The application discloses application of FTY720 as a PP2A
phosphatase activator in preparation of a
medicine for treating type I neurofibromatosis. The application first uses FTY720 for treatment of type I neurofibromatosis, and proves that FTY720 significantly inhibits formation of neurofibromas by non-specifically activating PP2A
phosphatase. In-vivo experimental results show that FTY720 as a single
drug can significantly inhibit
tumor growth, and a synergistic effect is presented when FTY720 is combined with a
MEK inhibitor, and
tumor growth is almost completely inhibited. In-vitro
cell experiments show that FTY720 as a single
drug or in combination with MEKi treatment can inhibit tumor Schwann cells from forming tumor spheres, inhibit
cell proliferation and migration, and induce
tumor cell apoptosis. The application overcomes the
drug resistance problem existing in the prior art
MEK inhibitor, and provides a new
treatment strategy for type I neurofibromatosis. FTY720 is an FDA-
approved drug, and has good safety and drugability, and has high clinical conversion potential.