By Leah MacLennan
Hundreds of Australians annually are heading overseas in search of treatments not available locally for multiple sclerosis (MS), despite knowing there are risks and high costs.
Among them is South Australian woman Sarah Tilbury, who is about to head to the United States for a treatment not easily accessible in Australia.
When she was in hospital this year after giving birth to her son Oliver, the 26-year-old woman was diagnosed.
In the months before the birth she had started having falls.
“Not just like someone who’s a bit clumsy, my whole right side would get weak and I’d, you know, without warning be on the ground,” she said.
Ms Tilbury said that, as an otherwise healthy young woman, she was told she was unlikely to experience a rapid onset of symptoms but she was still having problems.
“I ended up really not being able to walk without assistance. Tom [my husband] would pretty much have to hold me up and I was dragging my leg a lot of the time,” she said.
Her health problems meant she could no longer manage in her two-storey house in the state’s south-east, so the Tilburys started looking for other treatment options.
‘Confronting’ TV story led to treatment search
Ms Tilbury saw a TV show about Kristy Cruise, a woman with MS who had travelled to Russia for a treatment called an autologous haematopoietic stem cell transplant, or HSCT.
“I found it incredibly confronting to watch,” she said.
“It was just like me. Like me on a bad day was what they were showing and that is incredibly confronting because I think still I was in a bit of denial.”
Like Ms Tilbury, Kristy Cruise was a young mother living with multiple sclerosis.
After seeing the TV report, the SA mother got in touch with Ms Cruise and started researching HSCT.
Before her treatment overseas, Kristy Cruise was fatigued and needed a walker or wheelchair for mobility.
Now, more than a year after the treatment, she no longer feels like she has MS.
“Physically I’m back to how I was 10 years ago. I feel great. I have lots of energy,” Ms Cruise said.
“I can make plans for five, 10 years from now with my children.
“I don’t nap any more. I’m not on any medications and I feel really fit and healthy.”
Overseas treatment very costly
Without HSCT treatment options in Australia, Sarah Tilbury decided to try Chicago where Dr Richard Burt pioneered treatment for MS and other autoimmune diseases.
But treatment in the United States is expensive and Ms Tilbury’s medical bill alone will be $140,000.
There also will be the emotional cost as Sarah and Tom Tilbury leave young Oliver at home with his grandparents for two months.
But for the Tilburys, the sacrifice will be worth it if Sarah can again be a healthy and able-bodied woman.
“Our whole life has been thrown upside down by this disease and quality of life is huge, so if I come back and I’ve had my disease halted, which it [this treatment] has been proven to do, I’ll be ecstatic,” Ms Tilbury said.
“I do have certain goals ahead of me and one of them is walking down the beach with Oliver, when he can start walking. That’s a big one for me.”
In Australia, Colin Andrews is a Canberra-based neurologist who has performed HSCT on MS patients in the past.
The procedure involves harvesting stem cells from the patients and giving them doses of chemotherapy, then replacing the stem cells in the patient’s body.
“MS is thought to be largely an autoimmune disease, mainly involving T-cells so what we do is we harvest cells and treat them and then knock out the marrow and knock out the T-cells in the brain, which are producing the disease, and then the patient reboots their immune system with the implanted cells,” he explained.
“It’s as though they get a whole new immune system without the offending T-cells.”
HSCT has a 1 per cent mortality rate, and Dr Andrews no longer provides the treatment because the hospital in Canberra deemed it high risk.
In Australia, the only such treatment is via a small clinical trial being done at St Vincent’s Hospital in Sydney.
Professor Trevor Kilpatrick is a neurologist who is one of Australia’s leading experts in treatment of MS.
He said HSCT appeared to be effective in certain circumstances, but there was not yet definitive evidence.
“Haematopoietic stem cell transplant has a defined mortality, unfortunately between 1-2 per cent of people, that is one to two out of every 100 people who are to take this intervention will unfortunately die,” he said.
“That is a very, very important message to give to people that there are significant complications to be considered in relation to this intervention.”
Treatment dangers weighed against hopes
Ms Cruise said she weighed up the dangers of her own treatment against her hopes.
“You’re doing it because you’re fighting for your life and you want to have quality of life free of MS,” she said.
Since appearing on television, she has been inundated with people wanting to know more and now travels around Australia addressing forums for people with MS and their families.
“I feel for the people with MS who haven’t heard of this treatment so what I’m motivated to do is just to let people know that this is an option for them, so they can make their own informed decision,” she said.
Professor Kilpatrick said progress with MS treatment meant there now were other options people should consider ahead of HSCT.
He said Dr Burt in Chicago was assessing those treatments against the procedure he had pioneered.
“Richard has been doing this in a very systematised and standardised way such that the data which he has accumulated, looking at what’s happened after the transplantation, is probably the world’s best data at this point of time,” Professor Kilpatrick said.
“He’s instigated a clinical trial in which there will be randomisation for patients between standard therapy and haematopoietic stem cell therapy to understand who fares better.”
As a patient, Ms Tilbury said she was unwilling to wait for those trials to be completed while her MS worsened.
She said the risks of getting the US treatment paled compared with what was motivating her.
“Obviously my biggest concern is leaving Oliver,” she said.
“The treatment is nowhere near as scary as flying to the other side of the world without my son, so that would be my answer to that, and I think that really tells you why I’m doing it, doesn’t it?”