CHARLOTTESVILLE, Va., Jan. 6, 2016 /PRNewswire/ -- HemoShear Therapeutics, LLC, (HemoShear) a privately held discovery-stage biotechnology company, and Children's National Health System, a non-profit leading research and treatment center for children with rare diseases, today announced study results that demonstrated successful restoration of propionic acidemia (PA), a rare metabolic disorder, using explanted liver tissue from a child with the disease. This study, published in the Journal of Molecular Genetics and Metabolism, demonstrated a novel method of recreating the disease in the laboratory for discovery of treatments for PA that may extend the lives of these children and possibly cure the disease.
This study resulted from a collaboration between HemoShear Therapeutics, Children's National, MedStar Georgetown University Hospital, and the child's parents, who want to encourage efforts to discover treatments for PA. PA is an inherited genetic disorder in basic cellular metabolism which, untreated, leads to the rapid buildup of life-threatening toxins in the body. Over time the disorder also leads to cardiovascular and neurological complications and eventual organ failure. PA has no approved drug therapy and is treated through strict diets and support medications that nearly always fail.
"HemoShear has the scientific expertise necessary to develop a biologically responsive model for interrogating this rare disease," said Brian Wamhoff, Ph.D., HemoShear Therapeutics' head of R&D. "If HemoShear is successful in discovering a treatment for PA, then the doors will inevitably open for us to discover treatments for other organic acidemias and rare diseases of the liver, as well as to address broader applications in precision medicine."
Children born with PA often require liver transplants at an early age, and their livers are usually discarded. The research study became possible when a liver from an 8-year-old child with PA was transplanted. With the approval of the child's parents, Children's National and MedStar Georgetown University Hospital, the child's diseased liver tissue was sent to HemoShear.
Using its REVEAL-Tx™ discovery platform and working closely with the team from Children's National, HemoShear recapitulated the child's disease in the laboratory. Data from the study showed restoration of the in vivo disease condition, a physiologically-relevant response to nutrients, and elevated levels of toxins consistent with what is observed in patients. HemoShear has subsequently initiated drug discovery programs to treat and potentially cure PA.
"To my knowledge, this is the first time anyone has been able to use liver tissue from a patient with propionic acidemia to recreate the biology of the disease in the laboratory," said Marshall Summar, M.D., Chief of the Division of Genetics and Metabolism and the Margaret O'Malley Chair of Molecular Genetics at Children's National. "It is very difficult to gain meaningful response data about this disease from animal studies."
The article was published in the December 2015 issue of the Journal of Molecular Genetics and Metabolism and can be found on-line: K.A. Chapman, et al., Recapitulation of metabolic defects in a model of propionic acidemia using patient-derived primary hepatocytes, Mol. Genet. Metab. (2015), http:/dx.doi.org/10.1016/j.ymgme.2015.12.008.
About HemoShear Therapeutics
HemoShear Therapeutics is a biotechnology company focused on discovering drugs that treat metabolic disorders. Our transformational tissue models allow us to understand human diseases at a level not possible until now. Our current drug discovery programs are focused on organic acidemias, a group of rare genetic metabolic disorders, and nonalcoholic steatohepatitis (NASH).
At HemoShear Therapeutics, we believe that precision matters: best in class models of human disease lead to meaningful drug targets and optimal therapies. We THINK HUMAN.
REVEAL-Tx™, HemoShear Therapeutics' proprietary discovery platform, is agnostic to therapeutic modality and provides a powerful lens to interrogate biological mechanisms of human disease to identify novel targets and biomarkers and to discover superior therapeutics.
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SOURCE HemoShear Therapeutics