NCT05306457

Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) Delivered to the Motor Cortex for the Treatment of Amyotrophic Lateral Sclerosis

Active, enrolled Phase 1 Last updated 14 August 2025

What this trial tests

Phase 1 trial testing CNS10-NPC-GDNF in Amyotrophic Lateral Sclerosis in 16 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

8 May 2022

Primary endpoint
1 October 2026

1 October 2026

Quick facts

Lead sponsor	Cedars-Sinai Medical Center
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	8 May 2022
Primary completion	1 October 2026
Estimated completion	1 October 2026
Sites	1 location across United States

Drugs / interventions tested

• CNS10-NPC-GDNF — full drug profile →

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →

Sponsor

Cedars-Sinai Medical Center

Who can join

18 and older, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety, as evaluated by the incidence of Adverse Events and Serious Adverse Events and their relationship to the treatment
  Time frame: 12 months post-operatively
• Safety, as evaluated by changes from baseline in the brain MRI
  Time frame: 12 months post-operatively

Sponsor's own description

The investigator is examining the safety of transplanting cells, that have been engineered to produce a growth factor, into the motor cortex (brain) of patients with Amyotrophic Lateral Sclerosis (ALS). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neural cell. The growth factor is called glial cell line-derived neurotrophic factor, or GDNF. GDNF is a protein that promotes the survival of many types of neural cells. Therefore, the cells are called "CNS10-NPC-GDNF." The investigational treatment has been tested in people by delivering it to the spinal cord. However, it has only been delivered to the motor cortex of animals. In this study, we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the motor cortex (brain) of people.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Advancing cell therapy for neurodegenerative diseases.
   Temple S. · · 2023 · cited 147× · PMID 37084729 · DOI 10.1016/j.stem.2023.03.017
2. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial.
   Baloh RH, Johnson JP, Avalos P, Allred P, et al · · 2022 · cited 108× · PMID 36064599 · DOI 10.1038/s41591-022-01956-3
3. Cellular rejuvenation: molecular mechanisms and potential therapeutic interventions for diseases.
   Ji S, Xiong M, Chen H, Liu Y, et al · · 2023 · cited 89× · PMID 36918530 · DOI 10.1038/s41392-023-01343-5
4. New Insights into Oxidative Stress and Inflammatory Response in Neurodegenerative Diseases.
   Scarian E, Viola C, Dragoni F, Di Gerlando R, et al · · 2024 · cited 61× · PMID 38473944 · DOI 10.3390/ijms25052698
5. Microglia and Astrocytes in Amyotrophic Lateral Sclerosis: Disease-Associated States, Pathological Roles, and Therapeutic Potential.
   You J, Youssef MMM, Santos JR, Lee J, et al · · 2023 · cited 31× · PMID 37887017 · DOI 10.3390/biology12101307
6. Stem cell therapeutics and gene therapy for neurologic disorders.
   Chen KS, Koubek EJ, Sakowski SA, Feldman EL. · · 2024 · cited 26× · PMID 39096590 · DOI 10.1016/j.neurot.2024.e00427
7. Astrocytes: Dissecting Their Diverse Roles in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia.
   Valori CF, Sulmona C, Brambilla L, Rossi D. · · 2023 · cited 21× · PMID 37296571 · DOI 10.3390/cells12111450
8. Neural Cells for Neurodegenerative Diseases in Clinical Trials.
   Fan Y, Goh ELK, Chan JKY. · · 2023 · cited 17× · PMID 37487111 · DOI 10.1093/stcltm/szad041