SCIENTIFIC UPDATE FOR THE MIAMI PROJECT
Recent Program Accomplishments
This has been another outstanding year for the Miami Project to Cure Paralysis. Our multidisciplinary team continues to make significant discoveries in translational and clinical investigations. Ongoing programs and recent progress in brain and spinal cord injury research are briefly summarized in this scientific update.
- First completed FDA approved Phase I cell therapy trial for subacute SCI subjects transplanted with human Schwann cells. We reported no significant risk factors along with evidence for conversion of one subject from complete to incomplete status at 1 year.
- Based on encouraging results, FDA permission was obtained to move into the chronic phase for testing the safety and benefits of Schwann cell transplantation combined with an extensive exercise / rehabilitation protocol. Subjects with complete or incomplete thoracic injuries were successfully transplanted with no risk factors reported. Importantly, small sensory or motor improvements have been demonstrated in some transplanted subjects which is very encouraging.
- Based on these exciting results, the FDA approved moving forward in the same trial with our transplantation studies targeting complete and incomplete cervical SCI Several individuals have been enrolled and are successfully undergoing extensive locomotor training including functional electrical stimulation. Transplanted complete cervical subjects have demonstrated no complications and additional subjects are being scheduled for transplantation this year.
- The Miami Boot Camp, a highly successful structured multi-modal exercise program developed to improve multiple body systems and a significant component of our chronic SCI cell therapy program has been published so other programs can benefit from our experiences.
- In another FDA approved trial, a novel peripheral nerve bridging strategy with Schwann cells seeding sural nerve autografts in individuals with severe leg trauma was tested. Treated individuals are experiencing increased sensation and movements in their legs and walking after 1 year.
- We completed the recruitment for a multi-center human stem cell transplantation trial for chronic SCI sponsored by StemCells Inc. At our University of Miami Miller School of Medicine site, individuals with cervical injuries were transplanted with neural stem cells with encouraging results published in a scientific Journal.
- Persons living with SCI-induced neuropathic pain, an experimental Deep Brain Stimulation approach was tested to target this quality of life issue. Some subjects are experiencing reduced pain sensation below the injury level due to the stimulation protocol.
- New Department of Defense funding is supporting a multicenter randomized trial to test the benefits of therapeutic hypothermia in complete and incomplete SCI patients. This trial is based on encouraging UM data showing a significant percentage of treated SCI subjects showing evidence of conversion from complete to incomplete paralysis one year after injury.
- In the area of severe TBI, patients are being enrolled in a multi-center trial for therapeutic hypothermia treatment that require early decompression surgery (HOPES Trial). Patient recruitment is ongoing at Miami, Pittsburgh, Houston, and sites in Japan and China.
- To treat male fertility problems after SCI, a new treatment strategy using oral administration of probenecid has been reported to promote increased sperm motility.
- With Medtronics, a new Brain/Computer Interface strategy to improve upper limb function in high cervical injured subjects is being developed with future testing planned.
- In the emerging field of neuromodulation, transcranial magnetic stimulation is being tested to stimulate the corticospinal tract in SCI subjects to enhance circuit plasticity. Future SCI clinical studies will combine similar neural stimulation strategies with rehabilitation and cell transplantation to enhance function.
- We continue to conduct prevention research funded by the State of Florida for Road Accident Prevention Safety to educate children and adults in road crossing safety that has been shown to reduce injuries due to road accidents.
- A powerful 3D imaging approach for the visualization of nervous system circuits has been developed and is allowing scientists to evaluate therapeutic interventions to promote successful axonal regeneration.
- The importance of body temperature on the behavioral consequences of experimental concussion has been reported for the first time that may have important implications for why some concussed patients experience prolonged symptoms.
- A new molecular sequencing technique to study gene expression and gene therapies is clarifying the fundamental biologics behind failure for CNS regeneration that could provide new strategies for promoting axonal regeneration after brain and spinal or injury.
- The discovery of a significant fibroblast component of the glial scar surrounding a spinal cord lesion that inhibits axonal regeneration has been published which provides a novel cellular target to enhance repair.
- Tumor necrosis factor (TNF) soluble receptor type has been discovered to be a new therapeutic target for MS.
- Our Educational/Outreach programs total over 3500 people on our research volunteer database targeting paralysis and our Annual Miami Project Open house is being scheduled for 2018.
- The new Christine E. Lynn Rehabilitation Hospital currently under construction will house cutting edge Miami Project and University of Miami rehabilitation approaches for people with brain and spinal cord injury.
- Miami Project faculty are training over 75 postdoctoral, graduate and undergraduate students in the areas of discovery, translational and clinical neuroscience.
- Faculty published over 100 peer reviewed manuscripts and chapters this past year and presented their latest findings at national and international conferences. These types of academic activities are important for sharing new knowledge with scientific colleagues and to promote collaborations with other research groups.
- The total amount of extramural federal funding by the faculty was the highest in the history of The Project.
These are truly exciting times in the Miami Project and we thank our colleagues for their continued interest and support for our scientific and clinical programs. In the following pages we list recently published peer-reviewed scientific articles by our dedicated faculty and trainees.
Dalton Dietrich, PhD
Scientific Director, The Miami Project to Cure Paralysis
Senior Associate Dean for Discovery Science
Professor of Neurological Surgery, Neurology, Biomedical Engineering and Cell Biology