By James DeGiulio --
Last year, we reported on the revival of the therapeutic potential of gene therapy, after the technique hibernated for years due to questions of safety and inefficient gene delivery (see "Gene Therapy Experiencing a Revival"). The revival continues with the announcement of a new gene therapy clinical trial initiated by San Diego startup Renova Therapeutics. Renova announced that researchers at the VA San Diego Healthcare System are entering Phase II testing of a very promising adenovirus-based gene replacement therapy for patients with Congestive Heart Failure (CHF). If the human trials follow the animal data, Renova's therapy could substantially extend the lifespan of humans suffering from CHF. The NIH has shown great faith in the gene therapy project, contributing $23 million so far. However, if Phase II proves successful, Renova would likely look to partner up with a larger company to move into Phase III trials. The Phase II data is expected sometime in 2012.
CHF is a condition where the heart is unable to pump sufficiently to provide the body with enough blood flow to match its needs, since the hearts of CHF patients contain low levels of cAMP. The prognosis for CHF is dismal -- 50% of those diagnosed die within 4-5 years. CHF afflicts more than 5 million Americans. The project began when Dr. Kirk Hammond (at left), Renova founder and a cardiologist at the VA San Diego Healthcare System and Professor of Medicine at the University of California, San Diego, discovered that the adenylyl cyclase type 6 (AC6) gene was downregulated in CHF patients. Dr. Hammond later discovered that increasing the heart's content of AC6 improves heart function. Dr. Hammond and his group sequenced and patented the human AC6 gene.
The gene therapy technique uses the same adenovirus vector (Ad5.hAC6) as previous gene therapy candidate Generx, which reached Phase IIb/III studies before Schering determined that the protocol could not deliver enough gene product after 400 patients were screened. However, Renova's technique improves expression of AC6 gene product by delivering the adenovirus via a coronary catheter to the arteries of the heart, similar to angiography, which is preferable because the technique does not require surgery. This gene delivery technique is covered by U.S. Patent No. 5,792,453. The results from the preclinical animal studies were excellent, the data showing that mice with cardiomyopathy (a type of CHF) who received the AC6 gene therapy lived as long as normal, healthy mice. Whether human CHF patients experience an extension in lifespan remains to be seen.
The first trial site invites active and retired military personnel to volunteer in the clinical trial. They must be between 18 and 80 years old, and diagnosed with CHF. A second clinical trial site has recently opened at the William Beaumont Hospital in Royal Oak, Michigan, which is open to all subjects and is not limited to military. More information on the gene therapy clinical trial can be found on the AC6 Gene Transfer page at the ClincalTrials.gov site as well as on Renova's website.
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