Cryptosporidiosis causes severe diarrhoea and primarily affects babies and young children, and people with low immunity, such as HIV or transplant patients. Annually, more than 800,000 deaths are caused by diarrhoeal diseases, including cryptosporidiosis.
There is currently no vaccine and the one approved treatment is not effective in treating babies or patients with compromised immunity.
Writing in Nature, researchers supported by Wellcome report on the discovery and promising early trials of a potential drug to treat the disease.
Dr Stephen Caddick, Wellcome’s Director of Innovation, says: "Cryptosporidiosis is one of the leading causes of severe childhood diarrhoea. Developing new treatments has been particularly challenging. This study is a really important step forward in addressing an urgent need for new and safe drugs to treat the huge numbers of infants affected by this debilitating and life-threatening disease."
How the research was done
Professor Boris Striepen at the University of Georgia began studying the parasite that causes the disease – Cryptosporidium or crypto – more than a decade ago.
LinkCryptosporidium is most commonly spread through contaminated drinking water. Outbreaks have also been linked to contaminated water in swimming pools and water parks.
Discovery of the potential drug, KDU731, began with screening 6,200 compounds at the Novartis Institutes for Tropical Diseases (NITD).
New techniques were also developed to genetically modify the parasite to make it easier to carry out laboratory tests. These included manipulating the parasite so that it emits light and is easier to detect and measure.
The genetically modified organisms have been made available to researchers around the world to encourage other scientists to study the parasite.
"This is an important problem," Professor Striepen says. "No one institution can solve it alone. It needs significant investment, and it needs a lot of people with good ideas."
Manjunatha Ujjini, Senior Investigator at the NITD, adds: "The discovery of this compound represents an important step towards urgently needed treatment for seriously sick children around the world."
Read the research report in Nature – A Cryptosporidium PI(4)K inhibitor is a drug candidate for cryptosporidiosis.