Rare brain tumors were successfully reduced in a small study using targeted treatment.

Rare brain tumors were successfully reduced in a small study using targeted treatment.

Unprecedented Success: Targeted Treatment Shrinks Rare Brain Tumor


In an incredible breakthrough, a targeted treatment has achieved unprecedented success in shrinking a rare brain tumor, offering new hope to patients. The clinical trial results demonstrate remarkable outcomes for patients with papillary craniopharyngiomas (PCPs) who received a drug combination known as vemurafenib/cobimetinib. Each of the 15 patients who participated in the trial showed positive response to the targeted therapy, with an average reduction of 91% in tumor size.

Addressing a Health Challenge

PCPs can lead to significant health problems, and the standard treatment options of surgery and radiation have limitations. Incomplete removal of the tumor and the toxicity associated with radiation treatment often result in lifelong challenges for patients, including neuroendocrine dysfunction, vision impairment, and memory loss. Consequently, the success of this targeted therapy presents a game-changing moment in brain tumor treatment.

“All patients who completed one or more cycles of therapy responded to treatment, which is the highest response rate to date of any medical therapy for brain tumors,” said Dr. Priscilla Brastianos, co-author of the study and director of the Central Nervous System Metastasis Center at Mass General Cancer Center. She further stated, “These unprecedented results signal a paradigm shift for targeting brain tumors because they show that, with the right target and the right drugs, precision medicine can have a dramatic impact on brain tumors.”

The Power of Precision Medicine

This groundbreaking study, led by the Mass General Cancer Center, marks the first multicenter treatment protocol for PCPs. It builds upon previous laboratory discoveries that identified genetic drivers of PCP growth, leading researchers to discover that existing cancer medications can disrupt faulty genes in PCPs, halting their progression and significantly reducing their size.

One significant finding by Dr. Brastianos and her colleagues is that approximately 95% of PCPs have a BRAF V600E mutation, a key driver of their cancerous activity. This same mutation is found in some forms of melanoma. By leveraging recent FDA-approved therapies that inhibit the BRAF and MEK genes for treating melanoma and other cancers, the researchers hypothesized that a BRAF/MEK inhibitor might also be effective for treating PCPs.

To test this hypothesis, the researchers screened PCP patients across the country for BRAF V600E mutations. Sixteen patients from nine centers were enrolled, and 15 patients completed at least one cycle of the 28-day targeted therapy.

Extraordinary Results and Long-Term Benefits

Over the course of four treatment cycles, the average reduction in tumor size was an astounding 91%. Some patients experienced reductions ranging between 68% and 99%. What sets this treatment apart is its potential long-term benefits. Seven patients did not receive any additional treatment after discontinuing the targeted therapy, and nearly two years later, six of them showed no evidence of tumor progression. No patient’s tumor progressed while on the medication, and no deaths occurred.

Though some patients experienced adverse effects, such as rashes, the majority tolerated the drugs well and continued treatment beyond the initial four cycles. However, more research is needed to determine the optimal number of treatment cycles and further establish the effectiveness of this targeted therapy.

“This study demonstrated that national, biomarker-driven trials are feasible for patients with brain tumors,” remarked Dr. Brastianos. She emphasized that advancing the treatment of rare brain tumors necessitates a multi-institutional and multidisciplinary effort, as showcased by the collaboration and research conducted for this study.

Future Implications and Hope

The remarkable success of this targeted treatment for shrinking PCPs not only offers hope to patients facing this rare brain tumor but also highlights the power of precision medicine in fighting complex diseases. As researchers continue to uncover genetic drivers and develop targeted therapies, new possibilities for treating various cancers and disorders come to fruition.

The study, supported by the U.S. National Cancer Institute and others, was published in the New England Journal of Medicine, cementing its importance and impact in the field. The findings hold promise not only for brain tumor patients but also for researchers and medical professionals who are working tirelessly to improve treatments and outcomes in the realm of precision medicine.