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Sanford Burnham Prebys and Salk co-organize third annual La Jolla Aging Meeting

AuthorMonica May
Date

April 11, 2019

Malene Hansen, PhD, and Peter Adams, PhD; and Salk’s Jan Karlseder, PhD

“Excellent presentation!”
“We should connect—we have more samples coming soon.” 
“Feel free to reach out, we’re looking to partner so I’d love to hear more about your research.”

These exchanges, overheard at the 3rd annual La Jolla Aging Meeting held on March 29, 2019, at the Salk Institute, illustrate the importance of uniting scientists focused on a common goal: In this case, uncovering the root causes of aging. 

As in previous years, the 2019 meeting was co-organized by Sanford Burnham Prebys’ Malene Hansen, PhD, and Peter Adams, PhD; and Salk’s Jan Karlseder, PhD A key goal of the event is to feature research by local scientists studying the molecular mechanisms of aging, while fostering connections and building relationships that advance new discoveries. More than 200 researchers, primarily from the San Diego area, attended the meeting. 

Aging is the main risk factor for many of the serious diseases our society faces today, including cancer, heart disease and Alzheimer’s. As the U.S. population grows older due to the natural aging of the Baby-Boomer generation, the need to understand the underlying causes of aging becomes more urgent. The number of Americans who are age 65 or older is projected to double from nearly 48 million in 2015 to more than 90 million in 2060, according to the United States Census Bureau. 

The diversity of presentations given—from the role of supportive brain cells called astrocytes and cellular recycling (autophagy) to long-lived proteins in mitochondria (the cell’s power generator)—reflects the complexity of the field. A poster session was also held during the symposium, providing an opportunity for up-and-coming scientists to share their recent data. 

La Jolla Aging Meeting tweet

For scientists new to the area, the meeting provided a key opportunity to build relationships.

“It’s unlikely there will be any single factor responsible for aging,” says Matt Kaeberlein, PhD, professor at the University of Washington and the symposium’s keynote speaker. “I am encouraged to see so many people interested in diverse aspects of aging biology at this symposium. By advancing each of these distinct research areas, and working together, we will make progress in understanding the underlying aging process.”

The full list of speakers follows. Make sure to save the date for next year’s meeting, which will be held on March 27, 2020. 

  • Matt Kaeberlein, PhD, University of Washington (keynote) – New insights into mechanisms by which mTOR modulates metabolism, mitochondrial disease and aging
  • Isabel Salas, PhD, Allen lab, Salk – Astrocytes in aging and Alzheimer’s disease
  • David Sala Cano, PhD, Sacco lab, Sanford Burnham Prebys – The Stat3-Fam3a axis regulates skeletal muscle regenerative potential
  • Tina Wang, Ideker lab, University of California, San Diego – A conserved epigenetic progression aligns dog and human age
  • Rigo Cintron-Colon, Conti lab, Scripps Research – Identifying the molecules that regulate temperature during calorie restriction
  • Nan Hao, PhD, University of California, San Diego – Programmed fate bifurcation during cellular aging
  • Shefali Krishna, PhD, Hetzer lab, Salk – Long-lived proteins in the mitochondria and their role in aging
  • Sal Loguercio, PhD, Balch lab, Scripps Research – Tracking aging with spatial profiling
  • Alva Sainz, Shadel lab, Salk – Cytoplasmic mtDNA-mediated inflammatory signaling in cellular aging
  • Anthony Molina, PhD, University of California, San Diego – Mitochondrial bioenergetics and healthy aging: Advancing precision healthcare for older adults
  • Alice Chen, Cravatt lab, Scripps Research – Pharmacological convergence reveals a lipid pathway that regulates C. elegans lifespan
  • Robert Radford, PhD, Karlseder lab, Salk – TIN2: Communicating telomere status to mitochondria in aging
  • Jose Nieto-Torres, PhD, Hansen lab, Sanford Burnham Prebys – Regulating cellular recycling: role of LC3B phosphorylation in vesicle transport

Prizes for the best poster presentations were awarded to the following scientists: 

  • Hsin-Kai Liao, PhD, Juan Carlos Izpisua Belmonte’s lab, Salk  
  • Yongzhi Yang, PhD, Malene Hansen’s lab, Sanford Burnham Prebys
  • Tai Chalamarit, Sandra Encalada’s lab, Scripps Research 

Thank you to our generous sponsor, the Glenn Foundation for Medical Research, and to NanoString for donating the prizes received by the poster presenters.  

