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Targeted molecular therapy for untreatable NF1 tumors

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 Targeted molecular therapy for untreatable NF1 tumors

A targeted molecular therapy has been used successfully by researchers from Cincinnati Children’s Hospital Medical Center to treat nerve tumours that appear in people with neurofibromatosis type 1. The results of this study were published in the Journal of Clinical Investigation.

Nancy Ratner, PhD, principal investigator and the program leader for the Cancer Biology and Neural tumors Program, said it is the first time researchers could reduce the size of neurofibromas with a molecularly targeted therapy. She added that so far there was no treatment for neurofibromas. Surgery may be a solution to remove neurofibromas that compress organs but resection is sometimes difficult.

 NF1 tumors

NF1 tumors

Neurofibromatosis type 1 is a genetic disorder that is manifested by hyperpigmented skin spots and tumors of the nervous system. Patients with neurofibromatosis type 1 have cafe au lait spots, neurofibromas, speech disorders, epilepsy, optic nerve glioma, bone disorders and others. The disease is progressive, some signs appear at birth while others appear with age. Complications of this disease are chronic pain due neurofibroamas that compress nerves, blindness, chronic hypertension, and other neurological symptoms that occur due to brain tumors.

Neurofibromas are benign tumors localized on peripheral nerves and occur in approximately half of patients with neurofibromatosis. Although initially not cause major symptoms, however there are cases where neurofibromas increase dramatically and cause compression of the surrounding organs. There is a possibility that they turn into malignant tumors and become the cause of death of these people. Mutations of the NF1 are also found in other cancers such as glioblastoma, lung adenocarcinoma and ovarian cancer.

Researchers tested an experimental drug called PD0325901, which inhibits the protein MEK, and it was proved to reduce tumors sizes in neurofibromatosis in 80% of treated mice. MEK is part of a signaling pathway that promotes cell growth and it is involved in tumour formation. PD0325901 is now in clinical investigations in humans to treat cancer.

Researchers found that the drug can reduce tumor size in just 60 days. In addition, it was also showed that PD0325901 can reduce the size of malignant tumors in mice implanted with human MPNSTs (malignant peripheral nerve sheath tumors) and doubled the length of survival, that is to 52 days.  However, the researchers stressed that the size reduction  of malignant tumors compared with benign tumors is modest,  and that it is likely that other signaling pathways are involved. It is possible that in the future PD0325901 to be used in combination with other drugs to treat cancer.