cancer Archives - Page 5 of 11 - Sanford Burnham Prebys

Tag: cancer

Institute News

Meet cancer researcher Karina Barbosa Guerra

AuthorMonica May
Date

February 3, 2021

Karina Barbosa Guerra in the lab

Barbosa Guerra is working to find better treatments for a deadly leukemia

For Karina Barbosa Guerra, touring a lab and meeting scientists as part of her Girl Guides troop—Mexico’s equivalent of the Girl Scouts—was a life-changing experience. Suddenly, she could see herself as a scientist.

Today, Barbosa Guerra is a graduate student in the Deshpande lab at Sanford Burnham Prebys, where she’s working to find better treatments for a blood cancer called acute myeloid leukemia (AML). We caught up with Barbosa Guerra as she prepares to take the virtual stage at the Diversity and Science Lecture Series at UC San Diego (DASL) to learn more about when she decided she wanted to be a scientist and where she can be found when not in the lab.

Tell us about the moment you realized you wanted to be a scientist.
According to my mother, I stated that I wanted to become a chemist to develop vaccines when I was ten years old. However, it wasn’t until middle school that I started cultivating my own sense of scientific curiosity. At that time, I was in a Girl Scouts program centered on HIV/AIDS peer education, so I began to read a bit more about viruses. It was incredibly amazing that they could linger undetected in our bodies—and that many questions about their biology remained unanswered. The more I learned, the less I felt I knew, and I wanted to follow that endless string of questions.

What do you study, and what is your greatest hope for your research?
I study a cancer called acute myeloid leukemia—specifically, subtypes that are hard to treat. Certain cancer cells, like stem cells, are pretty resilient and can self-renew. This enables them to resist therapy, so we want to discover better ways to target this particular feature. My research aims to find ways in which we can treat these leukemias based on their stem cell–like capabilities. My hope is that we can ultimately benefit the patients enduring harsh treatments and disease relapse, and along the way, illuminate the fascinating aspects of the biology behind effective treatments.

What do you wish people knew about science?
That it’s a team effort. The current coronavirus pandemic has really shown us that collaboration is at the heart of transformative science. I think that great ideas are best developed through discussion—and the thrill of putting the pieces together is way more enjoyable with company.

How do you think your lab colleagues would describe you?
Maybe as the girl with a bunch of notebooks. I like to make notes of everything. My notebooks are way more reliable than my memory.

What is the best career advice you’ve ever received?
Early in the graduate program, one of my mentors told me, “Be there,” meaning that I had to spend time with my science. If I were to discover something or make a great insight, I had to be there to do it, think it or see it.

What do you wish people knew about Sanford Burnham Prebys?
That this is such a welcoming community. I felt this the very first time I visited the campus, and I feel so at home here as a student. There are plenty of opportunities to engage with others and help each other out. I really enjoy the collaborative spirit of our little community.

Learn more about the Institute’s Graduate School of Biomedical Sciences.

Institute News

Scientists uncover protein that empowers antibodies

AuthorMonica May
Date

January 11, 2021

antibodies illustration

Discovery may lead to better vaccine strategies and improve treatments for cancer and autoimmune disorders

Antibodies are the heroes of our immune system. They protect us from viruses, like SARS-CoV-2 (which can lead to COVID-19), as well as bacteria and other pathogens. They can provide lifelong protection from future infections—if they are strong enough. But, like any hero, they are fallible, and certain cancers or autoimmune disorders can arise when things go wrong.

Now, Sanford Burnham Prebys scientists have revealed that a protein called cyclin D3 tells antibody-producing B cells to start dividing—opening new research avenues that could improve vaccine development or the treatment of B cell lymphoma and autoimmune disorders. The discovery was published in Cell Reports.

Antibodies get their power from a complicated process. When an “intruder” is detected in the body, B cells—which produce antibodies—are activated. Each B cell is unique—they contain slight genetic variations to produce a diverse set of antibodies to attack the “intruder.” Later, they undergo optimization through a “survival of the fittest” process to identify the most protective versions.

“Our findings reveal that cyclin D3 is the ‘go’ signal for B cells to start rapidly dividing and producing a set of diverse antibodies,” says Parham Ramezani-Rad, PhD, a postdoctoral researcher in the Tumor Microenvironment and Cancer Immunology Program at Sanford Burnham Prebys and the lead author of the study. “This information might help scientists create better vaccine strategies in the future. On the flip side, researchers may be able to develop better weapons against B cell lymphoma and autoimmune disorders by removing malignant B cells.”

