Thousands of tiny hearts grown in lab to better test new drugs
When it comes to determining whether new drugs will be effective, Australian researchers looked to their hearts. Thousands of them.
Researchers from QIMR Berghofer Medical Research Institute have used stem cells to create thousands of samples of heart tissue to comprehensively test compounds which are being considered for new heart medication.
Head of QIMR Berghofer’s Organoid Research Laboratory, Associate Professor James Hudson, said they developed the method after an initial screening program using traditional methods indicated hundreds of different samples were viable.
“They initially screened five thousand compounds but far too many of them activated a response, over 100 activated a regeneration response,” Dr Hudson said.
“After that sort of study you usually have to go to other avenues such as pre-clinical animal models, but that was far too many candidates to move through the pipeline.”
Instead the drug company, AstraZeneca, teamed up with QIMR to use their stem cell method, which resulted in just two compounds being identified as promising to move to the next stage of development.
The method developed by QIMR creates tissue samples which behave almost exactly like human heart tissue, including beating regularly, as opposed to the inert cell cultures which are normally used.
The two potential drug candidates could help regenerate damaged heart tissue without negative side effects on heart function.
Dr Hudson said while this study involved heart tissue, the stem cell method could be used in a wide range of scenarios.
“This system is very versatile. There’s a whole array of different screens and different research projects we can use these tissues for,” he said.
“The intention is we have a human system rather than a rodent system to do the initial testing in.
“Also because we can generate all these tissues we can more quickly go through different conditions to find the best ones to move forward.”
The study also featured work from the Murdoch Children’s Research Institute and the University of Melbourne.
Co-lead author Enzo Porrello from the Murdoch Children’s Research Institute said the research in the current trial could eventually let doctors heal a broken heart.
“The study also showed that heart muscle would only regenerate if the drug treatment could turn on specific metabolic pathways – something that hadn’t been shown before,” Dr Porello said.
“It’s very early days, and there are years of testing ahead, but our research provides hope of finding therapeutics that could regenerate disease-damaged hearts.”
More broadly it’s hoped this new research method could eventually reduce the cost of drugs, by reducing the time and money spent chasing down research dead-ends.
“At the moment nine out of ten drugs fail in clinical trials and if we can improve that we could improve the cost of drugs,” Dr Hudson said.
“When you pay for a drug you’re not just paying for the drug you’ve bought, you’re paying for all the other drugs which failed during development.”
The research results have been published on Friday in the journal Cell Stem Cell.