FDA Gives Green Light for Phase 1 Clinical Trial

Phase 1 Clinical Trial Represents New Hope for Treating Spinal Cord Injury

July 31, 2012 — The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, has received permission from the Food and Drug Administration (FDA) to begin a revolutionary Phase 1 clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with recent spinal cord injuries.

Found mainly in the peripheral nervous system, Schwann cells are essential to sending appropriate electrical signals through the nervous system, and Miami Project scientists and supporters believe they are key to finding cures for paralysis. In what will be the only FDA-approved cell therapy-based clinical trial for sub-acute spinal cord injury in the United States, investigators plan to transplant a patient’s own Schwann cells at the injury site in the hope of ascertaining safety that will allow further trials to proceed.

“We believe today’s announcement is just as important to our field as man’s first step on the moon was to the space program,” said neurosurgeon Barth Green, M.D., Co-Founder and Chairman of The Miami Project. “When we started The Miami Project, the short-term goal was to improve the quality of life of people living with paralysis, but the long-term goal remains re-establishing function and finding a cure. Today the scientists, clinicians, and technicians who have made this day possible have taken a giant leap forward. The hundreds of millions of dollars and incalculable hours of bench and clinical work invested in this goal have been well spent.”

“This is truly one small Schwann cell for a human, and one giant leap for humankind and the search for cures for paralysis,” said Pascal J. Goldschmidt, M.D., Senior Vice President for Medical Affairs and Dean of the Miller School of Medicine, and CEO of UHealth-University of Miami Health System. “The FDA decision validates the commitment of a family who has turned their own tragedy into hope, and the vision of scientists who have never wavered in their quest to reverse the catastrophic consequences of spinal cord injury.”

Nick Buoniconti, Co-Founder of The Miami Project, said, “It has been 26 years since my family made a promise to Marc and all those living with paralysis that nothing will stand in the way of us finding a cure, absolutely nothing. FDA approval of this clinical trial is allowing us to follow through on this incredibly important commitment that impacts millions of families each day. This validation of the tireless efforts being undertaken at The Miami Project offers real hope and shows we are closer than ever to curing paralysis.”

“Back in 1985, all we had was Dr. Green’s dream, my family’s determination, a lot of hope, and a ton of hard work ahead of us,” said Miami Project President Marc Buoniconti. “To be at this point today, receiving FDA authorization for this Schwann cell trial, is so rewarding to me, my family, and everyone who has ever stood by our side and supported this important work. This is another of the many monumental steps we’ve undertaken these past 26 years that will lead us to our ultimate goal of curing paralysis.”

Led by W. Dalton Dietrich, Ph.D., the Schwann cell clinical trial team at The Miami Project is composed of a multi-disciplinary group of basic science and clinical faculty members, scientific staff, and regulatory personnel focused on advancing the trial.

“Obtaining clearance from the FDA to initiate this important Phase I clinical trial is a vital step for the field of SCI research, and for the Miami Project team that has been working diligently on this therapeutic concept for more than a quarter of a century. This trial, when completed successfully, will lay the critical foundation for future cell-based therapies we plan to target SCI,” said Dr. Dietrich, Scientific Director of The Miami Project.

The clinical trial will enroll eight participants with an acute thoracic SCI. Newly injured patients brought to the trauma center would have to meet the stringent criteria and agree to participate in further screening within five days of their injury. At that point, the participant will undergo a biopsy of a sensory nerve in one leg to obtain his or her own Schwann cells. The Schwann cells will then be grown in a culturing facility for three to five weeks to generate the number of cells necessary for transplantation, and to undergo the strict purification process. By the time the Schwann cells are surgically transplanted into the injury site, participants will be 26-40 days post-injury.

All procedures will be conducted in Miami at University of Miami Hospital, Jackson Memorial Hospital, and The Miami Project to Cure Paralysis. Each participant will be followed intensely for one year after receiving the transplantation surgery, and their neurologic status, medical status, pain symptoms, and muscle spasticity will be evaluated. It is expected that it could be at least two to three years from the time the first subject is enrolled until the final subject is one year post-transplantation. All participants will continue to be monitored for years under a separate clinical protocol. This Phase I trial is the foundation upon which The Miami Project will develop future trials targeting different types of injuries, times post-injury, and therapeutic combinations.

The Miami Project has developed life-enhancing SCI programs used by hospitals and clinics across the nation and around the world. For example, male fertility for paraplegics and quadriplegics was a research effort and is now clinical practice; the monitoring of a patient’s spinal cord in the operating room was a concept, and is now an FDA-approved reality; the use of functional electrical stimulation in rehabilitation of paralyzed or partially paralyzed muscles is now FDA-approved and effective because of work by The Miami Project; the use of computerized walking systems is now FDA-approved; and the use of cooling to protect the nervous system is now an approved and commonly used practice in cardiology and vascular surgery and is rapidly being advanced in the areas of brain and spinal cord injury due to the work of The Miami Project.

MEDIA CONTACTS: Scott Roy, 305-243-8939 or sroy@miami.edu and Dan Rene, Levick Strategic Communications, 202-973-1325 or drene@levick.com

SCIENCE/TRIAL CONTACT: Kim Anderson, Ph.D., 305-243-7108 or mpinfo@med.miami.edu