Parham Ramezani-Rad, PhD

​Parham Ramezani-Rad, a postdoctoral researcher at Sanford Burnham Prebys and lead author of the study.

Diving into the “dark zone”

After infection, B cells grow and divide in special structures called germinal centers that form in our spleen and lymph nodes. In this structure, a “dark zone”—referring to what scientists saw under the microscope in the 1930s—and a “light zone” are visible. Now researchers know the dark zone is where B cells are rapidly expanding, and this cell density appeared darker in the original microscope studies. After proliferating in the dark zone, B cells head to the light zone where the best potential antibody options are selected—while less desirable options are eliminated.

Cell Reports cover

Parham Ramezani-Rad designed the image that was featured on the cover of Cell Reports. The image is an artistic impression of the dynamics occurring inside of the germinal center, where antibody-producing B cells undergo a “survival of the fittest” selection process.

​Ramezani-Rad made the discovery when studying B cell lymphoma, a blood cancer that often contains a mutation that leads to hyper-stable cyclin D3. Using mice and sophisticated CRISPR gene editing technology, he discovered that cyclin D3 regulates the expansion or contraction of B cells specifically in the dark zone of germinal centers—and not the light zone. He also identified other regulatory aspects involved in this process that scientists might be able to harness for the benefit of human health.

“B cell lymphoma is often treated with an intensive chemo and immunotherapy combination. The side effects of this treatment can be immense, and relapses may occur,” says Ramezani-Rad. “Our findings about cyclin D3 could form the basis for a more tailored medicine that targets exactly what goes wrong during B cell lymphoma, and is potentially less toxic and more effective.”

Ramezani-Rad also designed the image that was selected for the journal cover, which is his artistic impression of the dynamics occurring inside the germinal center. He finds many parallels between scientists and artists.

“As a scientist, I see myself describing what already exists in nature,” explains Ramezani-Rad. “Musicians and painters are also describing the world. They are just using instruments or paint strokes to express emotions, whereas scientists use data to express knowledge.”

Institute News

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

Meet neuroscientist Paloma Sánchez Pavón

AuthorMonica May
Date

October 7, 2020

Paloma Sánchez Pavón in lab

Paloma’s research aims to protect premature babies from brain damage

Newborns have a new scientist in their corner: Paloma Sánchez Pavón, a graduate student in the lab of Jerold Chun, MD, PhD Paloma is working to find a medicine that could protect the still-developing brains of premature babies, which are incredibly delicate and prone to swelling. Called hydrocephalus, the condition is common—affecting one in 1,000 newborns—and repeated brain surgery is the only treatment.

We caught up with Paloma to learn more about what makes her tick, including why she decided to become a scientist and what she wishes people knew about research.

  • Did you always know you wanted to be a scientist? When you were a child, did you ever imagine you would be in the role you are today?
    I always knew I wanted to become a scientist, but I didn’t imagine I would be in the position I am today. Growing up, I was obsessed with the idea of becoming a marine biologist. I was fascinated by how much we didn’t know about the ocean. My plan was to move closer to the beach and enroll in a program that would allow me to learn more about it. Nevertheless, I soon realized that I was both mesmerized and terrified of the ocean (sharks, especially), and that I would never be able to spend enough time diving and exploring the water, which is what such a career would require. I was still passionate about biology and science in general, so I decided to study the most unknown (and equally unexplored) organ in the human body—the brain.
  • What do you study, and what is your greatest hope for your research?

    I study hydrocephalus, a condition that often affects premature infants. These newborns are extremely fragile and often accumulate fluid in their heads, which can cause brain damage or death. The only treatment is invasive brain surgery, required multiple times throughout individuals’ lives, to insert a shunt in their brains and drain the excess fluid so it is reabsorbed somewhere else in the body. This procedure is extremely uncomfortable for the patients and, like any other surgery, is associated with several risks that endanger their lives. I’m trying to understand the disease so we can find a better, less invasive treatment.

