The exciting state of regenerative medicine
Regenerative medicine has the power to transform healthcare, incorporating a wide variety of therapies. When incorporated into clinical practice, regenerative therapies may save USD 250 billion per year in the US alone, according to Gil Van Bokkelen, CEO of Athersys, speaking at the Biotech Showcase™ 2012 workshop on regenerative medicine.
“It will take time, but the next wave in the industry will be cell therapy and regenerative medicine,” predicted Robin Smith, CEO of NeoStem. Here’s a bit of what’s coming:
• Aastrom Bioscience’s therapy for critical limb ischemia is entering Phase III, based on success in changing the course of the disease. It has multiple applications.
• Advanced Cell Technology is transforming stem cells into retinal pigment epithelial cells to treat macular degeneration. Trials are showing cell migration and engraftment.
• Aldagen is in Phase II trials, using cell therapy six to eight hours after stroke. Results so far indicate multiple modes of action and improved perfusion and repair of brain tissue.
• Mesoblast is bringing a small molecule development environment to the development of off-the-shelf mesenchymal stem cell therapies with a low cost and a clear regulatory approval path.
Regenerative therapies must improve upon the standard of care and also demonstrate cost effectiveness to find a market. The recent success of ABH-Shire‘s Dermograft is a prime example. In a notable demonstration, “we took a retrospective look at three years of diabetic foot ulcers and built a model with more than 48,000 patients and one trillion data points, comparing the standard care with Dermograft. It showed Dermograft was cost effective after only six months,” said Dean Tozer, Senior VP of corporate development.
Results like that are feeding large pharmaceutical companies’ interest in regenerative medicine. Johnson & Johnson, among others, “keeps an eye out for new technology,” according to Jay Siegel, CBO, Johnson & Johnson. Many form dedicated teams as they build, buy and partner their way toward regenerative therapeutic pipelines.
Panelists agreed that regulatory hurdles exist, but they are not overwhelming. Tim Mayleben, President and CEO at Aastrom Biosciences, said the FDA is concentrating on the differences between autologous and allogeneic products as it reviews Aastrom’s submission. “The hurdles are to produce a consistent, allogeneic product. There’s a fine line to follow,” he said. Siegel added that “the agency is trying to look at multidimensional risk, and doesn’t always get it right across the huge spectrum of products. Therefore, the role of the industry is to engage and educate regulatory bodies.”
Companies also must engage and educate physicians. “It’s important to create space operationally for innovative therapies,” stressed Leanna Caron, VP and GM of cell therapy and regenerative medicine at Genzyme. That means training physicians to use the new products, as well as providing a rationale for their use.
Much of the life science industry is bent upon removing the risk from product development. Several organizations, notably the New York Stem Cell Foundation, are doing that. The Foundation is working with 60 postdocs and early career investigators on high risk/high reward projects and other resources, all focused on translational research. As Susan Solomon, CEO, explained, “Advancing regenerative medicine requires a longer view. By doing proof of concept studies for cutting edge work, projects can be scaled upon by pharma, biotech or government more easily.”
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