Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease in the human motor system characterized by the selective involvement of spinal cord anterior horn cells, brainstem motor nuclei, and the corticospinal tract. It predominantly presents as concurrent damage to upper and lower motor neurons.
Induced pluripotent stem cells (iPSCs) are a type of induced pluripotent stem cell derived from autologous or allogeneic cell sources. They can differentiate into various functional cell types, including specific motor neuron cells. iPSCs are used for stem cell replacement therapy. iPSCs hold significant clinical potential for ALS treatment. The iPSC database with human leukocyte antigen characteristics may represent a promising technology. This technology has the potential to obtain high-quality cell products and reduce the risk of graft rejection. Moreover, human iPSCs have demonstrated a certain degree of efficacy in the transplantation of neural stem/progenitor cells derived from ALS rodent models.
The potential mechanisms of iPSC therapy for ALS include: the differentiated motor neuron precursor cells can replace damaged motor neurons, and restore motor conduction function; by secreting neurotrophic factors, they protect neurons; through immune regulation, they inhibit inflammatory reactions, and slow the progression of ALS.
Xellsmart Biomedical (Suzhou) Co., Ltd. is developing an injectable solution for ALS treatment using human iPSC-derived motor neuron precursor cells to address the pressing need for ALS therapy.
Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the Informed Consent Form (ICF).
Age between 18 and 60 years, inclusive, with no gender restrictions.
Diagnosed with ALS according to the World Federation of Neurology criteria, and the initial diagnosis date is between 6 to 24 months before the screening date.
Patients who have received standard treatment in the past with poor efficacy or disease progression.
Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS.
Patients diagnosed with severe cognitive impairment, clinical dementia, or major psychiatric disorders, including but not limited to schizophrenia, bipolar disorder, or severe depression, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V).
Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy.
Patients with a history of malignant tumors or a previous diagnosis of malignancy.
Single group
iPSC-MNP
DrugUsing iPSC-MNP Cells for the Treatment of ALS Patients
Number of participants with adverse event (AE) and serious adverse event(SAE)
AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE were evaluated by laboratory examination and imageological examination, measuring method： 1. Head MRI, whole spine MRI, chest CT examination; 2. Changes in vital signs, 12-lead electrocardiogram, ultrasound (thyroid, abdomen, heart, genitourinary system) examination, and neurological signs; 3. Changes in blood routine, urine routine, stool routine, finger end blood oxygen saturation, serum immunoglobulin, cerebrospinal fluid routine, liver function, kidney function, electrolyte, muscle enzyme, lipid, blood glucose, glycated hemoglobin, coagulation function, tumor markers, various hepatitis viruses, treponema lutei antibodies, HIV antibodies and other indicators.
effective of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell
ALS functional assessment: Changes in ALSFRS-R scale score; The ALSFRS-R includes 12 different tasks designed to provide a representative picture of the physical abilities most commonly affected by ALS. These tasks include speaking, salivation, swallowing, writing, climbing stairs, turning in bed, walking, dressing and hygiene, and three different measures of respiratory capacity. Each task is rated on a scale of 0 to 4-0 indicates the inability to perform the task, and 4 indicates the ability to perform it without difficulty. These ratings are then summed to yield a total score ranging from 0 to 48, with 0 being the worst and 48 being the best.
Click on the lead sponsor to view all of their trials.
