MSK-DA01 Cell Therapy for Parkinson's Disease

No longer recruiting at 2 trial locations

Overseen By Jeffrey Lickteig

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: BlueRock Therapeutics

Must be taking: Levodopa

Must not be taking: Anti-coagulants

Disqualifiers: Epilepsy, Stroke, Alzheimer's, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new cell therapy, MSK-DA01, to determine its safety for people with Parkinson's disease. Researchers will inject dopamine-producing nerve cells directly into the brain and monitor for side effects. It targets individuals diagnosed with Parkinson's 3 to 20 years ago who experience issues with their current medication, such as wearing off or dyskinesia (uncontrolled movements). Participants must attend all study visits and have a study partner to assist with ongoing consent. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must be able to temporarily stop anti-platelet or anti-coagulant medications without serious risk.

What prior data suggests that MSK-DA01 and the MSK-DA01 Cell Delivery Device are safe?

Research shows that the treatment MSK-DA01, which involves injecting dopamine-producing cells into the brain, has demonstrated promising safety results in early studies. In previous research, these cells remained in the brain and did not spread to other parts of the body, indicating a positive safety profile.

Additionally, earlier studies found no major toxic effects or serious side effects from the treatment, suggesting that patients in those studies tolerated it well. It's important to note that this clinical trial is in the early stages, with the primary goal of learning more about its safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Parkinson's disease?

Unlike the standard treatments for Parkinson's Disease, which often focus on managing symptoms through medications like Levodopa or dopamine agonists, MSK-DA01 offers a novel approach by using cell therapy. This treatment is unique because it involves using specially developed cells to potentially replace the dopamine-producing neurons that are damaged in Parkinson's. Researchers are excited because this innovative strategy could address the root cause of the disease rather than just alleviating symptoms, offering hope for more effective, long-term relief.

What evidence suggests that MSK-DA01 Cell Therapy might be an effective treatment for Parkinson's disease?

Research shows that MSK-DA01, a cell therapy for Parkinson's disease, looks promising in early tests. Previous studies have demonstrated that similar treatments can improve or maintain patients' mobility. These therapies inject dopamine-producing cells into the brain to replace those lost due to Parkinson's disease. In early lab tests, these cells developed into the type needed to potentially alleviate symptoms. Initial tests found the treatment to be safe and well-tolerated. Overall, early findings suggest that MSK-DA01 could enhance movement and overall function in people with Parkinson's. ^{2 4 6 7 8}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Able to participate in all study visits and evaluations, including brain MRI and PET scan

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

I am between 50 and 78 years old.

Exclusion Criteria

You weigh more than 350 pounds or have a condition that makes it difficult to have a PET/MRI scan.

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgical Transplantation

Subjects undergo surgical transplantation of dopamine-producing cells into the putamen under general anesthesia

1 day

1 visit (in-person)

Immunosuppression Treatment

Subjects take medicines to partially suppress their immune system to prevent cell rejection

1 year

Follow-up

Participants are monitored for safety, tolerability, and evidence of cell survival using MRI and PET scans, and effect on Parkinson's disease symptoms

2 years

What Are the Treatments Tested in This Trial?

Interventions

• MSK-DA01
• MSK-DA01 Cell Delivery Device

Trial Overview 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.

How Is the Trial Designed?

Group I: MSK-DA01 Experimental Treatment 2 Interventions

Who Is Running the Clinical Trial?

BlueRock Therapeutics

Lead Sponsor

Trials

4

Recruited

270+

Memorial Sloan Kettering Cancer Center

Collaborator

Trials

1,998

Recruited

602,000+

Published Research Related to This Trial

Cell replacement therapy (CRT) shows promise for long-term motor improvements in Parkinson's disease by potentially preventing or slowing neurodegeneration and increasing the number of surviving dopaminergic neurons, as confirmed by imaging techniques.

Various cell sources, including embryonic stem cells and induced pluripotent stem cells, offer potential for effective dopaminergic neuron replacement, but ethical concerns and complications like graft-induced dyskinesias limit the use of some sources like fetal ventral midbrain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current Developments in Cell Replacement Therapy for Parkinson's Disease. Guo, X., Tang, L., Tang, X.[2021]

The STEM-PD phase I/IIa clinical trial for Parkinson's disease has shown promising safety and efficacy, with no adverse effects reported in a 39-week rat safety study, indicating the product's potential for safe use in humans.

In pre-clinical studies, the transplanted pluripotent stem cell-derived dopaminergic neurons led to full functional recovery in a rat model of Parkinson's disease, and the product demonstrated consistent efficacy across different manufacturing batches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson's disease, STEM-PD. Kirkeby, A., Nelander, J., Hoban, DB., et al.[2023]

Dopaminergic progenitors (DAPs) derived from a clinical-grade human iPSC line were found to be safe, showing no tumorigenicity or toxicity in pre-clinical studies with immunodeficient mice.

Transplanting these DAPs into the striatum of rats with induced Parkinson's disease led to significant behavioral improvements, supporting their potential efficacy as a treatment for Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor cells for Parkinson's disease. Doi, D., Magotani, H., Kikuchi, T., et al.[2022]

Stem cell-derived dopamine neuron transplantation shows promise as a potential curative therapy for Parkinson's disease, addressing the loss of dopaminergic neurons and aiming to restore dopamine levels in the brain.

Factors such as the source and quality of stem cells, patient age, disease progression stage, surgical techniques, and immunosuppression practices significantly influence the outcomes of cell transplantation, highlighting the need for careful consideration in clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current Status of Stem Cell-Derived Therapies for Parkinson's Disease: From Cell Assessment and Imaging Modalities to Clinical Trials. Jang, SE., Qiu, L., Chan, LL., et al.[2020]

Citations

1. nature.com nature.com/articles/s41586-025-08845-y

Phase I trial of hES cell-derived dopaminergic neurons for ... Secondary and exploratory clinical outcomes showed improvement or stability, including improvement in the Movement Disorder Society Unified ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04802733

Phase 1 Safety and Tolerability Study of MSK-DA01 Cell ... This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and ...

3. bluerocktx.com bluerocktx.com/bluerocks-phase-i-study-with-bemdaneprocel-in-patients-with-parkinsons-disease-meets-primary-endpoint/

BlueRock's Phase I study with bemdaneprocel in patients ... Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson's disease.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7903922/

Preclinical Efficacy and Safety of a Human Embryonic Stem ... Data on thawed cells from MSK-DA01 showed a high percentage (90.1–94.4%) of FOXA2, a floor plate marker that is maintained throughout the development of mDA ...

5. apdaparkinson.org apdaparkinson.org/article/understanding-stem-cell-therapy-in-parkinsons-disease-treatment/

Stem Cell Therapy in Parkinson's Disease | APDA The study demonstrated that the treatment was safe and well-tolerated. Transplantation of the cells was feasible and resulted in successful cell ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04802733

Phase 1 Safety and Tolerability Study of MSK-DA01 Cell ... This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S1934590921000047

Preclinical Efficacy and Safety of a Human Embryonic Stem ... These data suggest that the grafted MSK-DA01 cells were confined to the brain and rarely trafficked to areas outside the brain, including the ...

8. cell.com cell.com/cell/fulltext/S0092-8674(25)01041-4

Phase 1/2a clinical trial of hESC-derived dopamine ... No dose-limiting toxicities or graft-related adverse events were observed. At 12 months, off-medication Movement Disorder Society Unified ...