Lymphatic vascular (cyan) and blood vessels (red) are revealed in this confocal micrograph of a mouse lung. The lymph system collects excess fluid in tissues and returns it to bloodstream.
Image courtesy of Yurin Seo, Mark Looney and Simon Cleary, UCSF/Nikon Small World.
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PERCEPTION proves a predictable NCI milestone
AuthorScott LaFee
Date
May 9, 2025
A false-colored scanning electron micrograph of a breast tumor spheroid (cluster of cells). The spheroid has been treated with the anti-cancer drug doxorubicin, which is causing some cells to die (yellow). Healthy cells are colored pink and violet. Image courtesy of Khuloud T. Al-Jamal, David McCarthy and Izzat Suffian, Wellcome Collection.
PERCEPTION is the acronym for PERsonalized Single-Cell Expression-Based Planning for Treatments In Oncology, an artificial intelligence-based tool that, in findings first reported last year, was able to predict tumor response to targeted therapy using single-cell datasets.
The work, published in Nature Cancer, is the result of first study author Sanju Sinha, PhD, assistant professor in the Cancer Metabolism and Microenvironment Program at Sanford Burnham Prebys, with senior authors Eytan Ruppin, MD, PhD, and Alejandro Schaffer, PhD, at the National Cancer Institute (NCI), part of the National Institutes of Health, and colleagues.
The researchers said PERCEPTION not only helped predict which anti-cancer drugs are most effective for individual patients, but also tracked the evolution of drug resistance over the course of the disease and treatment — something never before achieved.
“A tumor is a complex and evolving beast. Using single-cell resolution can allow us to tackle both of these challenges, Sinha said when their findings were published. “PERCEPTION allows for the use of rich information within single-cell omics to understand the clonal architecture of the tumor and monitor the emergence of resistance.” (In biology, omics refers to the sum of constituents within a cell.)
“The ability to monitor the emergence of resistance is the most exciting part for me. It has the potential to allow us to adapt to the evolution of cancer cells and even modify our treatment strategy.”
PERCEPTION was previously named among the National Institutes of Health director’s highlights for 2024.
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Science in Pictures
AuthorScott LaFee
Date
May 5, 2025
Confocal micrograph of mouse aortic endothelium stained for beta-catenin (green), laminin (purple), smooth muscle actin (red) and Hoechst (cyan). Endothelial cells constitute the inner lining of blood vessels in the heart.
Image courtesy of Florian Alonso, University of Bordeaux/Nikon Small World.
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Kelly Kersten awarded Melanoma Research Alliance grant to support research on melanoma immunotherapy
AuthorGreg Calhoun
Date
May 2, 2025
Kelly Kersten, PhD, is an assistant professor in the Cancer Metabolism and Microenvironment Program at Sanford Burnham Prebys. Credit: Sanford Burnham Prebys
The newly created Paul Walks – MRA Young Investigator Award in Memory of Chad Johnson is part of the alliance’s $9.3 million commitment to melanoma research funding in 2025.
Kelly Kersten, PhD, an assistant professor in theCancer Metabolism and Microenvironment Program at Sanford Burnham Prebys, was awarded a new type of grant from the Melanoma Research Alliance (MRA). The funding will support Kersten’s research on reactivating “exhausted” immune cells within melanoma tumors to restore their cancer-fighting ability and improve the effectiveness of melanoma immunotherapy.
“Inside tumors, immune cells often lose their strength to attack cancer,” said Kersten. “Our work is focused on understanding and reversing this exhaustion to make therapies more effective for more people.”
The MRA is the world’s leading nonprofit funder of melanoma research. The organization created the Paul Walks – MRA Young Investigator Award in Memory of Chad Johnson to providesupport for the next generation of scientists driving innovation against melanoma.
“Our Young Investigator Awards fuel the creativity and drive of early-career scientists whose work can redefine the future of melanoma research.” said Joan Levy, PhD, MRA Chief Science Officer.
The new grant pays tribute to Chad Johnson, a beloved friend and surfer who died from his melanoma diagnosis at age 55. Funding for the award was made possible through “Paul Walks,” a community fundraiser organized by Chad’s lifelong friend, Paul Giobbi.
The Paul Walks – MRA Young Investigator Award in Memory of Chad Johnson is part of MRA’s $9.3 million commitment to fund melanoma research in 2025, supporting more than 30 researchers across the U.S., Europe and Australia. Melanoma remains the deadliest form of skin cancer, with more than 100,000 people expected to be diagnosed this year and one death every hour in the U.S. alone.
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Science in Pictures
AuthorScott LaFee
Date
April 28, 2025
Myelinated axons are depicted in a rat spinal root. Myelin is a type of fat that forms an insulating sheath around the axon to protect it from losing electrical current needed to transmit signals. Axoplasm inside the axon is shown in pink.
Image courtesy of the National Center for Microscopy and Imaging Research at UC San Diego.
