Stem Cell Therapy for Parkinson's Disease

(CT1-DAP001 Trial)

Overseen By Christian Fulinara

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of California, San Diego

Must not be taking: Antiparkinsonian drugs

Disqualifiers: Dementia, Epilepsy, Diabetes, others

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

Compensation: Varies

Number of Visits: 5

7 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment using special stem cells to help people with Parkinson's disease, a condition that affects movement. The goal is to determine if these stem cells can safely improve symptoms by being transplanted into a part of the brain involved in movement control. This trial is open to individuals who have had Parkinson’s for at least 5 years and find that their current medications aren't effective enough. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that you should not have hypersensitivity or contraindications to certain drugs like tacrolimus, levodopa, and carbidopa, which might suggest that some medications could be continued. Please consult with the trial team for specific guidance.

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

In a previous study, researchers found that special cells capable of producing dopamine were created from human stem cells. These cells did not cause tumors and survived well after transplantation. They also produced dopamine, which is crucial for individuals with Parkinson's disease. Another study showed that these cells did not cause any negative effects in mice over nine months.

Currently, the treatment undergoes testing in humans to assess its safety. As this trial is in its early stages, the primary goal is to identify any safety issues. Researchers are closely monitoring for potential problems.

Overall, these early studies suggest that the treatment is potentially safe, but further research is needed to confirm this. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Parkinson's disease, which often involve medications like Levodopa to manage symptoms, CT1-DAP001 introduces a groundbreaking approach by using human induced pluripotent stem cell-derived dopaminergic progenitors. These cells aim to directly replace the lost dopamine-producing neurons in the brain, potentially addressing the root cause of the disease rather than just alleviating symptoms. Researchers are excited because this treatment could lead to more sustained improvements in motor function and possibly slow down the progression of Parkinson’s disease, offering hope for a fundamentally different and more effective treatment strategy.

What evidence suggests that this treatment might be an effective treatment for Parkinson's disease?

Research has shown that a new treatment using special cells, called CT1-DAP001, may help people with Parkinson's disease. In a study, researchers transplanted these cells into the brain, where they survived, produced dopamine (a chemical that helps control movement), and did not cause tumors. This is important because people with Parkinson's often have low dopamine levels. These findings suggest that CT1-DAP001 could help improve movement problems by increasing dopamine in the brain. While more research is needed, early results are promising for people with Parkinson's disease. ^{1 2 3 5 6}

Who Is on the Research Team?

Joseph Ciacci, MD

Principal Investigator

University of California, San Diego

Are You a Good Fit for This Trial?

This trial is for individuals with Parkinson's Disease who are eligible to undergo a procedure where new cells are implanted into the brain. Specific eligibility criteria details were not provided.

Inclusion Criteria

I do not have uncontrolled movements.

My Parkinson's disease is at least stage 2.5, showing more symptoms when medication wears off.

My current medication isn't working for my condition.

Exclusion Criteria

My brain MRI shows signs of a condition that is not Parkinson's disease.

My immune system does not work properly.

Subject is pregnant or lactating or does not agree to avoid pregnancy throughout the study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transplantation

Transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors into the corpus striatum

Immediate

1 visit (in-person)

Post-Transplantation Monitoring

Participants are monitored for safety and efficacy, including adverse events and graft expansion

24 months

Regular visits at 4 weeks, 12 weeks, 6 months, 12 months, 18 months, and 24 months

Follow-up

Participants are monitored for long-term safety and effectiveness after the main monitoring period

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001)

Trial Overview The study tests transplanting special cells derived from human stem cells (CT1-DAP001) into a part of the brain affected by Parkinson's, aiming to assess safety and potential improvement in symptoms.

How Is the Trial Designed?

Group I: Single-center, open-label, uncontrolled Experimental Treatment 1 Intervention

To evaluate the safety and efficacy of transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors, CT1-DAP001, into the corpus striatum in patients with Parkinson's disease

Who Is Running the Clinical Trial?

University of California, San Diego

Lead Sponsor

Trials

1,215

Recruited

1,593,000+

Kyoto University

Collaborator

Trials

38

Recruited

26,500+

Sumitomo Pharma Co., Ltd.

Industry Sponsor

Trials

29

Recruited

5,100+

CiRA Foundation

Collaborator

Trials

1

Recruited

7+

Sumitomo Pharma America, Inc.

Industry Sponsor

Trials

244

Recruited

51,500+

Jatin Shah

Sumitomo Pharma America, Inc.

Chief Medical Officer since 2024

MD from an unspecified institution

Tsutomu Nakagawa

Sumitomo Pharma America, Inc.

Chief Executive Officer since 2024

MBA from Waseda University

Published Research Related to This Trial

A new method for creating human induced pluripotent stem cells (hiPSCs) has been developed, which ensures high quality and safety for use in treating Parkinson's disease by generating midbrain dopamine (mDA) progenitors.

