b'UNITED TO ADVANCE OURindustry Thanks in part to the clinical testbed available on our campus,out to multiple pharma companies to validate and develop novel led by Richard Zane, MD, professor and chair of emergencytherapeutics based on the results of this collaborative project.medicine at the CU School of Medicine and chief innovationThe real innovative part in all of this is that Dr. Smith officer at UCHealth, big ideas like this are put into practiceaccomplished in three months what would have normally taken and honed for patient care. Zane oversees innovation for thethree years by using data science to examine millions of pieces The Next Generation of Digitallyhospital system as well as all the emergency departments,of data to determine the right drug for the right patient in the urgent care centers, and virtual health, and his leadership of Enabled Healthcare Innovations those efforts helped make this project possible.right circumstances, said Zane. The initial data from this project is very exciting and can hopefully At the University of Colorado Anschutz Medical Campus,Starting with a group of 100 patients, the team did exactly whatbe used to transform the treatment of AML and other blood putting our patients first is in our DNA. One of the manythey set out to do and analyzed 10 million data points. Whencancers. But before big ideas can materialize into clinical use, ways we seek to provide exceptional healthcare is throughRich Zane, MD (left), Clay Smith, MD (center), and Kimberly Muller (right)you do the math, as Smith quipped, thats roughly 50 millionresearch funding is key. cross-campus collaboration and partnerships with experts.review data on a computer screen at the CU Anschutz Medical Campus. floppy disks worth of data! CU Innovations exists to do just that. By bringing togetherBecause Smiths project was so unique and exploratory, it was A new frontier in science is reliable ways to take advantageout of scope of what traditional grant funding mechanisms industry partners, entrepreneurs, and investors, CUtechnologies and treatments for AML. Together, they came upof all the cancer data weve acquired and generate insightsserve. Thats why CU Anschutz developed alternative funding Innovations is helping our researchers create biomedicalwith an idea to use cutting-edge technologies to look at single-celland hypotheses from that data to move the field forward,sources through the Chancellors Discovery and Innovation technology that improves patient care.DNA, RNA, and protein changes in single cellstermed multi-omicssaid CU School of Medicine Dean John Reilly, MD. TheresFund (CDIF). With an initial $10 million in institutional and We see ourselves as the main gateway to a large research andanalysis. Looking at the single-cell level with multi-omics isbeen a real thrust over the last 18 months on this campus toprivate philanthropic resources, the CDIF exists to provide clinical infrastructure, explained Kimberly Muller, executive directorpotentially key, as relapse in AML and other cancers typicallyexpand our informatic capabilities and Dr. Smiths project is aprofessionally managed early-stage seed funding that helps of CU Innovations. The whole concept of what we do is to createcome from small numbers of cells that have previously beenperfect example of how we can use big data to offer a moreadvance the commercialization of new discoveries. the next generation of healthcare products, whether its drugs,invisible to traditional technologies. personalized treatment plan for patients. The result? In less devices, or redesigning care delivery. We bring industry partnersDr. Jordan and I approached CU Innovations with an idea ofthan a year, the team had the largest AML dataset in the world.When we have novel ideas such as Smiths AML work, and pair them with clinicians to figure out how we can create thosehow to apply these new cutting-edge technologies to improvephilanthropy is key to moving those ideas forward; investment Using advanced informatic science, we were able to developfunds like the CDIF are critical to seeing those ideas come to next-generation products that can ultimately reach patient care. care of patients with AML and other forms of blood cancers,an initial approach for stratifying patients based on whetherfruition. The success of Smiths research project is a testament Thanks to hospital partners at Childrens Hospital Colorado andsaid Smith. Our hypothesis was to specifically look at thethey were likely to respond to chemotherapy or another newerto the power of partnership fueled by philanthropy. An innovative the UCHealth system, CU Innovations has established a livingleukemic stem cell with these technologies, which we andtreatment termed venetoclax/azacytidine, Smith explained.idea