CNS10-NPC-GDNF Delivered to the Motor Cortex for the Treatment of ALS

What is the Purpose of this Study?

This study focuses on individuals who have been diagnosed with amyotrophic lateral sclerosis (ALS), with symptoms present for less than 3 years. The purpose of the study is to examine the safety and effectiveness of transplanting cells that have been genetically engineered to produce a growth factor into the motor cortex of ALS patients to determine whether these cells are safe and help protect the motor neuron cells that are damaged. The cells are called neural progenitor cells (NPC). A growth factor is a substance that is required for the stimulation of growth in living cells. The growth factor produced by the cells used in this study is called glial cell line-derived neurotrophic factor, or GDNF. Therefore, the investigational cells are called “CNS10-NPC-GDNF.”

Researchers aim to determine the safety of delivering CNS10-NPC-GDNF cells into the motor cortex (brain), as well as which dose of the cells is safe to use in people. The cells will be delivered to one side of the brain, into the area that controls movement of one arm and specifically, the hand.

Eligibility

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

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

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Where can I participate?

Beverly : Pablo Avalos

More about this Clinical Trial

What is the full name of this clinical trial?

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

Study Details

Disease Type/Condition

Amyotrophic lateral sclerosis (ALS), Neural Progenitor Cells, Stem Cells

Principal Investigator

Lewis, Richard

Age Group

Adult

Phase

I/II

IRB Number

STUDY00000278

ClinicalTrials.gov ID

NCT05306457

Key Eligibility

ClinicalTrials.gov

How do I learn more about this study?

Name

Pablo Avalos

Pablo.Avalos@cshs.org