Experimental Gene Therapy Successful In Certain Lymphomas And Leukemia
MD安德森新闻发布2014年12月08日
Sleeping Beauty gene study shows encouraging results in patients with lymphoid malignancies
MD安德森新闻发布12/08/2014
研究CD19针对嵌合抗原受体（轿车）治疗的研究结果使用睡美的非病毒转导系统来改变T细胞已经证明了晚期血液学恶性肿瘤患者的进一步承诺。
患有急性淋巴细胞白血病（全部），非霍奇金淋巴瘤（NHL）或慢性淋巴细胞白血病（CLL）的患者是临床试验的一部分The University of Texas MD Anderson Cancer Center使用睡美人基因转移系统最初在明尼苏达大学发现。
研究的结果呈现在美国血液学学会（ASH）年度会议的第56次年会in San Francisco and were published in the Dec. 5 issue of the ASH journalBlood.
睡美观基因被命名为其“唤醒”一种灭绝的转座子 - DNA，可以复制并将复制恢复到基因组中。这允许将基因转移到称为质粒的DNA分子中。一种称为转座酶的酶与质粒结合，将转座子和基因从质粒中切割并粘贴到靶DNA序列中。该基因转移系统是MD Anderson临床试验的基础。
Using the Sleeping Beauty gene transfer system, Laurence Cooper, M.D., Ph.D., professor of pediatrics andPartow Kebriaei, M.D.,副教授干细胞移植和细胞疗法, were able to plug a gene into T cells, creating an artificial or chimeric antigen receptor (CAR) on the T cell that recognizes and binds to CD19, a cell surface on B cells. The resultant product known as CAR T cells are produced at MD Anderson and are being employed in the Sleeping Beauty clinical trials.
“我们正在使用汽车T细胞与常规血液干细胞移植组合使用汽车T细胞治疗晚期CD19阳性血液学恶性肿瘤的患者，”凯布赖厄说。“我们还对患有活跃疾病但没有接受血液干细胞移植的患者。”
Patients were recipients of autologous (patient’s own cells) or allogeneic (donor cells) stem cell transplantations, which were administered in combination with CAR. Kebriaei reported no acute or long-term toxicity in the 33 patients treated.
“Five patients at high risk for relapse were treated with CAR T cells along with autologous stem cell transplant, and four of those patients remain in complete remission with a median follow-up of 12 months,” she said. “Among 13 patients treated with donor CAR T cells after allogeneic stem cell transplantation, six remain in complete remission with a median follow-up of 7.5 months.”
Kebriaei also reported that five out of 14 patients treated with CAR T cells for active disease who did not undergo blood stem cell transplantation showed disease regression with a median follow-up of six months.
MD Anderson学习参与者包括Helen Huls，Harjeet Singh，Ph.D.，Simon Olivares，Matthew Figliola，Sourindra Maiti，Ph.D.，Su Shihuang，Ph.D.，Pappanebank Kumar，Bipulendu Jena，Ph.D.，SonnyAng，Ph.D.和Jackson Rineka，所有与儿科;Marie Andree忘了，黑色素瘤医学肿瘤;刘刘，伊恩麦尼，博士，加布里埃拉朗顿M.D.，Chitra Horings，M.D.，伊丽莎白Shpall，M.D.和Richard Champlin，M.D.，干细胞移植和细胞疗法;威廉威尔达，M.D.，Ph.D.，Susan O'Brien，M.D.，Michael Keating，MBB和Hagop Kantarjian，M.D.，白血病。还参加了明尼苏达大学的佩里哈克特，博士学位，博士学位，遗传细胞生物学和发展。
The study was funded by the National Institutes of Health and the Cancer Prevention and Research Institute of Texas. Continued development of this approach is proceeding under MD Anderson’s Moon Shots program to accelerate the pace of converting discoveries into clinical advances that reduced cancer deaths.