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Our top 10 discoveries of 2020

AuthorMonica May
Date

December 14, 2020

notebook with Top 10 written at top of page

This year required dedication, patience and perseverance as we all adjusted to a new normal—and we’re proud that our scientists more than rose to the occasion.

Despite the challenges presented by staggered-shift work and remote communications, our researchers continued to produce scientific insights that lay the foundation for achieving cures.

Read on to learn more about our top 10 discoveries of the year—which includes progress in the fight against COVID-19, insights into treating deadly cancers, research that may help children born with a rare condition, and more.

  1. Nature study identifies 21 existing drugs that could treat COVID-19

    Sumit Chanda, PhD, and his team screened one of the world’s largest drug collections to find compounds that can stop the replication of SARS-CoV-2. This heroic effort was documented by the New York Times, the New York Times Magazine, TIME, NPR and additional outlets—and his team continues to work around the clock to advance these potential treatment options for COVID-19 patients.

  2. Fruit flies reveal new insights into space travel’s effect on the heart

    Wife-and-husband team Karen Ocorr, PhD, and Rolf Bodmer, PhD, shared insights that hold implications for NASA’s plan to build a moon colony by 2024 and send astronauts to Mars.

  3. Personalized drug screens could guide treatment for children with brain cancer

    Robert Wechsler-Reya, PhD, and Jessica Rusert, PhD, demonstrated the power of personalized drug screens for medulloblastoma, the most common malignant brain cancer in children.

  4. Preventing pancreatic cancer metastasis by keeping cells “sheltered in place”

    Cosimo Commisso, PhD, identified druggable targets that hold promise as treatments that stop pancreatic cancer’s deadly spread.

  5. Prebiotics help mice fight melanoma by activating anti-tumor immunity

    Ze’ev Ronai, PhD, showed that two prebiotics, mucin and inulin, slowed the growth of melanoma in mice by boosting the immune system’s ability to fight cancer.

  6. New test for rare disease identifies children who may benefit from a simple supplement

    Hudson Freeze, PhD, helped create a test that determines which children with CAD deficiency—a rare metabolic disease—are likely to benefit from receiving a nutritional supplement that has dramatically improved the lives of other children with the condition.

  7. Drug guides stem cells to desired location, improving their ability to heal

    Evan Snyder, MD, PhD, created the first drug that can lure stem cells to damaged tissue and improve treatment efficacy—a major advance for regenerative medicine.

  8. Scientists identify a new drug target for dry age-related macular degeneration (AMD)

    Francesca Marassi, PhD, showed that the blood protein vitronectin is a promising drug target for dry age-related macular degeneration (AMD), a leading cause of vision loss in Americans 60 years of age and older.

  9. Scientists uncover a novel approach to treating Duchenne muscular dystrophy

    Pier Lorenzo Puri, MD, PhD, collaborated with scientists at Fondazione Santa Lucia IRCCS and Università Cattolica del Sacro Cuore in Rome to show that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring.

  10. New drug candidate reawakens sleeping HIV in the hopes of a functional cure

    Sumit Chanda, PhD, Nicholas Cosford, PhD, and Lars Pache, PhD, created a next-generation drug called Ciapavir (SBI-0953294) that is effective at reactivating dormant human immunodeficiency virus (HIV)—an approach called “shock and kill.”

Institute News

A year in review: Our top 10 discoveries of 2019

AuthorMonica May
Date

December 4, 2019

hands holding up Most Popular sign

At Sanford Burnham Prebys, we uncover the origins of disease and launch bold new strategies that lay the foundation for achieving cures. This year our scientists made significant progress—revealing new insights into how we treat some of the deadliest cancers, address neurological disorders such as Parkinson’s and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) and more.

Read on to learn more about our top 10 discoveries of the year. To receive more frequent updates on our discoveries, subscribe to our monthly newsletter at the bottom of this page.