Paloma Sánchez Pavón at the beach in front of the ocean at sunset

When Paloma isn’t working in the lab, she can be found enjoying one of San Diego’s many beautiful beaches

  • What is one scientific question you wish you had an absolutely true answer to?
    To answer this question, I will step away from biology and turn to the universe. What is there beyond our galaxy? Will we be able to inhabit other planets? If we have so many things to still learn about the ocean and the brain, the universe is in a completely different category, with so many possibilities ahead of us.
  • What do you wish people knew about science?
    That it is fun. Experiments are about testing limits and going beyond what is known. I think that is really exciting. Also, science advances because we’re constantly asking new questions. Curiosity is what keeps this field in continuous evolution. And never be afraid to ask questions because science can be understood by everybody—it just needs to be explained well.
  • When you aren’t working in the lab, where can you be found? Where is your happy place?
    You will find me at the beach, walking along it or watching a sunset. One of the main reasons why I decided to move to San Diego is because I fell in love with its sunsets. You will also find me having brunch (my favorite American tradition) with my friends or enjoying a beer after work with them, especially around Encinitas or downtown San Diego.
  • What is the best career advice you have ever received?
    Never stop pushing the boundaries of knowledge. A curious mind is what keeps a scientist passionate about their job. Experiments usually don’t work the first time. You have to keep asking new questions and learning from your mistakes. Finishing a project takes time, but every day is unexpected and exciting because you don’t know what you’re going to find. That is the thrilling part about being a scientist.
  • What do you wish people knew about Sanford Burnham Prebys?
    What a great community Sanford Burnham Prebys is. I’ve never been in such a collaborative environment, where you work closely not only with students and postdocs, but also with faculty members. Everyone is always willing to help, whether that is lending reagents or advising about different techniques. As a student, this is what I value the most because it helps me develop as a scientist in an extremely enriching way.

Learn more about our Graduate School of Biomedical Sciences.

Institute News

Scientists “turn back time” on cancer using new stem cell reprogramming technique

AuthorMonica May
Date

August 21, 2020

human cells

Discovery opens new research avenues that may help catch cancer early and identify potential preventive treatments

Scientists at Sanford Burnham Prebys Medical Discovery Institute have reprogrammed cancer cells back into their pre-cancer identity—opening new doors for studying how cancer develops and how it might be prevented. The research, published in Stem Cell Reports, may lead to tests that identify cancer early on, when it can be more easily treated, and uncover preventive treatments that stop cancer before it starts.

“We believe we have been able to contribute to one of the major goals of modern cancer research: creating next-generation models for studying how cancer develops from its earliest state,” says Evan Snyder, MD PhD, professor and director of the Center for Stem Cells & Regenerative Medicine at Sanford Burnham Prebys and senior author of the study. “We essentially took an adult cancer that has accumulated many mutations and pushed it back to the earliest stages of development, allowing us to emulate a tumor’s premalignant state. Then we watched cancer emerge from normal cells before our eyes.”

Turning back the clock on cancer 

In the study, the scientists set out to transform cells from anaplastic thyroid tumors—an aggressive, fast-growing cancer that is nearly always diagnosed at late stages—into induced pluripotent stem cells (iPSCs). These cells model the embryonic cells that are present at the earliest stages of human development and can become any cell in the body. While iPSCs are used today to create unlimited supplies of cells for research and therapeutic purposes—usually to correct abnormalities—the scientists recognized that tumor-derived iPSCs could be used to study the development of cancer.

However, this feat turned out to be easier said than done. The standard reprogramming method didn’t work, requiring the researchers to hunt for a different method that would induce the cancer cells to reset. Inhibiting a protein called RAS was the key ingredient that coaxed these thyroid cancer cells to become normal iPSC cells.

“We have named the pathway that is critical for making a cancer cell act as if it were a normal cell its ‘reprogram enablement factor,’” explains Snyder. “That factor will likely be different for every cancer and, in fact, may help in defining that cancer type.

“For this cancer type, which we examined in our study as a proof-of-concept, the reprogram enablement factor turned out to be blunting an overactive RAS pathway,” Snyder continues. “Our results suggest that losing control of RAS was the ‘big bang’ for this cancer—the very first event that leads to out-of-control cell growth and development of a tumor.”

The scientists next plan to reprogram additional cancers—including brain and lung cancer—into iPSCs to determine their “reprogram enablement factors.” If successful, they will next map the molecular changes that occur immediately before and after the tumors develop, which could reveal early signals of cancer and new preventive or early treatment measures.

“Unlike other cells, cancer cells are notoriously resistant to reprogramming,” says Snyder. “Our study is the first to successfully reprogram cancer cells into completely normal iPSCs, which opens new doors for cancer research.”

A team effort

The first author of the study is Yanjun Kong of Sanford Burnham Prebys and Shanghai Jiao Tong University. Yang Liu of Sanford Burnham Prebys is a co-corresponding author. Additional study authors include Ryan C. Gimple of UC San Diego; Rachael N. McVicar, Andrew P. Hodges and Jun Yin of Sanford Burnham Prebys; and Weiwei Zhan of Shanghai Jiao Tong University.

