有希望的胰腺癌新疗法向前发展

SALT LAKE CITY – Even among cancers, pancreatic cancer is an especially sinister form of disease. 一年存活率极低, 治疗进展也落后于许多其他恶性肿瘤.

A study published today in the journal Nature Medicine led by researchers at 亨茨曼癌症研究所 （HCI）在 犹他大学 (U of U) describes a new therapeutic approach with potential for patients with pancreatic cancer. These researchers discovered a combination drug therapy that may effectively comb在 disease. HCI researchers first observed anti-cancer impacts in a laboratory setting 和, 随后, 第一次用在人类病人身上.

The study has already progressed to a clinical trial that is now open at HCI 和 will soon be open at other sites in the United States. 有关这项名为THREAD的临床试验的详细信息，请参见 国家临床试验编号03825289. The combination therapy uses two drugs already approved for use by the Food 和 Drug Administration for other diseases, 包括癌症. 这种新组合药物是通过口服药丸给药的.

胰腺肿瘤的特征是KRAS基因的突变. 当KRAS以这种方式突变时, it sends constant signals that promote abnormal cell division 和 growth in cancer cells. 结果，肿瘤就会失去控制. 同时, 像所有细胞一样, pancreatic cancer cells must recycle their components to provide building blocks for new growth in an essential cell function known as autophagy. Previous studies to combat pancreatic cancer that were focused either on the role of KRAS or on impacting autophagy were not effective.

新的HCI研究, using an approach that simultaneously targets both abnormal KRAS signaling 和 the autophagy process, shows a strong response in mouse models 和 may be a promising therapy for patients with pancreatic cancer. 柯南金赛，医学博士，博士, a physician-scientist at 亨茨曼癌症研究所 和 the Department of Internal Medicine 在 U of U 和 马丁·麦克马洪博士, a cancer researcher at HCI 和 Professor of Dermatology 在 U of U, led the study.

“大发娱乐能够观察到这两种药物的结合, 单独使用时, don’t have much of an impact on the disease – appears to have a very potent impact on the growth of pancreatic cancer,麦克马洪说. “大发娱乐在实验室的培养皿中观察到了这一点, 然后在小鼠模型中, 现在在一个胰腺癌患者的治疗中. 事实上, we proceeded from a petri dish to a patient in less than two years – a timeline that is rarely seen in medical science."

The HCI-led research is bolstered by a separate study published in the same issue of the journal. This study outlines complementary findings regarding the effects of autophagy in pancreatic cancer in the laboratory setting 和 was led by Channing Der, 博士学位, 莎拉·格雷厄姆·凯南, 博士学位, 柯尔斯顿·布莱恩特, 博士学位, 在 University of North Carolina (UNC) Lineberger Comprehensive Cancer Center. McMahon 和 Der learned about the parallel nature of their research programs at a scientific meeting one year ago. Given the critical need for advances in pancreatic cancer therapies 和 the promise of their collective findings, 他们在同伴的基础上共同努力推进学习.

在大发娱乐的论文中, we show the response of a pancreatic cancer patient who had received surgery 和 multiple lines of chemotherapy prior to this combination,金赛说, 谁也是病人的医生. “这个病人, 此后又有谁死于这种疾病, 尽管如此，对这些药物有几个月的显著反应. We need to carefully evaluate this new combination therapy in the context of clinical trials to better underst和 if good responses might be seen in multiple patients. We also need to identify the specific features of any patient who may benefit, 在提出任何关于大规模使用的建议之前."

These preliminary findings are being rigorously scrutinized in clinical trials to observe 和 underst和 whether the combination of these drugs is safe 和 effective for pancreatic cancer patients. The trial is underway at HCI 和 is underway or planned 在 University of California, 旧金山, 以及纽约的哥伦比亚大学.

"In publishing how this new approach of mixing two medicines typically associated with malaria 和 melanoma can treat pancreatic tumors with KRAS mutations, we are hoping the clinical trial supported 由胰腺癌协会大发娱乐提供’s New Therapies Challenge Grant will help us learn how to best administer these medicines, 这样大发娱乐就能让尽可能多的病人受益,大卫·特维森说, MD, 博士学位, Lustgarten的首席科学家, 冷泉港实验室癌症中心主任, 集体的共同科学领袖.

hci主导的这项研究得到了美国国家癌症研究所的支持, including grant P30 CA042014; the Melanoma 研究 Alliance; 5 For The Fight; 和 Huntsman Cancer Foundation. 一项最近授予HCI的麦克马洪的资助, 与Eric Collisson合作, 医学博士（加州大学旧金山分校）, 由胰腺癌协会大发娱乐提供, a partnership between the Lustgarten Foundation 和 St和 Up To Cancer (SU2C), will carry forward future laboratory work on this project 和 also support the THREAD clinical trial. The UNC-led study was supported by the Pancreatic Cancer Action Network (PanCAN) 和 included collaborating partners from The Translational Genomics 研究 Institute (TGen).