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.
- 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
- 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
- History of a chronic onset of a progressive motor weakness of less than two years
- 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.
- 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
- 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
- Use of herbal medications, nutritional supplements or other unapproved drugs or
investigational medicinal products being used or studied for the treatment of ALS.
- Unwilling to forgo initiating the use of any new supplements during participation in
- 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
- Men or women of childbearing potential who are unwilling to employ adequate
- Chronic low back pain requiring invasive procedures (i.e. epidural injections or
lumbar spine surgery)
The Goal of the Proposed Study is to perform an open label, 60 subject, Phase II multi-site clinical trial to investigate the safety and efficacy of intrathecal treatment of aaMSCs in ALS. Patients will be treated with 10-100 million aaMSCs every 3 months for a total of 4 intrathecal injections over 12 months. Reduced dose treatments will be allowed based on specific adverse events. Multiple biomarkers will be tracked throughout the clinical trial and correlated with response to treatment. This study was initially performed at Mayo Clinic in Rochester and subsequently expanded to the two other Mayo Clinic sites in Arizona and Florida. All biopsies and stem cell injections take place at Mayo Clinic Rochester, regardless of where the subject initially enrolls into the study.