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

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Inspiring future scientists at the STEM EXPO

AuthorMonica May
Date

March 25, 2019

STEM EXPO 2019-Sanford Burnham Prebys

Armed with wiggly worms and striped zebrafish, on Saturday, March 2, more than 20 volunteers from Sanford Burnham Prebys helped kids and their families learn about the power of DNA at the San Diego Festival of Science & Engineering’s EXPO Day. 

One of the largest STEM (Science, Technology, Engineering and Math) festivals in the U.S., this year’s event featured more than 130 interactive exhibits designed to ignite a passion for science in K–12 students. Despite an uncharacteristically rainy morning, an estimated 17,000 people attended. 

For Joseph Lancman, PhD, a postdoctoral researcher at our Institute who was the first in his family to graduate from college, the festival was an opportunity to provide children with the experience he wishes he’d had as a kid.

“Growing up, I knew I was interested in human health, but I had no idea that research was an option,” Lancman says. “Like many kids, I thought I wanted to be a doctor. But in college, I quickly learned that I wanted to know more. I wanted to know what causes disease and how scientists go about finding cures.” 

STEM EXPO 2019 Joseph Lancman and son

Dr. Lancman and his son

At our booth, postdoctoral researchers, graduate students and staff helped children don paper lab coats and explore DNA-themed activities. 

Children were able to see live worms with DNA mutations that affect their movement, courtesy of the lab of Malene Hansen, PhD, professor in the Development, Aging and Regeneration Program. Compared to normal worms, some mutant worms moved mindlessly in circles, and others remained relatively immobile—illustrating how changes in a DNA sequence can dramatically affect life.

At the adjacent station, provided by the lab of Duc Dong, PhD, assistant professor in the Human Genetics Program, children squinted through microscopes and peered into fish tanks to observe how DNA changes can dramatically affect the heartbeat of zebra fish—one of the most powerful model organisms used to study vertebrate biology. 

Lancman, who works in Dong’s lab, took care to explain the exhibit in child-friendly language (he credits his four-year-old son for helping him develop this skill). 

“I want kids to know that science is like a puzzle,” he explains. “It takes time to put all the pieces together, but when you’re done, you can see the big picture—and that big picture can lead to improving human health.”

Institute News

Families find hope at our 10th Annual Rare Disease Day Symposium

AuthorMonica May
Date

March 25, 2019

Rare Disease Day 2019-Sanford Burnham Prebys

The unofficial theme of Sanford Burnham Prebys’ 10th annual Rare Disease Day symposium can be summarized in one word: hope. 

This year’s event focused on rare bone disorders and bone cancers, with a special emphasis on a condition called multiple hereditary exostoses (MHE) that causes numerous bone tumors in children. Until now, children with MHE had only one treatment option: repeated surgeries to remove these tumors. 

Now, the first medicine, called palovarotene, that may be able to slow or halt this bone growth is being tested in a clinical trial—potentially saving these children from a lifetime of painful surgeries. Yu Yamaguchi, MD, PhD, symposium chair and professor in Sanford Burnham Prebys’ Human Genetics program, participated in the key research needed in order for the clinical trial to begin.

“For the first time, a diagnosis doesn’t feel like a life sentence,” says Greta Falkner, who attended the symposium with her 8-year-old son, Jackson Falkner. Jackson has MHE and has undergone 13 surgeries to date. “Today we have hope for a cure.”

