CCF proudly presented the 2023 Giant Food Next Gen Award, a grant of $100,000, to Yiouli Ktena, M.D. at our November 2023 Gala. In January 2024, Dr. Ktena kindly met with me over Zoom to discuss her current research and the path that got her there. While I understood her work with leukemia and bone marrow transplants to be groundbreaking, I did not fully appreciate how instrumental CCF funding is to her research, advancing treatments and identifying cures with global implications.
Finding her passion
At 17, Dr. Ktena moved from the island of Cyprus to Athens, Greece to begin studying medicine. In 2012, following six years of medical school, Dr. Ktena arrived in the U.S. to pursue a career in pediatrics. She spent her first year at the NIH conducting research, followed by a residency in Pediatrics at the Children’s National Hospital in Washington, DC.
In 2016, Dr. Ktena accepted a fellowship at the Johns Hopkins Sidney Kimmel Cancer Center in Pediatric Hematology and Oncology. During this time, Dr. Ktena found her passion — researching and treating patients with high-risk leukemias and lymphomas using bone marrow transplantation (BMT). Dr. Ktena sees patients who have relapsed (the cancer has returned) or are battling a rare and aggressive leukemia or lymphoma. For the majority of the cases she sees, the patient’s only hope for survival is to undergo a BMT. Dr. Ktena dedicates approximately 20% of her time seeing young oncology patients, who range from infants to young adults. About 80% of her time is in the lab, researching how to make BMTs safer and more effective.
Dr. Ktena kindly explained the process to me as follows:
Leukemias and lymphomas typically present themselves through symptoms such as bone pain, fevers, weight loss, low blood counts, or a very high white blood cell count. These diseases originate from progenitor cells that have “gone bad”, and these progenitor cells typically reside in the bone marrow, the spongy cavity within our bones. Healthy bone marrow makes all of our blood and immune cells, but a leukemic cell population can take over the entire cavity.
The goal of a BMT is to have the donor bone marrow take over the patient’s immune system to make healthy cells necessary for survival and eradicate the cancer. Unlike an organ transplant, a bone marrow transplant does not require surgery, but instead, a patient receives the donated marrow (from a related or unrelated volunteer donor)* intravenously. The process begins with the patient receiving the “conditioning regimen.” This is typically a cycle of chemotherapy and/or radiation and it helps in two ways– to eradicate any residual cancer and to “make space” for the new stem cells and prevent rejection. After the conditioning, the stem cells from the donor are infused into the patient’s vein. The cells find their way into the bone marrow cavity after about 3-4 weeks and start growing. Once the donor cells find the cancer cells, the “graft v. tumor” effect begins to take place, killing off the malignant cells. Unfortunately, there are cases where the graft v. tumor effect is not adequate, and the cancer will return. In other cases, the donor cells can attack the patient’s organs, causing a condition known as graft v. host disease (GVHD). Unfortunately, current therapies for GVHD are less than ideal, require significant immune suppression, exposing one to life-threatening infections, and compromise graft v. tumor effects. Dr. Ktena’s goal is that every child who needs a BMT can receive it in a way that is consistently safe and always effective.
CCF Funding
The CCF research Grant has enabled Dr. Ktena and her lab to continue research to unlock how epigenetics control the donor’s immune response. Epigenetics is the study of how cells control gene activity without changing the DNA sequence itself. Her research has shown that the way the immune system works after a bone marrow transplant relies heavily on epigenetic modifications. To test how this works, the team works with mouse models receiving a BMT. By removing an important epigenetic modifier from a donor’s immune system and implanting it into a patient mouse, Dr. Ktena’s team has demonstrated that epigenetics inform the immune response from the donor; this includes both GVHD and the graft v. tumor phenomenon.
A big concern in this field is finding pathways to treat GVHD that are not harmful to the patient or to the graft v. tumor activity. Essentially, using this approach, Dr. Ktena is seeking a path to identify smarter ways to make transplants safer and more effective by understanding how to guide the donor immune system towards attacking the cancer (graft v. tumor) instead of the patient (GVHD).
Ktena noted that private funding, including the NextGen grant, makes her research possible. Her lab would not be able to conduct the research they are undertaking without this funding.
BMT Advancements
A generation ago, a diagnosis of Acute Lymphoblastic Leukemia (ALL) was nearly a death sentence, and BMTs were extremely dangerous. Bone marrow transplants are not new as far as treatments go; the first ever successful BMT was performed in 1956 on a child with leukemia. Today, ALL is a curable disease and BMT outcomes have dramatically improved, but remain a treatment of last resort. Dr. Ktena is dedicated to making ALL a 100% curable disease and preventing cancer from returning (as relapses, especially after a BMT, are much harder to treat). Ideally, all patients in need of a bone marrow transplant can receive one, without regard to geography, ability to pay, or any other barriers such as donor availability.
CCF is honored to be funding the important research of Dr. Ktena. We know we are making a difference in the landscape of how leukemias are treated. I was personally humbled to speak to Dr. Ktena and learn more about how the CCF funding is making a direct impact on how bone marrow treatments can be managed to ensure better survival outcomes.
We are truly all in this fight together. I left our conversation feeling inspired and motivated to keep at it. We must identify the funds to ensure that treatments and cures are available for, and to, all. No child should have to suffer from pediatric cancer or its treatments.
*You can also be a stem cell donor and save a life! Volunteer donors are identified as a match to the patient through a national registry. If you are between the ages of 18-40, you can join the registry simply with a free cheek swab– a simple process that could save a life. More can be found here: Be the Match. A bone marrow donation can treat and potentially cure over 75 diseases including leukemia, lymphoma, and sickle cell disease.