For the first time since its establishment 20 years ago, the Life Raft Group (LRG)—which advocates for people with gastrointestinal stromal tumor (GIST)—has new leadership. Sara Rothschild, a 17-year employee of the charity, recently took the helm following the retirement of founder Norman Scherzer.
“I’ve watched Life Raft Group grow from a kitchen-table organization in a basement in a rundown building to what it is today,” Rothschild, now executive director, told Rare Disease Advisor from the group’s headquarters in Wayne, New Jersey. “It’s been wonderful to see all the fruits of our labor, to see how we can reach out to the global community, help patients through their rare disease diagnosis and treatments, and support their families along the way.”
On its website, LRG describes itself as “a love story written by and for patients and caregivers who fight the daily battle against GIST with incredible courage and dignity.”
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LRG, a nonprofit, traces its roots back to 2000, when patients in the early trials of imatinib (Gleevec®) began sharing their experiences online. Many had been misdiagnosed with leiomyosarcoma and were learning they had an entirely different cancer.
The group launched a newsletter, created a website, and formally incorporated in June 2002 as a nonprofit organization. At its head was Scherzer, whose wife, Anita, had GIST and eventually died of the disease.
“There is no doubt the LRG would not be where it is without the vision and leadership of Norman,” Rothschild said. “Norman’s public health background—coupled with his natural curiosity about a rare disease and passion to save his soulmate Anita’s life—shaped the LRG into the strong organization it is today.”
A Disease Encompassing Many Subtypes
Rothschild has a master’s degree in public health from the University of Michigan, Ann Arbor. After graduation, she worked at the US Centers for Disease Control & Prevention, where she assessed racial and ethnic approaches to community health on infant mortality intervention. Rothschild also spent 4 months in Israel, where she did a comparative analysis of health care rationing.
“I was very interested in global health issues and did a lot of research on the health care system while I was there,” she said. “That helped shape my passion for when I did end up working at the Life Raft Group about understanding health systems, culture, and access issues. I felt like that armed me with the compassion and understanding I needed to advocate for our patients.”
Between 3000 and 5000 Americans are diagnosed each year with GIST. Most types of GIST—classified as a soft tissue sarcoma—affect adults over 60 years old, though a few types specifically target children. Appearing anywhere in the digestive tract, GIST normally occurs in the stomach (40-70% of cases), the small intestine (20-40%), and the colon and rectum (5-15%), as well as in the esophagus (under 5%).
At diagnosis, between 80% and 85% of GISTs are localized, though as the disease progresses, it may travel to the liver or other distant organs. The term GIST itself was first used in 1983, though until 2000, the disease was often misdiagnosed as other types of soft-tissue sarcomas.
“I’ve seen patients suffer through a diagnosis and hop from treatment to treatment and hospitalizations. It’s important that the personalized touch does not get lost, because that’s how we started, as a group of patients who were in this together,” Rothschild said. “As our membership has grown into the thousands, we still do a good job about keeping in touch with patients and supporting them along the way.”
Based on its registry, LRG tracks about 2500 patients a year, though its membership extends beyond that; people with GIST consult the organization from all over the world. LRG operates on an annual budget of around $2 million.
Last year, its website attracted over 208,000 page views from 206 countries and territories. The group conducted 14 webinars and held remote “Days of Learning” sessions, providing hundreds of patients from Pittsburgh, Pennsylvania, to Phoenix, Arizona, with the latest information on GIST. And its “GIST Awareness Day” social media campaign—held annually on July 13—reached over 9000 people this year.
Rothschild’s Research Passion: SDH-Deficient GIST
The overall 5-year survival rate for GIST is currently 83%, based on those diagnosed between 2010 and 2016. This varies according to whether the cancer is localized (93%), has spread to nearby tissues (80%), or has metastasized (55%).
“It depends. If a patient has a tumor rupture, and they have a very aggressive form of the disease, it could be shorter. Sometimes those are things that we can’t help,” Rothschild said. “We have seen patients living over 10 years with the disease. That gives people hope—especially those who are newly diagnosed coming to us and never hearing about the disease, and not knowing what to expect.”
Formerly vice president of program services at LRG, Rothschild has organized in-person and virtual events for patients and families, including GIST Days of Learning, Life Fest, New Horizons GIST, and “Salúd con Datos” meetings globally. She also launched the first in-person clinic for pediatric patients with GIST at the National Institutes of Health (NIH), and when funding dried up, she turned to online technology, creating the collaborative NIH/LRG Virtual Tumor Board.
More recently, Rothschild convened a Pediatric & SDH-Deficient GIST Research Symposium that focused specifically on SDH-deficient GIST—a very rare subtype of the disease that affects children, usually girls. SDH-deficient GIST accounts for about 10% of all GIST cases annually.
“What’s been so wonderful over the years is to see how much we’ve learned through science about understanding GIST,” Rothschild said. “These young patients or their mothers and families were asking us to help us understand it more. We didn’t know back then it was called SDH‑deficient GIST.”
A meeting with Lee J. Helman, MD, who then headed the pediatric oncology branch at the National Cancer Institute, led to the development of a clinic at NIH to further study the disease subtypes.
“Because of that clinic, we learned about all the different SDH subtypes. Since then, clinical trials have been initiated for that subtype, though there have been no official approvals yet,” she said.
“We continue to support researchers along the way and do our own research with our patient registry and our tissue bank. I would like to continue to marry the worlds of our internal research and external research and see how we can increase that collaboration.”
