Celsion (NSDQ:CLSN) and Children’s National Health System are teaming up to tackle refractory solid tumors in pediatric patients using a heat-activated chemotherapy drug and noninvasive ultrasound. The Phase I clinical trial, which launched last November, is the 1st of its kind in the U.S.
Lawrenceville, N.J.-based Celsion’s ThermoDox is a heat-activated liposomal doxorubicin. In combination with MRI, high-intensity focused ultrasound is used to direct sound wave energy and heat the tumor. When the liposomal chemotherapy drug comes into contact with the heated tumor, the liposome changes structure and releases doxorubicin into the tumor.
The study, supported by the National Institutes of Health, is designed to determine the appropriate dose range of ThermoDox for pediatric patients with refractory solid tumors.
Dr. AeRang Kim, principal investigator at Children’s National, told Drug Delivery Business News that the trial is expected to have 3 or 4 dose levels with up to 6 children each, ultimately enrolling 20 patients. This is the most recent study from the Image-Guided Non-Invasive Therapeutic Energy program, representing the exciting potential for a non-invasive, combination therapy for a challenging cancer.
“The real mission is to improve the quality of life and outcomes for patients through the development and clinical introduction of noninvasive or minimally invasive technology and combination therapy approaches,” Kim explained. “Ultimately the goal is to be able to incorporate targeted position medicine into up-front treatment protocols.”
ThermoDox is also in late-stage clinical trials for patients with primary liver cancer and recurrent chest wall breast cancer. Celsion’s chairman, president & CEO, Michael Tardugno, told us that he envisions a variety of potential applications for the heat-activated drug, including metastatic liver and non-muscle invading bladder cancers.
“The ThermoDox formulation is really ideal. We deliver a systemic dose that is safe,” he said. “What we are able to do is concentrate the drug at the site of the tumor, thereby avoiding some of the systemic toxicities associated with the toxic agent like doxorubicin. We’ve conducted studies now that include almost 700 patients. We’re impressed with the side effect profile, similar in many ways to doxorubicin. We haven’t seen some of the more serious toxicities like the concentration in the heart muscle, the cardiotoxicity, that may be evident in long-term exposure in patients treated with doxorubicin.”
Tardugno told us that he anticipates that the company could be filing a new drug application as early as the 2nd half of 2018.