May 10, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Siddhartha Mukherjee, perhaps best known as the Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer, co-founded and launched a biotech company today, Vor BioPharma, which licensed technology from his laboratory at Columbia University.
An assistant professor of medicine at Columbia, and an oncologist, Mukherjee is the scientific co-founder, launched by PureTech Health (PRTC), a cross-disciplinary healthcare company that creates and spins out biotech companies. Vor BioPharma will focus on immuno-oncology research, specifically working with chimeric antigen receptor T-cell (CAR T-cell) therapies.
“CAR T-cell therapies have shown remarkable progress in the clinic, yet their applicability beyond a small subset of cancers is currently very limited,” said Sanjiv Sam Gambhir, member of the Vor Scientific Advisory Board, and professor of radiology and bioengineering, and chair of the department of radiology, and director of the Canary Center for Cancer Early Detection, and director of the Molecular Imaging Program at Stanford University, in a statement. “This technology seeks to address bottlenecks that prevent CAR T-cell therapy from becoming more broadly useful in treating cancers outside of B-cell cancers.”
The technology is still in the process of patent applications. With that in mind, Mukherjee described the research to Forbes, saying, “We’ve studied literally hundreds of patient samples with pre-leukemia and leukemia, myeloid leukemia. In the process of doing that, we began to find that there were antigens that were specific for these leukemias and that we could create CAR-Ts that would not kill normal cells but would kill uniquely the cancer cells.”
The technology is still in the process of patent applications. With that in mind, Mukherjee described the research to Forbes, saying, “We’ve studied literally hundreds of patient samples with pre-leukemia and leukemia, myeloid leukemia. In the process of doing that, we began to find that there were antigens that were specific for these leukemias and that we could create CAR-Ts that would not kill normal cells but would kill uniquely the cancer cells.”
He goes on to say, “I think that the crucial realization came from understanding the target, and that’s the understanding that you could take even the standard CAR-T technologies and modify them appropriately to recognize unique, novel cancer cells…. In the process of doing that, we asked to also activate new kinds of CAR-T cells. It’s really a combination of two kinds of technologies, the cancer cell and the immune cell.”
So far the technology has only been tested in test tubes and petri dishes, and the company will push it into a mouse model and, with any luck, eventually into some level of clinical trials.
Joseph Bolen will join Vor’s scientific advisory board and be acting chief scientific officer. He is formerly the president and chief scientific officer of Moderna Therapeutics.
Sanjiv Sam Gambhir, professor of radiology, materials science & engineering, and bioengineering at Stanford University, is also on the scientific advisory board. Mukherjee is on the board as well, and Derrick Rossi, associate professor in the Stem Cell and Regenerative Biology Department at Harvard Medical School and Harvard University is on the scientific advisory board as well.
CAR-T is a relatively new area, but not so new that there aren’t companies far ahead, including Novartis , Juno Therapeutics and Kite Pharma . Which begs the question of what Vor is doing, especially so far behind the starting line, that’s going to be different.
“What’s published in a lab and what’s not published in a lab—the actual work going on—are two completely different things,” Rossi told STAT. “There’s a lot of really exciting science coming that hasn’t seen the light of publication yet.”
Which is a slightly oblique way of saying that the founders of the company believe the technology—as yet patented or published—brings something new to the table. Although this is completely speculative, Mukherjee’s description at least hints that possibly they have found some CAR-T cells that respond to universal antigens on certain types of cancers. If—a very big if, given what is being described so far—the technology would get around one of immuno-oncology’s biggest hurdles, which is the labor-intensive and individualized nature of these types of treatments.
