Mayo’s Precision Chemotherapy Delivery Platform Attracts Investor Attention
Precisely targeting cancerous cells with chemotherapy drugs while leaving healthy cells untouched is a long-sought but elusive holy grail among disease researchers. If a successful way could be found to deliver a “payload” of designer drugs directly into the belly of cancer cells without side effects, it could change all kinds of medical and market paradigms.
One Mayo Clinic researcher is making progress on just that front, and the technology platform has already been licensed by famed biotech serial entrepreneur Steve Gorlin, who is raising millions for a new start-up firm.
In the fight against cancer, current chemotherapy is a blunt instrument: The drugs used can indeed kill cancerous tumors, but they cannot be given in large enough doses to be consistently effective because of the side effects they cause. Healthy cells that also divide quickly, such as red blood cells in bone marrow, are also swept up in the toxic effects of chemo drugs, thus severely limiting their usefulness.
Instead, an increasingly popular cancer research focus has been on “targeted therapies” in which drugs are tailored to zero in on differences between cancer cells and healthy cells, such as an overexpression of vascular endothelial growth factor (VEGF), a protein associated with cell growth.
Common chemotherapy drugs such as aflibercept and bevacizumab can inhibit VEGF and thus control or slow tumor growth, and if delivered precisely on target, can be rendered more effective than they are currently.
Enter Dr. Svetomir Markovic, a Mayo Medical School professor and faculty researcher at its Cancer Center in Rochester. One of his focus areas is the development of a new kind of molecular platform that has been shown capable of carrying what he calls a “car bomb” of anti-cancer drugs into tumor cells – and then delivering their “payloads” once inside.
The delivery platform, he said, is “a commercially available nanoparticle called Abraxane, which after years of effort, we can now ‘coat’ with monoclonal antibodies. The antibodies can direct this sphere into directions that we want to take it, for instance, to target VEGF, which is highly expressed in human cancer cells.
“We were able to load these nanoparticles of Abraxane with roughly 700 antibodies in the appropriate orientation so that their ‘binding sites’ toward the VEGF were pointing outward. Thus this ‘sea urchin’ could actually attach specifically to where we wanted it go, and release its payload within the belly of the tumor cell.”
The upshot of mouse studies, chronicled this month in the journal of the American Association for Cancer Research, is that the platform is able to produce more antitumor efficacy from existing chemotherapy drugs by increasing the amount of them aimed directly at tumor cells.
“In mice, we were able to get more of the cancer drug into the tumor and away from normal tissue,” Markovic said. “We took this to the FDA and now we are just one patient away from completing a Phase One human clinical trial of our first-generation molecule for treatment of advanced melanoma. What I can tell you is that the results so far are encouraging enough that we’re opening the study up to include patients with ovarian cancer.
“We’ll also be seeking approval from the FDA to include breast cancer patients and other types of cancer as well.”
The prospect of developing an effective method of precisely targeting tumors with chemotherapy drugs is a cherished one – something that has the potential to finally solve the vexing conundrum of toxic side effects and truly unlock the efficacy potential of a long menu of anti-cancer drugs.
Whether the Mayo platform will ultimately be the answer, of course, remains to be seen, but it has at least one major believer: well-known biotech venture capitalist and “serial entrepreneur” Steve Gorlin.
Dr. Markovic confirmed the platform technology has been licensed by a Georgia-based entity called Vavotar Life Sciences LLC. According to SEC filings, Gorlin, of Aspen, Colorado, is an executive officer and board member of the firm, which was founded last year.
A Regulation D filing from May 16 indicated Vavotar had raised $3.5 million in an equity financing round that began in March.
Described in media accounts as a longtime behind-the-scenes power broker in the world of biotech commercialization, Gorlin has been involved in a plethora of start-ups since the 1980s, some of which went on to lucrative pay-offs.
According to his official bio, these include Hycor Biomedical Inc. (acquired by Agilent Technologies), Theragenics Corp. (NYSE: TGX), CytRx Corp. (Nasdaq: CYTR), Medicis Pharmaceutical Corp. (sold to Valeant Pharmaceuticals for approximately $2.6 billion), EntreMed Inc. (Nasdaq: ENMD), MRI Interventions, DARA BioSciences Inc. (Nasdaq: DARA), MiMedx (Nasdaq: MDXG), and Medivation Inc. (Nasdaq: MDVN).