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.
This Is Nothing Less Than the Future of Cancer Therapy
When cells of any sort grow old, sick, or damaged, the immune system creates large hunter/scavenger cells called macrophages that find, engulf, and devour them. The process is called phagocytosis.
Nearby healthy cells avoid this fate because they carry significant amounts of a signal protein, CD47, on their surfaces, which acts like a flashing sign that says, "Do not eat me!"
Unhealthy cells lose much of their ability to express CD47 and also give off other signals that attract hungry macrophages. They quickly become lunch.
But cancer cells, even though they're diseased, have developed the ability to make a lot of CD47 and camouflage themselves with it to avoid certain destruction.
That suggests a new approach to fighting the disease: CD47 blockade. Currently, scientists are developing ways to prevent cancer cells from producing the protein, leaving them with no place to hide.
Much of the research is being done by a group of researchers under the leadership of Irving Weissman, M.D., at Stanford University, where they're working to develop a monoclonal antibody that will unmask cancer cells for hunting macrophages.
Of more interest to investors, however, is the CD47 inhibitor under development at an immuno-oncology development biotech called Trillium Therapeutics Inc. (NASDAQ: TRIL).
Trillium's inhibitor, SIRPαFc, is a fusion protein that binds with CD47 and prevents it from broadcasting its "don't eat me" signal.
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The company is currently planning to begin a phase 1 clinical trial in H2 2015 to test the efficacy of SIRPαFc on acute myeloid leukemia (AML). It has already demonstrated that SIRPαFc binds well with AML-expressed CD47 in xenograft models. (A xenograft is a human tumor that has been grafted onto an animal host.)
This approach to fighting blood cancers is extremely promising, but it's not the end of the story. As it turns out, CD47 blockade may be a treatment that works against all cancers - there are more than 200 variations - because all malignant tumors, no matter the type, express CD47 for survival. Until now, researchers have assumed there would never be this kind of "magic bullet."
The science behind this approach is well established, and the possibility of efficacy in humans is promising. The more immediate question, however, concerns safety, because theoretically, CD47 inhibitors could bind with healthy red blood cells, making them targets for phagocytosis.
With SIRPαFc, this turns out not to be the case, as it binds well with cancer cells, but poorly with blood cells. This may give it a safety advantage over the monoclonal antibody approach at Stanford, and also a similar program at Celgene Corp. (NASDAQ: CELG), in partnership with a privately owned biotech called Inhibrx.
The Best Investment Opportunity of Them All
Trillium is a remarkable investment opportunity. For AML alone, assuming an annual treatment cost of around $75,000 per year, averaged worldwide, and a peak market penetration of 23%, sales could top $900 million for this indication alone.
But TRIL isn't stopping with AML. It's also looking into indications for other blood cancers and solid tumors as well.
If CD47 blockade works as well as the science predicts it should, TRIL's earning potential will be unlimited.
All in all, Juno and Bellicum remain sound investment opportunities for now, but in the long run, Trillium may well win the race.
More from Ernie
They call it the "Boomer Virus" and it kills every 58 seconds. A highly contagious virus is sweeping the globe and has made its way to the United States. It's 1,000 times more deadly than Ebola and kills every 58 seconds. Millions of Americans are already infected and don't even know it... and Boomers are five times more likely to contract this infection. Fortunately, a tiny $7 company is providing a patented medical breakthrough that could save 75 million Americans from a grisly death. Do not ignore this warning. It's a matter of life and death. Continue reading here...
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.