Introduction

Unlike the commonly held belief of 30 or 40 years ago, we know today that there are more than 100 distinct types of cancer. We are also learning that several of these have subtypes with unique molecular characteristics that influence how they develop and progress and how they can be effectively prevented, detected, and treated.

While the majority of the research supported by NCI has broad application across various types of cancer, we also have in place plans for ensuring coverage of essential research directed toward specific types of cancer. This disease-specific focus is absolutely critical to achieving our Challenge Goal and is bolstered by recent gains in understanding the molecular features both shared by and unique to specific cancers. Ultimately, we look for ways to apply all of our research results to the development and delivery of disease-specific interventions.

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NCI's Planning and Evaluation Process for Disease-Specific Research

NCI's planning and evaluation process for disease-specific research involves three distinct phases:

• Developing recommendations through external Progress Review Groups (PRGs).
• Planning for and implementing initiatives that respond to these recommendations, with advice from internal implementation working groups.
• Reporting on progress.

This comprehensive and integrated approach to planning and evaluation helps us demonstrate progress toward our Challenge Goal and the wise use of resources to the scientific community and the public. Through these and other crosscutting efforts, NCI has established a framework for accountability that is consistent with the President's Management Agenda (PMA) and the congressionally mandated Government Performance and Results Act (GPRA).

During the past 7 years, NCI has called upon a number of PRGs, each composed of prominent members of the scientific, clinical, industry, and advocacy communities, to assess the state of the science and recommend future research-related priorities for specific types or groups of related cancers. Go to prg.nci.nih.gov for additional information about and reports from each of these PRGs.

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Impacts of PRGs on Disease-Specific Research Agendas

PRG recommendations are central to sustaining the best possible science and making the fastest advances against specific cancers, and have proven to be a valuable asset to NCI strategic planning. For example:

• PRGs have helped shape research directions. PRG reports have had substantial reach. NCI divisions and programs use them in planning and developing new initiatives. Extramural scientists cite them in their grant applications. They are cited regularly in Congressional documents. And they are used by advocates, researchers, and members of professional societies to attract funding from other sources.



• PRGs have led to an expanded understanding and awareness of opportunities related to cancer research. PRG members and roundtable participants report that new collaborative research relationships have been formed as a result of their interactions with a diverse, multidisciplinary group of people involved in all aspects of cancer research.



• PRGs have led to the development of new information tools. Developed in response to needs identified by the groups, new tools are now available to aid both NCI staff and the community in learning about and responding to needs and opportunities for cancer research. For example, the Common Scientific Outline is a classification system that provides a consistent approach to comparing and assessing cancer research supported by different funding organizations. The Cancer Research Initiatives Website provides user-friendly access to a database of selected NCI research funding opportunities that can be searched by type of research or type of cancer. And the Cancer Research Portfolio Website provides a structure for searching, organizing, and analyzing NCI-supported research by type of cancer and/or type of research.



• PRGs have provided new opportunities for interaction between NCI and the extramural community. Participants have found the PRG venue a very effective way to gain input from the wider community and for individuals to influence both NCI priority setting and overall disease-specific priority setting.

The NCI PRG process has been highly successful as a model for gaining outside input and feedback focused on opportunities, needs, and gaps in research and fostering greater understanding of common issues across diseases. Similar approaches have now been adopted by other research funders including:

• National Institute of Neurological Disorders and Stroke (Stroke Research)
• National Institute of Diabetes and Digestive and Kidney Diseases (Bladder Disease Research)
• National Institute on Aging (Exploring the Role of Cancer Centers for Integrating Aging and Cancer Research)
• Department of Health and Human Services (Cancer Health Disparities)
• National Cancer Research Institute of the United Kingdom

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Collaborations for Assessing PRG Recommendations

NCI recognizes that its efforts represent only part of the global cancer research enterprise and works to engage other organizations that fund cancer research in addressing PRG recommendations and collaborating on relevant initiatives in order to leverage and optimize collective research efforts. To this end, NCI is bringing together groups of cancer funders to focus on a research. The objectives of these meetings are to:

• Share and discuss information on ongoing and proposed initiatives related to specific cancers in order to identify common areas of emphasis, eliminate duplication, and address gaps in critical research areas.
• Establish consensus on where the most serious gaps are and identify organizations that will be able to fill them.
• Identify opportunities for collaboration or coordination in areas of mutual interest.
• Establish a plan of action that includes mechanisms for continued communication and reporting on progress.
• Optimize deployment of resources among participating groups.

