Center for Experimental Therapeutics : Major Accomplishments

Therapeutic or diagnostic innovations often take ten to 20 years to develop, and ultimately very few inventions progress from laboratory research to clinical trials. MSK researchers have an extraordinary track record of generating practice-changing medical products, many of which were developed with support from the Center for Experimental Therapeutics. Some of our proudest achievements are listed below.

Genetically engineered T cells

This exciting cancer treatment breakthrough, known as CAR (chimeric antigen receptor) therapy, is based on genetically enhanced T cells. CAR therapy is an effective new treatment option for patients with leukemia, and potentially for those with other cancers. The Center for Experimental Therapeutics has supported research on engineered T cells led by physician-scientists Renier Brentjens, Michel Sadelain, and Isabelle Rivière for more than a decade. CAR therapies are now being developed by Juno Therapeutics.

Androgen receptor inhibitors

Physician-scientist Charles L. Sawyers discovered and developed enzalutamide for prostate cancer while he was working at the University of California, Los Angeles. Enzalutamide works by preventing testosterone production to starve tumors of the fuel they need to grow. Phase I and phase III clinical trials led by MSK clinicians Michael Morris, Dana Rathkopf, and Howard Scher resulted in the FDA approval of enzalutamide in 2012. In addition, Dr. Sawyers formulated a drug similar to enzalutamide, ARN-509, with a similar mechanism of action and improved safety profile. ARN-509 has been licensed to Johnson and Johnson for continued development.

HSP-90 inhibitors

In the early days of the Center for Experimental Therapeutics, chemist Gabriela Chiosis studied and manipulated a class of small molecule inhibitors of heat shock proteins. Her research led to the discovery and development of PU-H71, a promising new cancer drug that is in first-in-human clinical trials. PU-H71 labeled with radioactive iodine is being tested as an imaging agent for patients with non-Hodgkin lymphoma and solid tumors, and as a therapeutic agent. The technology is being developed by Samus Therapeutics.


Immunologist James P. Allison, who spent more than a decade of his career at MSK, conceived ipilimumab (Yervoy®) while working at the University of California, Berkeley. He continued studying its mechanism of action at MSK and, together with medical oncologist Jedd Wolchok, helped guide the development of the drug from the first laboratory studies through the late-stage clinical trials that led to its FDA approval for treatment of advanced melanoma. Today ipilimumab is owned by Bristol-Myers Squibb.

Pralatrexate (Folotyn®)

This new small molecule drug was discovered at MSK by molecular pharmacologist Francis M. Sirotnak, who later collaborated with Owen O’Connor of Columbia University Medical Center to conduct clinical trials of the drug. In 2009, pralatrexate was approved by the FDA for treatment of relapsed or refractory peripheral T cell lymphoma, a form of lymphoma for which no approved therapeutic options existed previously. Funding by the Center for Experimental Therapeutics allowed the advancement of the drug toward FDA approval. The drug will also be tested for treatment of additional blood-related cancers as well as solid tumors.

WT1 Treatments

The Wilms tumor 1 (WT1) antigen is an intracellular, oncogenic protein that is overexpressed in a wide range of leukemias and solid cancers, making it an attractive therapeutic target. Research led by David A. Scheinberg focuses on different strategies to target WT1 in leukemias and solid tumors. Favorable results were obtained in an ongoing clinical trial by Drs. Peter Maslak and Lee M. Krug with a WT1 multi-peptide vaccine, which targets the antigen within the cell. In addition, Dr. Scheinberg’s team pursues preclinical development of ESK1, a novel monoclonal antibody that targets fragments of WT1 located on the cell surface, in partnership with Novartis.

Overcoming Cancer Adaptive Resistance Responses

Research led by pharmacologist Neal Rosen into the complex and complicated pathway activation and feedback systems that control melanoma tumor growth has created a rationale for using two different pathway inhibitors to treat the disease. A phase III clinical trial combining the MEK inhibitor trametinib and BRAF inhibitor dabrafenib showed that the combination therapy significantly improved response rates when compared to treatment with dabrafenib alone. The combination received accelerated FDA approval. Physician-scientist Paul Chapman has added an important companion goal to the regimen by correlating tumor DNA profiles with patient data, creating predictive models for assigning treatment and improving patient outcomes.

3F8 and 8H9

Physician-scientist Nai-Kong Cheung is researching the use of monoclonal antibodies to treat pediatric tumors of the central nervous system, such as neuroblastoma, glioma, and brain metastases. He is also developing effective models for mimicking the physiology of this uniquely sensitive area. Dr. Cheung has pioneered the administration of the monoclonal antibodies 3F8 and 8H9 systemically and compounded as radioimmunotherapy, providing effective, precisely targeted therapy and sparing the surrounding brain tissues from toxicity.


The Center for Experimental Therapeutics has supported the work of many investigators to develop epothilones, a family of novel anticancer agents. Chemist Samuel Danishefsky has pioneered the research for synthesizing epothilones, and the lab of cell biologist Malcolm A. S. Moore has conducted preclinical evaluations of several epothilone analogs. Ultimately, a first-in-human clinical trial was opened by clinicians Mrinal Gounder and Naiyer Rizvi in partnership with Bristol-Myers Squibb.