New radical cancer therapies are popping up throughout the biotech industry, some of them with such original, even ingenious approaches that they can make you wonder who could even think of using such tactics, let alone engineer them successfully. What follows is a list of four radical and promising cancer treatments I see as plotting a new global course in oncological treatment. Some of these began in small university-based or privately sponsored clinical trials that have already attracted Big Pharma's attention. Others are still small to midsized operations. The former group is the safest in terms of investment with the most solid backing and the latter riskier, but with the most potential upside.
CTL-019, using HIV to kill cancer
CTL-019 was thought up by a University of Pennsylvania biologist named Dr. Carl June. He had what one could describe as a crazy idea - to take a gutted form of HIV, reprogram it with cancer killing genes and mix it with a cancer patient's T-Cells. HIV is very good at invading human T-Cells and staying there, one of the reasons that the disease is still incurable. The idea is that the HIV invades the T-Cells, reprograms them with its cancer-killing genes, and then those cells are released back into the blood stream.
A phase I clinical trial held at Penn administered by one Dr. Stephen Grupp included 12 patients. nine of them are now cancer free. The treatment, however, is not without risk. In activating a patient's immune system to attack its own cancerous blood cells (and there can be pounds of them in the body), the chemical reactions are so fierce and violent that the treatment can itself be life threatening.
CTL-019 has since been acquired by Novartis (NVS), a massive $167B company, which will push the drug through further clinical trials and give June the financial backing to continue development. Novartis' history shows that the company has some savvy when it comes to picking successful oncology drugs. The best example is the company's bestselling drug Gleevec, which raked in $4.7B in 2012. Important to note is that Gleevec's smashing success began much like CTL-019, as a partnership between Novartis and Oregon Health and Science University. Gleevec also holds the record for fastest approved drug by the FDA. If the same story happens with CTL-019 therapy, it could be a huge success.
The Oncovex herpes cancer cure
Using HIV to cure cancer comes across as daringly original. Using a genetically altered deadly virus to reprogram the immune system almost sounds like a microbiology action thriller. HIV, however, isn't the only virus being used to battle cancer. Currently at the tail end of a phase III trial for melanoma patients is Oncovex GM-CSF, a therapy using genetically modified herpes to target cancer cells and destroy them. The 439-patient phase III trial is expected to be completed in September 2014. Oncovex was acquired by Amgen (AMGN) from its creators at Biovex in January 2011 after a 50 patient phase II trial for melanoma showed 10 out of 50 patients, some even with terminal prognoses, undergoing complete remission. That was enough to have Amgen, a $68B company, fork over up to $1B for all of Biovex.
Worldwide, there are 160,000 cases of melanoma diagnosed each year, with an estimated 48,000 deaths. If those phase II results are repeated on a global scale at 20%, Amgen could save close to 10,000 lives every year with Oncovex.
The potential of Oncovex does not stop at melanoma however. Back in 2010, a phase I/II trial of Oncovex for head and neck cancer showed only 2 of 13 patients relapsing. The treatment has also shown effectiveness in battling pancreatic cancer. With this kind of potential being backed by Amgen, I'd say investment is a safe one. The only negative is that Amgen is right near all-time highs, but with a $1.44 yearly dividend, ups and downs can be weathered until September 2014 when the FDA will decide on Oncovex.
Seattle Genetics cancer smart bombs
Seattle Genetics (SGEN) is a different kind of investment opportunity. This is a struggling midcap on the way up, with increasing revenues though still operating at consistent, though shrinking, quarterly losses. Seattle Genetics is currently conducting three different phase III trials for its antibody drug conjugates, on various lymphomas. The runaway costs of three concurrent phase III trials are obviously a big part of the company's losses despite revenue growth, as a midcap conducting the most expensive type of clinical trial, let alone three of them at the same time, can really cause financial hardship on a company this size.
There is light at the end of the tunnel though for Seattle Genetics. The already approved versions of ADCETRIS, the trade name for these antibody drug conjugates, are already increasing SGEN's revenues. Antibody drug conjugates are a fascinating cancer treatment where cancer killing drugs are chemically attached to antibodies that are programmed to latch on to cancer cells. Once the antibody latches on, it releases the drug into the cancer cell, targeting it alone, and killing it. Think of it like a cancer smart bomb equipped with a cancer homing device. These trials have some time before completion, estimated at 2016. Once they are over, SGEN has the real potential of approaching large cap territory. With no debt to speak of on its balance sheet, the company is healthy despite its net losses.
OncoSec's cancer electroshock therapy
OncoSec (ONCS.OB) is an $18M microcap with no revenues, but armed with an elegantly simple technology which, if the Novartis and Amgen takeover examples are any indication, could attract the attention of Big Pharma sooner rather than later. OncoSec calls its cancer treatment platforms Immunopulse and Neopulse. The idea of both of them is to inject either drugs or immune receptors directly into melanoma tumors and then zap the tumors with electricity which opens the cancer cell membranes making them much more susceptible to the cancer treatments. In the case of Immunopulse, the electricity helps the cells absorb an immune response marker that causes the body to target the cancer. In the case of Neopulse, it opens the cells to a toxic cancer drug, helping the drug target only those cells. In the case of Immunopulse, the treatment has led to a systemic immune response in some patients.
The most encouraging thing is that 16% of patients in a phase I trial for Immunopulse showed complete regression, and phase II enrollment is scheduled for completion by the end of this quarter. It only took a 20% melanoma complete regression rate at phase II for Amgen to put up $1B for Biovex. Keep your eyes on this phase II with your fingers on the trigger.
What I'd really like to see is these therapies combined in some meta-approach to finally rid the world of the cancer scourge. With the progress biotech has been making of late, I don't see that as out of the realm of possibility.