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CAR T-cell therapies show amazing potential in treating blood cancers, but there’s a lot of work to do before the cells are ready for commercialization. Alan Leong, senior analyst with BioWatch, tells The Life Sciences Report about the latest in CAR T-cell therapeutics and how some companies are hedging their bets by developing bispecific antibodies. He also describes the reality behind the growing excitement in regenerative medicine, and what investors should know about some very innovative, but often overlooked, small companies.

The Life Sciences Report: To begin, tell our readers about chimeric antigen receptor (CAR) T-cells and why they are generating so much excitement in the oncology field, particularly among companies targeting hematologic (blood) cancers.

Alan Leong: #39;re seeing remarkable and well-documented response rates for CAR T-cell therapies. These therapeutics are still in the early stages of their lifecycles, so they are likely to evolve and be refined over the next five to 10 years.

What is amazing is the incredible array of companies developing CAR T- cell therapies. They include Juno Therapeutics (JUNO:NASDAQ), Kite Pharma (KITE:NASDAQ),Bellicum Pharmaceuticals Inc. (BLCM:NASDAQ), Intrexon Corp. (XON:NYSE),bluebird bio Inc. (BLUE:NASDAQ), Celgene Corp. (CELG:NASDAQ), Selexis SA (private) and many others. These companies are very good at stimulating T cells to be highly active cancer cell killers. Each company is trying to develop its own process and targets, but what excites everyone is the chance to target the CD3 receptor to activate T cells.

Activating T cells through the CD3 receptor, however, has drawbacks that may limit CAR T-cell therapeutics to hematologic cancers. Because CD3 activation highly stimulates the T cells, we sometimes see something called cytokine release syndrome. In this syndrome, the body mounts an extreme response, in which T cells target major organs in an autoimmune shutdown. This reaction is potentially fatal, so currently, when the leading CAR T-cell companies enroll patients in a CAR T-cell trial, those patients are put into intensive care wards immediately. Companies are trying to find solutions to minimize that risk. If they succeed, they may be able to target solid tumors.

If I had a caution about CAR T-cell therapy, it would be that the cost is extremely high. To put this in context, Provenge (sipuleucel-T), by Dendreon Corp. (DNDN:NASDAQ), debuted in 2010 at $93,000 ($93K) for a three-infusion regimen, and was criticized for being so expensive. CAR T-cell therapy is much more complex and, therefore, even more expensive. In some cases CAR T-cell therapies also are less homogenous, so there may be substantial batch-to-batch variation.

TLSR: If the CAR T-cell therapeutics are as effective as early results indicate they may be, would their higher costs be worthwhile for payers?

AL: Right now, the actual costs of CAR T-cell therapeutics are somewhat opaque, but people are talking about an initial price of $250K per patient. Some suggest payers will provide reimbursement based on Gilead Sciences Inc.’s (GILD:NASDAQ) Sovaldi (sofosbuvir) experience: A 12-week regimen for that hepatitis C therapeutic costs $84K. I always retort, “Well, if there were three or four Sovaldis out at the same time, would they have paid it?” The answer is no.

I think we’ll see some interesting movement over the next year or two as people raise questions regarding the ability to commercialize CAR T-cell therapies. Nonetheless, this is a time to be excited. We’re seeing response rates we only dreamed of years ago.

TLSR: Do you see any alternative therapies in development?

AL: We’re entering the age of targeted medicines. This is an exciting time. In the past decade Seattle Genetics (SGEN:NASDAQ) brought out Adcetris (brentuximab vedotin), which elicits amazing response rates in lymphomas. Then Medivation Inc. (MDVN:NASDAQ) introduced a prostate cancer medication. Now we have a plethora of targets and technologies.

CAR T-cell therapy is expensive, but many companies are creating bispecific antibodies that may recreate what CAR Ts do at a fraction of the cost. Bispecific antibodies are engineered to go after two or more targets. In their favor, bispecifics are much less expensive and much more standardized than CAR Ts, so they probably don’t have to be as effective to be commercially successful. They just have to beeffective. Therefore, I raise a caution regarding CAR Ts and bispecifics because they’re going after very similar targets. Perhaps both can be successful.

TLSR: Are you seeing any trends among all the companies that have the CAR T-cells out in late-stage research?

AL: On the business side, we’re seeing an interesting number of collaborations occurring with one major partner or multiple smaller partners. That’s true for bispecifics, too. Juno and bluebird each recently concluded deals with Celgene. So companies are getting support, whether from the equity markets or from partners, to help them reach their next milestones.

That funding is being used primarily to improve their processes. That includes identifying the source of the cells; the most effective methods to condition, transect or modify the cells to become CAR T cells; criteria to select T cells; and the best ways to replicate them in vitro. The methodologies used today will continue to improve.