Ependymoma is a rare type of brain tumor that occurs in the brain or spinal cord. While treatments like surgery, radiation, and chemotherapy have helped improve outcomes for some patients, ependymoma remains difficult to treat and often comes back after initial therapy. However, several new drug therapies are now in clinical trials that could offer hope for more effective long-term control of this disease.
Current Treatment Landscape
The current standard of care for ependymoma involves maximal safe surgical resection of the tumor, followed by radiation therapy and sometimes chemotherapy. While this multi-pronged approach can achieve remission in many cases initially, recurrence is still common. According to statistics from seminal clinical trials, up to 60% of patients experience the tumor recurring within 5 years after initial therapy.
When ependymoma returns, salvage treatments are limited and outcomes tend to be poor. Additional surgery may sometimes still be possible depending on the location and extent of regrowth. But repeat radiation is typically not an option due to risks of further neurological toxicity. Chemotherapy has shown only modest benefit in recurrent settings. As a result, average survival after relapse is estimated to be around 1-2 years, emphasizing the need for more effective long-term therapies.
Novel Targeted Drugs in Testing
Scientists have gained deeper insights into the molecular drivers of ependymoma in recent years. Several key genetic alterations have been identified that appear central to tumor growth. This has opened doors to developing targeted therapies aimed at specifically inhibiting these cancer-promoting pathways. A number of such drugs are now progressing through early and mid-stage clinical testing.
One promising approach involves blocking signals from a receptor tyrosine kinase called MERTK. Overexpression of MERTK is common in Ependymoma Drug , and early research suggests it plays a key role in tumor cell proliferation and invasiveness. A MERTK inhibitor called UBL3027, developed by Ultragenyx Pharmaceutical, entered phase 1 testing last year for recurrent ependymoma and other solid tumors. Preliminary data indicates the drug has acceptable safety and hints of anti-tumor activity.
Another trial launched in 2021 is evaluating an investigational PI3K inhibitor called lenvatinib in children and young adults with recurrent ependymoma. The PI3K-Akt pathway is hyperactivated in around 40% of ependymoma cases, driving unchecked growth signals. By blocking this pathway using lenvatinib, researchers hope to rein in tumor growth more precisely. Early results from the dose escalation phase have demonstrated a favorable safety profile so far.
Immunotherapy Approaches Explored
In parallel with targeted agents, immunotherapy is also being rigorously tested as a way to harness the body's immune system against ependymoma. One approach involves chimeric antigen receptor (CAR) T cell therapy, where a patient's own T cells are engineered to recognize and attack tumors. A phase 1 clinical trial of CAR T cells targeting the marker IL-13Rα2 in recurrent ependymoma has been accruing patients since 2020. Several participants have shown objective responses so far with this personalized cellular therapy approach.
Combining immunotherapy with targeted drugs is another strategy under active investigation. For example, a phase 1 trial will evaluate the PI3K inhibitor lenvatinib together with the immunotherapy pembrolizumab, combining immunotherapy with precision targeting of tumor growth signaling. The goal is to amplify antitumor effects through dual modes of action. Results of these pioneering combination studies could help pave the way for entirely new standards of care.
Looking Towards a Cure
While large controlled trials are still required to prove lasting benefit, the flurry of new therapeutic agents now in clinical testing brings much needed hope to the ependymoma community. If some of these targeted therapies and immunotherapies demonstrate durable responses, they may potentially start shifting the current treatment paradigm from temporary remission to long-term control and even cure for some patients.
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Summary Researcher clinical trial report, “Ependymoma Global Clinical Trials Review, H1, 2020" provides an overview of Ependymoma Clinical trials scenario.
This report provides top line data relating to the clinical trials on Ependymoma.
The report offers coverage of disease clinical trials by region, country (G7 & E7), phase, trial status, end points status and sponsor type.
Report also provides prominent drugs for in-progress trials (based on number of ongoing trials).
GlobalData Clinical Trial Reports are generated using proprietary database - Pharma eTrack Clinical trials database.
Clinical trials database undergoes periodic update by dynamic process.
