Transplanting engineered stem cells into the brain for ALS treatment
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
This study is testing if transplanting specially modified stem cells into the brains of people with ALS is safe and could help improve their condition.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 16 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Cedars-Sinai Medical Center Academic / other |
| Locations | 1 site (Los Angeles, California) |
| Trial ID | NCT05306457 on ClinicalTrials.gov |
What this trial studies
This study investigates the safety of transplanting genetically modified neural progenitor cells that produce a growth factor called GDNF into the motor cortex of patients with Amyotrophic Lateral Sclerosis (ALS). It is a Phase 1/2a, single-center trial that will assess two escalating doses of these cells delivered to the brain's 'hand-knob' area. Participants will be enrolled sequentially, with a minimum interval between surgeries to monitor safety and adverse events. The study aims to determine if this novel approach can be safely implemented in humans, as it has only been tested in animals previously.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of ALS and progressive weakness in upper extremities.
Not a fit: Patients with a longer duration of ALS symptoms or those currently taking riluzole or edaravone may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with ALS, potentially slowing disease progression.
How similar studies have performed: While this approach is novel in humans, previous studies have shown promise in animal models using similar cell-based therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion: 1. Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria) 2. Duration of ALS symptoms ≤ 36 months 3. Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities 4. Forced Vital Capacity ≥50% of predicted normal in supine 5. Age: 18 years or older 6. Able to provide Informed Consent 7. Be geographically accessible to the study site and able to travel to study site for required visits 8. Have caregiver to assist in the transportation and care required by participation in the study 9. Not taking riluzole and/or edaravone or on a stable dose for ≥ 30 day 10. For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial. 11. Medically able to undergo craniotomy as determined by the site PI and/or investigators 12. Medically able to tolerate the immunosuppression regimen as determined by the site PI Exclusion: 1. Using invasive ventilatory assistance 2. Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI 3. Presence of any of the following conditions: 1. Current drug or alcohol abuse 2. Any known immunodeficiency syndrome 3. Unstable medical condition 4. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening 4. Persons of child bearing capacity not willing to practice birth control 5. Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy 6. Any condition in the upper extremities that precludes serial strength or coordination testing 7. Any condition that the investigators feel may pose complications for the surgery 8. Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints 9. Allergy to Beta-Lactam antibiotics 10. Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20% 11. Contraindications to MRI
Where this trial is running
Los Angeles, California
- Cedars-Sinai Medical Center — Los Angeles, California, United States (Recruiting)
Study contacts
- Principal investigator: Richard Lewis, MD — Cedars-Sinai Medical Center
- Study coordinator: Pablo Avalos
- Email: Pablo.avalos@cshs.org
- Phone: 310-248-8584
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.