  1. One-two punch drug combination offers hope for pancreatic cancer therapy. Ze’ev Ronai, PhD, identified a combination of two anti-cancer compounds that shrank pancreatic tumors in mice—supporting the immediate evaluation of the drugs in a clinical trial. The study was published in Nature Cell Biology.
  2. Targeted treatment shrinks deadly pediatric brain tumors. Robert Wechsler-Reya, PhD, reported that a targeted therapy that blocks a protein called LSD1 shrank tumors in mice with a form of pediatric brain cancer known as medulloblastoma. LSD1 inhibitors are currently under evaluation in clinical trials for other cancers, which could speed their potential path to children. The study was published in Nature Communications.
  3. Epigenetic change causes fruit fly babies to inherit diet-induced heart disease. Rolf Bodmer, PhD, showed that reversing an epigenetic modification or over-expressing two genes protected fruit fly children and grandchildren from the negative heart effects of their parents’ fatty diet. These findings help explain how obesity-related heart failure is inherited and uncover potential targets for treatment. The study was published in Nature Communications.
  4. Amyotrophic lateral sclerosis (ALS) research reveals new treatment approach. Huaxi Xu, PhD, extended the survival of mice with ALS-like symptoms by elevating levels of a protein called membralin using a gene therapy approach. The study was published in the Journal of Clinical Investigations.
  5. How prostate cancer becomes treatment resistant. Jorge Moscat, PhD, and Maria Diaz-Meco, PhD, identified how prostate cancer transforms into an aggressive, treatment-resistant subtype called neuroendocrine prostate cancer (NEPC) following treatment with anti-androgen therapy. Their findings uncover new therapeutic avenues that could prevent this transformation from occurring and reveal that an FDA-approved drug holds promise as an NEPC treatment. The study was published in Cancer Cell.
  6. Boosting muscle stem cells to treat muscular dystrophy and aging muscles. Alessandra Sacco, PhD, uncovered a molecular signaling pathway that regulates how muscle stem cells decide whether to self-renew or differentiate—an insight that could lead to muscle-boosting therapeutics for muscular dystrophies or age-related muscle decline. The study was published in Nature Communications.
  7. Functional hair follicles grown from stem cells. Alexey Terskikh, PhD, created natural-looking hair that grows through the skin using human induced pluripotent stem cells (iPSCs), a major scientific achievement that could revolutionize the hair growth industry. Stemson Therapeutics has licensed the technology.
  8. Potential targeted treatment for acute myeloid leukemia identified. Ani Deshpande, PhD, showed that a protein called BMI1 is a promising drug target for an AML subtype in which two normally separate genes fuse together. The findings, published in Experimental Hematology, provide a rationale for evaluating a BMl1-inhibiting drug that is currently in clinical development as a potential treatment for this subtype.
  9. Antimicrobial protein implicated in Parkinson’s disease. An immune system protein that usually protects the body from pathogens is abnormally produced in the brain during Parkinson’s disease, Wanda Reynolds, PhD, reported in Free Radical Biology & Medicine. The discovery indicates that developing a drug that blocks this protein, called myeloperoxidase (MPO), may help people with Parkinson’s disease.
  10. Digestion-aiding herbs alter gut microbiome. Scott Peterson, PhD, found that four herbs—turmeric, ginger, long pepper and black pepper—promoted strong shifts in the gut bacteria that are known to regulate metabolism, providing insights that could help us protect our health. The study was published in Evidence-Based Complementary and Alternative Medicine.
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Nobel laureate Michael Rosbash presents his latest fruit-fly research at Sanford Burnham Prebys

AuthorMonica May
Date

February 8, 2019

Michael Rosbash and Rolf Bodmer, SBP

You may want to reconsider swatting that pesky fruit fly: Despite appearances, we share more than half of our genes with the tiny insect. For this reason—and their shorter life span and simpler genome—researchers often use the flies as models for human health and disease. 

Nobel laureate Michael Rosbash, PhD, is one such scientist. A self-described “fly chauvinist,” this month he visited SBP to present his latest research on the circadian rhythm of fruit flies. The event quickly became standing room only. 

Rosbash received the 2017 Nobel Prize in Physiology or Medicine, along with Jeffrey Hall and Michael Young, for their work uncovering the molecular timekeepers behind circadian rhythm. Their work was conducted in fruit flies, but has since unlocked new discoveries in animals and plants. 

Rolf Bodmer, PhD (pictured left), who introduced Rosbash (pictured right), is using fruit flies to uncover how our heart develops and ages, with a particular focus on a heart rhythm disorder called AFib. Nearly 10 percent of people over the age of 65 develop the condition, a leading cause of stroke, but we don’t know its cause, and there is no cure. By studying AFib in fruit flies, Bodmer and his team, which includes a cardiologist at Scripps Clinic, are hoping to learn the cause of the disorder and find effective treatment(s). 

