CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS
Amyotrophic Lateral Sclerosis
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Stem cells, Growth Factor, Regenerative, Neural Progenitor Cells, NPC, Transplantation
Eligibility Criteria
Inclusion:
- Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria)
- Duration of ALS symptoms ≤ 36 months
- Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities
- Forced Vital Capacity ≥50% of predicted normal in supine
- Age: 18 years or older
- Able to provide Informed Consent
- Be geographically accessible to the study site and able to travel to study site for required visits
- Have caregiver to assist in the transportation and care required by participation in the study
- Not taking riluzole and/or edaravone or on a stable dose for ≥ 30 day
- For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial.
- Medically able to undergo craniotomy as determined by the site PI and/or investigators
- Medically able to tolerate the immunosuppression regimen as determined by the site PI
Exclusion:
- Using invasive ventilatory assistance
- Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI
Presence of any of the following conditions:
- Current drug or alcohol abuse
- Any known immunodeficiency syndrome
- Unstable medical condition
- Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
- Persons of child bearing capacity not willing to practice birth control
- Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy
- Any condition in the upper extremities that precludes serial strength or coordination testing
- Any condition that the investigators feel may pose complications for the surgery
- 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
- Allergy to Beta-Lactam antibiotics
- Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20%
- Contraindications to MRI
Sites / Locations
- Cedars-Sinai Medical CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
CNS10-NPC-GDNF - Group A
CNS10-NPC-GDNF - Group B
CNS10-NPC-GDNF - Group C
Unilateral, Motor Cortex, 0.25x10^6 cells in 10 µL/site, 21 sites (5.25x10^6 total cells) - Motor cortex corresponding to the non-dominant hand
Unilateral, Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the non-dominant hand
Unilateral Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the dominant hand