报告从政策环境、市场行情、厂商动态、科技研发、国际资讯、行业数据、分析评论等几个方面对医药行业进行了分析评论。

报告从医药行业事件分析、竞争环境、竞争对手、行业数据等几个方面进行了分析评论。

Fabry disease is an inheritable lysosomal storage disease characterized by excessive accumulation of GL-3 in lysosomes. It results from the deficiency of α-Gal and can result in symptoms such as fatigue, angiokeratoma, tinnitus, kidney failure, heart disorders and anervous system problems. Fabry disease results from mutations in the GLA gene resulting in decreased activity or complete absence of α-Gal.

Prostate cancer is cancer in the prostate, which is an exocrine gland present in the male reproductive system. The main function of the prostate gland is to make fluid for semen, a white substance that contains sperm. The risk of developing prostate cancer is age-dependent. Prostate cancer affects men mostly over the age of 65 and rarely occurs in men younger than 40 years of age.

Inverse planning is at the heart of prostate Volumetric Modulated Arc Therapy (VMAT) treatment procedure and critically determines its level of success. As practiced now, the capacity of VMAT is greatly underutilized because of inferior computing performance of existing optimization methods. An alternative mathematical approach that improves both the efficiency and the efficacy is needed and is the center of this research. We propose to develop a new innovative inverse planning tool, based on the novel idea of superiorization, to replace the classical constrained optimization approaches employed in clinics today for prostate VMAT cases. Year 1 of the training award focused on formulating the VMAT problem as a constrained superiorization problem and on the development of a framework of fast converging inverse planning algorithms. Empty solution sets and infeasibility constraints that often exist in real-world applications were incorporated into the model. The new framework was proven mathematically and its efficacy was demonstrated when it was compared to a classical optimization method. The superiorization methodology was Implemented, tested and evaluated on a previously treated prostate case where good initial results were obtained.

Previously, we completed the molecular/ biochemical characterization of several shortlisted candidate gene fusions from the transcriptome sequencing of over 70 previously validated samples. From these studies, we identified two rare but recurrent gene fusions in breast cancer cell lines and tissues involving the MAST and Notch genes. Both of these fusion genes are potentially targetable and patients harboring MAST or Notch fusions may benefit from MAST or Notch inhibitors. We also describe a novel study of cancer-specific pseudogenes, including those in breast cancer. Most recently, through our clinical sequencing initiative, we discovered a series of activating mutations in the estrogen receptor (ESR1) in breast cancer patients. These activating mutations in ESR1 are a key mechanism in acquired endocrine resistance in breast cancer therapy. Overall, these discoveries made over the funding period contribute towards the understanding of the molecular and genetic etiology of breast cancer that will advance the development of targeted therapies.

This grant prepares DANA (Defense Automated Neurobehavioral Assessment), the next-generation neurocognitive assessment tool (NCAT), for transition into operational military use. DANA is a clinical decision support tool developed for and funded by the Department of Defense (DOD) for use in field and clinical settings. The effort is organized around two foci science and transition. The science concentrates on CONUS-based studies such as testing DANA in clinical drug, fatigue/alertness, concussion and/or depression protocols. The second thrust, transition, includes obtaining FDA clearance for DANA (DOD has determined it is a medical device), and positioning DANA to be operationally deployed into the military.