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The Sarcoma Progress Review Group

In 2003, NCI launched a PRG to focus on sarcomas, a general group of less common cancers in which the cancer cells arise from or resemble normal cells in the body known as "connective tissues." Normal connective tissues include, fat, blood vessels, nerves, bones, muscles, deep skin tissues, and cartilage.

Sarcomas in general are rare tumors, accounting for less than 5 percent of adult malignancies and less than 20 percent of pediatric cancers. Nearly half of all sarcoma patients die of the disease.

The most common forms of sarcoma include:

• Soft tissue sarcomas that originate in connective tissues, fat, blood vessels, nerves, joints, muscles, cartilage, and deep dermal tissue. They occur more often in adults.
• Osteosarcoma, which is the most common type of bone cancer. In children, it occurs most commonly in the bones around the knee. Although osteosarcoma occurs most often in adolescents and young adults, about 10 percent of cases develop in people in their 60s and 70s.
• Kaposi's sarcoma (KS), which usually develops in the skin or in the lining of the mouth, nose, or eye. In the last 20 years, the vast majority of KS cases have developed in association with human immunodeficiency virus (HIV) infection and Acquired Immunodeficiency Syndrome (AIDS).

As with past efforts, the Sarcoma PRG is soliciting extensive input from the research and advocacy communities and is examining NCI's research portfolio as well as research funded by other institutes and organizations for these cancers. The goal is to identify scientific priorities and resource needs for making progress against all sarcomas. Following are the Sarcoma PRG's next steps:

• A written report describing the group's findings and recommendations will be presented to the NCI and disseminated widely within the cancer community.
• The PRG will then meet with NCI leaders to discuss NCI's plan for addressing the recommendations.

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Modified Chemotherapy Regimen Improves Survival for Ewing's Sarcoma Patients

I'm only 13 years old, and now they tell me I have cancer? I knew I was sick, but I thought I would just get better. I never thought about this. Almost nobody gets this disease. Is anybody working on a better treatment?

Ewing's sarcoma is a rare malignant bone tumor that occurs in children and adolescents, with highest known incidence among Caucasian males. About 150 new cases are reported each year in the United States. Prior to the treatment regimen highlighted here, 50 percent to 60 percent of patients without metastases and about 25 percent with metastasis have survived at least 5 years.

Since the introduction in the 1970s of adjuvant chemotherapy for patients with Ewing's sarcoma, survival rates for patients with this disease increased dramatically but are still considerably low. Recently, scientists have discovered that the addition of the drugs isofamide and eposide to standard chemotherapy seems to improve treatment outcomes for relapsed patients with either Ewing's sarcoma or the closely related primitive neuroectodermal tumor of the bone. These findings led investigators to test this treatment regimen in newly diagnosed patients, with the following results:

• Although outcomes for patients with metastatic disease were not improved, the added drugs significantly improved survival in patients with newly diagnosed nonmetastatic disease.
• After 5 years, 69 percent of patients who received the experimental drug regimen were disease free, compared to 54 percent of those treated with standard therapy.
• Overall 5-year survival was 72 percent in the experimental therapy group, compared with 61 percent in the group treated with standard therapy.
• Although patients receiving experimental therapy suffered more infections and spent more time in the hospital, overall toxicity levels were similar between the two groups.

These findings hold promise for patients with Ewing's sarcoma and primitive neuroectodermal tumor of the bone.

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