Study reveals that a breast cancer drug may increase survival rates for children with brain tumors.

A study has discovered that a drug used to treat breast cancer, ribociclib, may slow the advancement of certain pediatric high-grade gliomas, a type of aggressive childhood brain cancer.

Kisqali, also known as ribociclib, is a CDK4/6 inhibitor that targets specific enzymes, including CDK6, to halt cancer cell growth.

A 10-year-old patient with a second relapse of pediatric high-grade glioma, who had no other treatment options, experienced 17 months of "progression-free survival" after taking ribociclib, as reported in a study published in the journal Cancer Cell.

A diffuse hemispheric glioma (DHG) typically develops in the frontal, parietal, and temporal lobes of the brain.

The Institute of Cancer Research (ICR) in London reports that the early results are impressive despite the poor prognosis for children with aggressive brain tumors.

This type of tumor usually progresses within 3½ months of recurrence.

The female patient has been receiving further treatment for four years since her diagnosis, according to the release.

"We are finally starting to see more targeted therapies come out for different forms of brain cancer."

Dr. Gregory Nalesnik, a pediatric oncologist at Memorial Sloan Kettering Cancer Center in New York, stated that ribociclib was selected for the patient based on their tumor's genetic profile, which exhibited relevant markers.

Ribociclib was found to be effective in mice, with all treated mice surviving for 110 days post-treatment, compared to only 25% of mice given a placebo.

The research, conducted by ICR and the Dana-Farber Cancer Institute in Boston, was partially funded by The Brain Tumour Charity and Cancer Research UK.

Targeted therapies for various forms of brain cancer are now becoming more visible, according to 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, as stated in the press release.

"Our patients really need these new treatment options."

In March 2017, the U.S. Food and Drug Administration (FDA) approved ribociclib for the treatment of specific advanced or metastatic breast cancer cases in postmenopausal women.

What to know about high-grade gliomas

Brain and spinal cord tumors known as high-grade gliomas (HGGs) can be aggressive and affect both children and adults.

Some 10% of pediatric brain tumors are made up by this type of tumor, according to the American Brain Tumor Association (ABTA).

Central nervous system tissue is often difficult to treat because tumors in this area tend to grow.

The survival rate for children with a high-grade glioma is less than two years, with only 30% of patients surviving for five years.

The ABTA pointed out that while current treatments for cancer include surgery, radiation, and chemotherapy, these drugs have shown greater effectiveness in adults rather than children.

Results show promise, more research is needed

Dr. Marc Siegel, a clinical professor of medicine at NYU Langone Medical Center and a senior medical analyst for Planet Chronicle, offered his opinions on the drug's potential, despite not being involved in the study.

He informed Planet Chronicle Digital that this therapy specifically targets growth factors on the surface of breast cancer.

"A rare but deadly childhood glioma (malignancy in support cells of the brain) is showing some promise in slowing progression after several months."

"This research helps us have a better understanding of what is driving this devastating disease in children."

Siegel pointed out that on a broader level, the drug could potentially affect more growth factor proteins on cancer cells.

He suggested that this method could be used in conjunction with other therapies, such as surgery and radiation, to increase lifespan.

The study highlights the growing prevalence of targeted therapy, Nalesnik commented.

The prevalence of therapy guided by molecular tumor markers in oncology is increasing, as he stated to Planet Chronicle Digital.

"These treatments, referred to as "targeted therapies," are more precise than conventional chemotherapies, which indiscriminately harm all living cells."

Nalesnik claims that targeted therapies, such as ribociclib, which aim to tackle the underlying cause of cancer cells' harmful actions, typically result in fewer side effects compared to traditional chemotherapy.

Experts agree that more research is necessary to fully understand the effectiveness of targeted therapies in treating aggressive cancers.

While more clinical trials are required to establish the effectiveness and side effects (short-term and long-term) of the treatment, I commend the authors for their application of translational research and the use of targeted therapy for pediatric high-grade glioma, as stated by Nalesnik.

In the future, the researchers anticipate that this research will lead to clinical trials involving a larger number of patients.

Dr. Simon Newman, chief scientific officer at The Brain Tumour Charity, stated in a press release that this research aids in comprehending the factors contributing to the devastating disease in children.

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"Although current treatments are not effective, discovering weaknesses in cancer cells could lead to new, targeted treatments that could improve children's lives. While this is still in the early stages, we hope it will provide some data to inform clinical trials in the future."

The study researchers and ABTA were contacted by Planet Chronicle Digital for comment.