Guest Update by Dr. John E. Niederhuber
Supporting Cancer Drug Development
NCI has an important role to play in the drug development process in the United States. From its expansive clinical trials program to the drug discovery research it performs and funds, the institute believes it has an essential duty to expedite the discovery and development of interventions that will save lives.
Outside of academic and industry circles, very little attention is paid to the multiplicity of steps and tasks required to take a molecule of interest or a therapeutic vaccine concept from initial laboratory investigations to preparation of patient-grade agents ready for testing in appropriate patients. But the public expects - and we want to deliver - more effective and less toxic interventions to prevent and treat cancer, and we are determined to dramatically increase the speed of this process.
Two efforts aimed at doing just that are the Rapid Access to Interventions Development (RAID) and Rapid Access to Preventive Intervention Development (RAPID) programs. These programs don't provide grants to investigators who apply to the programs; instead, they offer important resources and access to expertise and core services that are essential to the early development of a drug, biologic, or vaccine.
RAID, which operates out of NCI's Division of Cancer Treatment and Diagnosis, was launched in 1998 and has provided support and services to more than 100 investigators working on the development of small-molecule drugs and biologics. Of those projects, 13 small-molecule and 11 biologic agents have proceeded to clinical trials.
The services provided through RAID often entail highly specific expertise in areas such as toxicity testing, pharmacodynamics, histopathology, and the production of materials that meet FDA Good Manufacturing Practice (GMP) standards for testing in humans.
For example, Dr. Elizabeth Jaffe and colleagues at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins originally developed a novel therapeutic vaccine for pancreatic cancer through another NCI program called an NCDDG. But it took the RAID program to provide the researchers with GMP-quality material for use in the phase I trial of this vaccine. Of the 14 patients in that trial, 3 are still alive 7 years later, one of Dr. Jaffe's colleagues, Dr. Daniel Laheru, recently reported. Data from the phase II trial demonstrated an astounding 76-percent survival rate after 2 years.
A similar program called RAPID has become an important resource for researchers investigating promising chemopreventive agents. Run by NCI's Division of Cancer Prevention, RAPID, although much smaller in scope than RAID, also offers essential services, such as toxicology studies and the development of GMP-grade material, to successful applicants.
More than 20 projects have received support through RAPID since its launch in 2000. One of the agents developed through this work, Se-methylselenocysteine, has demonstrated potent chemopreventive effects in prostate cancer models and will be tested in a phase I trial at Roswell Park Cancer Institute this year. Several other intriguing agents developed with assistance from RAPID look to be on the same track.
In a testament to the success and popularity of RAID, the National Institutes of Health, under the auspices of the Roadmap initiative, has launched its own pilot RAID program, which is also managed by NCI's Developmental Therapeutics Program.
At the moment, not nearly as many anticancer agents are reaching patients as quickly as we would like. But successful programs like RAID and RAPID are serving as catalysts for a new generation of interventions that will herald a new era of hope in our efforts to thwart this disease.