Two key, independent proof-of-concept studies conducted for NellOne
Therapeutics, Inc. (NellOne) demonstrate the potential efficacy of the
company’s lead therapeutic candidate for both skeletal muscle wound
healing and myocardial infarction.
“Following further
proof-of-concept studies this year, we hope to be ready to commence
preclinical testing and toxicology studies for both protein treatments
derived from the Nell1 platform.”
The regenerative medicine company, which was spun out of the U.S.
Department of Energy’s Oak Ridge National Laboratory (ORNL) in 2008, is
developing novel therapeutic
treatments that restore both mass and function to damaged human
tissues, such as heart and skeletal muscle. These treatments are based
on the discoveries of company founder Dr.
Cymbeline Culiat, who, as an ORNL systems genetics researcher,
identified the role
that the Nell1 pathway plays in tissue growth and maturation.
The two efficacy studies, in vivo for skeletal muscle and in
vitro for heart muscle, sought to establish the Nell1 protein
therapeutic’s promise to effectively improve wound healing and to
restore cardiac tissue while also preventing further damage to heart
cells, explained Dr. Culiat, who also is chair of the NellOne Scientific
Advisory Board.
“Evaluation of the protein for skeletal muscle wound healing in a murine
diabetic model showed that Nell1 significantly increased new
blood-vessel formation and muscle regrowth,” she said, adding that the
results were consistent with earlier genetic and genomic studies she
conducted at ORNL—that Nell1 triggers the production of several
extracellular matrix (ECM) proteins that support cell proliferation and
maturation in muscle and the blood-vessel network.
The cardiac study evaluated the protein as a treatment for myocardial
infarction by testing if Nell1 can protect cardiomyocytes (heart muscle
cells) in a low-oxygen (hypoxic) environment. “These conditions mimic
what heart-muscle cells are subjected to during a heart attack,” Dr.
Culiat continued.