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These Promising Cancer Therapies Are a Biotech "Triple Play"

On Jan. 7, 2015, CytRX Corp. (NASDAQ: CYTR) announced positive results from a study of its experimental brain cancer drug, aldoxorubicin, and the company's stock immediately jumped 15.8%. Then, over the next four months, it nearly doubled.

Over the months since June 2013, when Clovis Oncology Inc. (NASDAQ: CLVS) released positive trial data for both a lung cancer and a lymphoma drug, CLVS share value has soared 255%.

And just last summer, when Puma Biotechnology Inc. (NYSE: PBYI) released positive results from a clinical trial of its breast cancer drug, neratanib, PBYI shares jumped 300% in a single session.

It's clear that investors can do very well with the right cancer drug.

But there, of course, is the rub: Finding the right cancer drug is not so easy.

I'm about to show you three companies – all sound investments – running strong in the race to fight this scourge, and the one that could very well take the prize…

Biotech Investing's Biggest Challenge

According to a study published in the September 2013 issue of Clinical Pharmacology and Therapeutics, only 9.8% of all experimental drugs designed to treat solid tumors make it to final FDA approval – fewer than one in 10.

So the odds are not in your favor.

The same study, however, pinpointed one area of cancer research where success isn't so elusive: treatments for blood cancers, such as leukemias and lymphomas. In fact, more than one out of every three (36%) of these compounds will make it to market.

Some companies are making significant strides in the war against these diseases right now.

One approach, called chimeric antigen receptor (CAR) T-cell therapy, represents the cutting edge of current oncology. It involves removing T cells (immune cells produced by the thymus gland) from a cancer patient, genetically altering them so they can detect the individual's specific cancer, and injecting them back into the bloodstream, where they can directly attack the disease. It's a combination of immunotherapy, cell therapy, and gene therapy.

CAR-T has yielded some impressive results.

Researchers at Juno Therapeutics Inc. (NASDAQ: JUNO), for example, announced that in an ongoing phase 1 trial of its CAR-T cell therapy,  JCAR015, 24 of 27 adults with acute lymphoblastic leukemia (ALL), all of whom had ceased responding to other therapies, went into remission. Six of those patients remained disease-free for more than a year. Without CAR-T, most would likely have died within weeks or months.

As a result, Juno had a huge IPO in 2014 and saw its valuation grow from $2 billion to $4.7 billion in the first month. The company plans to test CAR-T cells in clinical trials against six different types of cancer by the end of next year.

Bellicum Pharmaceuticals Inc. (NASDAQ: BLCM), another small biotech developing CAR T-cell therapies, is creating CAR-Ts that not only recognize cancer cells, but also respond to a medical "on-off switch," a drug called rimiducid, which, in the presence of cancer, can awaken the CAR-Ts from a dormant state and send them to work.

If either the drug or cancer cells are absent, the T cells turn off. Having the switch helps control the toxicity of the therapy. The company calls their cells "GoCAR-Ts."

Bellicum's cell therapy for hematologic cancers, BPX-401, is in preclinical development for the treatment of acute ALL, chronic lymphocytic leukemia (CLL), and certain types of non-Hodgkin's lymphoma. The company says, "We have generated preclinical proof-of-principle data in vitro showing that BPX-401 has significant CAR-T cell activation and proliferation potential, and may be more effective in killing cancer cells compared to other CAR-T constructs."

Bellicum's share price currently hovers around $23, with a market cap of $597 million, but those numbers should jump considerably higher as the company's CAR-T compounds move into clinical trials.

The "Fly in the Ointment"

For all of their promise, current CAR-T therapies come with significant downside: They can kill you.

A number of deaths have occurred in CAR-T trial studies, both at JUNO's Labs and at the University of Pennsylvania. The deaths resulted from cytokine syndrome, a release of inflammatory immune substances such as interferon, interleukin, and various growth factors, in response to T-cell activation.

These so-called "cytokine storms" can result in high fever, muscle aches, low blood pressure, delirium, and excess fluid in the lungs.

Both Juno and Bellicum are working on strategies to lower the risk of this kind of toxic response, but whether their strategies will work remains to be seen.

So for all of its promise, CAR-T may represent merely a transitional point to another type of therapy altogether – one that is safer and more effective, and may even prove to be a "magic bullet" for all cancers.

