Keystone Nano said today that the FDA approved its investigational new drug application for the ceramide nanoliposome as a treatment for solid tumors. The company will launch a phase I trial at 3 sites to evaluate safe dosing levels and the product’s efficacy as a cancer therapy.

Ceramide is a lipid that has demonstrated efficacy in liver cancer, breast cancer, leukemia and pancreatic cancer. Preclinical evidence has shown that Keystone’s nanoliposome is an effective delivery system for the bioactive compound, targeting cancer cells and ignoring healthy cells.

“This approval represents the conclusion of 1 phase of developing ceramide, a truly novel anti-cancer agent, and the beginning of the very important human testing phase,” CEO Jeff Davidson said in prepared remarks.  “We are excited by the efficacy and lack of toxicity ceramide nanoliposome has shown in preclinical testing, and we look forward to continuing to evaluate ceramide nanoliposome for human use.  This is a significant step forward in the development of ceramide nanoliposome for the treatment of cancer.”

Keystone reported that the phase I trial will recruit patients with solid tumors, while the phase II study will focus on liver cancer. The State College, Penn.-based company was granted orphan drug status for its ceramide nanoliposomes in October last year.

“The anti-cancer activity of ceramide has long been known in the laboratory,” VP of research Mylisa Parette added. “Until now, however, no-one has had an effective method for delivery of ceramide, which is insoluble and has an extremely short half-life in the body.  The proprietary formulation we are evaluating uses ceramide integrated into the membrane of a nanoliposome to overcome these barriers and allow us to exploit the novel properties of ceramide as an anti-cancer therapy.”