A Hawaii cancer researcher and mainland colleagues are exploring use of a drug approved for African sleeping sickness to treat neuroblastoma, an aggressive tumor that affects about 650 children annually in the United States.
"It has the advantage, if it works, which it seems to, that it can be put in a clinic much, much faster, and it’s cheaper because we don’t have to go through the whole development process as we have to do with a new project," said Andre Bachmann, a Cancer Research Center of Hawaii associate professor and molecular biologist.
Hawaii is playing a key role in the drug’s development as a cancer fighter, he said.
Bachmann recently received the first Weinman Innovator Award for Translational Research with $50,000 to support his work on cancer in nerve tissue cells. The annual award is part of a $1.7 million Weinman Foundation Fund for Innovation endowment provided to the center by Virginia and Barry Weinman.
Bachmann, in the Cancer Center’s Natural Products and Cancer Biology Program, is working with University of Vermont pediatric oncologist Giselle Sholler on a drug trial for neuroblastoma. The collaboration developed in 2008 after he met the parent of a child with the cancer at a conference in San Diego.
The man’s son had relapsed, and he was looking at the presentations for new drug developments, said Bachmann, who was presenting some of his research on a poster.
"He came to my poster and broke down in tears," said Bachmann. "If kids relapse, it’s basically a death sentence. Nothing works right now."
The Hawaii scientist was presenting his work on a drug using a molecule called Syringolin A to interfere with proteasome, an important part of a cancer cell.
He said he told the father that drug was not at a stage to help his son, but he had a project on another drug which might be available sooner. It involves re-purposing alpha-difluoromethylornithine for neuroblastoma, a tumor that develops from nerve tissue.
The drug was discovered in the 1970s and approved by the Food and Drug Administration to treat African sleeping sickness. It was shown to be safe, with few side effects, Bachmann said.
It targets a protein produced by a parasite responsible for the sleeping sickness. The protein also produces molecules called polyamines that transform normal cells to cancerous cells if present in high amounts, he said.
Bachmann moved ahead on his research after receiving $1.2 million in 2006 from the National Cancer Institute, and "people started to take notice of us because of publications," he said.
Physicians at two Philadelphia hospitals — the Children’s Hospital and St. Jude Children’s Research Hospital — who approached him also started to do research, he said.
"Three of us independently reached the same conclusion: It is a very, very effective drug," he said.
The father he met in San Diego suggested he talk to the pediatric oncologist in Vermont who treats children with neuroblastoma, which led to the drug trial, he said.
He said the FDA approved the trial, and it opened for patients in February at the University of Vermont.
The child of the man he met in San Diego died two weeks before the trial began, Bachmann said.
"But he feels today he jump-started something." Bachmann said. "Because of his son, all of this happened, all of these people came together. … Other kids hopefully will profit from that."
He said a second clinical trial site opened in Florida.
Hawaii has a key role in the drug development, Bachmann said. Tumor samples from patients in the mainland clinical trials are being sent to the Cancer Research Center here for biological analysis before and after they are treated, he said.