Clinical trial finds new immunotherapy improves MS symptoms

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 most 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, 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.

Prof. 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 those patients showed improvements. Without this treatment, their symptoms would have continued to get worse,” Prof. 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.”

Prof. 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 for us to develop similar cellular immunotherapies for certain other autoimmune conditions like rheumatoid arthritis.

“From this phase I trial, we have also discovered exactly what produces 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 September 2014. A phase II trial is due to begin in 2019 at 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 brains lesions of MS patients.

The results of the clinical trial have been published in JCI Insight.