UNC SCI Conference: November 7‐8, 2014 2014 Research Program Update Marc A. Buoniconti President The Miami Project to Cure Paralysis The Buoniconti Fund Most Exciting / Productive Year Scientists and Clinicians Our educational and mentoring programs This has been the most exciting and continue to help train the next generation of productive year in the history of The scientists and clinicians in the area of Miami Project with increased extramural Neuroscience. Our faculty have national grant funding combined with the prominence in the area of CNS injury and successful translation of several of our Repair research and serve on numerous discoveries targeting brain and spinal cord national and international scientific boards as injury patients. well as NIH, DoD, VA and Foundation funding panels. FDA Approval 2014 Sub-acute / Autologous Schwann Cells We have now transplanted 4 sub-acute spinal cord Accomplishments injured subjects with autologous Schwann cells. We received FDA approval for this Phase I and clinical trial that will test the safety of this unique cellular transplantation procedure. This is a major Highlights accomplishment for The Miami Project since it represents the first academic institution that has received FDA approval for transplanting cells into spinal cord injured people. 1 UNC SCI Conference: November 7‐8, 2014 Chronically Injured Peripheral Nerve Injury Autologous Schwann Cells Last month we received approval from the We are using Schwann cells to target FDA to transplant autologous Schwann peripheral nerve injury to promote cells into chronically injured subjects. regeneration. This experimental bridging Preclinical data from our rodent and pig SCI approach was combined with nerve grafts studies supports safety and efficiency. targeting return of leg function. Deep Brain Stimulation (DBS) Adult Mesenchymal Stem Cells Deep brain stimulation (DBS) is being evaluated for the first time to target FDA approved trial in the testing of the neuropathic pain in spinal cord injured effect of adult mesenchymal stem cells subjects. Our first subject was successfully transplantation; the subject has been treated. transplanted and is being monitored New Floating Catheter Therapeutic Hypothermia We have submitted an addendum to the We have initiated therapeutic hypothermia FDA to use a new floating catheter for the in 35 severe spinal cord injured subjects. next series of subjects. This approach is One year outcome measures indicate that a safer and more appropriate for future 45% conversion from complete to chronic SCI studies. incomplete paralysis which is thus far the best hope for treating early SCI. We have submitted a multi-center trial proposal to NIH to continue these studies. 2 UNC SCI Conference: November 7‐8, 2014 Therapeutic Hypothermia Boot Camp TBI Patients The Miami Project has initiated a new Boot Therapeutic hypothermia is being utilized in Camp for chronically injured spinal cord severe TBI patients. We have initiated a injured patients. We intend to use a very three site multi-center trial (Miami, advanced conditioning/rehabilitation Houston, and Pittsburgh) to test the efficacy strategy to improve the quality of life of of therapeutic hypothermia in this patient people living with paralysis and combine population (HOPES Trial). this Boot Camp rehabilitation program with transplantation of Schwann cells in the chronic setting. Miami Project Boot Camp Preparing People with Chronic SCI for Cellular Therapies Epidural Stimulation Other combination approaches including epidural stimulation appears to maximize the beneficial Upper Limb Conditioning Motor Rehabilitation effects of Schwann Cell transplantation. Endurance Balance and Conditioning SOF Brain Computer/Interface Red Light Camera Bill We are in the final stages of receiving FDA approval for our Brain/Computer interface State of Florida funding continues to be work targeting spinal cord injured patients. another source of support for our brain and This investigation has resulted from an spinal cord injury studies. We will receive alliance with Medtronic, Inc. which will supply money from the SOF and the Red Light state-of-the-art integrators that will allow Camera Bill. neuronal impulses from the brain to be transmitted around the injured spinal cord to the muscles of the upper extremity. 3 UNC SCI Conference: November 7‐8, 2014 Discovery Science Programs State-of-Art Research Our discovery science programs are also very successful. We are publishing in high- • We continue to train many pre-doctoral and tier journals on neuroprotection, axonal postdoctoral fellows to conduct state-of-the- regeneration and repair. This year, The art research. Miami Project published over 135 peer • Our faculty organizes and teaches in many reviewed articles in many of our leading undergraduate, graduate and other medical journals. school courses. Neurosurgery and New Rehab Center Sylvester Cancer Center Neurosurgery and the Sylvester Cancer The Chrystine E. Lynn Center have initiated a new Brain Rehabilitation Center Tumor Laboratory in the Lois Pope LIFE Center. This brain tumor laboratory will for utilize state-of-the-art cell culture and The Miami Project to Cure Paralysis. screening approaches to develop specialized therapeutic interventions targeting brain tumors. The Miami Project Faculty 2014 Coleen Atkins Allan D.O.Levi Radiology John R. Bethea Daniel Liebl Neurology John Bixby Alberto Martinez Pathology Murray Blackmore Paula Monje Diabetes Research Institute Nancy L. Brackett Mark S. Nash Interdisciplinary Stem Cell Helen M. Bramlett Brian R. Noga Institute Roberta Brambilla Kevin Park Miami Project Cores Mary Bartlett Bunge Damien Pearse Alex Marcillo M. Ross Bullock Jacqueline Sagen Beata Frydel Pablo de Rivera Vaccari Robert Keane Edelle Field-Fote Christine K. Thomas Supported by NIH, DOD, State of Barth A. Green Pantelis Tsoulfas Florida, Craig Neilsen Foundation, Jim Guest Michael Wang Miami Project to Cure Paralysis and the Buoniconti Fund Jae Lee Eva Widerstrom-Noga Vance Lemmon Patrick M. Wood 4 UNC SCI Conference: November 7‐8, 2014 Thank You 5.