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Immunotherapy Combo Halts Rare, Stage 4 Sarcoma in Teen

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HACKENSACK, N.J., Oct. 15, 2020 /PRNewswire/ -- A patient with end-stage and rapidly progressing soft-tissue cancer whose tumor did not respond to standard treatment, had a "rapid and complete response" to a novel combination of immunotherapy, according to new research published by a team of scientists from John Theurer Cancer Center at Hackensack University Medical Center and the Georgetown Lombardi Comprehensive Cancer Center, both of whom are part of the Georgetown Lombardi Comprehensive Cancer Center Consortium.

The immunotherapies targeting the immune checkpoints T-lymphocyte-associated protein 4 (CTLA-4) and programmed cell death protein 1 (PD-1) were administered to a 19-year-old patient with stage 4 epithelioid sarcoma. The patient, whose tumor responded within two weeks after receiving the combination, resumed normal activity and was in a complete remission at the time of the report. The single case was reported online August 18, 2020 in the Journal of Immunotherapy (with the patient's consent).

"Epithelioid sarcoma is a rare cancer, and the outcome was not expected to be so positive," said Andrew Pecora, M.D., F.A.C.P., C.P.E., the division chief of skin cancer and sarcoma services at John Theurer Cancer Center. "The breakthrough in this patient's care was the result of the close collaboration between clinician scientist of the Consortium to elucidate the underlying mechanisms that suggested potential sensitivity to the checkpoint inhibitors."

"The teamwork in this case, which involved experts from our Consortium, turned things around for this patient," said Louis M. Weiner, M.D., director, Georgetown Lombardi Comprehensive Cancer Center and the MedStar Georgetown Cancer Institute. "The team will follow the patient closely in hopes that we can continue to keep the cancer at bay."

The patient was first diagnosed with the soft-tissue sarcoma along the spine in 2017, as a 17 year old. Chemotherapy, radiation and standard of care drugs, were administered, and surgery was performed to achieve partial response. The patient was admitted to the hospital in April 2019 in severe pain and his cancer had progressed to stage 4.

Most cases of epithelioid sarcoma show an inactivation of a protein-coding gene known as SMARCB1. That inactivation leads to the suppression of INI1, a gene that codes for a tumor-suppressing protein, effectively causing a biological chain reaction that promotes tumor growth.

The doctors at John Theurer Cancer Center, working as a team with Georgetown's experts, obtained a compassionate use authorization to try two checkpoint inhibitors, ipilimumab (anti-CTLA4) and nivolumab (anti-PD1) in May 2019.

By October, the patient was in complete remission. As of his last visit in June 2020, he has resumed normal activities and normal physical examination and is essentially asymptomatic.

According to the report, the patient's tumor was reliant on the lack of INI1. But the checkpoint inhibitors apparently unmasked the immune system, making the cancer cells susceptible to the body's natural defenses again.

The researchers write that more about the dramatic reversal of the patient's cancer needs to be further investigated, particularly what effect the chemotherapy before and after the checkpoint inhibitors may have had.

"Immune checkpoint inhibitors are finding a way to effect previously impossible outcomes and we are trying to learn about the precise indicators that suggest clinical utility of the checkpoint inhibitors," said senior author Jeffrey Toretsky, M.D., a professor of oncology and pediatrics at Georgetown Lombardi and chief of MedStar Georgetown University Hospital's Division of Pediatric Hematology/Oncology. "This patient opens a window for this exploration. We're proud to be able to successfully treat patients in this way."  

"We are eager to see what our science can do for our sickest patients," said Andre Goy, M.D., M.S., physician-in-chief of Oncology, Hackensack Meridian Health. "It seems like almost anything is becoming possible."

In addition to Dr. Pecora and Dr. Toretsky, authors of the report include Steven Halpern, M.D.; Melinda Weber, DNP, RN, APN, AOCN; Elli G. Paleoudis, MS, Ph.D.; David Panush, M.D.; and Francis Patterson, M.D.  All authors have declared that there are no financial conflicts of interest with regard to this work.

About Hackensack Meridian Health

Hackensack Meridian Health is a leading not-for-profit health care organization that is the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care.

Hackensack Meridian Health comprises 17 hospitals from Bergen to Ocean counties, which includes three academic medical centers – Hackensack University Medical Center in Hackensack, Jersey Shore University Medical Center in Neptune, JFK Medical Center in Edison; two children's hospitals - Joseph M. Sanzari Children's Hospital in Hackensack, K. Hovnanian Children's Hospital in Neptune; nine community hospitals – Bayshore Medical Center in Holmdel, Mountainside Medical Center in Montclair, Ocean Medical Center in Brick, Palisades Medical Center in North Bergen, Pascack Valley Medical Center in Westwood, Raritan Bay Medical Center in Old Bridge, Raritan Bay Medical Center in Perth Amboy, Riverview Medical Center in Red Bank, and Southern Ocean Medical Center in Manahawkin; a behavioral health hospital – Carrier Clinic in Belle Mead; and two rehabilitation hospitals - JFK Johnson Rehabilitation Institute in Edison and Shore Rehabilitation Institute in Brick.

Additionally, the network has more than 500 patient care locations throughout the state which include ambulatory care centers, surgery centers, home health services, long-term care and assisted living communities, ambulance services, lifesaving air medical transportation, fitness and wellness centers, rehabilitation centers, urgent care centers and physician practice locations. Hackensack Meridian Health has more than 36,000 team members, and 7,000 physicians and is a distinguished leader in health care philanthropy, committed to the health and well-being of the communities it serves.

The network's notable distinctions include having four of its hospitals are among the top hospitals in New Jersey for 2020-21, according to U.S. News & World Report. Additionally, the health system has more top-ranked hospitals than any system in New Jersey. Children's Health is again ranked a top provider of pediatric health care in the United States and earned top 50 rankings in the annual U.S. News' 2020-21 Best Children's Hospitals report.   Other honors include consistently achieving Magnet® recognition for nursing excellence from the American Nurses Credentialing Center and being named to Becker's Healthcare's "150 Top Places to Work in Healthcare/2019" list.

The Hackensack Meridian School of Medicine, the first private medical school in New Jersey in more than 50 years, welcomed its first class of students in 2018 to its On3 campus in Nutley and Clifton. The Hackensack Meridian Center for Discovery and Innovation (CDI), housed in a fully renovated state-of-the-art facility, seeks to translate current innovations in science to improve clinical outcomes for patients with cancer, infectious diseases and other life-threatening and disabling conditions.

Additionally, the network partnered with Memorial Sloan Kettering Cancer Center to find more cures for cancer faster while ensuring that patients have access to the highest quality, most individualized cancer care when and where they need it.

Hackensack Meridian Health is a member of AllSpire Health Partners, an interstate consortium of leading health systems, to focus on the sharing of best practices in clinical care and achieving efficiencies.

To learn more, visit www.hackensackmeridianhealth.org.

About John Theurer Cancer Center at Hackensack University Medical Center

John Theurer Cancer Center at Hackensack University Medical Center is New Jersey's largest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to John Theurer Cancer Center for cancer care than to any other facility in New Jersey. John Theurer Cancer Center is a member of the Georgetown Lombardi Comprehensive Cancer Center Consortium, one of just 16 NCI-approved cancer research consortia based at the nation's most prestigious institutions. Housed within a 775-bed not-for-profit teaching, tertiary care, and research hospital, John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish John Theurer Cancer Center from other facilities. For additional information, please visit www.jtcancercenter.org.

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SOURCE Hackensack Meridian John Theurer Cancer Center

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