Neural Stem Cell Therapy for Stroke

Overseen By Clinical Research Coordinator

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Gary Steinberg, MD, PhD

Must be taking: Tacrolimus

Disqualifiers: Neurological disease, Cancer, Seizures, others

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

Compensation: Varies

Number of Visits: 1

Duration: Single administration

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called NR1, a neural stem cell therapy, for individuals who have experienced a stroke in specific brain areas. The primary goal is to assess its safety and how the body processes it when injected directly into the brain. Participants will receive varying doses to determine the optimal and safest amount. The trial seeks individuals who had a stroke within the last 6 months to 5 years and are willing to take tacrolimus for a short period. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in humans.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, you must be willing to take tacrolimus (Prograf) starting 2 days before and continuing for 2 months after the transplant.

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

Research has shown that neural stem cell therapy, such as the NR1 treatment under study, is generally safe and well-tolerated. In earlier studies, patients who received NR1 for chronic stroke experienced no serious side effects.

Another study on stem cell therapy for stroke patients found that it improved their mobility and was safe. In simpler terms, it was both safe and enhanced their movement over time.

This treatment has been tested in people before, with positive results regarding safety. While this doesn't guarantee the same outcome for everyone, it suggests that neural stem cell therapy like NR1 could be a promising and safe option for those considering participation in such trials. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike standard treatments for stroke, which often focus on clot removal or blood flow restoration, Neural Stem Cell Therapy offers a regenerative approach. This treatment involves injecting neural stem cells directly into the brain, aiming to repair and regenerate damaged brain tissue. Researchers are excited because this method could potentially restore lost neurological function and offer recovery options beyond the acute phase of stroke, something current treatments can't achieve.

What evidence suggests that NR1 might be an effective treatment for stroke?

Research has shown that neural stem cell therapy, such as NR1, may aid stroke recovery. In one study, patients who had a stroke and received neural stem cell transplants demonstrated improved brain and movement function a year later. This suggests that NR1 might repair brain damage caused by a stroke. Other studies indicate that stem cell therapy can enhance long-term recovery for stroke patients. These findings offer hope that NR1 could be an effective treatment for those recovering from a stroke. ^{1 4 5 6 7}

Who Is on the Research Team?

Gary K Steinberg, MD, PhD

Principal Investigator

Professor, Neurosurgery Department

Are You a Good Fit for This Trial?

This trial is for adults aged 18-75 who've had a stroke in specific brain arteries between 6 months to 5 years ago. Participants must understand the study, consent to it, and be willing to take tacrolimus medication before and after receiving stem cell treatment. People with very small or large stroke areas, recent certain types of strokes, major neurological diseases other than stroke, active cancer (except some skin cancers), seizures, or those pregnant/breastfeeding cannot join.

Inclusion Criteria

I had a stroke in a specific part of my brain between 6 months and 5 years ago.

Ability of subject to understand and provide written Informed Consent

I am willing to take tacrolimus before and after my transplant.

Exclusion Criteria

I have had cancer before, but it wasn't skin cancer.

I have had seizures in the past.

Pregnant or breast-feeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intracerebral transplantation of Neural Stem Cells (NR1) at a single time-point post-injury

Single administration

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of treatment-related adverse events and changes in Fugl Meyer motor score

12 months

What Are the Treatments Tested in This Trial?

Interventions

• NR1

Trial Overview

The trial is testing the safety of different doses of Neural Stem Cells (NR1) injected directly into the brain one time post-injury in people with chronic ischemic subcortical strokes. It aims to see how well participants tolerate this potential new treatment.

How Is the Trial Designed?

Group I: Neural Stem cells injected intracerebrally Experimental Treatment 1 Intervention

Subject cohorts will be treated with increasing doses of Neural Stem Cells injected intracerebrally using a traditional 3+3 trial design

Who Is Running the Clinical Trial?

Gary Steinberg, MD, PhD

Lead Sponsor

Trials

1

Recruited

30+

Gary Steinberg

Lead Sponsor

Trials

1

Recruited

30+

California Institute for Regenerative Medicine (CIRM)

Collaborator

Trials

70

Recruited

3,300+

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12181957/

How neural stem cell therapy promotes brain repair after stroke

This review explores the latest advancements in neural stem cell therapy for stroke, highlighting research insights in brain repair mechanisms.

2.

healio.com

healio.com/news/neurology/20250217/neural-stem-cell-transplant-improves-outcomes-for-chronic-ischemic-stroke-at-12-months

Neural stem cell transplant improves outcomes for chronic ...

Transplantation of neural stem cells improved neurologic and motor function for adults with chronic ischemic stroke at 12 months, according to a study.

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04631406

A Safety and Tolerability Study of Neural Stem Cells (NR1) ...

The study will be a dose escalation using up to 4 cohorts each at a single dose level, of intracerebral administration of NR1 cells.

4.

nature.com

nature.com/articles/s41598-025-04405-6

Efficacy and safety of stem cell therapy for acute and ...

Stem cell therapy for acute/subacute ischemic stroke within 1 month of onset is safe and significantly improves long-term functional outcomes, ...

5.

bmcneurol.biomedcentral.com

bmcneurol.biomedcentral.com/articles/10.1186/s12883-025-04246-w

The efficacy and safety of stem cell therapy for ischemic stroke

A total of nineteen papers had complete outcome data, no selective reporting of results, and other risks of bias were unclear. Seven papers ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11943215/

Safety and Efficacy of Stem Cell Therapy in Ischemic Stroke

Conclusions: SCT offers a safe and effective approach to improving functional outcomes in stroke patients, particularly with early intervention.

7.

ahajournals.org

ahajournals.org/doi/10.1161/str.56.suppl_1.26

Abstract 26: First-in-human Phase 1/2a Study of ...

Intraparenchymal transplantation of NR1 cells in chronic stroke patients appears safe and well tolerated. Results suggest improved motor function starting at 1 ...