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 U.S. biotech firm Celladon hopes that its Mydicar treatment can
help patients like Lee Adams who have advanced heart failure and
rely on so called Left Ventricular Assist Devices (LVAD) to keep
them alive until a donor heart becomes available.
Celladon's treatment works by inserting a gene called SERCA2a - the
lack of which makes hearts pump weakly - directly into heart cells
via a catheter to repair them. The trial will evaluate how much of
the gene is getting to the heart muscle and how well it is working.
Adams, from Hertfordshire, north of London, is the first of 24
patients with heart pumps who will be given either the gene therapy
or a placebo as part of a clinical study partially funded by the
British Heart Foundation and sponsored by Imperial College London.
"Advanced heart failure is a progressive condition that results in a
poor quality of life and shortened life expectancy," said Dr Nick
Banner, the consultant cardiologist at Harefield Hospital who
carried out the first infusion.
 "The best treatment currently available is a heart transplant but
the shortage of donor organs in the UK means that many patients will
die on the transplant waiting list."
Adams is on the waiting list and has been living for more than two
and a half years with an LVAD, which must always be connected to an
external power supply via a lead through his abdomen.
"You can't just jump in the shower...and it's difficult sleeping
whilst being attached to it. Everywhere I go I have to carry the
power supply and spare batteries in a backpack," he said.
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"Of course the best thing that could happen would be...for the gene
therapy to prove to be a 'miracle cure' for myself and other
patients."
Plans for the study were first announced last year and complement an
ongoing 250-patient trial, also funded by Celladon, which is
investigating the benefits of gene therapy in 250 people with less
advanced heart failure from Europe and the United States.
That trial is expected to report results next year.
The success of the experimental Mydicar gene therapy program is
critical to Celladon, which listed on the United States' Nasdaq
stock market in January. Mydicar is the company's most advanced
product.
(Reporting by Ben Hirschler; Editing by Sophie Walker)
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