PhD student Madeleine White and Dr Anne La Flamme

MIS416 is a drug that is already showing its effectiveness against progressive multiple sclerosis in on-going clinical trials in New Zealand and Australia.

Multiple sclerosis (MS) is a genetically linked neurological disease of the central nervous system, including the brain, spinal cord and optic nerves. It is thought to be an autoimmune disorder, meaning the immune system incorrectly attacks healthy tissue, causing inflammation followed by scarring (sclerosis) as the inflammation heals. The inflammation usually causes damage to the insulating myelin sheath that covers nerve fibres (like the insulation on an electrical wire) but can also damage the nerve fibres (axons) themselves. Symptoms may be mild such as numbness in the limbs or severe such as paralysis or loss of vision. These problems may be permanent or may come and go. However, the disease often leads to progressively worsening disability for sufferers over months or even years. The disease affects more than 3000 New Zealanders.

MIS416 is a microparticulate, myeloid-directed, immune response modifier developed by New Zealand biotech company Innate Immunotherapeutics. In this latest study, the researchers used mouse models of MS to investigate how and why MIS416 was able to modify autoimmune responses and thus alter the progression of disease. “Our studies clearly indicate that even a single dose of MIS416 administered either on the day of immunization or after disease onset is effective at reducing disease severity and furthermore, that this effect is augmented by regular administration,” write the researchers in their published paper.

In a press release from Victoria University, Dr Anne La Flamme, an Associate Professor in Victoria’s School of Biological Sciences and head of the MS Research Programme at the Malaghan Institute of Medical Research, said, “We know this drug works, but we are not sure why. This study has helped us understand the pathways that are driving the disease and how the medication alters the immune system, giving us a better idea of why MIS416 works as well as insight into how to treat patients and predict who will do better on this sort of medication,” says Dr La Flamme.

Most people believe MS revolves around T cells, says Dr La Flamme, but the study reveals that targeting other cells in the central nervous system can significantly reduce advanced forms of MS.

The Biotech Learning Hub first reported on the development of the drug MIS416 in August 2012 as the drug moved to its second clinical trial.

Get news story: NZ-developed MS drug moves to second clinical trial

In December 2012, TVNZ’s ONE News reported on the positive outcome for patients fortunate enough to be receiving the drug in trials. A patient interviewed reported improved hand-eye co-ordination and eyesight and an ability to stand for longer periods within hours of receiving the first of his weekly injections. He expressed nervousness about giving up the drug at the conclusion of the trial, as he didn’t want his earlier symptoms to return. Following the trial, several subjects did again deteriorate but were allowed to return to using the drug for compassionate reasons.

“We certainly aren’t taking the patients all the way back to being in perfect health,” says Innate Immunotherapeutics Chief Executive Simon Wilkinson in the ONE News interview, “but what we do seem to be doing is reversing disabilities that have been acquired over the preceding 6–12 months.”

The latest research into the drug was published in the 31 January issue of the international research journal PLoS ONE.