HemoShear Therapeutics

We all greatly desire new treatments to improve the quality of life for people with organic acidemias. In order for progress to be made, scientists need to better understand how these rare diseases work and identify ways to reduce the build-up of toxic acids that cause so much damage. HemoShear Therapeutics is a biotechnology company that is generating important insights that may lead to new therapies for propionic acidemia (PA), methylmalonic acidemia (MMA) and maple syrup urine disease (MSUD).

Modeling Human Disease to Discover Therapies
HemoShear, in collaboration with the Children’s National Rare Disease Institute (CN-RDI), has recreated models of organic acidemias in the laboratory by using tissue from patients who have donated their livers after transplant. The company’s unique technology is able to mimic human diseases to enable their scientists to study errors in the metabolic process and gain deep understanding of the course of diseases.

“To successfully meet the challenge of developing treatments for complex diseases like PA and MMA, we are advancing beyond animal research and static cells in a petri dish,” said Brian Wamhoff, PhD, Head of Innovation at HemoShear. “Our innovative platform combines the power of dynamic human biology and computational science to generate insights that may lead to new therapies to improve the lives of these children.”

Through these insights, HemoShear is now in the early stages of drug discovery — designing and assessing drugs to bypass errors in metabolism and lower the levels of toxic substances that build up throughout the body. They began their drug discovery program in 2016 and are progressing rapidly to identify compounds that may be effective at reducing the symptoms associated with PA, MMA and related diseases.

“HemoShear has already advanced our understanding and treatment of PA, MMA and MSUD,” said Marshall Summar, MD, director of the CN-RDI. “These diseases strike right at basic human metabolic function, leading to a cascade of complications from birth through the entire lifespan. If HemoShear can uncover the fundamental functioning of metabolism that is so important in these diseases, then we will be far down the path toward developing targeted treatments.”

Steps to the Clinic
Once HemoShear has identified a promising drug candidate through rigorous assessment with their disease model in the laboratory, followed by additional nonclinical testing, then they may apply for permission to test this compound in clinical trials. Clinical trials assess the safety and effectiveness of drugs in patients who volunteer to be participants at medical research institutions. This process can take a few years as clinical trials with experimental drugs must demonstrate substantial evidence of safety and efficacy before being considered for approval by the FDA for treating patients.

Meet the Company
Members of the HemoShear research team will attend our July meeting, where they will lead a workshop on the drug discovery and clinical trial process. This will give our families the opportunity to learn more from them directly and ask questions about their progress. 
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You can learn more by visiting the company’s website at www.hemoshear.com