At the Regenerative Medicine Institute (Remedi), researchers are working on a promising adult stem cell therapy for osteoarthritis, a novel technique for extracting stem cells from bone marrow, and a manufacturing facility to produce clinical-grade stem cells. All in all, Remedi is doing rather well for a research institute that started from scratch less than five years ago.
The institute, based at the National University of Ireland in Galway, specializes in applying gene therapy and adult stem cell technology to enhance tissue regeneration and repair. Established in 2003, it is one of nine Centers for Science, Engineering and Technology funded by Science Foundation Ireland (SFI) and industry.
Scientific director Frank Barry is most excited about the new therapy for osteoarthritis that is currently undergoing efficacy and safety trials in animals, and the as-yet unpublished technique to "pull" mesenchymal stem cells from human bone marrow using cloned chicken antibodies. "There are lots of groups looking for new and better ways to extract stem cells from bone marrow. We're the first to do it with this new antibody approach. So that's novel, and we've filed for patents on it," says Barry.
This work follows from a finding of Barry and colleagues showing that injecting mesenchymal stem cells into arthritic joints in goats helped regenerate damaged tissue and slow progressive degeneration (Arthritis Rheumatol, 48:3464-74, 2003). They are now investigating two therapeutic approaches. "We can inject stem cells direct into the arthritic joint, or deliver the antibodies and have them pull the stem cells into the joint," says Barry. The aim in both cases is to harness stem cell signaling to trigger tissue regeneration and repair. If all goes well, he expects clinical trials to start in 2010.
Cardiovascular disease is another target, and Remedi scientists had initial success with gene therapy in animals, using a virus to deliver a nitric oxide synthase gene to cells in blood vessels. Nitric oxide levels rose, and the animals' arterial channels widened. However, given growing concerns about the safety of viral vectors, they are now investigating nonviral methods such as liposomes and sonication.
Remedi's team is currently in the process of applying for its second five-year tranche of funding from SFI. So far, in addition to core funding of €20 million ($30.9 million US) for five years, they have gathered €40 million ($61.8 million US) in external funding, much of it from industrial partners such as Smith & Nephew and Medtronic, alongside grants from the European Union and Ireland's Health Research Board. By the end of their second five years in 2013, they will need to be financially independent of SFI, which seems an achievable goal.
Tim O'Brien, Remedi's clinical director, and Barry both worked in the United States before returning to Ireland, so the institute retains strong collaborations with several US institutions, including Arnold Caplan's team at Case Western Reserve University in Ohio. Caplan, a pioneer of adult stem cell science, ranks the Galway institute among the world's best centers for innovative therapies. They have "very promising data and creative, multipronged approaches to the treatment of osteoarthritis," he says.
Barry credits the positive review in part to what he calls Ireland's enabling environment. "The funding is there, and the infrastructure is getting there, and it is absolutely important that this continues." And Galway, he adds, is a great place to live and do science.