In the developed world, brain cancer ranks as the second most common cause of death for children. Standard therapies, especially for young children and babies, have long-term effects on the development and quality of life of the children who survive.
In preclinical models tested in mice, a possible novel targeted therapy for children brain cancer has been found to be successful in infiltrating and destroying tumor cells, according to research from Emory University and QIMR Berghofer Medical Research Institute.
The new medication CT-179 was demonstrated in the Nature Communications publication to target a particular population of tumor cells that cause recurrence and resistance to treatment in pediatric brain cancer. The results may result in less harmful and more effective treatments, increasing young patients’ chances of survival and quality of life.
According to the primary researchers, the results could revolutionize the treatment of medulloblastoma, the most prevalent juvenile brain cancer, and may also be applicable to other brain malignancies such diffuse intrinsic pontine glioma (DIPG) and glioblastoma (GBM).
The results of the study represent a major breakthrough in our knowledge of the biological mechanisms underlying tumor growth and recurrence, according to Timothy Gershon, a professor at Emory University, a pediatric neurologist at Children’s Healthcare of Atlanta, and the director of the Children’s Center for Neurosciences Research in the United States.
Current treatments, including radiation and chemotherapy, often eliminate most of the tumor, but sometimes fail to eliminate cancer stem cells,
These cancer stem cells can regrow the tumor after treatment, causing fatal recurrence. We show that CT-179 treatment specifically disrupts cancer stem cells. Combining CT-179 with treatments such as radiation therapy treats the whole tumor more effectively, including both stem cells and tumor cells that are not stem cells. Adding CT-179 to combinations of treatments may bring new efficacy to brain tumor therapy.
Timothy Gershon
The study teams worked with Curtana Pharmaceuticals, a U.S. pharmaceutical company that created the investigational small molecule medication known as CT-179. They discovered that the medication successfully targets the protein OLIG2, a recognized stem cell marker that is essential to the development and spread of brain tumors.
Professor Bryan Day, co-director of the Children’s Brain Cancer Centre in Australia and head of QIMR Berghofer’s Sid Faithfull Brain Cancer Laboratory, called the results a breakthrough that is all the more noteworthy because they come from separate research.
Children with brain cancer urgently need more effective and less toxic treatments,
Our study demonstrated that the drug CT-179, used in combination with standard radiation therapy can cross the blood brain barrier and penetrate the tumour. It prolonged survival in a range of preclinical medulloblastoma models, delayed recurrence of the disease, and increased the effectiveness of radiotherapy. Brain cancer is an incredibly tough puzzle to solve. As researchers, what gets us out of bed every day is trying to solve that puzzle. This global research could potentially lead to new combination therapies that improve outcomes for these young patients.
Bryan Day
The results of a different study conducted by Professor Peter Dirks from the University of Toronto, who is the neurosurgeon-in-chief and a senior scientist at the Hospital for Sick Children (SickKids) in Canada, and published in Nature Communications, are complemented by the findings from QIMR Berghofer and Emory University.
The study’s focus was on medulloblastomas, a prevalent brain tumor in children. The OLIG2 protein was found to be a crucial regulator of the tumor’s development transitions by the study using cutting-edge techniques like CRISPR gene editing, single-cell RNA sequencing, and cooperative drug testing. The results offer a new therapeutic perspective, emphasizing a move away from treating tumors generally and toward targeted therapies that target the cells that cause tumors.
Our study demonstrated that the OLIG2 protein is a critical driver of the complex early stages of medulloblastoma tumor formation, making it a highly promising treatment target,
We showed that inhibiting the OLIG2 protein with the CT-179 drug prevented cancer stem cells from changing to a proliferative state, effectively blocking the growth and recurrence of tumors. This could have potentially profound implications for treatment in the future.
Peter Dirks
Dr. Yuchen Li, a postdoctoral researcher at QIMR Berghofer and a co-first author on the study conducted by Emory University and QIMR Berghofer, is optimistic that this translational research can eventually enhance the quality of life for kids with brain cancer.
This has been a long-running study, and it is very rewarding to see it published. We are incredibly grateful for the funding support that helped make this research possible, including from the Children’s Hospital Foundation in Australia.
Dr. Yuchen Li
Clinical studies are the next step, according to Day.
We’ve been working with our collaborators, particularly in the U.S. and Australia, and we’re very hopeful that the culmination of all this work has paved the way for the first in-human clinical testing of CT-179 in patients with brain cancer.
Bryan Day
Source: Emory University – News
Journal Reference: Li, Yuchen, et al. “Suppressing Recurrence in Sonic Hedgehog Subgroup Medulloblastoma Using the OLIG2 Inhibitor CT-179.” Nature Communications, vol. 16, no. 1, 2025, pp. 1-23, DOI: https://doi.org/10.1038/s41467-024-54861-3.
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Graduated from the University of Kerala with B.Sc. Botany and Biotechnology. Attained Post-Graduation in Biotechnology from the Kerala University of Fisheries and Ocean Science (KUFOS) with the third rank. Conducted various seminars and attended major Science conferences. Done 6 months of internship in ICMR – National Institute of Nutrition, Hyderabad. 5 years of tutoring experience.
