Autologous Mesenchymal Stem Cell Therapy in Progressive Supranuclear Palsy: a Randomized, Double-blind, Controlled Clinical Trial

Date de début de l'étude : 1 décembre 2012

Inclusion Criteria: diagnosis of 'probable Progressive Supranuclear Palsy - Richardson's disease subtype' according to current diagnostic criteria (Litvan et al. 1996 and 2003) age at onset at least 40 years; disease duration 12 months to 8 years; supranuclear ophthalmoplegia; postural instability or falls within 3 years from disease onset positive MRI for PSP criteria (Quattrone et al, 2008) Stable pharmacological treatment for at least 90 days Lack of response to chronic levodopa (at least 12-month treatment). Able to stand in upright posture without assistance for at least 30 seconds Written informed consent (including video taping) Exclusion Criteria: Idiopathic Parkinson's disease; Cerebellar ataxia Symptomatic autonomic dysfunction Evidence of any other neurological disease that could explain signs; History of repeated strokes with stepwise progression of parkinsonian features; History of major stroke; Any history of severe or repeated head injury; A history of encephalitis; A history of neuroleptic use for a prolonged period of time or within the past 6 months; Street-drug related parkinsonism; Significant other neurological disease on CT-scan/MRI; Oculogyric crises; Major signs of corticobasal degeneration; Signs of Lewy body disease; Other life-threatening disease likely to interfere with the main outcome measure; Any clinically significant laboratory abnormality, with the exception of cholesterol, triglycerides and glucose; Renal failure (serum creatinine more than 300 mM/l); Transaminase elevation more than twice the upper limit of normal; Any concomitant disorder associated with bone marrow function impairment Any concomitant disorder that requires chronic treatment with immunosuppressors, anti-inflammatory agents, and/or growth factors dementia (MMSE < 24 according to Folstein 1975 or defined according to DSM-IV TR criteria) any other disorder that could interfere with the evaluation of treatment or that could make intra-arterial infusion inadvisable any other features that, according to the investigator, could reduce adherence to protocol procedures or prevent rapid access in case of emergency; women of child-bearing age participation in another clinical trial with experimental treatment in the last 30 days brain MRI evidence of severe vascular abnormalities, space-occupying lesions or normal pressure hydrocephalus