Monday, August 11, 2014

Could Gene Therapy Replace the Need for a Heart transplant?

Could gene therapy help avoid a heart transplant?  We’ll soon be one step closer to knowing the answer thanks to a groundbreaking trial in the UK.  Lee Adams, 37, is the first of an eventual 24 patients who will take part in a trial to see if Mydicar, a treatment from U.S. biotech firm Celladon, can help overcome advanced heart failure. 

The treatment is designed to deliver a spike in SERCA2a protein in the heart muscle.  The SERCA2a protein is responsible for making heart muscle contract and low levels have been known to make the heart pump weakly.  The protein will be delivered to the heart via harmless virus.  Researchers plan to take a samples after an initial six month period to measure the presence of the gene.  For those that undergo a subsequent transplant, the researchers will be able to examine the actual heart as well.

Gene Therapy Heart Transplant SERCA2a Protein TreatmentOf the 24 patients the study plans to involve, 16 will receive the actual treatment while the other eight are given placebos.  Like Adams, these patients have advanced heart failure and rely on Left Ventricle Assist Devices (LVAD) to keep them alive while they await a transplant. 

The trial, led by Imperial College London and funded by British Heart Foundation as well as Celladon, claims to be the first in the world to investigate the use of gene therapy to correct heart failure. 

Says Professor Sian Harding, who helped develop the treatment, “It's important to remember that the therapy is not correcting a gene defect. We are working much more downstream, which means that no matter what the cause of the heart failure, the therapy should be equally beneficial for patients whether their heart problems stem from genes, lifestyle or the environment or a mixture of all of these."

Adams has a reserved optimism towards the study.  "Of course the best thing that could happen would be for my heart function to show signs of improvement and for the gene therapy to prove to be a 'miracle cure' for myself and other patients. But I'm not building up my hopes too much because, for all I know, I might have had the placebo.”

Here’s to hoping it is the “miracle cure”.

What else is new in the field of cell therapy?  Join us for the Cell Therapy Bioprocessing conference, September 15-16, Boston, MA.  Download the agenda to see what’s on tap.

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