Daijiworld Media Network- New Delhi
New Delhi, Feb 5: In a ground-breaking development, researchers from Emory University and QIMR Berghofer Medical Research Institute in Queensland, Australia, have unveiled a promising new drug, CT-179, that has shown remarkable effectiveness in treating medulloblastoma (MB), the most common malignant brain tumor in children. Published in nature communications, the study reveals that CT-179 successfully targets and destroys cancer stem cells, which are often resistant to traditional therapies.
Current treatments for medulloblastoma, such as radiation and chemotherapy, can eliminate most tumor cells but often fail to eradicate cancer stem cells. These cells are responsible for tumor regrowth and fatal recurrences. Dr Timothy Gershon, professor of neuro-oncology at Emory University School of Medicine and one of the lead researchers, explained, “CT-179 treatment specifically disrupts cancer stem cells. When combined with radiation therapy, it treats the tumor more comprehensively, targeting both stem cells and regular tumor cells.”
CT-179 works by inhibiting a protein known as OLIG2, a critical marker associated with cancer stem cells involved in the initiation and recurrence of brain tumours. The drug has shown particular effectiveness against the Sonic Hedgehog (SHH) subtype of medulloblastoma, which is notably resistant to conventional treatments and prone to recurrence. SHH-driven medulloblastoma accounts for approximately 25% of all MB cases, primarily affecting infants and young children.
Traditional treatments for MB, while life-extending, often cause long-term cognitive and physical side effects, especially in younger patients. CT-179’s targeted approach offers a significant advantage, as it focuses on the specific stem cells responsible for tumor regrowth, thereby reducing harm to healthy brain tissue and minimizing side effects.
The researchers conducted extensive preclinical studies using human MB cell lines, patient-derived tumor organoids, and genetically modified mice. Results showed that CT-179 effectively inhibits OLIG2, leading to the destruction of tumor cells and preventing their ability to regenerate. Additionally, when combined with radiation therapy, CT-179 demonstrated the ability to cross the blood-brain barrier, penetrate tumours, prolong survival, delay disease recurrence, and enhance the effectiveness of radiation treatment.
Dr Bryan Day, senior author of the study and group leader at QIMR Berghofer Medical Research Institute, highlighted that CT-179’s potential extends beyond medulloblastoma. The drug has also shown promising results against other aggressive paediatric brain tumours, including glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG), both of which present significant treatment challenges. “This global research could lead to new combination therapies that improve outcomes for young patients,” Dr Day remarked. “What motivates us is the drive to solve the puzzle of brain cancer.”
While the initial results are promising, researchers caution that further studies are needed to confirm CT-179’s safety and efficacy in humans. One concern is its potential impact on normal brain function, particularly in relation to myelination, which is vital for healthy brain development. However, preliminary findings indicate that CT-179 does not adversely affect myelinating cells, suggesting it may be safe for use in paediatric patients.
The next step for the research team is to launch clinical trials to determine the optimal combination therapies for CT-179. They are also exploring the possibility of pairing the drug with other targeted therapies, such as Palbociclib, to prevent the development of treatment resistance.
This breakthrough offers renewed hope for children battling medulloblastoma and other brain cancers, marking a significant step forward in the quest for more effective and less harmful cancer treatments.