PARIS - Synt:em has released the results of animal studies demonstrating the efficacy of its Pep:trans technology for delivering drugs into the brain, confirming its potential in the treatment of central nervous system (CNS) diseases.

The tests involved the delivery of the anticancer agent doxorubicin, and showed that using Pep:trans as a vector increased the brain uptake of the drug by a factor of 20. At the same time, pharmacological studies indicated that Pep:trans delivery resulted in far lower concentrations of doxorubicin in the heart, a significant factor since one of the main adverse side effects of doxorubicin is its cardiotoxicity.

Pep:trans is an intracellular delivery system, consisting of small vectors derived from natural peptides that deliver bioactive molecules direct into human cells. As well as enhancing the uptake of therapeutic molecules at an intracellular level in general, this technology has been found to be particularly effective in delivering drugs through epithelial barriers, such as the blood-brain barrier (BBB). Because they cross the BBB without compromising the barrier's integrity, they are especially suited for the delivery of therapies for CNS diseases.

Synt:em said it has obtained even greater improvements in brain uptake with other drugs in recent in-house research in which it used proprietary computational methods to optimize specific Pep:trans vectors. Moreover, according to its director of sciences, Tony Rees, "The Pep:trans vectors developed by Synt:em not only effectively carry drugs across the blood-brain barrier, but at the same time improve the overall biodistribution of the drug in other tissues."

Synt:em does not intend to take this technology into clinical development itself but is currently negotiating development deals with pharmaceutical companies. Chairman and CEO Michel Kaczorek told BioWorld International that "at least one agreement will be signed" with big pharma this year. He also pointed out that, whereas in these animal studies Pep:trans was administered intravenously, the company had started work on developing an oral delivery version and was talking to drug delivery companies specialized in this area with a view to a collaboration.

Based in Nnmes, in southern France, Synt:em is a drug discovery company that uses powerful proprietary computer software to identify and develop new compounds and technologies with therapeutic potential. The company is focusing in particular on CNS diseases, including age-related degenerative diseases such as Alzheimer's and Parkinson's.