The launch of a study evaluating palovarotene as an MHE treatment is the result of decades of hard work and collaboration between scientists, clinicians, Clementia Pharmaceuticals (now Ipsen) and the MHE Research Foundation—a patient advocacy and support group. 

The symposium featured an all-star lineup of distinguished speakers in the field of skeletal biology and MHE research, including keynote speaker Henry Kronenberg, MD, of Massachusetts General Hospital and Maurizio Pacifici, PhD, of Children’s Hospital of Philadelphia, whose research formed the scientific rationale for the use of the drug in MHE. The event was sponsored by the MHE Research Foundation and Clementia Pharmaceuticals. 

“We are so grateful that Sanford Burnham Prebys holds this Rare Disease Day symposium to bring together all the important players in rare disease drug discovery: scientists, doctors, families and drug companies. It is literally ‘bench to bedside’ at one event,” says Sarah Ziegler, who co-founded the MHE Research Foundation with Craig and Susan Eaton after Sarah’s son was diagnosed with MHE in 1993 (when little to no information was available on the disease). “Foundations like ours rely on the research of scientists like Drs. Yamaguchi and Pacifici to find new medicines for patients—and the arrival of the first potential treatment for MHE is the perfect example of the power of this research.” 

Multiple parents and children with MHE, some of whom are enrolled in the clinical trial, attended the event. For most of these children, this was the first time they’d met another person with their condition. 
 

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

Institute News

5 takeaways from Insights: Heart Disease

AuthorMonica May
Date

February 15, 2019

SBP Insights:Heart Disease

It’s easy to forget about the fist-sized organ in our chest. But the heart is arguably the most important muscle in the body. We can’t live without it, after all. 

To help educate the public about heart health and share the latest scientific advances, this month Sanford Burnham Prebys Medical Discovery Institute (SBP) invited the San Diego community to a free panel discussion focused on the heart. 

More than 70 community members attended the event, whose speakers included cardiologist Anthony N. DeMaria, MD; Jack White, chair in Cardiology, professor of Medicine, founding director, Sulpizio Cardiovascular Center at UC San Diego Health; Donna Marie Robinson, an individual living with heart failure; and heart researcher Karen Ocorr, PhD, assistant professor, Development, Aging and Regeneration Program at SBP. Jennifer Sobotka, executive director at the American Heart Association San Diego, moderated the discussion.

In a special introduction provided by Rolf Bodmer, PhD, director and professor in the Development, Aging and Regeneration Program at SBP, he explained that his heart research uses model organisms such as the fruit fly. He quipped, “Which some of you didn’t even know had a heart.” 

The ensuing discussion was robust and insightful. Below are five important takeaways: 

  1. Heart disease is the number-one killer of Americans. Nearly half of American adults have some form of heart or blood vessel disease.
  2. Obesity is an epidemic in America. In the 1960s, approximately 13 percent of American adults were obese, according to the Centers for Disease Control (CDC). Today, that number has tripled to nearly 40 percent. DeMaria illustrated this point with a colored map showing obesity’s prevalence during each decade, which drew gasps from the crowd. 
  3. Know your numbers. Donna Marie was healthy and fit, so she didn’t think that a fainting episode could have been heart disease. “My cardiologist saved my life,” she said. Now, she encourages everyone to “know your numbers, including your cholesterol level and your blood pressure.” 
  4. Rethink swatting that pesky fruit fly. We share 80 percent of disease-causing genes with the tiny insect, including ion channels that keep the heart pumping. For this reason scientists are studying fruit-fly hearts in an effort to learn about the many mysteries of the heart, such as how the rhythm disorder atrial fibrillation (AFib) arises. 
  5. Consider moving to Italy. Just about everyone wants to know which science-backed diet to follow for optimal health. DeMaria explained that the most robust data supports eating a Mediterranean diet rich in fruits, vegetables and olive oil.

Read the La Jolla Light’s coverage of the event. 

