World-first clinical trial for treating spinal cord injury

Since the passing of Professor Emeritus Alan Mackay-Sim AM in 2023, his ground-breaking legacy research is about to be realized with a Phase I human clinical trial commencing to test the efficacy and safety of the transplantation of olfactory cell nerve bridges to treat chronic spinal cord injury.

Griffith University researcher, Professor James St John, said olfactory ensheathing cells are the specialized cells within the olfactory nerve within the nose that have numerous therapeutic properties for repairing and regenerating nerves.

"With more than 20,000 Australians living with spinal cord injury, and another 300 or more people having spinal cord injury each year, now is the time to translate this therapy into the clinic," Professor St John said.

"We have designed the trial in close consultation with the spinal injury community, clinicians and industry partners to ensure the trial meets the needs and expectations of the trial participants, and has the best chance of success."

The trial is a blinded and randomized control study which will test the olfactory nerve bridge transplantation combined with long-term intensive rehabilitation.

To encourage and reinforce regeneration, participants will undergo intensive rehabilitation for up to one year.

"These cells have been previously tested for treating spinal cord injury in a human clinical trial in 2002 in Queensland by the late Professor Emeritus Mackay-Sim, the 2017 Australian of the Year," Professor St John said.

"Since then, other trials around the world have also tested the cells but while there were some encouraging results, technical difficulties in preparing and transplanting the cells have been limiting factors."

Perry Cross Spinal Research Foundation (PCSRF) Executive President Perry Cross said, "30 years ago, I injured my spinal cord, and I am paralyzed from the neck down, unable to move, unable to feel, unable to breathe.

"It is incredible that we are now on the cusp of developing a treatment that may allow us to repair the damage to the spinal cord and regain function," Mr. Cross said.

"It would be life changing for any of the participants in this trial to regain the ability to stand and hug a loved one again, to feel again.

"I am excited to see the trial commence and to see this brilliant research help people with a spinal cord injury.

"I am grateful to all of our generous donors who have supported this work for many years."

The Spinal Injury Project team within the Clem Jones Center for Neurobiology and Stem Cell Research used a translational research strategy to create a new therapeutic approach to overcome the previous limitations.

The critical improvements include an enhanced cell purification procedure, an award-winning innovative cellular nerve bridge, and a dedicated long-term intensive rehabilitation program.

Professor St John, Head of Griffith's Clem Jones Center for Neurobiology and Stem Cell Research, said the high-purity cells and innovative nerve bridges are the critical components of the therapy.

"Despite decades of worldwide research to find a treatment for spinal cord injury, there is still no clinically available treatment," he said.

"Our innovative nerve bridges, combined with the high purity olfactory cells, offer what we think is the best hope for treating spinal cord injury.

"The preclinical research has clearly demonstrated the olfactory nerve bridges are effective in repairing spinal cord injury in animal models.

"We have also successfully tested the intensive rehab in two separate clinical trials that showed people with spinal cord injury enjoyed the program."

The trial is seeking volunteers who are living with chronic acquired spinal cord injury who have had their injury for at least four months, although the trial will start with people whose injuries are at least 12 months.

Participants will be from South East Queensland, northern New South Wales, Sydney or Melbourne and will undergo the rehab program at a rehabilitation partner in those areas.

Spinal Life Australia CEO Mark Townend welcomed the start of the trial and the potential life-changing impacts the research could have for people with acquired spinal cord injuries.

"We have been closely following the progress of this research for many years and it is exciting to see all the hard work come to fruition with this clinical trial," Mr. Townend said.

"I know many of our members will be interested in participating and we look forward to hearing about the outcomes, and what this will mean for the future of spinal cord injury treatment and rehabilitation."

Professor St John said, "To have a cell transplantation therapy progressing to clinical trial after only eight years is testament to the benefits of the strategic translational research program the team has used."

Provided by Griffith University