An Auckland-based doctor's ground-breaking research, involving lab-cultivated, living, beating heart cells, could land a blow against a major killer of Kiwi children.
A new effort has been launched to keep Swedish paediatric cardiology expert Dr Annika Winbo's pioneering work here in New Zealand.
Winbo's studies aim to better treat children who suffer cardiac inherited diseases, particularly long QT syndrome (LQTS), which mostly affects children and young adults with frequent faints, but sometimes results in sudden death.
Each year, an estimated 100 people aged between one and 35 years old die from sudden or unexpected cardiac death.
Because the syndrome, characterised by abnormal electrical activity in the heart, is typically inherited, each child of an affected parent has a 50 per cent chance of suffering it.
"Some children can live normal lives when they're on medication, but I've talked to some parents of children who are sick, and they've never, ever, left their kids with a babysitter," said Winbo, who shared her new insights at an event at Auckland Hospital today.
Other parents were afraid to run around and play with their children, for fear of over-stimulating them.
As part of an international effort, Winbo has helped create a type of stem cell model derived from the blood of five young Swedish LQTS sufferers.
Alongside University of Auckland physiologist Associate Professor Johanna Montgomery, Winbo has developed the stem cells, which can be matured into virtually any type of cell in the human body, into heart cells.
Remarkably, this has meant that living, beating human heart cells with the exact same genetic makeup as the LQTS patient donors can be tested for arrhythmia susceptibility and that potential treatments can also be tested against the new models.
Winbo and Montgomery are now pushing forward with another model, where both human heart cells and the human neurons responsible for the so-called "fight-or-flight" response, important for triggering life-threatening arrhythmia in LQTS, could be characterised and assessed while growing together.
By better understanding the links between neurons and heart cells, the researchers hope to reveal the arrhythmia mechanics at play, and ultimately guide the way to new, disease-specific therapies for the syndrome.
There was further exciting potential in steering their scientific groundwork toward other inherited child diseases that involved a cellular basis.
Winbo said the Cardiac Inherited Disease Group (CIDG) she's part of draws on a registry of Kiwi patients, with genetic profiles unique to New Zealand.
Since its formation, the CIDG has become the gold standard for similar efforts around the globe and has proven instrumental in saving many young peoples' lives.
"This work is taking off but my fellowship ends in July; I'd definitely like to continue my research here, but if not, I'll have to carry on back in Sweden within my current contract."
The charity Cure Kids, which has contributed more than $1.5 million to the CIDC in more than a decade, is now raising funds toward Winbo's work so she can remain here.
While Cure Kids was targeting large donations, people interested in contributing to the programme could email firstname.lastname@example.org.