以下为最近福布斯文章，发表于最近ASH（美国血液学协会）大会之后。在该会议期间，几家主要的CAR-T的生物科技公司包括Kite、Juno都公布了最新临床进展。整体看结果不错，同时可以预期诺华的产品可以在2017年出来，Kite和Juno可能在2016年争取获批。
作为保守投资者，本人目前持有诺华，也持有不少与Kite建立合作关系的安进。现在正考虑少许买进Kite和Juno作为风险投资。仍然有些举棋不定。其中担心的一点是，复发率。目前的临床结果观察的时间在几个月，复发率需要长期观察，会不会大量复发是个担心。另外，Kite的实验再次造成一位病人死亡，致使前段时间股价大跌。既然是VC，就要承担股价风险，这也是个道理。
同样关注这些股票的生物科技爱好者请多给意见！并欢迎加入我的股票群。
Killer T-Cells Provide Hope For Treating Toughest Blood Cancers
- Forbes
At the 57th annual meeting of the American Society of Hematology (ASH), wrapping up today in Orlando, FL, much of the excitement centered around cancer-killing immune cells known as chimeric antigen receptor T-cells, or CARTs. And it’s no wonder. Three companies working on the technology presented promising data of their CARTs from clinical studies in a variety of blood cancers, led by Swiss pharma giant Novartis, which confirmed it is on track to file for FDA approval of its investigational CART, called CTL019, in 2017.
CART therapy involves removing T-cells from the blood of patients and reprogramming those cells so they can recognize and destroy cancer. Novartis drummed up excitement for the technology back in 2014, when 27 out of 30 patients with leukemia—most of them children—went into complete remissions in a trial of CTL019.
Now the number of patients who have been treated with CARTs is well into the hundreds, with heartening results for who are working in the field. “What we’re seeing at this ASH is really a maturing of the data,” says Seth Ettenberg, who spent 10 years on the oncology team at Novartis before joining Boston-based startup Unum Therapeutics, which is also developing T-cell therapies to treat cancer. “As the patient numbers expand, the response rates seem to be holding. Sometimes as you increase patient numbers, it’s hard to maintain that rate, so it’s nice to see it holding.”
Novartis is testing CTL019 in adult and pediatric patients suffering from leukemia or lymphoma. At ASH, the company announced that in an ongoing Phase IIa study, seven out of 15 adults with a common type of hard-to-treat non-Hodgkin lymphoma—diffuse large B cell lymphoma (DLBCL)—responded well to the engineered T-cells. And eight out of 11 patients with follicular lymphoma also responded. The results were similar to previously released data, leading the company to predict the on-time FDA filing.
Separately, researchers at the University of Pennsylvania reported that 55 out of 59 pediatric patients with acute lymphoblastic leukemia (ALL) that had recurred or was not responding to treatment went into remission with CTL019. Of those, 18 were still in remission after a year and nine experienced remissions of more than two years.
Novartis is reporting progress in clinical trials of its engineered T-cell therapy to treat some blood cancers (Credit: FABRICE COFFRINI/AFP/Getty Images).
Some of Novartis’ up-and-coming rivals also had good showings at the ASH meeting. Kite Pharma, which saw its stock shoot up 14% in November to $70, said it had four complete remissions in a Phase I study of seven patients with the same type of lymphoma being investigated in the Novartis trial. The company also announced that the therapy, called KTE-C19, had received “breakthrough” designation from the FDA, which could help speed up its development timeline. Kite says it could be ready to file for approval in 2016.
Juno Therapeutics also presented new data at ASH from trials of two of its experimental CARTs. In a trial of JCAR014, 57% of leukemia patients and 64% of lymphoma patients who were given the most commonly used dosing regimen experiencedcomplete responses. Another experimental T-cell-based therapy from Juno, JCAR015, achieved an 82% complete response rate in patients with ALL.
As is often the case with cancer innovations, the subject of cost came up at the ASH meeting. Because the CARTs that are furthest along in development are personalized to each patient and involve complex manufacturing challenges, they are expected to cost as much as $450,000. Juno CEO Hans Bishop, who spoke at the Forbes Healthcare Summit a few days before the ASH meeting, says worrying about cost is a bad idea.“Just focusing on cost scares me,” he said during the Forbes event. “It will undoubtedly dis-incentivize innovation.”
There are plenty of rivals lining up behind the CART leaders, each with a different approach to the technology. Cellectis has been making waves in the CART space with its version of T-cell therapy, called UCART19, which is designed to be an off-the-shelf option—meaning it would not have to be specially tailored to each patient. At ASH, the company presented a poster describing the first patient to receive the treatment, a baby girl with ALL who went into remission on the therapy.
Celgene and Bluebird Bio are also developing a T-cell therapy, this one designed to recognize a protein present on some cancer cells called B-cell maturation antigen (BCMA). During ASH, an investigator from the National Cancer Institute presented data from a small trial in patients with multiple myeloma, in which one patient who was on the highest dose achieved a complete remission. The two companies are planning larger trials next year.
Unum is in early-stage testing of its platform, called an antibody-coupled T-cell receptor, which involves genetically engineering T-cells and then attaching them to antibodies that target particular tumors. The idea is to improve the ability of T-cells to home in on cancer cells while sparing healthy tissues, which could help reduce the risk of dangerous immune responses, in addition to making T-cell therapy applicable to a wider range of cancers, Ettenberg says.
Unum did not present data at ASH but Ettenberg and other executives attended. Learning from the experiences of Novartis and other companies working on CARTs is helping pave the path for Unum, he says—and bolstering the startup’s confidence in the field of T-cell therapy. “When you enter rooms at ASH where there are presentations on CARTs, they’re packed,” Ettenberg reports. “There’s incredible enthusiasm and interest in the field.”
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