CHOP doctors named 'Disruptors of the Year' for groundbreaking sickle cell gene therapy
CHOP doctors Alexis Thompson and Stephan Grupp are named "Disruptors of the Year" in the second annual Philadelphia Citizen of the Year Awards.
Dr. Thompson and Dr. Grupp are recognized for being in the forefront of innovating gene therapies for patients suffering from sickle cell disease.
The gene therapy is specifically designed to treat the underlying cause of sickle cell by using a patient's own blood stem cells.
PHILADELPHIA - The Philadelphia Citizen of the Year Awards celebrate all-star citizens who inspire Philadelphians make change and pursue solution-focused action.
Among the honorees are two doctors from the Children's Hospital of Philadelphia: Dr. Alexis Thompson and Dr. Stephan Grupp who have been named Disruptors of the Year.
What we know
Dr. Thompson is the Chief of the Division of Hematology at CHOP and Dr. Grupp is the Director of Cell Therapy and Transplant at CHOP.
Both Thompson and Grupp have been in the forefront of innovative research and treatment for sickle cell disease. In 2023, the FDA approved two gene therapies after rigorous clinical trials that took place at CHOP and other sites.
"To see what people have been working on in the laboratory for so long, and to actually see it finally reach a patient and for a patient to have a great outcome, it's just everything we could possibly hope for," said Dr. Thompson. "It's a rigorous process up front, but long term we hope this allows them to move through the rest of their lives with less of the burden of sickle cell disease. The need for hospitalization, the need for continuous medications that all of those things are in the past for them, and that's what we're really hoping more and more patients will enjoy."
Sickle cell disease is an inherited blood disorder that can be debilitating and disproportionately affects children of African descent. The disease can cause intense pain due to deformed blood cells that can cause blockages in blood vessels.
The gene therapy is specifically designed to treat the underlying cause of sickle cell disease, according to Dr. Grupp.
"Sickle cell disease is a disease which traditionally has been very difficult to treat. Even though we know exactly what's wrong genetically that causes the disease, it hasn't led to anything in terms of better treatment for these patients. This is a group of patients who are fairly underserved by the medical system," said Dr. Grupp. "We can cure their disease by essentially using gene therapy. What we're doing is, collecting cells from patients. We're correcting what's wrong with the cells that produces the sickle cedll disease. We're putting those cells back into patients, and then once those cells grow within the patients, they now produce normal red blood cells which are the cause of the sickle cell disease."
Tema Oladeji started receiving care at CHOP as a toddler for sickle cell disease and related complications.
In 2019, her doctor recommended a clinical trial gene therapy transplant. Now in 2025, Oladeji is a first-year college student living a full life surrounded by family and friends.
"I was really liberated and now I'm not tied to sickle cell," said Oladeji. "The fact that now kids are growing up are going to have that hope, they can get gene therapy treatment, and they don't have to live a life with sickle cell for the rest of their life is crazy and it's amazing. I'm so happy that future generations have amazing doctors like Dr. Thompson and Dr. Grupp to help them through this."
"There are so many times when disruption is not necessarily a welcome thing. I think in this instance being able to change the arc of sickle cell disease, to be able to take science and make it work for patients has been a welcome disruption from my point of view and I really, truly am grateful for the honor," said Dr. Thompson.
"We like to think that we've disrupted the care of sickle cell. I think it's really important to understand while we're at the beginning of this the big step, we need to take the time now to scale it out. Make it available to more patients," said Dr. Grupp. "This is a real opportunity to help a group of patients that the medical system has not helped as well as it should have and to use the most cutting-edge technology."
What's next
The Citizen of the Year Awards is on Tuesday February 25. Eagles quarterback Jalen Hurts and former Governor Ed Rendell will also be honored at the event.
The Source
The information in this story is from CHOP doctors Alexis Thompson and Stephan Grupp and their patients.
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