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Ruxolitinib, a Jak 1/2 inhibitor, significantly reduced tumorigenesis in a PJS mouse model. Based on the Inventors' preclinical results, they expect Ruxolitinib to also reduce tumorigenesis in PJS patients. Other Jak inhibitors such as Itacitinib are also being engaged to reduce or stop polyposis and tumorigenesis.
Rapamycin (Sirolimus), an mTOR inhibitor, is a promising candidate in treatment and prevention of PJS polyps. PJS is caused by mutation of LKB1 gene which leads to increased activity of mTOR pathway.
Combo approach. Use the identified FDA approved compounds with Rapa and the mutation in cell lines and or animal models for treatment and repurposing.
Polyps are reduced in Ruxolitinib treated LKB1 knockout mice.
Gene therapy and editing.
Repair mutated LKB1/STK11 tumor suppressor gene.
Utilize mRNA approach. Intestine delivery pill identified. Develop a vaccine to recognize the mutation and stop formation of polyps. Checkpoint immunotherapy may also be considered as a combo therapy.
Stem cell therapy. Infuse corrected or donated stem cells to be proliferated as healthy T cells to supplement protein and correct deficiencies.
Liquid Biopsy.
Lessen the burden of ongoing intensive screening and risks for patients.
Germline mutations of the LKB1 tumor suppressor gene result in Peutz–Jeghers Syndrome (PJS) characterized by intestinal hamartomas and increased incidence of epithelial cancers.
LKB1 is commonly thought of as a tumor suppressor gene because its hereditary mutation is responsible for cancer syndrome, and somatic inactivation of LKB1 is found in non-small cell lung cancer, melanoma, and cervical cancers.
Increasing evidence has highlighted that deficiency of LKB1 in cancer cells induces extensive metabolic alterations that promote tumorigenesis and development.
Physiologically, LKB1 possesses multiple cellular functions in the regulation of cell bioenergetics metabolism, cell cycle arrest, embryo development, cell polarity, and apoptosis.
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