Alex's Lemonade Stand Foundation for Childhood Cancer

Brenda Weigel

Project Overview

The overarching research goal and objective is to expand our pediatric phase I drug development program to bring new agents from the laboratory into clinical practice for children with cancer. Our specific research objectives with this award are: 

1. To develop a sustainable infrastructure for implementing, monitoring, and completing early-phase trials in pediatric oncology, with specific emphasis on brain tumors, sarcomas, and leukemia.
2. To develop and implement a streamlined process for trial development, monitoring, and completion that will allow a uniform and standardized process for investigator-initiated trials, industry-sponsored trials, and consortia trials to be efficiently completed at the University of Minnesota.
3. To utilize a process for rapid implementation and completion of early phase clinical trials in pediatric oncology partnering with laboratories to build on our understanding of the mechanisms of action of the drug.
4. To utilize this infrastructure as a platform to obtain more resources through industry collaborations and federal funding sources for early phase trials in pediatric oncology.

Currently our program is actively developing agents in three areas: brain tumors, leukemias, and sarcomas. These trials are local investigator-initiated trials that have local, national, and/or pharmaceutical support. These three areas form the pillars of our pediatric drug development program. The agents in development marry the best in scientific development at the UMN, namely cellular and immune-based therapies with clinical trials expertise. This award would allow us to expand these initiatives and develop the infrastructure for sustained support of these types of novel early-phase trials in pediatric oncology.

Project Update 01/01/2012

Submitted by Dr. Brenda Weigel

Our Program Infrastructure Award from Alex’s Lemonade Stand Foundation was sought to expand the breadth and depth of cancer therapy that is available to children and young adults with cancer in the Upper Midwest.

The University of Minnesota (UMN) sees a large number of children each year with recurrent and/or hard-to-treat cancer because:

• It is a regional center for children with all cancers.
• It is the only Children’s Oncology Group Phase I Center in the Northern United States between Chicago and Seattle.
• It is a national and international center for blood and marrow transplantation.

Within our Cancer Center, new therapies for both solid tumors and leukemias must be moved from the laboratory to our patients in a safe yet timely fashion. The growth of our program expands the therapeutic options for children who come to the UMN for novel therapies.

We are using this award to have an impact on moving research from “bench to bedside” more quickly by adding capacity in the following ways:

• Additional staff was hired to process and maintain data for new studies, which means that more research can be undertaken and the results can be translated into useful recommendations faster. Since receiving the award, the addition of staff has allowed us to:
  • Proceed with the planned opening of a second novel trial for patients with brain tumors using a tumor vaccine.
  • Proceed with opening MIBG therapy for patients with neuroblastoma. (MIBG is the name of the chemical used, meta-iodo-benzyl-guanidine.)
• We plan to add more clinical research assistants, who help to design, implement and plan clinical research studies, and who collect and review data obtained from the studies. This is important for early and accurate scientific reporting of results.
• We have added a nurse who is dedicated to the pediatric phase I trial patients. This provides:
  • Comprehensive, knowledgeable care of these patients, and
  • Safe and accurate trial participation.

Currently our program is actively developing agents in three areas: brain tumors, leukemias and sarcomas. These three areas form the pillars of our pediatric drug development program. New treatment agents are researched through local-investigator-initiated trials that have local, national and/or pharmaceutical support. Developing these agents for use in patients combines the best in scientific development at the UMN, namely cellular therapies, immune-based therapies and clinical trials expertise.

The Alex’s Lemonade Stand Foundation award is allowing us to develop and expand the infrastructure for sustained support of these novel early phase trials in pediatric oncology.