2014年12月6日讯 /生物谷BIOON/ --嵌合抗原受体-T细胞（CAR-T）疗法代表着当今最先进的肿瘤免疫细胞治疗技术，在相关临床试验中取得了巨大的成功，该领域已成为药品研发最热门的领域之一，也是投资合作的大热点。目前，在CAR-T领域，诺华（Novartis）处于领先地位，其临床试验中有开发用于白血病、淋巴瘤、间皮瘤和胰腺癌的实验性产品。今年7月，该公司开发的CAR-T免疫疗法CTL019收获了FDA的突破性疗法认定。
在CAR-T领域中，诺华的主要竞争对手为生物技术新贵Juno Therapeutics，该公司开发的一款CAR-T免疫疗法JCAR015近日也获得了FDA的突破性疗法认定，其他跟进者包括凯特药业（Kite Pharma）、基因治疗先驱蓝鸟生物（Bluebird bio）、生物技术巨头新基（Celgene）。今年6月，辉瑞与Cellectis签署高达29亿美元协议宣布进入CAR-T领域，蓝鸟生物7月收购Pregenen意在进一步奠定CAR-T领域地位。而强生（JNJ）在11月与Transposagen签署3亿美元合作，也宣布进军CAR-T领域。
近日，Juno Therapeutics与美国国家癌症研究所（NCI）达成一项免疫肿瘤学交易，从NCI授权获得了一种互补性的CAR-T产品，意在扩充CAR-T资产。此次交易，也标志着Juno公司CAR-T资产增添了第4种临床CAR-T产品。除了FDA已授予突破性疗法认定的JCAR015，该公司另外2个产品，JCAR017开发用于ALL和NHL，JCAR014目前正处于早期研究开发用于各种B细胞恶性肿瘤。
从NCI授权获得的互补性CAR-T产品，与Juno公司的产品类似，其原理是从患者体内获得T细胞，并利用嵌合抗原受体（CARs）武装它们，使这些T细胞能够结合癌细胞表达的抗原。之后，经过武装并增殖的T细胞重新注入患者体内，这些细胞便能够追踪恶性肿瘤细胞并发起攻击。
除了与Juno合作外，NCI已联合Opus启动了一项I期临床研究的患者招募，调查该CAR-T产品用于复发性/难治性急性淋巴细胞白血病（ALL）和非霍奇金淋巴瘤（NHL）儿童和青少年患者的治疗。
英文原文：Juno expands its CAR-T arsenal with latest immuno-oncology deal
Seattle's Juno Therapeutics, at work on treatments that train the immune system to better fight cancer, has signed a deal to expand its pipeline of therapies, licensing a complementary candidate developed by the National Cancer Institute.
Under a deal with NCI partner Opus Bio, Juno has paid an undisclosed sum to license an immunotherapy that targets CD22, a protein expressed on B cell leukemias and lymphomas. Juno's work in the field has thus far focused on CD19, another protein common among blood cancers.
Much like the rest of Juno's pipeline, the NCI treatment is a CAR-T immunotherapy, in which scientists remove T cells from a patient's blood and equip them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting cells are reinjected into the patient, at which point they track down malignancies and attack them as they would any commonplace infection.
NCI, in collaboration with Opus, has already begun enrollment in Phase I study testing the candidate in children and adolescents with relapsed/refractory acute lymphoblastic leukemia (ALL) or non-Hodgkin's lymphoma (NHL).
The deal adds a fourth clinical CAR-T candidate to Juno's portfolio of potential therapies. Leading the way is JCAR015, an ALL treatment whose stellar Phase I results led the FDA to grant a breakthrough therapy designation last month, promising an expedited regulatory review. Behind that is JCAR017, in development for ALL and NHL, followed by JCAR014, which is undergoing early studies on various B cell malignancies.
Meanwhile, Juno is queued up to make a splash on Wall Street after filing for a $150 million IPO last month. The biotech has turned heads since its launch last year, raising more than $300 million in venture cash and reporting excellent clinical results for its cadre of cancer treatments, including complete remission rates above 90% in ALL.
Now it's locked in competition with Novartis ($NVS), which is developing a similar--and similarly promising--CAR-T therapy alongside the University of Pennsylvania. And the technology's promise has brought a host of contenders to the table, including oncology heavyweight Celgene ($CELG), which reached out to gene therapy pioneer bluebird bio ($BLUE) to get to work in the space, and Santa Monica, CA's Kite Pharma ($KITE), which grossed more than $140 million in a June IPO on the strength of its approach to T cell modification. Late-comer Johnson & Johnson ($JNJ) has shouldered its way into CAR-T with a $292 million deal signed last month.