Transplanting these mDA cells into rodent models of Parkinson's disease significantly restored motor function without causing tumor formation, indicating a promising approach for personalized autologous cell therapy in humans.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human autologous iPSC-derived dopaminergic progenitors restore motor function in Parkinson's disease models. Song, B., Cha, Y., Ko, S., et al.[2020]

Induced pluripotent stem cells (iPSCs) derived from idiopathic Parkinson's disease (PD) patients do not show significant vulnerability compared to those from healthy individuals, indicating their potential for safe use in cell transplantation therapy.

PDiPSC-derived dopaminergic neurons successfully survived and functioned in PD model rats without causing pathological α-Synuclein accumulation, suggesting they could be effective for autologous transplantation in treating PD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Idiopathic Parkinson's disease patient-derived induced pluripotent stem cells function as midbrain dopaminergic neurons in rodent brains. Kikuchi, T., Morizane, A., Doi, D., et al.[2018]

Human pluripotent stem cell (hPSC)-derived dopamine neurons show promise as a potential therapy for Parkinson's disease, offering a new regenerative approach to address the lack of effective treatments for disease progression.

Clinical-grade midbrain dopamine neuron precursors have been developed under strict manufacturing conditions and are moving towards clinical testing, indicating significant progress in translating hPSC technology into potential therapies for patients with Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pluripotent Stem Cell Therapies for Parkinson Disease: Present Challenges and Future Opportunities. Kim, TW., Koo, SY., Studer, L.[2021]

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 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]

Human induced pluripotent stem cells (iPSCs) can be effectively expanded and differentiated into functional dopaminergic neurons using a defined, xeno-free culture system, showing similar growth and pluripotency to human embryonic stem cells (hESCs).

Transplantation of iPSC-derived dopaminergic neurons into rats with Parkinson's disease improved behavioral deficits, indicating their potential efficacy for treating neurological disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions. Swistowski, A., Peng, J., Liu, Q., et al.[2021]

Induced pluripotent stem (iPS) cells derived from Parkinson's disease patients can be successfully differentiated into dopaminergic neurons, which survive and function well when transplanted into rodent models, showing potential for treating PD.

Transplantation of these patient-derived dopaminergic neurons resulted in significant functional improvements in an animal model of PD, indicating their potential therapeutic efficacy, although further development is needed to enhance their growth and integration in the host brain.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Differentiated Parkinson patient-derived induced pluripotent stem cells grow in the adult rodent brain and reduce motor asymmetry in Parkinsonian rats. Hargus, G., Cooper, O., Deleidi, M., et al.[2022]

A new human induced pluripotent stem cell (iPSC) line, named UNAMi001-A, was created from the skin cells of a Mexican patient with sporadic Parkinson's disease, marking the first iPSC line derived from a Mexican individual.

This iPSC line shows normal characteristics and can differentiate into various cell types, making it a valuable tool for studying the mechanisms of dopaminergic neuron loss in Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Generation of a human induced pluripotent stem cell line (UNAMi001-A) from a Mexican patient with sporadic Parkinson's disease. Flores-Ponce, X., López-Ornelas, A., Escobedo-Avila, I., et al.[2023]

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]

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]

Stem cell therapy for Parkinson's disease aims to restore dopamine production and improve synapse formation, with both embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) showing promise as donor cell sources.

Research has successfully generated dopaminergic neurons from ESCs and iPSCs, and these cells have demonstrated functionality in animal models of Parkinson's disease, paving the way for potential safe and effective transplantation in humans.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Cell replacement therapy for Parkinson's disease using iPS cells]. Takahashi, J.[2019]

Citations

1. 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.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06482268

Study Details | NCT06482268 | Transplantation of Human ... The primary objective of this study is to evaluate the safety of CT1-DAP001 in subjects with Parkinson's disease by determining the incidence and severity of ...

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

Phase 1/2a clinical trial of hESC-derived dopamine ... We generated high-purity dopaminergic progenitors (A9-DPCs) from human embryonic stem cells and evaluated their safety and exploratory efficacy ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7794047/

iPS cell-based therapy for Parkinson's disease: A Kyoto trial We performed a pre-clinical study using a clinical-grade iPS cell line and finally started a clinical trial to treat Parkinson's disease patients in August ...

5. sumitomo-pharma.com sumitomo-pharma.com/news/20240328.html

Initiation of Company-sponsored Clinical Study on iPS Cell ... Initiation of Company-sponsored Clinical Study on iPS Cell-derived Dopaminergic Progenitor Cells for Parkinson's Disease in the United States.

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1934590925000062

Pre-clinical safety and efficacy of human induced ... Clinical-grade hiPSCs were successfully generated from four PD patients · These hiPSC-derived mDA cells showed no adverse effects in mice up to 9 ...