Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)

Prior treatment

• Evidence of any concurrent illness or treatments limiting the safety to participate or any condition that the neurosurgeon feels may pose complications for the surgery;
• Cancer within the previous 10 years;
• Patient has been treated previously with any stem cell or somatic cells therapy;
• Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.

Other illness

• Psychiatric disease or other neurological diseases different from ALS;
• Immunosuppressive therapy within 12 weeks of screening; active autoimmune disease or infection (including hepatitis B, hepatitis C, or HIV);
• Cognitive impairment;

Other

• Contraindications to perform MRI scans, CSF withdrawal and Skin biopsy;
• Patient unable to understand informed consent form;
• Pregnancy and breast feeding;

• Patient provides written informed consent, informed consent signature collection prior to any study procedure (patient has good acceptance and understanding of the informed consent);
• Definite, probable diagnosis according to the revised El Escorial criteria;
• Age: 18-65 years;
• FVC \>70%;
• Onset ≤ 24 months;
• Patients with an ALSFRS-R score of at least 26; overall, including a score of at least 2 on each of the 1-9 ALSFRS-R individual component items and of at least 3 of the 10-12 individual components items;
• Evidence of fast progression of the disease. We exclude slow progressors at the time of screening defined as Patient with an ALSFRS-R total score progression between onset of the disease and screening of \< 0.3 per month. We document the fast progression of the disease defined as ALSFRS-R total score decrease of ≥ 1 point per month during a 12 week run-in period between screening and randomization;
• Patient should be on a stable dose of Riluzole for \> 30 days from pre-screening visit or not taking riluzole at all, nor plan to begin riluzole during the study period;
• Patient is medically able to tolerate transient immunosuppression regimen;
• Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines.