权益分类	功能权益	普通用户	{{item.name}}会员
{{category.name}}	{{benefitItem.name}}

Identification of the Antisickling Compounds in SCD-101

SCD-101 中抗镰化化合物的鉴定

基本信息

批准号：
9407759
负责人：
Robert Swift
金额：
$ 22.47万
依托单位：
INVENUX, LLC
依托单位国家：
美国
项目类别：
财政年份：
2017
资助国家：
美国
起止时间：
2017-07-17 至 2020-01-16
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9407759
关键词：
Acute Adult Adverse effects Affect Age American Antineoplastic Agents Antisickling Agents Biological Assay Birth Blood Botanicals Cells Cessation of life Child Clinical Research Colorado Deoxygenated Sickle Hemoglobin Developing Countries Development Disease Disease Progression Dose Drug Kinetics Erythrocytes Event Fatigue Fractionation Genes Goals Grant Hemoglobin Hereditary Disease Hospitalization Human In Vitro Libraries Liquid Chromatography Mass Fragmentography Measures Medical Molecular Mus Mutation Neurocognitive Opiates Organ Organ failure Pain Participant Pathogenesis Patients Pediatric Hospitals Pharmaceutical Preparations Pharmacodynamics Pharmacology Pharmacotherapy Phase Philadelphia Placebos Plasma Polymers Property Proteomics Quality of life Renal function Research Sickle Cell Sickle Cell Anemia Signs and Symptoms Sorghum Sorghum vulgare Spleen Stroke Structure Symptoms Testing Transgenic Mice Transgenic Organisms United States United States Food and Drug Administration Universities Variant Visit Work beta Globin chemical fingerprinting chronic pain clinical development common symptom cost drug development follow-up hydroxyurea in vivo metabolomics mortality mouse model multiorgan damage polymerization sickling trait treatment duration

项目摘要

ABSTRACT Sickle cell disease (SCD) affects approximately 100,000 people in the United States and millions worldwide. Symptoms appear shortly after birth, and in less developed countries the majority of children with SCD die before the age of five. In the U.S., SCD patients suffer chronic pain and fatigue, severe acute painful crises requiring hospitalization and opiates, strokes, and multi-organ damage, and have an average mortality in their 40s. The only FDA approved drug for adults with SCD is the anticancer drug hydroxyurea. New treatments are desperately needed for both children and adults with SCD. SCD-101 is a botanical drug that inhibits red blood cell sickling in vitro and in vivo. The formation of sickle shaped red blood cells, caused by the polymerization of deoxygenated sickle hemoglobin, is the primary and causative event in the molecular pathogenesis of sickle cell disease; therefore, inhibition of sickling by SCD-101 should ameliorate the signs, symptoms, and slow the progression of sickle cell disease. In a Phase 1B study, SCD-101 was shown to be safe and reduced pain and fatigue, the two most common symptoms of sickle cell disease. The compounds in SCD-101 responsible for the antisickling activity are unknown, as is the mechanism of antisickling. Identification of the antisickling compounds would assist in clinical development and may enable the development of a more potent and effective drug for the treatment of sickle cell disease. Identification of the antisickling compounds would assist in the elucidation of the mechanism by which SCD-101 inhibits red blood cell sickling and point the way to a new target for antisickling drugs, and the development of a new class of compounds to treat sickle cell disease.

摘要