While he has all the reason in the world to have an ego, Rosbash remains humble and down-to-earth. He ended his presentation by thanking his lab, without whom he would “not have a job and such prizes.” 

Interested in keeping up with SBP’s latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.

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Preuss School interns get an “A” grade at SBP

AuthorHelen I. Hwang
Date

August 4, 2017

SBP Board Trustee Malin Burnham with Arturo Torres Jimenez and Josué Barragán

“I got to do things I never thought I could do,” said Yadira Gomez Rangel, 16, a rising junior at Preuss School in San Diego. “I got a chance to dissect a fly, which I didn’t think I could do,” she told the audience at Sanford Burnham Prebys Medical Discovery Institute (SBP), which included SBP Trustees Malin Burnham and Wain Fishburn as well as CEO Perry Nisen, MD, PhD

Rangel is one of seven students from the prestigious Preuss School, who completed a two-week internship. Students from the Preuss School, affiliated with UC San Diego, strive to become the first in their families to graduate from college. The SBP Preuss program is designed to introduce young scientists-in-training to medical research by working hand in hand with our scientists.

The group of 16-year-olds got a chance to rotate among four different labs at SBP. The other students included Michelle Villa Bardales, Josué Barragán, Edizandro Morales Herrera, Arturo Torres Jimenez, Jenny Nguyen and Natalie Nguyen. Students presented posters in English and Spanish, received a certificate and a stipend for their hard work.

Fishburn said the Preuss program at SBP was “inspirational” as he hoped the young teens would continue their path in science. At the celebratory luncheon with students, their families and SBP staff, Fishburn chatted with Tommy Le, a Preuss School graduate. Le was part of the SBP Preuss program for the two-week internship, followed by a six-week internship the following year, and is now doing a summer internship at SBP before entering UC San Diego in the fall where he’ll major in biochemistry.

Each summer, SBP also hosts a six-week internship for rising Preuss seniors, sponsored by the NIH CURE program. Two of the seven interns (who happen to be all female), Gizelle Avitia Mejica and Julieta Morales Ornelas, also completed the two-week Preuss program, which inspired them to apply again at SBP. “About 90 percent of what I learned in the lab I wouldn’t have been taught in the classroom,” says Mejica.  

During the internship, the teenagers studied several aspects of medical research. They examined the correlation between obesity and heart disease in fruit flies in the laboratory of Rolf Bodmer, Ph.D. Also, the kids studied zebrafish and tackled the challenge of curing diabetes in the laboratory of Duc Dong, Ph.D. They looked at how to use C. elegans worms to understand the aging process in the laboratory of Malene Hansen, Ph.D. Finally, in the laboratory of Jing Crystal Zhou, Ph.D., the young scientists learned about RNA modification, a process that occurs in all living organisms and can influence how diseases occur.

With hands-on training and in-depth laboratory involvement, the Preuss students gained invaluable skills and networking opportunities. The program is made possible by founding philanthropists Peggy and Peter Preuss and Debby and Wain Fishburn. Jimenez said, “It’s been a wonderful experience!”

Preuss School Internship Program with SBP Trustees​

 

Institute News

2017 BIO features SBP scientists

AuthorKristen Cusato
Date

June 19, 2017

Bio2017

Two SBP researchers will be sharing their knowledge and insights at the 2017 BIO International Convention, which will be held June 19th to the 22nd at the San Diego Convention Center.

Rolf Bodmer, PhD, professor in the Development, Aging and Regeneration Program, will be part of a panel on June 21st titled, “Reimagine Old Age: New Frontiers in the Science of Aging.”  Bodmer will highlight new biomedical research, as other panelists report on drugs that are presently in development and discuss how pharmaceutical companies are approaching new treatments for diseases related to aging.

 

“With our population aging, it becomes more important to understand and find ways to remedy the rapid decline and diseases that come with getting older,” Bodmer says. “We don’t necessarily need to live that much longer, but we need to find out how to have all of our organ systems work their best, to live healthier and happier as we age.”

 

Bodmer will also talk about the use of fruit fly models in his lab at SBP to study different aspects of aging, including cardiac function.

