Project Update June 2013

Submitted by Dr. Brenda Weigel

Our 2011 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 as 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 expansion 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 award is allowing us to develop and expand the infrastructure for sustained support of these novel early phase trials in pediatric oncology.