MUSC pediatric oncology researcher instrumental in newly FDA-approved neuroblastoma drug

Charleston, S.C. (Dec. 18, 2023) – WorldMeds USWM LLC announced that the U.S. Food and Drug Administration (FDA) has approved IWILFIN, also known by generic name eflornithine or DFMO, a groundbreaking oral maintenance therapy for high-risk neuroblastoma in pediatric patients. This life-changing therapy helps to prevent relapse so children can return to school and live normal lives with full heads of hair after undergoing chemotherapy, surgery, tandem stem-cell transplants, radiation and immunotherapy.

Jacqueline Kraveka, D.O., a pediatric hematologist-oncologist at MUSC Children’s Health and a cancer researcher at MUSC Hollings Cancer Center was the institutional principal investigator for the DFMO trials. Kraveka is a national leader in neuroblastoma research and an integral part of the executive committee and scientific committee for the Beat Childhood Cancer Research Consortium (BCC). She was a member of the core team that designed and conducted the clinical trials that led to FDA approval.

The WorldMeds USWM LLC press release stated that 700 to 800 cases of neuroblastoma are diagnosed in the U.S. each year, with 90% of diagnoses coming before the age of 5. The study demonstrated that the addition of IWILFIN improved event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma. At four years following immunotherapy, EFS in the IWILFIN-treated patient group was 84% compared with 73% of patients in the external control group, and 96% of patients treated with IWILFIN were alive compared with 84% of external control patients. This corresponded to a 52% reduction in the risk of relapse and a 68% reduction in the risk of death.

“The global impact of the eflornithine approval for pediatric patients and families cannot be underestimated,” said Kraveka. “This is the first drug approved to prevent relapse for a pediatric cancer. It gives new hope for our patients. IWILFIN is well-tolerated with minimal side effects. This drug approval will be life-changing for families, and I am honored to have helped to lead this research endeavor.”

“This incredible treatment option is going to add quality of life to pediatric neuroblastoma patients and their families across the globe,” said MUSC Hollings Cancer Center director Raymond N. DuBois, M.D., Ph.D. “These strides by Dr. Jacqueline Kraveka and her colleagues reinforce our unwavering commitment at Hollings to deliver optimal care options and pursue cutting-edge science and ultimately, to work relentlessly toward the eradication of cancer.”

Hollings Cancer Center (HCC) is one of 72 National Cancer Institute-designated (NCI) cancer centers in the nation and the only one in South Carolina. The prestigious NCI designation recognizes the exemplary research and patient care HCC offers adult and pediatric patients across the Southeast region and beyond. Kraveka’s contributions to cancer research and the IWILFIN trial highlight the level of innovation and expertise at HCC as well as MUSC’s leadership in patient care powered by groundbreaking research.

MUSC Shawn Jenkins Children’s Hospital is ranked 26th in the nation for pediatric cancer care. The hospital, on the same campus as MUSC Hollings Cancer Center, includes a floor dedicated to children with cancer.

Michelle Hudspeth, M.D., director of adult and pediatric blood and marrow transplantation and division chief of Pediatric Hematology / Oncology, praised Kraveka’s role in this important approval. “Dr. Kraveka’s efforts as our institutional primary investigator and a national leader in pediatric oncology research have been essential for the drug approval. I respect no one more for their dedication to patients and families and challenging clinical cancer care.”

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About MUSC Hollings Cancer Center

MUSC Hollings Cancer Center is South Carolina’s only National Cancer Institute-designated cancer center with the largest academic-based cancer research program in the state. The cancer center comprises more than 130 faculty cancer scientists and 20 academic departments. It has an annual research funding portfolio of more than $44 million and sponsors more than 200 clinical trials across the state. Dedicated to preventing and reducing the cancer burden statewide, the Hollings Office of Community Outreach and Engagement works with community organizations to bring cancer education and prevention information to affected populations.

Hollings offers state-of-the-art cancer screening, diagnostic capabilities, therapies and surgical techniques within its multidisciplinary clinics. Hollings specialists include surgeons, medical oncologists, radiation oncologists, radiologists, pathologists, psychologists and other clinical providers equipped to provide the full range of cancer care. For more information, visit hollingscancercenter.musc.edu.

About MUSC Children’s Health 

MUSC Children’s Health is changing what’s possible for children and their families by providing the pediatric clinical and research expertise every child deserves. From promoting healthy lifestyles to offering life-saving treatments, MUSC Children’s Health delivers comprehensive and compassionate care to children throughout South Carolina and beyond. 

MUSC’s integrated children’s health care system consists of the 250-bed  Shawn Jenkins Children’s Hospital (SJCH) and Pearl Tourville Women’s Pavilion located on the Charleston peninsula, which opened in February 2020; the Charles P. Darby Research Institutewhere research teams conduct widely-published pediatric studies discoveries that change how care is provided; and the R. Keith Summey Medical Pavilion in North Charleston, which offers an ambulatory care and surgery center, specialty care and after-hours clinics. MUSC Children’s Health also offers an expanded depth and breadth of expertise in multiple locations throughout the state, providing primary, urgent and specialty care in person or via telehealth.  

To provide the best care for children of South Carolina now and in the future, Children’s Health puts the needs of patients and their families first. This intentional approach aligns with the emerging national best practice of offering children’s ambulatory and specialty services within local communities and the delivery of more acute care at a major flagship hospital. As a result, families can more easily access the specialized pediatric care they seek. For more information, visit musckids.org.