Stem cell transplantation study for the treatment of ALS – Phase 2
The phase 2 trial focused on the safety and maximum tolerated dose of Human Spinal Cord Derived Neural Stem Cell Transplantation. This study expanded on the work of the phase 1 study directed by Eva L. Feldman, M.D., Ph.D., who is the principal investigator and director of the first-ever FDA-approved human clinical trial of stem cells injected directly into the spinal cords of ALS patients.
Phase 1 of the trial, designed to study the safety of the procedure, was completed in 2013 with no significant adverse side effects to patients. And follow-up patient evaluations have produced some extraordinary data: Several participants in the trial, who were treated early in their disease, were determined to have had little or no significant progression of ALS for more than 700 days post-surgery.
Fifteen patients were studied in the phase 2 study, the last 3 of which received 8 million stem cells injected into the lumbar spinal cord followed by 8 million stem cells injected into the cervical spinal cord.
All procedures have been completed and the trial is still ongoing. No data has been released.
Inflammation of the brain and nervous system is thought to be involved in the disease process of ALS. This project involves brain imaging with positron emission tomography (PET) and magnetic resonance imaging (MRI) in patients with amyotrophic lateral sclerosis (ALS). The primary purpose of this study is to test if brain “pictures” made with a new radiotracer, C-PBR28, can provide satisfactory measures of inflammation activity in the brains of ALS subjects. Pictures of C-PBR28 in the brain will be made using PET scans. In addition, comparison research pictures of brain structure will be made with MRI.
Diaphragm Ultrasound study
This study uses ultrasound in ALS populations to aid in the development of biomarkers for more definitive ALS disease staging. ALS patients will have up to 5 ultrasounds of their diaphragms; these will occur every 3 to 4 months. During the ultrasounds patients will be asked to perform a few breathing assessments – quiet breathing, deep inspiration, and sniff testing. The 2-D images will allow the physician to observe changes over time, if any. Pulmonary function tests (PFTs) will be completed to help us evaluate patients’ respiratory system. We will collect basic vital signs – age, gender, height, and weight.
Michigan Brain Autopsy Program
Patients seen in our clinic are eligible to donate their brain and cervical spinal tissue as well as their teeth (molars only) to the Michigan Brain Bank after death. Family members will receive an autopsy report and the tissue will be stored and made available to scientists wishing to study ALS. These tissue donations will also be correlated with blood or skin biopsies that may have been given for another ALS study. These studies may lead to improved understanding and advances in the diagnosis and treatment of neurodegenerative disease.
Cervical MRI (ALS recruitment just completed)
ALS patients participated in this MRI study which is designed to see if MRI imaging can accurately provide an early diagnosis of ALS. The MRI magnet is used slightly differently, though not noticeable to the patient, and looks at chemical concentrations and pathways as well as the microstructure in the spinal cord at the neck level. Researchers will use this data and compare it to scans done on healthy adults to identify differences to gather more information on diagnosing ALS sooner.
Discrimination Study (R01)
This upcoming study will be available to ALS patients with a goal to combine several brain imaging techniques to develop a new diagnostic test to help with an earlier diagnosis of ALS. Researchers will use the MRI magnet slightly differently than normal, though not noticeable to the patient, to determine brain chemical concentrations, brain structure, and brain resting state of activity. That data will then be compiled and compared to corresponding measures in healthy adults.
Validation Study of the ALS Specific Quality of Life – Short Form Instrument
This study validates a shortened version of the ALS Specific Quality of Life – Short Form Instrument (ALSSQOL-SF). The ALSSQOL-SF is a short questionnaire that asks broad questions about emotional, spiritual, and support related issues. We will compare the ALSSQOL-SF to other tools we currently use to determine the best tool for ALS patients. At one clinic appointments patients will answer questions from several surveys. The answers to these surveys will help researchers assess concerns that are not necessarily related to patients’ physical well-being. Additional demographic and health history information will also be collected.