Promising Therapeutic Target found for Medulloblastoma

In 2013 Michael Gustafson passed away, 5 years after his medulloblastoma diagnosis. Michael decided to donate his tissue to research in the hopes that other children would not experience the suffering of brain cancer. His decision gave him peace and purpose at the end of his life. This “Master Plan” of Michael’s became the inspiration for Gift from a Child.

Medulloblastoma is the most common type of pediatric brain cancer, accounting for 20% of diagnoses. There are four identified groups of this aggressive cancer, one of which is the sonic hedgehog (SHH) group. The SHH group is the most common form of medulloblastoma in patients less than three years old and overall accounts for 30% of all medulloblastomas.

In a recent breakthrough, researchers at Johns Hopkins Kimmel Cancer Center have identified a new molecule specific to SHH medulloblastomas. This molecule could potentially be used for early detection and as a new therapeutic target. A scientific article on this finding was published in Acta Neuropathologica Communications in March, 2023.

The researchers merged publicly available genetic data from 175 samples of medulloblastoma tissue and looked for significantly expressed circular RNAs (circRNAs) in each medulloblastoma classification group. CircRNA is a noncoding RNA that is believed to play a part in the development of different cancers and is seen as a good target for cancer drug discovery.

Researchers found a circRNA, known as circ_63706, that showed a significantly higher expression in the SHH group medulloblastomas. They transplanted medulloblastoma cells that did not express circ_63706 into the brains of rats. Those mice ultimately had significantly smaller tumors and prolonged survival when compared to mice with unmodified medulloblastoma.

Lipid metabolism, how the body burns fat, is known to be a key factor in tumor growth. Researchers found that when circ_63706 was not expressed, lipid metabolism increased. Lipid metabolism is toxic to cancer and ultimately leads to cancer cell death. This indicates the potential for a targeted therapy that blocks circ_63706 and causes tumor cells to die.

These findings represent a much needed step forward in changing the prognosis for medulloblastoma. This research would not have been possible without tissue samples and illustrates the immense impact of tissue donation.

Read more about this exciting breakthrough in Science Daily News.