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New Cancer Drug Trial For Neurofibromatosis

Micdametinib is in investigation (Phase 3 clinical trials) as an oral allosteric treatment for neurofibromatosis. It is a small molecule MEK inhibitor. It is hypothesized to be used as a monotherapy treatment in people with neurofibromatosis that have plexiform neurofibromas and low-grade glioma. Also as a combined therapy in treating many subsets of biomarker-defined metastatic solid tumors.

Micdametinib has been developed to inhibit MEK1 and MEK2, these occupy substantial significant places within the MAPK pathway.

In DNA, the MAPK pathway triggers key networks which regulate both growth and longevity of cells. Playing an important role in many cancers in which MAPK dysregulates cell development.

In NF1, a genetic disorder that rarely occurs but happens when there are mutations within the NF1 gene. NF1 effects any or several systems within the body, existing from birth, lasts all throughout life. Effected persons have 30%-50% chance during their lifetime to develop plexiform neurofibromas (PN)

PN are nerve sheath tumors chronically leading to severe pain, lowered physical function, compression of major organs, poor quality of life and disfigurement. These are not malignant but have potential to mutate into malignant peripheral nerve sheath tumours. People with neurofibromatosis 1 that have this outcome can succumb to a shortened lifespan.

Phase 2 trials of Micdametinib in both adults and adolescents demonstrated good efficacy, well tolerated side effects. In inoperable NF1-PN patients phase 2b clinical trials is showing tolerability, tumor volume reductions and fast onset, sustained pain relief with higher quality of life in people of 2 years and older.

MEK inhibitors bring promise to the future treatment of lung cancer, melanoma, pancreatic cancer, endometrial cancer, ovarian cancer and colorectal cancer. Any cancers impacted by the MAPK pathway.

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