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Hot and Freeze
AuthorScott LaFee
Date
April 25, 2025
We’re on Radiolab! Hudson Freeze, PhD, director of the Sanford Children’s Health Research Center joins the legendary science podcast to recount a discovery that changed biology forever.
For years, scientists thought nothing could live above 73℃/163℉.At that temperature, everything boiled to death. But scientists Tom Brock and Hudson Freeze, PhD, now director of the Sanford Children’s Health Research Center and William W. Ruch Distinguished Chair at Sanford Burnham Prebys, weren’t convinced.
What began as their simple quest to trawl for life in some of the hottest natural springs on Earth would, decades later, change the trajectory of biological science forever, saving millions of lives.
Listen to Freeze recount a transformative moment in his early career on the latest Radiolab podcast.
A Conversation About Aging and Alzheimer’s Disease at Sanford Burnham Prebys
AuthorCommunications
Date
April 23, 2025
Event recording now available for panel discussion with scientists held on April 16, 2025
Sanford Burnham Prebys recently welcomed members of the San Diego community for an engaging afternoon focused on the intersection of aging and Alzheimer’s disease. Hosted as part of the A Conversation About series, the event featured a panel discussion with leading experts from the Center for Neurologic Diseases and Conrad Prebys Center for Chemical Genomics. The afternoon offered valuable insights into how the biology of aging contributes to neurodegenerative disease and how research at Sanford Burnham Prebys is helping to shape the future of prevention and treatment.
Panelists included Dr. Su-Chun Zhang, Dr. Timothy Huang, and Dr. Anne Bang, who discussed current findings, ongoing research collaborations, and the role of translational science in accelerating progress against Alzheimer’s. The panel also included author and caregiver advocate Muffy Walker, who introduced her new novel, Memory Weavers, which draws on her personal experience with Alzheimer’s disease.
The event was introduced by Reena Horowitz, founder of Group of 12 and Friends at Sanford Burnham Prebys, whose support has been instrumental in fostering dialogue around science and health within our community.
Watch Event Recording
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Science in Pictures
AuthorScott LaFee
Date
April 21, 2025
In the world of animal models, naked mole rats are supermodels despite their novel appearance. They are extremely long-lived, sometimes more than 30 years. They rarely get cancer. And they can survive up to 18 minutes without oxygen.
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Sanford Burnham Prebys scientist shared research on pancreatic cancer podcast
AuthorGreg Calhoun
Date
April 18, 2025
Cosimo Commisso, PhD, is interim director and deputy director of the NCI-Designated Cancer Center at Sanford Burnham Prebys.
Cosimo Commisso discussed his lab’s contributions to the pancreatic cancer research community on the Project Purple Podcast
Commisso discussed his lab’s work studying pancreatic cancer with Project Purple CEO and founder Dino Verrelli, including efforts to understand pancreatic cancer cells’ appetite for glutamine, one of the 20 amino acids used to build proteins throughout the body. The Commisso lab has shown that feeding tumors a glutamine copycat starves them of the fuel they need to survive and grow.
Commisso and Verrelli also discussed:
The complexity of pancreatic cancer, including changes in cellular metabolism and the microenvironment surrounding tumors.
The systemic effects from factors such as diet and aging on pancreatic cancer.
The importance of developing personalized diagnostics and therapies that account for individual differences.
The pancreatic cancer research community’s willingness to collaborate.
Opportunities to progress research more quickly to benefit patients as soon as possible.
Project Purple is a nonprofit organization focused on pancreatic cancer research and support for patients and their families. Since 2008, Project Purple has raised more than $3.5 million to support pancreatic cancer research efforts, including improving early detection and finding new curative treatments.
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Cancer drug finds new purpose in the brain
AuthorGreg Calhoun
Date
April 14, 2025
These representative MRIs of responding patients show tumors shrinking throughout the course of avapritinib therapy.
Scientists show that an established cancer drug travels to and shrinks some brain tumors, which may lead to new therapies for a disease with few treatments
Brain tumors are the leading cause of cancer-related death in childhood. The deadliest of these tumors are known as high-grade gliomas, with the grade referring to how quickly certain tumors grow and spread throughout the central nervous system.
Treatment options for high-grade gliomas are limited. Surgical removal is typically the first option depending on the tumor size and location. Radiation often follows to kill any remaining cancer cells to prevent another tumor from forming.
“Drug options to pair with surgery and/or radiation are few and far between,” saidLukas Chavez, PhD, associate professor in theCancer Genome and Epigenetics Programat Sanford Burnham Prebys. “A big reason for this is the blood-brain barrier being as formidable a boundary as the mythological River Styx.”
The blood-brain barrier can, at times, mean the difference between life and death. It protects the brain and spinal cord from potential toxins and pathogens circulating in the bloodstream. However, in its vigilance, it also blocks beneficial drugs from reaching the brain. This presents a major challenge, since most medications are designed to travel through the bloodstream after being ingested or injected.