Institute News

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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SBP scientist presents at Fleet Science Center to help the public better understand precision medicine

AuthorMonica May
Date

January 29, 2019

Chess pieces-SBP

From how much coffee we can tolerate to the amount of weight we can lift, our bodies differ from one another in myriad ways. But when it comes to medicine, historically every patient with the same disease or condition has received the same treatment—even though individual responses vary wildly. 

Now, technological advances are enabling medicine to move from “one size fits all” to tailored treatments based upon one’s specific genes. This approach is called precision medicine. 

This December, under the dome of the Fleet Science Center’s IMAX theater, Jessica Rusert, PhD, a postdoctoral researcher in the lab of Robert Wechsler-Reya, PhD, explained the promise and potential of precision medicine to a crowd of nearly 100 people as part of the museum’s Senior Monday presentation series (if interested, check out the 2019 schedule). 

We caught up with Rusert to learn more about the goals of precision medicine and her work in the Wechsler-Reya lab. 

Before the advent of precision medicine, how did doctors typically treat patients? 
In the past, all patients who had the same disease—say, breast cancer—received the same treatment, which was most likely surgery followed by chemotherapy and radiation. Your treatment might change if you have more advanced disease—in which case the approach would be more aggressive. But typically treatment was based upon physical criteria: the location of the tumor, your symptoms or how the tumor looks under a microscope. That’s not to say this is a bad approach; it was simply the only information doctors and scientists had to work from. 

What catalyzed the movement from one size fits all to personalized treatment?
The invention of DNA sequencing has revolutionized personalized medicine. As the cost of sequencing decreases and the use of the technology swells, we will glean even more information from the genome, and personalized medicine will expand further to new areas. 

What is the ultimate goal of precision medicine? 
Precision medicine aims to treat the right patient, with the right drug, at the right time.

How far away is precision medicine from this goal? 
We are making inroads, but it is still early days for precision medicine. Currently, we are making the most progress in cancer. But despite these advances, the vast majority of cancers—including the pediatric brain cancer our lab studies—are treated with surgery, chemotherapy and radiation. 

As scientists learn more about the underlying cause of disease(s), precision medicine will expand to new cancers and new disease areas. These advances are happening now, and for some cancers, the outlook is already much better than it used to be. Perhaps one day we will have personalized treatments for schizophrenia and autism. The approach is mostly limited by how much we know about a disease. 

What is an example of a precision medicine? 
The breast cancer treatment Herceptin® is a great example of a precision medicine. 

Cells in our body use molecular antennae called receptors to sense and respond to their environment. One receptor, called HER2, controls cellular growth, and is involved in the development of breast cancer. 

Herceptin works by blocking the HER2 receptor. Then, the receptor can’t tell cells to grow, and tumor growth stops. 

However, this medicine only works if your tumor cells have this receptor. HER2-positive breast cancer means your cells have this receptor. HER2-negative breast cancer means you do not have the receptor, and thus Herceptin wouldn’t work for you. So people with breast cancer are tested to ensure they have the HER2 receptor before receiving Herceptin. 

Herceptin has saved thousands of lives. It is a true testament to the power of precision medicine. 

What was a popular question from the audience?
A lot of people wondered if there’s a way to prevent cancer. This is understandable—cancer is scary, and we want to do all we can to stop it. But it’s a difficult question to answer. The science isn’t there yet. We are only just now starting to understand how to help people who have acute disease. We may understand how to prevent cancer one day—but that will take decades, not years. 

How do you use precision medicine in your work?
In the Wechsler-Reya lab, we are working to find personalized treatments for children with brain cancer. We are studying the most common malignant pediatric brain cancer, called medulloblastoma. 

Children with medulloblastoma undergo surgery to remove the tumor and then undergo chemotherapy and radiation treatment. This treatment is hard for adults to go through—and even more devastating for a developing child. The treatment leaves long-term effects, including cognitive impairment and increased risk of other cancers due to the DNA damage caused. A treatment that reduces or eliminates these side effects is urgently needed. 

