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Breast Cancer Drug Could Help Prolong Survival For Children With Brain Tumors, Study Finds
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Having trouble? Click here.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.
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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 gliomasHigh-grade gliomas (HGGs) are aggressive tumors in the brain and spinal cord that can occur in children and adults.
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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 neededDr. 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.
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"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.
Old Chemo Drug, New Pancreatic Cancer Therapy?
Cold Spring Harbor Laboratory Professor Douglas Fearon discovered that treating pancreatic cancer with a common chemotherapy drug makes immunotherapy more effective. Folinic acid elevates levels of two anti-cancer immune molecules within pancreatic tumors: natural killer T cells and type-I interferons. In mice, this led to a more effective immune response, slower tumor growth, and longer survival.
The fight against cancer is an arms race, and one of the most effective weapons in clinicians' arsenals is immunotherapy. Immune checkpoint therapy has become the standard for treating several types of cancer. However, the Nobel Prize-winning strategy is ineffective for most pancreatic ductal adenocarcinoma (PDAC) patients.
"Immune checkpoint therapy is only an option in rare cases of PDAC," Cold Spring Harbor Laboratory (CSHL) Professor Douglas Fearon says. "It's only effective for patients with a specific subtype of PDAC -- that's less than 5% of all cases."
Until recently, it was thought that PDAC didn't trigger any kind of immune response. In 2023, Fearon and his team confirmed the opposite. Immune cells do go on the attack. But they struggle to infiltrate the deadly tumors, which allows PDAC to avoid destruction. Now, Fearon and former CSHL postdoc Jiayun Li have discovered that a common chemotherapy supplement called folinic acid weakens the cancer's defenses in mice. They found that folinic acid elevates levels of two anti-cancer immune molecules within PDAC: natural killer T (NKT) cells and type-I interferons. In mice, this leads to a more effective immune response, slower tumor growth, and longer survival.
"We discovered that NKT cells enabled type-I interferon to be produced and, as a consequence, adaptive immune killing and expansion of T cells would occur," says Fearon. "T cells respond to tumors, but they typically cannot get in there unless type-I interferon is produced. Folinic acid enhances that response."
PDAC resists immune cells, using a protective shield built from two proteins -- CXCR4 and CXCL12. This defensive wall is virtually impenetrable. But when the team treated PDAC tumors with folinic acid, cracks were revealed. The resulting elevated levels of NKT cells and type-I interferons acted like trail markers, highlighting a way past PDAC's defenses. Cancer-killing immune cells that had been kept outside the wall were able to slip into the tumor and start fighting back.
The Fearon lab now aims to translate its discovery into new therapeutics. They've recently partnered with biotech company Autobahn Labs to develop potential drugs targeting CXCR4 and CXCL12. These may one day make immune checkpoint therapy a regular option in the fight against PDAC.
"Translating observations in mice into human therapy has been difficult," Fearon says. "But if we're successful, immunotherapy may one day become a viable choice for all patients with PDAC -- and every other solid tumor -- not just in the rare cases we see today."
U.K. Charities Give $36 Million To Start New Drugs For Childhood Tumors
Sept. 5, 2024
General Assignment Reporter
For three decades, adults have benefited from a revolution in oncology treatment, as immunotherapies and targeted drugs have joined or replaced older, blunter tools like chemotherapy and radiation. Children have been less fortunate.
Their tumors are far rarer than adult cancers and attract little interest from industry. Pediatric cancer researchers hoping to advance care have had to rely largely on repurposing newer drugs developed for adults — a limited strategy, as pediatric malignancies often have unique biology that require unique drugs.
On Thursday, though, two U.K. Charities announced they would invest £28 million — $36 million — to create an international consortium capable of designing new drugs specifically for childhood tumors.
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