Novel Medical Food for the Management of Iron Deficiency Anemia

治疗缺铁性贫血的新型医疗食品

• 批准号: 9136952
• 负责人: Darren Wolfe
• 金额: $ 22.48万
• 依托单位: SIDERO BIOSCIENCE, LLC
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-01 至 2019-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9136952
• 关键词: Address; Adverse effects; Adverse event; Affect; Agreement; Anemia; Animal Model; Attention deficit hyperactivity disorder; Bioavailable; Blood - brain barrier anatomy; Bolus Infusion; Brain; Cadmium; Cardiomyopathies; Child; Chronic; Clinical Data; Clinical Research; Constipation; Consumption; Contracts; Data; Diet; Discipline of Nursing; Dizziness; Dose; Dried Yeast; Elderly; Enzymes; Epigastric; Fatigue; Female of child bearing age; Ferritin; Ferrous Sulfate; Food; Food Industry; Formulation; Functional disorder; General Practitioners; Government; H ferritin; Health; Health Food; Heart; Hospitals; Human; Human Milk; Human body; Impaired cognition; Individual; Infant; Injection of therapeutic agent; Internet; Iron; Iron Compounds; Iron deficiency anemia; Kidney; Knowledge; L Forms; L-ferritin; Laboratories; Legal patent; Letters; Licensing; Liver; Malnutrition; Marketing; Measures; Mediating; Medical center; Metals; Methods; Milk; Mitochondria; Mothers; Nature; Nausea; Nutrition Disorders; Nutritional; Oral; Organ; Organic Iron Compounds; Pain; Pallor; Pathway interactions; Patients; Pharmacologic Substance; Phase; Plants; Population; Production; Proteins; Public Health; Reporting; Rights; Risk; Rodent; Safety; Salts; Scientist; Serum; Shortness of Breath; Small Business Technology Transfer Research; Soil; Source; Symptoms; System; Technology; Testing; Toxic effect; Tuberculosis; United Nations; Universities; Vomiting; Work; World Health Organization; Yeasts; absorption; base; commercial application; commercialization; cost; design; dietary supplements; functional food; human data; human subject; improved; indexing; innovation; interest; iron deficiency; medical food; meetings; nonhuman primate; novel; novel strategies; phytate; pre-clinical; preclinical study; primary outcome; programs; public health relevance; standard of care; toxic metal; treatment strategy

 DESCRIPTION (provided by applicant): We will perform a clinical study to test a novel, patented medical food to manage iron deficiency anemia. The project is significant because it addresses the number one nutritional disorder in the world; and a health problem on the global scale that is second only to tuberculosis in cost worldwide. Estimates are that approximately 2 billion people, more than 30% of the population, suffers from iron deficiency anemia. Iron deficiency affects infants, young children and women of child bearing age and is increasing in significance among the elderly. The implications of iron deficiency anemia range from (IDA) fatigue, to cardiomyopathy to increased risk of ADHD in children and cognitive decline in the elderly. Oral iron therapy is first line treatment for patients with IDA. The current treatment strategies for iron deficiency are inadequate for a number of reasons chief among them is poor absorption. Adverse events (AE) are reported in at least 40% of individuals receiving oral iron and include significant GI discomfort, nausea, epigastric discomfort, pain, vomiting and constipation. These AE's result in low treatment persistency. We are proposing a novel iron delivery system that is based on our discovery that iron can be taken up by a number of organs including the brain via H-ferritin. We hypothesized that H-ferritin expressed in nutritional yeast could be a significant source of oral iron. We have performed successful preclinical studies in two animal models. Our product is innovative on a number of levels. First of all, our choice to use the H-subunit of ferritin is novel and differentiates our product from those using plant ferritins which are more similar to the L-subunit of ferritin. We and others have shown that L-ferritin is not readily taken up by organs in the body. Absorption of iron from plant ferritin is n better than from iron salts which are the current standard of care. Secondly, our product can be produced economically in large scale and is grown in controlled laboratory conditions allowing for consistent quantities of iron and ferritin. This approach also differentiates our product from the bioagriculture approach where the amount of iron in the soil is inconsistent and cannot be controlled. Plant ferritin can also accumulate metals from the soil other than iron including potentially toxic metals such as cadmium. In addition, plants have phytates that limit iron absorption. Thus this project is innovative because we have a novel approach to make iron more bioavailable in a food that is commonly consumed directly by many cultures. By providing iron in human ferritin, we are providing a safe, natural and efficient mechanism for delivering iron in the diet; an approach that mimics iron delivery from breast milk. In this study, we propose a Phase 1b ascending dose trial in humans to determine safety and tolerability of our product. Clinical data are expected to generate significant interest among those in the functional food and medical foods industry with whom we can partner to provide the world with a treatment for iron deficiency.

 描述（由申请人提供）：我们将进行一项临床研究，以测试一种新型的专利医疗食品，以管理缺铁性贫血。该项目意义重大，因为它解决了世界上头号营养失调问题;这是一个全球范围的健康问题，在全世界范围内的成本仅次于结核病。据估计，约有20亿人，即超过30%的人口患有缺铁性贫血。铁缺乏影响婴儿、幼儿和育龄妇女，在老年人中的影响日益严重。缺铁性贫血的影响范围从（IDA）疲劳到心肌病，再到儿童多动症风险增加和老年人认知能力下降。口服铁剂治疗是IDA患者的一线治疗。目前治疗缺铁症的策略是不充分的，原因有很多，其中最主要的是吸收不良。在至少40%接受口服铁的个体中报告了不良事件（AE），包括显著的GI不适、恶心、上腹部不适、疼痛、呕吐和便秘。这些AE导致治疗持续时间较低。我们提出了一种新的铁递送系统，该系统基于我们的发现，即铁可以通过H-铁蛋白被包括大脑在内的许多器官吸收。我们假设，在营养酵母中表达的H-铁蛋白可能是口服铁的重要来源。我们已经在两种动物模型中进行了成功的临床前研究。我们的产品在很多层面上都是创新的。首先，我们选择使用铁蛋白的H-亚基是新颖的，并且将我们的产品与使用与铁蛋白的L-亚基更相似的植物铁蛋白的产品区分开来。我们和其他人已经表明，L-铁蛋白不容易被体内器官吸收。从植物铁蛋白中吸收铁比从铁盐中吸收铁更好，铁盐是目前的护理标准。其次，我们的产品可以大规模经济地生产，并在受控的实验室条件下生长，从而使铁和铁蛋白的数量保持一致。这种方法也将我们的产品与生物农业方法区分开来，后者土壤中的铁含量不一致，无法控制。植物铁蛋白还可以从土壤中积累铁以外的金属，包括潜在的有毒金属，如镉。此外，植物含有限制铁吸收的植酸盐。因此，这个项目是创新的，因为我们有一个新的方法，使铁更生物可利用的食物，通常是直接消费的许多文化。通过在人类铁蛋白中提供铁，我们提供了一种安全，天然和有效的机制，用于在饮食中提供铁;一种模拟母乳铁输送的方法。在这项研究中，我们建议 一项1b期人体剂量递增试验，以确定我们产品的安全性和耐受性。临床数据预计将在功能性食品和医疗食品行业引起极大的兴趣，我们可以与他们合作，为世界提供治疗缺铁症的方法。