What side effects are associated with this type of intervention?

Common treatments for Parkinson's Disease, such as levodopa and dopamine agonists, have several known side effects. Levodopa can cause nausea, somnolence, dizziness, headache, confusion, hallucinations, delusions, agitation, psychosis, and orthostatic hypotension. Dopamine agonists, including pramipexole, ropinirole, and rotigotine, may lead to edema, somnolence, constipation, dizziness, hallucinations, and nausea. These treatments aim to restore dopamine levels but can also result in motor complications like dyskinesia and motor fluctuations over time.

What prior FDA approvals exist?

The FDA has approved several treatments for Parkinson's Disease that aim to restore dopamine levels. Levodopa, often combined with carbidopa, is the most commonly used treatment and helps replenish dopamine. Dopamine agonists such as pramipexole, ropinirole, and rotigotine mimic dopamine's effects in the brain. Additionally, MAO-B inhibitors like selegiline and rasagiline help prevent the breakdown of dopamine. While these treatments manage symptoms by enhancing or mimicking dopamine, they do not involve the transplantation of dopamine-producing cells, which is still under investigation.

What other types of research exist?

Promising novel alternatives to dopamine-producing nerve cell transplantation for Parkinson's Disease patients include: 1) Gradual tapering of dopamine agonists (DAs) with close monitoring and potential increase of levodopa to manage motor symptoms, 2) Cognitive Behavioral Therapy (CBT) for patients with persistent Impulse Control Disorders (ICDs) despite discontinuation of DAs, 3) Use of amantadine, although its efficacy and tolerability vary, 4) Exploration of stem cell therapies, such as induced pluripotent stem cells (iPS) for personalized treatment, and 5) Ongoing clinical trials investigating the safety and efficacy of various stem cell-derived treatments and other innovative pharmacologic agents.

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Cell Therapy

MSK-DA01 Cell Therapy for Parkinson's Disease

Phase 1

Waitlist Available

Research Sponsored by BlueRock Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Taking levodopa, but with complications of therapy such as wearing off and/or dyskinesia

Age 50-78 years old (Canada)

Must not have

Previous or currently active malignant disease within the past 5 years, except basal cell carcinoma or in situ uterine cervical carcinoma that have been treated

Prior Deep Brain Stimulation, lesion therapy, or gene therapy for PD

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to 1 year post-transplant and baseline to 2 years post-transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if surgically placing dopamine-making cells into the brains of Parkinson's patients is safe and effective. The goal is to help manage their symptoms by increasing dopamine levels. In an effort to improve the clinical signs of Parkinson's disease, dopamine cells have been implanted into patients' brains.

Who is the study for?

This trial is for Parkinson's disease patients aged 50-78 in Canada or 60-78 in the US, who've been diagnosed between 3 to 20 years ago and are experiencing complications from levodopa therapy. Participants need a study partner and must be able to undergo MRI and PET scans. Exclusions include other neurodegenerative diseases, past brain surgeries or therapies, high risk conditions for immunosuppressive drugs, inability to stop certain medications, pregnancy/breastfeeding, contraindications to surgery/anesthesia, recent cancer except specific types.

What is being tested?

The trial is testing the safety of MSK-DA01 Cell Therapy for advanced Parkinson's Disease by surgically injecting dopamine-producing nerve cells into the brain. It aims to assess tolerability and monitor side effects that may arise from this novel intervention.

What are the potential side effects?

Potential side effects might include reactions related to surgery or anesthesia risks such as infection or bleeding at the injection site; immune response against implanted cells; changes in motor function due to cell placement; general surgical risks like blood clots.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am on levodopa and experiencing side effects like sudden loss of effect or involuntary movements.

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I am between 50 and 78 years old.

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I am between 60 and 78 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had cancer, except for skin or cervical cancer that was treated, in the last 5 years.

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I have had brain stimulation, lesion, or gene therapy for Parkinson's.

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I cannot stop my blood thinning medications without serious risk.

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I cannot undergo surgery or be given general anesthesia due to health risks.

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I have a condition that makes it risky for me to take drugs that weaken my immune system.

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I have been diagnosed with a condition like epilepsy, MS, Alzheimer's, or another similar neurological disorder.

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I have had surgery or radiation therapy on my brain or spinal cord.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 1 year post-transplant and baseline to 2 years post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to 1 year post-transplant and baseline to 2 years post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 1 year post-transplant and baseline to 2 years post-transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and Tolerability

Secondary study objectives

Changes in Motor Function

Changes in Waking Hours in "Off" State

Continued Safety and Tolerability

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MSK-DA01Experimental Treatment2 Interventions

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

MSK-DA01

2021

Completed Phase 1

~20

MSK-DA01 Cell Delivery Device

2021

Completed Phase 1

~20

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Parkinson's Disease aim to restore or mimic the function of dopamine, a neurotransmitter that is significantly depleted in PD patients. Levodopa, often combined with carbidopa, is a precursor to dopamine and is converted to dopamine in the brain, helping to replenish its levels. Dopamine agonists, such as pramipexole and ropinirole, mimic dopamine by stimulating dopamine receptors directly. MAO-B inhibitors, like selegiline and rasagiline, prevent the breakdown of dopamine, thereby increasing its availability. The trial involving dopamine-producing nerve cell transplantation seeks to restore dopamine levels by directly introducing cells that produce dopamine into the brain. This approach could potentially offer a more sustained and natural restoration of dopamine levels, addressing both motor and non-motor symptoms of PD and improving the quality of life for patients.

Infusion of apomorphine into the dorsocentral striatum produces acute drug-induced recovery from neglect produced by unilateral medial agranular cortex lesions in rats.