July 25, 2017

expert reaction to study showing gene therapy for muscular dystrophy symptoms in a dog model

In a new study published in Nature Communications scientists report gene therapy improving dystrophic symptoms, for over two years, in a canine model of Duchenne muscular dystrophy.

Prof. Dame Kay Davies, Director of the MRC Functional Genomics Unit & Dr Lee’s Professor of Anatomy, Oxford Neuromuscular Centre, University of Oxford, said:

“This is a very exciting and promising step in the pursuit of an effective gene therapy protocol of Duchenne Muscular Dystrophy boys.  The sustained expression after two years is also very encouraging and paves the way for human clinical trials.”

Prof. Darren Griffin, Professor of Genetics, University of Kent, said: 

“This is really a very exciting study indeed. Duchenne Muscular Dystrophy is a horrible, wasting, life-ending disease of young people. By making use of the canine model and showing genuine improvement in the animals treated, then real hope is present for the prospect for disease treatment in humans.  The disease has long been a target for gene therapy and it is only to be hoped that sufficient funds can be awarded for this research to reach its natural conclusion and go into full clinical trials.

“The press release does indeed accurately and succinctly reflect the study and makes the point that success in a large mammal (i.e. dogs) is an essential step in paving the way, subject to efficacy and safety trails in humans, for full clinical implementation.”

Dr John Counsell, Research Associate in Gene Transfer Technology Group, UCL, said:

“In the past, gene therapy has often suffered from the body’s immune system rejecting parts of the drug, consequentially limiting therapeutic efficiency. Here, we see that gene therapy can be well tolerated in dogs with muscular dystrophy, whose symptoms remain improved two years after a single dose. This is very encouraging, as current treatments for muscular dystrophy are merely palliative and patients are under constant medical care throughout their life. Further preclinical trials will be required to show that this treatment can be effective in patients, and the long-term side-effects are difficult to predict, but this paper demonstrates the enormous potential of gene therapy for severe, life-threatening diseases, for which treatments are extremely limited.”

* ‘Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy’ by Le Guiner et al. published in Nature Communications on Tuesday 25 July.

Declared interests

Prof Dame Kay Davies: “I am co-founder and shareholder of Summit Therapeutics developing utrophin modulation for Duchenne Muscular Dystrophy (DMD).”

Prof. Darren Griffin: No conflicts of interest.

Dr John Counsell: “I have no conflict of interest or anything to declare.”