No strong evidence for tamoxifen as a treatment for Duchenne muscular dystrophy – TAM-DMD analysis concludes

Duchenne UK, along with our charity partners Alex’s Wish and Joining Jack, funded a trial called TAM-DMD in 2017 to assess the safety and efficacy of tamoxifen. This drug, which is currently used to treat breast cancer, had shown promise for Duchenne muscular dystrophy (DMD) in preclinical research studies.  

After receiving preliminary data from the trial in September 2021, Duchenne UK has worked with the study lead researcher at University Children’s Hospital in Basel, Switzerland, and other DMD data expert groups in the UK and the US on extensive data analyses to determine whether tamoxifen had an effect on the boys treated in the trial.  

We were disappointed to conclude that the trial did not show evidence for tamoxifen’s effectiveness as a DMD treatment.  

The trial, which took place over 48 weeks, involved both ambulant (Group A) and non-ambulant boys (Group B). Half of the 72 Group A boys were given tamoxifen and the other half placebo, as a comparison. Tamoxifen did not show a clinically significant effect in either Group A or Group B patients against the outcome measures used. 

For this reason, Duchenne UK will not pursue further research into tamoxifen as a treatment for DMD.  

While this trial did not provide the desired results, it has nonetheless provided learnings to help inform future DMD research.  

Emily Reuben, CEO of Duchenne UK, said “There is so much activity in the DMD field right now, and while the outcome of the tamoxifen trial is a disappointment for us, as I am sure it will be for the wider community, we are optimistic around the current pipeline of DMD drug products. Duchenne UK is very active in making new treatments accessible to UK patients and in supporting companies globally to drive their programmes forward. 

We strongly believe that this was a risk worth taking, because we have now a conclusive answer on the effect of tamoxifen in DMD, and this information will help patients and families make better informed decisions moving forward.” 

A scientific paper with full results will be published in the coming months and Duchenne UK will share this with the community when available.  

We would like to thank all the patients and families who have taken part in the study, and our partner charities for their support in making this research happen. 

If you have any further questions about the study results, please get in touch with us [email protected] 

Visit our Research Glossary for help understanding the terms used in DMD research