Preclinical Development of Tacrolimus for Radiation Cystitis

他克莫司治疗放射性膀胱炎的临床前开发

• 批准号: 8888531
• 负责人: JONATHAN H KAUFMAN
• 金额: $ 64.85万
• 依托单位: LIPELLA PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-05-12 至 2017-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8888531
• 关键词: Basic Science; Biological Availability; Bladder; Canis familiaris; Caring; Cessation of life; Clinical; Clinical Research; Collaborations; Cyclophosphamide; Cystitis; Data; Development; Dinoprostone; Dose; Drug Formulations; Drug Kinetics; Dysuria; Foundations; Funding; Goals; Health; Hematuria; Hemorrhage; Histopathology; Hospitals; Immunosuppressive Agents; Incidence; Inflammatory; Intellectual Property; Interleukin-2; Intravenous; Intravesical Administration; Kidney; Kidney Function Tests; Lead; Letters; Life; Liposomes; Marketing; Medical; Modeling; Oral; Orphan; Patients; Pelvic Cancer; Pharmaceutical Preparations; Pharmacologic Substance; Phase; Property Rights; Quality of life; Radiation; Radiation therapy; Rare Diseases; Rattus; Reaction; Research; Research Proposals; Research Support; Rights; Route; Safety; Small Business Innovation Research Grant; Staining method; Stains; Tacrolimus; Testing; Therapeutic; Therapeutics for Rare and Neglected Diseases; Tissues; Toxic effect; Toxicology; United States National Institutes of Health; Universities; Urinary tract; Urine; Urothelium; Woman; Work; commercialization; effective therapy; follow-up; human WFDC2 protein; innovation; intravesical; lower urinary tract symptoms; lymph nodes; medical schools; men; novel; phase 1 study; pre-clinical; programs; research study; urologic

DESCRIPTION (provided by applicant): This project, entitied "Preclinical Development of Tracrolimus for Radiation Cystitis" will fund key experiments to advance the commercial development of LP-10. LP-10 is a liposomal tacrolimus formulation for local (topical) intravesical administration to the urinary bladder. The formulation provides active drug levels in the bladder with significantly reduced systemic levels. Radiation cystitis is a rare disease defined by lower urinary tract symptoms that include dysuria, hematuria, and hemorrhage. There are currently no approved therapies to treat RC, which can severely degrade a patient's quality of life, require long-term follow-up treatment, and, in some patients, lead to death. This project is public-private collaboration between Lipella Pharmaceuticals Inc., the University of Pittsburgh School of Medicine, and William Beaumont Hospital. Lipella owns intellectual property rights to the LP-10 formulation as well as rights associated with its recent receipt of orphan designation from the FDA. The FDA has also provided Lipella with pre-clinical requirements for IND consideration of LP-10. These requirements constitute the studies described in this proposal. The scope of work includes further pharmaceutical and pharmacological characterization of LP-10 (Phase I) and IND-enabling studies (Phase II). This research is translational, and bridges basic research to clinical development. Progress in this direction will directly support our ultimate goal of developing a safe and effective therapy ready for commercialization.

描述（由申请人提供）：该项目名为“曲克莫司治疗放射性膀胱炎的临床前开发”，将资助关键实验，以推进LP-10的商业开发。LP-10是一种用于膀胱局部（局部）膀胱内给药的他克莫司脂质体制剂。该制剂提供膀胱中的活性药物水平，同时显著降低全身水平。放射性膀胱炎是一种罕见的疾病，其下尿路症状包括排尿困难、血尿和出血。目前还没有批准的治疗RC的疗法，RC会严重降低患者的生活质量，需要长期随访治疗，并且在一些患者中导致死亡。这个项目是公私合营的 Lipella Pharmaceuticals Inc.，匹兹堡大学医学院和威廉博蒙医院。Lipella拥有LP-10配方的知识产权，以及最近从FDA获得孤儿药资格的相关权利。FDA还向Lipella提供了LP-10 IND考虑的临床前要求。这些要求构成了本提案中所述的研究。工作范围包括LP-10（I期）和IND使能研究（II期）的进一步药物和药理学表征。这项研究是转化性的，是基础研究与临床开发的桥梁。这一方向的进展将直接支持我们开发安全有效、可商业化的治疗方法的最终目标。