Stem Cell Summit Preview
Five years ago, the prevailing wisdom was that stem cell therapies were at least a decade away from commercialization. Embryonic stem cells, according to media reports, were the predominate form of stem cells. Finally, five years ago we held the first Stem Cell Summit—in a small hotel near Chicago’s O’Hare airport.
At that first meeting we said that stem cell commercialization was imminent and that sales within five years would reach $100 million. Today, five stem cell branded products are on the market, sales this year will likely exceed our estimates from five years ago and, we estimate, one in ten surgeons in the United States has started employing stem cells therapeutically. At this pace, by 2015, virtually every surgeon in the United States will be incorporating stem cells in either autologous, allograft or cultured forms into their practices.
What a difference five years makes.
RRY Publications’ Stem Cell Summit in New York has found a home in the historic South Seaport section of Manhattan. South Seaport is where Fulton Street and South Street meet. It’s where the tall ships dock and cobblestone streets take visitors back to an earlier time. For the fifth annual New York Stem Cell Summit, entrepreneurs from Europe, Israel, Latin America, Asia, and the U.S. are finalizing their PowerPoint presentations, six key scientific leaders will bring all attendees up to date on the science and as many as six NEW stem cell products will be introduced.
This year’s meeting will have more attendees, more presentations and cooler give-aways than last year (Hint: the pens are multi-functional). It is still one full, total-immersion day with, as was the case last year, outstanding networking opportunities during lunch and the breaks.
Five Preview Highlights
Having surveyed the presentations here are five preview highlights although, as we learned last year, there are still plenty of surprises expected from the podium.
Spine Disc Rehydration – Rehydrating the spine nucleus with stem cells, with or without bone morphogenic proteins, has been bouncing around the periphery of stem cell science for a decade or longer. A single study in 2009 changed all that. Degradation of the spine disc from either acute or chronic disc injury and/or degeneration is the basis for most of today’s spinal implant sales. If physicians could rehydrate a degenerated disc reliably with stem cell injections it could transform the business of selling spinal fusion implants and instruments. But the nucleus is not a benign environment. Could stem cells survive the ph levels, the compressive and stress forces or avascular nature of the nucleus? Early in 2009 data emerged from a small canine study (n=12) that adipose stem cells in a hyaluronic cocktail could rehydrate the disc to near normal levels. Then on September 10th, a stronger study was presented at the Osteoarthritis Research Society International meeting in Montreal. The sheep study used a single, direct low-dose injection of allogeneic or "off-the-shelf" adult stem cells into the degenerated disc nucleus. At the six-month follow up, discs that were initially severely damaged and degenerated had regenerated and “become indistinguishable from healthy non-degenerated discs in their histopathology, cartilage content, height, and structure” (quote from the study’s principal investigator and one of the Stem Cell Summit’s presenters). The control group of severely degenerated discs was either not injected or was injected with hyaluronic acid. The control group had significantly lower disc height (P<0.01), disordered disc structure (P<0.01), disrupted histopathology (P<0.01), and reduced cartilage content (P<0.05) at the end of six months.
Clinical Study Set-Backs – Not all news in 2009 was good news. In August, the company that had received the first FDA approval for an embryonic stem cell trial announced that its IND for a study of its cell therapy for sub acute spinal cord injury had been stopped. Geron announced last August that its clinical study of GRNOPC1 in patients with spinal cord injuries was placed on hold by the FDA. With respect to embryonic stem cell commercialization, the future keeps stretching out into a vaguely defined future. Osiris, which started 2009 as the market leader in both market value and FDA approved cultured stem cells in the market, reported TWO clinical study setbacks. On June 23rd, the Osiris company reported that chronic obstructive pulmonary disease patients receiving Prochymal, the company’s formulation of adult stem cells, did not have significantly improved pulmonary function. The patients DID experience decreased systemic inflammation and Prochymal was shown, again, to be safe. But…no improved pulmonary function. Then, three months earlier, Osiris stopped enrollment in its study of Prochymal for patients with Crohn’s disease. Turns out there was a design flaw in the study. Patients were asked how they felt. If they answered a particular way, they got long-term Prochymal treatment. Even though the study was double-blinded, it administered Prochymal in two phases. So patients and their physicians who participated in the first phase of the study could figure out who had Prochymal and who did not. At the end of the first treatment phase, all of the patients were asked, “How do you feel?” By answering a certain way, the patients could ensure enrollment in the long-term Prochymal treatment arm. So, many patients in the study used the magic words to get into the long-term treatment arm. Oops. The good news is that Prochymal is safe and a powerful anti-inflammatory treatment. Slam dunk works. Ten million patients a year can use this drug. Right now. But getting this product to market is proving to be a real puzzle in the United States. Osiris CEO Randall Mill’s presentation at the New York Stem Cell Summit will certainly be one of the most anticipated presentations.
