MSK-DA01 Cell Therapy for Parkinson's Disease

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

12 Participants Needed

Trial Summary

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 data supports the effectiveness of the MSK-DA01 treatment for Parkinson's Disease?

Research shows that transplanting dopamine-producing cells into the brain can help restore function in animal models of Parkinson's Disease, and early clinical trials have shown some positive effects in humans. However, more studies are needed to confirm long-term safety and effectiveness. ^{1 2 3 4 5}

What safety data exists for MSK-DA01 Cell Therapy for Parkinson's Disease?

Pre-clinical studies of similar cell therapies for Parkinson's disease, like those using dopaminergic progenitor cells derived from stem cells, have shown no tumor formation or toxicity in animal models, suggesting they are generally safe for further clinical trials. ^{6 7 8 9 10}

How is the MSK-DA01 treatment different from other Parkinson's disease treatments?

MSK-DA01 is unique because it uses midbrain dopamine neurons derived from human embryonic stem cells to replace lost neurons in Parkinson's disease, aiming to restore dopamine levels and improve motor function, unlike conventional treatments that mainly manage symptoms without addressing neuron loss. ^{11 12 13 14 15}

What is the purpose of this trial?

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.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

References

1. Switzerland 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. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Gene transfer of trophic factors and stem cell grafting as treatments for Parkinson's disease. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Cell therapeutics in Parkinson's disease. [2021]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Assessing the Efficacy of Cell Transplantation for Parkinson's Disease: A Patient-Centered Approach. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Cellular replacement therapy for Parkinson's disease--where we are today? [2017]

6. England pubmed.ncbi.nlm.nih.gov

Pre-clinical study of induced pluripotent stem cell-derived dopaminergic progenitor cells for Parkinson's disease. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Preclinical and dose-ranging assessment of hESC-derived dopaminergic progenitors for a clinical trial on Parkinson's disease. [2023]

8. England pubmed.ncbi.nlm.nih.gov

Towards translational therapies for multiple system atrophy. [2022]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Mitomycin-treated undifferentiated embryonic stem cells as a safe and effective therapeutic strategy in a mouse model of Parkinson's disease. [2020]

10. United States 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. [2023]

11. United States pubmed.ncbi.nlm.nih.gov

Using human pluripotent stem cell-derived dopaminergic neurons to evaluate candidate Parkinson's disease therapeutic agents in MPP+ and rotenone models. [2013]

12. United States pubmed.ncbi.nlm.nih.gov

Current Developments in Cell Replacement Therapy for Parkinson's Disease. [2021]

13. England pubmed.ncbi.nlm.nih.gov

Purification of functional human ES and iPSC-derived midbrain dopaminergic progenitors using LRTM1. [2021]

14. India pubmed.ncbi.nlm.nih.gov

One-step cell biomanufacturing platform: porous gelatin microcarrier beads promote human embryonic stem cell-derived midbrain dopaminergic progenitor cell differentiation in vitro and survival after transplantation in vivo. [2023]

15. United States pubmed.ncbi.nlm.nih.gov

Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01. [2022]