Published: Wednesday, November 20th, 2013

Gene therapy trial begins at the Golden Jubilee




The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital (GJNH); with the first candidate recently being administered with a dose of the MYDICAR treatment.

The GJNH, home of the Scottish National Advanced Heart Failure Service (SNAHFS), announced in April 2013 that it would be participating in the international trial, helping gather a total of 200 patients from 50 institutions worldwide, to determine the effectiveness of the gene therapy treatment, MYDICAR, in advanced heart failure patients.  


Studies have shown that there is a clear connection between depletion of the SERCA2a gene in the heart and the progression of end-stage heart failure.


SERCA2a is a protein which controls the uptake of calcium – a key signalling molecule – into specialised stores in heart cells. MYDICAR is a genetically-targeted enzyme replacement therapy which aims to restore this function, thereby improving many of the symptoms associated with heart failure and reducing hospitalisations.


In a previous phase of the trial, carried out on 39 individuals, frequency of death, worsening heart failure, hospitalizations, transplants and the need for a mechanical heart pump was dramatically reduced among patients being treated with a high dosage of MYDICAR.


The trial aims to establish the efficiency and safety of this treatment by comparing findings of individuals treated with the gene therapy against those who have been administered with a placebo (dummy) infusion.


Dr Mark Petrie, Director of the Scottish National Advanced Heart Failure Service, and the Golden Jubilee’s Principal Investigator for CUPID2, who performed the procedure, commented: “The Golden Jubilee National Hospital provides a national service for individuals suffering from the most serious heart failure.


“It is, therefore, our responsibility to ensure we are at the forefront of new innovations and research developments, ensuring we are providing a world class standard of care for all of our patients.  Close collaboration with Professor Baker in Glasgow University has allowed this project to be conducted in Scotland as one of only two selected UK sites. 


“We are very excited to be a part of the first UK gene therapy trial for patients with chronic heart failure. As part of this trial we hope to establish the effectiveness and safety of this new treatment, allowing us to contribute to advances in medicine which have the potential to make a significant improvement in quality of life for our patients in the years to come.”

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