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

Study Purpose:

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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

autologous mesenchymal stem cells



Phase 1

Study Chair(s)/Principal Investigator(s):

Anthony Windebank, MD, Mayo Clinic

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Mayo Clinic

Rochester, Minnesota, 55905 United States

Full Study Summary:

The primary objective of this study is to determine the safety of intrathecal delivery of autologous mesenchymal stem cells (MSCs) to the cerebrospinal fluid (CSF) of patients with ALS using a dose-escalation study. The trial will include 25 adult, non-ventilator-dependent patients with clinically definite amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue, expanded ex vivo and then, after ~8 weeks, intrathecal (IT) autologous delivery of MSCs will be performed. There will be 5 treatment groups of up to 5 patients each. Groups 1, 2, and 4 will receive a single dose of cells. Groups 3 and 5 will receive 2 doses of cells separated by 1 month. Groups will be completed sequentially so that patients will not be enrolled into the next treatment group until at least 3 patients in the preceding group have completed the treatment and 1 month of additional observation without significant toxicity. All patients will be followed on a regular basis until death or for a minimum of 2 years after completion of the final infusion. Initial clinical follow-up will be weekly with scheduled blood, CSF and magnetic resonance imaging (MRI) evaluations. After 1 month, patients will have clinical evaluations at 3 month intervals, or earlier if indicated by clinical status.

Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.

Study Sponsor:

Mayo Clinic

Participant Duration:

Follow up: minimum of 2 years after completion of the final infusion.

Estimated Enrollment:


Estimated Study Start Date:

04 / 30 / 2012

Estimated Study Completion Date:

01 / 31 / 2019

Posting Last Modified Date:

09 / 03 / 2019

Date Study Added to neals.org:

05 / 31 / 2012

Minimum Age:

18 Years

Maximum Age:


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

Mayo Clinic

Rochester, Minnesota 55905
United States