About Duchenne Treatments In The Pipeline Nutraceuticals Nutraceuticals are products derived from food sources that provide extra health benefits, in addition to the basic nutritional value found in the food. Many nutraceuticals are thought to have anti-inflammatory or anti-oxidant effects. We know that DMD progression is be worsened by inflammation and oxidative stressed, therefore it is suspected many nutraceuticals could have some therapeutic benefit to patients. A 2016 review showed that, in the United States, 80% of caregivers reported using alternative medicine such as nutraceuticals for the treatment of Duchenne muscular dystrophy, in conjunction to their traditional medicine. It is therefore vital for us to understand which nutraceuticals have a benefit to patients with Duchenne muscular dystrophy. We have granted more than half a million pounds to projects investigating nutraceuticals, or compounds derived from nutraceuticals. Prof Winder- Soy products (£135,000) One nutraceutical product which has been popular with some members of Duchenne community is Haelan 951. This fermented soybean beverage is extremely expensive and has an unpleasant taste, however it has been said to slow down DMD disease progression. In 2016, Duchenne UK commissioned Professor Steve Winder at the University of Sheffield to carry out a study in mice to build up some evidence for the use of Haelan 951 to treat DMD. The results of this study clearly showed that Haelan 951 did not have a significant benefit on grip strength in the mouse model of DMD. However, when BBI, a component of Haelan 951, was isolated it significantly improved grip strength in the mdx mouse. Duchenne UK has now extended this grant to Professor Winder to carry out. Dose escalation study for the use of BBI in mdx mice. This project highlights the importance of funding research into the use of nutraceuticals for DMD. Thousands of families have been spending money on Haelan 951, a product which now been shown to have no benefit to those with DMD. Read more here. University of Western Australia- Taurine (£237,362) Taurine is an amino acid that is found in all animal tissues. It can also be bought as a health food supplement. There is strong pre-clinical data to suggest taurine improves muscle strength, reduces inflammation and protects against muscle wasting in the DMD (mdx) mouse model. This project will do further animal experiments to better understand how taurine would work in patients. The project will then look at gathering information on human urine and blood samples that would be needed to plan a potential clinical trial to look at the safety and efficacy of Taurine as a treatment for DMD. Read more here. Nutraceuticals- Keith Foster (£179,520) Dr Fosters ongoing nutraceuticals project is investigating the use of a selection of different nutraceuticals on their own and in combination, to define the optimum combinational treatment protocols for Duchenne muscular dystrophy. Unfortunately this project has been terminated due to practical and technical issues. For more information, please click here.