Little Lilly Lloyd-Morgan, aged 8, lives with a debilitating condition which stops her swallowing food. But there is hope on the horizon thanks to a potential stem cell therapy being developed by Dr Lincon Stamp and Dr Marlene Hao at the University of Melbourne.

Lilly’s parents Rhys and Marianne are leading the charge to further fund Lincon and Marlene’s work in partnership with the National Stem Cell Foundation of Australia.

The disease, known as achalasia, causes the oesophagus to stop functioning. Food builds up in the oesophagus, unable to enter the stomach. There is no cure. The most common treatment is surgery, which is only partially effective – 70% of patients still experience chest pain and difficulty swallowing, and they remain at a higher risk of developing oesophageal cancer.

Oesophageal achalasia kills nerve cells at the base of the oesophagus. Its causes are not known. Lilly’s identical twin Bella does not have the disease. These nerve cells control the pulsating motion that squeezes food through our digestive system and cause the oesophagus to open to allow food to enter the stomach.

Lincon and Marlene are developing a therapy that involves making healthy nerve cells from stem cells. These cells would be transplanted into patients like Lilly to replace the damaged or lost nerve cells and restore function.

Rhys and Marianne are taking advantage of the Foundation’s Matched Funding Program whereby, once $50,000 is raised, the Foundation will match it dollar-for-dollar to provide a total of $100,000.

They have raised $30,000 so far and so are looking for donations to reach their target of a further $20,000.

Lilly with her father Rhys, mother Marianne and siblings Bella and Nathaniel

“A stem cell-based cure would be a godsend to Lilly and other people affected by this disease,” says Rhys, a Brisbane-based lawyer.

He says Lilly has gained some relief from her symptoms thanks to surgery, but the condition hasn’t gone away and still significantly impacts her day-to-day life.

“She still has to eat and drink very, very slowly and carefully, because she’s relying on gravity to get the food down and has to sleep elevated,” Rhys says.

“Occasionally, things do get stuck, and she’ll have to throw them up, and she gets fairly regular chest pain as well.”

And, of course, the heightened cancer risk remains.

“She’s dealing with it really well, though,” says Rhys. “She has her moments, but she just keeps on keeping on. She's amazing and an inspiration to the whole family.”

Even with surgical intervention, the disease remains progressive. Symptoms often worsen in the longer-term and some sufferers may ultimately need to have their oesophagus removed (a condition known as end-stage achalasia).

“This disease completely changes people’s way of life,” Lincon says. “The operation is not curative at all and drugs are not particularly helpful.”

Based on his and Marlene’s previous work on Hirschsprung disease, where babies are born without nerve cells in their bowel, Lincon believes stem cell therapies are also possible with achalasia.

“We've been working to develop a stem cell therapy for Hirschsprung disease for quite a while now and things are going well. But Marlene and I think that we should be able to apply the principles of that kind of cell therapy to achalasia,” he says.

Lincon says there is progress in the field, which could conceivably lead to clinical application of his research within 10 years.

“Things are moving fast and there is interest from industry. We've already partnered with Takeda Pharmaceuticals over the last couple of years around the Hirschsprung project. So, they've got an interest in this field; there is some momentum in the space.”

You can help Rhys and Marianne reach their target by donating to support this research.

If you would like to make a donation of $1,000 or more, please email the Foundation’s general manager Graeme Mehegan on [email protected] to get direct transfer details. For other amounts, please donate online here and, when prompted, specify ‘Stamp for Lilly’ as the specific project.