CAR T-cell therapy
美国东部时间1月30日，美国临床肿瘤学会（ASCO®）发布了《2018临床肿瘤学进展》，过继性细胞免疫治疗（CAR-T疗法）被评选为最具影响力的“年度进展（Advance of the year）”CAR-T细胞疗法是使用患者自身的白血细胞，在实验室进行基因重编后回输患者体内以对抗癌症。
2017年8月，FDA批准了首个过继性细胞免疫治疗——CAR-T疗法以及首个癌症基因治疗tisagenlecleucel（用于编辑免疫细胞）。Tisagenlecleucel可能是首个真正改变急性淋巴细胞白血病(ALL)治疗的疗法。ALL是儿童最常见的癌症之一，在一项临床试验中，tisagenlecleucel治疗5例患儿后，有4例获得了疾病缓解。
2017年10月，FDA批准了第二项CAR T细胞疗法——axicabtagene ciloleucel获批治疗某些特定类型的淋巴瘤（弥漫大B细胞淋巴瘤等）成年患者。
此外，在多发性骨髓瘤的研究中，CAR-T疗法也显示了令人鼓舞的前景。
CAR-T疗法代表了治疗手段上令人兴奋的创新，有可能对临床实践产生变革性的影响。
参考英文文献源自Clinical Cancer Advance of the Year Is CAR T Cells - Medscape - Jan 30, 2018.具体如下：
网页
Cancer experts looking back over the past 12 months have highlighted adoptive immunotherapy with chimeric antigen receptor (CAR) T cells as the most important clinical cancer advance of the year.
The announcement comes in Clinical Cancer Advances 2018, the annual report from the American Society of Clinical Oncology (ASCO) in which a team of cancer experts reviewed developments from November 2016 through October 2017.
In the course of that year, 31 new therapies for more than 16 types of cancer were approved by the US Food and Drug Administration (FDA).
Highlighted as the ASCO Advance of the Year is the first CAR T-cell therapy to be approved — tisagenlecleucel (Kymriah, Novartis) for acute lymphoblastic leukemia (ALL).
This product scored a double first, the report notes — it was the first adoptive cell immunotherapy and the first gene therapy for cancer to be approved by the FDA.
"Even more important than the historic significance of this achievement is the medical need this unique new therapy is poised to fill," the authors write.
"Tisagenlecleucel may be the first treatment to truly turn the tables on recurrent pediatric acute lymphoblastic leukemia (ALL), one of the most common cancers in children," they write. "In a clinical trial, in four of five patients, cancer went into remission after the treatment."
This represents one of the most remarkable advances in the treatment of childhood cancer in the last decade.
This product "can eradicate relapsed ALL in children. This represents one of the most remarkable advances in the treatment of childhood cancer in the last decade and could dramatically change treatment paradigms for this disease," the report authors enthuse.
The report notes that a second CAR T-cell therapy, axicabtagene ciloleucel (Yescarta, Kite Pharma), was approved to treat adults with certain types of lymphoma. More are in development, targeting other hematologic malignancies, including multiple myeloma.
"CAR T-cell therapy represents an exciting innovation that has the potential to transform cancer care," the report states. However, "it also raises the ongoing issue of cost and reminds us that, as a community, we need to find solutions that will assure that every patient with cancer has access to the care they need."
As previously reported by Medscape Medical News, CAR T-cell therapies come with an astronomical price tag (Kymriah is priced at $475,000 and Yescarta at $373,000), and there have been problems with arranging for reimbursement.
血液病患者未来一片光明