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

Trial Summary

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.

What data supports the effectiveness of the treatment Human induced pluripotent stem cell-derived dopaminergic progenitors (CT1-DAP001) for Parkinson's Disease?

Research shows that dopaminergic progenitor cells derived from human induced pluripotent stem cells can improve motor function in animal models of Parkinson's Disease. These cells have been shown to survive, integrate into the brain, and produce dopamine, which is crucial for motor control, without causing tumors. ^{1 2 3 4 5}

Is stem cell therapy for Parkinson's disease safe for humans?

Pre-clinical studies show that stem cell therapies for Parkinson's disease, like those using dopaminergic progenitors from induced pluripotent stem cells, have not shown any signs of causing tumors or toxicity in animal models. These findings suggest that the treatment is generally safe, as no adverse effects were observed in long-term animal studies. ^{1 4 6 7 8}

How is the treatment CT1-DAP001 unique for Parkinson's disease?

CT1-DAP001 is unique because it uses human induced pluripotent stem cell-derived dopaminergic progenitors to potentially restore dopamine production in the brain, which is different from traditional treatments that mainly focus on managing symptoms rather than addressing the underlying cause. ^{4 9 10 11 12}

What is the purpose of this trial?

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

Research Team

Joseph Ciacci, MD

Principal Investigator

University of California, San Diego

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

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]

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]

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]

References

1. United States 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. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Efficient generation of functional dopaminergic neurons from human induced pluripotent stem cells under defined conditions. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Pluripotent Stem Cell Therapies for Parkinson Disease: Present Challenges and Future Opportunities. [2021]

4. England pubmed.ncbi.nlm.nih.gov

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

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

Human autologous iPSC-derived dopaminergic progenitors restore motor function in Parkinson's disease models. [2020]

6. England 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. [2023]

7. 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]

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

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

9. Japan pubmed.ncbi.nlm.nih.gov

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

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

Seq-ing Markers of Midbrain Dopamine Neurons. [2021]

11. Japan pubmed.ncbi.nlm.nih.gov

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

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

Idiopathic Parkinson's disease patient-derived induced pluripotent stem cells function as midbrain dopaminergic neurons in rodent brains. [2018]