New Discoveries and Honors

Read about the latest discoveries by Damon Runyon scientists and honors received by scientists in the Damon Runyon scientific community.

December 4, 2017

Craig J. Ceol, PhD (Damon Runyon Fellow '05-'07) of the University of Massachusetts Medical School, Worcester, and colleagues, have identified a new protein that is involved in metastatic melanoma, the most deadly form of skin cancer. The protein, GDF6, is part of a class of proteins called “growth differentiation factors” that helps cells divide and differentiate into specific cell types. Looking at human melanoma cells, the researchers found that 80 percent of patient tumors had elevated levels of GDF6 and these higher levels correlated to the aggressiveness of the cancer. Patients with less GDF6 had a lower risk of metastasis and a better chance of survival. The next step for the scientists is to find a way to turn off GDF6 that could potentially be used for treating patients. The study was published in the Journal of Clinical Investigation.

November 30, 2017

Chuan He, PhD (Damon Runyon Fellow '00-'02) of the University of Chicago, Chicago, was named one of this year's winners of the 2017 Paul Marks Prize for Cancer Research. The award recognizes promising investigators aged 45 or younger at the time of nomination for their efforts in advancing cancer research.

November 23, 2017

Matthew L. Meyerson, MD, PhD (Damon Runyon Fellow '95-'98), of the Dana-Farber Cancer Institute, Boston, and colleagues, reported a study that provides clues to the role Fusobacteria may play in the development of human colon cancers. Meyerson was one of the first scientists to discover that Fusobacteria, which normally inhabit the mouth, are closely associated with colon cancer cells but not normal colon cells. Researchers have now confirmed the presence of Fusobacteria in up to half of all colon tumors. They went on to show colon cancer cells that had metastasized to the liver continued to be infected with Fusobacteria. Interestingly, tumor cells given an antibiotic called metronidazole that fights Fusobacteria showed decreased cancer cell proliferation and slowed tumor growth. The paper, published in Science, suggests that targeted approaches for colorectal cancer treatment directed at Fusobacteria should be pursued.

November 9, 2017

This month, Damon Runyon scientists published promising findings on “liquid biopsies.” This non-invasive method isolates and studies circulating tumor DNA (ctDNA)-- free-floating pieces of DNA found in blood plasma that are shed from tumor cells. These studies demonstrated that liquid biopsies are becoming an important tool for monitoring cancer progression, as well as identifying treatment strategies and drug resistance earlier than traditional approaches.

A cross-disciplinary group from leading research centers in Boston recently reported on a new approach to capture ctDNA and perform broad genetic sequencing. Matthew L. Meyerson, MD, PhD (Damon Runyon Fellow '95-'98), of the Dana-Farber Cancer Institute, is a pioneer in cancer genomics and was one of the leaders of this study, which established the feasibility and accuracy of profiling ctDNA from patients with advanced metastatic cancer. The researchers developed an efficient method for capturing and quantifying tumor DNA from blood prior to sequencing, thereby making blood biopsies cost-effective and scalable. The study demonstrates that nearly 90 percent of the genetic features of a tumor can be detected in blood by sequencing all the protein encoding genes. Eliezer M. Van Allen, MD (Damon Runyon Clinical Investigator '15-'18), also contributed to this study, published in Nature Communications.  

Separately, Christine M. Lovly, MD, PhD (Damon Runyon Clinical Investigator '13-'18), of Vanderbilt University Medical Center, Nashville, and colleagues, have found a method of monitoring the progression of small-cell lung cancer (SCLC) by using liquid biopsies. They were able to identify specific gene mutations associated with relapse of SCLC before the cancer growth could be discovered through standard imaging.  This study was published in The Journal of Thoracic Oncology.

November 8, 2017

Ronald J. Buckanovich, MD, PhD (Damon Runyon Clinical Investigator ’08-’11) of the University of Pittsburgh, and colleagues, have developed a process that can grow hundreds of cultured cell masses, called spheroids, from just a few tumor cells derived from a patient. This 3D method yields cells that grow and multiply just as they would inside the body. Currently, researchers are limited to two-dimensional cells grown in petri dishes, which often do not respond to medicines the same way as ovarian cancer cells inside the body. The researchers administered cancer drugs to the cultured cancer spheroids and compared their response to that of ovarian cancer cells from the same patient that were implanted into mice. They showed that the response of the cultured spheroids accurately mirrored that of the natural cells implanted in the mice. The process is still being developed for clinical application and may be helpful for informing personalized medicine for cancer patients. The study was published in the journal Clinical Cancer Research.

November 2, 2017

Increasing evidence shows that diet plays a major role in the development of some cancers. Andrew T. Chan, MD, MPH (Damon Runyon Clinical Investigator ‘08-‘13), of Harvard Medical School and Massachusetts General Hospital, Boston, and colleagues, found that eating more fiber after colorectal cancer diagnosis is associated with a lower risk of dying from colorectal cancer. The researchers collected data on 1,575 men and women who took part in the Nurses' Health Study and Health Professionals Follow-up Study, and who had been treated for colon or rectal cancer that had not spread. Specifically, the study looked at total fiber consumption in the eight years after the participants' cancer diagnosis. The greatest benefit was attributed to fiber from cereals and whole grains, according to the report. Vegetable fiber was linked to an overall reduction in death, but not specifically in death from colon cancer, and fiber from fruit was not linked to a reduction in death from any cause.  The report was published in JAMA Oncology.

October 18, 2017

A new class of treatments called CAR-T therapy is providing options for patients who have all but lost hope in their fight against cancer. This form of immunotherapy is based on genetically enhancing a patient’s own immune cells to target and kill their cancer. The Food and Drug Administration approved Yescarta for adults with a form of blood cancer called non-Hodgkins lymphoma. This second CAR-T therapy follows closely on the heels of Kymriah, which was approved in September to treat certain lethal blood and bone marrow cancers in children. Damon Runyon scientists are front and center in the development and advancement of this immunotherapy approach that is having dramatic results in patients. This newly approved therapy was developed by Kite Pharma under the leadership of Chief Medical Officer David D. Chang, MD, PhD (Damon Runyon Fellow '88-'90).

October 16, 2017

The National Academy of Medicine announced the election of 80 new members. Election to the Academy is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. Damon Runyon congratulates the four alumni who were elected this year:

Scott A. Armstrong, MD, PhD (Clinical Investigator ’03-’08), Dana-Farber Cancer Institute

Howard Y. Chang, MD, PhD (Scholar ’06-’08), Stanford University

Rachel Green, PhD (Fellow ’93-’96), Johns Hopkins School of Medicine

Michael E. Greenberg, PhD (Fellow ’83-’84), Harvard Medical School

October 5, 2017

Gordon J. Freeman, PhD (Damon Runyon Fellow ’79-’81), Dana-Farber Cancer Institute, Boston, is one of five scientists honored by the 2017 Warren Alpert Foundation Prize for discoveries focused on “Immune Checkpoint Blockade and the Transformation of Cancer Therapy.” Collectively, their work has elucidated foundational mechanisms in cancer’s ability to evade immune recognition through the CTLA-4 and PD-1 pathways and, in doing so, has profoundly altered the understanding of disease development and treatment. Their discoveries have led to the development of effective immune therapies for several types of cancer.

October 5, 2017

The National Institutes of Health’s High-Risk, High-Reward Research program, funded 86 awards to exceptionally creative scientists proposing to use highly innovative approaches to tackle major challenges in biomedical research. The program is designed to accelerate scientific discovery by supporting high-risk research proposals. Applicants of the program are encouraged to think outside-the-box and to pursue exciting, trailblazing ideas. Four Damon Runyon scientists are recipients of this year’s awards:

Pioneer Award

Feng Zhang, PhD (Damon Runyon-Rachleff Innovator ‘12-‘14), the Broad Institute, Cambridge

New Innovator Award

Jaehyuk Choi, MD, PhD (Damon Runyon Clinical Investigator ’16-’19), Northwestern University, Chicago

Raymond E. Moellering, PhD (Damon Runyon Fellow ’11-‘13, Dale F. Frey Breakthrough Scientist ’13-‘15), University of Chicago, Chicago

Jakob von Moltke, PhD (Damon Runyon Fellow ’13-’16, Dale F. Frey Breakthrough Scientist ’17-‘18), University of Washington, Seattle

September 29, 2017

Liron Bar-Peled, PhD (Damon Runyon Fellow ‘14-‘17) of the Scripps Research Institute, La Jolla, developed a new proteomics-based approach to discover small-molecule inhibitors that could be used as anti-cancer therapies. The approach is based on the fact that certain amino acids on proteins have a special chemical reactivity that allows them to form irreversible covalent bonds with suitably designed probe or "scout" molecules. Scientists can apply such molecules to a large collection of cellular proteins to quickly identify proteins that have these reactive amino-acids -- which would be considered potential drug targets. The researchers applied this strategy to identify potential drug targets in non-small-cell lung cancers (NSCLCs) that depend on over-activity of the transcription factor NRF2. The study was published in the journal Cell.

September 13, 2017

Alexandra Zidovska, PhD (Damon Runyon Fellow ‘10-‘12) of New York University, New York, has discovered the “internal clock” of live human cells using state-of-the-art fluorescence microscopy. Previously, the only way to tell the precise point of a cell in its life cycle was by studying dead cells. Alexandra’s lab has found that the nuclear envelope, which separates the nucleus with the DNA from the rest of the cell, has a previously undetected type of motion: it fluctuates in shape every few seconds. As the cell ages, these fluctuations become measurably less and less dramatic. Moreover, this motion marks the first physical feature that systematically changes with the cell cycle. “Therefore, this process can serve as an internal clock of the cell, telling you at what stage in the cell cycle the cell is,” explained Alexandra. Understanding how the behavior of the nuclear envelope changes over time could have important implications for research and treatment of diseases such as cancer, which is characterized by cells with longer live spans and uncontrolled cell divisions. This study was published in the Proceedings of the National Academy of Sciences (PNAS).

September 11, 2017

Kristopher R. Bosse, MD (Damon Runyon Physician Scientist '16-'20) of the Children's Hospital of Philadelphia, is engineering a new drug to selectively target neuroblastoma cells and deliver a chemotherapeutic agent into the cells. Neuroblastoma is a cancer of the developing nervous system that usually occurs as a solid tumor in a child's chest or abdomen, and is the most common cancer in infants. After discovering a protein found on the surface of cancer cells called GPC2, he and his colleagues created an antibody that can recognize GPC2 and linked it to chemotherapy drug to form an “antibody-drug conjugate” (ADC). When tested in human cell cultures and mouse models, it successfully killed cancer cells while sparing healthy ones. The study was published in the journal Cancer Cell, which featured their findings as the cover story.

August 15, 2017

Five researchers have been announced as the recipients of the Albany Medical Center Prize in Medicine and Biomedical Research for 2017. They have made important contributions to the development of CRISPR-Cas9, a gene engineering technology that harnesses a naturally occurring bacterial immune system process. The technology has revolutionized biomedical research and provided new hope for the treatment of genetic diseases and more. The awardees include Feng Zhang, PhD (Damon Runyon-Rachleff Innovator ‘12-‘14) of the Broad Institute and Massachusetts Institute of Technology, Cambridge, and Jennifer A. Doudna, PhD (Damon Runyon Fellowship Sponsor, Former Fellowship Award Committee Member) of the University of California, Berkeley.

