Luminesce Alliance held its annual Paediatric Precision Medicine Forum on the 26 September 2019 with over 50 participants attending. The Forum provided an opportunity to interact and discuss the key activities that are occurring across the entire Precision Medicine Program as well as identifying key strategies for further collaboration and innovation.

Key speakers included:

• Professor Ian Alexander – Head, Gene Therapy Unit, Children’s Medical Research Institute (Australia)
• Professor Patrick Tam – Head, Embryology Unit, Children’s Medical Research Institute (Australia)
• Dr Shafagh Waters -Lead Scientist, Australasian Centre for Personalised Cystic Fibrosis Medicine (ACPCFM), School of Women’s and Children’s Health, University of New South Wales Sydney
• Dr Anai Gonzalez Cordero – Children’s Medical Research Institute
• Associate Professor Mark Cowley – Group Leader, Computational Biology Group, Children’s Cancer Institute
• Vanessa (Ness) Tyrrell – Co-Head, Personalised Medicine Theme, Children’s Cancer Institute
• Associate Professor Tracey O’Brien – Director, Kids Cancer Centre, Sydney Children’s Hospital Network
• Dr Luciano Dalla-Pozza – Head and Senior Staff Specialist, Cancer Centre for Children, The Sydney Children’s Hospital Network
• Professor Natasha Nassar – Financial Markets Foundation for Children Chair in Translational Childhood Medicine at the Children’s Hospital at Westmead, Clinical School
• Ms Cate Smith – Associate Director Research Operations, Sydney Children’s Hospitals Network
• Professor Chris Cowell – Director of Research, Sydney Children’s Hospitals Network
• Professor Deborah Schofield, Director GenIMPACT: Centre for Economic Impacts of Genomic Medicine and Professor and Chair of Health Economics, Macquarie University
• Associate Professor Michelle Farrar – Paediatric Neurology, School of Women’s and Children’s Health, University of NSW Sydney & Lead, Neuromuscular Diseases Clinical and Research Program Sydney Children’s Hospital Network

Personalised mini-organs are changing the way health experts understand Cystic Fibrosis (CF), the most common inherited disorder affecting Australian children.

Professor Adam Jaffe, John Beveridge Professor of Paediatrics and Head of School of Women and Children’s Health at the University of New South Wales (UNSW) and Clinical Academic, Respiratory Medicine at Sydney Children’s Hospitals Network along with Dr Shafagh Waters, Molecular and Integrative Cystic Fibrosis (miCF) Research Centre ® laboratory group leader and senior research associate at UNSW and Sydney Children’s Hospital discussed the importance of these mini-organs in a recent interview on ABC Radio National.

 

The newest lab to be established at Children’s Medical Research Institute is really at the cutting edge of modern medicine, using stem cells to provide regenerative medicine for future generations of children.

The Stem Cell Medicine Group has been started by Dr Anai Gonzalez Cordero, who joined CMRI from London, earlier in the year.
Dr Gonzalez Cordero studied Developmental Biologist at University College in London and was awarded a PhD in retinal regeneration. Her thesis, which established that you could use stem cells as a treatment for blindness, was published in the prestigious Nature Biotechnology.

When she was asked to move to Australia and start up a new lab at CMRI, Dr Gonzalez Cordero jumped at the opportunity.

“CMRI offered me a wonderful opportunity to set up a whole new field of research at the Institute,’’ she said. “It’s a challenging task to establish a whole new group and facility at the same time, but one that I embrace with enthusiasm.

“CMRI has excellent research facilities. There are few other centres in the world that have this concentrated level of research expertise including gene therapy and ocular disease infrastructure. It provides an ideal environment in which to translate laboratory-based stem cell research, especially of the eye, into clinical studies. CMRI has an environment that allows for collaborations between numerous areas of expertise.”

Dr Gonzalez Cordero said her vision for the next five years is very clear.

“There are currently many incurable childhood diseases, and stem cell approaches offer significant promise towards therapies in these conditions. My expertise with differentiation of pluripotent stem cells protocols was crucial to allow for an easy transition from the retina to the ear. Therefore, I envisage that in collaboration with other group leaders at CMRI we will be able differentiate stem cells into a variety of other organ systems for disease modelling purposes.”

She said she would like to see stem cell medicine used more widely for regenerative purposes.

“In the long term, I hope to be able to develop impactful translational research to address significant unmet clinical needs.”