Compositions for treating CMT and related disorders
Inventors
Cohen, Daniel • Nabirochkin, Serguei • Chumakov, Ilya
Assignees
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Publication Number
US-11672796-B2
Publication Date
2023-06-13
Expiration Date
Abstract
The present invention relates to compositions and methods for the treatment of the Charcot-Marie-Tooth disease and related disorders.
Core Innovation
The invention relates to pharmaceutical compositions and related therapeutic approaches for Charcot-Marie-Tooth disease, including Charcot-Marie-Tooth Disease Type 1A, using baclofen, sorbitol, and naltrexone in a pharmaceutically suitable excipient or carrier. The document also describes unit dosage forms and patient packs that include a liquid formulation for oral administration comprising these active ingredients.
The compositions are described as ternary drug combinations centered on baclofen plus sorbitol plus naltrexone, including stereospecific forms such as RS-baclofen and D-sorbitol. In the disclosed experimental and model context, the ternary combinations are reported to downregulate PMP22 expression in Schwann cell assays and to show synergistic increases in MBP/myelination markers in PMP22 transgenic DRG/Schwann co-culture.
The document also reports in vivo functional outcomes in CMT animal models, including behavioral readouts such as bar test latency, grip strength, and gait/inclined plane performance, along with reduced pmp22 RNA and correlation to the behavioral metrics. Additionally, the disclosed therapeutic use includes prevention or reduction of chemotherapy-induced neuropathy, and treatment of ALS in an SOD1G93A rat model.
Claims Coverage
The independent claims cover a ternary pharmaceutical composition, a unit dosage form, and a patient pack. Across these independent claims, the main inventive concept is the same three-ingredient active set (baclofen, sorbitol, naltrexone) with pharmaceutically acceptable salts and excipient/carrier, applied in particular formulation and packaging contexts.
Ternary pharmaceutical composition of baclofen, sorbitol, and naltrexone
A pharmaceutical composition comprising baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof, and a pharmaceutically suitable excipient or carrier.
Unit dosage form with baclofen, sorbitol, and naltrexone
A unit dosage form comprising baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient or carrier.
Patient pack with liquid oral formulation of baclofen, sorbitol, and naltrexone
A patient pack comprising a liquid formulation for oral administration, comprising a composition comprising baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof, a pharmaceutically acceptable excipient or carrier, and optionally instructions for administration of the liquid formulation to a human subject.
Taken together, the independent claims define the same active-ingredient core (baclofen + sorbitol + naltrexone with pharmaceutically acceptable salts) in three deliverable forms: a pharmaceutical composition, a unit dosage form, and a patient pack containing a liquid oral formulation (optionally with instructions).
Stated Advantages
Not explicitly described in patent.
Documented Applications
Charcot-Marie-Tooth Disease Type 1A treatment using the disclosed ternary baclofen/sorbitol/naltrexone combinations.
Prevention or reduction of chemotherapy-induced neuropathy.
Treatment of ALS in an SOD1G93A rat model.
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