Cancer cells achieve their pathogenicity by changing which genes are on and off. To maintain these changes in gene expression, cancer cells rely on proteins that interact with DNA or modify chromatin. My group investigates how such factors sustain the aberrant capabilities of cancer cells, thereby identifying new therapeutic targets.
Cancer can be understood as a disease of dysfunctional gene expression control. Research in Chris Vakoc’s lab investigates how transcription factors and chromatin regulators cooperate to control gene expression and maintain the cancer cell state. This work makes extensive use of genetic screens to reveal cancer-specific functions for transcriptional regulators, as well as genomic and biochemical approaches to identify molecular mechanisms. One theme that has emerged from their efforts is that blood cancers are often vulnerable to targeting transcriptional coactivators, such as BRD4 and the SWI/SNF chromatin remodeling complex. Vakoc’s team demonstrated that chemical inhibition of BRD4 exhibits therapeutic effects in mouse models of leukemia, a finding that has motivated ongoing clinical trials in human leukemia patients. The Vakoc lab has also developed a CRISPR-Cas9 screening approach that can reveal individual protein domains that sustain cancer cells. Their lab is now deploying this technology in a diverse array of human cancers to reveal therapeutic opportunities and basic mechanisms of cancer gene control.
AACR Outstanding Achievement in Cancer Research Award
Forbeck Scholar Award
"V Scholar" by The V Foundation for Cancer Research
Burroughs Welcome Fund Career Award for Medical Scientists
Pershing Square Sohn Prize
Leukemia and Lymphoma Society Scholar Award
Christina Renna Foundation gives $35k for pediatric cancer research
February 7, 2019
On January 26th, the Christina Renna Foundation donated $35,000 to Cold Spring Harbor Laboratory (CSHL) at its 12th annual Angel’s Wish Gala. The funds will go to continued research into rhabdomyosarcoma (RMS), a rare and often fatal form of pediatric cancer. This research is part of the CSHL Sarcoma Research Project. CSHL Professor Chris Vakoc,...
The year of CRISPR
December 26, 2018
It’s hard to have missed the acronym CRISPR this year! Headlines in the news have heralded game changing possibilities in biomedicine. Controversy and debate continue to sizzle worldwide among scientists and policymakers over the ethical implications of gene editing in humans. At Cold Spring Harbor Laboratory (CSHL), though, CRISPR isn’t just about headlines. It is...
Inconspicuous protein key to deadly blood cancer
November 29, 2018
Cold Spring Harbor, NY — Five percent of acute leukemia cases are diagnosed as mixed lineage leukemia (MLL). MLL is an aggressive blood cancer that predominantly occurs in infants and has been difficult to treat. Now, researchers at Cold Spring Harbor Laboratory (CSHL) have identified a very common protein as the single key to the...
Pancreatic cancer’s addiction could be its end
November 13, 2018
Cold Spring Harbor, NY — Cancer cells are often described as cells “gone bad” or “renegade.” New research reveals that in some of the deadliest cases of pancreatic cancer, these rebellious cells have an unexpected addiction. Now, scientists are investigating if that addiction can be used to bring about a tumor’s end. Cold Spring Harbor...
Friends of T.J. donate $50k for sarcoma research
October 9, 2018
On September 23rd, Nancy and Tom Arcati of the Friends of T.J. Foundation presented a donation of $50,000 to Cold Spring Harbor Laboratory (CSHL) Associate Professor Chris Vakoc and his team of rhabdomyosarcoma researchers. Vakoc’s lab studies cancers like rhabdomyosarcoma to pinpoint genetic weaknesses and find therapeutic targets. Three members of his lab specifically focus...
Researchers discover new type of lung cancer
June 25, 2018
Cold Spring Harbor, NY — Researchers have discovered a new kind of small-cell lung cancer (SCLC). The discovery paves the way for developing personalized medicine approaches to target this previously unnoticed form of the disease. “Cancer is not one thing, it’s actually hundreds of distinct diseases.” This common refrain helps explain the frustrating experience oncologists...
