Promising target for treating brain tumors in children

November 28, 2017, Ann & Robert H. Lurie Children's Hospital of Chicago

Findings published in Oncotarget offer new hope for children with highly aggressive brain tumors like atypical teratoid/rhabdoid tumor (AT/RT) and medulloblastoma. Previously, the authors of the study have shown that an experimental drug that inhibits polo-like kinase 4 (PLK4) stopped pediatric brain tumor growth in vitro. Now, they have demonstrated its success in an animal model - the drug shrank the tumor and increased survival. Importantly, the PLK4 inhibitor was able to cross the blood-brain barrier, which means that the drug can target cancer cells in the brain even when taken orally, avoiding the need for injection into the spinal fluid. In this new study, researchers also demonstrated that when they associate a PLK4 inhibitor with traditional chemotherapy drugs, they kill tumor cells with significantly lower drug doses.

"We are very excited with the outcome of this work," said lead author Simone Treiger Sredni, MD, PhD, Associate Professor of Pediatric Neurosurgery at Northwestern University Feinberg School of Medicine and cancer researcher at the Stanley Manne Children's Research Institute of Ann & Robert H. Lurie Children's Hospital of Chicago. "This promising treatment strategy may also help us reduce toxicity when used with other chemotherapy agents."

In a previous study, Sredni and colleagues were the first to describe PLK4 as a therapeutic target for brain tumors and in pediatric cancer. They found that, in these particular types of tumors, PLK4 regulates and the tumor's tendency to metastasize.

AT/RT is a therapy resistant brain tumor that mostly occurs in infants. Medulloblastoma is the most frequent in children and current treatments for it are highly toxic. Both of these types of have a high mortality rate.

"New targeted therapies are urgently needed for children with AT/RT and medulloblastoma," said Sredni. "We expect that targeting PLK4 will prove to be an effective and less toxic treatment approach for these children, and we hope to move to a clinical trial soon."

Explore further: New potential treatment for aggressive brain cancer in children

Related Stories

New potential treatment for aggressive brain cancer in children

April 11, 2017
Chicago...Using state-of-the-art gene editing technology, scientists from Ann & Robert H. Lurie Children's Hospital of Chicago have discovered a promising target to treat atypical teratoid/rhabdoid tumor (AT/RT) - a highly ...

New target found to attack an incurable brain tumor in children

June 6, 2017
A study published in Molecular Cancer Research reveals that a tumor suppressor gene p16 is turned off by a histone mutation (H3.3K27M), which is found in up to 70 percent of childhood brain tumors called diffuse intrinsic ...

New pathway identified as a target for precision medicine against a common brain tumor

November 2, 2017
St. Jude Children's Research Hospital scientists have discovered a promising target for precision medicines to block a mechanism that drives several cancers, including about 30 percent of cases of the brain tumor called medulloblastoma. ...

PID1 gene enhances effectiveness of chemotherapy on brain cancer cells

April 11, 2017
Investigators at The Saban Research Institute of Children's Hospital Los Angeles have found that the gene PID1 enhances killing of medulloblastoma and glioblastoma cells. Medulloblastoma is the most commonly occurring malignant ...

Potential treatment for brain cancer as drug shrinks tumours

August 14, 2017
An international team of researchers has found a drug previously approved to treat breast cancer could also be used to shrink medulloblastoma, a common form of childhood brain tumour.

Molecule stops fatal pediatric brain tumor

February 27, 2017
Northwestern Medicine scientists have found a molecule that stops the growth of an aggressive pediatric brain tumor. The tumor is always fatal and primarily strikes children under 10 years old.

Recommended for you

Pushing closer to a new cancer-fighting strategy

December 11, 2018
A molecular pathway that's frequently mutated in many different forms of cancer becomes active when cells push parts of their membranes outward into bulging protrusions, Johns Hopkins researchers report in a new study. The ...

Scientists have identified and modelled a distinct biology for paediatric AML

December 11, 2018
Scientists have identified and modelled a distinct biology for paediatric acute myeloid leukaemia, one of the major causes of death in children.

HER2 mutations can cause treatment resistance in metastatic ER-positive breast cancer

December 11, 2018
Metastatic breast cancers treated with hormone therapy can become treatment-resistant when they acquire mutations in the human epidermal growth factor receptor 2 (HER2) that were not present in the original tumor, reports ...

Loss of two genes drives a deadly form of colorectal cancer, reveals a potential treatment

December 11, 2018
Colorectal cancers arise from earlier growths, called polyps, found on the inner surface of the colon. Scientists are now learning that polyps use two distinct molecular pathways as they progress to cancer, called the "conventional" ...

Successful anti-PD-1 therapy requires interaction between CD8+ T cells and dendritic cells

December 11, 2018
A team led by a Massachusetts General Hospital (MGH) investigator has found that successful cancer immunotherapy targeting the PD-1 molecule requires interaction between cytotoxic CD8+ T cells, which have been considered ...

Taking uncertainty out of cancer prognosis

December 11, 2018
A cancer diagnosis tells you that you have cancer, but how that cancer will progress is a terrifying uncertainty for most patients. Researchers at Cold Spring Harbor Laboratory (CSHL) have now identified a specific class ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.