Dopamine Neuron Transplantation for Parkinson's Disease

Overseen By Penelope Hallett, Ph.D.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Penelope J. Hallett, Ph.D.

Must be taking: Dopamine drugs

Must not be taking: Anticoagulants, Investigational drugs

Disqualifiers: Intracranial surgeries, Psychiatric disorders, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: Baseline to 12 months post-transplant

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for Parkinson's disease by transplanting special dopamine-producing cells into the brain. The goal is to determine if these cells are safe and potentially effective in managing symptoms like tremors and stiffness. The trial suits individuals who have had Parkinson's disease for at least 5 years and respond well to current dopamine medications. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that you cannot be on chronic anticoagulation medication (blood thinners) and should not have poorly controlled blood pressure or diabetes. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that using a person's own cells to create dopamine-producing brain cells holds potential. In tests with monkeys, these cells safely integrated into the brain without causing tumors. A study with humans found that these cells also survived, produced dopamine, and did not lead to tumors. This treatment remains in the early stages, requiring further research to confirm these results in humans. However, findings so far suggest that the treatment is generally safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about autologous iPSC-derived dopamine neuron transplantation for Parkinson's Disease because it offers a groundbreaking approach to treatment. Unlike standard options like Levodopa or deep brain stimulation, which mainly manage symptoms, this therapy aims to address the root cause by replenishing the brain's depleted dopamine neurons. By using the patient's own cells (autologous), the risk of immune rejection is minimized, potentially leading to more effective and long-lasting results. This cell therapy also introduces a regenerative aspect, providing hope for not just symptom relief but also disease modification.

What evidence suggests that autologous iPSC-derived dopamine neuron transplantation might be an effective treatment for Parkinson's disease?

Research shows that using a person's own cells to create new dopamine-producing brain cells could help treat Parkinson's disease. In studies with monkeys, these lab-grown cells blended into the brain and functioned like natural brain cells. Examinations after these studies revealed that the transplanted cells survived well and reached the areas of the brain affected by Parkinson’s. This trial will investigate autologous iPSC-derived dopamine neuron transplantation to replace lost dopamine cells in people with Parkinson’s, potentially improving symptoms by restoring dopamine levels. Although human trials remain in the early stages, research suggests this approach might effectively manage the disease. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals with Parkinson's Disease who are eligible to undergo a surgical procedure where investigational cells will be injected into their brain. Specific eligibility criteria details were not provided.

Inclusion Criteria

No gross abnormalities on MRI, including hydrocephalus or extensive white matter disease

I have been diagnosed with Parkinson's disease for 5 years or more.

No significant untreated depression (Beck Depression Inventory 2)

Exclusion Criteria

My condition is a type of Parkinsonism that is not typical Parkinson's disease.

Inability to have an MRI

Life expectancy < 6 months due to concomitant illnesses

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive autologous iPSC-derived dopamine neuron transplantation into the putamen

Baseline to 12 months post-transplant

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of adverse events and changes in Parkinson's disease symptoms

18 months following transplantation

What Are the Treatments Tested in This Trial?

Interventions

• Autologous iPSC-Derived Dopamine Neuron Transplantation

Trial Overview The study is testing the safety of transplanting dopamine neurons derived from a patient's own induced pluripotent stem cells (iPSCs) into their brain, as a potential treatment for Parkinson's Disease.

How Is the Trial Designed?

Group I: Autologous midbrain dopamine neurons Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Penelope J. Hallett, Ph.D.

Lead Sponsor

Trials

1

Recruited

6+

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials

1,403

Recruited

655,000+

Oryon Cell Therapies

Collaborator

Trials

1

Recruited

6+

Published Research Related to This Trial

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]

Transplanting dopaminergic neurons derived from human pluripotent stem cells shows potential as a treatment for Parkinson's disease.

Recent studies have identified specific markers for midbrain dopaminergic progenitors, which can enhance the process of deriving these neurons and help predict the amount of dopamine-producing neurons after they are transplanted.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Seq-ing Markers of Midbrain Dopamine Neurons. Osborn, T., Hallett, PJ.[2021]

The study demonstrated that autologous induced pluripotent stem cell (iPSC)-derived dopamine neurons can improve behavior in a cynomolgus monkey model of Parkinson's disease, indicating potential efficacy for treating the condition.

No immunosuppression was needed for the grafts, and histological analysis showed a significant number of surviving dopamine neurons without any overgrowth, suggesting a safe approach for cell replacement therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous iPSC-derived dopamine neuron transplantation in a nonhuman primate Parkinson's disease model. Wang, S., Zou, C., Fu, L., et al.[2020]

A groundbreaking study demonstrated the successful transplantation of dopamine neurons derived from a patient's own skin cells into a patient with Parkinson's disease, highlighting the potential for personalized cell therapy.

This approach shows promise for autologous transplantation, meaning the patient's own cells are used, which may reduce the risk of rejection and improve treatment outcomes for dopamine cell replacement.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

From Skin to Brain: A Parkinson's Disease Patient Transplanted with His Own Cells. Parmar, M., Björklund, A.[2021]

Stem cell therapy for Parkinson's disease aims to restore dopamine production and improve synapse formation, with fetal ventral-midbrain cells showing promise in alleviating symptoms.

Induced pluripotent stem cells (iPSCs) can be effectively used to generate dopaminergic neurons for transplantation, providing a safe and efficient alternative donor cell source for treating Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

iPS cell therapy for Parkinson's disease. Takahashi, J.[2017]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4462124/

Successful function of autologous iPSC-derived dopamine ... Post-mortem analyses demonstrated robust survival of midbrain-like dopaminergic neurons and extensive outgrowth into the transplanted putamen. Our proof of ...

2. nature.com nature.com/articles/celldisc201512

Autologous iPSC-derived dopamine neuron ... In this study, we tested the safety and efficacy of autologous induced pluripotent stem cell (iPSC)-derived DA cells for treatment of a cynomolgus monkey PD ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S1353802025005747

Allogenic transplantation therapy of iPS cell-derived ... This is a minireview featuring the transplantation therapy of iPS cell-derived dopamine progenitors for Parkinson's disease.

4. hpscreg.eu hpscreg.eu/browse/trial/169

Autologous iPSC-Derived Dopamine Neuron ... This trial will test whether new dopamine neurons made from blood cells from subjects with Parkinson's disease are safe when surgically injected into the area ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6994130/

Human autologous iPSC–derived dopaminergic progenitors ... While this treatment improves PD patients' quality of life, the effects fade with disease progression and prolonged usage of these medications often (>80%) ...

6. nature.com nature.com/articles/s41586-025-08700-0

Phase I/II trial of iPS-cell-derived dopaminergic cells for ... This trial (jRCT2090220384) demonstrated that allogeneic iPS-cell-derived dopaminergic progenitors survived, produced dopamine and did not form tumours.