The Miami Project Submits Its First IND to the FDA

A Clinical Trial Involving Schwann Cell Transplantation Following Subacute Spinal Cord Injury

September 21, 2011 — The Miami Project to Cure Paralysis, at the University of Miami Miller School of Medicine has submitted today its first Investigational New Drug (IND) application to the Food and Drug Administration (FDA) requesting permission to begin a Phase I clinical trial to evaluate the safety of autologous human Schwann cell transplantation in the subacute spinal cord injury (SCI) setting.

The clinical protocol of the Phase I trial is strictly focused on safety outcomes. Therefore, the proposed clinical trial is designed to enroll 8 participants with complete thoracic SCI. Newly injured patients would have to meet the stringent inclusion/exclusion criteria and agree to participate in further screening within 5 days after their injury, which is considered the acute phase. At that point, the participant will have a biopsy of a sensory nerve in one leg to obtain his or her own Schwann cells. The Schwann cells will then need to grow in a culturing facility for 3 to 5 weeks to generate the number of cells necessary for transplantation, and to undergo the strict purification process. By the time the Schwann cells are transplanted into the site of spinal cord injury, the participant will be 26-40 days post-injury, which is considered the sub-acute phase.

Led by W. Dalton Dietrich, Ph.D., Scientific Director, The Miami Project to Cure Paralysis, the Schwann cell clinical trial team is comprised of a multi-disciplinary group of faculty level scientists, neurosurgeons, and staff focused on advancing the trial.

“This submission to the FDA is a vital step for the field of SCI research, and for The Miami Project team that has been working diligently for more than a quarter of a century to get us to this point. This trial, when approved and completed, will lay the critical foundation for future cellular therapies we plan to target SCI,” said Dr. Dietrich.

All procedures will be conducted in Miami at the University of Miami Hospital, and at Jackson Memorial Hospital. Participants will be followed for 1 year after the transplantation surgery, and their neurologic status, medical status, pain symptoms, and muscle spasticity will be evaluated. They will continue to be monitored for multiple years under a separate clinical protocol.

Because the Schwann cells, which are found in the peripheral nerves, are obtained from a nerve biopsy from the participant, there is reduced fear of rejection and no need for immunosuppressant medication. Once the cells are removed from the participant, they have to be handled in accordance to Good Manufacturing Practices (GMP). Because the cells will eventually be injected into the spinal cord, this process is required to ensure that the cells and every single thing that touches them are not contaminated with anything that could be dangerous after they are transplanted.

As is the case with all IND applications, once the FDA receives the package they have thirty days to respond. If after thirty days we receive no response from the FDA, the clinical trial will proceed.

MEDIA CONTACT: Scott Roy, 305-243-8939 or email, sroy@miami.edu

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