# Parkinsons disease scalable iPSC autologous cell therapy

> **NIH NIH U01** · MCLEAN HOSPITAL · 2022 · $164,000

## Abstract

Abstract:
The NINDS CREATE Bio Development Track U01 work will complete IND-enabling studies to progress cell
replacement paradigms into the clinic using induced pluripotent stem cell (iPSC)-derived dopamine (DA)
neurons, and a first-in-man clinical trial for autologous transplantation in Parkinson’s disease (PD). Cell
replacement therapy with midbrain dopamine (mDA) neurons provides cellular and synaptic repair in the
parkinsonian brain, and addresses both the motor symptoms of PD as well as levodopa-induced dyskinesias.
Our previous fetal cell transplantation work shows that in PD patients transplanted mDA neurons remain healthy
and can provide remarkable therapeutic benefit for decades. While fetal cell transplantations are not scalable for
a larger patient population and require immunosuppression, iPSCs are a promising alternative cell source. iPSCs
generated from PD patients can be differentiated into midbrain dopaminergic cells, frozen and used for
autologous transplantation.
The U01 proposal over 5 years consists of milestones within four Specific Aims, that includes a Phase I clinical
trial in human patients with PD. In Specific Aim 1 we will transfer the remaining mDA neuron product quality
control assays for qualification in the cGMP facility, perform FDA-guided quality control of excipients for the
clinical product, and produce mDA neurons to be used in IND-enabling studies. In Specific Aim 2, definitive IND-
enabling studies will be performed to test the safety (tumorigenicity and biodistribution) and efficacy of human
iPSC-derived frozen-thawed mDA neurons in rodents, as well as testing of the planned clinical delivery device
in non-human primates. Specific Aim 3 will include IND package preparation and filing for an Investigator-initiated
Phase I clinical trial, recruitment of patients with PD and generation of autologous cGMP iPSCs and mDA
neurons as well as release criteria testing of the cryopreserved clinical product. Finally, Specific Aim 4 is a first-
in-human clinical Phase I interventional, open-label clinical trial in 6 patients with sporadic PD, to test the safety
and efficacy of autologous transplantation of frozen-thawed mDA neurons.
This highly innovative autologous CMC iPS cell technology U01 proposal for cell replacement clinical trials in PD
patients provides a necessary step and exploration for the development of successful cell therapy for PD and
several neurological disorders.

## Key facts

- **NIH application ID:** 10543901
- **Project number:** 3U01NS109463-03S1
- **Recipient organization:** MCLEAN HOSPITAL
- **Principal Investigator:** PENELOPE Jane HALLETT
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $164,000
- **Award type:** 3
- **Project period:** 2020-01-01 → 2022-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10543901

## Citation

> US National Institutes of Health, RePORTER application 10543901, Parkinsons disease scalable iPSC autologous cell therapy (3U01NS109463-03S1). Retrieved via AI Analytics 2026-06-14 from https://api.ai-analytics.org/grant/nih/10543901. Licensed CC0.

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