Clinical trial: CNS10-NPC-GDNF for the Treatment of ALS (NCT02943850)

Eligibility criteria:
Inclusion Criteria:

          1. Confirmed diagnosis of ALS (Lab-supported Probable, Probable or Definite EI Escorial
             Criteria)

          2. Duration of symptoms ≤ 36 months

          3. Progressive weakness in lower extremities, with EMG supported evidence of denervation
             in both lower extremities.

          4. Forced Vital Capacity >60% of predicted normal in supine.

          5. Male/Female; Age: 18 and 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 or on a stable dose for ≥ 30 days

         10. For women of child bearing capacity, negative pregnancy test prior to surgery

         11. Medically able to undergo thoracolumbar laminectomy or laminoplasty as determined by
             the site PI and Neurosurgeon

         12. Medically able to tolerate the immunosuppression regimen as determined by the site PI

        Exclusion Criteria:

          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 past 30 days or any previous
             exposure to stem cell therapy

          6. Any condition in the lower extremities which precludes serial strength testing

          7. Any condition that the Neurosurgeon feels 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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.


Patient description (medical language): A 47-year-old man comes to the clinic for the follow up of his neuromuscular disease. He experienced gradual, progressive weakness of the left upper extremity over the last year.  Over the last few months, he has also noticed weakness in the right upper extremity.  BP is 120/75, PR is 80 and temperature is 37 C. Reflexes are brisk in the upper extremities, and the plantar responses are extensor.  Mild gait ataxia is present. The patient is under treatment of Riluzole 50 mg BID with the diagnosis of ALS.

Patient description (patient's own words): I've been suffering from a neuromuscular disease for a while now, and I went to my doctor's office. I'm now a 47-year-old man and over the past year I experienced a progressive and gradual weakness of my left upper extremity, and over the past month, I also noticed a weakness over my right upper extremity. My heart rate was 120/75, and my  PR was 80 with 37°C for temperature. My reflexes are not good in my upper extremities, and I have trouble with my balance. I'm also under Exservan 50 mg for my sclerosis.

Does the patient match the trial criteria? 1