August 1, 2017

The FDA approved Idhifa for acute myeloid leukemia (AML), the result of important contributions from Hai Yan, MD, PhD (Damon Runyon Scholar ‘05-‘07), of Duke University Medical Center, Durham, and Omar Abdel-Wahab, MD (Damon Runyon Clinical Investigator ‘13-‘16), of Memorial Sloan Kettering Cancer Center, New York. They made independent discoveries about mutations in the IDH2 gene and how these contribute to the development of AML. In clinical trials, Idhifa treatment led to complete remission in some patients and a reduction in the need for both red-cell and platelet transfusion.

July 21, 2017

Marcela V. Maus, MD, PhD (Damon Runyon-Rachleff Innovator ’17-’18) at Massachusetts General Hospital, Boston, published results from her clinical trial with glioblastoma patients showing for the first time that CAR (chimeric antigen receptor) T cells cross the blood-brain barrier to reach tumors and appeared to be safe. These CAR T cells were targeted to EGFR variant III in glioblastoma patients. Her lab is working on next steps such as combining CAR T cells with other drugs to increase the potency of this treatment to shrink tumor size. The results of this trial indicate that CAR T cell therapy is a viable option for treating glioblastoma. The study was published in Science Translational Medicine.

July 5, 2017

Catherine J.Wu, MD (Current Damon Runyon Physician-Scientist Mentor and Clinical Investigator '07-'12) of Dana-Farber Cancer Institute, Boston, has led one of the first studies that demonstrates the potential of personalized cancer vaccines. The study focused on six people with advanced melanoma, a type of skin cancer. The participants had surgery to remove their tumors, but about half of all such patients face a recurrence of cancer. The researchers formulated a personalized vaccine for each person that contained up to 20 protein fragments corresponding to the mutations in their individual tumors. Thirty-two months later, four of the patients were cancer free. Two patients had their tumors re-grow, but experienced complete remission when subsequently treated with a PD-1 inhibitor, a drug that blocks the ability of cancer cells to hide from the immune system. Two years after treatment, the patients’ blood still carried anti-melanoma immune cells, suggesting the vaccine’s benefits last. Currently, cancer patients begin treatment with traditional therapies about three to six weeks after an initial diagnosis. Her goal is to treat a patient with an individualized cancer vaccine in that same window. While this is a small-scale study, the promising results mean researchers can move ahead with larger clinical trials, which could potentially lead to new, more effective cancer treatments. The results were published in the scientific journal Nature.

May 3, 2017

C. Ryan Miller, MD, PhD (Damon Runyon Clinical Investigator ’09-’12) of the UNC Lineberger Comprehensive Cancer Center, Chapel Hill, and colleagues, reported two studies on the genetics underlying brain tumors. The first study showed that mutations in MAPK and PI3K affect how cancer starts in glial cells, brain cells that provide support and insulation for neurons. These mutations triggered tumor initiation and produced increasingly dense low-grade gliomas that quickly progressed to aggressive and often deadly glioblastoma (GBM). The other study, conducted in cell lines and mouse models, tested a combination of targeted drugs as a potential therapy against glioblastoma by inhibiting the MAPK and PI3K cellular pathways. While the treatments overcame resistance in cells grown in the laboratory, they did not reach high enough concentrations to be effective when tumors were in the brain. One of the fundamental challenges in treating brain cancer with drugs is overcoming the blood-brain barrier, a membrane that separates circulating blood from the fluid in the central nervous system. This barrier works to protect the brain from toxins; however, this security system is so effective at protecting the brain that it prevents many life-saving drugs from reaching the cancer. The studies were published in the journal Neuro-Oncology.

