Clinical trial: Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS) (NCT03268603)

Eligibility criteria:
Inclusion Criteria:

          -  All patients will have ALS diagnosed as possible, laboratory-supported probable,
             probable, or definite as defined by the World Federation of Neurology criteria for the
             diagnosis of ALS.

          -  Examination and neurophysiological testing confirm a pure motor syndrome compatible
             with the diagnosis of ALS. All other possible causes of weakness have been excluded by
             extensive investigations.

          -  Age greater than 18 years, if female, must be post-menopausal, had a hysterectomy, or
             agree to two forms of birth control.

          -  Permanent resident or citizen of the United States.

          -  Geographic accessibility to the study site and willingness and ability to comply with
             follow-up.

          -  History of a chronic onset of a progressive motor weakness of less than two years
             duration.

          -  Subjects must be taking a stable dose of riluzole for at least 30 days prior to
             enrolment or not be on riluzole, and not have been on it for at least 30 days prior to
             enrolment (riluzole-naïve subjects are permitted in the study).

          -  Able to comply with protocol requirements, including MRI testing.

          -  Can provide written informed consent.

        Exclusion Criteria:

          -  Use of Radicava® (edaravone) within 30 days of screening or intent to use Radicava® at
             any time during the course of the study including the follow up period.

          -  Any clinically significant medical condition (e.g., within six months of baseline, had
             myocardial infarction, angina pectoris, and/or congestive heart failure) that, in the
             opinion of the investigator, would compromise the safety of patient.

          -  Pulmonary Slow Vital Capacity (SVC) less than 65% of predicted for age, gender, and
             body type.

          -  Autoimmunity, including Crohn's disease or rheumatoid arthritis

          -  Current use of immunosuppressant medication or use of such medication within 4 weeks
             of Screening visit (Visit 1).

          -  Malignancy 5 years prior to enrollment, including melanoma,with the exception of
             localized skin cancers (with no evidence of metastasis, significant invasion, or
             re-occurrence within three years of baseline).

          -  Active systemic or local infection near the lumbar puncture site.

          -  Inability to lie flat for the duration of intrathecal cell transplantation, or
             inability to tolerate study procedures for any other reason.

          -  Other active systemic disease as defined by laboratory abnormalities delineated in
             Appendix IV.

          -  Use of herbal medications, nutritional supplements or other unapproved drugs or
             investigational medicinal products being used or studied for the treatment of ALS.

          -  Enrolled in an investigational drug trial within 30 days of baseline visit

          -  Prior stem cell therapy of any kind

          -  Kokmen Short Test of Mental Status score <32

          -  Presence of a tracheostomy

          -  Ventilator dependent

          -  Pregnancy

          -  Men or women of childbearing potential who are unwilling to employ adequate
             contraception
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