A Study to Evaluate Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis


About this study

The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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).
  • Current use of any of the following investigational medicinal products or nutritional supplements being used or studied for the treatment of ALS:
    • Phenylbutyrate;
    • Retigabine;
    • Inosine;
    • Rasagiline;
    • Interleukin 2(Aldesleukin, Proleukin®);
    • N-acetyl-cysteine;
    • Acetyl carnitine;
    • Tudca (tauroursodeoxycholic acid);
    • BASIS (nicotinamide riboside and pterostilbene);
    • L-Serine;
    • Biotin;
    • Methylcobalamin (B12);
    • Calogen.
  • Unwilling to forgo initiating the use of any new supplements during participation in the study.
  • 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.
  • Unwillingness to discontinue herbal medications or other unapproved drugs.
  • Enrolled in an investigational drug trial within 30 days of baseline visit.
  • Prior stem cell therapy for a neurological disease.
  • 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.
  • Chronic low back pain requiring invasive procedures (i.e., epidural injections or lumbar spine surgery).

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Nathan Staff, M.D., Ph.D.

Closed for enrollment

Contact information:

Michelle Turner



Jacksonville, Fla.

Mayo Clinic principal investigator

Bjorn Oskarsson, M.D.

Closed for enrollment

Contact information:

Lisa Thuro


Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Iryna Muzyka, M.D.

Closed for enrollment

Contact information:

Thomas Osgood CCRP

(480) 301-4992


More information


Publications are currently not available

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