Targeting Cancer Stem Cells By Metformin To Treat Malignant Neuroblastoma


Reference #: 01167

The University of South Carolina is offering licensing opportunities for a metformin-based drug formulation (alone or in combination with other existing drugs) to inhibit cancer metastasis and treat pediatric cancers such as neuroblastoma.

Invention Description:

The subject invention utilizes metformin to induce apoptotic cell death in neuroblastoma stem cells that cause tumor growth. It also inhibits the self-renewal and differentiation potential of new neuroblastoma stem cells to prevent the further occurrence and progression.

Advantages and Benefits:

Metformin is cost effective, safe, and shows no harmful side effects in patients compared to existing anti-cancer drugs, which are often toxic and have considerable side effects. Treating pediatric cancers such as neuroblastoma using metformin-based drugs alone or in combination with other drugs in a metformin-based formulation is a novel approach to stem cell therapy that eliminates the chance of re-occurrence as well as regrowth of tumors in patients.

Potential Applications:

  • Treatment of neuroblastoma and other pediatric cancers


The majority of cancer therapies available are generally developed for adults and their efficacy in treating childhood cancers is unknown. From 1948 to 2003, regulators approved 120 new cancer therapies. However, only 15 contained information about pediatric use. Pediatric cancers differ significantly from adult cancers. For example, childhood cancers are found in different areas of the body than adult cancers. Additionally, unlike adult cancers, childhood cancers are not strongly linked to environmental or lifestyle risk factors but depend more on genetic factors. Although chemotherapy and radiation therapy are used to treat pediatric cancer, these methods may cause long-term side effects, so children who have had cancer will need careful attention for the rest of their lives.

Thus, there is urgent need to develop safer drugs and therapeutic approaches specific for the treatment of childhood cancers. Among various types of pediatric cancers, neuroblastoma is the most common cancer in babies in younger than 12 months and the second most common tumor present in children. Neuroblastoma is a malignant tumor that occurs in sympathetic ganglia and adrenal medulla and metastasizes to other locations including lungs, bones, the chest, abdomen, etc. Despite recent advances in systemic therapies including chemotherapy, radiation, and surgery, a high proportion of patients with metastases suffer from poor outcomes and relapse.


In vitro and in vivo preclinical mouse model studies confirmed that metformin reduces the growth of tumors generated from neuroblastoma stem cells enriched-spheroids. These observations also suggest metformin inhibited the self-renewable and differentiation potential of neuroblastoma stem cells, which are the main characteristics of stem cells.


Patent Information:
For Information, Contact:
Technology Commercialization
University of South Carolina
Ambrish Kumar
Ugra Singh
Donald Dipette
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