Developmental Therapeutics and Pharmacology Program

Program Leaders

Program Mission

The mission of the Developmental Therapeutics and Pharmacology program is two-fold. First, we seek to provide novel, safe, effective therapeutic options to children with high-risk, relapsed, and refractory malignancies. We are carrying out clinical, genomic, and biological research that will enhance care for future generation of children.  We are utilizing a combination of UNC investigator initiated, cooperative group, and pharmaceutical led trials to offer a range of options when precision medicine approaches are indicated, or when standard therapies have failed. Second, we are utilizing the strengths of the UNC School of Pharmacy and the Lineberger Cancer Center to investigate dosing and toxicity of traditional agents, pharmacology of new molecules, and novel drug delivery mechanisms.

We are active members of the following consortiums offering clinical trials to patients with high-risk, relapsed and refractory malignancies:


Developmental Clinical Trials - Currently Open 

DiagnosisSponsorStudy Title with ClinicalTrails.Gov Link
Acute Myeloid Leukemia - Relapsed or Refractory 

COG/Celgene A Phase 2, Multicenter, Singe Arm, Openlabel Study to Evaluate the Activity, Safety and Pharmacokinetics of Lenolidomide (Revlimid) in Pediatric Subjects from 1 to ≤ 18 Years of Age with Relapsed or Refractory Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia - Ph-Like with JAK Pathway Alteration COG/Incyte A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia
Acute Lymphoblastic Leukemia - Relapsed or Refractory

COG Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND# 117467, NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)
Hodgkin Lymphoma - Relapsed or Refractory 

UNC Phase I Study of the Administration of T Lymphocytes Expressing the CD30 Chimeric Antigen Receptor (CAR) for Prevention of Relapse of CD30+ Lymphomas after High Dose Therapy and Autologous Stem Transplantation (ATLAS)
Neuroblastoma - Relapsed or Refractory 

COG A Phase II Randomized Trial of Irinotecan/Temozolomide with Temsirolimus (NSC# 683864, IND# 61010) or Chimeric 14.18 Antibody (ch14.18) (NSC# 623408, IND# 4308) in Children with Refractory, Relapsed or Progressive Neuroblastoma
Osteosarcoma - Relapsed or Refractory 

COG Phase 2 Study of Denosumab (IND# 127430, NSC# 744010), a RANK Ligand Antibody, for Recurrent or Refractory Osteosarcoma
Solid Tumors  - Relapsed or Refractory 

COG/GSK A Phase II Study of Pazopanib (GW786034, NSC# 737754) in Children, Adolescents and Young Adults with Refractory Solid Tumors
Solid Tumors  - Relapsed or Refractory

SUNSHINE A Phase I Trial of Dose Escalation of Metformin in Combination with Vincristine, Irinotecan, and Temozolomide in Children with Relapsed or Refractory Solid Tumors
Solid Tumors  - Relapsed or Refractory with NTRK fusions Loxo Oncology A Phase II Basket Study of the Oral TRK Inhibitor LOXO-101 in Subjects with NTRK Fusion-Positive Tumors
Low Grade Glioma - Relapsed or Refractory COG A Phase II Randomized Trial of Lenalidomide (NSC # 703813, IND # 70116) in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

Developmental Clinical Trials - In Process of Opening 

DiagnosisSponsorStudy Title
Acute lymphoblastic leukemia - Relapsed or Refractory                                 AbbVie A Phase 1 Dose Escalation, Open-Label Study of Venetoclax in Combination with Navitoclax and Chemotherapy in Subjects with Relapsed Acute Lymphoblastic Leukemia
Acute myeloid leukemia - Relapsed or Refractory St. Jude A Phase I And Expansion Cohort Study Of Venetoclax In Combination With Chemotherapy In Pediatric Patients With Refractory Or Relapsed Acute Myeloid Leukemia
Hodgkin Lymphoma - Relapsed or Refractory COG / BMS Risk adapted, response based Phase 2 trial of nivolumab and brentuximab vedotin followed by brentuximab vedotin and bendamustine for pediatric and young adults with relapsed or refractory CD30 positive classic Hodgkin lymphoma
Acute lymphoblastic leukemia - Relapsed or Refractory COG A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND# 133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
Neuroblastoma – Relapsed or Refractory Jubilant An open label, expanded access protocol using 131i-metaiodobenzylguanidine (131I-MIBG) therapy in patients with refractory neuroblastoma, pheochromocytoma, or paraganglioma
Sarcoma – Relapsed or Refractory  SUNSHINE Phase II Study of nab-Paclitaxel in Combination with Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults
Neuroblastoma – Relapsed or Refractory NANT Phase I study of Lorlatinib, an oral small molecule inhibitor of ALK/ROS1, for patients with ALK-driven Relapsed or Refractory Neuroblastoma
Neuroblastoma – Relapsed or Refractory NANT NANT Precision Trial
Neuroblastoma – Relapsed or Refractory NANT Phase I Study of SF1126 for Patients with Relapsed or Refractory Neuroblastoma
Neuroblastoma – Relapsed or Refractory NANT A Phase I Study of Sorafenib and Cyclophosphamide/Topotecan in Patients with Relapsed and Refractory Neuroblastoma


Recent publications; for additional publications from Drs. Thompson or Alexander, please click here.

  1. Opportunities for expanding clinical trial enrollment for relapsed and refractory pediatric acute myeloid leukemia in the United States and Canada. Alexander, T.B., Bhakta, N., Kolb, E.A. & Rubnitz, J.E. (2017). Pediatric Blood & Cancer. 
  2. Adverse effects of pegaspargase in pediatric patients receiving doses greater than 3,750 IU. Lebovic, R., Pearce, N., Lacey, L., Xenakis, J., Faircloth, C.B., & Thompson, P. (2017). Pediatric Blood & Cancer. 
  3. Vincristine toxicity with co-administration of fluconazole during induction therapy for pediatric acute lymphoblastic leukemia. Smitherman, A.B., Faircloth, C.B., Deal, A., Troy, M., & Gold, S.H. (2017). Pediatric Blood & Cancer. 
  4. Phase I Study of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination With Fludarabine and Cytarabine, in Pediatric Relapsed or Refractory Acute Leukemia. Alexander T.B., Lacayo N.J., Choi J.K., Ribeiro R.C., Pui C.H., Rubnitz J.E.. (2016) J Clin Oncol