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 realised 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 specialised cells within the olfactory (sense of smell) 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 randomised control study which will test the olfactory nerve bridge transplantation combined with long-term intensive rehabilitation.

Cells for the transplantation will be harvested from the patient’s own nose, purified, and prepared into specialised nerve bridges which are then transplanted into the injury site within the spinal cord.

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.”

Nerve bridges made from olfactory ensheathing cells which replicate a nerve obtained from a chicken
Perry Cross Spinal Research Foundation (PCSRF) Executive President Perry Cross said: “30 years ago, I injured my spinal cord, and I am paralysed 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, which 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 Centre 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 Centre 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 funded by the Perry Cross Spinal Research Foundation which 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.

CEO of the Clem Jones Foundation, Peter Johnstone, said the latest phase of the project illustrated the benefits of philanthropic support for long-term research.

“The Clem Jones Foundation has been pleased to be involved with others from the philanthropic sector in funding this world-class research,” Mr Johnstone said.

“It is proof of the progress that can be made through long-term funding commitments for highly specialised work with the potential for changing lives around the world.”

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.”

“To be able to develop the therapy in Queensland is thanks to the incredible support from our funding partners, in particular the Motor Accident Insurance Commission (MAIC) as the major funder, along with the Perry Cross Spinal Research Foundation, the Clem Jones Foundation, NHMRC, MRFF, and the dedicated spinal injury community which has been the inspiration and driving force behind the therapy development,” Professor St John said.

The trial is funded by the Medical Research Future Fund, Perry Cross Spinal Research Foundation, The Clem Jones Foundation, Queensland Government, Nicola and Andrew Forrest, Brazil Family Foundation, Terry and Rhonda White, and Griffith University.

If you are interested in the trial, more information can be sought by emailing scitrial@griffith.edu.au

An experimental and controversial stem cell transplant, which aims to treat traumatic spinal cord injury, has shown promising new results in a phase I clinical trial.

Not only was the single-dose spinal injection safe and well-tolerated by all 10 patients with paralysis, it also resulted in some improved movement and sensation for 7 volunteers.

While that’s seriously hopeful, the type and degree of injury seems to matter. Researchers at the Mayo Clinic in the US admit that in all likelihood, only about five percent of patients with a complete injury to their thoracic spine would regain any feeling or movement with this therapy, based on their results.

Nevertheless, neurosurgeon Mohamad Bydon says: “In spinal cord injury, even a mild improvement can make a significant difference in that patient’s quality of life.”

Stem cell transplants are cutting-edge treatments that have recently emerged as a possible way to treat spinal cord injuries.

First, a patient’s own stem cells are taken from their abdomen or thigh and multiplied in the lab before being injected back into the patient’s spine. These cells, called mesenchymal stem cells, hold the potential to rebuild bone, cartilage, muscle, fat, or even aspects of the human central nervous system.

For patients with a traumatic spinal cord injury, who have few other treatment options, the possibility that these injections can help the healing process is exciting. But that hope needs to be tempered by the available science.

While other small clinical trials on the experimental therapy have produced results that also suggest it is safe and effective for spinal cord injury, some scientists argue that these trials are not rigorous or long enough to fully assess either of those outcomes just yet.

In 2019, Japan made the controversial decision to approve mesenchymal stem cell therapy for commercial use in treating spinal cord injury. Many scientists continue to criticize that decision as premature. Even those directly involved in the research, like those at the Mayo Clinic, have called for larger and more rigorous trials.

Phase I clinical trials are not designed to specifically test how well a treatment works; they are mostly concerned with safety.

However, several patients in the current clinical trial demonstrated improved sensory and motor performance when tested roughly a year and 10 months after receiving the lumbar injection.

Nobody got worse as a result of the treatment, although there were some minor side effects, such as headache and musculoskeletal pain. This bodes well for phase II studies.

Still, there’s reason to wait for further evidence before getting too excited. The current trial, like many previous trials, focused mostly on patients who had suffered a spinal injury in the past year or so, usually from vehicle accidents or falls.

People’s movement and sensation can improve slightly in the first year or so after a spinal injury. That means when looking at the results, it’s impossible to tease apart the healing effect of time and the healing effect of the stem cell therapy specifically.

What’s more, patients in the trial were placed under the care of a highly specialized multidisciplinary team to help them rehabilitate, and this could very well have impacted their health outcomes over the course of the study.

That said, one participant had a cervical spine injury that had occurred 22 months before the stem cell injection, which is long after general improvement naturally stops. This patient also showed improved movement and sensation following the stem cell transplant.

“As a result,” the researchers at the Mayo Clinic conclude, “there is a need for future, larger randomized controlled trials employing various clinimetric tools and quality of life measures, to investigate the potential benefits of [these] injections in late-stage neurological recovery for spinal cord injury.”

The Mayo Clinic’s phase II trial is now underway. It aims to assess physical therapy outcomes after stem cell transplants, as well as safety.