A multi-institutional team composed of researchers from Skaggs School of Pharmacy and Pharmaceutical Sciences at the University of California, San Diego has uncovered a more effective treatment for medulloblastoma as used in the mouse models for their experiment. The said drug is more popularly known for treating a type of leukemia where the bone marrow produces white blood cells excessively.

Their study, published in the journal PLOS One on September 20, 2019, had yielded positive results in targeting cancer cells when they used a single drug known as nilotinib. The researchers pointed out that their experiment has proven the drug to be the better choice when treating a deadly pediatric brain tumor. The US Food and Drug Administration has approved Nilotinib as therapy for chronic myeloid leukemia.

The researchers explained that the cancer cells that were targeted had an abnormal activation of the Hedgehog pathway, a cellular communication system. The team later added that using a single drug became more effective and lessened its toxicity as compared to using a combination of two or more drugs.

Ruben Abagyan Ph.D., a professor at the Skaggs School of Pharmacy and the senior author of the study, stated that the previously unknown activity of nilotinib could be utilized to treat childhood brain cancer known as medulloblastoma. The professor pointed out that although more research needed to be done, this pharmaceutical could be used to treat several cancer types. 

The Hedgehog signaling pathway is a key cellular system that regulates embryonic development. The said system is also instrumental in adult tissue regeneration. This puts the impairment of the Hedgehog signaling pathway as a critical matter that could lead to various types of cancer including some types of basal cell carcinoma, myeloid leukemia, and medulloblastoma among others. Agbayan added that a quarter of cancer cases which led to death was due to the said abnormality in malignancies. 

Agbayan explained that only a fraction of medulloblastoma patients have a positive response to the treatments that target the "Smoothened," a cell-surface receptor which is over-produced by cancer cells during the impairment of the Hedgehog signaling pathway. The professor pointed out that their goal was to inhibit the pathway through the use of a single drug. 

Their study has revealed that tumor growth in their mouse models was reduced. The subjects, which bore a human medulloblastoma tumor, did not show any resistance to the drug that the team used. The team reported that not only did Nilotinib inhibit the Smoothened, but it also reduced the occurrence of several protein kinases which played a critical role in tumor growth.