May 2, 2017

Election to the National Academy of Sciences is one of the highest honors that can be earned by a U.S. scientist.  In recognition of their distinguished and continuing achievements in biomedical research, members of the Damon Runyon community of scientists were inducted this month:  

Ardem Patapoutian, PhD (Damon Runyon Scholar ’03-‘05, Fellow ’96-‘99), Scripps Research Institute, La Jolla

Guillermina Lozano, PhD (Former Fellowship Award Committee Member), M.D. Anderson Cancer Center, Houston

April 13, 2017

Feng Zhang, PhD (Damon Runyon-Rachleff Innovator ’12-’14) and colleagues at the Broad Institute, Cambridge, have developed a new CRISPR-based genetic diagnostic tool that may make it faster, less expensive, and easier to diagnose acute and chronic diseases like Zika, Ebola, cancer, and other hereditary disorders. The new tool dubbed SHERLOCK (Specific High-sensitivity Enzymatic Reporter unLOCKing) can detect extremely low concentrations of Zika virus and cancer DNA in blood, urine, and saliva samples. Previous CRISPR systems target DNA, but this system targets and degrades RNA. While scientists have recognized the potential for such a diagnostic tool, this is the first time it is possible to use the system widely to detect small amounts of RNA that could indicate the onset of cancer or other disease. The study was published in the journal Science.

April 10, 2017

Jedd D. Wolchok, MD, PhD (Damon Runyon-Lilly Clinical Investigator ‘03-‘08) at Memorial Sloan Kettering Cancer Center, New York, and colleagues, reported that matching the size of a tumor to the body's immune response could help doctors tailor immunotherapy treatments for melanoma patients whose disease has spread. Immunotherapy activates tumor-fighting T cells to target cancer, but these immune cells can lose their effectiveness or become “exhausted.” The study identified three different ways by which PD-1 blocking immunotherapies can fail: the drug doesn't re-invigorate exhausted T-cells, an immune response is not strong enough for the size of the tumor, and the drug is off target. Using these factors, the researchers developed a scoring system to more accurately predict how likely it is a treatment will work. The study was published in the scientific journal Nature.

March 29, 2017

David M. Livingston, MD (Damon Runyon Board Member), of Dana-Farber Cancer Institute, Boston, has been named the 20th recipient of the Pezcoller Foundation-AACR International Award for Cancer Research. Dr. Livingston is honored for his fundamental contributions to the field of basic cancer research. His work has been pivotal to the understanding of retinoblastoma pathway of cell cycle control as well as the transcriptional co-activation function of the key regulatory proteins, p300 and CBP. He is also credited with the landmark discovery of the central functions of the tumor suppressors, BRCA1 and BRCA2, which has revolutionized the fields of breast and ovarian cancer research. He will deliver his award lecture at the AACR Annual Meeting this April in Washington, DC.

March 24, 2017

At the junction of growth and starvation stands a signaling protein called mechanistic Target of Rapamycin Complex 1 (mTORC1). Inside the cell, mTORC1 regulates metabolism, growth, protein and organelle recycling (autophagy), proliferation, and survival. When something goes wrong in the pathway, various diseases such as cancer, obesity, and type 2 diabetes, can develop. An outstanding challenge is to understand how mTORC1 becomes activated in response to a wide variety of stimuli including nutrients, growth factors, and stressors, in normal and cancer states. Roberto Zoncu, PhD (Damon Runyon-Rachleff Innovator ’16-’17), and colleagues at the University of California, Berkeley, recently identified a lipid, cholesterol, as a novel nutrient input to mTORC1. They found that when cholesterol levels in organelles known as lysosomes are high, mTORC1 activity is switched on to initiate growth signals. Lysosomes are membrane-bound compartments where waste products are broken down and recycled. Lysosomes also serve as decision-making centers, where mTORC1 is switched on or off and therefore can dictate whether cells should grow or not. The way cholesterol, an essential building block for cellular growth and proliferation, activates mTORC1 appears to be highly specific and requires dedicated cholesterol-sensing proteins that reside at the lysosome. The study was published in the prestigious journal Science.

