by What is Ependymoma?
Ependymoma is a type of brain tumor that starts from cells called ependymal cells that line the ventricles and passageways within the brain and spinal cord where cerebrospinal fluid is produced and circulated. This rare cancer mostly affects children under 10 years of age. The exact cause of ependymoma is unknown but genetics and exposure to radiation are considered risk factors. Tumors can develop in the brain or spinal cord. Surgery followed by radiation and chemotherapy are the current standard treatments but the tumors often grow back, making long-term treatment difficult.
A New Targeted Drug
Researchers have been trying to find more targeted therapies that can specifically attack cancer cells while sparing healthy tissues. In the case of ependymoma, scientists studied the genetic makeup of tumors and identified a protein called BRAF as an important driver of cancer growth. A drug called vemurafenib was then tested that specifically inhibits the BRAF protein. This drug is already FDA-approved for another cancer called melanoma where BRAF mutations also play a role.
Early Trial Results Look Promising
In the first phase 1 clinical trial, 36 children and young adults with recurring Ependymoma Drug tumors were given vemurafenib. Researchers observed that the drug was well-tolerated with minimal side effects in most patients. Excitingly, brain imaging showed the cancer had stabilized or partially responded in 19 out of 36 patients based on decrease in tumor size or slow growth over months. No complete remissions were noted yet but these early signs suggest the drug may be able to curb ependymoma progression.
Larger Trials Still Needed
While the initial results are encouraging, doctors caution that more research is still warranted before vemurafenib can be designated as a standard ependymoma treatment option. The phase 1 study had a small sample size and short follow-up duration. To truly assess drug effectiveness and safety, larger phase 2 and 3 trials with control groups are essential. These would prove if vemurafenib alone or in combination with other therapies can significantly extend patient survival compared to existing regimens. Scientists are also exploring mechanisms of drug resistance to further augment treatment outcomes.
Hope for Patients and Families
For families of children battling ependymoma, this emerging targeted therapy offers renewed optimism. If successful in later trials, vemurafenib could finally provide a much-needed oral treatment alternative that may shrink tumors without harsh side effects of chemotherapy or long hospital visits for radiation. It could potentially help delay cancer recurrence after initial therapy or even control it for years. Experts believe even minor improvements in the bleak prognosis of ependymoma would have a major psychological impact on patients and caregivers. The scientific community is cautiously optimistic but also committed to speed up research to help save more young lives.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
