Funding Update

October 18, 2018 2:50 pm

Alex’s Wish has been working with Duchenne UK to invest and fund in some very important projects over the last couple of years and here is an update of our funding.

Tamoxifen TAMDMD trial – £25K

This international clinical trial is investigating the use of tamoxifen, a well-known breast cancer drug, to reduce the progression of DMD. Alex’s Wish gave £25K to this project which will fund 3 patients through the clinical trial for a year at Alder Hey Children’s Hospital, Liverpool.

We are pleased to announce that 9 patients have now been dosed on this trial. The trial is due to begin in the UK at Alder Hey Children’s Hospital and Leeds Children’s Hospital later this year.

Expanding clinical trial capacity at Birmingham Heartlands Hospital – £10K

Our funding is helping to provide consultant time which will be dedicated to supporting DMD clinical trials as well as funding physiotherapy equipment for use in these clinical trials. This funding was provided through Duchenne UK’s DMD Hub, an initiative set up to increase clinical trial capacity in the UK for DMD.

Combination therapy with Tamoxifen – £35K

This pre-clinical work is looking at the effect of using a combination of drugs for the treatment of DMD. The researchers will look at combining tamoxifen (a breast cancer drug), metformin (a diabetes drug) and L-citrulline (an amino acid) to treat mice with DMD. They will then also explore whether there is an advantage to combining these compounds with prednisolone, the most commonly prescribed steroid in DMD.

John Bourke Heart Study- £37K

This award has been granted to consultant cardiologist Dr John Bourke at Newcastle Hospital to carry out a study of cardiac arrhythmias, to understand the causes of heart disease and sudden death in patients with DMD.

Alder Hey Neuromuscular Research Nurse- £25K

Alex’s Wish are part funding a Research Nurse to run DMD clinical trials at Alder Hey Children’s Hospital for 2-years. The new recruit, Tim Henderson, is helping increase clinical trial capacity in the UK for DMD trials.



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