Scientists are learning that medulloblastoma is not one cancer, but actually four clearly defined subgroups. We are working to better understand these subgroups so we can develop targeted treatments that are customized to each cancer type (read the lab’s recent discovery). 

Where is precision medicine heading in the future? 
Right now, most precision medicine focuses on our DNA, but there are many other ways we differ from one another. But increasingly, scientists are working toward precision medicine that also takes into account RNA, proteins, our metabolism, the epigenome (molecular tags on DNA) and more.

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

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World-renowned scientists speak at SBP’s symposium on a booming research area: epigenetics

AuthorMonica May
Date

November 28, 2018

Epigenetics Symposium-Lorenzo Puri, Ani Deshpande, Peter Adams

DNA contains more information than scientists previously thought. A booming field of research called epigenetics is uncovering DNA alterations that affect whether genes are turned on or off—without changing a single base pair. 

These epigenetic changes may be physical, such as winding or unwinding DNA to change its accessibility—akin to opening or closing a door. Other changes can be chemical, such as adding molecular tags that turn genes on or off—like a highlighter calling out special text to a reader. 

On October 29, 2018, more than 300 scientists gathered at SBP’s 39th Annual Symposium to hear the latest connections between epigenetics and development, aging, and diseases such as cancer. Researchers are particularly interested in this field because modifying epigenetic changes could be easier than editing the body’s underlying genetic code—and advances are leading to new medicines. 

Organized by SBP professors Peter Adams, PhD; Ani Deshpande, PhD; and Lorenzo Puri, MD, PhD, the event’s speakers hailed from Harvard Medical School, Massachusetts Institute of Technology, Stanford University and other world-renowned institutions. 

Tweet by scientists praising the symposium ​
  USC assistant professor Bérénice A. Benayoun, PhD,
  praised the symposium on Twitter

After a welcome by SBP’s President Kristiina Vuori, MD, PhD, talks commenced on topics ranging from the role of epigenetics in prostate cancer, development of the heart and aging. Unpublished work was frequently presented, meaning that attendees received the most up-to-date results available. 

A full list of speakers is listed below. To read more insights, follow #SBPsymposium on Twitter. And be sure to look out for next year’s symposium, which will focus on neurodegenerative diseases.

  • Anne Brunet, Stanford University – Epigenetic and metabolic regulation of aging 
  • Chris Glass, University of California, San Diego – Nature and nurture of tissue resident macrophages 
  • Cigall Kadoch, Dana-Farber Cancer Institute and Harvard Medical School – Structure and function of mammalian SWI/SNF complexes in human cancer 
  • Manolis Kellis, Massachusetts Institute of Technology – From genomics to therapeutics: dissection and manipulation of human disease circuitry at single-cell resolution 
  • Peter Lewis, Wisconsin Institute for Discovery – Mechanistic dissection of oncogenic histone mutations 
  • Barbara Meyer, University of California, Berkeley – Dynamic control of X-chromosome topology and gene expression during development via chromatin modification and condensing 
  • Raul Mostoslavsky, Harvard Medical School – Linking epigenetics, metabolism and cancer: new clues from SIRT6
  • Bing Ren, University of California, San Diego – Remodeling of chromatin organization during human cardiomyocyte differentiation 
  • Peggy Farnham, University of Southern California – CRISPR-mediated deletion of prostate cancer risk elements identifies repressive chromatin loops 
Institute News

2018 Fishman Award Ceremony honors postdoctoral scholars

AuthorSusan Gammon
Date

September 26, 2018

Fishman Fund Awards 2018

More than 100 supporters of SBP came out to celebrate this year’s Fishman Award recipients: Usue Etxaniz Irigoien, PhD, Koen Galenkamp, PhD, Laura Martin-Sancho, PhD, and Ee Phie Tan, PhD The annual awards ceremony, held September 20 at the Sanford Consortium, recognizes postdocs who have made extraordinary efforts and encourages their passion for careers in science.