Scientists from an international team including Sanford Burnham Prebys, the University of Michigan, Dana Farber Cancer Institute, the Medical University of Vienna and many other institutions published findings March 13, 2025, in Cancer Cell demonstrating that the drug avapritinib could treat certain brain tumor cells. And, like the Styx’s ferryman Charon, the medicine is one of the rare few that can cross the blood-brain barrier known to prevent the passage of more than 98% of small molecule drugs.
The researchers selected avapritinib—which is approved by the Food and Drug Administration for treating gastrointestinal and other cancers—after finding it was the strongest commercially available drug for inhibiting the gene Platelet-derived growth factor receptor alpha (PDGFRA), which is found to be mutated in 15% of high-grade gliomas.
Lukas Chavez, PhD, is an associate professor in the Cancer Genome and Epigenetics Program at Sanford Burnham Prebys.
In addition to showing that avapritinib inhibited PDGFRA in cancer cells and mouse brain tumors, the research team tested its effects on eight human pediatric and young adult high-grade glioma patients through a compassionate-use program. The treatment was found to be safe and investigators observed that the drug caused tumors to shrink in three patients.
“More research is needed to better understand how to best repurpose this drug for high-grade gliomas,” said Chavez. “We’ll learn a lot from the ongoing Rover study, a phase 1/2 multicenter trial of avapritinib based on these findings that will include more participants.”
The authors of the new study also highlighted the need to study combining multiple targeted therapies to overcome acquired resistance to any single treatment.
Mariella G. Filbin, MD, PhD, assistant professor of Pediatrics at Harvard Medical School and research co-director of the Pediatric Neuro-Oncology Program at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, is the lead contact on the study.
Carl Koschmann,MD, ChadTough Defeat DIPG Research Professor and associate professor of Pediatric Neuro-Oncology at the University of Michigan Medical School, and Johannes Gojo, MD, PhD, head of Pediatric Precision Oncology CNS and ITCC-Lab/Clinical Trials Unit at the Medical University of Vienna, are corresponding authors along with Filbin.
Lisa Mayr, Sina Neyazi, Kallen Schwark and Maria Trissal share first authorship of the study.
Additional authors include:
Owen Chapman, Sunita Sridhar, Rishaan Kenkre, Aditi Dutta, Shanqing Wang, and Jessica Wang from Sanford Burnham Prebys
Jenna Labelle, Sebastian K. Eder, Joana G. Marques, Carlos A.O. de Biagi-Junior, Costanza Lo Cascio, Olivia Hack, Andrezza Nascimento, Cuong M. Nguyen, Sophia Castellani, Jacob S. Rozowsky, Andrew Groves, Eshini Panditharatna, Gustavo Alencastro Veiga Cruzeiro, Rebecca D. Haase, Kuscha Tabatabai, Alicia Baumgartner, Frank Dubois, Pratiti Bandopadhayay and Keith Ligon from the Dana-Farber/Boston Children’s Cancer and Blood Disorder Center and Harvard Medical School
Liesa Weiler-Wichtl, Sibylle Madlener, Katharina Bruckner, Daniel Senfter, Anna Lammerer, Natalia Stepien, Daniela Lotsch-Gojo, Walter Berger, Ulrike Leiss, Verena Rosenmayr, Christian Dorfer, Karin Dieckmann, Andreas Peyrl, Amedeo A. Azizi, Leonhard Mullauer, Christine Haberler and Julia Furtner from the Medical University of Vienna
Jack Wadden, Tiffany Adam, Seongbae Kong, Madeline Miclea, Tirth Patel, Chandan Kumar-Sinha, Arul Chinnaiyan and Rajen Mody from the University of Michigan Medical School
Alexander Beck from Ludwig Maximilians University Munich
Jeffrey Supko and Hiroaki Wakimoto from Massachusetts General Hospital
Armin S. Guntner from Johannes Kepler University
Hana Palova, Jakub Neradil, Ondrej Slaby, Petra Pokorna and Jaroslav Sterba from Masaryk University
Louise M. Clark, Amy Cameron and Quang-De Nguyen from the Dana-Farber Cancer Institute
Noah F. Greenwald and Rameen Beroukhim from the Broad Institute of MIT and Harvard
Christof Kramm from University Medical Center Gottingen
Annika Bronsema from University Medical Center Hamburg-Eppendorf
Simon Bailey from Great North Children’s Hospital and Newcastle University
Ana Guerreiro Stucklin from University Children’s Hospital Zurich
Sabine Mueller from the University of California San Francisco
Mary Skrypek from Children’s Minnesota
Nina Martinez from Jefferson University
Daniel C. Bowers from the University of Texas Southwestern Medical Center
David T.W. Jones, Natalie Jager from Hopp Children’s Cancer Center Heidelberg