New Allograft Stem Cell Products – Stem cells from human allograft placental material? Pluristem, the Israeli company developing adult stem cell products from placental material is one of the most innovative new stem cell companies in years. Bill Prather, VP Corporate Development will make the presentation on behalf of Pluristem. Then two of the leading allograft processing companies—MTF Foundation and AlloSource—earlier announced innovative new allograft based stem cell products and we will hear about at least one of those from the podium at the Summit. Through its distribution partner Orthofix, MTF announced that it had developed a second generation allograft stem cell product called Trinity Evolution (TE). Like the original Trinity, TE is an allograft bone matrix with concentrated progenitor MSCs derived from cadaveric donors. AlloSource, the rapidly growing and large allograft processor based in Colorado, has formed a distribution partnership with NuVasive for the original allograft stem cell product, Osteocel. We expect to hear many comments regarding the coming evolution of allograft stem cell products.
Cosmetic Stem Cell Applications – Five companies representing a wide range of cosmetic applications of stem cells will be presenting. Where will commercialization of stem cells likely go next? The really big markets and, therefore, development capital may well be in plastic surgery and cosmetic applications. One of the highlights will certainly be Dr. Angela Christiano’s review of her work with hair follicle stem cells. Dr. Christiano, who is Professor of Dermatology and Director of Research at Columbia University, gave one of the most compelling presentations NOT heard at last year’s New York Stem Cell Summit. Her presentation regarding the rich stem cell environment in hair follicles came the day after the Summit at the Columbia University stem cell symposia we attended. But leaders in the race to commercialization are among the following strong presenters. For dermal regenerative therapies the leaders are Garnet BioTherapeutics and Histogen. For breast reconstruction and aesthetic body contouring Cytori Therapeutics will present both a facilitating technology and the science of using adipose tissues with their rich stem cell component to address these very, very large markets.
Cardiovascular Stem Cell Wins – It may not receive quite as much attention, but the gains in cardiovascular stem cells keep piling up. For years, literally, we have been collecting patient stories regarding the use of autologous stem cells to treat various forms of heart disease. Typically, these are patients who are nearing the end stages of their disease and the heart muscle is progressively deteriorating. Several strong studies have been released over the years that describe exactly why the anecdotal information is what it is. In 2009, the Proceedings of the National Academy of Sciences waded in and published a study from Brigham and Women’s Hospital that stated unequivocally that mesenchymal stem cells (MSCs) exhibit vascular smooth muscle and endothelial cell differentiation, contribute to large and small vessel formation, reduce infarct size and increase ejection fraction. To quote directly from the study’s conclusions; “Importantly, MSC engraftment correlated with functional recovery in contractility (R = 0.85, P < 0.05) and MBF (R = 0.76, P < 0.01). Together these findings demonstrate long-term MSC survival, engraftment, and trilineage differentiation following transplantation into chronically scarred myocardium.” From the podium at this year’s New York Stem Cell Summit, we will hear from five companies about their latest successes treating heart disease with autologous, cultured and, perhaps, allograft stem cells.
The New York Stem Cell Summit will start promptly at 8:00 am on February 16th at the Bridgewater’s facility at the South Seaport. We hope to see you all there.