A new class of anticancer drugs developed by researchers at Nationwide Children’s Hospital will be licensed by Open Therapeutics, according to a company announcement.
The drug, amphiphilic amines (RCn), has shown promise against human cancer cell lines and mouse models of cancer, including pediatric and adult lymphoma, neuroblastoma, and sarcoma.
“We are excited about this small molecule drug opportunity. Making it freely available to the global research community can fundamentally change the way therapeutics are developed and delivered to the world’s population of patients,” Jason E. Barkeloo, chairman of Open Therapeutics, said in a news release.
The drugs’ beneficial effects were recently described in a previous study, where researchers demonstrated that one particular derivative of RCn, called RC16, was the most active compound, showing 10 times more activity against tumor cells than healthy cells.
In addition, due to its particular properties — they are formed by a water-soluble part and a water-insoluble part — amphiphilic amines can organize into lipid structures with the ability to carry approved anticancer chemotherapy drugs, including Adriamycin (doxorubicin), VePesid (etoposide), and Taxol (paclitaxel). This suggests they can provide a new means to safely deliver chemotherapy agents specifically into cancer cells.
Dr. Timothy P. Cripe, MD, PhD, RCn co-investigator and developer, and principal investigator at Columbus, Ohio’s Nationwide Children’s Hospital Center for Childhood Cancer and Blood Diseases, said “RCn represents a novel type of therapy that destroys the energy centers of the cancer cells.”
In fact, RCn targets specific residues found in the mitochondria of cancer cells and induced damage to these organelles, markedly reducing the cell’s ability to produce energy.
“An important aspect of RCn is its ability to accumulate into the mitochondria of cancer cells, thereby disrupting their metabolism and inducing apoptosis, that is a form of cell suicide,” said Isabella Orienti, PhD, professor of pharmacy and biotechnology at the University of Bologna in Italy, the RCn primary investigator and developer.
“In addition, RCn may function as a micellar nanocarrier for traditional antitumor drugs, allowing the combination of a mitochondria-directed therapy with the conventional antitumor therapy,” she said.
“Our collaboration with the team at Nationwide Children’s Hospital is a first great example of how open licensing can quickly broaden the availability of new therapies,” said Open Therapeutics CEO Jerome Hamilton. “As our platform grows and these new cooperative philosophies gain traction, we look forward to supporting further development of such treatments.”