Treating Parkinson's Disease Through Transplantation of Autologous Stem Cell-Derived Dopaminergic Neurons
Phase I Trial of Autologous Induced Pluripotent Stem Cell-derived Dopaminergic Progenitor Cell Transplantation for Parkinson's Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 45 Years to 80 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Procedures mentioned: MRI scans
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "The study will assess the safety and tolerability of the cell transplant procedure through clinical assessments and neuroimaging (CT, MRI and 18F-DOPA PET) over a 2-year follow-up period."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the safety of surgery to place new cells into the brains of people with Parkinson’s disease. The cells are made from each person’s own skin cells and are meant to develop into cells that make dopamine, a brain chemical. The study is for adults ages 45–80 who meet specific health and symptom requirements.
The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Have Parkinson’s disease that meets the study’s diagnosis rules, and be 45–80 years old.
- Have had movement symptoms for at least 5 years. Symptoms must be stage 3–4 on a Parkinson’s rating scale when medicine is not working.
- Take at least 300 mg of levodopa each day and have at least a 30% improvement in movement scores when medicine is working.
- Have at least 3 hours each day when Parkinson’s medicine is not working well. Parkinson’s medicines must stay the same for at least 4 weeks before screening.
- Understand enough English to discuss the study’s risks and benefits and give consent. Be willing and able to attend all visits and complete study tasks.
- Have blood counts, blood clotting, liver tests, and kidney tests that meet the study’s surgery safety limits.
- Have a brain scan showing loss of dopamine nerve activity in an area called the putamen.
- Agree to delay optional brain surgery and certain treatments delivered by tubes or pumps until the study’s main outcome check is complete.
Who can't join
- Cannot give consent because of dementia or psychosis, or score below 26 on a thinking and memory test. Also excluded: psychosis in the past 12 months, suicidal thoughts with a plan or intent in the past 12 months, or a suicide attempt in the past 5 years.
- Have a parent, sibling, or child with Parkinson’s, a known gene change that raises Parkinson’s risk, or a different condition that causes Parkinson’s-like symptoms.
- Have moderate or severe unwanted movements caused by levodopa, another brain problem that could affect study participation or testing, a past stroke or mini-stroke, bleeding around the brain, or past surgery inside the skull.
- Have had cancer in the past 5 years, except treated basal cell or squamous cell skin cancer that has not spread.
- Take blood-thinning medicines that cannot safely be stopped for 1 week before and 2 days after surgery, or take long-term medicines that weaken the immune system, including steroids.
- Cannot safely have an MRI or its contrast dye, are pregnant or nursing, or have alcohol or drug addiction.
- Have recent active heart or brain blood vessel disease; certain heart failure, chest pain, heart attack, or serious heartbeat problems; past heart or blood vessel surgery; or major blood vessel disease.
- Have blood pressure that stays above 160/100 despite treatment, severe blood pressure drops when standing, or poorly controlled diabetes: A1c above 9% or fasting blood sugar at least 11.1 mmol/L.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * Diagnosis of Parkinson's Disease consistent with the Movement Disorders Society 2015 Parkinson's diagnostic criteria. * Age 45 - 80 years * English proficiency sufficient to understand the consent form and participate in a discussion of risks and benefits * At least 5 years since Parkinson's disease motor symptom onset * Modified Hoehn and Yahr stage 3-4 in "off"-medication state * Motor symptoms responsive to levodopa and/or dopamine agonist, defined as taking at least 300 mg/day of levodopa and exhibiting improvement between "off" and "on" MDS-UPDRS of at least 30% * At least 3 hours of cumulative "off" time per day * Stable regimen of Parkinson's medications, including levodopa and dopamine agonists, for at least 4 weeks prior to screening. * Acceptable surgical laboratory values including: 1. Platelets \> 100×109/L (transfusion independent) 2. Prothrombin time / partial thromboplastin time in normal range and international normalized ratio ≤ 1.3 3. Aspartate aminotransferase and alanine aminotransferase \< 2.5x the upper limit of normal 4. Serum creatinine ≤ 1.5mg/dL 5. White blood cell count \< 12×109/L. 6. Estimated glomerular filtration rate ≥ 30 mL/min/1.73m2 * Subject agrees to defer elective neurological surgery, including deep brain stimulation or lesional procedure for PD, invasive treatments, including levodopa or apomorphine infusion, or pump- pump-administered levodopa intestinal gel, until after the study's primary outcome is completed. * Findings on baseline 18F-DOPA PET imaging consistent with dopaminergic denervation of the putamen * Subject is willing and able to comply with all study visits and procedures in the opinion of the Investigator. Exclusion Criteria: * Subjects unable to give consent due to dementia or psychosis. * Montreal Cognitive Assessment (MoCA) score \< 26 * Subjects with a first-degree relative with Parkinson's disease or with a known genetic mutation predisposing to the development of Parkinson's disease (i.e. this initial study is confined to the more common "sporadic" vs a "genetic" form of the disease). * Atypical Parkinsonism (Parkinson's-Plus syndrome, secondary parkinsonism) * Moderate or severe levodopa-induced dyskinesias in any body segment (such patients were found to be more prone to graft-induced dyskinesias in the fetal tissue studies that are proof of priniciple for this therapy) * Neurologic history or imaging demonstrating brain pathology not directly related to Parkinson's disease that is likely to interfere with study compliance or assessment of Parkinson's related motor disability. * History of stroke or transient ischemic attack * History of subarachnoid hemorrhage * Presence or history of psychosis within 12 months of screening * Suicidal ideation associated with intent or plan in the past 12 months (an answer of "yes" to C-SSRS questions 4 or 5) or with a previous history of suicide attempts in the past 5 years. * History of intracranial surgery including deep brain stimulation, focused ultrasound, stereotactic or radiosurgical lesion therapy * History of malignancy within 5 years. Exceptions will be made for treated cutaneous squamous cell or basal cell carcinoma without evidence of metastasis. * Use of anticoagulation / antiplatelet agents that cannot be stopped for one week in advance of and two days following surgery without significant risk to the subject * Use of chronic immunosuppressive therapy including chronic steroids * Contraindication to MRI or MRI contrast agents * Pregnant or nursing women * Subjects with active cardiovascular and cerebrovascular disease within 6 months prior to signing the informed consent form. * History of severe heart failure (congestive heart failure of New York Heart Association Class II or above or left ventricular ejection fraction \< 35% by any examination method), unstable angina pectoris and myocardial infarction/ * Severe arrhythmia * History of cardiovascular surgery (cardiac, vascular stent surgery, angioplasty) * Patients with major vascular diseases (aortic aneurysm, aortic dissecting aneurysm, internal carotid artery stenosis) * Hypertensive patients with poorly controlled blood pressure (defined as blood pressure consistently above 160/100 mmHg despite treatment with antihypertensive drugs) and patients with severe postural hypotension * Diabetic patients with poorly controlled blood glucose (glycosylated hemoglobin \> 9.0%, or fasting plasma glucose (FPG) ≥ 11.1 mmol/L); * Subjects with alcohol or drug addiction
Study sites (1)
MA
- Massachusetts General Hospital · Boston, MA 02114Jeffrey S Schweitzer, MD, PhD · Study contact · 617-726-1799 · jschweitzer1@mgh.harvard.eduDolores Smith · Study contact · 617-726-1799 · vprathivadi@mgh.harvard.eduMore trials in Boston