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Current and Future Treatments for Lysosomal Storage Disorders
David P W Rastall1, Andrea Amalfitano2,3
1Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, 48824, USA.
Current Treatment Options in Neurology
|November 5, 2017
Summary
Novel therapeutics for lysosomal storage disorders (LSDs) are advancing, with new enzyme replacement (ERT) and substrate reduction therapies (SRT) approved. Gene therapy and hematopoietic stem cell transplant (HSCT) show promise for treating these genetic conditions.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Lysosomal storage disorders (LSDs) are genetic conditions.
- Novel therapeutics are crucial for LSDs.
- Current treatments include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).
Purpose of the Study:
- To review recently approved drugs for LSDs.
- To examine clinical trials of gene therapy and hematopoietic stem cell transplant (HSCT) for LSDs.
- To discuss the future landscape of LSD therapeutics.
Main Methods:
- Literature review of approved drugs and clinical trials.
- Analysis of recent FDA and EU regulatory decisions.
- Synthesis of findings on gene therapy and HSCT efficacy and challenges.
Main Results:
- Two substrate reduction therapies (SRTs) are now approved for LSDs.
- Gene therapy (adeno-associated virus, adenovirus) and HSCT show promising clinical results.
- Some advanced therapies face challenges with adoption and economic viability.
Conclusions:
- 11 ERTs and 2 SRTs are approved in the USA for LSDs.
- Gene therapy and HSCT are at the forefront of LSD treatment innovation.
- Key challenges include immune response, blood-brain barrier penetration, and end-organ damage reversal.
Keywords:
Clinical trialsEnzyme replacement therapyGene therapyHematopoietic stem cell transplantationLysosomal storage disordersSubstrate reduction therapyMore Related Videos
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