Janus kinase inhibitor
Janus kinase inhibitors, also known as JAK inhibitors or jakinibs, are a type of medication that functions by inhibiting the activity of one or more of the Janus kinase family of enzymes, thereby interfering with the JAK-STAT signaling pathway.
These inhibitors have therapeutic application in the treatment of cancer and inflammatory diseases such as rheumatoid arthritis. There is interest in their use for various skin conditions. JAK3 inhibitors are attractive as a possible treatment of various autoimmune diseases since its functions is mainly restricted to lymphocytes. Development for a selective JAK3 inhibitors are ongoing.
Mechanism of action
s play key roles in controlling cell growth and the immune response. Many cytokines function by binding to and activating type I and type II cytokine receptors. These receptors in turn rely on the Janus kinase family of enzymes for signal transduction. Hence drugs that inhibit the activity of these Janus kinases block cytokine signalling.More specifically, Janus kinases phosphorylate activated cytokine receptors. These phosphorylated receptors in turn recruit STAT transcription factors which modulate gene transcription.
The first JAK inhibitor to reach clinical trials was tofacitinib. Tofacitinib is a specific inhibitor of JAK3 thereby blocking the activity of IL-2, IL-4, IL-15 and IL-21. Hence Th2 cell differentiation is blocked and therefore tofacitinib is effective in treating allergic diseases. Tofacitinib to a lesser extent also inhibits JAK1 and JAK2 which in turn blocks IFN-γ and IL-6 signalling and consequently Th1 cell differentiation.
One mechanism is that the blocking of Jak-dependent IL-23 reduces IL-17 and the damage it causes.
Molecule design
Some JAK1 inhibitors are based on a benzimidazole core.Examples
Approved compounds
- Ruxolitinib against JAK1/JAK2, for myelofibrosis and polycythemia vera Approved by the U.S. FDA in November 2011 for myelofibrosis and polycythemia vera, in patients with an inadequate response or intolerance to hydroxyurea.
- Tofacitinib against JAK3 for psoriasis and rheumatoid arthritis. U.S. FDA approved it in November 2012 for rheumatoid arthritis in patients who had an inadequate response or intolerance to methotrexate.
- Oclacitinib — against JAK1 for the control of pruritus associated with allergic dermatitis and the control of atopic dermatitis in dogs at least 12 months of age.
- Baricitinib against JAK1/JAK2 for rheumatoid arthritis.
- Peficitinib mainly inhibits JAK3 and used for treatment of rheumatoid arthritis. Approved for use in Japan in 2019.
- Fedratinib is a JAK2 inhibitor for treatment of primary myelofibrosis, or secondary myelofibrosis against JAK1 for rheumatoid arthritis. Approved by U.S FDA on 16 August 2019.
In clinical trials
- Filgotinib against JAK1 for rheumatoid arthritis and Crohn's disease.
- Cerdulatinib dual SYK/JAK inhibitor for hematological malignancies.
- Gandotinib against JAK2 for myeloproliferative neoplasms.
- Lestaurtinib against JAK2 for acute myeloid leukemia.
- Momelotinib against JAK1 and JAK2 for myeloproliferative disorders and relapsed/refractory metastatic pancreatic cancer.
- Pacritinib against JAK2 for relapsed lymphoma and advanced myeloid malignancies, also myelofibrosis, myeloproliferative neoplasms and myelodysplastic syndrome.
- Abrocitinib against JAK1 for atopic dermatitis and moderate to severe psoriasis. Currently in phase III.
- BMS-986165 against TYK2 for psoriasis, psoriatic arthritis, inflammatory bowel disease and systemic lupus erythematosus. Currently in Phase III trials for psoriasis and Phase II for other conditions.
- Ruxolitinib cream against JAK1/2 for vitiligo and atopic dermatitis. Phase III trials are completed or ongoing.
Experimental drugs/indications
- Cucurbitacin I.
- CHZ868 — a type II JAK2 inhibitor for use in myeloproliferative disorders and chronic myelomonocytic leukemia.
- Tofacitinib for alopecia universalis.
- Topical tofacitinib and ruxolitinib for alopecia.