CRISPR-based system identifies important new drug targets in a deadly leukemia
March 8, 2018
Cold Spring Harbor, NY — Scientists at Cold Spring Harbor Laboratory (CSHL) have discovered a way to rein in an overactive protein that drives some aggressive leukemias. The renegade molecule, MEF2C, belongs to a class of proteins that is notoriously difficult to manipulate with drugs. But the new research suggests an opportunity to develop therapies...
Molecular decoy helps researchers halt and reverse acute leukemia in mice
January 8, 2018
Cold Spring Harbor, NY — Cancer researchers today announced they have developed a way of sidelining one of the most dangerous “bad actors” in leukemia. Their approach depends on throwing a molecular wrench into the gears of an important machine that sets genes into motion, enabling cancer cells to proliferate. In tests in mice, the...
Friends of T.J. present $50,000 gift for ongoing rhabdomyosarcoma research
November 7, 2017
Cold Spring Harbor, N.Y. — On November 1st, The Friends of T.J. Foundation presented CSHL Associate Professor Chris Vakoc with a check for $50,000 for his ongoing rhabdomysarcoma (RMS) research. Dr. Vakoc heads the RMS research initiative at Cold Spring Harbor Laboratory. The Friends of T.J. Foundation is one of a group of local funders...
Halfway around the world, a reunion of friends opens door to a cancer discovery
August 31, 2017
fter interviewing for a position in a pancreatic cancer lab at Cold Spring Harbor Laboratory, 7,000 miles from his hometown in South Korea, Chang-il Hwang did what many of us would: he posted about it on Facebook. Little did he know that he was rekindling a friendship that would pave the way to a long-sought...
Roe, J. S. and Hwang, C. I. and Somerville, T. D. D. and Milazzo, J. P. and Lee, E. J. and Da Silva, B. and Maiorino, L. and Tiriac, H. and Young, C. M. and Miyabayashi, K. and Filippini, D. and Creighton, B. and Burkhart, R. A. and Buscaglia, J. M. and Kim, E. J. and Grem, J. L. and Lazenby, A. J. and Grunkemeyer, J. A. and Hollingsworth, M. A. and Grandgenett, P. M. and Egeblad, M. and Park, Y. and Tuveson, D. A. and Vakoc, C. R. (2017) Enhancer Reprogramming Promotes Pancreatic Cancer Metastasis. Cell, 170(5) pp. 875-888.
Hohmann, A. F. and Martin, L. J. and Minder, J. L. and Roe, J. S. and Shi, J. and Steurer, S. and Bader, G. and McConnell, D. and Pearson, M. and Gerstberger, T. and Gottschamel, T. and Thompson, D. and Suzuki, Y. and Koegl, M. and Vakoc, C. R. (2016) Sensitivity and engineered resistance of myeloid leukemia cells to BRD9 inhibition. Nat Chem Biol, 12(9) pp. 672-9.
Shen, Chen and Ipsaro, Jonathan J and Shi, Junwei and Milazzo, Joseph P and Wang, Eric and Roe, Jae-Seok and Suzuki, Yutaka and Pappin, Darryl J and Joshua-Tor, Leemor and Vakoc, Christopher R (2015) NSD3-Short Is an Adaptor Protein that Couples BRD4 to the CHD8 Chromatin Remodeler. Molecular Cell, 60(6) pp. 847-859.
Shi, J. and Wang, E. and Milazzo, J. P. and Wang, Z. and Kinney, J. B. and Vakoc, C. R. (2015) Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains. Nat Biotechnol, 33(6) pp. 661-67.
Zuber, J. and Shi, J. and Wang, E. and Rappaport, A. R. and Herrmann, H. and Sison, E. A. and Magoon, D. and Qi, J. and Blatt, K. and Wunderlich, M. and Taylor, M. J. and Johns, C. and Chicas, A. and Mulloy, J. C. and Kogan, S. C. and Brown, P. and Valent, P. and Bradner, J. E. and Lowe, S. W. and Vakoc, C. R. (2011) RNAi screen identifies Brd4 as a therapeutic target in acute myeloid leukaemia. Nature, 478(7370) pp. 524-528.Additional materials of the author at
CSHL Institutional Repository