Young brain cancer patient inspires drug discovery project


  • Carrie Printz

Violet, a little girl who died from inoperable brain cancer when she was 11 years old, is the inspiration for a drug discovery project recently launched by the Fred Hutchinson Cancer Research Center in Seattle, Washington.

“Project Violet” is using crowdfunding to gain community support for developing a new class of anticancer compounds from natural sources such as violets, scorpions, and sunflowers to attack cancer cells without harming healthy cells. The goal of these targeted treatments is to spare patients the harmful side effects of chemotherapy.

The group is involving “citizen scientists” from around the world by enabling them to “adopt” a drug candidate for $100. Participants can choose from 1 of 6 organisms, including violet, scorpion, locust, sunflower, spider, or human, and receive an image of their drug molecule. Investigators will test the drug candidates and, if one fails, it can be replaced by a new one.

Before Violet died last year, she and her family requested a rapid autopsy to help create important research tools from her brain tumor in an effort to help others, according to her physician, pediatric brain specialist Jim Olson, MD, PhD, of Fred Hutchinson and Seattle Children's Hospital.

Dr. Olson developed “tumor paint,” a molecule derived from scorpion venom that safely travels through the body and causes cancer cells to “light up” so that surgeons can visually distinguish them from normal tissue. This development led him and his colleagues to explore substances produced by other plants and animals. Sunflower petals, for example, are not eaten by bugs because they make a compound that protects them from insects. They also found other examples of drug-like substances made by potatoes, spiders, cone snails, sea slugs, and violets.

Inspired by these natural properties, Dr. Olson and other researchers developed a new class of drugs known as optimized peptides, or “optides,” which are potentially less expensive and more effective than many nextgeneration therapies. These tiny molecules can bind to certain types of cancer cells and also can be attached to chemotherapy drugs, thus helping them to spare healthy cells. Specifically, investigators are attempting to target brain cancer, melanoma, breast cancer, and tumors of the neck and throat. The drugs also may offer treatments for epilepsy, depression, anxiety, and other central nervous system disorders.

Although using molecular scaffolds from nature is not entirely new, many previous efforts have been unsuccessful because there are many barriers to making thousands of optides inexpensively and quickly enough. Dr. Olson's team has overcome some of these barriers with a new optide production system that rapidly synthesizes optide variants. They are able to make 12,000 peptide drug candidates each month and are working to speed up that process.