I’m not an academic, but I’ve been to many scientific conferences and read many more papers over the past 10 years or so. I know how important data is in research. I also know how important it is in communications (where visits, likes or shares are currency). Strangely, though, I had not considered its ubiquity in the commercialization context (apart from the rather obvious figures around investment). So, while the opening sessions at the World Stem Cells & Regenerative Medicine Congress, taking place this week in London, were themed “global commercialization” and “investment” they could just as easily (and perhaps should) have been called “Data is King” or “Show me the data and I’ll show you the money”, by virtue of the fact that nearly all of the presentations spoke about data as a key driver in moving stem cell and regenerative medicine forward into the clinic.
Betting on data
In a refreshing change from the typical plenary model, congress Chairman Chris Mason served as interviewer with a series of keynote guests. In his words, the aim was to get to the “nitty-gritty”, to uncover those nuggets of information that you don’t get from a powerpoint presentation. He was right; for the most part, the informality of a talk show format allowed keynote guests to detach from well-practiced corporate presentations and provide more insight into the factors that are driving decisions.
In the hot seat, Cell Therapy Catapult CEO Keith Thompson took us to the track, first by outlining his strategy of importing people who already had a strong track record in order to create a translation centre that could hit the ground running. Early on, CT Catapult has secured some strong projects and unveiled a state-of-the-art, highly modular cell manufacturing facility as the first step towards the goals of “debottlenecking the industry” and “moving the UK from a good science base into a clinical translation space.” Critical to this is an increased enthusiasm in the sector, which Thompson credited to better data. This data has removed some of the risk for investors (such as government) and regulators thereby creating opportunities to streamline processes and build the new facilities. Thompson likened it to horse racing – the only race he ever puts money on is the Grand National, where you understand the odds and the risk. People are now beginning to understand the bet in regenerative medicine – they know what they’re investing in.
On the back of better regulations, better facilities and better data, Thompson pointed out that clinical trials have gone up 20% in past year, a trend he predicted will continue.
Volume of data
Two presenters corroborated Thompson’s observation about clinical trials from an investment standpoint. Navid Nalik of Cenkos Securities showed several indicators of the positive trends in regenerative medicine, including very strong performances by companies in the stem cell space based on Nasdaq data from early 2014. He also cited regulatory changes in Japan, the UK, and the US as an impetus for movement, which is very reassuring for investment. But he stressed the need for more early phase clinical data to continue to propel this trend forward.
Less optimistic, at least in terms of investment figures, was Greg Bonfiglio of Proteus Venture Partners, who noted that while 2013 was a record year, up 43% over a very dismal 3-year period ending in 2012, there was a market correction in April of this year, and since then a bit of a downward trend. Still, the picture is positive overall – biotech is finally profitable and venture capital investment has improved, albeit still at historic lows.
The challenge for the sector, when it comes to attracting investment, said Bonfiglio, is that venture capital still wants pristine deals. The Valley of Death is still where it is. While there were a few examples of investment in companies based on very early phase data (phase 1 or 1/2), phase 2 data should be considered an absolute requirement in order to get the attention of venture capital to fund phase 3 trials. He suggested several strategies to help make this happen, including keeping research inside institutions for as long as possible in order to leverage the capacity, brain trust and other resources of the university, maximizing collaborations and take advantage of the newly emerging translation centres, such as CT Catapult in the UK, CCRM in Canada and other such as Wake Forest in the US.
Getting good data
The need for pristine deals does also raise the question of the quality of data. Most readers will be familiar with trial outcomes with less than optimal results. So what if some of these results are due to the parameters of the patient recruitment process? This was a question posed by Arnold Caplan of Case Western Reserve University, who pointed out that quite often, the patients who are enrolled in cell therapy trials are well advanced in the disease – and have often (unsuccessfully) been through innumerable drug options before being either eligible or selected for a cell therapy trial – all of which makes them less likely to respond to the cell therapy. Caplan argued that the patients who should be enrolled in cell therapy trials should not be the failed cases, but the newly diagnosed. “These are the people we need to be testing. These are the ones that will give us the data to move forward.”
Looking at data in new ways
Of the many noteworthy afternoon talks in the clinical trials stream, one is worth commenting on in terms the presentation of data. Kristin Comella, Chief Scientific Officer at Bioheart wowed the crowd with quick summaries of several clinical trials using muscle and adipose stem cells. Remarkable due to the breadth of conditions treated (including heart failure as well as a range of ischemic conditions) and the company’s ability to adapt to regulatory hurdles, the key message from Comella with regard to data, which she emphasized more than once, is that the sector needs to view the outcomes of regenerative medicine trials differently. Cell therapies are not the same as drug therapies: they need to be measured not just at the 3 and 6 month endpoints (though noted that her 6-month data showed some impressive outcomes for patients in the trials profiled), but at longer term endpoints such as 12 months or more, since the benefits of the therapy are long term and cumulative. This is in contrast to drug therapies, which typically show benefits only during the course of the therapy, with declines once the drug is stopped and leaves the patient’s system. She coupled this with the suggestion that the sector also needs to address a critical regulatory hurdle, which assesses cell therapies based on single outcomes and treatments administered separately, when in fact, the complexity of many conditions would benefit greatly from bundling more than one cell therapy into a single delivery system. A problem worth chewing on.
Day two at the congress suggests a focus on reimbursement and regulation, amid which I expect there will a great deal of additional data.
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