Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
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Publication Number
US-12239733-B2
Publication Date
2025-03-04
Expiration Date
Abstract
Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.
Core Innovation
The document describes methods and formulations for ameliorating a symptom in a subject having spinocerebellar ataxia (SCA) associated with a polyglutamine repeat mutation. The methods include parenterally administering a pharmaceutical formulation comprising trehalose as the sole active ingredient, with trehalose concentration between about 0.1% (w/v) and about 50% (w/v) and osmolality from about 280 to about 330 mOsm/Kg.
The administration is completed within less than 120 minutes, and the subject is administered about 10 to about 50 grams of trehalose per week. The document further discusses trehalose in the context of diseases associated with abnormal protein aggregation, including tauopathies and polyglutamine/polyalanine-related conditions, with emphasis on OPMD and SCA.
The document also describes monitoring the effect of the trehalose pharmaceutical formulation by measuring an effective serum level of trehalose or a derivative thereof in a subject with SCA associated with a polyglutamine repeat mutation. In this monitoring method, the formulation trehalose concentration, osmolality, weekly trehalose amount, and administration completion time are maintained within the stated ranges.
Claims Coverage
The partial claim set includes two independent claims. Both independent claims center on trehalose as the sole active ingredient administered parenterally for SCA associated with a polyglutamine repeat mutation, with quantitative constraints on formulation concentration, osmolality, weekly dose, and administration completion time. The second independent claim additionally requires measuring an effective serum level of trehalose or a derivative to monitor effect.
Parenteral trehalose-only formulation for SCA amelioration
Parenterally administering to a subject having spinocerebellar ataxia (SCA) associated with a polyglutamine repeat mutation a pharmaceutical formulation comprising trehalose as the sole active ingredient, wherein the formulation has a trehalose concentration between about 0.1% (w/v) and about 50% (w/v) and an osmolality from about 280 to about 330 mOsm/Kg, the subject receiving about 10 to about 50 grams of trehalose per week, with administration completed within less than 120 minutes.
Monitoring effect by measuring effective serum trehalose level
Administering to a subject in need thereof a pharmaceutical formulation comprising trehalose as the sole active ingredient, for a subject having spinocerebellar ataxia (SCA) associated with a polyglutamine repeat mutation, and measuring an effective serum level of trehalose or a derivative thereof, wherein the formulation has a trehalose concentration between about 0.1% (w/v) and about 50% (w/v), an osmolality from about 280 to about 330 mOsm/Kg, the subject is administered about 10 to about 50 grams of trehalose per week, and administration is completed within less than 120 minutes.
Across the independent claims, the core coverage is a trehalose-only pharmaceutical formulation administered parenterally to subjects with SCA associated with a polyglutamine repeat mutation, with specified quantitative constraints on trehalose concentration, osmolality, weekly trehalose amount, and administration completion time. One independent claim covers ameliorating a symptom, and the other covers monitoring effect via measurement of an effective serum level of trehalose or a derivative.
Stated Advantages
Higher bioavailability and muscle/plasma exposure compared with oral trehalose, attributed to overcoming intestinal trehalase degradation.
Documented Applications
Treating diseases associated with abnormal protein aggregation, including spinocerebellar ataxia (SCA) associated with a polyglutamine repeat mutation and conditions emphasized in the document such as OPMD and tauopathies.
Monitoring the effect of the trehalose pharmaceutical formulation in a subject with spinocerebellar ataxia (SCA) associated with a polyglutamine repeat mutation by measuring an effective serum level of trehalose or a derivative thereof.
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