Combined use of biotin and thiamine in the treatment of huntington's disease
Inventors
Lucas Lozano, José Javier • PICÓ DEL PINO, Sara • PARRAS RODRÍGUEZ, Alberto • SANTOS GALINDO, María
Assignees
Consejo Superior de Investigaciones Científicas • Centro de Investigación Biomédica en Red
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Publication Number
US-12616680-B2
Publication Date
2026-05-05
Expiration Date
Abstract
The present invention relates to the use of a combination of vitamins, more specifically to the combined use of biotin and thiamine, for the treatment of Huntington's disease. More specifically, the present invention explains that treatment with a combination of biotin and thiamine can improve the neurological, neuroimaging and spectroscopic symptoms associated with Huntington's disease.
Core Innovation
The invention relates to a method for the treatment of Huntington's disease using a composition comprising biotin and thiamine. It links Huntington's disease pathology to thiamine deficiency by reporting altered cytoplasmic polyadenylation element binding protein expression, including altered CPEB1/CPEB4 expression and transcript poly(A) shortening. It further identifies SLC19A3 as a deadenylated target associated with thiamine handling, with decreased SLC19A3 protein and reduced thiamine forms in cerebrospinal fluid and brain, including reduced TPP fraction.
In Huntington's disease settings, the disclosure connects thiamine-related changes with molecular and neurochemical readouts and provides evidence in HD mouse models, including R6/1 and zQ175. The reported results indicate that combined biotin and thiamine improves motor coordination and phenotypes, including rotarod performance, and attenuates striatal atrophy. The disclosure also reports restoration of striatal phosphocreatine measured by magnetic resonance spectroscopy.
Claims Coverage
The provided claims include one independent claim directed to administering an effective amount of a biotin-and-thiamine composition for treatment of Huntington's disease, with dependent claims adding specific ingredient constraints, formulation components, optional additional active ingredients, and administration routes and timing.
Biotin and thiamine treatment of Huntington's disease
A method for the treatment of Huntington's disease comprising administering to a subject in need thereof an effective amount of a composition comprising biotin and thiamine.
Minimum biotin and thiamine amounts
The composition comprises biotin at least 0.40 mg/kg/day and thiamine at least 2 mg/kg/day.
Composition with excipient and/or pharmacologically acceptable carrier
The composition further includes at least one excipient and/or a pharmacologically acceptable carrier.
Optional additional active ingredients selected from named drugs
The active ingredient is chosen from tetrabenazine, haloperidol, chlorpromazine, risperidone, quetiapine, amantadine, levetiracetam, clonazepam, citalopram, escitalopram, fluoxetine, sertraline, olanzapine, valproate, carbamazepine, lamotrigine and/or any combinations thereof.
Permitted routes of administration
The composition is administered via one or more of the oral, sublingual, parenteral, intravenous, intraperitoneal and/or intramuscular routes.
Together, separately, or sequential administration of compounds
The compounds of the composition are formulated or administered together, separately, or sequentially.
The claim coverage centers on treating Huntington's disease by administering an effective amount of a composition comprising biotin and thiamine, with refinements for minimum amounts, excipients and/or pharmacologically acceptable carriers, optional additional named active ingredients, and specified routes and administration timing.
Stated Advantages
Improves motor coordination and phenotypes as indicated by rotarod performance.
Attenuates striatal atrophy.
Restores striatal phosphocreatine as measured by magnetic resonance spectroscopy (MRS).
Documented Applications
Prevention and/or treatment of Huntington's disease in a subject in need thereof using a pharmaceutical composition comprising biotin and thiamine.
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