that turned into something real and tangible, and offers clinical laboratory for testing and validating the best healthcareothers think are the most important cells out of all the AMLAnd in less than two years, we were able to get those findingshope for many other disease treatments.technologies. One recent partnership with Clay Smith, MD,cells for determining whether a patient will relapse or not. director of the Blood Disorders and Cell Therapies Center atHowever, applying this multi-omics approach generates more University of Colorado Hospitalthan 10 million data points for and associate chief, Division ofevery patient. To successfully Hematology, CU School of Medicine,This project was so importantanalyze such large amounts of What are the biggest challenges in cell and gene therapyrevolved around developing newbecause it set a paradigm fordata, we then applied machineMICHAEL VERNERIS, MDand how are the partners on campus poised to overcome?therapies for adults with leukemia. learning and AI to achieve twoCELLULAR THERAPY OPERATIONS PROGRAM,His work specifically focuses onwhat we can do when we allaims: figuring out which patientsSCIENTIFIC & MEDICAL DIRECTOR Producing and testing new cellular therapies comes with several will respond to which availablechallenges. For example, moving a laboratory concept into acute myeloid leukemia (AML), acome together, as a campustherapies and equally important,the clinic requires a team well-versed in good manufacturing type of cancer that affects the bone marrow and blood and is the secondand with industry and hospitalto identify new targets andWhat are the objectives of the practices, regulatory requirements, and the challenges of drugs that would overcomeCellular Therapy Operations Program? first-in-human clinical trials. There are also affordability issues most common leukemia type foundpartners, to solve the mosttreatment resistance in patientsThe Cellular Therapy Operations Program (CTOP) facilitatesas these treatments and their testing can be expensive. We are in adults. pressing problems in healthcarewho dont respond to currentthe production of investigator-initiated cellular therapies andfortunate the University and Medical School leadership have AML is also one of the mosttherapies. helps bring these therapies to patients on the CU Anschutzinvested in these transformative therapies.aggressive types of cancer and sousing data-driven solutions. Medical Campus. The CTOP team engages with laboratory is its treatment: chemotherapyHow to get 10 million dataFitzsimons Innovation Community is building a commercial and, if necessary, a bone marrow KIMBERLY MULLER, CU INNOVATIONS, EXECUTIVE DIRECTOR points per patient and figure and clinical investigators to understand the technology andmanufacturing site to support the growth of cell and gene therapy transplant. What makes AMLout how to analyze them was the projects development. In collaboration with leaders at theinnovation. How does this align with the CU Anschutz Medical particularly cruel is that its caused not just by cancer cells, butan arduous task to say theGates Biomanufacturing Facility, we determine what studies willCampus investment in cell and gene therapy capabilities?by leukemia stem cells, which resist chemotherapy. The bigleast. It all started with getting the right people together. be needed for investigational new drug (IND) submission andIts an exciting addition to our campus community. We continue question to solve was how to come up with a therapy that isTo accomplish this, Drs. Smith and Jordan put together a largewhen and how to start the technology transfer. In conjunctionto build a critical mass of researchers and clinicians with interest precisely targeted at the cancer and has the fewest side effects multi-disciplinary team, complete with clinical, laboratory,with the investigator, we compile the IND application, write theand expertise in producing and clinically testing cell and gene on healthy cells.computer science, and bioinformatics expertise. From there,clinical trial, and manage regulatory requirements needed fortherapy through this development. CTOP will be beneficial for CU Innovations brought in one of the leading groups in dataclinical testing. Finally, we engage with clinical research teamsany commercial phase manufacturing venture and will further Smith was already working with another researcher on campus,analysis and AI and formed a collaboration with UCHealth toand physicians at Childrens Hospital Colorado and UCHealth tostrengthen our commitment to change the practice of medicine. Craig Jordan, PhD, chief, Division of Hematology, on newtackle this problem of treatment resistance in AML. conduct clinical trials. 8Fitzsimons Innovation Community Annual Innovation Report 9'