Australian MS trials highly promising, despite drawbacks

Younger patients with inflammatory relapsing-remitting multiple sclerosis benefit most from the therapy.

Nerve cells. ‘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,’ haematologist John Moore says.

The Australian medical breakthrough that has led to some multiple sclerosis patients discarding their walking sticks began with animal studies in the 1960s and 70s.

First, mice with a form of MS were successfully treated with a variant of the treatment; then doctors began noticing that MS patients treated with chemotherapy for cancer were coincidentally reporting a slowing of their auto-immune disease as well.

More recently, clinical trials using the procedure to treat MS patients have had mixed success. Treatment of patients with advanced MS, known as the secondary progressive phase of the disease, has not been as successful as that of young, early stage MS patients with the inflammatory relapsing-remitting type of the disease.

The St Vincent’s trial was the first successful Australian trial using chemotherapy and blood stem-cell transplants to treat mainly young, early stage MS patients. The trial’s lead scientist, haematologist John Moore, describes it as “really promising”.

“We’ve been able to show that the immune system in MS patients corrects, and the cells and immune system seem to be more anti-inflammatory than inflammatory after the stem-cell transplants,” Moore says.

“So we’re quite excited about those findings because that’s the first time anyone has ever shown that.”

The trial patients — 48 so far — have been examined regularly for years after the treatment, and 85 per cent to 90 per cent have “done really well”, Moore says, without drugs and with no MS progression.

Technically known as autologous haematopoietic stem-cell transplant, the treatment also has been tested in a large and yet unpublished US trial, which Moore says has also had “extremely positive” results. Two further randomised international trials are in the planning stages and due to start soon.

“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,” Moore says. “Our objective is to stop patients having relapses and deteriorating function, but what we did notice in virtually only relapsing-remitting patients is that about half of them get an improvement in their disability.”

Unfortunately the treatment does not have the same effect for patients with the secondary progressive type of MS. “These findings are important, but we’ve got to caution also that we need more phase three (randomised controlled trial) data,” Moore says. “And, second, this is about chemotherapy, so there are obvious toxicities. Patients lose their hair. They have low blood counts, they put themselves at risk of infection and they have to spend three to four weeks in an isolation ward in hospital.”

Women of child-bearing age, he adds, usually become infertile after the treatment.

About 20 per cent of patients in the trial had organisms growing in their blood cultures or urine after the treatment. Ulcers in the throat or bowel and persistent diarrhoea were problems dealt with during the recovery period, along with shingles and urinary infections, although patients began to recover as soon as their new immune systems kicked into gear.

The treatment is also cost-effective, Moore says. One cost-benefit analysis found the cost to the community of looking after one patient with MS was about $48,000 a year, compared with a one-off cost of about $60,000 for the AHSCT treatment.

Sian Powell