Novogen, an oncology drug development company, is collaborating with Australian CRO Genea Biocells to accelerate testing of its super-benzopyran (SBP) drugs for degenerative diseases of the nervous system and muscles.

Preliminary research conducted by both companies found super-benzopyrans appear to be effective at promoting the “normalization” of stem cells associated with some forms of neurodegeneration and muscular dystrophy, genetic disorders that result in progressive deterioration of brain function or muscle strength and function.

"SBPs have already been shown to be highly effective at killing cancer stem cells, which were previously considered resistant to anti-cancer therapy. But in some of our studies, we found in certain instances some of these drugs actually appeared to normalize both the behavior and appearance of the cancer cells. It was that observation that set us on the path to testing their ability to do the same thing with stem cells carrying genetic disorders," said Graham Kelly, CEO of Novogen.

Genea Biocells focuses on embryonic stem cells with genetic disorders. The cells have been sourced from embryos donated by couples undergoing in vitro fertilisation and pre-implantation genetic diagnosis (PGD). PGD enables the genetic testing of embryos before they are implanted to make sure babies born are not affected by a genetic disease. Genea is using those stem cell lines to test compounds to identify potential clinical development candidates.  

"Super-benzopyrans are the first drug class we have seen with the ability to selectively modulate misbehaving stem cells," said Uli Schmidt, general manager, Genea Biocells.

"Effected embryos identified during the PGD process cannot be used for implantation, but can instead be donated by patients to develop stem cell lines carrying the genetic disease. These disease-specific cell lines now can be used to test the effectiveness of exciting new compounds that are thought to counteract the disease. The testing of new molecules on individual stem cells holds great promise in the pursuit of new therapies," said Schmidt.

The collaboration will test the molecules in laboratory models across a range of degenerative diseases including infantile neuraxonal dystrophy, fascioscapularhumeral dystrophy, amyotrophic lateral sclerosis (motor neurone disease), Sanfillipo syndrome and Alzheimer's disease. It will pool respective resources, with each company retaining its own intellectual property rights and commercial opportunities.