brain cancer Archives - Sanford Burnham Prebys

Tag: brain cancer

Institute News

Sanford Burnham Prebys researchers awarded Curebound grants

AuthorMiles Martin
Date

March 20, 2023

Aninyda Bagchi, PhD, Linda Bradley, Lukas Chavez, PhD, Nicholas Cosford, PhD, and Michael Jackson, PhD

Each year, Sanford Burnham Prebys joins Padres Pedal the Cause, an annual fundraising event that raises money for Curebound which awards collaborative cancer grants in the San Diego area.

These grants include Discovery Grants, which provide seed funds for high-risk/high-reward research in the earliest phases, and Targeted Grants, which are larger awards ($500K) that help translate promising discoveries into treatments for the clinic.

In the 2022-2023 Curebound Research portfolio, five researchers from Sanford Burnham Prebys were awarded grants: Associate Professor Anindya Bagchi, PhD, Professor Linda Bradley, PhD, Assistant Professor Lukas Chavez, PhD, Professor Nicholas Cosford, PhD, and Professor Michael Jackson, PhD

2022 Discovery Grant: Treating incurable pediatric brain tumors 
Bagchi and Chavez will collaborate to advance a new therapeutic approach for medulloblastoma, the most common childhood brain tumor. They will be focusing on a gene called MYC, found only in the deadliest forms of medulloblastoma. This form of brain cancer is currently untreatable, but Bagchi and Chavez recently discovered a molecule that can help control the activity of the MYC gene and potentially inhibit the growth of medulloblastoma tumors. The researcher holds promise to reveal a new treatment approach for this incurable cancer. 

The grant is titled “Decoding the Role of the Long Non-Coding RNA PVT1 in Medulloblastoma.”

2023 Targeted Grant: Discovering a new immunotherapy drug for melanoma
Bradley will be working with Soo Jin Park, MD, from UC San Diego Health to advance a new immunotherapy approach for malignant melanoma. Despite recent advances, this type of skin cancer still causes thousands of deaths in the U.S. each year. The goal of their project is to develop a new drug for melanoma that can reactivate the tumor-killing properties of the patient’s own immune system. This therapeutic approach has the potential to destroy tumors that are resistant to existing therapies, which could help save lives.

The grant is titled, “Advancing Immune Checkpoint Inhibition of PSGL-1 for Treatment of Malignant Melanoma.
 

2022 Discovery Grant: Developing drugs for bone-metastatic prostate cancer
Cosford will work with Christina Jamieson, PhD, from the University of California, San Diego, to advance a new treatment approach for prostate cancer that has spread to the bones. Bone is the most common place for prostate cancer to metastasize, and this form of cancer is currently incurable. The researchers will look for drugs that can kill tumor cells by inhibiting autophagy, a process that promotes tumor progression. The results of the study could identify a new drug ready for clinical trials.

The grant is titled “Pre-Clinical Development of New Autophagy Targeting Drugs for Bone Metastatic Prostate Cancer.”

2022 Discovery Grant: Repurposing drugs for deadly childhood brain cancer
Jackson and Chavez will collaborate to identify new treatment options for ependymoma, an aggressive pediatric brain tumor and leading cause of death among childhood cancer patients. The researchers will screen patient tumor cells against drugs already approved by the FDA for other conditions, looking for drugs that could be repurposed to fight these tumors. Because FDA-approved drugs are known to be safe for humans, this may prove to be the quickest way to help patients currently living with this cancer. 

The grant is titled “High Throughput-Screen for Inhibitors of Pediatric Ependymoma.”

Institute News

Is cloud computing a game changer in cancer research? Three big questions for Lukas Chavez

AuthorMiles Martin
Date

February 22, 2023

Lukas Chavez, PhD

As an assistant professor at Sanford Burnham Prebys and director of the Neuro-Oncology Molecular Tumor Board at Rady Children’s Hospital, Lukas Chavez, PhD, leverages modern technology for precision diagnostics and for uncovering new treatment options for the most aggressive childhood brain cancers.