The evening began with a warm welcome from founder Reena Horowitz, who described how she began the Fishman Fund with the late Mary Bradley (her dear friend) to honor Dr. William and Lillian Fishman. The Fishmans were the founders of SBP and firmly believed in helping young postdoctoral scientists become great principal investigators—and these awards help boost young researchers to meaningful careers in science.

Jeanne Jones, the Fishman Fund’s co-founder designee, shared that since the fund’s inception, 64 recipients have been awarded. The career development awards—bestowed annually to three postdocs—provide a generous $10,000 stipend to allow them to attend workshops and travel to national and international conferences to learn the latest developments in their research fields. Additionally, a Fishman Fund Fellowship Award, which provides salary support for two years in addition to benefits and a career-development stipend, was awarded this year for the second time in SBP history.

The keynote speaker was Aman Mann, PhD, who won the Fishman Award in 2011. Today, Mann is a research assistant professor at SBP and an entrepreneur and founder of AivoCode, a neuroscience company that licenses technology originally developed at SBP. Mann reflected on how the award gave him added confidence and resources to pursue his career goals—and he encouraged this year’s recipients to similarly follow their dreams.

If you are interested in donating to the Fishman Fund, click here.

The 2018 Fishman Fund Fellowship winner, Usue Etxaniz Irigoien, PhD, was presented with her award by Reena Horowitz. Irigoien, originally from the Basque region of Spain, is the first in her family to pursue a graduate degree in science. She is currently a postdoctoral researcher in the laboratory of Lorenzo Puri, PhD, and is studying the communication between nerves and muscles. Irigoien’s research is providing insights that may lead to improved therapies for neuromuscular diseases such as ALS. In the future, she hopes to become a principal investigator of her own lab.

The Fishman Fund Awardees:

Koen Galenkamp, PhD, was presented with his award by Andrew Viterbi, a distinguished scientist and former SBP trustee. Galenkamp works in the laboratory of Cosimo Commisso, PhD, and specializes in seeking ways to starve pancreatic tumors of the food they need to survive and grow. Originally from Amsterdam, Galenkamp thanked his colleagues and wife—who is also a postdoc in San Diego—and shared his plans to use the funds to attend a comprehensive pancreatic cancer conference at Cold Spring Harbor Laboratories.

Laura Martin-Sancho, PhD, a postdoc in the lab of Sumit Chanda, PhD, received her Fishman Fund Award from Armi Williams, a Fishman Fund Board member and longtime SBP supporter. Martin-Sancho is working to develop antiviral medicines that will combat infectious diseases such as influenza, dengue, and West Nile and Zika viruses. Martin-Sancho’s career goal is to be an independent investigator in an academic organization. She will use her stipend to attend a Keystone RNA virus conference in Dublin, Ireland.

Ee Phie Tan, PhD, originally from Malaysia, joined SBP in 2016 to work in the lab of Malene Hansen, PhD Tan is studying autophagy—a cell process that tidies up cell debris and recycles parts to maintain health. Defects in autophagy are linked to many aging-related diseases such as Alzheimer’s and Parkinson’s disease, and even cancer. Tan received her award from Reena Horowitz and will use the funds to attend a Gordon Research Conference in Texas and a Keystone Symposium in Santa Fe, New Mexico. 
 

Institute News

Duc Dong honored at Alagille Syndrome Alliance Gala

AuthorSusan Gammon
Date

July 10, 2018

Duc dong

Associate Professor Duc Dong, PhD, was the guest of honor at the Gala of Dreams, the inaugural fundraiser for the Alagille Syndrome Alliance held June 30 at the San Diego Marriott Del Mar. Dong is a trailblazer in the field of Alagille syndrome research—he is working toward a cure for the extremely rare genetic condition that affects approximately one in 30,000 births.

Babies born with Alagille syndrome have too few bile ducts—which are essential for the transport of waste out of the liver. This causes toxins build up in the liver and throughout the body, leading to constant severe itching, and more critically, liver damage and failure. Alagille syndrome patients also have many other life-threatening developmental defects in other parts of their bodies, including the heart, kidneys, vertebrae, and blood vessels. There is no cure for this debilitating disease, and up to 50 percent of patients eventually need a liver transplant, often during childhood.

