Renowned Cancer Specialist Starts CAR-T Biotech Firm

Marcia Frellick

May 19, 2016

Siddhartha Mukherjee, MD, PhD, world-renowned cancer physician and Pulitzer Prize–winning author, has launched a biotechnology firm spun from his work in developing chimeric antigen receptor (CAR-T) cell therapy.

Dr Mukherjee, an assistant professor of medicine at Columbia University in New York City, who most famously wrote The Emperor of All Maladies: A Biography of Cancer, for which he won a Pulitzer in 2011, has teamed up with innovator PureTech Health and a team of top cancer researchers to form Vor BioPharma. The immuno-oncology start-up was announced May 9.

Vor has a worldwide exclusive license with Columbia University built on technology Dr Mukherjee developed to use the body's own immune system to fight cancer. It involves removing a patient's disease-fighting T cells and reengineering them to hunt down and attack cancer. The CAR-T cells are then injected back into the body and unleashed on cancer cells.

CAR-T has been in development in other laboratories for more than 5 years, but recently has expanded into the public consciousness.

"I think immunotherapy in general, and CAR-T therapy specifically, are generating a tremendous excitement among the research community and patients," Saed Usmani, MD, a hematologist with the Levine Cancer Institute in Charlotte, North Carolina, told Medscape Medical News.

"The first CAR-T investigations have been done with leukemia and lymphoma. There are several platforms being developed in other diseases, such as multiple myeloma," he said.

One of the key challenges for scientists is overcoming the toxicity threat the cells present. Therefore, the people selected for clinical trials will be chosen very specifically, Dr Usmani said.

                                                               

Dr Siddhartha Mukherjee. Source: Fredy Perojo/Medscape

                       

"It won't be something every patient can get," he emphasized.

Another challenge is figuring out how to sustain the power of the reengineered cells, which can essentially exhaust themselves, Dr Usmani said.

Crowded Field

Boston-based Vor BioPharma already has many competitors. Swiss drug maker Novartis, which has teamed with the University of Pennsylvania, has demonstrated clinical results with the technology in lymphoma, and US firms including Juno Therapeutics and Kite Pharma have treated human tumors with their technology and are moving toward regulatory filings.

As reported earlier in Medscape Medical News, last year saw the first success of CAR-T therapy in a solid tumor: pancreatic cancer. Results were reported at the American Society of Clinical Oncology 2015 Annual Meeting in Chicago, Illinois.

Companies have primarily shown clinical success with certain advanced leukemias such as advanced acute lymphoblastic leukemia and chronic lymphocytic leukemia.

Vor's founders aim to expand the targets.

Among Vor's founding scientific advisory board members is Derrick Rossi, PhD, associate professor in the Stem Cell and Regenerative Biology Department at Harvard Medical School and Harvard University in Boston, Massachusetts.

Dr Rossi told Medscape Medical News that although CAR-T therapies have yielded lasting remissions in patients with advanced B-cell cancers, success has been limited in other cancers.

"We believe that Vor's unique technology has the potential to broaden the applicability and success rate of this promising therapy beyond B-cell malignancies by removing key barriers that currently limit its use," he said.

Current CAR-T therapies result in loss of cell and tissue functionality, Dr Rossi said. "For example, CD19-targeted CAR-T cell therapies result in B-cell aplasia," he noted, adding that Vor seeks to preserve healthy cell functionality while maintaining potent anticancer effects.

Dr Rossi acknowledged the company is limited in what it can say about methodologies until their work is published, but said that Vor is focused on therapies that have the potential for lasting remissions or cures for patients with advanced cancers that have failed other options.

Vor's technology is a platform for potentially generating multiple products addressing multiple cancer types, he said.

"As of right now, Vor is not announcing a specific lead indication, but we are prioritizing hematologic malignancies that are not well-addressed using existing CAR-T technologies," he said.

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