New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy
Abstract Over the past three decades the lysosomal storage diseases have served as model for rare disease treatment development. While these efforts have led to considerable success, important challenges remain. For example, no treatments are currently approved for nearly two thirds of all lysosomal...
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doaj-b30fbcc639c8423a8b0eb5d2efb78c142021-03-28T11:10:32ZengBMCOrphanet Journal of Rare Diseases1750-11722021-03-011611610.1186/s13023-021-01779-4New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategyEdward H. Schuchman0Maria D. Ledesma1Calogera M. Simonaro2Department of Genetics and Genomic Sciences, Icahn School of Medicine At Mount SinaiCentro Biologia Molecular Severo OchoaDepartment of Genetics and Genomic Sciences, Icahn School of Medicine At Mount SinaiAbstract Over the past three decades the lysosomal storage diseases have served as model for rare disease treatment development. While these efforts have led to considerable success, important challenges remain. For example, no treatments are currently approved for nearly two thirds of all lysosomal diseases, and there is limited impact of the existing drugs on the central nervous system. In addition, the costs of these therapies are extremely high, in part due to the fact that drug development has focused on a “single hit” approach – i.e., one drug for one disease. To overcome these obstacles researchers have begun to focus on defining common disease mechanisms in the lysosomal diseases, particularly in the central nervous system, with the hope of identifying drugs that might be used in several lysosomal diseases rather than an individual disease. With this concept in mind, herein we review a new potential treatment approach for the lysosomal storage diseases that focuses on modulation of the endocannabinoid system. We provide a short introduction to lysosomal storage diseases and the endocannabinoid system, followed by a brief review of data supporting this concept.https://doi.org/10.1186/s13023-021-01779-4Lysosomal storage diseasesEndocannabinoidsTreatment |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Edward H. Schuchman Maria D. Ledesma Calogera M. Simonaro |
spellingShingle |
Edward H. Schuchman Maria D. Ledesma Calogera M. Simonaro New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy Orphanet Journal of Rare Diseases Lysosomal storage diseases Endocannabinoids Treatment |
author_facet |
Edward H. Schuchman Maria D. Ledesma Calogera M. Simonaro |
author_sort |
Edward H. Schuchman |
title |
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
title_short |
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
title_full |
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
title_fullStr |
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
title_full_unstemmed |
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
title_sort |
new paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy |
publisher |
BMC |
series |
Orphanet Journal of Rare Diseases |
issn |
1750-1172 |
publishDate |
2021-03-01 |
description |
Abstract Over the past three decades the lysosomal storage diseases have served as model for rare disease treatment development. While these efforts have led to considerable success, important challenges remain. For example, no treatments are currently approved for nearly two thirds of all lysosomal diseases, and there is limited impact of the existing drugs on the central nervous system. In addition, the costs of these therapies are extremely high, in part due to the fact that drug development has focused on a “single hit” approach – i.e., one drug for one disease. To overcome these obstacles researchers have begun to focus on defining common disease mechanisms in the lysosomal diseases, particularly in the central nervous system, with the hope of identifying drugs that might be used in several lysosomal diseases rather than an individual disease. With this concept in mind, herein we review a new potential treatment approach for the lysosomal storage diseases that focuses on modulation of the endocannabinoid system. We provide a short introduction to lysosomal storage diseases and the endocannabinoid system, followed by a brief review of data supporting this concept. |
topic |
Lysosomal storage diseases Endocannabinoids Treatment |
url |
https://doi.org/10.1186/s13023-021-01779-4 |
work_keys_str_mv |
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