Background:

Werdnig Hoffman (WH), a hereditary neurodegenerative disorder of lower motoneurons associated with progressive muscle weakness is the most common genetic cause of infant mortality. There is no effective treatment for WH exists. The field of translational research is active now, and clinical trials or case studies are ongoing. We present a phase 1 clinical trial in patients with WH who received side population adipose-derived mesenchymal stem cells (SPADMSCs). Methods The intervention group administered with three intrathecal administrations of escalating doses of SPADMSCs. The safety analysis was assessed by controlling the vital signs and efficacy analysis performed by the Ballard score and EMG test. These tests were performed previous to treatment and at the end of the follow-up. Results The treatment well-tolerated, without any adverse event related to the stem cell administration. Patients showed significant improvement in the amplitude response of motor in the tibial nerve (0.56 mV; p 0.029). The weight of patients, ventilation days, and number of hospitalizations were not meaningful parameters in the response of patients in the intervention and control groups. One patient in the intervention group is still alive after 36 months. He gained a normal weight and has a normal growth rate. The patient can breathe without ventilator aid. Conclusion The present study for stem cell therapy shows safety and efficacy in WH patients, mainly in the recovery of the tibial nerve, respiratory system, and length of life.