Children living with sickle cell disease and other blood disorders face lifelong challenges—but that’s changing thanks to research at Children’s Healthcare of Atlanta and Emory University. At the Aflac Cancer and Blood Disorders Center, Dr. Francesca Vinchi is studying how iron and heme (a molecule that contains iron) affect the body and is looking for new therapies that could reduce complications and improve quality of life for kids with sickle cell disease in Georgia and beyond.

A Lifelong Passion for Discovery

At five years old, Dr. Vinchi knew she wanted to be a scientist. “It was my dream to become a scientist,” she said. “I have always been driven to do this. My entire trajectory and career development focused on becoming a scientist.”

It was a dream inspired by her parents, though not directly. Her father worked as an engineer and her mother as an artist. “Being a scientist allows me tap into both sides,” she said. “It gives me a chance to be rational and logical, like an engineer, and creative like an artist.”

Although she knew she wanted to be in the lab, she wasn’t as clear about what she wanted to study. That came later, during her second year at university. “Almost by chance, I attended a lab focused on mechanisms related to heme and sickle cell disease,” she said. “I kept attending because it sparked my curiosity, and that ended up being my path.”

That path led to an international research career, including postdoctoral training at the University Hospital and European Molecular Biology Laboratory in Heidelberg, Germany, and directing the Iron Research Laboratory at the New York Blood Center. She came to Children’s and Emory in 2025, drawn by the unique environment for research that exists here.

Today, as a researcher within Aflac Cancer and Blood Disorders Center, a Georgia Research Alliance Distinguished Investigator in Sickle Cell Disease, and an Associate Professor in the Department of Pediatric Hematology/Oncology of Emory University School of Medicine, Dr. Vinchi works alongside clinicians to translate laboratory discoveries into better treatments for children. “I am interested in understanding the mechanisms at the root of disease, and in the idea of using that knowledge to develop new drugs so we can better treat it,” she said.

Understanding How Iron and Heme Affect Children’s Health

Even today, Dr. Vinchi still works toward answering the research question she first asked in that lab during her second year of university. It’s a question that has allowed her to develop her entire career: How does heme contribute to disease severity?

Iron and heme are essential to building hemoglobin, the protein that carries oxygen in red blood cells. But when levels are abnormal—as in sickle cell disease and other blood disorders—they can harm cells and trigger inflammation.

Working closely with clinicians and industry partners, her translational lab evaluates therapies designed to reduce iron- and heme-related damage and improve quality of life for children with blood disorders.

Dr. Vinchi’s research has shown that free heme not only damages cells but also interferes with their function, worsening organ injury. Her team demonstrated that in some patients, hemopexin, a protein that binds free heme, can limit toxic effects and may help treat sickle cell disease. Hemopexin is an investigational therapy that has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of sickle cell disease.

She also identified a link between iron overload and the severity of myelodysplastic syndromes (MDS). Her studies found that vamifeport, a drug that lowers iron levels, can improve anemia and reduce symptoms in preclinical models—highlighting its potential for treating conditions such as MDS, beta thalassemia and sickle cell disease. Vamifeport has also received orphan drug designation for the treatment of sickle cell disease.

Her lab continues to explore how iron and heme influence sickle cell disease, thalassemia and other blood disorders—and how personalized treatments can reduce complications and improve long-term health for children. “The more we learn about the mechanisms, the more we can improve treatments,” she said. “From there, we can create personalized medicine.”

Advancing Sickle Cell Research in Georgia

For many researchers, Georgia is ground zero when it comes to studying sickle cell disease. The state has one of the nation’s largest populations of people living with sickle cell disease, and the Aflac Cancer and Blood Disorders Center is home to the largest pediatric sickle cell disease program in the country.

In 2021, Gov. Brian Kemp announced a Georgia Research Alliance initiative to advance exploration into sickle cell disease, including new treatments and therapies. Georgia’s Solve Sickle Cell Initiative (SSCI) aims to leverage the state’s considerable strengths in university research and healthcare and break new ground in knowledge, treatment and cures. Children’s, Emory and Morehouse School of Medicine are part of the SSCI and have committed to adding mindpower by recruiting top researchers like Dr. Vinchi, lab infrastructure, and clinical and market translation.

“SSCI is a unique opportunity to focus on sickle cell disease, and Georgia is the place where we see so many people impacted,” she said. “Finding better treatment is important to people in this state. Here, patients and researchers are surrounded by clinicians who are working directly with children who have sickle cell disease. It is easy to see the importance of the work.”

While Dr. Vinchi understands there is still much to learn about sickle cell disease, she is inspired by the leaps she’s seen in the time she has worked as a researcher.

“I’ve had the privilege to grow in the science,” she said. “In my first lab, we started to work on protein that is a scavenger for heme. Now, 23 years later, we are thinking about that protein as a potential treatment. It is inspiring to see how far we’ve come, and exciting to think about the discoveries to be made ahead.”