Sanford Children's Health Research Center Archives - Sanford Burnham Prebys
Institute News

Where science meets patients: Sanford Children’s Research Center hosts inaugural symposium

AuthorMiles Martin
Date

May 10, 2023

The event celebrated 16 years of progress at the Center and connected scientists with the people most impacted by their work.

The Sanford Children’s Health Research Center at Sanford Burnham Prebys recently hosted its first-ever Children’s Health Research Symposium, which brought scientists and families together to learn about the latest research tackling childhood diseases.

“We’re all here because we want to improve the health of children,” said President and CEO David A. Brenner, MD, during his opening comments. “But this event also shows the amazing amount of collaboration and collegiality across San Diego, because we have all types of people together from different backgrounds who want to develop therapies and cures for children affected by disease.”

The Sanford Children’s Health Research Center was established in 2008 with the help of a generous gift from Institute namesake T. Denny Sanford. Since then, the Center has been a world leader in children’s health research.

Professor Hudson Freeze with the Omler family

“T. Denny Sanford made an investment in children’s health 15 years ago, and I think that investment has paid off pretty well so far,” said Center director Hudson Freeze, PhD, in his introduction to the first scientific session. Freeze is among the world’s leading experts on congenital disorders of glycosylation (CDG), a rare group of genetic disorders that can cause serious, sometimes fatal, malfunctions of different organs and systems in the body.

“We’ve published over 600 scientific papers, and about half of those are translational studies, which means they’re helping turn scientific discoveries into real treatments for patients,” adds Freeze.

The day included presentations from researchers at Sanford Burnham Prebys, as well as from other research organizations studying childhood diseases. However, the highlight of the event was the afternoon reception, in which scientists had the opportunity to mingle and share a meal with families affected by rare childhood diseases.

Professor José Luis Millán (center) with the Fischer family (left) and the Britt family (right)

Each researcher briefly introduced the family affected by the illness the scientist studies. This list included many longtime friends of the Institute, such as Damian Omler, who lives with a rare form of CDG; and Morgan Fischer, who was born with soft bone disease. Today, thanks to the help of a drug developed based on the research of Institute professor José Luis Millán, PhD, Morgan is a thriving teenager. This drug is also helping other children living with soft bone disease, including 10-year-old Aubrey Britt, who was in attendance with her family as well.

“Something so important that we keep as a tradition for scientific events at our Institute is to involve families that have been touched by the work of our faculty,” said Freeze. “They’re why we’re all here.”
 

The full list of talks included: 

Sanford Children’s Health Research Center

  • José Luis Millán, PhD “Developing therapeutics for soft bones and ectopic calcification disorders”
  • Duc Dong, PhD “From hope for few to drug for many—why rare is precious”
  • Evan Snyder, MD PhD “A clinical trial using human neural stem cells for neuroprotection in perinatal asphyxia, a major cause of cerebral palsy in kids”
  • Anne Bang, PhD “Drug screens of human-induced pluripotent stem cell (hiPSC) derived neuronal networks on multi-electrode arrays”
  • Pamela Itkin-Ansari, PhD “Proinsulin misfolding in diabetes”
  • Yu Yamaguchi, MD PhD “Multiple hereditary exostoses—from genetics to potential drug targets”
  • Hudson Freeze, PhD “Fucose therapy: Revising bedrock biochemistry”

Sanford Health

  • David Pearce, PhD “From rare diseases to type-1 diabetes: Research that impacts children at Sanford Health”

Frontiers in Congenital Disorders of Glycosylation Consortium

  • Eva Morava, MD, PhD “Clinical trials in Glyco-land”
  • Ethan Perlstein, PhD “Precision drug repurposing: Patient avatar to pioneer study to Phase 3 trial”

UC San Diego

  • Lars Bode, PhD “Human milk-based therapeutics and diagnostics to protect preterm babies from necrotizing enterocolitis”
  • Stephanie Cherqui, PhD “Hematopoietic stem cell gene therapy for cystinosis: Mechanism of action and clinical trial update”
Institute News

Former postdoc Suzanne Graham now leads business development at diagnostic company

AuthorJessica Moore
Date

July 8, 2016

Suzanne Graham, PhD, did postdoctoral research at SBP from 2004-2012, during which time she worked in two labs. With Fred Levine, MD, PhD, professor and director of the Sanford Children’s Health Research Center, she studied cell cycle control in pancreatic beta cells, and with Rolf Bodmer, PhD, professor and director of the Development, Aging, and Regeneration Program, she explored how metabolic syndrome affects heart function. She has served as senior manager of business development at Genection, which develops and sells genetic tests, in San Diego since June 2012.

What’s your job like— what do you do every day?

I’m responsible for managing relationships with clients (generally pharmaceutical companies)—making sure we’re getting the right data to them, answering their questions, and trying to get more business.

What drew you to business development?

I’m more sociable than the average R&D scientist—I wanted to be involved in lots of conversations, interfacing with all parts of the company.

What do you enjoy most about it and why? 

It’s exciting to help our clients find tools that help them meet their goals. For example, if a company is running a trial of a drug and they can tell it’s working for some patients but not others, I can help them either find a diagnostic test or work with our company to make one that helps them identify the likely responders.

Plus, since I’m working in industry, everything moves quickly. It’s really rewarding to see things through to completion.

What do you hope to accomplish in your career? 

Since I’m familiar with how genomics fits into pharmaceutical development, I’d love to move to a pharma company in a strategy role to help them expand into that area.

What did you gain from your postdoc experience at Sanford Burnham? 

My scientific training is instrumental in my work—it really helps me understand and define the questions our clients have, which is crucial for finding the right solution.

How did you find your first job after your postdoc? Was it challenging? 

I used a job placement company because it is VERY hard for postdocs to get into industry. They helped make my resume convey my skills in a way that speaks to biotech companies, understand what kind of jobs to look for, and create a strategy to connect with potential hirers.

What advice do you have for current postdocs looking for a job?

PhD scientists can fulfill a lot more roles than they might think. Almost any job related to biotech benefits from a deep understanding of science—even designing websites or doing social media for executives.

Institute News

A new way to generate insulin-producing cells in type 1 diabetes

Authorsgammon
Date

July 31, 2014

A new study by researchers at Sanford-Burnham Medical Research Institute (Sanford-Burnham) has found that a peptide called caerulein can convert existing cells in the pancreas into those cells destroyed in type 1 diabetesinsulin-producing beta cells. The study, published online July 31 in Cell Death and Disease, suggests a new approach to treating the estimated 3 million people in the U.S., and over 300 million worldwide, living with type 1 diabetes. Continue reading “A new way to generate insulin-producing cells in type 1 diabetes”