Developing an olfactory cell therapy for spinal cord injury

Phase I/IIa clinical trial and NEXT StePs

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THE SPINAL INJURY PROJECT IS Progressing THE PRE-CLINICAL RESEARCH with the aim of commencing a PHASE I/IIA HUMAN CLINICAL TRIAL.

 Since Griffith University’s Phase I clinical trial in 2002, the work has continued and expanded with a new generation of scientists. With a scientific team led by Associate Professor James St John and Associate Professor Jenny Ekberg—and involving more than

40 scientists and clinicians across a range of fields and universities including international collaborators —the time has arrived to take the next giant leap in the development of this therapy. What is now needed is to make the therapy more effective and generate

higher consistency in results. 


The aim is to conduct a Phase I/IIa clinical trial of 25 patients with chronic spinal cord injury.

First, patients will conduct a sustained functional activity-based therapy to prime their spinal nerves, brain and muscles for the cell transplantation.


Then a 3D nerve bridge made of olfactory cells will be implanted into patients with chronic spinal cord injury, without the need for invasive surgery. 


The 3D nerve bridge maximises the efficacy of the olfactory cells, reduce the existing scar tissue, and promotes the regeneration of the nerve cells. 


This methodology ensures that patients receive optimal restoration of motor and sensory function and that outcomes are as consistent as possible.


Intensive and sustained functional therapy will be performed starting immediately after surgery for at least 12 months to maximise the regeneration of motor and sensory neural connections. Functional recovery is expected to be detected in patients within several months after the transplantation. 


The clinical trial is anticipated to start in south-east Queensland in late 2020. The complete Phase I/IIa clinical trial is expected to cost $20 m over six years.

Participants

 To obtain more information about our partners click on the red box below. For example, you can find out more about the progress of our research and the progress of other research into spinal cord injury around the world by clicking the link to the Perry Cross Spinal Research Foundation

Phase I/IIa Clinical Trial key outcomes expected

Whilst it is not possible to provide guaranteed outcomes in any clinical trial, the

outcomes this trial are aiming to achieve include:

• Showing that the therapy works—achieving an improvement in motor and sensory function in patients (without any detrimental effects)

• Understanding whether the therapy works across different types of injuries

(quadriplegic, paraplegic, minor/partial injuries, full injuries etc)

• Development of a 3D olfactory cell nerve bridge that improves regeneration

• Identifying the optimal surgical procedures for transplantation

• Identifying the optimal rehabilitation treatment

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2019 and beyond - the next steps

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In order to be fully prepared to start a clinical trial in 2020, Griffith University requires funding in addition to that provided by the Queensland State Government through the Motor Accident Insurance Commission. 

1. Sustained Functional Therapy

 As a key component of the therapy, the sustained functional therapy needs to meet the needs and abilities of patients. To ensure that, we need to conduct an extensive feasibility study for delivery of a sustained functional therapy rehabilitation program. We have to

ensure that participants can maintain the intensive level throughout the program. For this, we need to identify barriers to be addressed by conducting the sustained functional therapy with people who have different types of spinal cord injury. In this way, when the cell transplantation clinical trial commences, we can be confident that the rehabilitation program be can tailored to suit individual needs.

2. Clinical Trial Planning and Management

 A Contract Research Organisation is a professional body that drives the scoping and  approvals needed for a clinical trial and manages the independent assessment and reporting of outcomes of a clinical trial. We want to engage a CRO to ensure that the 2020 trial is scoped, planned and executed as professionally as possible. This will further the chance of success for the trial. 

3. Funding Applications for Clinical Trial

 Obtaining funding for clinical trials, e.g. from the Medical Research Futures Fund, is a competitive process. The best possible case has to be put forward. For this, we require an expert with experience in high level government submissions to write the respective business case.

4. Socio-Economic Case

To develop the business case for our clinical trial, an updated socio-economic analysis of spinal cord injury in Australia is required. The last analysis was reported in 2009 (by Access Economics) with an overall cost of $2 billion per annum to the Australian community. We anticipate that the cost to the community is now considerably higher.

These four programmes will enable the key Phase I/IIa clinical trial to commence.


Funding to proceed with the Phase I/IIa clinical trial is needed. All aspects of the trial need funding, including the production of the cells and nerve bridge, the transplantation procedure and medical care, on-going sustained functional therapy and the assessment and

reporting of outcomes. The full cost will be determined by the CRO and is initially estimated to be $20 million. The therapy will likely be improved upon over time, particularly as we

obtain early results from the clinical trial. Funding for on-going research beyond mid-2020 is needed to retain the key staff and research teams of the SIP team.