{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T07:02:32Z","timestamp":1791270152792,"version":"4.1.0"},"reference-count":48,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2016,6,3]],"date-time":"2016-06-03T00:00:00Z","timestamp":1464912000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Parkinson\u2019s Disease"],"published-print":{"date-parts":[[2016,6,3]]},"abstract":"<jats:p>Background: We evaluated the effects of low doses of the tyrosine kinase Abelson (Abl) inhibitor Nilotinib, on safety and pharmacokinetics in Parkinson\u2019s disease dementia or dementia with Lewy bodies.<\/jats:p>\n                  <jats:p>Objectives: The primary outcomes of this study were safety and tolerability; pharmacokinetics and target engagement were secondary, while clinical outcomes were exploratory.<\/jats:p>\n                  <jats:p>Methods: Twelve subjects were randomized into 150\u200amg ( n\u200a=\u200a5) or 300\u200amg ( n\u200a=\u200a7) groups and received Nilotinib orally every day for 24 weeks.<\/jats:p>\n                  <jats:p>Results: This study shows that 150\u200amg and 300\u200amg doses of Nilotinib appear to be safe and tolerated in subjects with advanced Parkinson\u2019s disease. Nilotinib is detectable in the cerebrospinal fluid (CSF) and seems to engage the target Abl. Motor and cognitive outcomes suggest a possible beneficial effect on clinical outcomes. The CSF levels of homovanillic acid are significantly increased between baseline and 24 weeks of treatment. Exploratory CSF biomarkers were measured.<\/jats:p>\n                  <jats:p>Conclusions: This small proof-of-concept study lacks a placebo group and participants were not homogenous, resulting in baseline differences between and within groups. This limits the interpretations of the biomarker and clinical data, and any conclusions should be drawn cautiously. Nonetheless, the collective observations suggest that it is warranted to evaluate the safety and efficacy of Nilotinib in larger randomized, double-blind, placebo-controlled trials.<\/jats:p>","DOI":"10.3233\/jpd-160867","type":"journal-article","created":{"date-parts":[[2016,7,12]],"date-time":"2016-07-12T12:58:28Z","timestamp":1468328308000},"page":"503-517","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":222,"title":["Nilotinib Effects in Parkinson\u2019s disease and Dementia with Lewy bodies"],"prefix":"10.1177","volume":"6","author":[{"given":"Fernando","family":"Pagan","sequence":"first","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michaeline","family":"Hebron","sequence":"additional","affiliation":[{"name":"Department of Neurology, Laboratory for Dementia and Parkinsonism, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Georgetown University Medical Center, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellen H.","family":"Valadez","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasar","family":"Torres-Yaghi","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xu","family":"Huang","sequence":"additional","affiliation":[{"name":"Department of Neurology, Laboratory for Dementia and Parkinsonism, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Georgetown University Medical Center, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reversa R.","family":"Mills","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara M.","family":"Wilmarth","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hellen","family":"Howard","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Connell","family":"Dunn","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexis","family":"Carlson","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Abigail","family":"Lawler","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. MedStar Georgetown Hospital Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean L.","family":"Rogers","sequence":"additional","affiliation":[{"name":"Department of Neurology, National Parkinson\u2019s Foundation Center for Excellence, Translational Neurotherapeutics Program. Movement Disorders Program. 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