We spoke to Chavez about his work and asked him how modern technology—particularly cloud computing—is shifting the approach to cancer research.

How are you using new technologies to advance your research?

New technologies are helping us generate a huge amount of data as well as many new types of data. All this new information at our disposal has created a pressing need for tools to make sense of it and maximize their benefits. That’s where computational biology and bioinformatics come into play. The childhood brain cancers I work on are very rare, which has historically made it difficult to study large numbers of cases and identify patterns.

Now, data for thousands of cases can be stored in the cloud. By creating data analysis tools, we can reveal insights that we would never have seen otherwise. For example, we’ve developed tools that can use patient data in the cloud to categorize brain cancers into subtypes we’ve never identified before, and we’re learning that there are many more types of brain tumors than we’ve previously understood. We’re basically transforming the classic histo-pathological approach that people have studied for decades by looking at tumor tissues under the microscope and turning that into data science.

How is cloud computing improving cancer research in general?

Assembling big datasets delays everything, so I believe the main idea of cloud computing is really to store data in the cloud, then bring the computational tools to the data, not the other way around.

My team did one study where we assembled publicly available data, and basically downloaded everything locally. The data assembly process alone took at least two to three years because of all the data access agreements and legal offices that were involved.

And that is the burden that cloud computing infrastructures remove. All of this personalized cancer data can be centrally stored in the cloud, which makes it available to more researchers while keeping it secure to protect patient privacy. Researchers can get access without downloading the data, so they are not responsible for data protection anymore. It’s both faster and more secure to just bring your tools to the data.

Are there any risks we need to be aware of?

Like any new technology, we need to be clear about how we use it. The technology is another tool in the toolbox of patient care. It will never entirely replace physicians and researchers, but it can complement and assist them.

Also, because we use costly and sophisticated tools that are being built and trained on very specific patient groups, we need to be careful that these tools are not only helping wealthier segments of society. Ideally, these tools will be expanded worldwide to help everybody affected by cancer.

Institute News

Brain cancer researcher Jia Zack Shen wins 2022 Eric Dudl scholarship

AuthorMiles Martin
Date

October 18, 2022

Professor Cosimo Comisso, Brett Dudl, Dr. Jim Dudl, Barbra Dudl, and scholarship winner Zack Shen

For the first time since the pandemic, Sanford Burnham Prebys presented the Eric Dudl Endowed Scholarship Award in person at last week’s Cancer Center Open House. This year’s recipient, selected by leaders at the Cancer Center, was Jia Zack Shen, PhD, a staff scientist in the lab of Charles Spruck, PhD The award pays tribute to Eric Dudl, a postdoctoral researcher who succumbed to cancer in 2006, when he was just 33.

“Eric was ill at such a young age, but he was also very lucky because he knew exactly what his dream job was and what his life meant,” says Shen. “Eric’s compassion and dedication to cancer research has been inspiring and encouraging for our postdocs here at Sanford Burnham Prebys. Thank you to the Dudl family for helping me continue my career here.”

Honoring Eric Dudl

In 2005, Eric Dudl had just begun his postdoctoral research in a cancer lab at Sanford Burnham Prebys, where he was known as kind, helpful and a fast learner. Then, at age 32, he was diagnosed with non-small cell lung cancer. This only fueled his fiery passion for cancer research.

“Even when he was very ill, Eric wanted to make a contribution in the time he had,” says Jim Dudl, MD, Eric’s father. “One day I looked at Eric and asked, ‘Why don’t you take some time off work? Get your energy back and go back in when you feel better?’ He looked up at me and said, ‘Why would I do that? This is the best job in the world!’”

Tragically, Eric Dudl would pass away in 2006, at age 33. The next year, his parents established the Eric Dudl Endowed Scholarship Fund to support young cancer researchers like their son.
“These talented scientists pick up where Eric had to leave off,” says Barbara Dudl, Eric’s mother.

