Breakthrough Innovations

“Smart Bomb” for Lung Cancer

Challenge: Some 15% of people diagnosed with lung cancer have a type called small cell lung cancer. Few new therapies have been developed for this disease in the past several decades.

Breakthrough: MSK scientists participated in the preclinical development and led the first clinical trial of the drug, rovalpituzumab tesirine (Rova-T). Rova-T targets a protein called DLL3 that is highly expressed on small cell lung cancers. This therapy works like a Trojan horse—after the antibody seeks out and binds to the protein on the cancer cell, it delivers the toxin to the inside of the cell and destroys it. Specifically targeting the cancer cells limits toxicity to the normal tissues of the body, minimizing side effects.

Impact: Results from clinical trials have been encouraging. Rova-T could become a new treatment for patients with recurrent small cell lung cancer.



Challenge: The immune system is not designed to fight cancer cells—as it does infections—because cancer cells are not foreign: they are a patient’s own cells that have gone awry. The immune system needs to be taught to identify and attack cancer cells.

Breakthrough: Cell-based immunotherapy uses a patient’s own immune cells to battle cancer. One promising type is called chimeric antigen receptor, or CAR T cell therapy. CAR T cells are immune cells that are removed from a patient’s body and, in the laboratory, receive new DNA with instructions to find and destroy cancer. The altered cells are then placed back in the patient’s body to fight cancer. These cells live indefinitely within the patient and are only administered once.

Impact: MSK doctors have successfully used CAR T  therapy to treat leukemia and lymphoma. They are now investigating whether it can work in other cancers as well.


Memorial Sloan Kettering physician leads innovations in cancer treatment


Checkpoint Inhibitors

Challenge: One reason the immune system does not naturally fight cancer is that a type of immune cells, called T cells, have “brakes” that stop them from attacking cells that originate in one’s own body—such as cancer.

Breakthrough: MSK scientists and their collaborators discovered a class of drugs that releases the brakes on T cells so they can identify and fight cancer as they would foreign germs. These drugs are called checkpoint inhibitors.

Impact: MSK has led clinical trials showing that checkpoint inhibitors can be effective against melanoma, some types of lymphoma, and lung, kidney, bladder, and head and neck cancers. These drugs are now being tested at MSK against several other types of cancer, including sarcoma. MSK researchers guided the clinical trial of checkpoint inhibitor ipilimumab, which was approved by the FDA in 2011—the first drug ever to improve the survival rate of people with advanced melanoma. Combinations of these medicines have also been pioneered by MSK researchers and have resulted in the 2015 approval of the first combination immunotherapy to treat melanoma.


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Prostate Cancer Drug Discovery Using Epigenetics

Challenge: While most men with prostate cancer respond well to standard hormone therapy and chemotherapy, those cancers that don’t respond—or stop responding—are notoriously difficult to treat.

Breakthrough: MSK physician-scientists are investigating the signaling pathways that promote the growth of cancer cells, with an eye toward designing new treatments based on an emerging understanding of epigenetics. Epigenetics is the process by which different cells and tissues receive specific instructions—or “blueprints”—to function in unique roles, despite sharing the same DNA. Epigenetics is illuminating previously unknown aspects of how and why genes turn on and off, and expanding the possibilities for targeted drug intervention when these processes malfunction—with the goal of reversing them back to normal.

Impact: MSK researchers developed the drug enzalutamide, which is proving effective in treating an aggressive form of therapy-resistant prostate cancer. This drug works by targeting a key factor that governs gene regulation in prostate cancer cells.


Rethinking the Microbiome for Bone Marrow Transplants

Challenge: Bone marrow transplant (BMT)—when a patient’s cancerous cells are replaced with healthy cells from another person—can help people with blood cancers that haven’t responded to other treatments. But BMT poses serious risks—including graft-versus-host disease (GVHD), which can strike if the newly transplanted cells attack the “foreign” tissue. GVHD can cause serious organ damage and is life-threatening.

Breakthrough: MSK physician-scientists have recently discovered that certain bacterial strains commonly found in the gut (microbiome) can help prevent this dangerous condition. While a link has been known to exist between the microbiome and GVHD since the 1970s, no one had identified specific bacteria or understood their roles—until now. With the help of new DNA sequencing technologies, researchers have learned that patients with even a small amount of bacteria called Blautia in their gastrointestinal tract have a significantly lower chance of developing GVHD.

Impact: MSK clinicians are exploring ways to help patients keep this important bacteria by prescribing antibiotics that do not kill it, and by encouraging a wholesome diet during transplants rather than relying on IV nutrition. Researchers are evaluating the benefits of a fecal microbiota transplant prior to a bone marrow transplant. And in one trial, patients have their own microbiota stored before beginning treatment, so they can get it back later when needed.