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A year in review: Our top 10 discoveries of 2019

AuthorMonica May
Date

December 4, 2019

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.
Institute News

West Coast meets East Coast: Dr. Wechsler-Reya makes special trip to thank students

AuthorMonica May
Date

July 17, 2019

In the summer of 2015, 5-year-old William Schultz began to experience odd and increasingly worrying symptoms, including frequent vomiting. After two emergency-room visits, doctors ushered his parents, Jim and Margaret Schultz, into a small office and gave an unimaginable diagnosis: William had a brain tumor, ultimately revealed as medulloblastoma—the most common malignant childhood brain cancer. Standard treatment proved ineffective. The tumor returned, and William later died due to treatment complications.

William’s parents quickly channeled their pain into action. Mr. and Mrs. Schultz launched William’s Warriors, a foundation that supports art therapy for all children battling cancer, both in and out of the hospital, and raises funds in support of a cure for pediatric brain cancer—the deadliest form of childhood cancer. Even with aggressive treatment, many children don’t survive, and those who do often suffer severe long-term side effects from the therapy. Half of the foundation’s fundraising goes to William’s Superhero Fund, which supports the work of Robert Wechsler-Reya, PhD, professor and director of the Tumor Initiation and Maintenance Program at Sanford Burnham Prebys, and program director of the Clayes Center for Neuro-Oncology and Genomics at the Rady Children’s Institute for Genomic Medicine.

On May 31, 2019, Wechsler-Reya traveled to New York’s Bay Shore High School, where Mrs. Schultz teaches art—and many teachers and students volunteer with the foundation—to provide an update on his research. As part of his goal to develop safer and more effective treatments for pediatric brain cancer, his lab explores potential personalized treatments based on a child’s specific tumor type, nanotechnology approaches that improve drug delivery and immunotherapy to train patients’ immune systems to eradicate the cancer.

The visit, organized by William’s Warriors volunteer and New York State Master Science Teacher Erin Garland, provided students with a unique opportunity to learn firsthand about the drug development process and how their funds directly impact medical research. During the trip, Wechsler-Reya addressed an assembly of students and teachers, attended a student-centered science symposium, met one-on-one with science students and participated in a question-and-answer session with STEAM teachers. Following his address, the Bay Shore Girls Basketball Team and Brother Sister Organization, presented Wechsler-Reya with a donation of funds they raised in support of his research.

“From the bottom of our hearts, we sincerely thank Dr. Wechsler-Reya for taking time out of his very busy schedule to make this visit. But, more importantly, we are grateful for his unwavering commitment to finding a cure for childhood brain cancer,” says Mrs. Schultz. “While William’s life couldn’t be saved, knowing that researchers are working to find a treatment that might help children like him means the absolute world to us.” 

Read William’s story

Donate to William’s Superhero Fund 

The Bay Shore Girls Basketball Team and Brother Sister Organization presented Wechsler-Reya with a donation of funds they raised in support of his research.

The Bay Shore Girls Basketball Team and Brother Sister Organization presented Wechsler-Reya with a donation of funds they raised in support of his research.

Institute News

New drug combination may lead to treatment for childhood brain cancer

AuthorJessica Moore
Date

March 14, 2016

Researchers at SBP have identified a new combination therapy for the most aggressive form of medulloblastoma, a fast growing type of pediatric brain cancer. The study, published  in Cancer Cell, is expected to lead to a clinical trial to confirm the benefits of the novel drug combination. Continue reading “New drug combination may lead to treatment for childhood brain cancer”

Institute News

Why new therapies are failing some kids with brain cancer

Authorjmoore
Date

February 1, 2016

The most common type of malignant brain cancer in children is medulloblastoma, a fast growing tumor located in the cerebellum—the lower, rear portion of the brain. Although the standard treatment, an aggressive combination of surgery, radiation, and high-dose chemotherapy, cures more than 70 percent of patients, many survivors are left with profound long-term side effects, including cognitive deficits and increased incidence of other cancers. Continue reading “Why new therapies are failing some kids with brain cancer”