Eric Dudl

Eric Dudl

“We are so grateful to the Institute for their compassion for Eric during his illness and helping us create this scholarship. The scientists who are now working on new discoveries might one day save the life of someone like Eric.”

The award presentation was emceed by Cosimo Commisso, PhD, and featured comments from Eric’s parents, as well as his brother, Bret.

“This scholarship fund is the best way we can honor Eric, because he was so passionate about education and supporting others,” says Bret. “The fact that he now helps other postdocs further their work to fight cancer is exactly what he would have wanted.”

Meet the recipient: Jia Zack Shen

Shen started at the Institute as a postdoctoral researcher in 2016, winning a Fishman Fund Award that year. He has since transitioned to a permanent role as a staff scientist, and the funding from this award will help continue to support his role.

“Sanford Burnham Prebys has a great, noncompetitive atmosphere, and the resources we have here are excellent,” says Shen. “Combining cancer research with the drug discovery capabilities we have at SBP is my dream.”

Shen’s research focuses on killing cancer stem cells by shutting off their ability to self-renew and by promoting a response from the immune system, delivering what Shen and his colleagues call a “one-two punch.” Shen has been working specifically on glioblastoma, one of the most aggressive brain cancers.

“Glioblastoma is one of the most devastating diseases, and there is a desperate need for better ways to treat it,” says Shen. “I am working hard to leverage the immune system to fight glioblastoma and save thousands of lives.”

Institute News

Heating up cold brain tumors: An emerging approach to medulloblastoma

AuthorMiles Martin
Date

July 6, 2022

Cancer cells

Immunotherapy has revolutionized cancer treatment, but it doesn’t work on many childhood brain tumors. Researchers from Sanford Burnham Prebys are working to change that.

Brain tumors account for about a quarter of all cancer cases in children. Medulloblastoma, a particularly aggressive form of childhood brain cancer, often develops resistance to radiation and chemotherapy. Researchers from Sanford Burnham Prebys are working to solve this problem by harnessing the power of the immune system.

They describe the potential of this approach in their recently published paper in Genes & Development. 

“The brain’s location makes it very difficult to target medulloblastoma tumors with current therapies,” says first author Tanja Eisemann, PhD, a postdoctoral associate in the lab of Robert Wechsler-Reya, PhD “They’re also immunologically cold, which means they’re good at evading the immune system.” 

The researchers hypothesize that it may be possible to enhance the body’s immune response to medulloblastoma and help the body’s immune cells enter the brain, making treatment with immunotherapy possible.

“Immunotherapy has so much potential as a  cancer treatment, but its scope is limited right now,” says Eisemann. “We want to bring the benefits of this therapy to medulloblastoma patients and their families.”

Eisemann has been studying this approach in mice, and although the research is still at an early stage, she and her colleagues are highly optimistic about its potential.

“The brain has long been considered immune privileged, hidden from immune-system surveillance and immune responses. But we’re starting to see that this isn’t the case,” says Eisemann. “This is a rapidly evolving field, and I’m excited to be working in a lab on the forefront of that research.”

Institute News

Boosting immunotherapy in aggressive brain cancer

AuthorMiles Martin
Date

November 3, 2021

Blue and orange illustration of human brain against black background

Researchers from Sanford Burnham Prebys have collaborated the University of Pittsburgh Cancer Institute to reveal a new approach to enhance the effects of immunotherapy in glioblastoma, one of the most aggressive and treatment-resistant forms of brain cancer.

The study, published recently in Cancer Discovery, describes a novel method to ‘turn off’ cancer stem cells—the malignant cells that self-renew and sustain tumors—enabling the body’s own defense system to take charge and destroy tumors.

“Tumors are more than just masses of cells—each one is a complex system that relies on a vast network of chemical signals, proteins and different cell types to grow,” says senior author Charles Spruck, PhD, an assistant professor at Sanford Burnham Prebys. “This is part of why cancer is so difficult to treat, but it also presents us with opportunities to develop treatment strategies that target the machinery powering tumor cells rather than trying to destroy them outright.”