This study was funded by the Stem Cell Research Center & Core Facility at Sanford Burnham Prebys and by the China Scholarship Council (201606230202). The study’s DOI is 10.1016/j.stemcr.2020.07.016.

Institute News

Sanford Burnham Prebys researchers awarded 2020 Padres Pedal the Cause grants

AuthorSusan Gammon
Date

July 9, 2020

Sanford Burnham Prebys - Padres Pedal the Cause; team riders at SBP

We are pleased to announce that Padres Pedal the Cause (PPTC) has awarded three collaborative research grants to Sanford Burnham Prebys, Moores Cancer Center at UC San Diego Health and the Salk Institute. Funding for the research comes from the record setting $3.1 million raised in the 2019 event and brings the lifetime raise for PPTC to over $13 million.

PPTC’s goal is to leverage the strengths of San Diego—home to three nationally recognized NIH cancer institutions and a renowned pediatric hospital. Each grant unites scientists at beneficiary institutions and aims to advance research toward developing therapies to attack and cure cancer.

Congratulations to the recipients!

  • Robert Wechsler-Reya, PhD, (SBP) and John Crawford, MD, (Moores Cancer Center/Rady Children’s) will work on a new approach to treat medulloblastoma—the most common malignant brain tumor in children.
  • Garth Powis, D. Phil., (SBP) Pradipta Ghosh, MD, (Moores Cancer Center) and Michael Bouvet, MD, (Moores Cancer Center) are joining forces to find medical treatments for gastric cancer—a disease for which no therapy exists. 
  • Nicholas Cosford, PhD, (SBP) Hatim Husain, MD, (Moores Cancer Center) and Reuben Shaw, PhD, (Salk Institute) will perform a first-of-its-kind study for lung cancer—the number one cause of cancer-related deaths per year.

The PPTC event featured multiple cycling courses, a 5K run or walk, spin classes and kid-friendly activities. The number of participants reached an all-time high of nearly 3,000 in 2019.

Congratulations to everyone who worked, played and cycled their way to success!

Read the full list of 2020 grants funded by Padres Pedal the Cause.

Institute News

Padres Pedal the Cause donates $3.1 million to cancer research

AuthorMonica May
Date

February 18, 2020

Garth Powis (fourth from left) who heads our National Cancer Institute (NCI)-designated Cancer Center—one of only seven in the nation—joined the beneficiaries onstage for a check presentation ceremony.

Garth Powis, who heads our NCI-designated Cancer Center, joined representatives from the beneficiary institutes onstage for the check presentation ceremony.

Padres Pedal the Cause (PPTC) hit it out of the ballpark, revealing that its 2019 event at Petco Park raised a record $3.1 million to accelerate local cancer research. 

The amount, revealed in an evening ceremony held on Thursday, February 6, brings the total raised to $13.2 million. Garth Powis, D. Phil., director of Sanford Burnham Prebys’ National Cancer Institute (NCI)-designated Cancer Center—one of only seven in the nation—joined representatives from the beneficiary institutes onstage for the check presentation ceremony. 

The audience in the packed auditorium gave the news a standing ovation. Most guests, who included hundreds of event participants, San Diego business leaders, top donors and fundraisers, as well as Padres Pedal founders Bill and Amy Koman, have firsthand experience with cancer—the number-one cause of death in San Diego. 

Typically held in November, PPTC features multiple cycling courses, a 5K run or walk, spin classes and kid-friendly activities. The number of participants has steadily increased since the event’s launch in 2013—reaching an all-time high of nearly 3,000 individuals last year. 

The nonprofit’s goal is to leverage the incredible strengths of San Diego—home to three nationally recognized NIH cancer institutions and a renowned pediatric hospital—to bring us closer to cancer cures. Each PPTC grant unites scientists at two or more of the four beneficiary institutions, which include Sanford Burnham Prebys, Moores Cancer Center at UC San Diego Health, Rady Children’s Hospital and the Salk Institute for Biological Studies. Past grants have accelerated our research into cancers of the breast, skin, brain, colon, pancreas and more. 

The 2020 event date will be revealed in mid-April. Visit www.gopedal.org for the latest details.

Institute News

How to help children survive—and thrive—after a brain cancer diagnosis

AuthorMonica May
Date

January 13, 2020

Scientist Robert Wechsler-Reya of Sanford Burnham Prebys and Rady Children’s Institute for Genomic Medicine, clinician John Crawford of Rady Children’s Hospital–San Diego, pediatric brain cancer survivor Travis Selinka, patient advocate Lynne Selinka, and patient advocate Tony Selinka

Lynne Selinka knew in her heart that something was seriously wrong with her 10-year-old son, Travis. For months he had experienced dizziness, vomiting and headaches, despite his doctor’s best efforts to find a cause. A visit to Rady Children’s Hospital-San Diego revealed a heartbreaking diagnosis: Travis had a malignant brain tumor. He was operated on the next day and then endured two months of radiation treatment followed by six rounds of chemotherapy.

