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Methods for treating mucopolysaccharidosis

a technology of mucopolysaccharidosis and compositions, applied in the field of compositions and methods for treating mucopolysaccharidosis, can solve the problems of insufficient production of enzymes required to degrade gags, defective enzymes that do not function properly, and undegraded or partially degraded gags, etc., to achieve the effect of increasing enzyme expression and increasing idua activity

Inactive Publication Date: 2019-03-14
UNIV OF MIAMI
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The present invention provides methods for treating various forms of mucopolysaccharidosis (MPS) using stilbenoid compounds, such as resveratrol and piceatannol. These compounds can increase the activity of the enzyme IDUA, which is deficient in MPS. The stilbenoid compounds can cross the blood-brain barrier, which is important for addressing the neurological manifestations of MPS. The technical effect of the invention is to provide an effective treatment for MPS that targets the underlying cause of the disease.

Problems solved by technology

MPS patients either do not produce enough of the enzymes required to degrade GAGs or produce defective enzymes that do not function properly.
As a result, undegraded or partially degraded GAGs accumulate and interfere with the normal function of cells, tissues, and organs, and affect the normal growth and development of individuals.
Hurler syndrome is characterized by a near total absence of α-L-iduronidase activity, resulting in heart and liver diseases and mental deterioration as well as death in childhood.
Currently, the only available treatment of MPS is enzyme replacement therapy, although this treatment is very expensive and therefore not available for many afflicted with an MPS condition.

Method used

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  • Methods for treating mucopolysaccharidosis
  • Methods for treating mucopolysaccharidosis
  • Methods for treating mucopolysaccharidosis

Examples

Experimental program
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Effect test

example 1

Administration of Resveratrol Restores IDUA Expression and / or Activity

[0071]A cell culture experiment was performed in MPS-I patient-derived fibroblast cells. Cells were cultured overnight in a 384-well plate, at a concentration of 20,000 cells per well in advanced DMEM media (Life Technologies) supplemented with 15% FBS (Life technologies), 1× Glutamax (Life Technologies) and beta-mercaptoethanol (1:125000 dilution). The next day, cells were treated with the indicated compound with final concentrations including, but not limited to: 100 μM, 30 μM, 10 μM, 3.3 μM, 1.1 μM, 0.37 μM and 0.12 μM depending on experiment. Treated cells were then incubated for 48 hours at 37° C., 5% CO2, 95% humidity. Cells were then washed with HBSS buffer, and 25 μM of the fluorogenic substrate 4-Methylumbelliferyl α-L-iduronide (free acid) (Santa Cruz Biotechnology) were added in the presence of 0.2M CHNaO2, 0.1% BSA, and 0.1% Triton X. After the addition of the 4-Methylumbelliferyl α-L-iduronide substra...

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Abstract

The present invention provides methods and compositions for the treatment of mucopolysaccharidoses (MPS).

Description

CROSS-REFERENCE TO RELATED APPLICATION[0001]This application is a National Stage Application of International Patent Application No. PCT / US2016 / 041564, filed Jul. 8, 2016, which claims the priority benefit of U.S. Provisional Patent Application No. 62 / 190,935, filed Jul. 10, 2015, the disclosure of which is incorporated herein by reference in its entirety.FIELD OF THE INVENTION[0002]The present invention relates to compositions and methods for treating mucopolysaccharidosis (MPS).BACKGROUND[0003]Mucopolysaccharidosis (MPS) constitutes a class of lysosomal storage disorders characterized by the excessive accumulation of glycosaminoglycans (GAGs) within the lysosomes of various tissues. MPS patients either do not produce enough of the enzymes required to degrade GAGs or produce defective enzymes that do not function properly. As a result, undegraded or partially degraded GAGs accumulate and interfere with the normal function of cells, tissues, and organs, and affect the normal growth ...

Claims

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Application Information

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IPC IPC(8): A61K31/05A61K9/00A61P3/00
CPCA61K31/05A61K9/0019A61K9/0053A61P3/00A61K45/06A61K31/03A61K31/09A61K31/166A61K31/185A61K31/194A61K31/235A61K31/36A61K31/423A61K31/69A61K31/7004
Inventor BROTHERS, SHAUNWAHLESTEDT, CLAESVOLMAR, CLAUDE HENRY
Owner UNIV OF MIAMI
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