BioWorld International Correspondent

LONDON - NovaThera Ltd. has teamed up with the Texas Heart Institute to scale up production of its stem cell-derived cardiomyocytes and take them to clinical trials in treating the aftereffects of heart attack and stroke.

"What we are interested in is the problem of how you go from small-scale production of cardiomyocytes, which we can do, to something that uses standardized processes to scale up production and get enough cells to treat hundreds of thousands of people," Gareth Roberts, NovaThera's CEO, told BioWorld International.

The Texas Heart Institute has experience using patients' own bone marrow as a source of replacement cells and has the facilities for completing preclinical studies, including animal models of ischemia and infarction. In 2004, it was the first center in the U.S. to get FDA approval for a human trial of adult stem cell therapy to treat congestive heart failure.

The institute has held preliminary talks with the FDA about the regulatory pathway for NovaThera's cardiomyocytes. "These discussions are still at an early stage, though there has been progress. But parts of the regulatory requirements can only be clarified as we move through the development of the process," Roberts said. "Until you make practical progress, it is only possible to have generic discussions; we need to agree on specifics."

The parties will bear their own costs in the collaboration, with NovaThera being responsible for producing the cells and the institute for carrying out all preclinical work and getting regulatory approvals for human trials.

Roberts said it is useful to develop and manufacture the cells at a distance from the hospital at which they will be transplanted, as it is a crucial element for showing that the manufacturing and shipping systems are robust and reliable. "Making cells in situ is never going to be commercially viable approach. You've got to show that the cells can be produced in a central facility, whether you are shipping them 10 miles or 10,000."

Apart from distance, the other attraction of working with the Texas Heart Institute is its massive experience with cardiac surgery and the fact that it has secured FDA approval for cells transplants.

Ed Yeh, chair of the department of cardiology at the institute, said it is clear that the use of embryonic stem cells could provide a new approach to heart repair. "This [collaboration] allows us to continue our first-class research, initially by testing the cells in animal models of heart injury and finally by carrying out full clinical trials."

NovaThera was spun out of Imperial College London to commercialize research from the Tissue Engineering and Regenerative Medicine Center. Apart from cardiomyocytes, the company has reliably differentiated blood and lung cells, using the same basic process for encouraging stem cells to differentiate into other cell types. The company in January was awarded a government grant of £3.75 million (US$6.9 million) to develop bioprocesses for generating cells at an industrial scale.

Roberts estimated it will be two years before the cardiomyocyte cell line will be ready for human trials. "It has already been shown that cell transplants are useful, and there are limited side effects. Now we are trying to build the momentum. The key thing that needs to be done next is the real translational research to show how stem cell research can benefit patients. The cellular biology is spectacular, but we've got to show that stem cells can improve health care."