image: Dr. Nancy Klauber-DeMore's innovative research laid the foundation for the development of the first potential new treatment for osteosarcoma in over 30 years. Photo by Sarah Pack view more
Credit: Medical University of South Carolina
Innova Therapeutics Inc., a biotechnology company dedicated to developing innovative cancer therapies for patients who have inadequate treatment options, recently announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for IVT-8086 for the treatment of osteosarcoma.
MUSC Hollings Cancer Center researcher Nancy Klauber-DeMore, M.D., who is the co-founder and chairman of the Scientific Advisory Board for Innova Therapeutics, based in Charleston, South Carolina, was instrumental in the development of the research that led to the therapy. IVT-8086 is a humanized monoclonal antibody (mAb) with a high affinity receptor for secreted frizzled-related protein 2 (SFRP2) and is believed to be the only SFRP2 antagonist in development.
Under the Creating Hope Act passed into federal law in 2012, the FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and includes fewer than 200,000 people in the U.S.
Robert Ryan, Ph.D., president and chief executive officer of Innova Therapeutics, said the FDA's rare pediatric disease designation for IVT-8086 for the treatment of osteosarcoma highlights the significant unmet medical needs of patients with this devastating and life-threatening disease.
"Receiving rare pediatric disease designation from the FDA is a significant milestone for this program and underscores the critical value of our work," Ryan said. "The entire Innova team is encouraged by this designation and will continue to work diligently toward determining the efficacy and registration for IVT-8086 for the treatment of osteosarcoma."
Klauber-DeMore, who holds the BMW Endowed Chair in Cancer Research at MUSC, said the FDA designation of their drug is a huge breakthrough for osteosarcoma research. "This designation is an enormous validation of the work that we've been doing. It's really exciting because there have been no breakthroughs in osteosarcoma treatments in decades," she said. "IVT-8086 has the potential to become the first FDA-approved therapy for individuals with osteosarcoma in over 30 years."
Hollings Cancer Center director Raymond N. DuBois, M.D., Ph.D., helped form the collaboration, capitalizing on the innovative research of Klauber-DeMore and the expertise of Ryan, who has extensive experience in rare diseases. "This is very exciting for the cancer center. This is a new designation approved by the FDA for a terrible disease in children -- osteosarcoma," DuBois said. "Hollings values these kinds of partnerships that help to advance the great science happening here in our laboratories. Also, translating our basic science into the clinic is a major goal of Hollings, especially when there is such a great unmet need."
Klauber-DeMore and her team at Hollings Cancer Center have been studying IVT-8086 for the treatment of osteosarcoma, and preclinical results are encouraging. Her laboratory is focused on the role of SFRP2 in metastatic cancers. Her research shows that blocking SFRP2 with IVT-8086 reduces tumor growth in preclinical models. SFRP2 is also a potential marker of prognosis in osteosarcoma, as patients with high expression levels of SFRP2 have been shown to have poor survival. The results of her studies on osteosarcoma led to the rare pediatric disease designation for IVT-8086.
IVT-8086 currently is in late-stage pre-investigational new drug (pre-IND) development. The next step is to move the development of the therapy into the clinic and confirm the safety and efficacy of the drug in osteosarcoma patients. Klauber-DeMore, Ryan and colleagues are dedicated to developing IVT-8086 as an osteosarcoma therapy.
Osteosarcoma is a primary malignant bone tumor that generally arises in the long bones and, more rarely, in soft tissues. Osteosarcoma affects approximately 40,000 Americans, predominantly children, teenagers and young adults. It is a highly metastatic cancer wherein up to 20% of patients have detectable lung metastasis upon initial diagnosis, with a greater incidence in pediatric patients compared to adult patients, and 40% of patients develop lung metastasis in later stages. In the last 20 years, there has been little improvement in prognosis, even with available treatments, so new therapies are needed to improve the outcomes of patients with this serious and debilitating disease.
"We're passionate about trying to move the field forward and hopeful that we can find something to improve survival for these patients," Klauber-DeMore said.
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About MUSC
Founded in 1824 in Charleston, MUSC is the oldest medical school in the South, as well as the state's only integrated, academic health sciences center with a unique charge to serve the state through education, research and patient care. Each year, MUSC educates and trains more than 3,000 students and nearly 800 residents in six colleges: Dental Medicine, Graduate Studies, Health Professions, Medicine, Nursing and Pharmacy. The state's leader in obtaining biomedical research funds, in fiscal year 2019, MUSC set a new high, bringing in more than $284 million. For information on academic programs, visit musc.edu.
As the clinical health system of the Medical University of South Carolina, MUSC Health is dedicated to delivering the highest quality patient care available, while training generations of competent, compassionate health care providers to serve the people of South Carolina and beyond. Comprising some 1,600 beds, more than 100 outreach sites, the MUSC College of Medicine, the physicians' practice plan, and nearly 275 telehealth locations, MUSC Health owns and operates eight hospitals situated in Charleston, Chester, Florence, Lancaster and Marion counties. In 2019, for the fifth consecutive year, U.S. News & World Report named MUSC Health the No. 1 hospital in South Carolina. To learn more about clinical patient services, visit muschealth.org.
MUSC and its affiliates have collective annual budgets of $3.2 billion. The more than 17,000 MUSC team members include world-class faculty, physicians, specialty providers and scientists who deliver groundbreaking education, research, technology and patient care.
About MUSC Hollings Cancer Center
MUSC Hollings Cancer Center is a National Cancer Institute-designated cancer center and the largest academic-based cancer research program in South Carolina. The cancer center comprises more than 100 faculty cancer scientists and 20 academic departments. It has an annual research funding portfolio of more than $44 million and a dedication to reducing the cancer burden in South Carolina. Hollings offers state-of-the-art diagnostic capabilities, therapies and surgical techniques within multidisciplinary clinics that include surgeons, medical oncologists, radiation therapists, radiologists, pathologists, psychologists and other specialists equipped for the full range of cancer care, including more than 200 clinical trials. For more information, visit http://www.hollingscancercenter.org.