Developmental & Experimental Therapies

Developmental and Experimental Therapies

The Developmental and Experimental Therapeutics program focuses on translational and clinical application of new therapeutic strategies in early-phase clinical investigation. This program is designed to gauge the safety, feasibility, dose, schedule and early efficacy of new therapeutic strategies during experimental clinical investigation in children, adolescents, and young adults, with both malignant and non-malignant blood disease-related disorders. 

Our areas of expertise include:

• Phase I/II Therapy
•  Bench to Bedside Therapy (Translational)
•  Humoral Immunotherapy
•  Radioimmunotherapy
•  Cellular immunotherapy
•  Stem Cell Transplantation
•  Targeted Cellular Therapy
•  Immunotoxin Therapy 

These specialties yield the opportunity for students, residents, and fellows to pursue scholarly research in this field. Most importantly, this program provides personal and customized medical therapy and care to children, adolescents, and young adults. 

Diseases:

This program is focused on many malignant and non-malignant blood disorders including: 

• acute lymphoblastic leukemia
• acute myelogenous leukemia
• chronic myeloid leukemia
• neuroblastoma
  
• Wilms Tumor
  
• sarcomas
  
• brain tumors
  
• hepatic and germ cell tumors
  
• sickle cell disease
  
• bone marrow failure syndromes
  
• immunodeficiencies
  
• metabolic diseases
  
• hemostasis and thrombosis disorders
  
• histiocytic disorders
  
• vascular malformations
  

Hematotherapy

Clinical services don’t have to feel so…clinical. Our full-service apheresis and transfusion center provides care to patients of all ages, including children at Maria Fareri Children’s Hospital. The newly-renovated, kid-friendly outpatient center has individual patient bays for comfort and privacy, flat-screen televisions and complimentary Wi-Fi. We also provide comprehensive inpatient services. 

We offer consultation and treatment plans for many types of apheresis including:

• Therapeutic plasma exchange
•  Peripheral stem cell collection
•  Red cell exchange
• Extracorporeal photopheresis
• Leukapheresis and plateletpheresis
• Blood transfusions 

The Hematotherapy Center has equipment to transfuse blood components and infuse blood product derivatives and medications, such as:

• Red blood cell or platelet infusions for outpatients with cancer and other diseases
•  Factors, iron and other infusion treatments
•  Therapeutic phlebotomy for conditions such as hemochromatosis or polycythemia vera 

As with all clinical services, physicians are available 24 hours a day.

Unique Features:

• COG Phase II Center
• Several FDA IND Approved Clinical Trials
• Several Phase I Clinical Investigations
• CTEL Laboratory (GMP Quality)
  
• Experienced Clinical Research Staff
  
• Sickle Cell Center Consortium (Lead Center)•Lymphoma Cell Therapy Consortium (Lead Center)
  
• High Risk Leukemia Consortium (Lead Center)
  
• Therapeutic Lymphoma Consortium (Lead Center)
  
• Sarcoma Cell Therapy Consortium (Lead Center)
  
•  Multidisciplinary
  
• Translational Research
•  Personalized and Customized Care

Research and Education Highlights:

• ACGME-Accredited Pediatric Hematology, Oncology, and Stem Cell Transplantation training program
• Student, resident and allied health professionals programs elective/selective
  
• COG Phase II Center
  
• Lead center for multiple Phase I/II clinical consortiums
  
• Pediatric Blood and Marrow Transplantation Consortium Member Institution
  
• Basic Pharmacology Department
  
• Dedicated Clinical Pharmacist
  
• Bench to Bedside Research and Translational Medicine