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Novartis, University of Georgia and Washington State University identify drug candidate for cryptosporidiosis.

M2 EQUITYBITES-June 1, 2017-Novartis, University of Georgia and Washington State University identify drug candidate for cryptosporidiosis

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Experts in infectious disease from Novartis (VTX: NOVN), who teamed up with the University of Georgia and Washington State University last year, have announced the discovery and early validation of a drug candidate for treating cryptosporidiosis, Novartis reported on Thursday.

Cryptosporidiosis is a diarrhoeal disease that is a major cause of child mortality in lower-income countries and is responsible for some 800,000 deaths per year. Currently, there are no effective treatments or vaccines.

Epidemiological research has determined that in order to combat the disease, pharmacologists must target the protozoan parasite Cryptosporidium, which victims are often exposed to through contaminated waterways and or substandard supplies.

The only available medicine for the illness, Nitazoxanide, has demonstrated poor efficacy when used in the treatment of vulnerable infants and immune-compromised patients.

Boris Striepen from the University of Georgia has been studying the parasite for over a decade.

"Cryptosporidiosis is largely a disease of poverty," he said.

"Globally, it primarily affects infants in developing countries, but there are patients in the US -- those with weakened immune systems, such as HIV/AIDS or transplant patients -- that would benefit greatly from new therapeutics."

However, researchers face a significant challenge in fighting the protozoan parasite Cryptosporidium, due to the fact it perishes relatively quickly in labs and scientists spearheading the research have lacked adequate research tools necessary to identify drug candidates.

So far, the team has developed a new drug discovery process that uses transgenic parasites and novel disease models. UGA's lead researchers Striepen and Sumiti Vinayak used a mouse model to demonstrate that oral administration of the drug dramatically reduced intestinal infection of immunocompromised mice.

Moreover, further research led by Jennifer A. Zambriski at Washington State University found that that treatment with KDU731 also caused the rapid resolution of diarrhoea and dehydration in neonatal calves, a clinical model of cryptosporidiosis. This has resulted in the successful identification and validation of the Cryptosporidium PI(4)K inhibitor candidate KDU731. The discovery and preclinical findings were published in a recent issue of Nature.

"There's a lot of uncertainty when embarking on drug discovery for a notoriously intractable parasite such as Cryptosporidium, the cause of cryptosporidiosis," commented Thierry Diagana, head of the Novartis Institute for Tropical Diseases (NITD).

He continued: "Thanks to the commitment of our funding collaborators and urgent action of our academic colleagues, we've made an important step toward advancing a new treatment."

Swiss multinational Novartis hopes that through collaboration with leading industry experts, it will be able to significantly advance treatment options for the disease. Novartis is working alongside the global health community and its scientists at NITD towards this end.

Diagana encapsulated this intention by stating: "This is an important problem. No one institution can solve it alone. It needs significant investment, and it needs a lot of people with good ideas."

"The discovery of this compound represents an important step toward urgently needed treatment for gravely ill children around the world," he concluded.

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Publication:M2 EquityBites (EQB)
Date:Jun 1, 2017
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