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Regenerative Medicine: Has It Reached a Tipping Point?

By Peter Winter
Editor, BioWorld Insight

SAN FRANCISCO – For more than a decade now the technologies associated with the regenerative medicine and cell therapy sectors have been hailed as the "future of medicine."

While that lofty projection still holds true, investors have started to become impatient with companies developing therapies and their slow rate of progress, a fact acknowledged by Geoff MacKay, president and CEO of Organogenesis Inc., and the incoming chairman of the Washington-based Alliance for Regenerative Medicine (ARM).

He was participating in an industry panel for the Alliance's third annual State of the Industry briefing during the Biotech Showcase 2013 event, which is running at the same time as the J.P. Morgan Healthcare Conference.

Speaking to a packed audience of pharma and biotech company delegates as well as industry analysts and media, he noted that the sector is starting to finally mature and already products are now reaching the market.

"The maturity is reflected in the fact that there are now seasoned management teams heading successful companies and new constituents are entering in the space such as translational medicine centers. These include the new London-based Cell Therapy Catapult center and the Toronto-based Center for Commercialization of Regenerative Medicine. Both of which have a CEO and a mandate to help support the development of foundational technologies that accelerate the commercialization of stem cell therapies and related technologies."

With multiple products already on the market targeting a range of diseases and a critical mass of compounds in clinical trials the "debate on the future of this industry is now moot, as it is already arrived," he added.

MacKay cited ARM analysis showing that product sales have almost doubled in the last two years, from $460 million in 2010 to an estimated $900 million in 2012.

Another panelist, Susan Soloman, CEO of the New York Stem Cell Foundation, noted that she has seen a significant change in the industry over the past year and reiterated the notion that the future is here.

The next phase is to industrialize the production of stem cells to enable their advancement into human trials and to effectively deliver the quantities needed for drug discovery purposes. Her own foundation is funding research in that area in order to be able to produce identical stem cell lines in large quantities.

This "industrialization" theme was echoed by Robert Shaw, commercial director at the Stem Cell Initiative, EMD Millipore. Manufacturing scale-up and standardization along with scientific validation remain major barriers to the success of the sector. He said he believed the market continues to be in development and will be coming to fruition in the years 2015 to 2020.

The stigma that the industry is just "Cells 'R' Us" with a history of money-losing projects needs to be removed, said Jeffrey Jonas, president of Shire Regenerative Medicine. In order to do that, it will require companies to have "reproducible clinical data with a meaningful effect size." That needs to be combined with a clearly defined regulatory and reimbursement pathway.

Reflecting the upbeat mood of the panelists, Chris Calhoun, CEO of Cytori Therapeutics Inc., said the sector has reached a tipping point for regenerative medicine.

After a decade in development, the industry is seeing late-stage clinical trials being undertaken with the promise of significant data reads from those trials emerging as early as next year. Another encouraging sign for the sector is that the safety of products is being established and there is starting to be a continual stratification of peer group companies working to develop cell therapies.

Well-defined business models are being identified by companies so that investors can now establish qualified metrics to track the progress that is being made.

Gil Van Bokkelen, CEO of Athersys Inc., of Cleveland, and the outgoing chairman of ARM, agreed. "We have reached that inflection point; aggregate revenues have doubled, and there is the potential for enormous growth going forward."