{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T02:06:04Z","timestamp":1789610764726,"version":"build-2803163510"},"reference-count":31,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,11,20]],"date-time":"2020-11-20T00:00:00Z","timestamp":1605830400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["movementdisorders.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Movement Disorders"],"published-print":{"date-parts":[[2021,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Nilotinib is US Food and Drug Administration\u2013approved for leukemia, and this open\u2010label study investigated the safety, tolerability, and potential clinical effects of nilotinib in medically optimized patients with Parkinson's disease.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Safety and tolerability were the primary objectives, and clinical outcomes were exploratory.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A total of 63 patients completed a 15\u2010month phase 2, double\u2010blind, placebo\u2010controlled study and were rerandomized 1:1 into an open\u2010label study of nilotinib 150\u2009mg versus 300\u2009mg for 12\u2009months.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Nilotinib was safe and tolerated, and no adverse effects seemed to be related to the drug, and no differences in adverse events were observed between groups. Exploratory clinical outcomes showed that nilotinib 300\u2009mg was remarkably stable from baseline to 27\u2009months using partial and total Unified Parkinson's Disease Scale (UPDRS). Nilotinib 150\u2009mg versus 300\u2009mg, significantly declined using partial or the sum of UPDRS Parts I and II. There was no significant difference in nilotinib 150\u2009mg versus 300\u2009mg using UPDRS Part III (<jats:italic>on<\/jats:italic> levodopa) and total UPDRS Parts I to III. Subgroup analysis showed that late\u2010start nilotinib 150\u2009mg significantly worsened using the sum of UPDRS Parts II\u2009+\u2009III and total UPDRS Parts I to III compared with late\u2010start nilotinib 300\u2009mg. Quality of life using the Parkinson's Disease Questionnaire in nilotinib 150\u2009mg significantly declined between 15 and 27\u2009months compared with nilotinib 300\u2009mg, and there was no change in cognition using the Montreal Cognitive Assessment between groups.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>This study provides evidence that nilotinib is safe and tolerated in Parkinson's disease. The exploratory clinical data will inform an adequately powered larger study to evaluate the efficacy of nilotinib 300\u2009mg in Parkinson's disease. \u00a9 2020 The Authors. <jats:italic>Movement Disorders<\/jats:italic> published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society<\/jats:p><\/jats:sec>","DOI":"10.1002\/mds.28389","type":"journal-article","created":{"date-parts":[[2020,11,20]],"date-time":"2020-11-20T15:40:39Z","timestamp":1605886839000},"page":"740-749","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":58,"title":["Long\u2010Term Safety and Clinical Effects of Nilotinib in Parkinson's Disease"],"prefix":"10.1002","volume":"36","author":[{"given":"Fernando L.","family":"Pagan","sequence":"first","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"},{"name":"Movement Disorders Clinic, Department of Neurology MedStar Georgetown University Hospital  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Wilmarth","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"},{"name":"Movement Disorders Clinic, Department of Neurology MedStar Georgetown University Hospital  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasar","family":"Torres\u2010Yaghi","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"},{"name":"Movement Disorders Clinic, Department of Neurology MedStar Georgetown University Hospital  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaeline L.","family":"Hebron","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanjana","family":"Mulki","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dalila","family":"Ferrante","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Matar","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaeil","family":"Ahn","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, Bioinformatics and Biomathematics Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charbel","family":"Moussa","sequence":"additional","affiliation":[{"name":"Translational Neurotherapeutics Program, Laboratory for Dementia and Parkinsonism, Department of Neurology Georgetown University Medical Center  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,11,20]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jneuroim.2017.07.009"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40268-019-0266-z"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M112.419945"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddt192"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.4172\/2155-9899.1000259"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00109-013-1112-3"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2018.06.005"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.neuroscience.2015.07.070"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1326-9-46"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddaa177"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2008.11.014"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.chembiol.2011.11.001"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1158\/0008-5472.CAN-08-1008"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201707918"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejphar.2008.10.014"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbapap.2009.11.008"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25775"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.3233\/JPD-160867"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2019.4200"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.470"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(02)08029-7"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1931"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.parkreldis.2018.06.034"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-019-02023-x"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1002\/pro.3445"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0176235"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ceb.2009.05.007"},{"key":"e_1_2_12_29_1","doi-asserted-by":"publisher","DOI":"10.1002\/emmm.201302771"},{"key":"e_1_2_12_30_1","doi-asserted-by":"publisher","DOI":"10.2967\/jnumed.118.224667"},{"key":"e_1_2_12_31_1","doi-asserted-by":"publisher","DOI":"10.1111\/jnc.13763"},{"key":"e_1_2_12_32_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002923"}],"container-title":["Movement Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mds.28389","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mds.28389","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/movementdisorders.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mds.28389","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T11:37:13Z","timestamp":1760960233000},"score":1,"resource":{"primary":{"URL":"https:\/\/movementdisorders.onlinelibrary.wiley.com\/doi\/10.1002\/mds.28389"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,11,20]]},"references-count":31,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2021,3]]}},"alternative-id":["10.1002\/mds.28389"],"URL":"https:\/\/doi.org\/10.1002\/mds.28389","archive":["Portico"],"relation":{},"ISSN":["0885-3185","1531-8257"],"issn-type":[{"value":"0885-3185","type":"print"},{"value":"1531-8257","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,11,20]]},"assertion":[{"value":"2020-08-14","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-10-26","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-11-20","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}