A 10-year-old girl who took the drug lived for 17 months with no disease progression
A drug called ribociclib, currently used to treat breast cancer, could slow the progression of certain pediatric high-grade gliomas, an aggressive type of childhood brain cancer, a new study has found.
Ribociclib (brand name Kisqali) is a CDK4/6 inhibitor, a type of drug that targets specific enzymes — including the protein CDK6 — to stop cancer cells from growing.
In the study, which was published in the journal Cancer Cell, a 10-year-old patient who had experienced a second relapse of pediatric high-grade glioma — and had no other treatment options — experienced 17 months of "progression-free survival" after taking ribociclib.
The patient had a diffuse hemispheric glioma (DHG), which typically develops in the frontal, parietal and temporal lobes of the brain.
"These very early results are remarkable given the typically poor prognosis for children diagnosed with these aggressive brain tumors," according to a press release from the Institute of Cancer Research (ICR) in London.
A drug called ribociclib, which is currently used to treat breast cancer, could slow the progression of certain pediatric high-grade gliomas. (iStock)
This type of tumor usually progresses within 3½ months of recurrence.
It has been four years since the female patient received her diagnosis. She is currently receiving further treatment, the release stated.
"We are finally starting to see more targeted therapies come out for different forms of brain cancer."
"Ribociclib was chosen based on the genetic profile of the patient's tumor, which demonstrated relevant markers," said Dr. Gregory Nalesnik, a pediatric oncologist at Memorial Sloan Kettering Cancer Center in New York, who was not involved in the study but reviewed the findings.
The drug was also shown to be effective in mice — 100% of the mice treated with ribociclib survived for 110 days after treatment, compared to only 25% of the mice who received a placebo.
High-grade gliomas (HGGs) are aggressive tumors in the brain and spinal cord that can occur in children and adults. (iStock)
The study, which was led by ICR and the Dana-Farber Cancer Institute in Boston, was partly funded by The Brain Tumour Charity and Cancer Research UK.
"We are finally starting to see more targeted therapies come out for different forms of brain cancer," says senior author Mariella Filbin, M.D., PhD, co-director of the Brain Tumor Center of Excellence at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, in the press release.
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"Our patients really need these new treatment options."
Ribociclib was approved by the U.S. Food and Drug Administration (FDA) in March 2017 for the treatment of certain types of advanced or metastatic breast cancer in postmenopausal women.
What to know about high-grade gliomas
High-grade gliomas (HGGs) are aggressive tumors in the brain and spinal cord that can occur in children and adults.
This type of tumor makes up some 10% of pediatric brain tumors, according to the American Brain Tumor Association (ABTA).
They are generally difficult to treat because they often grow into the tissue of the central nervous system.
A 10-year-old patient (not pictured) who had experienced a second relapse of pediatric high-grade glioma — and had no other treatment options — experienced 17 months of "progression-free survival" after taking ribociclib. (iStock)
In children who are diagnosed with a high-grade glioma, the overall survival rate is less than two years, with less than 30% of patients surviving for five years.
Current treatments include surgery, radiation and chemotherapy, although these drugs have proven more effective for adults than children, noted the ABTA.
Results show promise, more research is needed
Dr. Marc Siegel, senior medical analyst for Fox News and clinical professor of medicine at NYU Langone Medical Center, was not involved in the study but shared his opinions on the drug’s potential.
"This is a targeted therapy that has been used in breast cancer to attack growth factors on the surface of the cancer," he told Fox News Digital.
"It is now showing some promise in slowing progression of several months in a rare but deadly childhood glioma (malignancy in support cells of the brain)."
"This research helps us have a better understanding of what is driving this devastating disease in children."
On a wider scale, the drug could target more growth factor proteins on cancer cells, Siegel noted.
"This approach may be combined with other treatments, including surgery and radiation, to help prolong life," he added.
In children diagnosed with a high-grade glioma, the overall survival rate is less than two years, with less than 30% of patients surviving for five years. (iStock)
The study highlights the growing prevalence of targeted therapy, Nalesnik commented.
"The use of therapy guided by molecular tumor markers is becoming more and more prevalent in the world of oncology," he told Fox News Digital.
"Sometimes called ‘targeted therapies,’ these treatments are much more specific than traditional chemotherapies, which are globally cytotoxic (toxic to living cells) in general."
"This research helps us have a better understanding of what is driving this devastating disease in children," a scientist said. (iStock)
Targeted therapies like ribociclib, which are designed to address the root cause of a cancer cell's harmful behavior, often have fewer side effects than conventional chemotherapy, according to Nalesnik.
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While targeted therapies show promise in treating aggressive cancers, experts agree that more research is needed.
"Although more clinical trials are needed to lend evidence to efficacy and information about side effects (both short-term and long-term), I applaud the authors in their application of translational research and the use of targeted therapy for pediatric high-grade glioma," Nalesnik added.
Looking ahead, the researchers hope that this study will pave the way to clinical trials that include more patients. (iStock)
Looking ahead, the researchers hope that this study will pave the way to clinical trials that include more patients.
"This research helps us have a better understanding of what is driving this devastating disease in children," said Dr. Simon Newman, chief scientific officer at The Brain Tumour Charity, in the press release.
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"We know that current treatments are not effective and that finding vulnerabilities in cancer cells could lead to new, targeted treatments to help children live longer and better lives. Although this is very early days, we hope that it will provide some data to inform clinical trials in the future."
Fox News Digital reached out to the study researchers and to ABTA for comment.
Melissa Rudy is senior health editor and a member of the lifestyle team at Fox News Digital. Story tips can be sent to