 

“Organisms like the fruit fly have an infinite box of genetic tools that can be used to figure out how our genetics and signaling pathways work. What we learn from them can help in the development of drugs for people with age-related diseases,” says Bodmer.

 

Rolf Bodmer, PhD

 

Reimagine Old Age: New Frontiers in the Science of Aging

 

June 21st 10:45am Room 6C upper level SDCC

Scott Peterson, PhD, professor in the Tumor Microenvironment and Cancer Immunology Program, will be on the “Microbiome 2.0: Going Beyond Bugs as Drugs” panel, also on June 21st.  Dr. Peterson’s focus will be on using prebiotics as a way of modulating or altering the gut microbiome to impact health.

Additional panelists will discuss new microbiome targets, novel approaches to regulate the gut-brain axis and how big data fits in.

“Beyond bugs as drugs means it’s no longer just about probiotics, now prebiotics have become part of the equation,” Peterson says. “We want to know if we can use pre-biotics to change someone’s microbiome to make them more responsive to certain drugs.”

Peterson says the translational aspect of microbiome research is starting to take form, and he is looking forward to talking about his research with potential new partners at BIO 2017 and beyond.

“NIH funding is hard to come by right now, and getting biotech interested in the microbiome will eventually benefit all of us.”

Scott Peterson, PhD

Microbiome 2.0: Going Beyond Bugs as Drugs

June 21st 4:15pm Room 6C upper level SDCC

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An “Odd” gene affects aging of the heart

AuthorJessica Moore
Date

February 1, 2017

hands holding heart

As we get older, our hearts change in ways that make it harder for them to pump blood. They become stiffer, less efficient at generating energy, and more likely to respond to damage with inflammatory chemicals. To help find new ways to slow that decline, researchers in the laboratory of Rolf Bodmer, PhD, professor and director of the Development, Aging and Regeneration Program at Sanford Burnham Prebys Medical Discovery Institute (SBP), are looking at how the heart ages at a molecular level.

Bodmer’s team recently discovered a new potential contributor to cardiac aging, a protein called Odd, opening up a novel direction for research on therapies to prolong heart health. In their study, published in the journal Aging Cell, the gene for Odd, which controls the activity of other genes by turning them on or off, was found to be turned up in the hearts of old fruit flies. Bodmer’s lab studies flies because their hearts deteriorate with age in the same ways that human hearts do, but their genetics are much simpler.

“It’s intriguing that Odd is linked to aging because its known function is in early development—it’s crucial for the heart to form properly, and, as we found here, is also important for preventing the heart from deteriorating prematurely,” says Bodmer.

Odd’s involvement in cardiac aging was uncovered by a genome-wide comparison of the genes that are active in the hearts of young and old flies. Odd was one of over 200 genes whose activity was significantly elevated in older flies. Remarkably, further analysis showed that in aging hearts, increasing Odd activity temporarily protects the heart from decline by supporting proper electrical function and heart rate.

“Our findings suggest that increased levels of Odd in older hearts may be a way to compensate for aging-associated loss of function,” comments Bodmer. “In combination with a companion paper showing that another gene-regulating protein, FoxO, helps preserve the adult heart, they support a growing body of evidence that genes that are crucial in development are also important to keep the heart running well into old age.”

Bodmer contributed to the other paper, from the lab of Anthony Cammarato, PhD, assistant professor at Johns Hopkins University School of Medicine, and previously a staff scientist in Bodmer’s lab. The paper showed that FoxO helps protect the aging heart by turning on genes that help get rid of unneeded proteins.

“Following up on the findings of both studies could point to ways to keep our hearts working better for longer,” Bodmer adds.

The Bodmer lab paper is available online here and the Cammarato lab paper is here.

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SBP’s 37th Annual Symposium: Aging and Regeneration

Authorsgammon
Date

November 3, 2015

Header image

On Friday, October 30, more 350 people came to SBP’s 37th Annual Symposium to hear leading scientists present their latest research on aging and regeneration.  The presenters, listed here, provided valuable insight into the latest studies on what causes aging, and strategies to repair injuries, prolong life, and prevent diseases.  The event was hosted by (from left to right): Rolf Bodmer, PhD, Malene Hansen, PhD, (in bee costume for Halloween) Alexey Terskikh, PhD

 

organizers-symposium-beaker

Many congratulations to Esther Minotti for successfully organizing the event!

symposium-photo-beaker

And many thanks to the Glenn Foundation for Medical Research for their support.

