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 'Radical' stem cell trial offers hope for MSers

Jason McIntyre's autoimmune system is dead. The rest of him isn't feeling much better. Eleven days ago he underwent an aggressive chemotherapy, not for the sake of killing cancer - but to knock out every skerrick of protection his body has against infection. Sitting in a freezer were 35 million stem cells that were shaken from Mr McIntyre's bone marrow by a combination of drugs. These were filtered from his blood about three weeks ago.
That process, he says, left him with aching bones. It was his birthday.

If he survives long enough - that is, if a piece of dust doesn't get in his eye and spark a fatal infection - the stem cells will this week be returned to his body, as building blocks for a brand new autoimmune system.

Mr McIntyre, 37, is only the sixth patient with multiple sclerosis to undergo this experimental therapy - known as an autologous haematopoietic stem cell transplant - in a small trial being conducted by St Vincent's Hospital in Sydney.

Thousands of stem cell transplants are performed worldwide to treat certain blood cancers in patients who have become resistant to regular therapies - but the numbers of MS sufferers treated with a stem cell transplant are in the hundreds.

It's a strategy reserved for people like Jason McIntyre whose form of MS is very aggressive and resistant to drug therapy.

About three years ago, the Melbourne truck driver arrived home with blurry vision. He told his wife, Kym, that he couldn't read the number plates on cars. Soon after, following a session at the gym, he was ''boiling hot and his vision went blurry again''.

Kym McIntyre says it all happened pretty quickly. He started dragging his left foot. He couldn't unscrew bottle tops and lost his co-ordination.

An eye doctor recognised the problem as multiple sclerosis, but Mr McIntyre was told by a specialist that he'd have to wait for another attack - another lesion on the brain to develop - for the diagnosis to be confirmed.

Meanwhile, the trucking company Mr McIntyre ran with his father, Peter, had to stay in business. Peter was meant to be retiring, but went back on the road. The McIntyres have two small children, and the medical bills aren't going away.

When the diagnosis was confirmed, Mr McIntyre underwent a series of injections to alleviate symptoms and slow the attacks. It didn't work. He was put on a more aggressive drug therapy. That didn't work either, and his body went into further decline. His specialist said he had run out of options.

''Jason's sister Stacey didn't cop that,'' says Ms McIntyre. ''She went online and found a transplant trial was happening in Canberra.'' However, that project collapsed because of lack of funding.

Then came word that the haematopoietic stem cell transplant team at St Vincent's Hospital in Sydney - where patients with blood cancers are routinely treated - were trialling the treatment with MS sufferers who weren't responding to traditional therapies.

Speaking from his hospital bed, Mr McIntyre said the chemotherapy was ''knocking me about a bit. Feeling tired. See how we go.''

His wife was upbeat - or perhaps driven is a better word. ''I'm sick of chasing the kids. I need him back in action.''

The children, Pyper, 6, and Ryder, 3, were at an apartment with Ms McIntyre's mother. They were anxious and rowdy. ''They just want everything to get back to normal.'' Stem cell transplants have a claimed 70 per cent success rate at halting some types of MS. However, researchers here and abroad can't get sufficient funding such that a randomised trial can be undertaken that would bring the treatment into the mainstream.

Lisa Melton, research development manager with MS Research Australia, said about 40 patients in Australia were known to have undergone the therapy, with mixed results. Most of the treatments were carried out at the discretion of treating doctors on compassionate grounds at different centres around the country.

''It's a dangerous procedure, highly aggressive, and carries considerable risks,'' says Dr Melton.

''There have been many attempts in Europe to mount good clinical studies to demonstrate definitively that the transplants are effective, but [the studies] weren't successful … each time they ran out of money,'' says Professor David Ma, the head of the St Vincent's haematopoietic stem cell transplant team, which is caring for Mr McIntyre.

The St Vincent's small-scale trial is part of an international effort to get enough runs on the board - and secure funding.

Multiple sclerosis, says Professor Ma, is a good fit for stem cell transplant therapy because it's an autoimmune disease.

The brain and spinal cord become inflamed with lesions - and these are then attacked and damaged by the autoimmune system. If the immune system can be effectively killed off, and replaced with a new system, then the brain has an opportunity to recover from the inflammation that caused the problem in the first place.

''It's a radical strategy, for sure,'' says Professor Ma. ''We're trying to get it past the experimental stage.''

Source: The Age National Copyright © 2013 Fairfax Media (28/10/13)


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