Children’s National and Celsion Announce Clinical Study to Treat Recurrent Childhood Tumors First in US to use ThermoDox® in combination with noninvasive magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU) in children November 15, 2016

WASHINGTON –  Children’s National Health System and Celsion Corporation (NASDAQ; CLSN), a leading oncology drug development company, announce the launch of a clinical study to evaluate the use of ThermoDox®, a heat-activated chemotherapy drug, in combination with noninvasive magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU) to treat refractory or relapsed solid tumors in children and young adults – a first in the U.S.
 
The primary objective of the investigator-sponsored Phase I study, partially funded by a National Institutes of Health (NIH) R01 grant, is to determine a safe and tolerable dose of ThermoDox®, a lyso-thermosensitive liposomal doxorubicin (LTLD), which can be administered in combination with MR-HIFU. Under the guidance of an MRI, the high-intensity focused ultrasound directs sound wave energy to heat the tumor and the area around the tumor. When heated, the liposome rapidly changes structure and releases doxorubicin directly into and around the targeted tumor. 

“There is currently no known cure for many patients with refractory recurring solid tumors, despite the use of intensive therapy, so we need to identify new, smarter therapies that can improve outcomes,” said AeRang Kim, MD, PhD, an Oncologist and member of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National, and Principal Investigator for the study. “Recent advances in the use of noninvasive MR-HIFU coupled with novel therapies, such as LTLD, may provide us with a mechanism to noninvasively administer high concentrations of the drug directly to the site where it is most needed and avoid toxicity to other areas of the body.” 

This is the first time LTLD is being combined with MR-HIFU and the first time it is being evaluated in children. 

"Celsion’s experience in combining ThermoDox® with HIFU, a noninvasive next generation heating technology, supports this very important research in childhood cancers. From a safe dose, ThermoDox’s proven ability to deliver high concentrations of an effective chemotherapy directly to a heated tumor makes it an ideal candidate for a trial involving children and young adults," said Michael H. Tardugno, Celsion's Chairman, President and Chief Executive Officer. "This study will further elucidate ThermoDox®’s potential in combination with ultrasound-induced hyperthermia, and highlight potential applications of ThermoDox® in combination with a broad range of heating technologies that could address an even larger population of patients." 

The study targeting the treatment of childhood sarcomas will be carried out as a multi-disciplinary collaboration between Children’s National, Celsion, and Dr. Bradford Wood’s team at the NIH. 

This is the latest study from the Image-Guided Non-Invasive Therapeutic Energy (IGNITE) program, a collaboration of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and the pediatric health system’s Divisions of Radiology, Oncology, Surgery, and Anesthesiology. The goal of the IGNITE program is to improve the quality of life and outcomes for pediatric patients through the development and clinical introduction of novel minimally invasive and noninvasive surgery technologies and combination therapy approaches. In 2015, doctors from Children’s National were the first in the U.S. to treat osteoid osteoma, a benign and painful bone tumor, using MR-HIFU.ThermoDox® is currently in late stage clinical trials in primary liver cancer and recurrent chest wall breast cancer. It is positioned for use with multiple heating technologies, and has the potential for applications in the treatment of other forms of cancer including metastatic liver and non-muscle invading bladder cancers.

Contact: Rebecca Porterfield | Children's National Health System | 202-476-450 | rporterfi2@childrensnational.org



About Children’s National Health System

Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is ranked in the top 20 in every specialty evaluated by U.S. News & World Report; one of only four children’s hospitals in the nation to earn this distinction. Designated a Leapfrog Group Top Hospital and a two-time recipient of Magnet® status, this pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.

About ThermoDox®

Celsion's most advanced program is a heat-mediated, tumor-targeting drug delivery technology that employs a novel heat-sensitive liposome engineered to address a range of difficult-to-treat cancers. The first application of this platform is ThermoDox®, a lyso-thermosensitive liposomal doxorubicin (LTLD), whose novel mechanism of action delivers high concentrations of doxorubicin to a region targeted with the application of localized heat at 40°C, just above body temperature. ThermoDox® has the potential to address a broad range of cancers.

Celsion's LTLD technology leverages two mechanisms of tumor biology to deliver higher concentrations of drug directly to the tumor site. In the first mechanism, rapidly growing tumors have leaky vasculature, which is permeable to liposomes and enables their accumulation within tumors. Leaky vasculature influences a number of factors within the tumor, including the access of therapeutic agents to tumor cells. Administered intravenously, ThermoDox® is engineered with a half-life to allow significant accumulation of liposomes at the tumor site as these liposomes recirculate in the blood stream.  In the second mechanism, when an external heating device heats tumor tissue to a temperature of 40°C or greater, the heat-sensitive liposome rapidly changes structure and the liposomal membrane selectively dissolves, creating openings that can release a chemotherapeutic agent directly into the tumor and into the surrounding vasculature. Drug concentration increases as a function of the accumulation of liposomes at the tumor site, but only where the heat is present. This method damages only the tumor and the area related to tumor invasion, supporting more precise drug targeting.

About Celsion Corporation

Celsion is a fully-integrated oncology company focused on developing a portfolio of innovative cancer treatments, including directed chemotherapies, immunotherapies and RNA- or DNA-based therapies. The Company's lead program is ThermoDox®, a proprietary heat-activated liposomal encapsulation of doxorubicin, currently in Phase III development for the treatment of primary liver cancer and in Phase II development for the treatment of recurrent chest wall breast cancer. The pipeline also includes GEN-1, a DNA-based immunotherapy for the localized treatment of ovarian and brain cancers. Celsion has two platform technologies for the development of novel nucleic acid-based immunotherapies and other anticancer DNA or RNA therapies, including TheraPlas™ and TheraSilence™. The Company has a Cooperative Research and Development Agreement (CRADA) with the NIH.  Any reference to NIH should not be viewed as an endorsement of Celsion, its products or services.For more information on Celsion, visit our website: http://www.celsion.com. (LTSL/ThermoDox®)

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