A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis


About this study

The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation 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 must have clinically-defined ALS as defined by the World Federation of Neurology criteria
  • Age greater than 18 years
  • If female, must be post-menopausal or had a hysterectomy
  • Permanent resident or citizen of the United States
  • History of a chronic onset of a progressive motor weakness of greater than one year, but less than two years duration
  • Must have vital capacity greater than 65% of predicated for age, gender, and body type
  • Able to comply with protocol requirements, including MRI testing
  • Can provide written informed consent

Exclusion Criteria:

  • 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.
  • Autoimmunity, including Crohn's disease, rheumatoid arthritis, psoriasis
  • Malignancy including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). Any other malignancy will not be allowed.
  • Active systemic or local infection near the lumbar puncture site
  • Other active systemic disease as defined by laboratory abnormalities
  • Use of herbal medications or other unapproved drugs
  • Enrolled in an investigational drug trial within 30 days of baseline visit
  • Kokmen Short Test of Mental Status score <32
  • Beck's Depression Inventory score >18
  • Presence of a tracheostomy
  • Ventilator dependent

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

Anthony Windebank, M.D.

Closed for enrollment

Contact information:

Pamela Vicari B.S.N., R.N.

More information


Publications are currently not available

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