I-Corps: Drug delivery systems for treating degenerative brain diseases

这个I-Corps项目的更广泛的影响/商业潜力是改善目前治疗帕金森病（PD）的护理标准，使PD患者和医疗保健从业人员受益。迄今为止，没有治愈方法可以逆转PD的病程，目前的护理标准通常侧重于管理运动症状（震颤，僵硬和步态丧失）。 开发了一种新的药物递送平台，以有效地将促智剂递送到PD患者的大脑，以减轻症状并可能逆转疾病的进程。 这种新的药物输送平台旨在逆转症状和认知能力下降，同时通过一种非侵入性设备提高患者的依从性，这种设备可以以负担得起的方式补充或取代当前的标准方法，而不会引起副作用。除了帕金森病，新的药物输送平台可用于治疗阿尔茨海默病和路易体痴呆症。这个I-Corps项目开发了一个平台，用于重新配制口服形式的促智药，用于帕金森病（PD）患者的鼻内输送和使用。食用这些促智剂的口服形式的主要限制之一是这些化合物不能有效地到达血脑屏障，并且在肝脏和胃肠道隔室中被广泛代谢。 这种无法到达靶器官的情况限制了药物在人类中的潜在认知和脑保护能力，并限制了药物在治疗脑退行性疾病（如PD）方面的疗效。先前的临床前研究工作表明，新的鼻内制剂可以在大脑中递送高浓度的促智剂，以有效激活脑保护信号通路并提高能量输出，从而更好地管理PD症状。为了通过鼻途径有效和安全地输送到大脑，鼻内制剂与一系列鼻内装置兼容，包括鼻喷雾剂，涂抹器和雾化器。该奖项反映了NSF的法定使命，并通过使用基金会的知识价值和更广泛的影响审查标准进行评估，被认为值得支持。

The broader impact/commercial potential of this I-Corps project is the improvement of the current standard of care for treating Parkinson's disease (PD), benefiting PD patients and healthcare practitioners. To date, there is no cure to reverse the course of PD and the current standard of care often focuses on managing motor symptoms (tremors, rigidity and loss of gait). A new drug delivery platform is developed to efficiently deliver nootropic agents to the brain in PD patients to alleviate symptoms and potentially reverse the course of disease. This new drug delivery platform was designed to reverse symptoms and cognitive decline while enhancing patient compliance though a non-invasive device that could complement or replace the current standard methods in an affordable manner without causing side effects. Beyond PD, the new drug delivery platform may be used to treat Alzheimer's disease and Lewy Body Dementia.This I-Corps project develops a platform for reformulating oral forms of nootropic agents for intranasal delivery and use by Parkinson's disease (PD) patients. One of the major limitations of consuming oral versions of these nootropic agents is that these compounds do not efficiently reach the blood brain barrier and are extensively metabolized in the liver and gastrointestinal compartments. This inability to reach the target organs limits the drug's potential cognitive and brain-protective abilities in humans and limits drug efficacy in managing brain degenerative disorders such as PD. Prior preclinical research endeavors showed that the new intranasal formulations can deliver nootropic agents in high concentration in the brain in order to efficiently activate brain-protective signaling pathways and elevate energy output which can lead to better management of PD symptoms. For efficient and safe delivery to the brain via the nasal route, the intranasal formulations are compatible with a range of intranasal devices including nasal sprays, applicators, and atomizers.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.