“That year, Travis asked Santa, ‘Can I please be done with chemo before Christmas?’” Lynne said. “It was by far the hardest year of our life.”

Brain tumors are the most common cause of cancer-related death in children—recently surpassing leukemia. To help the public learn about the latest efforts to develop better treatments for pediatric brain cancer, our Institute teamed up with the Fleet Science Center to host a panel discussion on Sunday, December 8. Travis and his parents, Lynne and Tony, shared their story alongside the clinician who treated Travis, John Crawford, MD, director of Pediatric Neuro-Oncology at Rady Children’s Hospital-San Diego; and a scientist working on personalized treatments for pediatric brain cancer, Robert Wechsler-Reya, PhD, of Sanford Burnham Prebys and Rady Children’s Institute for Genomic Medicine. 

As the speakers explained, while aggressive therapies have improved outcomes for children with brain tumors (today Travis is a junior in high school), one in four children with a malignant brain tumor does not survive. Children who do survive have an increased risk of severe long-term side effects from undergoing aggressive treatment at such a young age, including developing additional cancers or experiencing intellectual disability. Six years after he was declared cancer-free, Travis was diagnosed with chronic myeloid leukemia, a type of blood cancer caused by his previous chemotherapy. So far, his new treatment is working.

Wechsler-Reya hopes his work to develop personalized therapies based upon an individual’s tumor could help spare children from this painful experience. By analyzing patient tumor samples—obtained from Rady Children’s Hospital—his team works to understand the cancer at a molecular level, studying the tumor’s DNA mutations, changes in gene expression, responses to drugs, and much more. Armed with this information, the scientists then work to find therapies that are customized to a child’s specific tumor—and may be more effective and less toxic.

“For pediatric brain cancer, success doesn’t just mean better treatments. It also means developing treatments with fewer long-term side effects,” says Wechsler-Reya. “If successful, this work might help more children not only survive brain cancer, but also live a long, healthy life after treatment.

Travis and his family welcome this work with open arms.  

“We try to look for a silver lining in every day. Travis has become an amazing public speaker and now shares his story with other children fighting brain cancer. But each part of our journey has been so hard—from receiving the diagnosis, seeing Travis go through a painful surgery and then chemo, not knowing if the treatments would work, and then being diagnosed with another cancer almost six years later,” said Lynne. “We are so grateful for the efforts of researchers who are working toward a world where a child doesn’t have to go through what Travis did—or at least is spared from some of the hardest parts of the journey.”

This event was the last of our five-part “Cornering Cancer” series at the Fleet Science Center. Read about our past discussions focusing on lung, blood, breast and pancreatic cancers.

Institute News

Prestigious Forbeck Scholar Award granted to Sanford Burnham Prebys cancer researcher

AuthorMonica May
Date

December 23, 2019

Brooke Emerling, PhD, headshot

Breast cancer expert Brooke Emerling, PhD, an assistant professor at Sanford Burnham Prebys, has been named a Forbeck Scholar by the William Guy Forbeck Research Foundation.

This prestigious award recognizes early-career cancer researchers for their achievements, research and dedication to the field. As an award winner, Emerling receives rare access to several three-day “think tank” events featuring the world’s top cancer clinicians and scientists.

“My goal is to create therapies that help more breast cancer patients survive cancer,” says Emerling. “The opportunity to discuss my ideas and research with the absolute leaders in my field is incredible and only accelerates my work toward that end.”

Emerling is working to find treatments for triple-negative breast cancer, which is treatable only with standard surgery, chemotherapy and radiation. The lack of specific treatments means that it has a mortality rate three times higher than the other types of breast cancer. Emerling is working to find a personalized medicine that blocks several proteins she identified that allow the triple-negative breast cancer to grow, called PI5P4Ks.

The William Guy Forbeck Research Foundation was established in 1985 by George and Jennifer Forbeck in honor of their son, who succumbed to a rare childhood cancer at age 11. Today the foundation promotes advances in cancer research through collaboration. The foundation began the Forbeck Scholar award as a way to recognize early-career cancer researchers with great future promise. Past Forbeck Scholar award winners hail from Dana-Farber Cancer Institute, the Broad Institute, Cold Spring Harbor Laboratory and other top-tier institutes.

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.