Unproven stem cell treatments provide expensive last resort in families’ search for hope – ABC News

Updated September 18, 2019 06:37:06

It is a quiet Monday morning, and as people are returning to desks and lining up for coffee, Doreen Deede is zig-zagging between bars, collecting cans and bottles emptied over the weekend.

Cashing in refundable containers, hosting charity events, even selling donuts at a local market Ms Deede tried all options as she inched towards a fundraising goal of $35,000 to fund overseas medical treatment.

"For the past nine years I've been Emma's sole carer," she said.

"I call myself her fundraising manager because that's what I've been doing."

Emma, aged nine, suffered a brain injury at birth and has been living with cerebral palsy and dystonia a condition where her muscles contract uncontrollably.

Ms Deede's social life all but vanished as her daughter grew up and significant problems with her mobility, eating, and speech began to register.

"That's when I decided that I wanted to be a mum that gives everything to my daughter," she said.

"The choice included that I live for her, that I do everything in my power to give her all the chances that she can get to one day live independently."

This week, the small family will take another of those chances when they travel from Cairns to the United States for a stem cell treatment to be administered to Emma via two lumbar punctures.

Even with Ms Deede's exhaustive fundraising regime the procedure is so expensive she has had to take out a bank loan and then there is the fact that the procedure has not been clinically proven.

"Everyone that is a parent will know that you do everything for your child," Ms Deede said.

"I would feel guilty if I don't try."

In Australia, the only stem cell treatments classed by the Therapeutic Goods Administration as safe and effective have a narrow application. They generally involve the use of umbilical cord blood or bone marrow to treat disorders of the blood or immune system.

These treatments rely on the function of stem cells, which are unallocated, to replace cells that may be lost or damaged.

Also seizing on that potential has been a burgeoning market of private clinics offering solutions to disorders broadly ranging from neurological conditions to ageing.

As the deputy director of the University of Melbourne's Centre for Stem Cell Systems, part of Megan Munsie's job has been to help inquisitive families understand the sometimes significant gap between what these clinics offered and what was actually sold.

"One of my biggest challenges in this field is the fact that stem cells are somehow seen as magical," she said.

"It's an example of non-evidence-based practice that's gone mainstream."

Ten years ago, this practice sometimes termed stem cell tourism was confined to a handful of clinics in exotic locations.

Since then Dr Munsie has witnessed growth in the number of clinics offering these treatments around the world.

In some cases, they may have been supported only by effusive patient testimonies or materials that blurred the line between medical science and marketing sometimes at the expense of communicating that these procedures were invasive and potentially harmful.

"Our responsibility as a research community is to try to contextualise the science and paint a clearer picture about where exactly we are," Dr Munsie said.

"Unfortunately that means that most of those conversations start with a disclaimer that we don't have any proven treatments for that condition."

On the other side of the equation were families like Ms Deede's, buoyed by hope and leaving no stone unturned.

Success stories may add to the guilt some parents feel.

Even in Cairns, another local family has credited two rounds of treatment with helping their son, who also lives with cerebral palsy, learn to speak and improve his fine motor skills.

"What we do know is there's certainly a lot of promise in this area, but we do still need more data to answer some of the many questions that remain," said Megan Finch-Edmondson, a stem cell researcher with the Cerebral Palsy Alliance Research Institute.

"It's a very interesting and complex question."

Like Dr Munsie, Dr Finch-Edmondson has been in frequent contact with families keen to explore their options.

An Australian-first trial conducted by Melbourne's Murdoch Children's Research Institute has been investigating the use of umbilical cord blood to treat cerebral palsy.

Even though it was only a trial and the selection criteria was rigorous, the limited spaces were highly coveted.

"That is one of the reasons that we are so committed to trying to progress this research and conduct more rigorous clinical trials in Australia," Dr Finch-Edmondson said.

"We do need to operate within [rigorous regulatory guidelines] to get any new therapy, or treatment that's like a drug, approved for use in Australia.

"It does require those very rigorous trials that can show enough data to those regulatory bodies to warrant that this treatment should be available."

Until then, families hamstrung between a long regulatory process and the promises of unproven treatments may continue to take the leap.

"I'm doing it because I love my daughter and I want to give her the best life and the best chance of improvement," Ms Deede said.

"I don't want to be in a situation where I haven't done everything I could and in 10 years' time I might be thinking 'What if?'."

Topics:medical-sciences,health-policy,medical-procedures,healthcare-facilities,medical-research,cerebral-palsy,stem-cells,cairns-4870,university-of-melbourne-3010,melbourne-3000,united-states

First posted September 18, 2019 06:34:23

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Unproven stem cell treatments provide expensive last resort in families' search for hope - ABC News