Panel Envisions New Translational Program to Hasten Hematologic Cancer Drug Research and Development

BETHESDA, Maryland—A National Cancer Institute (NCI) panel, which explored how to improve survival among leukemia, lymphoma, and myeloma patients and stem the soaring death rates for those with non-Hodgkin’s lymphoma and multiple myeloma, has recommended giving priority to 10 areas of research.

The group also called for the creation of a formal, NCI-supported system to be known as the Cancer Translational Research Allied Consortium (C-TRAC), to speed the discovery and development of new therapies for hematologic cancers.

"The ultimate goal of the C-TRAC will be to shorten drug development time from 5 to 10 years to 2 years through a novel alliance among academic, industry, government, and patients," the report said.

Temporary Halt to New PRGs

NCI has temporarily suspended naming new Progress Review Groups (PRGs). These groups identify scientific research opportunities for a specific cancer, set a list or research priorities, and make recommendations on how best to exploit these opportunities to reduce morbidity and mortality.

The PRG program has provided excellent recommendations in recent years, Robert Wittes, MD, director of NCI’s Division of Cancer Treatment and Diagnosis, told the National Cancer Advisory Board.

"The challenge for us is converting these recommendations into operational plans, implementation strategies, and then assessing what we have done," he said. "Most of our energies have gone into the actual formulation of the recommendations and translating them into things that we are going to do."

Indeed, Dr. Wittes said, the PRGs are much better at making recommendations and putting action plans in place than in analyzing their results. "So we decided to temporarily call a halt to empanelling new PRGs, with the idea of concentrating intensively on what we have already put in motion and making sure that we are on top of it," he said.

NCI also plans to rethink the PRG process itself "to make the initial stage less prolonged and less labor intensive." 

The panel attributed the current long-development time to many barriers that "ultimately are related to the absence of adequate infrastructure for the development of the therapeutics." The C-TRAC concept is not meant to replace the research infrastructure of the pharmaceutical industry, but to create a parallel system for the rapid translation of research findings, the report said.

The 80-page report is the result of a 10-month effort by the 34-member Leukemia, Lymphoma, and Myeloma Progress Review Group (PRG), which included experts from NCI and from academia, industry, and advocacy organizations.

Priority Research Areas

The 10 priority research areas identified by the review group are:

1. Understand the interaction among genotype, immune function, infectious agents, environmental toxins, and lifestyle factors that can lead to hematopoietic malignancy.

2. Identify the basic mechanisms responsible for genome instability, chromosome translocations, and other mutations in hematologic malignancies.

3. Define the relationship between the development of hematologic malignancies and the host biologic environment.

4. Provide molecular characterization of hematologic malignancies, including the characterization of global patterns of genetic and epigenetic alterations and RNA and protein expression, as well as the validation of the molecular targets necessary for the survival, proliferation, and evolution of hematologic malignancies.

5. Further develop research on stem cells, both multi-lineage and single lineage.

6. Develop the required resources to translate "lead" structures and molecules into effective therapeutic agents. Hasten the translation of candidate-validated targets to lead compounds and subsequent clinical trials, and support the development of orphan therapeutic agents and diagnostics, including FDA approval.

7. Foster partnerships between the NCI and academia, advocates, cooperative groups, FDA, and industry to expedite drug development and availability.

8. Determine how to provide accurate, timely, and tailored information to patients to improve medical decision-making, access to clinical trials, quality of care during active treatment and follow-up, and quality of life.

9. Develop education and training programs for certification of physicians and centers for diagnosis, treatment, and clinical trials in hematologic malignancies.

10. Identify and target individuals and populations at high risk for adverse long-term outcomes, to define the biologic basis of identified associations and facilitate the design and testing of intervention and prevention strategies.

"If implemented, the research priorities proposed here will dramatically accelerate progress against these diseases and provide a bold new strategy for rapid translation of basic research into life-saving treatments," the report said.