“This work advances our understanding of how mTORC1 integrates its many inputs, and could point the way to novel therapeutic avenues in cancer, particularly pancreatic cancers, where lysosome function and potentially mTORC1 activity are intimately associated with cancer growth,” says Zoncu.

March 14, 2017

Anjana Rao, PhD (Damon Runyon Fellow ’79), and James Scott-Browne, PhD (Damon Runyon Fellow ’11-’13), at the La Jolla Institute for Allergy and Immunology, La Jolla, and colleagues, are focusing on a key issue of how tumor-fighting T cells can lose their effectiveness or become “exhausted.”  The researchers identified two proteins, NFAT and Nr4a, that can bind to the DNA of T cells and shut down their tumor-fighting activity.  Next steps will be to determine if these processes can be interfered with or reversed in order to re-motivate immune cells to eradicate a patient's tumor.  These results were published in the journal Proceedings of the National Academy of Science.

February 27, 2017

Don X. Nguyen, PhD (Damon Runyon Fellow ’05-’08), of Yale Cancer Center, New Haven, and colleagues, reported new findings that explain the propensity of latent lung adenocarcinoma (LUAD) to relapse. They showed that differential expression of extracellular matrix (ECM) molecules and their interacting proteins contributes to risk of relapse in distinct LUAD subtypes. One protein called hyaluronan receptor HMMR, when overexpressed, was associated with inflammation and poor prognosis. Blocking HMMR in LUAD cells lowered their ability to initiate lung tumors and distant metastases such as in the brain. This study reveals an important mechanism by which disseminated cancer cells can coopt the inflammatory ECM to persist, leading to brain metastases. The results were published in the journal Cancer Research.

February 23, 2017

Christine Iok In Chio, PhD (Damon Runyon Shirley Stein Fellow ’13-’17), in the laboratory of her sponsor David Tuveson, MD, PhD, and colleagues at Cold Spring Harbor Laboratory, Cold Spring Harbor, created a new 3D model of pancreatic cancer, which allowed them to identify two distinct stroma cell populations called cancer-associated fibroblasts  (CAF’s), that work together with cancer cells to protect and help the tumor grow. One reason pancreatic cancer is difficult to treat is the dense stromal tissue that envelops tumors and acts as a physical barrier that blocks drug delivery. Since CAFs secrete extracellular matrix, which surround the cancer cells and shape the stroma, CAFs are attractive targets for drug development of targeted therapies for pancreatic cancer. The findings were published in the Journal of Experimental Medicine.

January 25, 2017

Gregory L. Beatty, MD, PhD (Damon Runyon-Nadia’s Gift Foundation Innovator ’12-’15) and colleagues at the Abramson Cancer Center at University of Pennsylvania, Philadelphia, reported high levels of inflammatory compounds in mice with pancreatic tumors. These included CCL2, a signaling molecule that promotes recruitment of inflammatory white blood cells by tumors. This likely contributes to the protective tumor microenvironment that makes most pancreatic tumors resistant to treatment. CCL2 levels increased further after the mice received radiotherapy. However, treatment with chemo and a CCL2-blocking antibody resulted in dramatically slower tumor growth and reduced recruitment of inflammatory cell types. These treated mice survived roughly 25% longer than those that received radiation alone. The results were published in the journal Clinical Cancer Research.

January 24, 2017

Theodora S. Ross, MD, PhD (Damon Runyon Scholar ’01-’03), and colleagues at UT Southwestern, Dallas, reported that the BRCA1 gene is required for the survival of blood forming stem cells. This could explain why patients with BRCA1 mutations do not have an elevated risk for leukemia; the stem cells die before they have an opportunity to transform into a blood cancer. These results also suggest that these patients may be at higher risk for the serious side effects of chemotherapy. The study was published in Cell Reports.

