My clinical specialty is hematologic malignancies (leukemia/lymphoma), especially acute myeloid leukemia. I was drawn to pediatrics because of the opportunity to form long-lived therapeutic relationships with my patients and their families.

I pursued oncology in particular because childhood cancer presents an incredibly emotional challenge to the family and an intellectual one for the healthcare team. To me, oncology presented the chance to take care of a child and their family in a way that prioritized empathy and understanding but also incorporated cutting-edge scientific knowledge.

Langerhans cell histiocytosis (LCH) is a rare childhood disorder that biologically behaves much like a cancer. I’m studying how the hematopoietic (blood-forming) system is involved in severe cases and how to modify or combine targeted treatments to cure these children without using traditional cytotoxic chemotherapy. LCH is not particularly well-understood. We have only recently shown that severe forms of the disorder involve blood-forming cells. I hope our research will determine better ways to use targeted therapy to treat, and potentially eradicate, this disease.

I also study infant mixed-lineage leukemia (MLL- rearranged) leukemias. In particular, our lab has found that these leukemias depend on an RNA binding protein (MBNL1), which regulates alternative splicing of essential genes. We’re trying to better understand mechanistically what happens when MBNL1 is blocked, while developing potential drugs to achieve this effect.

We see infant leukemias, which biologically resemble another leukemia that sometimes occurs spontaneously with some regularity. Infant leukemia is particularly difficult to treat. It often returns after chemotherapy. Bone marrow transplant or immunotherapy are frequently ineffective. Patients often must spend prolonged periods of time in the hospital. We are trying to find a new way to target the core programs essential for this leukemia to survive.

I received a St. Baldrick's Foundation Scholar Award — a career development grant from the St. Baldrick's Foundation. This award provides me with protected research time and helps me continue to focus on my research.

MD: Emory University School of Medicine, Atlanta, GA, 2010.

Residency: Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, 2013.

Fellowship: Hematology/Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, 2016.


Leukemia/lymphoma - acute myeloid leukemia; Langerhans cell histiocytosis

Services and Specialties

Cancer and Blood Diseases, Leukemia


Biology of acute myeloid leukemia & infant leukemia; alternative splicing and RNA processing; MAP kinase signaling; cancer genomics; disease modeling with patient-derived xenografts

Research Areas

Cancer and Blood Diseases, Oncology

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MBNL1 regulates essential alternative RNA splicing patterns in MLL-rearranged leukemia. Itskovich, SS; Gurunathan, A; Clark, J; Burwinkel, M; Wunderlich, M; Berger, MR; Kulkarni, A; Chetal, K; Venkatasubramanian, M; Salomonis, N; et al. Nature Communications. 2020; 11:2369.


High-risk LCH in infants is serially transplantable in a xenograft model but responds durably to targeted therapy. Lee, LH; Krupski, C; Clark, J; Wunderlich, M; Lorsbach, RB; Grimley, MS; Burwinkel, M; Nelson, A; Kumar, AR. Blood Advances. 2020; 4:717-727.


Real-time genomic profiling of histiocytoses identifies early-kinase domain BRAF alterations while improving treatment outcomes. Lee, LH; Gasilina, A; Roychoudhury, J; McCormack, FX; Pressey, J; Grimley, MS; Lorsbach, R; Ali, S; Bailey, M; Stephens, P; et al. JCI insight. 2017; 2:e89473.

LAMP-5 is an essential inflammatory-signaling regulator and novel immunotherapy target for mixed lineage leukemia-rearranged acute leukemia. Gracia-Maldonado, G; Clark, J; Burwinkel, M; Greenslade, B; Wunderlich, M; Salomonis, N; Leone, D; Gatti, E; Pierre, P; Kumar, AR; et al. Haematologica: the hematology journal. 2022; 107:803-815.

TRAF6 functions as a tumor suppressor in myeloid malignancies by directly targeting MYC oncogenic activity. Muto, T; Guillamot, M; Yeung, J; Fang, J; Bennett, J; Nadorp, B; Lasry, A; Redondo, LZ; Choi, K; Gong, Y; et al. Cell Stem Cell. 2022; 29:298-314.e9.

An In Vivo CRISPR Screening Platform for Prioritizing Therapeutic Targets in AML. Lin, S; Larrue, C; Scheidegger, NK; Seong, BK A; Dharia, NV; Kuljanin, M; Wechsler, CS; Kugener, G; Robichaud, AL; Conway, AS; et al. Cancer Discovery. 2022; 12:432-449.

Treatment of posttransplant lymphoproliferative disorder with poor prognostic features in children and young adults: Short-course EPOCH regimens are safe and effective. Rubinstein, JD; Shah, R; Breese, EH; Burns, KC; Mangino, JL; Norris, RE; Lee, L; Mizukawa, B; O'Brien, MM; Phillips, CL; et al. Pediatric Blood and Cancer. 2021; 68:e29126.

Methylation profiling of hypomethylating agent response and treatment failure in pediatric and young adult MDS/AML. Jones, LM; O'Brien, MM; Breese, EH; Absalon, M; Burns, KC; Grimley, M; Mizukawa, B; Lee, LH; Perentesis, JP; Phillips, CL. Journal of Clinical Oncology. 2020; 38:e22502.

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