SPERIMENTAZIONE SU 12 MALATI DI SLA - FASE 1
Il primo ok alla sperimentazione sull'uomo è stato ottenuto dall'azienda Biotherapeutics Neuralstem di Rockville (Maryland), e i test saranno condotti in Georgia , presso la Emory University : sarà una sperimentazione di fase 1 sperimentazione sull'uomo ( test su 12 pazienti )basata sulle cellule staminali neurali per la cura della SLA. Si tratta di una delle prime autorizzazioni concesse in seguito alla selezione avviata nel settembre scorso dagli statunitensi National Institutes of Health (Nih).
STAFF della Struttura Complessa NEUROLOGIA - NOVARA
LEGGI NEI COMMENTI LE PRECISAZIONI DELLA DOTT.SSA LETIZIA MAZZINI
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We have just read, not without some interest, the NeuralStem communicate. The trial is presented as if it were the first clinical study with stem cells in ALS (“This is the first stem-cell approach for ALS” says Lucie Bruijin). However, this needs to be rectified as we conducted two phase 1 clinical trials one in 2001 (Mazzini et al., 2008)) and the other in 2007 (Mazzini L et al., 2009). The trials were approved respectively by the regional Ethical Committee and by the Italian Institute of Health and by the and were designed to test the safety and the feasibility of mesenchymal stem cell transplantation into the spinal cord of ALS patients.
MSC were isolated from patients’ bone marrow, in vitro expanded for 3-4 passages and evaluated for quality control as requested by national rules on advanced therapies. In neither of our trials were there any immediate or delayed transplant related toxicities. Stem cells were transplanted into the spinal cord at the thoracic levels with a surgical approach. Clinical, laboratory, and radiographic evaluations of the patients showed no serious transplant related adverse events. Magnetic resonance images (MRI) showed no structural changes (including tumor formation) in either the brain or the spinal cord.
Furthermore, we also demonstrated that expanded MSCs can survive and migrate after transplantation in the lumbar spinal cord of SOD1-G93A mice, where they prevent astrogliosis and microglial activation and delay the ALS-related decrease in the number of motor neurons, resulting in an amelioration of motor performance (Vercelli A et al., 2008 ). Therefore we concluded that MSCs represent a good source of stem cells for future ALS cell based clinical trials.
Thus, the NeuralStem trial, as it appears, shows this experimental design to be no different to our studies except for the cell type: neural stem cell derived from a 8-week fetus.
Letizia Mazzini, MD ALS Centre, Dpt of Neurology ,Eastern Piedmont University Maggiore della Carità Hospital Novara, Italy
Franca Fagioli, MD Stem Cell Transplantation and Cellular Therapy Unit Pediatric Onco-Hematology Department Regina Margherita Children’s Hospital Torino. Italy
References
1.Mazzini, L. & Mareschi, K.& Ferrero, I.& Vassallo, E.& Oliveri, G.& Nasuelli, N.& Oggioni, GD.& Testa, L.& Fagioli F. Stem cell treatment in Amyotrophic Lateral Sclerosis. Journal of the Neurological Sciences 265 (2008) 78–83
2. Mazzini L, Ferrero I, Luparello V, Rustichelli D, Gunetti M, Mareschi K, Testa L, Stecco A, Tarletti R, Miglioretti M, Fava E, Nasuelli N, Cisari C, Massara M, Vercelli R, Oggioni GD, Carriero A, Cantello R, Monaco F, Fagioli F Mesenchymal stem cell transplantation in amyotrophic lateral sclerosis: A Phase I clinical trial.Exp Neurol. 2009 Aug 13. [Epub ahead of print]
3. Alessandro Vercelli, Oana M Mereuta, MD; Diego Garbossa, MD; Giuseppe Muraca; Katia Mareschi; Deborah Rustichelli; Ivana Ferrero; Letizia Mazzini, MD; Enrico Madon, MD; Franca Fagioli, MD Human mesenchymal stem cell transplantation extends survival, improves motor performance and decreases neuroinflammation in mouse model of amyotrophic lateral sclerosis
Neurobiology of disease. 31 (2008) 395–405
Posted by: Mazzini Letizia | September 28, 2009 08:06 AM
Per completezza di informazioni pubblichiamo in allegato la nota, contenente alcune puntualizzazioni relative alle sperimentazioni con le cellule staminali già condotta sull'uomo in Italia, inviato alla rivista Nature dalla dottoressa Letizia Mazzini del Centro Sla-Dip. Neurologia dell'Università del Piemonte Orientale A. Avogadro di Novara.
La dottoressa Mazzini precisa che quella americana non è, in assoluto, la prima sperimentazione clinica con le cellule staminali in pazienti Sla. Insieme alla sue equipe, infatti, la dottoressa Mazzini ha già condotto in Italia nel 2001 e nel 2007 due trials clinici in fase 1 relativamente al trapianto di cellule staminali mesenchimali nel midolo spinale del tutto simili a quello proposto ora negli Usa, eccezion fatta per il tipo di cellule staminali utilizzate (mesenchimali invece che neurali derivate da un feto di 8 settimane).
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