New gene therapy trials aim to mend broken hearts

Published: Tuesday April 30, 2013 MYT 8:06:00 AM

New gene therapy trials aim to mend broken hearts

LONDON: British scientists are stepping up clinical tests of gene therapy in a bid to help people with advanced heart failure pump blood more efficiently. Researchers said on Tuesday they planned to enrol patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held U.S. biotech company Celladon. After more than 20 years of research, the ground-breaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for an rare metabolic disease last November. In the case of heart failure, the aim is to insert a gene called SERCA2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of SERCA2a leads to ever weaker pumping in people with heart failure. Although drugs offer some relief, there is currently no way of restoring heart function and the prognosis for those with advanced disease is worse than for many cancers.One of the studies, led by scientists at Imperial College London, is part of a wider mid-stage Phase II project sponsored by Celladon that involves 200 patients worldwide, some of whom have already been treated in the United States and Denmark.

The second trial, which is due to start in the summer, will test the same treatment in 24 British patients already fitted with mechanical heart pumps to see how the approach may help in this particular setting.

It promises to be a long haul, with extensive Phase III studies still needed once results of the current mid-stage tests are received, which Celladon expects in the first half of 2015.

Gene therapy has experienced a series of advances and setbacks over the decades. The most notable blow came in 1999 when an Arizona teenager died in a gene therapy experiment. More recent results, however, have been promising in fields ranging from immune system diseases to blindness.

“It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment,” said Peter Weissberg, medical director of the British Heart Foundation, which is co-funding the second trial.

Because gene therapy replaces or boosts the activity of a faulty gene, it offers the possibility of a one-time “fix” – and that creates an economic challenge.

Any gene therapy is bound to be expensive, since a single dose could last a lifetime and the manufacturer will have just one shot at recouping its investment.

But Alexander Lyon of Imperial College, lead investigator on both studies, said it could be a cost-effective solution in heart failure if it avoided the need for interventions such as heart transplants at 200,000 pounds each. – Reuters

About bambooinnovator
Kee Koon Boon (“KB”) is the co-founder and director of HERO Investment Management which provides specialized fund management and investment advisory services to the ARCHEA Asia HERO Innovators Fund (www.heroinnovator.com), the only Asian SMID-cap tech-focused fund in the industry. KB is an internationally featured investor rooted in the principles of value investing for over a decade as a fund manager and analyst in the Asian capital markets who started his career at a boutique hedge fund in Singapore where he was with the firm since 2002 and was also part of the core investment committee in significantly outperforming the index in the 10-year-plus-old flagship Asian fund. He was also the portfolio manager for Asia-Pacific equities at Korea’s largest mutual fund company. Prior to setting up the H.E.R.O. Innovators Fund, KB was the Chief Investment Officer & CEO of a Singapore Registered Fund Management Company (RFMC) where he is responsible for listed Asian equity investments. KB had taught accounting at the Singapore Management University (SMU) as a faculty member and also pioneered the 15-week course on Accounting Fraud in Asia as an official module at SMU. KB remains grateful and honored to be invited by Singapore’s financial regulator Monetary Authority of Singapore (MAS) to present to their top management team about implementing a world’s first fact-based forward-looking fraud detection framework to bring about benefits for the capital markets in Singapore and for the public and investment community. KB also served the community in sharing his insights in writing articles about value investing and corporate governance in the media that include Business Times, Straits Times, Jakarta Post, Manual of Ideas, Investopedia, TedXWallStreet. He had also presented in top investment, banking and finance conferences in America, Italy, Sydney, Cape Town, HK, China. He has trained CEOs, entrepreneurs, CFOs, management executives in business strategy & business model innovation in Singapore, HK and China.

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