Clinical Trials
Relapsed Acute Lymphoblastic Leukemia
The Pediatric Cancer Foundation launched a clinical trial in June 2011 focusing on relapsed acute lymphoblastic leukemia (ALL). ALL is the most common cancer in children. The Barredo lab at the University of Miami discovered a remarkable target in ALL known as AMPK. AMPK is a central switch in cells that regulates cell growth and division. In ALL, AMPK is turned off allowing the leukemia cell to grow out of control. This makes turning AMPK back on in ALL an attractive idea for treatment.
Metformin, commonly known as GlucophageTM, is a medication frequently used to treat Type II diabetes and also studied in humans to turn on AMPK. The combination of metformin and chemotherapy is a new way to target resistant leukemia cells. This trial will determine if combining metformin with chemotherapy is safe and effective at treating leukemia.
Eighteen children will be treated on this FDA approved clinical trial.
For more information regarding this trial, please visit http://clinicaltrials.gov/ct2/show/NCT01324180?term=relapsed+leukemia+and+miami&rank=1.
VIT & Metformin in Relapsed or Refractory Solid Tumors
The Pediatric Cancer Foundation opened a second clinical trial in the fall of 2012 focused on relapsed or refractory solid tumors. Solid tumors account for 60% of all childhood malignancies. Unlike other childhood cancers, minimal improvement in survival has been seen in children with solid tumors over the past 20 years. These disappointing results have prompted PCF to find new agents in the fight against this disease.
Metformin is an oral anti-diabetes medication. Recent data from research experiments and analyses of previous data suggest that Metformin has anti-cancer activity. The drugs vincristine, irinotecan and temozolomide, also known as VIT, are a combination of chemotherapy that have different ways of working and different side effects. This combination has shown to be safe and well tolerated in children with solid tumors. This trial will evaluate the tolerability and safety of increasing doses of Metformin in combination with vincristine, irinotecan and temozolomide in children with solid tumors.
Twenty-five children will be treated on this FDA approved clinical trial.
For more information regarding this trial, please visit, http://clinicaltrials.gov/ct2/show/NCT01528046?term=pediatric+cancer+foundation&rank=1.
Topotecan & Sorafenib in Relapsed or Refractory Solid Tumors
The Pediatric Cancer Foundation launched a third clinical trial in the fall of 2012 focused on relapsed or refractory solid tumors. Solid tumors account for 60% of all childhood malignancies. Unlike other childhood cancers, minimal improvement in survival has been seen in children with solid tumors over the past twenty years. These disappointing results have prompted PCF to find new agents in the fight against this disease.
Topotecan is a chemotherapy drug that has been used safely for over a decade in children with anti-cancer activity in leukemias, solid tumors, and brain tumors. Sorafenib is from a newer class of targeted chemotherapy drugs also with safety data in children when used alone and activity in multiple pediatric research models. The combination of these two agents was tested along with numerous others in sarcoma models and found to work well together to kill cancer cells. This trial will establish a safe dose, determine toxicities, and evaluate the effect of these agents on pediatric solid tumors.
Twelve to Twenty-four children will be enrolled in this clinical trial.
For more information regarding this trial, please visit, http://clinicaltrials.gov/ct2/show/NCT01683149?term=Topotecan+reed&rank=1.
Pediatric Total Cancer Care Protocol
In the beginning of 2013, the Pediatric Cancer Foundation launched a new trial focusing on tissue and blood collection to further personalized medicine for children with cancer. When cancer is suspected, a patient undergoes a biopsy to establish the diagnosis. For some cancers, the therapy is entirely based on the pathologist’s interpretation of this tissue. Other cancers have molecular testing done to either confirm the diagnosis or choose a type of therapy. Sometimes the results of testing have a significant impact on the patient’s choice of therapy. The goal of our latest protocol is to better understand the causes of cancer and perhaps identify additional or new therapies for individual patients. We hope to maximize the benefit and toxicity of these new therapies. This concept is known as personalized medicine.
There are many technologies currently being utilized to better identify the genetic underpinnings of cancer. The past two decades have been spent investigating doses and schedules of traditional chemotherapies without large changes in outcome for these patients. Often, there is also not a clear path towards new targeted therapies. This proposal will allow for a centralized collection of tumor tissue for molecular testing. The results may lead to a better understanding of a child’s malignancy or may lead to a better matching of therapy to the patient. Our hope is that this may guide the patient to a clinical trial from which they’d be predicted to derive the most benefit.
Contact Information
John M. Goldberg, MD
Principal Investigator, Relapsed Acute Lymphoblastic Leukemia Trial
Assistant Professor for Clinical Pediatrics
Director, Pediatric Oncology Early Phase Clinical Trials Program
University of Miami Sylvester Comprehensive Cancer Center
University of Miami Miller School of Medicine
Attending Physician, Holtz Children’s Hospital
jgoldberg2@med.miami.edu
305-243-2795
Jonathan Gill, MD
Principal Investigator, VIT & Metformin in Relapsed/Refractory Solid Tumors Trial
Assistant Professor of Pediatrics
Division of Pediatric Hematology/Oncology
Attending Physician, Montefiore Medical Center
jgill@montefiore.org
718-741-2339
Damon Reed, MD
Principal Investigator, Topotecan & Sorafenib in Relapsed/Refractory Solid Tumors Trial
Assistant Professor of Pediatrics
Assistant Member of the Department of Oncologic Sciences
Medical Director of the Sarcoma Program, Moffitt Cancer Center
Attending Physician, All Children’s Hospital
damon.reed@moffitt.org
813-745-2297