Mosquitoes are the deadliest animal on Earth. The tiny flying insects are responsible for more disease, death, and resulting economic loss than any other animal on the planet. One of the more common diseases carried by mosquitoes is Malaria, a parasitic blood infection that is one of the leading causes of death for children worldwide.

"Malaria is a global disease and nearly half of the world's population is at risk. In Africa, $12 billion are lost each year in gross domestic product due to the heavy toll malaria inflicts on regional economic systems. In 2019 alone, we saw 230 million cases and 400,000 deaths, 70 percent of which were deaths of children under the age of five," said Dr. Jared Hammill, an assistant research professor at the University of Kentucky College of Pharmacy, Department of Pharmaceutical Sciences.

At UK, Dr. Hammill works alongside Dr. R. Kip Guy, the Dean of the College of Pharmacy, on a new anti-malarial medicine called SJ733. After Dr. Hammill completed his post-doctoral fellowship with Dr. Guy at St. Jude Children's Research Hospital in Memphis (where SJ733 was discovered), they both moved to Kentucky in 2016 and have been able to see the medicine go from the bench to the bedside, successfully treating patients.

"As I was finishing graduate school, my grandfather was diagnosed with lung cancer, and I watched him pass away with no satisfactory treatments. It was that life event that made me dedicate the rest of my life to advancing cures for unmet clinical needs," said Dr. Hammill, whose focus is oncology and infectious disease.

In the case of SJ733, that lifelong goal is coming to fruition. SJ733 has successfully completed phase 1a and 1b clinical trials, finding that the medicine was safe and efficacious in healthy volunteers. Their phase 2a trial with malaria patients has begun in Peru with promising results.

"Everybody that we've given the drug to, to date, has had no serious adverse events, no life-threatening events. In fact, they have only had very minor or moderate negative side effects. Just a little bit of a headache or a little nausea," Dr. Hammill said.

"The phase 2a trial is just getting started, and we have a long road ahead, but so far SJ733 has been really well tolerated in real patients and no drug-related adverse events have been reported. SJ733 is really fast-acting. We've now finished treatment on four patients in Peru and all four of those patients were completely cured of their parasitemia in one day," he added. "All the modeling that we've done, all the science that we did in the lab accurately predicted what we're seeing now, and that has just been so exciting."

While there are several popular anti-malarial drugs in the market, one of the biggest concerns with the disease is parasites developing resistance to the available treatments.

"One of the major challenges that's been thematic in malaria treatment since we started generating drugs in the 1940s to combat the devastation of the disease is resistance," Dr. Hammill said. "What makes our drug special is it targets a new mechanism of action for which there's no resistance in the field."

According to Dr. Hammill, their drug targets a parasite proton-sodium antiporter (PfATP4), specifically causing a change in the patient's parasite-infected red blood cells while leaving their uninfected red blood cell untouched. These changes to the infected red blood cells cause the patient's body to recognize and clear only the infected cells. Taking this approach to combat malaria, the drug has shown to be safe, fast-acting (clearing parasites from patients in about a day), and in the lab has been active against drug-resistant parasite strains.

Now the team is focusing on additional clinic trials to determine whether their medicine is safe for pregnant women, effective for patients with severe malaria, and useful in the prevention of malaria (possibly aimed toward the travel industry or the military). Throughout the duration of their work on SJ733, they have raised approximately $15 million in grant funding and are actively applying for additional grants.

For Dr. Hammill, the thrill of seeing his work help people is unlike any other.

"This is the first program I've ever gotten to be a part of that went into humans. So, it is the first time I've been able to say something that I worked on actually cured another human of their disease. That was just a really powerful moment for me, it was the culmination of everything I set out to do when I decided that I wanted to pursue human health," he said.

"All the long nights, all the hard work, everything that we work for is to try to improve human health. To do that for someone with a real disease in a real setting, and to do it in a way that was safe, effective, and rapid, it was just the best. There's nothing better."

By: Erin Shea

Launch Blue nurtures promising startup founders and university innovators through intensive accelerator and incubator programs. Its funding partners are the University of Kentucky: Office of Technology Commercialization, KY Innovation, the U.S. Economic Development Administration, and the National Science Foundation.