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Novel model for cardiomyopathy paves the way for new therapies

Authorsgammon
Date

May 29, 2015

Header image

A new fruit fly model that captures key metabolic defects associated with cardiomyopathy could translate into more-effective treatments for this potentially deadly heart condition, according to a study conducted by researchers at Sanford-Burnham and the Universidad Autónoma de Madrid in Spain. The findings, published April 9 in Human Molecular Genetics, could also have broader clinical implications for human metabolic diseases affecting other organ systems such as the liver and skeletal muscle. Continue reading “Novel model for cardiomyopathy paves the way for new therapies”

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New insights into how the heart forms may help identify heart defects

AuthorGuest Blogger
Date

September 29, 2014

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This is a post by our guest writer Janelle Weaver, PhD

The formation of the heart during development is a highly complex process that requires precise coordination between cells and molecular signaling pathways. The fruit fly has been widely used for studying the underlying cellular and molecular mechanisms, and a great deal is known about how the fate of heart cells is controlled by signaling pathways and transcription factors—proteins that control gene activity. But beyond that, events that regulate heart formation have not been clear. Continue reading “New insights into how the heart forms may help identify heart defects”

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The bright side of free radicals

Authorsgammon
Date

September 17, 2014

Header image

In a new study by Rolf Bodmer, Ph.D., director of the Development, Aging, and Regeneration Program at Sanford-Burnham, and Hui-Ying Lim, Ph.D., assistant member of the Free Radical Biology and Aging Program at the Oklahoma Medical Research Foundation as lead author, researchers report a previously unrecognized role for reactive oxygen species (ROS) in mediating normal heart function. The findings show how under normal physiological conditions, ROS produced in non-muscle heart cells act on nearby muscle cells to maintain normal cardiac function. The results provide vital insight on how ROS direct cell communications, and in addition to the heart, may be important for the function of other organs.

“Until now, scientists knew that ROS in non-muscle heart cells affected nearby muscle cells in conditions of cellular damage and stress,” said Bodmer. “We have shown that ROS have an essential role in normal cardiac health. Understanding the fundamental communication systems in healthy and damaged hearts has important implications for developing protective and therapeutic interventions for cardiac diseases.”

ROS—a reputation of destruction ROS are free radicals that are usually associated with diseases such as cancer, cardiovascular, and neurodegenerative disorders. ROS have atoms with an unpaired electron in their orbit which can send them on a rampage to pair with other molecules, including DNA—causing mutations that contribute to disease. Antioxidants are molecules that soak up the extra electron and remove free radicals, raising the possibility that antioxidant vitamins and supplements might have a protective role in human health.

Opinions on antioxidant supplements are highly polarized. Several large-scale randomized trials of supplements have had inconsistent results and the antioxidant pendulum appears to be swinging from healthy to insignificant, and in some cases even toxic. More reliable data is needed to better define the role of antioxidants in the prevention of cardiovascular and other diseases.

ROS regulate cardiac function by cell-to-cell signaling The new study, published in Cell Reports, illustrates a previously unappreciated role for ROS signaling in the heart and supports the critical concept that optimal levels of ROS are needed in the body to provide protection to the heart and other organs.

“Interestingly, we found that ROS do not diffuse from non-muscle cells into cardiac muscle cells to exert their function. Instead, ROS in the non-muscle (pericardial) cells exert their function by starting a specific signaling cascade within the cell that in turn acts on nearby cardiac muscle cells to regulate their proper function,” said Lim. “Although the precise mechanism by which ROS maintain cardiac functions has yet to be established, our research provides a more complete understanding of the functional interactions between cardiac muscle cells and non-muscle cells—and possibly cell-to-cell (paracrine) communications in other tissues.”

The research team used Drosophila melanogaster—the common fruit fly—to decipher the ROS signals that impact the cell function. The Drosophila heart shares many of the same genes, proteins, and structural characteristics with humans, and has been used for decades as a model to understand the human genes that govern healthy development as well as those involved with disease.

A link to the paper can be found at: http://www.cell.com/cell-reports/abstract/S2211-1247(14)00143-0