treated diseases. Also, who says exon skipping can't/won't be able to solve problems for diseases other than duchenne? Same with Myostatin Inhibitors, Gene Therapy and Stem Cells to mention a few. Currently there is a changing tide in the economics of the pharma world and yes, they are still in the business of making a profit.

Also, many of us don't think of exon skipping as a "cure", we comprehend there will be a need to combine many chemestries to fix the complications of duchenne. Exon skipping can stop progression. So far, in the history of duchenne...that's monumental.

Jeff Sobel said:
This research is necessary and good but the money to bring any kind of exon skipping to market is astronomical. My take is that exon skipping has many more scientific issues and is further away from being a treatment than I wish it was (or than some seem to think). The systemic delivery issues remain. The inability to solve the cardiac problem remain. And the economics is not there. We are taking the small, total population of duchenne, and breaking it into even smaller subgroups. This makes the economics even more problematic. Even if drugs can be developed (lots of time and money), financing will be quite an issue. Once again, a very important area and one that is moving forward, but not something likely to come to fruition in the next several years. I hope I am wrong, but I don't think so.
Steve Wilton is one of the most dedicated researchers in DMD & BMD. A great speaker at conferences helping parents understand what is going on with their son(s) and what is being done to help them. Here's to you, Steve and here's to a cure for all our sons.
really great to hear steve wilton getting some money to help his research along. he was one of the first researchers i met years ago after austin and max were diagnosed, and the only one i have had the honor of sitting at the bar and discussing dmd with! he is truely dedicated, and i believe in him fully!

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