MS cure hope as drug reverses paralysis
MULTIPLE SCLEROSIS BREAKTHROUGH
AUSTRALIAN researchers have discovered a potential cure for multiple sclerosis that will progress to human trials after it allowed paralysed mice to walk again.
Monash University researchers have found the drug DITPA – approved by the US Food and Drug Administration for use in clinical trials to treat another genetic disorder called Allan-Herndon-Dudley Syndrome – can remyelinate, or rebuild, 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 needs $2 million to get the drug ready for human clinical trials.
MS affects about 2.5 million people worldwide and 25,000 in Australia. 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 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.”
Dr Petratos has since modified DITPA so that it can be effective at just one third of the dose used in the US Defence Department experiments on people with heart failure. Many treatments that work in mice fail when tried in humans so more testing is needed before it can enter a clinical trial.
“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.
Adelaide para-equestrian Elizabeth Griffiths, 28, who was diagnosed with MS just after she turned 16, said she had used various medications which helped for a while and later stopped working.
“Hearing that there’s a potential cure, it would just be life-changing for me. It’s just so exciting,” Mrs Griffiths said.
“For the people that haven’t had the opportunity to feel better or have a drug that’s worked for them, this could be a miracle for them.”
Hearing that there’s a potential cure, it would just be life-changing for me. It’s just so exciting