Dong and his team have been studying JAGGED1, the gene implicated in Alagille syndrome. Taking advantage of an unusual animal model, the zebrafish, he has been able to uncover a novel genetic mechanism for the disease—opening new potential therapeutic avenues. Further, his team has surprisingly discovered that the bile ducts lost can be regenerated after turning the affected gene back “on.”

“The implication is that these developmental defects in Alagille syndrome patients could potentially be reversible and therefore curable,” says Dong. “We will now start screening for drugs that may be used to restore the function of this genetic pathway and hopefully allow for these lost bile ducts to regenerate. We will continue to challenge the science of Alagille syndrome to move closer to a cure.”

The theme of the event, “The Dawn of a Dream,” signified new advances in Alagille syndrome research and the anniversary of the organization’s 25th year in existence. The evening gave advocates, families, doctors and pharma representatives an opportunity to interact in a fun, casual setting and participate in a silent auction to raise money for research. Dong’s lab was presented with the Champion of Alagille Syndrome Award and funds raised by the Alliance through crowd sourcing.

Institute News

Highlights from the 2018 Faculty Retreat

AuthorSusan Gammon
Date

June 4, 2018

Faculty Retreat

SBP’s annual Faculty Retreat was held May 30–31 in beautiful Carlsbad, California.

This year’s theme was “Fundamental Cellular Processes in Health and Disease,” and featured 20 speakers from SBP areas of expertise:

·         Development and Aging — Malene Hansen, PhD, Lorenzo Puri, MD, PhD, Karen Ocorr, PhD, Alexey Terskikh, PhD, and Rolf Bodmer, PhD

·         Immunity and Inflammation — Rob Weschler-Reya, PhD, Linda Bradley, PhD, Scott Peterson, PhD, and Jeff Smith, PhD

·         Technology Platforms — Dorit Hanein, PhD, Sumit Chanda, PhD, and Niels Volkmann, PhD

·         Drug and Disease Targets — Michael Jackson, PhD, Garth Powis, PhD, Wanda Reynolds, PhD, Elena Pasquale, PhD, and Matt Peroski, PhD

·         Share Resources Update — Craig Hauser, PhD

Special congratulations to Duc Dong, PhD, and Crystal Zhao, PhD, both of whom were officially promoted to associate professors.

Faculty promotion​
Pictured (left to right): Duc Dong, PhD, Elena Pasquale, PhD and Crystal Zhao PhD

Dong, in the Human Genetics Program, is advancing research in cell/organ replacement therapy that may ultimately benefit patients with diabetes and Alagille syndrome—a genetic disorder that can affect the liver, heart and other parts of the body.

Zhao, in the Tumor Initiation and Maintenance Program, is studying the regulation of neural stem cells—research that may lead to therapies for neurological diseases, including Alzheimer’s disease, Parkinson’s disease, and mental health disorders that affect cognitive abilities.

A highlight of the annual retreat is the presentation of the “WOW” award, which stands for Wonderful Original Work. This year, the WOW was given to Robert Wechsler-Reya, PhD, who won for his presentation titled “Overcoming Immune Evasion in Pediatric Brain Tumors.”

Wechsler-Reya’s research goal is to advance immunotherapy for medulloblastoma tumors—the most common malignant brain tumor in children. By increasing certain cell surface molecules called MHC on these cancer cells, he believes he can improve the ability of T cells to recognize the tumors and kill them.

Wechsler-Reya is a professor in the Tumor Initiation and Maintenance Program at SBP and is also the program director for the Joseph Clayes III Research Center for Neuro-Oncology and Genomics at Rady Children’s Institute for Genomic Medicine.

Cosimo Commisso, PhD, and Crystal Zhao, PhD, served as faculty organizers.

And many thanks to Wendy Lyon and Nelson, the research administrative specialists who organized the event.