Description
Children's Medical Research Institute is committed to finding cures and better treatments for a range of genetic diseases, birth defects, and cancers. How We’re Different
Not all research is the same.
CMRI conducts fundamental (meaning fundamentally important) medical and biological research. We ask the difficult questions. What causes cancer? How do we stop it? Why does development go wrong and how can we prevent it? How does the brain work? Can what we’ve learned about brain cells be put to use to treat epilepsy and other neurological disorders?
Our task is to make the future better.
CMRI scientists ask the difficult questions in order to gain the important answers. This knowledge will enable us build a better future and light the spark that will lead to tomorrow’s discoveries.
What we’ve accomplished in the last 20 years:
Found a single genetic defect can cause cleft lip and palate
Fate map of the early embryo to help us understand many developmental problems
Identification of the components of telomerase, which will be important for treating 85% of all cancers
Discovery of the Alternative Lengthening of Telomeres (ALT) mechanism, which can lead to treatments for the other 15% of cancers
Developing a blood test for ALT cancers that will allow physicians to diagnose and plan treatments for cancers such as aggressive glioblastoma brain tumours
Partnering with the Children’s Hospital Westmead on a cure for genetic liver disease, with clinical trials about to begin
Discovering and developing a new class of drugs for treating epilepsy
What we plan to accomplish in the next 20 years:
Develop epilepsy treatments that will help children (and adults) around the world
Develop new treatments for kidney disease and diabetes
Gene therapy cures for rare genetic diseases in children
New treatments for infectious diseases
Telomere research to help us understand predisposition to disease
Find new and better treatments for every type of cancer
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facebook.comChildren remain underrepresented in drug research, says expert
"Children continue to be underrepresented in drug and medical research, making them less likely to receive personalized health-care treatments for life-threatening conditions such as cancer and infectious disease." We are currently working to develop medicine for epilepsy and cancer in children at CMRI. With your help, we believe our work can greatly improve the outcomes for future generations.
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“In 1993, I had a healthy baby girl named Samantha. Then in 1997 I fell pregnant again. The [second] pregnancy was met with a lot of problems. I went to a paediatrician in Hobart who diagnosed the baby with congenital Myotonic Dystrophy (MD). At 35 weeks, the baby stopped moving and I went in for an emergency cesarean. 5 days after he was born, Jesse died. Due to Jesse’s condition and the possibility of it being hereditary, my mother, my sister and I were tested. We all came back positive for MD. Five years later, after suffering quite considerably with pain and being in and out of hospital, my mother died from heart complications secondary to MD. She was 63. In 2013, my sister died suddenly from heart complications secondary to MD. My sister was 52. I suppose the worst part of this disorder is that no one can tell you exactly what is going to happen, as everyone is different, even among family members, or how long your life will be. I sometimes read articles on the internet regarding MD and hope that one day perhaps there is an article there to say that a treatment has been found, but I don’t think [it will happen] in my lifetime. I believe research may be able to find answers to disorders such as MD and at least give families hope for the future.” - Carole Cross
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Please remember to spare a thought for families in need today, whose situation can often seem most difficult over the holidays. From CMRI to them, and all of you, we wish you a very happy and healthy Christmas!
Linke's Story
Every child should have a future, and medical research could grant them one. This is Linke's story <3 #CureChildhoodDiseases
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Katherine Pym and her husband Daniel were normal soon-to-be parents, dreaming of the life they would have with their first child. But on July 16, 2010, the unsuspecting couple were faced with a staggering new reality. “Initially it was shock. You have this dream when pregnant how everything will be and then that gets shattered. You go through a day-to-day process dealing with it and grieve the loss of this life you’ve made up. First it’s shock, then anger, grieving and then acceptance - well this is how life is.” Within the first 24 hours of their daughter Eva’s life, she was rushed to Neonatal Intensive Care and diagnosed with cardiomyopathy. But it was a second, much rarer, diagnosis that would send Eva’s parents on a hunt for answers. Little Eva was diagnosed with a disorder called '6q23.5 -25.1 deletion' - the only diagnosis of its kind anywhere in the world at the time. “All [the doctors] could do was point us in the direction of a unique organisation in the UK which had material on duplications and deletions. We went to them for information and anything we could find out about chromosome 6 and there was only one document. We were left in limbo.” “We’re constantly looking for answers. All we can do at the moment is go off what other families have been through, but even then every child is different." For Eva's family, continued medical research and development is the only hope they have for getting answers. So until then, a priority in the Pym household is to make every day as special as Eva.
Cheeki Monkey
Looking for some last minute gifts for a new mum in your life? Cheeki Monkey are donating $2 from each sale to us up until Christmas Eve. Shop away!
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When Ella was just 3 years old her parents started to notice something was wrong. She wasn’t gaining weight, despite eating normally, and seemed to tire too easily. At first doctors weren’t sure what was wrong but extensive examinations finally led to the discovery of a large, life-threatening tumour in her liver. The tumour was so far advanced that large volumes of blood were being diverted to it, putting Ella's heart under significant stress, and leaving her with a condition so rare that it challenged doctors across the country. “There are still so many unanswered questions for us in relation to Ella’s medical condition, but we live in hope." Ella's mum Kath says. "I just hate to think that other children might have to endure the same struggles that our darling little Ella has faced, it’s just so heart-breaking. We know just how vital the research work is, not just in terms of treating childhood cancer…but preventing it." Ella's parents hope that continued medical research will help the next family suffering from a traumatising diagnosis like theirs.
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"Alone we can do so little, together we can do so much." Today on #InternationalVolunteersDay we are sending an enormous thank you to all of our outstanding volunteers, with very special appreciation going to our committee and board members! Most important are the many families that have you to thank for your ceaseless commitment. Only together can we continue to create a better future for children everywhere. If you've not been involved with us before, find out how to start today! http://bit.ly/2gWH2Zp
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"Riley was a normal, naughty boy until age 6. Then something odd happened on the soccer field…all the boys took off after the ball, but he was just standing there with a funny look on his face. Months later he was diagnosed with epilepsy and given drugs, combinations of drugs - but nothing worked." We want to give children like Riley and his mum Ruth a better hope for the future this Christmas. Like and Share if you think they deserve our help! Find out how you can contribute: http://bit.ly/2gTNlj5
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For most families the holiday season is a time to celebrate with loved ones - but for families facing childhood disease it can be the toughest time of all. Every day at CMRI we work toward creating a future without childhood disease. Get involved this #GivingTuesday and help us bring a bit of hope to the families who need it most. <3 http://bit.ly/2gmiFU9
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Today is Universal #ChildrensDay - the UN recognised day to celebrate every child's right to life, health, education and play. Our mission is to create a future free of childhood disease - and for us that doesn't just happen today! It happens each and every day as our researchers strive to develop cures and medical advancements for children like Livia, whose battle for a healthy life is ongoing. Share if you're supporting every child's right to a healthy future alongside us! Jeans for Genes Australia FIT In Your Jeans for Genes
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21 years ago Linda Penn's daughter was born with a life threatening disease and was immediately rushed into emergency microsurgery. Two decades later, the Penn family continues to support organisations they believe helped their daughter survive her traumatic introduction to the world. “We felt it only fitting that we give something back to those who contributed to saving our daughter’s life and whose work would save the lives of those yet to be born babies with genetic disorders.” Last weekend Linda and her family hosted a large scale masquerade fundraiser with all proceeds going toward CMRI and our research to end childhood disease. Like if you applaud their generous efforts! Considering running your own event? Find out how here http://bit.ly/2f6lie6
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