Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Please see the detailed description in following content

Inclusion criteria

• Patients with a confirmed diagnosis of ALS, with an ALSFRS-R score ≥35 and a maximum vital capacity ≥65%
• Aged >35 years and <50 years, with no gender restrictions
• Karnofsky Performance Status (KPS) score ≥70, and Eastern Cooperative Oncology Group (ECOG) Performance Status ≤2
• Unrelated umbilical cord blood and the recipient demonstrate high-resolution HLA (-A, -B, Cw, DR, DQ) matching at ≥4/6, 5/8, or 7/10 loci, and the post-thaw CD34+ cell count in the cord blood unit is ≥1.2×10⁵/kg (recipient body weight)
• Willing and able to comply with the study procedures and conditions, demonstrating good compliance
• Willing to receive at least two years of treatment and follow-up, with detailed medical records maintained
• The patient and/or their legal guardian voluntarily participate in this clinical trial, sign the informed consent form, and are capable of completing all follow-up assessments as required by the protocol

Exclusion criteria

• Patients with positive results in the following etiological tests: Human Immunodeficiency Virus (HIV-1/2), Human Cytomegalovirus DNA (HCMV-DNA), Epstein-Barr Virus DNA (EBV-DNA), Hepatitis B (positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B virus DNA (HBV-DNA)), Hepatitis C antibody (HCV-Ab), or Treponema pallidum antibody (TP-Ab)
• Clinically significant active bacterial, viral, fungal, or parasitic infections as judged by the investigator during screening
• Patients who have previously received gene therapy or allogeneic hematopoietic stem cell transplantation
• First-degree relatives with known or suspected familial cancer syndromes (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary nonpolyposis colorectal cancer syndrome, familial adenomatous polyposis, etc.)
• Diagnosis of major psychiatric disorders or predisposition to such conditions that would significantly impair the ability to participate in the clinical study
• History of major organ impairment, including: Liver disorders: Liver function tests showing AST or ALT >3 × ULN; total serum bilirubin >2.5 × ULN; for cases consistent with Gilbert syndrome, total bilirubin >3 × ULN and direct bilirubin >2.5 × ULN; history of hepatic bridging fibrosis, cirrhosis, or active hepatitis; Cardiac disorders: Left ventricular ejection fraction (LVEF) <45% at screening; New York Heart Association (NYHA) Class III or IV congestive heart failure; severe arrhythmias requiring treatment; poorly controlled hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg despite antihypertensive therapy), or prior history of hypertensive emergencies, hypertensive encephalopathy, or unstable angina; history of myocardial infarction, coronary artery bypass graft surgery, peripheral arterial bypass graft implantation, or stent placement within 12 months prior to enrollment; clinically significant valvular disease; calculated eGFR <60 mL/min/1.73m²; Pulmonary function: FEV1/FVC <60% and/or diffusing capacity less than 60% of predicted; clinically significant evidence of pulmonary hypertension requiring medical intervention
• Uncorrectable coagulation dysfunction or history of severe bleeding disorders
• Any other condition deemed by the physician to render the subject unsuitable for hematopoietic stem cell transplantation
• Known hypersensitivity to the investigational drug or its components
• Participation in or ongoing participation in other interventional clinical studies within 3 months prior to screening
• Vaccination with live vaccines within 6 weeks prior to screening
• Pregnant or breastfeeding women
• History of solid organ transplantation
• Poor compliance of the subject with the study protocol
• Any other condition considered by the investigator as unsuitable for participation in this clinical trial
• Unwillingness of the subject to provide pre-existing valid diagnostic evidence before treatment or to undergo bone marrow, lumbar puncture, blood tests, and other examinations after treatment