Aperçu: G.M.
Pediatr Res. 2017 Oct 6. doi: 10.1038/pr.2017.254.
Cell therapy for diverse central nervous system disorders: Inherited metabolic diseases and autism
Sun JM1, Kurtzberg J1.
Author information
- 1
- Robertson Clinical and Translational Cell Therapy Program, Duke University, Durham, NC, USA.
Abstract
La notion d'utilisation de cellules humaines pour le traitement des affections médicales n'est pas nouvelle. Dans
sa forme la plus simple, la transfusion de produits sanguins comme
traitement d'une hémorragie sévère a été pratiquée depuis les années
1800. L'avènement
de la transplantation de cellules souches hématopoïétiques (HSCT) a
commencé par le développement de la transplantation de moelle osseuse
pour les tumeurs malignes hématologiques au milieu des années 1900 et
est maintenant un standard de soins pour de nombreux troubles
hématologiques. Au
cours des dernières décennies, HSCT s'est étendu à d'autres sources de
cellules donneuses, à une plus large gamme d'indications et au
développement de nouveaux produits cellulaires. Cette
trajectoire a suscité un intérêt croissant pour la recherche de
thérapies cellulaires novatrices pour traiter les maladies actuellement
incurables, y compris les troubles neurologiques. HSCT
est actuellement une thérapie établie pour certaines maladies
métaboliques héréditaires (IMD) dévastatrices neurologiquement, dans
lesquelles les cellules donneuses de greffe fournissent un
remplacement enzymatique tout au long de la vie qui empêche la
détérioration neurologique et prolonge considérablement la vie des
enfants touchés. Les
connaissances acquises grâce au traitement de ces troubles rares ont
permis d'améliorer les indications et le calendrier du HSCT, l'étude de
produits et de techniques cellulaires supplémentaires pour répondre à
ses limites et l'étude de thérapies cellulaires sans transplantation
pour traiter des affections neurologiques plus fréquentes telles que le "trouble du spectre de l'autisme" (TSA).
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematologic malignancies in the mid-1900s and is now standard of care for many hematologic disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products. This trajectory has sparked a rapidly growing interest in the pursuit of innovative cell therapies to treat presently incurable diseases, including neurologic conditions. HSCT is currently an established therapy for certain neurologically devastating inherited metabolic diseases (IMDs), in which engrafting donor cells provide lifelong enzyme replacement that prevents neurologic deterioration and significantly extends lives of affected children. Knowledge gained from the treatment of these rare conditions has led to refinement of the indications and timing of HSCT, the study of additional cellular products and techniques to address its limitations, and the investigation of cellular therapies without transplantation to treat more common neurologic conditions such as autism spectrum disorder (ASD)
.Pediatric Research accepted article preview online, 06 October 2017. doi:10.1038/pr.2017.254.
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplantation (HSCT) began with the development of bone marrow transplantation for hematologic malignancies in the mid-1900s and is now standard of care for many hematologic disorders. In the past few decades, HSCT has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products. This trajectory has sparked a rapidly growing interest in the pursuit of innovative cell therapies to treat presently incurable diseases, including neurologic conditions. HSCT is currently an established therapy for certain neurologically devastating inherited metabolic diseases (IMDs), in which engrafting donor cells provide lifelong enzyme replacement that prevents neurologic deterioration and significantly extends lives of affected children. Knowledge gained from the treatment of these rare conditions has led to refinement of the indications and timing of HSCT, the study of additional cellular products and techniques to address its limitations, and the investigation of cellular therapies without transplantation to treat more common neurologic conditions such as autism spectrum disorder (ASD)
.Pediatric Research accepted article preview online, 06 October 2017. doi:10.1038/pr.2017.254.
- PMID:28985203
- DOI:10.1038/pr.2017.254
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