Glioblastoma is an extremely aggressive form of cancer that affects the brain and the spinal cord. Occurring more often in older adults and forming about half of all malignant brain tumors, glioblastoma causes worsening headaches, seizures and nausea. And unfortunately for the thousands of people who receive this diagnosis each year, glioblastoma is most often fatal.

“We haven’t been able to cure glioblastoma with existing treatment methods because it’s just too aggressive,” says Spruck. “Most therapies are palliative, more about reducing suffering than destroying the cancer. This is something we hope our work will change.”

Immune checkpoint inhibitors—which help prevent cancer cells from hiding from the immune system—can be effective for certain forms of cancer in the brain, but their results in glioblastoma have been disappointing. The researchers sought a way to improve the effects of these medications.

“Modern cancer treatment rarely relies on just one strategy at a time,” says Spruck. “Sometimes you have to mix and match, using treatments to complement one another.”

The researchers used genomic sequencing to investigate glioblastoma stem cells. These cells are the source of the rapid and consistent regeneration of glioblastoma tumors that make them so difficult to treat.

The team successfully identified a protein complex called YY1-CDK9 as essential to the cells’ ability to express genes and produce proteins. By modifying the activity of this protein complex in the lab, the team was able to improve the effectiveness of immune checkpoint inhibitors in these cells. 

“Knocking out this transcription machinery makes it much more difficult for the cells to multiply” says Spruck. “They start to respond to chemical signals from the immune system that they would otherwise evade, giving immunotherapy a chance to take effect.” 

While the approach will need to be tested in clinical settings, the researchers are optimistic that it may provide a way to improve treatment outcomes for people with glioblastoma. 

“What our results tell us is that these cells are targetable by drugs we already have, so for patients, improving their treatment may just be a matter of adding another medication,” adds Spruck. “For a cancer as treatment-resistant as glioblastoma, this is a great step forward.”

Institute News

Conrad Prebys Foundation provides $3 million for pediatric brain cancer research

AuthorSusan Gammon
Date

April 7, 2021

Conrad Prebys Foundation Award

Conrad Prebys was an extraordinary man and a passionate philanthropist. Today, his generosity extends beyond his life through the Conrad Prebys Foundation.

This year, the Foundation provided $3 million to Robert Wechsler-Reya, PhD, and his team of researchers to advance a potential drug to treat medulloblastoma—the most common malignant brain tumor in children.

Children with medulloblastoma often receive aggressive treatment (surgery, radiation and chemotherapy), but many still die of their disease, and survivors suffer long-term effects from therapy. Safer and more effective therapies are desperately needed.

Wechsler-Reya recently combined forces with Michael Jackson, PhD, senior vice president of Drug Discovery and Development, to find a drug(s) that would inhibit the growth of Group 3 medulloblastoma, the most aggressive form of the disease. Using high-throughput screening technology, they identified a compound that reduces levels of a protein called MYC, which is found at exceptionally high levels in Group 3 medulloblastoma, as well as in cancers of the blood, breast, lung and prostate.

“An effective MYC inhibitor could have a major impact on the survival and quality of life of patients with medulloblastoma,” says Wechsler-Reya. “We identified a compound that reduces levels of MYC in medulloblastoma cells, but now we need to learn how it works to optimize it as an anti-cancer drug and advance studies toward the clinic.

“Historically, pharmaceutical companies and funding agencies have under-invested in childhood cancers, and the majority of drugs currently used to treat these cancers were originally developed for adult cancer,” adds Wechsler-Reya. “We believe that effective drugs for pediatric brain tumors must be developed—and this award from the Foundation will help us achieve this goal.”

“We are profoundly grateful to Conrad for his generosity over the years,” says President Kristiina Vuori, MD, PhD “He has a special legacy at our Institute, which was renamed Sanford Burnham Prebys in 2015 to honor him. We are now thankful to his Foundation for including us in their inaugural grant cycle, and for supporting the critical work we do to benefit children and others suffering from cancer.”

