A groundbreaking Australian clinical trial has halted the progression of multiple sclerosis in early-stage patients by using chemotherapy and stem cell transplants, with some of the patients showing marked physical improvement and even abandoning their walking sticks.
Published in the prestigious Journal of Neurology, Neurosurgery and Psychiatry, the results of the trial offer hope to many of the 25,000 Australians now living with the incurable and degenerative disease.
“What is very clear is that for those patients with active, inflammatory disease is that following treatment there is a sustained and almost complete absence of inflammation,” said one of the lead researchers, neurologist Dr Ian Sutton.
“This correlates with a marked reduction in clinical relapses of disease.”
Underway at St Vincent’s hospital in Sydney, the phase two trial has been running since 2011, and so far 48 patients have been treated, and 35 included in the published research paper.
“When you reduce inflammation, you also allow the brain and spinal cord a chance to recover, which can allow an improvement in disability,” Dr Sutton said. “If the procedure is undertaken early in the disease course when inflammation is most pronounced, this increases the chance of an improvement in disability”.
Stem cell treatment
Blood is first taken from the patients to harvest their stem cells, then the patients are admitted to hospital for intensive chemotherapy to effectively kill their immune systems.
Each patient’s harvested stem cells are then returned to him or her, via a drip, and the immune system effectively reboots, without the burden of multiple sclerosis.
Existing multiple sclerosis lesions remain on the brain and spinal cord, but inflammation is halted so in most cases, no new lesions are formed.
The chief researcher, haematologist Conjoint Associate Professor John Moore said some of the patients on the trial showed physical improvement, as well as a halt in the progression of the disease. “We have had patients who used sticks for walking, and now no longer need to,” he said.
The trial has tracked all patients over time, some over many years, and so far the progression of multiple sclerosis has remained in check for nearly all of them.
“Given these patients stay off MS medications after their stem cell transplants, we’re doing something to change the immune system that was previously attacking the central nervous system,” Professor Moore said, adding “we’re not seeing new lesions; the vast majority of patients remain lesion-free”.
The trial was successful for early-stage MS patients, with the relapsing-remitting type of the disease, when inflammation in the brain and spinal cord is active. It worked less well for patients who have had MS for longer and moved onto the progressive stage of the disease.
These chemotherapy treatments have been available to paying patients in Russia and Mexico for some time, and trials have been running in the US, Europe and here in Australia. An earlier trial in Western Australia was less successful because it focused on patients with secondary progressive MS.
“Our objective is to stop patients having relapses and deteriorating function, but what we did notice in virtually only relapsing remitting patients is about half of them get an improvement in their disability”, Professor Moore said, adding the improvement did not happen for the patients with secondary progressive MS.