Issue link: https://tmcpulse.uberflip.com/i/515661
t m c » p u l s e | m a y 2 0 1 5 12 We spend $400 million a year on biomedical research and yet we did not have much in the way of drug devel- opment or other experimental therapeutics. We have sort of been on both ends of the research spectrum; third-party clinical trials on the one side, basic science on the other and not that much in between. We are trying to bring forward that opportunity at several levels. We are hiring chemists and developing a center for drug discovery so that we can bring forward small molecule therapeutics. We are building the sup- port structure that would allow that to happen. In terms of pilot projects, we have the opening of an Innovation Development Center, and other commercialization efforts. We are trying to pull the most promising projects out of the lab and put them along a critical path towards some kind of therapeutic or diagnostic outcome. We are just getting started, so it is too early to judge our efforts. But it has been a lot of fun. Q | Do you believe the multi-institution institutes like Innovation and Clinical Research will serve as a springboard for the next generation of discovery and commercialization? A | Yes, I can see a future where all of the institutions interact robustly on the scale of the TMC, in much the way you have laid out in your innovation programs. That said, I don't see the individual efforts of the TMC institutions going away. I only see them strengthened. Because the larger entity—the interaction with the third parties, big pharma, and so forth—they will not want to develop every single project that comes out of all of the institutions. The institutions will want to ensure that their own individual projects have the maximum impact through their own natural course of development. I have seen enough of these projects now, just going back five years. I started as a chair of biochem- istry with one particular small molecule therapeutic starting in 2005, and am seeing how it is only now get- ting to phase one clinical trials over a 10 year period. These things have such a circuitous root. On the one hand, you wonder if it can only come to fruition in an academic setting. This project could have been shut down five times over if it was in industry, and it may have been rejected in a TMC-wide down-select pro- cess. But there are going to be these projects that indi- vidual institutions believe in. For whatever reason—the specific need that is being addressed, or the particular faculty member, or whatever set of criteria they have in their own institution—they will want to be able to drive that forward, independent of other exigencies. So I see that going forward we will have a hub-of-hubs sort of model of innovation. There will be hubs at the institu- tions whose projects may feed into the TMC program or be developed independently. Q | Someone once told me that on the east and west coasts, they worry most that the Texas Medical Center member institutions will actually come together programmatically, and in doing so, will become the dominant force in discovery and com- mercialization. Is that all talk, or do you believe there really is an opportunity here? A | I believe this 100 percent. The strong research institutions in the TMC are fully onboard and this will be a very attractive center in the coming years. The turnover in the leadership of all of the TMC institu- tions has really changed the culture toward commer- cialization. This is not just to generate revenue to support our missions, but to maximize the impact of what we do. The Baylor Miraca Genetics Lab is a great example of this. Q | Tell us more about that. I think it is a perfect example of something that has grown out of research and became a service that grew into a business. A | It started in the early '70s, with Tom Caskey and Art Beaudet starting to see pediatric patients with odd biochemical anomalies that they could trace back to a genetic cause—an inborn error in metabolism. That led to a more robust formulation of a biochemi- cal genetics laboratory and collaboration with Texas Children's Hospital. The unique features of this from the late '70s, when this was formalized and growing into the arena of molecular genetic diagnostics, were: patient volume; our world-class genetics department that was built over decades under Tom Caskey and, later, Art Beaudet; and the national Human Genome Sequencing Center under Richard Gibbs. Those three things together led to this unique opportunity to drive innovation in clinical genetic diagnostics. Art Beaudet saw this and started pushing the model of an academic reference lab, the Medical Genetics Lab (MGL), that would continually innovate and would be essentially forcing the conversation around the clinical utility of molecular genetic diagnostics. They would typically offer a test before most doctors knew it would be use- ful, and offered it to the academic physician scientists who had patients with complex cases with a suspected genetic component. In the late '90s, early 2000s, the vision was to replace karyotyping by chromosome spreads with chromosome microarrays. So we were the first to launch that commercially, and it was a spectacular success. For the first time you were able to diagnose not only large chromosomal rearrangements and trisomies, but also small deletions and duplications by basically reading a chip. The uptake of that was tremendous. It drove revenues for quite a while. So it developed into a $40-50 million a year revenue business. The latest innovation involved bringing Richard Gibbs and his team on board to create the first clinical whole exome sequence test. They were the first to launch a whole exome test in 2011, doing about 200 cases a month, with a 26 percent diagnostic rate. For you and I, having lived in the days in the mid-'80s when the suggestion that we would sequence even one human's genome was thought to be science fiction, this is remarkable. The thought that we can sequence the relevant genes of a patient over a few week period and then interpret the clinically relevant genetic vari- ants and return a diagnoses back to the patient really truly seems like science fiction. Q | Any closing thoughts? A | There are intense pressures on how academic medical centers are being funded and have been funded in the past. I think the public does not under- stand the threat that the TMC institutions face. Our institutions are where the innovative new medical treatments are developed and they are an invaluable national resource. The traditional fee-for-service medical revenues cross-subsidize our education and research programs, and they are under pressure and rapidly changing into a new model of medical care delivery. I view this generally as a good development, but it presents a major challenge for the next five to 10 years. We have to morph our old academic medical center model, with the traditional sources of support, into one where we develop new sources of support through carefully thought-out joint ventures and bring commercial funding into the mix in a meaningful way. To the extent we can align incentives between commercial interests and academic priorities, this should work. I think we have to take advantage of the market forces in biotech and pharma to drive what we do in better ways than we have in the past. I have spent the last five years making this argument to our faculty and they have responded positively, but it is too early to judge the outcome of our hospital joint venture with CHI, or our diagnostics joint venture with Miraca. And we have other JVs in the works. We are trying to lever- age our unique capabilities in research into some sort of a commercial format that brings a unique oppor- tunity to an outside venture but also brings a unique opportunity to support the academic enterprise. For the full interview, visit TMCNews.org The thought that we can sequence the relevant genes of a patient over a few week period and then interpret the clinically relevant genetic variants and return a diagnoses back to the patient really truly seems like science fiction.