A world-first clinical trial of a new cellular immunotherapy for multiple sclerosis (MS) has found that it improved symptoms and quality of life for the majority of patients.
The treatment targets the Epstein-Barr virus (EBV). It is based on the theory of Professor Michael Pender, from The University of Queensland and the Royal Brisbane and Women’s Hospital (RBWH), that MS is caused by an accumulation of EBV-infected cells in the brain and that a therapy targeting EBV can potentially stop the progression of MS.
The new cellular immunotherapy was developed by Professor Rajiv Khanna and his team at QIMR Berghofer Medical Research Institute. The phase I clinical trial was conducted in collaboration with Professor Michael Pender and his colleagues. The trial was funded by MS Queensland, MS Research Australia, Perpetual Trustee Company Ltd and donations from private individuals.
Professor Pender said a total of 10 patients – five with secondary progressive MS and five with primary progressive MS – received four doses of the cellular immunotherapy treatment at the RBWH.
“Seven of these patients showed improvements. Without this treatment, we would have expected their symptoms to continue to get worse,” Professor Pender said.
“Improvements ranged from reduced fatigue and improved productivity and quality of life to improvements in vision and mobility. Importantly, we found the treatment was safe and without serious side effects.
“Our findings add to the mounting evidence that EBV infection plays a role in the development of MS.”
Professor Khanna said it was the first time in the world a T cell immunotherapy had been used to treat any autoimmune disease.
“We have already used these cellular immunotherapies to treat different types of cancer and viral infections. This clinical trial is a breakthrough because for the first time we have found these treatments are safe and have had positive improvements in an autoimmune disease,” Professor Khanna said.
“This trial opens the door to develop similar cellular immunotherapies for certain other autoimmune conditions.
“From this phase I trial, we have also discovered what cell properties produce the best results for the patients. We can now apply this knowledge to cellular immunotherapies for other diseases to try to ensure the best results for all patients.”
The phase I clinical trial started in November 2015. A phase II trial sponsored by Atara Biotherapeutics is planned for several locations in Australia and the United States.
Multiple Sclerosis, which is a condition of the central nervous system, is estimated to affect more than 25 000 Australians. It can cause a range of symptoms including problems with coordination, balance, weakness, arm and leg functioning, cognitive problems and memory loss.
While the majority of people are diagnosed with a relapsing remitting form of the disease, some can go on to develop a secondary progressive form in which disability gradually worsens. A small proportion will be diagnosed with a primary progressive form of the disease from the outset.
While there are a range of treatments available to prevent attacks in relapsing remitting MS, there are currently only very limited treatment options for people with progressive forms of MS.
The cellular immunotherapy works by taking blood from patients, extracting their T (immune) cells, and “training” them in the laboratory to recognise and destroy the EBV present in the brain lesions of MS patients.
MS Research Australia’s CEO Dr Matthew Miles said the organisation was delighted to have supported Professor Pender’s fundamental research into the role that EBV plays in MS for nearly a decade.
“It is now very rewarding to have been able to support this innovative trial,” Dr Miles said.
“We hope that with further research, we may see this work translated into a potentially effective treatment for people with progressive MS.”
The Chair of the MS Queensland board, Roger Burrell, said he was extremely grateful for the philanthropic support of this trial.
“We are beyond grateful to our wonderful Queensland philanthropists and grant-makers who made this clinical trial a reality,” he said.
“This trial shows the very important role that Australia is playing in EBV research worldwide. We are excited for what the future holds for our MS community and look forward to the phase II study.”
The cellular immunotherapies were manufactured at QIMR Berghofer’s Q-Gen Cell Therapeutics, one of the largest cell therapy manufacturing facilities in Australia.
The results of the clinical trial have been published in JCI Insight.