Therapeutic potential of human embryonic stem cell transplantation in patients with cerebral palsy

Abstract

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.

A novel approach of human embryonic stem cells therapy in treatment of Friedrich's Ataxia

“…Other changes includes improved urinary bladder control in Case I, improved overall stamina and coordination in Case II and improved spine curvature in Case III. Our finding that the hESCs are capable of treating FRDA is supported by our previous studies, where hESCs have successfully treated the patients with cerebral palsy [12]. Human embryonic stem cells might have the potential to treat patients with neurodegenerative diseases such as cerebral palsy, Friedreich's ataxia, etc.…”

“…In our previous studies we have shown the improvement in patient's condition who were suffering from cerebral palsy and cortical visual impairment after hESCs therapy [12,13]. Herein, I report three cases of FRDA patients who were treated with hESCs therapy.…”

“…The treatment consisted of three phases in which 0.25 mL hESCs were administered through intramuscular route twice daily and 1 mL of hESCs were administered through intravenous route twice every 7th day for 12 weeks initially and then four weeks thereafter. The details of the technique have been elaborated elsewhere [12]. cAsE sErIEs case 1 and case 2: A 25-year-old female (Case 1) and a 28-year-old male (Case 2) were admitted at Nutech Mediworld on July 2011 with chief complaints of difficulty in walking, standing, and climbing stairs, weakness in lower limbs and slowness of speech.…”

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“…[35][36][37][38] The differentiation of embryonic stem cells has been explained and discussed in our previous paper. 39 Until now, we have used this cell line in > 1400 patients with various incurable conditions with remarkable results.…”

Use of Human Embryonic Stem Cells in the Treatment of Age-Related Macular Degeneration

“…Stem cells that have been explored in animal models for the treatment of AMD include embryonic stem cells (ESCs) and human induced pluripotent stem cells (iPSCs) and mesenchymal stem cells (MSCs) [2,6]. In our previous studies we have shown the improvement in the patient's condition suffering from cerebral palsy, cortical visual impairment (CVI), Lyme's disease, spinocerebellar ataxia, Friedreich's ataxia and spinal cord injury after human embryonic stem cells (hESCs) therapy [7][8][9][10][11][12].…”