Sanford-Burnham Medical Research Institute at Lake Nona, 6400 Sanger Road, Orlando, FL 32827, USA.
J Cardiovasc Transl Res. 2010 Oct;3(5):431-7. doi: 10.1007/s12265-010-9204-8. Epub 2010 Jul 13.
The flow of innovative, effective, and safe new drugs from pharmaceutical laboratories for the treatment and prevention of cardiovascular disease has slowed to a trickle. While the need for breakthrough cardiovascular disease drugs is still paramount, the incentive to develop these agents has been blunted by burgeoning clinical development costs coupled with a heightened risk of failure due to the unprecedented nature of the emerging drug targets and increasingly challenging regulatory environment. A fuller understanding of the drug targets and employing novel biomarker strategies in clinical trials should serve to mitigate the risk. In any event, these current challenges have evoked changing trends in the pharmaceutical industry, which have created an opportunity for non-profit biomedical research institutions to play a pivotal partnering role in early stage drug discovery. The obvious strengths of academic research institutions is the breadth of their scientific programs and the ability and motivation to "go deep" to identify and characterize new target pathways that unlock the specific mysteries of cardiovascular diseases--leading to a bounty of novel therapeutic targets and prescient biomarkers. However, success in the drug discovery arena within the academic environment is contingent upon assembling the requisite infrastructure, annexing the talent to interrogate and validate the drug targets, and building translational bridges with pharmaceutical organizations and patient-oriented researchers.
创新、有效和安全的新药从制药实验室治疗和预防心血管疾病的流动已经减缓到涓涓细流。虽然对突破性心血管疾病药物的需求仍然至关重要，但由于新兴药物靶点的前所未有的性质和日益具有挑战性的监管环境，开发这些药物的动力已经减弱，加上临床开发成本的不断增加，失败的风险也随之增加。更全面地了解药物靶点，并在临床试验中采用新型生物标志物策略，应该有助于降低风险。无论如何，这些当前的挑战引发了制药行业的变化趋势，为非营利性生物医学研究机构在早期药物发现中发挥关键的合作作用创造了机会。学术研究机构的明显优势在于其科学计划的广度，以及“深入”识别和表征新靶点途径的能力和动力，这些途径可以揭示心血管疾病的具体奥秘，从而产生大量新的治疗靶点和有先见之明的生物标志物。然而，在学术环境中成功进行药物发现取决于能否建立必要的基础设施，能否吸引人才来探究和验证药物靶点，以及与制药组织和以患者为中心的研究人员建立转化桥梁。
