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EAN 2026 | Findings from a Phase I trial of autologous SVF therapy in ALS

Zenglin Cai, PhD, Nanjing Medical University, Nanjing, China, discusses a Phase I trial (ChiCTR2400091754) exploring autologous stromal vascular fraction (SVF) therapy in amyotrophic lateral sclerosis (ALS). Dr Cai shares key findings on the efficacy and safety, demonstrating that SVF therapy is safe and effective in these patients. This interview took place at the 12th Congress of the European Academy of Neurology (EAN) in Geneva, Switzerland.

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Transcript

Okay. When we conduct a Phase I clinical trial, we aim to observe the safety and the efficacy for ALS patients. First, we observed the safety. About the endpoint, for 45 days after SVF treatment, all of the patients only had five mild adverse events: little subcutaneous bleeding, headache, and little fever. So it is very safe for patients. Second, we found most patients’ scores improved a little, although it is not statistically significant...

Okay. When we conduct a Phase I clinical trial, we aim to observe the safety and the efficacy for ALS patients. First, we observed the safety. About the endpoint, for 45 days after SVF treatment, all of the patients only had five mild adverse events: little subcutaneous bleeding, headache, and little fever. So it is very safe for patients. Second, we found most patients’ scores improved a little, although it is not statistically significant. But about 15 patients self-reported symptom improvement. Third, we observed the CSF biomarkers of patients. We observed that the biomarkers of NfL and GFAP are reduced. So we conclude that SVF treatment is effective for patients with ALS. First, we will do a larger placebo-controlled trial and expand for more patients to observe the efficacy. Second, for these patients, SVF has been processed in our laboratory. So we can isolate the exosomes from the SVF and formulate them into some intranasal spray for patients to use at home. In summary, we think SVF and its formulation will be easy to use and have some efficacy for ALS patients.

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