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Tamoxifen for Duchenne muscular dystrophy

6/9/2020

So, to shine UK we've adopted a slightly different approach to the drug development, and we're looking at repurposing, which is when you take a drug that's already been approved for use in a different disease like cancer, and you test it to see if it's going to have efficacy in Duchenne muscular dystrophy.

Tamoxifen for Duchenne muscular dystrophy

There are several advantages to repurposing for. First is that these drugs have already been through extensive testing for safety and efficacy. So, to test them for efficacy and Duchenne we only need to do one trial, compared to the normal three trials. Secondly, these drugs are cheap and readily available. So, if they do prove to be effective in treating Duchenne muscular dystrophy, then the cost will be much lower for patients, who want to apply them.

In the 1980s Tamoxifen was created to treat breast cancer. It's also been used to treat hormonal disorders within young boys.

“My name is Duke Fisher. I'm a consulting urologist at the University Children's Hospital in Basel, Switzerland. I'm leading the neuroma scar research group. Two years ago, when people from the research lab from Geneva contacted me, they had very good data on the model of Duchenne muscular dystrophy that have shown that Tamoxifen is improving the muscle function much more than other interventions and other drugs.

They asked me how to organize a trial and to submit a proposal willing to give us funding of about a million euro, but asking us to raise another half a million. Without their help we were not able to do the trial at all, because they stepped in, when we had very few time to find additional funding.”

I think that our support for the Tamoxifen study really represents our holistic approach to funding research. So, we are not only funding the clinical trial sites, where the trial is going to take place. We're funding the project manager for the trial, who's based in Switzerland. We're providing regulatory advice. And we're also funding the doctors and nurses in the UK, who will deliver that trial. And so we will also be expanding clinical trial capacity, so that more boys can take part in research in the UK.

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