Finding creates hope for MS cure

AUSTRALIAN researchers have discovered a potential cure for the devastating disease multiple sclerosis by repurposing an old drug used for another rare genetic illness.

Monash University researchers have found the drug DITPA – approved by the US FDA for use in clinical trials to treat a rare disorder called Allan-Herndon-Dudley syndrome (AHDS) – can remyelinate cells in the central nervous system.

Demyelination, or stripping nerve cells of their protective sheath, is a hallmark of MS which causes loss of motor control, muscular spasms, incontinence, weakness, memory loss and problems with thought and planning.

When the drug was used on a mouse model of multiple sclerosis, it tricked the mouse cells into remyelination and paralysed mice were able to walk again.

The research team now needs $2 million to be able to get the drug ready for human clinical trials.

MS affects about 2.5 million people worldwide and more than 25,000 in Australia.

At the moment, the best therapies can only limit relapses in patients with the early stage of the disease and there is no cure.

The researcher behind the breakthrough – Dr Steve Petratos – has spent 25 years on MS research and became interested because his grandfather died with the disease.

“There is a significant genetic linkage with MS. I suppose I’m a scientist first and foremost, but you are human and there is an emotional aspect to it,” he said.

In MS, oligodendrocyte cells are mistakenly targeted by the immune system; they play an important role in protecting nerve fibres so stopping this damage is central to treating the condition.

When they tested DITPA in a culture of human cells in a

THIS IS A POTENTIAL GAME-CHANGER FOR MS PATIENTS IN THE FUTURE, AS THERE IS ONLY ONE SUCCESSFUL TRIAL TO DATE DR STEVE PETRATOS

dish, Dr Petratos’ team found it stopped oligodendrocyte cells from dying.

Even better, it prompted stem cells to mature into new, healthy oligodendrocyte cells.

“It was the regenerative therapy which MS patients have been crying out for,” he said.

“By giving mice the drug, we can make them walk again,” he said.

“This is a potential gamechanger for MS patients in the future, as there is only one successful trial to date which has reported effective outcomes of limiting MS progression,” Dr Petratos said.

But more testing is needed before clinical trials take place.