January 23, 2017

Akinyemi I. Ojesina, MBBS, PhD (Damon Runyon Fellow ’08-’11), of University of Alabama at Birmingham, worked with The Cancer Genome Atlas Research Network to identify novel genomic and molecular characteristics of cervical cancer that will aid in the subclassification of the disease and may help define personalized therapies for each individual patient. The new study conducted a comprehensive analysis of the genomes of 178 primary cervical cancers, and found that more than 70 percent of the tumors had genomic alterations in either one or both of two important cell signaling pathways (PI3K/MAPK and TGF-beta). The researchers also found that a subset of tumors did not show evidence of human papillomavirus infection. Certain cervical cancers contained mutations in genes that normally put ‘brakes’ on the immune system, suggesting treatment with immunotherapy “checkpoint inhibitors” may be effective against these cancers. The report was published in the scientific journal Nature.

January 23, 2017

Elaine V. Fuchs, PhD (Damon Runyon Board Member, Damon Runyon Fellow ‘77-‘79) of The Rockefeller University, New York, has been named the recipient of the 2017 McEwen Award for Innovation. The prize, given by the International Society for Stem Cell Research, recognizes groundbreaking work pertaining to stem cells or regenerative medicine. Dr. Fuchs studies adult skin stem cells, how they make and repair tissues, and how cancers develop.

January 23, 2017

Pardis C. Sabeti, MD, DPhil (Damon Runyon Fellow ‘04-‘06) of Harvard University, Cambridge, will receive the 2017 Richard Lounsbery Award from the National Academy of Sciences. She is recognized for her groundbreaking contributions including the development of new methods to study evolutionary selection in humans and viruses; the creation of new collaborative models for combatting emerging diseases across disciplinary and national borders; and leadership of global efforts to increase data sharing in pandemics such as Ebola and Lassa Fever. She published landmark genomic studies describing Ebola virus transmission during the 2014 outbreak.

January 13, 2017

Elaine V. Fuchs, PhD (Damon Runyon Board Member, Damon Runyon Fellow ‘77-‘79) and Shruti Naik, PhD (Damon Runyon Fellow ’14-’18) at The Rockefeller University, New York, and colleagues, found that skin squamous cell carcinomas alter the protein-making machinery to preferentially use tumor-related mRNAs, leading to the production of proteins important for cancer progression. This switch is linked to a ribosome initiation factor called eIF2 and transition initiation factor eIF2A. The results suggest that these could represent new targets for anticancer drug development.  The study was published in the scientific journal Nature.

January 12, 2017

Trudy G. Oliver, PhD (Damon Runyon-Rachleff Innovator ’13-’15), and colleagues at the Huntsman Cancer Institute at the University of Utah, Salt Lake City, reported the generation of a new mouse model for studying small cell lung cancer (SCLC). They demonstrated that Myc oncogene expression cooperates with Rb1 and Trp53 loss in the mouse lung to promote aggressive, highly metastatic tumors that are initially sensitive to chemotherapy followed by relapse. Targeted drug screening found that SCLC with high MYC expression is vulnerable to Aurora kinase inhibition, which, combined with chemotherapy, strongly suppresses tumor progression and increases survival. This study identified molecular features for patient stratification and uncovers a potential targeted treatment approach for these specific cancers.  The study was published in Cancer Cell.

January 5, 2017

Feng Zhang, PhD (Damon Runyon-Rachleff Innovator ‘12-‘14) of the Broad Institute, Cambridge, and colleagues, reported the discovery of new types of RNA-targeting CRISPR systems, which utilize a novel Cas enzyme called Cas13b. Cas13b is capable of targeting and degrading RNA (rather than DNA, which is targeted by previous CRISPR systems), which will enable researchers to specifically manipulate RNA in a high-throughput manner and manipulate gene function more broadly. The characterization of Cas13b has the potential to create a suite of RNA manipulation tools for studying a wide-range of biological processes that could be important for understanding and treatment of cancer and other diseases. The findings were reported in the scientific journal Molecular Cell.