Every year around 200-400 children die from the incurable brain tumor, Diffuse Intrinsic Pontine Glioma (DIPG) in the US and Australia. The average age of diagnosis for DIPG is just seven years. There are no effective treatments, and average survival is from 12-18 months.
A paper published yesterday in the prestigious journal, Nature Communications, reveals a potential revolutionary drug combination that – in animal studies and in world first 3D models of the tumor – is “spectacularly effective in eradicating the cancer cells,” according to lead researcher and pediatric oncologist Associate Professor David Ziegler, from the Children’s Cancer Institute and Sydney Children’s Hospital. These promising results were the product of a research project funded by The Cure Starts Now Foundation, The Julian Boivin Courage for Cures Foundation, Hope for Caroline Foundation, The Cure Starts Now Australia, Reflections Of Grace, Aidan's Avengers, Austin Strong, The Brooke Healey Foundation, ChadTough Foundation, Cure Brain Cancer, Grant's Ginormous Gift , Jeffrey Thomas Hayden Foundation, Love Chloe Foundation, Musella Foundation, Operation Grace White, Pray Hope Believe, Ryan's Hope, Storm the Heavens Fund, Benny's World, Julia Barbara Foundation, Lauren's Fight for Cure, Lily Larue Foundation, The Isabella and Marcus Foundation (a partnership funding of The DIPG/DMG Collaborative).
In pre-clinical testing in mouse models, the researchers found that the promising drug combination led to survival in two thirds of the mice and that the drug combination completely halted growth of these highly aggressive tumors in these mice.
Importantly, the drug therapy, which is currently in early trials in adult cancer, is the most effective treatment ever tested in laboratory models of this incurable childhood cancer. The treatment is a combination of two drugs: difluoromethylornithine (DFMO), an established drug, and AMXT 1501, an investigational agent being developed by Aminex Therapeutics.
DFMO is increasingly getting attention as a treatment for difficult-to-control cancers like neuroblastoma, another aggressive childhood cancer, and colorectal cancer in adults. DFMO works by targeting the polyamine pathway – an important mechanism that allows tumor cells to grow.
Associate Professor Ziegler has shown for the first time that the polyamine pathway is critical to the growth of DIPG cells. Ziegler and his team developed Australia’s first research program into DIPG by using tumor cells donated by the parents of children who have passed away from the disease. From these, they created the first laboratory models of the tumor in order to test new drugs. These models have been used to show that DIPG can bypass the activity of DFMO by pumping polyamines into the cancer, essentially allowing the tumor to continue growing despite treatment with DFMO. They have now made the breakthrough discovery that treatment with a new developmental drug, AMXT 1501, potently blocks the transport of polyamines into the DIPG cancer cell. Treatment with AMXT 1501 was found to re-sensitize the DIPG cells to DFMO leading to what Associate Professor Ziegler said, “was a spectacular response in animal models, with a significantly increased survival and minimal toxicity (side effects).”
Associate Professor Ziegler said that clinical trials of the drug combination in DIPG are planned to begin this year in children in a global study led by the Children’s Cancer Institute and in combination with the CONNECT Consortium, a $12 million dollar, 16 hospital member trial collaborative in 4 countries and with operational funding by The Cure Starts Now Foundation, the Brooke Healey Foundation and the Reflections of Grace Foundation.
About The Cure Starts Now
The Cure Starts Now was started by parents in response to a desire to fund Homerun Cure™ and universal cure strategy research, starting with those cancers that experts believe we can learn the most from: DIPG/DMG, medulloblastoma and pediatric brain cancers. Today, The Cure Starts Now Foundation has over 40 locations in three countries. Believing in more than just awareness, The Cure Starts Now has funded over $15 million in cancer research, resulting in over 97 cutting edge research grants in 15 countries since 2007. Learn more about The Cure Starts Now and their mission to find the Homerun Cure™ for cancer at www.thecurestartsnow.org, and follow www.facebook.com/TheCureStartsNow/ for updates.
About The DIPG/DMG Collaborative
The DIPG/DMG Collaborative is a collection of foundations with the shared mission of efficiently funding and inspiring DIPG cancer research with the belief that through a cure for DIPG, significant advances in other cancer research will be made. As of 2021, membership included 28 independent foundations, dedicated to research funding with transparency, cooperation and the elimination of duplication. You can find more at www.dipgcollaborative.org.
About Children’s Cancer Institute
Originally founded by two fathers of children with cancer in 1976, Children’s Cancer Institute is the only independent medical research institute in Australia wholly dedicated to research into the causes, prevention, and cure of childhood cancer. Forty years on, our vision is to save the lives of all children with cancer and improve their long-term health, through research. The Institute has grown to now employ over 300 researchers, operational staff and students, and has established a national and international reputation for scientific excellence. More at www.ccia.org.au.
About Aminex Therapeutics
Aminex Therapeutics, Inc. is a clinical-stage biotechnology company focused on the development of a novel small molecule combination therapy for the treatment of broad range of cancer indications. Aminex has advanced AMXT 1501+ DFMO through target discovery, patenting, pre-clinical research and now into clinical development forthe potential benefit of cancer patients. Formore information,please visit www.aminextx.com