BackgroundThe present study evaluated the efficacy and safety of human embryonic stem cell (hESC) therapy in patients with CP.Materials and methodsThis analysis included patients (30 days-18 yr) with documented diagnosis of CP. The study consisted of four treatment phases (T1, T2, T3, T4) separated by gap phases. Efficacy of hESC therapy was evaluated based on Gross Motor Function Classification Scores Expanded and Revised (GMFCS-E & R; 1-good to 5-bad).ResultsNinety one patients were included and all received hESC therapy in T1, 66 patients returned for T2, 38 patients for T3, and 15 patients for T4. Overall, 30.2% patients achieved GMFCS-E & R score 1 during the study with different number of patients achieving GMFCS score 1 by the end of each treatment phase (T1: 6 [6.6%]; T2: 7 [10.6%]; T3: 11 [28.9%]; and T4: 5 [33.3%]). All patients in up to 2 yr (n = 10), 2-4 yr (n = 10), 4-6 yr (n = 9), and 6-12 yr (n = 8) age groups except one of the 5 patients in the age group of 12-18 yr transitioned from GMFCS-E & R score 5 to lower scores by end of T1. Most patients transitioned to GMFCS-E & R score 2 (n = 34) from higher scores by end of T2. Eleven patients achieved GMFCS-E & R score 1 by end of T3. No serious adverse events were observed.ConclusionUse of hESC therapy in patients with CP is effective and safe. hESC therapy has demonstrated significant improvement in GMFCS-E & R scale.

BackgroundHuman embryonic stem cells (hESCs) are pluripotent cells that have the potential to self-renew and differentiate into all types of human cells.PurposeThe present study was aimed at establishing the safety of hESC therapy in patients with terminal/incurable conditions.MethodsThis was a single cohort study conducted at Nutech Mediworld, New Delhi. The patients suffering from various degenerative diseases were included in the study from year 2002 to 2004. hESCs (0.25 mL) were injected under skin in the abdominal wall. The safety of hESC therapy was evaluated by assessing the AEs experienced by patients during the study. Any disabling symptom/ sign, teratoma or antigen-antibody reaction that a patient suffered post transplantation of hESCs was considered as an AE.ResultsA total of four, six and twenty three patients received hESC therapy in the year 2002, 2003 and 2004 respectively. Pain and fever were the most common AEs observed during the study. Other AEs included headache, mild pain in the abdomen, swelling of legs (edema), urinary tract infection (UTI), rash/erythema, pain at the lower back and limbs and body ache. All the AEs reported were mild in nature and resolved within one or two days with symptomatic medication and rest. No serious AEs were reported. The improvement in specific parameters of the patients was observed after the therapy.ConclusionhESCs used in the present study are safe for use in humans afflicted with incurable/terminal conditions. Future, prospective controlled studies to substantiate the present study are ongoing.

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