Some of you may recall what I hope was a heartwarming holiday column about one family, a doctor and their unified hope for a new beginning (in the Dec. 26, 2018 La Jolla Light, “Frontline Cancer : The season for family, health, hope and new beginnings” at lajollalight.com/scott-lippman/cm-ljl-frontline-cancer-jan3-2019-20190102-story.html)
I’d like to update you on their journey and share an amazing story of an uncommon partnership in rare disease that has accelerated strategic alliances and rare cancer research, specifically SDH-deficient Gastrointestinal Stromal Tumor (GIST) research, all in the last three years.
Merak Melikian Hatounian just turned 25 and continues to bravely battle a very rare form, known as SDH-deficient GIST (only 150-200 patients are diagnosed with SDH-deficient GIST in the United States each year). He is receiving seventh line therapy, a personalized therapy based on research being done by Dr. Jason Sicklick and his leading research team at UC San Diego Moores Cancer Center. This NCI-designated cancer center is a global destination for those with rare cancers like SDH-deficient GIST.
Merak’s mother, Debra Melikian, is the unstoppable driving force on a relentless mission to raise awareness and financial support to accelerate pioneering SDH-deficient GIST research being led by Sicklick. He, too, is an unstoppable force with a passion for research and genuine compassion for his patients like Merak. According to Debra, the day they met in November 2015 was the day that changed the course of Merak’s care and all of their lives forever.
Soon after, Debra asked Sicklick how she could help him get what he needed to help save Merak, her only child. He said it came down to funding the research. Without missing a beat, she told him that she could do that. In that very moment, her quest began, and their unique collaboration based in mutual respect and resolution was born.
Debra is inspirational in her encouragement and support of Moores’ team vision to solve the mystery of SDH-deficient GIST. “This has evolved into a true partnership between Debra and our GIST research team,” said Sicklick, who has thoughtfully and deliberately built a clinical and laboratory research program comprised of medical oncologists, gastroenterologists, radiologists, and pathologists, as well as MD/PhD scientists, geneticists, and bioinformaticians.
A major challenge in the past was the total lack of SDH-deficient models to unravel Merak’s rare cancer type in the laboratory, which seriously impeded meaningful advances in this field. Motivated by a race to fight Merak’s cancer, as well as those afflicting other teenagers and young adults, Sicklick and his team have been able to culture (grow) SDH-deficient cell lines in the laboratory, deciphering the biology of these tumors, thus far testing over 5,000 drugs against this elusive disease. With this recently accelerated research, they are developing personalized therapies for their patients, including Merak. We all remain hopeful.
Along this journey, Debra has created many strategic alliances including one with Global Genes, a rare disease advocacy organization based in Orange County that connects, inspires and empowers the rare-disease community. “Global Genes continues to be a great resource for me in the rare disease space,” she said. Their upcoming event, the RARE Patient Advocacy Summit, offers exceptional programming content including the opportunity to learn about how other rare diseases have been conquered through patient advocacy.
This summit is an annual gathering of patients, patient advocates, medical professionals and representatives from industries and universities around the world.
As a group, rare cancers remarkably account for 15 percent of all new cancers in the United States annually. This staggering burden — more than 200,000 or about 1 in 8 patients — yearly, does not include the striking increase in the number of rare forms of more common cancers, an inevitable result of the “omics” technology revolution. The perception that rare tumors are uncommon has led to little investment in this relatively uncharted area. A national leader in this space, Dr. Razelle Kurzrock leads one of the very few dedicated rare tumor clinics (at Moores), chairs the national NCI Southwest Oncology Group (SWOG) cooperative rare-tumor committee and, with Dr. Sandip Patel, leads the only NCI rare-tumor national trial.
Nicole Boice, founder of Global Genes, is an alumna of UC San Diego and was excited to learn about all the progress being made at her alma mater. According to Boice, “Research in rare disease and rare cancers is hard. This is why collaborations are key. This is why working with compassionate and committed champions for these communities is so critical. Without both, we will not succeed.”
Tapping into the patient advocacy power and strategic alliances that Debra brings, I recently invited Boice and Maureen McArthur Hart, PhD, strategic adviser at Global Genes, to a lunch meeting to share our vision for the Moores Cancer Center Rare Cancer Initiative and a collaboration with Global Genes. “We are honored to be partnering with these incredibly committed individuals at an institution that puts patients first. We are so grateful for the groundbreaking work being done at UC San Diego, and about our growing collaborations to support patients and their families on these difficult and complex rare journeys,” Boice said.
Global Genes will be presenting its eigth annual RARE Patient Advocacy Summit in San Diego on Sept. 18-20, globalgenes.org/event/patient-summit
I’m very proud to share that our own Sicklick (recipient of the 2018 RARE Champion of Hope Award in Medical Care and Treatment) and Kurzrock, a global leader in precision medicine, will be featured speakers at the 2019 Global Genes RARE Patient Summit. Debra will join an expert panel, a first, speaking on the impact of community engagement and Research Collaborations. According to Debra, who nominated him for the prestigious 2018 award, “Dr. Sicklick has been and will always be our RARE Champion of Hope.”
— Scott M. Lippman, MD, is director of UC San Diego Moores Cancer Center. His column on medical advances from the front lines of cancer research and care regularly appears in La Jolla Light. You can reach Dr. Lippman at email@example.com