The Conrad Prebys Foundation allocated $78 million in its inaugural grant cycle to fund 121 projects. The awards reflect areas of personal interest to Conrad Prebys—including visual and performing arts, higher education, health care, youth development and animal conservation.

Sanford Burnham Prebys joins a long list of recipients, which included other prominent San Diego institutions such as Rady Children’s Hospital, KPBS, San Diego State University, Scripps Research, Museum of Contemporary Art San Diego and the La Jolla Music Society.

Institute News

Mining “junk DNA” reveals a new way to kill cancer cells

AuthorMonica May
Date

February 11, 2021

DNA strand and Cancer Cell Oncology Research Concept 3D rendering, abstract background

Scientists unearth a previously unknown vulnerability for cancer and a promising drug candidate that leverages the approach

Scientists at Sanford Burnham Prebys have uncovered a drug candidate, called F5446, that exposes ancient viruses buried in “junk DNA” to selectively kill cancer cells. Published in the journal Cell, the proof-of-concept study reveals a previously unknown Achilles’ heel for cancer that could lead to treatments for deadly breast, brain, colon and lung cancers.

“We found within ‘junk DNA’ a mechanism to stimulate an immune response to cancer cells, while also causing tumor-specific DNA damage and cell death,” says Charles Spruck, PhD, assistant professor in the National Cancer Institute (NCI)-designated Cancer Center and senior author of the study. “This is a very new field of research, with only a handful of papers published, but this has the potential to be a game-changer in terms of how we treat cancer.”

Since the human genome was fully sequenced in 2003, scientists have learned that our DNA is filled with some very strange stuff—including mysterious, noncoding regions dubbed “junk DNA.” These regions are silenced for a reason—they contain the genomes of ancient viruses and other destabilizing elements. An emerging area of cancer research called “viral mimicry” aims to activate these noncoding regions and expose the ancient viruses to make it appear that a cancer cell is infected. The hypothesis is that the immune system will then be triggered to destroy the tumor.

A one-two punch to cancer

In the study, Spruck and his team set out to find the molecular machinery that silences “junk DNA” in cancer cells. Using sophisticated molecular biology techniques, they found that a protein called FBXO44 is key to this process. Blocking this protein caused the noncoding sections of DNA to unwind—but not for long.

“When we revealed noncoding regions, which aren’t meant to be expressed, this caused DNA breakage. This told the cell that something is deeply wrong, and it committed suicide,” explains Spruck. “At the same time, the DNA of the ancient virus was exposed, so the immune system was recruited to the area and caused more cell death. So, we really delivered a one-two punch to cancer.”

The scientists then showed that a drug that targets the FBXO44 pathway, called F5446, shrank tumors in mice with breast cancer. The drug also improved the survival of mice with breast cancer that were resistant to anti-PD-1 treatment, an immunotherapy that is highly effective but often stops working over time. Additional studies in cells grown in a lab dish showed that the drug stops the growth of other tumors, including brain, colon and lung cancers.

The scientists also conducted many experiments to show that this silencing mechanism only occurs in cancer cells, not regular cells. Analysis of patient tumor databases confirmed that FBXO44 is overproduced in many cancers and correlated with worse outcomes—further indicating that a drug that inhibits this protein would be beneficial.

Moving the research toward people

As a next step, the scientists are working with the Conrad Prebys Center for Chemical Genomics to design an FBXO44 pathway-inhibiting drug that is more potent and selective than F5446. This state-of-the-art drug discovery facility is located at Sanford Burnham Prebys.

“Now that we have a compound that works, medicinal chemists can make modifications to the drug so we have a greater chance of success when we test it in people,” says Jia Zack Shen, PhD, staff scientist at Sanford Burnham Prebys and co-first author of the study. “Our greatest hope is that this approach will be a safe and effective pan-cancer drug, which maybe one day could even replace toxic chemotherapy.”

 

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

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

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.