PFI: Partnerships for Biomarker Research and Innovation Enabled by a Flow Cytometry Center

这个创新合作伙伴关系（PFI）项目-凯克应用生命科学研究生院（KGI），NSF PFI毕业生（0332749）和哈维穆德学院（HMC）之间的III型（A：C）合作伙伴关系，一个新的PFI计划机构（定义为从未被PFI资助的机构）-寻求在KGI建立一个生物标志物研究中心，拥有最先进的流式细胞术设施，以加速发现和商业化新的人类罕见疾病的疾病特异性生物标志物。 对罕见病的关注很重要，因为常规商业化途径忽视了这一领域。 该中心将通过与患者倡导团体和疾病基金会合作，确定受影响患者人群的生物标志物，帮助降低治疗罕见疾病药物的开发风险。与国家罕见病组织（诺德）合作，将确定能够获得患者样本的罕见病组织，并探索合作行动。将在流式细胞术机构中处理从罕见疾病人群中获得的人类样本。将使用荧光抗体通过流式细胞术鉴定参与信号传导途径的蛋白质。将使用贝叶斯网络方法分析细胞计数结果，以识别可能是决定性疾病生物标志物的信号转导中的中心分子。这些活动将促进生物标志物的发现，并提供一种对疾病人群进行分类和分层的方法。通过选择罕见疾病作为生物标志物发现的重点，该项目将对所需治疗的发展产生更广泛的影响。该中心将创建一个罕见疾病生物标志物数据库，并根据生物标志物的应用，协助疾病组织开展风险慈善活动，以帮助消除风险。对特定条件高度区分的生物标志物将被确定用于专利申请和随后的许可活动。整个生物标志物数据库也将供学术界和企业界访问，以便进行后续数据挖掘。 理想情况下，有兴趣进一步开发这些数据集的公司将启动新的项目。此外，该项目将满足一个关键的工作需求：翻译科学家的教育，个人谁可以将发现转化为商业产品。 该中心将教育凯基大学的生物科学硕士（MBS）学生和HMC的本科工程学生开发生物标志物发现的诊断工具。该项目的合作伙伴是学术机构：凯克应用生命科学研究生院（KGI）（牵头机构）和哈维穆德学院（HMC）;以及私营部门组织：Beckman Coulter，Inc（BCI）和国家罕见疾病组织（诺德）。

This Partnerships for Innovation (PFI)project--a Type III (A:C) partnership between Keck Graduate Institute of Applied Life Sciences (KGI), a NSF PFI graduate (0332749), and Harvey Mudd College (HMC), an institution new to the PFI Program (defined as one that has never been a PFI grantee)--seeks to establish a Center for Biomarkers Research at KGI, with a state-of-the-art flow cytometry facility in order to accelerate discovery and commercialization of new disease-specific biomarkers for rare human diseases. The focus on rare diseases is important because routine commercialization pathways neglect this area. The Center will help take risk out of the development of drugs that treat rare diseases by working with patient advocacy groups and disease foundations to identify biomarkers in affected patient populations. Partnering with the National Organizations for Rare Diseases (NORD), rare disease organizations that have access to patient samples will be identified and collaborative operations will be explored. Human samples obtained from rare disease populations will be processed in the flow cytometry facility. Proteins involved in signaling pathways will be identified by flow cytometry using fluorescent antibodies. Cytometry results will be analyzed with a Bayesian network approach to identify central molecules in signal transduction that might be determinative disease biomarkers. These activities will facilitate biomarker discovery and provide a means of classifying and stratifying disease populations Through the choice of rare diseases as a focus for biomarker discovery, the project will achieve a broader impact on the development of needed treatments. The Center will create a database of rare disease biomarkers and assist disease organizations in their venture philanthropic efforts to help remove risk, based on biomarker applications. Biomarkers highly discriminating for specific conditions will be identified for patenting and subsequent licensing activity. The entire database of biomarkers will also be accessible to the academic and corporate communities for subsequent data mining. Ideally, companies interested in further developing these data sets will initiate new projects. Also, the project will meet a critical work need: the education of translational scientists, individuals who can translate a discovery into commercial products. The Center will educate Master of Bioscience (MBS) students at KGI and undergraduate engineering students at HMC in the development of diagnostic tools from biomarkers discovery.Partners at the inception of the project are Academic Institutions: Keck Graduate Institute of Applied Life Sciences (KGI) (lead institution), and Harvey Mudd College (HMC); and Private Sector Organizations: Beckman Coulter, Inc (BCI) and The National Organization for Rare Disorders (NORD).