Join the conversation. Click here to jump to comments…

About the Author

Ernie Tremblay has more than 25 years of experience in following and analyzing the latest developments in health, medicine, and related technologies. He understands the FDA approval process, as well as the "hard science" behind new, experimental drugs and the market demand for them - and has a comprehensive grasp of the complex dynamics that determine whether a new drug will be a breakthrough winner, or just another casualty of the FDA approval process.

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  1. Dieter Hovekamp | June 20, 2015

    There is something to learn: the cure for cancer is nonsense !
    As there is not ONE cancer to cure there will be not ONE magic bullet for "all" cancers.
    But we a close to many cures to many if not all cancers. CAR-T is certainly a good one – but there are many sorts of CAR-T already targeting different but also the same kind of cancers. Then there are checkpoint inhibitors and oncolytic virus therapies as well as all sorts of antibody bi- and tri-specifics etc. Common to all: they make use of the natural immune system.
    Closer to "magic bullets" are combinatons of these technologies to better/more effectively target tumors and – perhaps – one other kind of T-cell therapies focussed on TCRs, which bring about 9-fold as many targets in much finer grains as CAR-T and all the others.
    Juno and e.g. Kite are now looking in that TCR direction too, but the clear leader in this field is Adaptimmune together with its sister company Immunocore both UK spinouts of German Medigene AG, which is also developing its own set of immunotherapies – including one being a library of TCRs.
    The speciality of Adaptimmune and Immunocore is their sophisticated engineering of 'supernatural' TCRs for highly specific targets. They have developed about 30 such engineered TCRs together, 8-10 are validated now and 3 (targeting NY-ESO, LAGE, and gp100) are in the clinic – each for many different kinds of cancers and working against hematological as well as solid tumors. They aim to add 2-5 new TCR targets each year.
    (see, to understand the difference to CAR-T / antibodies etc or watch Medigenes instructive videos )

  2. Ernie Tremblay | June 22, 2015

    Dieter, thanks for your comments. You’ve brought up some interesting points.

    As I’m sure you’re aware, the scientific community once believed—or at least hoped—that each of the more than two hundred forms of human cancer would be associated with its own, discrete gene mutation. I say “hoped” because that would imply every tumor type would have a specific, identifiable therapeutic target. We would need to find a lot of cures, but it was a doable challenge.

    It turned out, however, that most cancers don’t arise from a single mutation. In fact, range of various mutations, working in combination, could produce the same cancer in different individuals. That meant tumors were highly individualized, and the number of therapies to treat them would be countless.

    Thus the advent of “personalized” or “specific” medicine, such as CAR-T therapy. Each tumor would have its own cure. Still, that presented a huge challenge—understanding the particular array of mutations in every patient’s cancer.

    Fortunately, at the same time, we came to learn that there was another, simpler approach—targeting biological pathways, instead of genes, that are disrupted by genetic mutations. That cut the number of therapeutic targets way down, because it turned out that groups of seemingly unrelated cancers had something in common—they disrupted the same pathways. So that implied on medication for multiple indications.
    Not a magic bullet yet, but much closer to one.

    Of course, tumors have other things in common—unrestrained cell proliferation, inhibited senescence, resistance to apoptosis, etc.—but it has been unclear how we might use those characteristics to fight the disease.

    Now researchers have discovered something all cancers, or at least the vast majority, have in common—CD47 expression. And that is a target. In xenotypes (murine transplantations of human malignancies), this approach has proven successful, in varying degrees, against a long list of cancers.

    So in fact, a magic bullet may now be in the realm of possibility.

    As to TCR, I agree with you, it’s a very interesting technology. But to say it’s “in the clinic,” may be a little misleading. That implies it’s been approved and is in regular therapeutic use. Adaptimmune has TCR clinical candidates in very early stage (phase 1/2) clinical trials for two indications: an extremely rare cancer called synovial carcinoma, and a hematologic cancer called multiple myloma.

    At this stage of research, the emphasis is on establishing safety and dosage rather than efficacy. It may turn out that TCR is in fact a leader in the field of therapeutic candidates, but we won’t know that until R&D is a little farther down the road.

    In the meantime, we do know that Juno’s (Nasdaq:JUNO) CAR has achieved complete responses in more than 90% of treated human patients, adults and children, with B cell malignancies, and that it will likely receive FDA approval in 2017. I would look for approval of Adaptimmune’s first product somewhat later than that.

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