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ATRT Tumor Research Focuses on Rare Pediatric Brain Cancer

5/13/2024

Slide showing blood vessel and tumor cells.

Central nervous system tumors are the leading cause of death among pediatric cancer patients. Atypical teratoid rhabdoid tumor, or ATRT, is a particularly rare and aggressive tumor most often seen in the cerebellum. Most children with ATRT are diagnosed before they reach their third birthday and face a survival rate of less than 15%.

Children’s Hospital Colorado hematologist/oncologist Jean Mulcahy Levy, MD, studies ATRT tumors in an effort to change these difficult statistics. Her team was recently awarded a grant to further this work from Hyundai Hope on Wheels, a program sponsored by the car manufacturer, which supports innovative childhood cancer research projects. Using this grant, Dr. Mulcahy Levy and her team will study a potential ATRT treatment that combines inhibitors of cyclin-dependent kinase 7, or CDK7, with antimetabolic therapy. CDK7 plays an important role in cell growth and division, but in ATRT, CDK7 becomes dysregulated and results in the rapid growth of tumor cells. Antimetabolic therapy is already key in ATRT treatment, but Dr. Mulcahy Levy hypothesizes that combining it with a CDK7 inhibitor could make it more effective at halting tumor growth.

“If successful, we have the opportunity to incorporate a new way of targeting cancer cells into pediatric cancer therapy and improving the survival of patients with ATRT.”

— Jean Mulcahy Levy, MD

By confirming the treatment’s viability against ATRT, Dr. Mulcahy Levy hopes the grant will help pave the way for a clinical trial. “If successful, we have the opportunity to incorporate a new way of targeting cancer cells into pediatric cancer therapy and improving the survival of patients with ATRT,” she says.

In addition to providing more hope for children and families facing this rare and aggressive diagnosis, Dr. Mulcahy Levy's research has the potential to cure other tumors that grow in a similar way. ATRT tumors result from a mutation in the SMARCB1 component of the SWI/SNF (SWItch/Sucrose Non-Fermentable) chromatin remodeling complex, and 25% of all cancers contain this specific type of mutation. Meaning, Dr. Mulcahy Levy's work on ATRT could potentially translate to other cancers beyond the brain.

If you'd like to learn more about Dr. Mulcahy Levy's ATRT research and goals at Children's Colorado, watch her discuss it in further detail in the below video.