The Miami Project to Cure Paralysis at the University of Miami Miller School of Medicine, announced today that the FDA has approved what the Project calls a “revolutionary” clinical trial to evaluate the safety of transplanting human Schwann cells to treat patients with recent spinal cord injuries.
Note: There is lots of excitement about this trial. It is important work and must be done to set the stage for actual treatments. Keep in mind it is a safety trial for newly injured people. It may lead to treatments for chronic SCI, but that’s not what this news is about.
The announcement came at a morning press conference broadcast over Internet, led by University President Donna Shalala.
“This is a big day,” said Marc Buoniconti, whose 1985 spinal cord injury sparked the creation of the Miami Project. “Twenty-six years ago my father made me a promise that there would be a cure for paralysis. Today we are taking a huge step to make that happen.”
This, the only approved cell therapy trial for spinal cord injury in the U.S., is indeed a milestone for the team at Miami, and for the SCI field. Said neurosurgeon and Project co-founder Barth Green: “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.”
The space shot analogy continues, this from Pascal J. Goldschmidt, dean of the med school: "This is truly one small Schwann cell for a human, and one giant leap for humankind and the search for cures for paralysis," said. “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.”
Attending the press conference was the science team, including MP research director Dalton Deitrich and the research team that did all the tedious preclinical work. Special recognition is much deserved by senior scientist Mary Bunge, whose late husband Richard came up with the idea that Schwann cells might be useful tools to treat spinal cord injury. That was in 1975. “It was his dream,” she said, “to move from Petri dishes to therapies that help people. So, kudos to Richard.”
The continuity is remarkable: Mary stayed with the research; two scientists on the Schwann cell team, Allan Levi and James Guest, were former students of Richard Bunge’s.
Noted Marc Buoniconti: “I know Richard Bunge is with us today, looking down at us here. It was his dream to see his research move toward benefiting people.”
This trial is looking only at safety. Eight patients will be enrolled, all in Miami, all completely injured paraplegics (T-3 to T-11). Participants will have to commit to the trial within five days of injury; at that time doctors will remove a small piece of nerve from each patient’s leg (sural nerve) to extract Schwann cells. These cells will then be cultivated and expanded. At one to two month post-injury, the cells will be injected into the epicenter of the injury, guided by intraoperative ultrasound. The syringe will be held by a fixed device.
When is it clear the process is safe? The Miami team won’t report trial data until one year after all eight patients have been treated. According to the group, that could take two and a half years.
What about Schwann cells for chronic SCI? Not yet, but they’re working on it, this from a prepared statement: “We are currently conducting more animal chronic transplantation preclinical studies and we will soon begin a combined exercise and locomotor rehabilitation study for chronically injured people to determine the minimum exercise and rehabilitation needed to prepare people for transplantation surgery and to ensure that they are neurologically stable. We will take all of that data and design another clinical trial specifically for people with a chronic injury. We will submit that in addition to safety data generated by the acute trial to the FDA for approval.”
The Schwann cell studies at the Miami Project were funded, in part, by the Reeve Foundation. Richard Bunge was the principal investigator for Miami's membership in the Reeve International Research Consortium for Spinal Cord Injury. Upon his death in 1996, Mary became PI. Damian Pearse got his start in science as an Associate member of the Consortium, under Mary Bunge.
While the Miami Project is self-contained as part of the university medical school, there are many connections to the SCI research community, including the Reeve Foundation. James Guest is the PI for the Reeve Foundation North American Clinical Trials Network affiliate at Miami. Robert Grossman, neurosurgery chairman at Methodist Hospital in Houston, PI of the NACTN program and member of the Reeve Consortium Advisory Panel, is on the Miami Project's Data Safety and Monitoring Board.
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