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Publication Number
US-8586770-B2
Publication Date
2013-11-19
Expiration Date
Abstract
The invention relates to methods to manipulate stem cells in vivo and in vitro to treat, e.g., a condition where cell or tissue repair is needed.
Core Innovation
The subject matter defines a steroid-based or steroid-like structure with substituents R1 through R5, optional double bonds, and stereochemical configurations including cis/trans and α/β relationships. The compounds are described with broad variable groups and structural embodiments, including aromatic A-ring embodiments, epoxide, cyclopropyl, spiro rings, acetal motifs, and related ring structures.
Permissible substituent options include hydroxyl, ester, ether, halogen, and optionally substituted amine selections, together with oxygen-, nitrogen-, sulfur-, and carbon-linked moieties. The disclosure further includes phosphate, phosphothioester, phosphonoester, thiophosphate, oxime, sulfite, sulfate, sulfonamide, sulfamate, sulfonate, sulfamide, sulfinamide, carbonate, carbamate, acyl, thioacyl, and protected forms such as amides and esters.
The document also frames the compounds as pharmaceutical compositions, medicaments, formulations, and excipients, and includes nonaqueous liquid formulations with very low water content. It further describes systemic delivery strategies with controlled peak timing and sustained exposure, depot formulations, multiple dosing regimens, and monitoring of immune response changes in therapeutic contexts.
Claims Coverage
The consolidated claim coverage centers on a steroid-based or steroid-like compound scaffold defined by optional double bond(s) and enumerated substituent selections for R1-R5. Across the provided items, four recurring inventive feature sets are present, with dependent claims further narrowing R4 and, in some claim sets, R2 and R3.
Substituent-defined steroid-like compound with R1-R5 functional-group restrictions
A compound having a steroid-like structure wherein R1 is OH, ester or ether; R2 is OH, an oxygen linked ester or an oxygen linked ether; R3 is OH, ester, ether or halogen; R4 is OH, ester, ether or optionally substituted amine; and R5 is CH3, CH2OH, CHO, C2H5 or C3H7.
Optional double bond steroid with R1-R5 constraints
A compound having the structure wherein the dotted line or dotted lines are optional double bond(s), with R1, R2, R3, R4, and R5 restricted to the enumerated hydroxyl, ester, ether, halogen, and optionally substituted amine options, and R5 restricted to CH3, CH2OH, CHO, C2H5 or C3H7.
R4-enumerated amine-containing substituent set
Dependent claim coverage further specifies R4 selections including O-C(O)-CH3, O-C(O)-CH2CH3, NH-C(O)-CH3, NH-C(O)-OCH3, NHCH3, NH(CH3)2, alkylamine, dialkylamine, an N-linked carbamate, an N-linked amino acid, or an N-linked heterocycle.
Restricted R2 and R3 options including fluorine
Dependent claim coverage narrows R2 and R3 to defined functional-group sets, including R2 as OH or O-C(O)-CH3 and R3 as OH, F, or O-C(O)-CH3 in some claim sets.
Across the independent claims, the inventive scope is directed to a steroid-based or steroid-like compound scaffold defined by optional double bond(s) and specific allowable substituent sets at R1-R5. Dependent claims further narrow permissible substituent selections, especially R4 and, in some claims, R2 and R3, while preserving the same core scaffold and enumerated functional-group framework.
Stated Advantages
The delivery strategies are described as obtaining defined peak timing and sustained exposure after dosing regimens, including short courses that still lead to sustained levels.
The patent states that systemic delivery is linked to targeting serum, blood, or tissue concentration ranges in the nM range and maintaining sustained levels after short courses.
The formulations are described as nonaqueous liquid formulations characterized by very low water content.
Modulation of chemokines and cytokines, including MCP-1, IL-1, IL-6, and TNF-α.
Reduced amyloid-β deposition.
Enhanced production, survival, and differentiation of hematopoietic cells.
Treatment or management of delayed radiation effects after ionizing radiation exposure.
The temperature and hematology approach is used to predict survival and lethality with stated confidence levels.
Documented Applications
Immune modulation and infection treatment.
Autoimmune, allergy, inflammation, cardiovascular, metabolic disorders, and respiratory or pulmonary conditions.
Hematopoiesis-related uses such as neutropenia and thrombocytopenia.
Viral infection contexts including HCV, HIV, SIV, and SHIV.
Core-body temperature monitoring with hematology endpoints to predict survival and lethality status profiles for subjects exposed to radiation.
Characterizing biological activity of formula 1 compounds by exposing subjects to LD50/30 or LD50/60 insults, treating with the compound, and measuring survival.
Therapeutic use for chronic/progressive neurological disorders, including multiple sclerosis and Alzheimer’s disease.
Treatment or management of skin inflammatory/atrophic/rash conditions using topical and systemic formulations.
Prevention or treatment of delayed radiation effects beginning at ≥2 weeks after ionizing radiation exposure.
Preventing or treating blood cell deficiencies, immune dysregulation, inflammatory and allergy-related conditions, immune suppression, infections, cancer, neurological disorders, cardiovascular disorders, pulmonary disorders, trauma, hemorrhage, bone loss, endocrine deficiencies, and congenital or hereditary disorders.
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