Novel Medical Food for the Management of Iron Deficiency Anemia

 描述(申请人提供)：我们将进行一项临床研究，以测试一种治疗缺铁性贫血的新型专利医疗食品。该项目意义重大，因为它解决了世界上头号营养失调问题；以及全球范围内的一个健康问题，在全球成本上仅次于结核病。据估计，约有20亿人患有缺铁性贫血，占总人口的30%以上。缺铁影响到婴儿、幼儿和育龄妇女，并且在老年人中的重要性正在增加。缺铁性贫血的影响范围从缺铁性贫血(IDA)疲劳，到心肌病，再到儿童ADHD风险增加和老年人认知能力下降。口服铁剂是缺铁性贫血患者的一线治疗方法。目前针对缺铁症的治疗策略并不充分，原因有很多，其中最主要的原因是吸收不良。据报道，在接受口服铁剂治疗的患者中，至少有40%出现不良事件，包括严重的胃肠道不适、恶心、上腹部不适、疼痛、呕吐和便秘。这些AE的结果是治疗的持久性低。我们正在提出一种新的铁输送系统，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。我们推测，在营养酵母中表达的H-铁蛋白可能是口腔铁的重要来源。我们已经在两个动物模型上成功地进行了临床前研究。我们的产品在许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，并将我们的产品与使用植物铁蛋白的产品区分开来，植物铁蛋白更类似于铁蛋白的L亚基。我们和其他人已经证明，L铁蛋白不容易被体内的器官摄取。从植物铁蛋白中吸收铁比从铁盐中吸收铁更好，后者是目前的护理标准。其次，我们的产品可以经济地大规模生产，并在受控的实验室条件下生长，从而确保铁和铁蛋白的数量保持一致。这种方法还将我们的产品与生物农业方法区分开来，在生物农业方法中，土壤中的铁量不一致，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，因为我们有一种新的方法，使铁在一种通常被许多文化直接消费的食物中更具生物利用率。通过在人类铁蛋白中提供铁，我们正在提供一种安全、自然和高效的机制来在饮食中输送铁；这种方法类似于从母乳中输送铁。在这项研究中，我们建议 1b期人体递增剂量试验，以确定我们产品的安全性和耐受性。临床数据预计将引起功能食品和医疗食品行业的巨大兴趣，我们可以与这些行业合作，为世界提供铁缺乏的治疗。

 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.