Methods for treatment of motor neuron diseases

Enrique Brandan; David Gonzalez

Abstract

In one aspect of the invention, a method is provided for treating a MND, The method comprises administering to a subject in need thereof, an effective amount of an anti-CTGF agent, thereby treating the MND, in some embodiments, the MND is selected from the grou consisting of amyotrophic lateral sclerosis, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, progressive bulbar palsy, pseudobulbar palsy, spinal-bulbar muscular atrophy, hereditary spastic paraplegia and post-polio syndrome. In particular embodiments, the MND is amyotrophic lateral sclerosis. In another aspect of the invention, a method is provided for preventing or reducing a clinical symptom of a MND. The method comprises administering to a subject in need thereof an effective amount of an anti-CTGF agent, thereby preventing or reducing a clinical syrnptom of "the MND.. in some embodiments, the clinical symptom of the MND is selected from the group consisting of dysphagia, dysarthria, weight loss, muscle weakness, muscle fatigue, muscle spasticity, hyperreflexia and joint or muscle contractures, fasciculation or pain. Another aspect, a method is provided for preventing the development of, or reducing the rate of progression of a MND . The method comprises administering to a subject in need thereof an effective amount of an anti-CTGF agent, thereby preventing the development of, or reducing the rate of progression of a MND. In a further aspect, a method is provided for preventing or reducing muscle damage in a subject with muscular dystrophy, the method comprising administering to the subject a therapeutically effective amount of an anti-CTGF agent, thereb preventing or reducing muscle damage in the subject. In certain embodiments, the muscle damage is skeletal muscle damage. These and other methods of the invention are accomplished by administering an anti-CTGF agent to the subject with a MND. In particular embodiments, the anti-CTGF agent is selected from the group consisting of anti-CTGF antibodies, anti-CTGF antibody fragments, antibody mimeties and anti-CTGF oligonucleotides. In some embodiments, the anti-CTGF agent is an anti-CTGF antibody. In further embodiments, the anti-CTGF antibody is a human or humanized antibody. In a specific embodiment, the anti-CTGF antibody is identical to CLN1 or mAbl as described in U.S.. Patent No. 7,405,274. In further embodiments, the anti-CTGF antibody binds to domain 2 of human CTGF. In other embodiments, the anti-CTGF antibody binds to the same epitope as CLN1 or mAbl. In additional embodiments, the anti-CTGF oligonucleotide is selected from the grou consisting, of anti-CTGF antisense oligonucleotides, anti-CTGF siRNAs, anti-CTGF shRNAs, anti-CTGF miRMAs and anti-CTGF rfbozymes, In certain embodiments, the anti-CTGF agent is used in combination with another therapeutic modality, in particular embodiments, the anti-CTGF agent; is used in combination with an effective amount of riluzole. These and other embodiments of the invention will readily occur to those of skill in the art in light of the disclosure herein, and ail such embodiments are specifically contemplated. Each of the limitations of the invention can encompass various embodiments of the. invention, it is, therefore, anticipated that each of the limitations of the invention involving any one element or combinations of elements can be included in each aspect of the invention. This invention is not limited in its application to the details of construction and the arrangement of components set forth in the following description or illustrated in the drawings. The invention is capable of other embodiments and of being practiced or of being earned out in various ways.

Más información

Fecha de publicación: 2017
Idioma: CTGF/CCN2, FG-3019, Amyotrophic Lateral Sclerosis, motor neuron diseases, fibrosis
URL: https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2017100193&tab=PCTDESCRIPTION
DOI:

WO/2017/100193