Clinical trial: Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (NCT01348451)

Eligibility criteria:
Inclusion Criteria:

          1. Have the ability to understand the requirements of the study, provide written informed
             consent, understand and provide written authorization for the use and disclosure of
             Protected Health Information (PHI) [per Health Insurance Portability and
             Accountability Act (HIPAA) Privacy Ruling] and comply with the study procedures.

          2. Subjects with sporadic or familial ALS diagnosed as laboratory-supported
             probable,probable or definite according to the World Federation of Neurology El
             Escorial Criteria (Appendix A), based on examination by the site PI.

          3. Age 18 years or older.

          4. Females must have a negative serum pregnancy test and practice an acceptable method of
             contraception or be of non-childbearing potential (post-menopausal for at least 2
             years or surgically sterile [hysterectomy, oophorectomy or surgical sterilization]).

          5. Geographic accessibility to the study center and the ability to travel to the clinic
             for study visits.

          6. Presence of a willing and able caregiver.

          7. Medically able to undergo lumbar or cervical laminectomy as determined by the
             Investigator, surgeon and anesthesiologist.

          8. Medically able to tolerate immunosuppression regimen consisting of basiliximab,
             tacrolimus, mycophenolate mofetil, and methylprednisolone as determined by the site
             Investigator.

          9. Agrees to the visit schedule as outlined in the informed consent.

         10. Not taking riluzole (Rilutek®) or on a stable dose for ≥30 days.

         11. All required vaccinations current: tetanus/diptheria (TDAP), herpes
             zoster/shingles(Vostavax®: within last 10 years and must be prior to surgery),
             pneumonia (Pneumovax®),seasonal/H1N1 flu vaccines (as appropriate for season) for
             Groups B-E.

        Exclusion Criteria:

          1. Etiology of paraplegia or weakness is due to causes other than ALS such as spinal
             ischemia, traumatic spinal injury, traumatic brain injury, multiple sclerosis,
             cerebral stroke, cerebral palsy, or infection.

          2. VC < 60% predicted normal by standard nomogram at the time of screening and VC < 50%
             predicted normal measured supine for age at the time of surgery.

          3. Current or peak Panel Reactive Antibody (PRA) due to alloantibodies > 20% receiving
             their first allograft.

          4. Any known immunodeficiency syndrome.

          5. Receipt of any investigational drug,device or biologic within 30 days of surgery.

          6. Any concomitant medical disease or condition limiting the safety to participate:

               -  Coagulopathy

               -  Active uncontrolled infection

               -  Hypotension requiring vasopressor therapy

               -  Previous spinal surgery at the site of planned transplantation except for
                  anterior cervical dissection fusion (ACDF)

               -  Skin breakdown over the site of surgery

               -  Malignancy (except for non-melanoma skin cancer)

               -  Primary or secondary immune deficiency

               -  Spinal stenosis.

          7. Creatinine >1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) > 2x the upper
             limit of normal, hematocrit/hemoglobin < 30/10, total WBC < 4000, uncontrolled
             hypertension (defined as systolic >180 or diastolic >100) or uncontrolled
             diabetes(defined as hemoglobin A1C >8), evidence of GI bleeding by hemoccult test,
             positive tuberculosis (TB test: PPD/Mantoux), hepatitis B or C, or human
             immunodeficiency virus (HIV).

          8. Presence of any of the following conditions:

               -  Current drug abuse or alcoholism

               -  Unstable medical conditions

               -  Unstable psychiatric illness including psychosis and untreated major depression
                  within 90 days of screening

               -  Positive blood test for hepatitis B or C.

          9. Any condition that the site PI feels may interfere with participation in the study.

         10. Any condition that the surgeon feels may pose complications for the surgery.

         11. Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, or
             methylprednisolone.

         12. Inability to provide informed consent as determined by screening protocol.

         13. Inadequate family or caregiver support as determined by the site PI.
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? Entailment