Friday, June 15, 2018

By Roxanna Van Norman 

Most people have never heard of Diamond-Blackfan anemia (DBA), a rare disorder of the bone marrow caused by a genetic mutation that results in a severe anemia during early childhood years. That’s why Stanford pediatric hematologist/oncologist, Anupama Narla, MD, is dedicating her research to learning more about this disease and seeking others who share her enthusiasm – and the challenges - in translating laboratory findings to better care for these patients.

Dr. Narla leads a team of researchers in the Division of Hematology and Oncology at the Stanford University School of Medicine. Her lab focuses on bone marrow failure syndromes like DBA and other blood cell development abnormalities in pediatric patients. She is also a physician at the Lucile Packard Children's Hospital Stanford and has the privilege of caring for young patients and their families dealing with unusual bone marrow failure findings.

She explains that while this rare disorder only affects a small group of children - about 750 kids diagnosed DBA in the country - to them, it’s the most important thing in their lives. “I don't think I'm going change the world, but I will have made a difference for this group of patients and their families,” she says.

Dr. Narla received her medical training at several leading pediatric hospitals, including the medical school at the Children’s Hospital of Philadelphia, residency at the University of California San Francisco, and a fellowship in hematology/oncology at the Boston Children’s Hospital. She did her research with Benjamin Ebert, MD, PhD in the division of hematology at Brigham and Women’s Hospital who encouraged her to pursue a career in non-malignant hematology and bone marrow failure.

“I was inspired by the work being done and how the field combines the continuity of primary care with the fascinating pathophysiology of hematology and oncology, all in a rigorous academic environment,” says Dr. Narla, who then became committed to a career as a translational physician-scientist in hematology. It became apparent to her there was a critical need for research in this area in order to advance better care for patients.

After spending seven years in Boston, she was recruited to Stanford University in 2013 as an assistant professor in the Division of Hematology and Oncology at the School of Medicine. She is currently the PI or co-PI on several clinical trials including one studying the effects of a new drug to treat patients with DBA.

When I go to a conference, I'm going to a hematology conference or a pediatric bone marrow failure conference, and we all understand each other's language...[...]..When you're working with rare diseases, it's hard to sell it to big audiences.

Dr. Narla points out that the transition to becoming an independent physician-scientist has been somewhat challenging for her, especially for someone who’s studying a rare disorder. There is a sense of isolation when you are siloed in a niche area and as with any career in scientific research, she adds, it’s challenging to keep up with finding funding sources and establishing local scientific relationships.

“When I go to a conference, I'm going to a hematology conference or a pediatric bone marrow failure conference, and we all understand each other's language,” Dr. Narla explains. Communicating with others who are not familiar or compelled by the impact of uncommon diseases like DBA can be hard. She puts it simply, “When you're working with rare diseases, it's hard to sell it to big audiences.”

That perspective changed when Dr. Narla attended a week-long course at the Eureka Institute for Translational Medicine in Siracusa, Italy that provides a facilitated curriculum for her and other emerging leaders in biomedical research to share their passion and struggles with their work. “I thought it'd be great to meet other people with diverse backgrounds and learn how to explain why I'm passionate about my research,” she recalls. “I went in with the expectations that it was going to challenge me a bit, expose me to different people, and help me learn new techniques for communication.”

The Eureka Institute for Translational Medicine provides professional and training opportunities for mid-level career scientists in translational medicine and is one of the educational offerings provided by the Stanford Child Health Research Institute (CHRI) to its members. The CHRI sponsored five Stanford participants, including Dr. Narla, to attend this year’s 10^th annual Eureka certificate course program in Italy.

“The Eureka program brought together a group of people in their early or mid-training who were passionate about translational research - whether that's from the drug discovery side, the basic science side, or the clinical side,” she explains.  More importantly, it was chance for her to meet other people who were also in the same mid-career training as her and to discuss the difficulties with finding solutions for uncommon diseases with a supportive peer group.

The Eureka program brought together a group of people in their early or mid-training who were passionate about translational research - whether that's from the drug discovery side, the basic science side, or the clinical side.

It also helped that she received the Tashia and John Morgridge Faculty Scholar award from the CHRI prior to going to the Eureka program, which comes with a $500,000 funding support over five years for faculty whose research interests are focused in maternal or child health. “The opportunity to be recognized as a CHRI Faculty Scholar, as well to receive the support, has helped strengthen my confidence and commitment to my work,” she says. And with the addition of the Eureka program set in a beautiful city of Italy, surrounded by brilliant scientists and leading experts in translational medicine, she was “re-inspired” by the tremendous impact she was doing that could lead to patient benefits.  

Over seven days, the Eureka program delivered an intensive curriculum in translational medicine and related topics covering problem-solving strategies, team building approaches, and communications skills. She was surprised to find that many of the sessions that would’ve seemed less relevant to her turned out to be the best part of the program. Many of the activities, she describes, pushed them to engage with others through team-building tasks, role-playing presentations, and even recording yourself giving an elevator pitch about your research and getting feedback from the group.

“It gets you out of your comfort zone, it makes you think about why you're actually doing this, and what you want get out of your job, and what you want to tell other people about it,” Dr. Narla says. Excited by the new peer network developed through the program who share her enthusiasm for the work that they do, she hopes others will find their Eureka inspiration and come back to the community reinvigorated with confidence to advance their work in translational medicine.

Now having completed the course, she feels, “It certainly has already changed the way I approach certain things, how I carry myself, and how I present my science,” she expresses. “I am very excited to be a part of the Stanford community and am very grateful for the lessons and support I have received from the CHRI. I look forward to contributing to scientific discoveries, mentoring junior physician-scientists, and caring for patients.”

Roxanna Van Norman is the marketing manager for the Child Health Research Institute.