Stem cells hold the promise of ushering in a new era of regenerative medicine, as scientists and engineers have made significant progress in directing these powerful cells towards use in drug screening models and replacements for failing tissues. More recently, scientists have developed even more efficient and alternative methods for reprogramming cells into their desired state.
But as panelists at the recent Washington Biotechnology & Biomedical Association (WBBA) symposium on the “Today and Tomorrow” of stem cells in regenerative medicine emphasized, the stem cell promise is an exciting but risky wager that seems extreme even by biotech standards. It is easy, even for those familiar with the field, to understate the incredible challenges imperiling the transition of stem cell therapies to the clinic. Yet against all odds, two companies have pursued clinical trials and many more are fast at their heels. What gives? And what distinguishes this challenge from the commercialization of more traditional biotech approaches?
First, take the FDA. According to Joseph Gold, the senior director of neurobiology and cell therapies research at Menlo Park, CA-based Geron Corp. (NASDAQ: GERN) and a panelist at the WBBA event, the U.S. regulatory body was not really “conversant” in embryonic stem cell-derived therapies before Geron submitted its 20,000-page proposal for beginning clinical trials among patients with spinal cord injuries. This application to start trials was the largest of its kind ever presented. To date, there has only been one other company to seek FDA clearance to study an embryonic stem cell therapy in humans: Advanced Cell Technology (OTCBB: ACTC). Clearly playing a world role in the oversight of a constantly evolving landscape in biomedical technology, the FDA must understand and demonstrate competence in effectively regulating stem cell treatments for present and future companies to have a prayer of getting them to market. But Joe Gold argues that Geron’s and ACT’s application processes have improved, and in fact given birth to, the FDA’s ability to regulate stem cells as a new type of therapy.
Then comes funding, scaleup and manufacturing: requirements for any biotech but which may in the context of stem cell therapies point to a unique reliance on startups in pushing the field forward. Although preclinical stem cell research is well funded by the National Institutes of Health, federal dollars don’t support the costs of current good manufacturing practice (cGMP) manufacturing and clinical trials. And on the other end of the spectrum, Big Pharma appears hesitant to move forward with internal cell therapy programs because, like venture capitalists, they remain underwhelmingly convinced of the commercialization potential of such an infant scientific development. Pfizer, Bayer, Merck, and others have expressed interest and developed programs for partnering with cell therapy collaborators in academia and early-stage industry, but are not about to go it alone. This parallels the unfavorability of innovation within large pharmaceutical companies increasingly even in the space they know best-pharmaceuticals-where in the modern framework it’s preferable to acquire the next Viagra from what will likely be a small company. The small company environment can catalyze the initial phases of cell therapy translation better than anywhere else, partly because the level of infrastructure is right for tackling the nontrivial scale-up and manufacturing of cGMP cells, a proof-of concept endeavor that is impossible in the university discovery setting, but which, once demonstrated, can be augmented with acquisition by Big Pharma. The current state of affairs suggests, then, that the rate-limiting step for pushing stem cell treatments into mainstream use will be capital investment at the startup phase.
So why, in the midst of an extended recession and with hurdles raised higher on all fronts than those for new pharmaceuticals, is the field (albeit slowly) advancing? Even with a clear translational path, investors in some cases remain reluctant while conditions are favorable enough in others to get the ball rolling. What is happening? One possibility is that we are looking at the cusp of translation, which has happened time and again in biotech and which often gives rise to a similar teeter-tottering of a new wave of medical therapies over the edge into the clinic. John Mendlein, the executive chairman of Fate Therapeutics and also a panelist at the WBBA event, posited an apt reminiscence of the challenges facing the implementation of monoclonal antibodies as pharmaceuticals 20 years ago. Roadblocks stifling the technology, manufacturing, funding, and regulation scared every facet of the industry at the outset. The promise of the field prevailed, and what was a process became a profit. That’s what biotech is all about, and maybe this time is no different.
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