St. Jude Children’s Research Hospital has discovered a fast-acting anti-malarial compound that is well-tolerated and exhibits promising anti-malarial effects in the first clinical studies. Called SJ733, researchers are exploring ways to increase and/or extend blood levels of the experimental drug to maximize its effectiveness. The investigators, working across continents and time zones in a seamless and collaborative manner, are in pursuit of an improved way to stop one of the leading causes of death in children.
Malaria is a Real Challenge
About 3.4 billion people live in areas of the world where malaria is regularly found. In many countries, the disease is one of the leading causes of illness and death. Despite this, there are limited number of drugs called antimalarials, that can be used to treat malaria and increasing reports of resistance to existing antimalarials.
Malaria is by a parasite transmitted by a mosquito, destroys red blood cells. Currently Artemisinin-based combination drug therapy is used as first-line treatment for malaria. However, its success is under severe pressure by emerging drug resistance. Figures vary but, according to World Health Organization (WHO), by 2018, over 220 million contracted malaria and over 405,000 died. A majority of the deaths occur in sub-Saharan Africa.
About 3.4 billion people live in areas of the world where malaria is regularly found. In many countries, malaria is one of the leading causes of illness and death. Despite this, there are a limited number of drugs, called antimalarials, that can be used to treat malaria and increasing reports of resistance to existing antimalarials.
SJ733 is one of the first in a new class of anti-malarial compounds to reach clinical trials. It works by disrupting the malaria parasite’s ability to remove excess sodium from red blood cells. As sodium builds up, infected cells become less flexible. The cells are removed by the immune system or get caught in small blood vessels.
The study was published in Clinicaltrials.gov but some of the details of the study, such as site locations, and total number of participants, adjusted as the study unfolded.
Sponsored by St. Jude Children’s Research Hospital, a total of 38 healthy volunteers were recruited as part of the Phase 1a study in Memphis, Tennessee and Phase Ib study in Brisbane, Australia. The 23 healthy study participants in Memphis received increasing doses of SJ733 as part of the first-in-human study to understand SJ733 dosing, safety profile and metabolism, including absorption reported the sponsor its website.
Based on the results of the 15 Australian volunteers received SJ733 after being infected with malaria to understand the anti-malarial effectiveness of this new drug. The participants later received a curative dose of conventional anti-malarial combination therapy. No significant SJ733 treatment-related side-effects were identified in any of the volunteers.
According to the principal investigator and senior study author Aditya Gaur, MD, with St. Jude Department of Infectious Diseases, “The results support further development of the compound SF733 as a fast-acting component of combination anti-malarial therapy.” He continued, “The drug was well tolerated and well absorbed with a rapid anti-parasitic effect.”
The study was funded by the Global Health Innovative Technology Fund; Medicines for Malaria Venture; and ALSAC, the fundraising and awareness organization of St. Jude.
About St. Jude Children’s Research Hospital
St. Jude Children’s Research Hospital is leading the way the world understands, treats and cures childhood cancer and other life-threatening diseases. It is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children. Treatments developed at St. Jude have helped push the overall childhood cancer survival rate from 20 percent to 80 percent since the hospital opened more than 50 years ago. St. Jude freely shares the breakthroughs it makes, and every child saved at St. Jude means doctors and scientists worldwide can use that knowledge to save thousands more children. Families never receive a bill from St. Jude for treatment, travel, housing and food — because all a family should worry about is helping their child live
Aditya Gaur, MD, St. Jude Department of Infectious Diseases
James McCarthy, MD, MBBS, QIMR Berghofer Medical Research Institute, Australia
Note, there were several other authors—these can be seen at the underlying source.
Call to Action: The St. Jude researchers and collaborators will continue to explore ways to increase and/or extend blood levels of SJ733 to maximize its effectiveness in patients. Interested in learning more? Why not connect with the investigators?