Spotlight on disease-specific cancer organizations

Despite challenges, they continue to pursue missions and make strides


  • Carrie Printz

Although scores of disease-specific cancer organizations nationwide often compete over charitable and government funding in these tough economic times, many also work collaboratively toward common goals. “There's so much data that crosses the border these days that the more we learn about one cancer, there's applicability to other cancers as well,” says Dan Zenka, vice president of communications for the Prostate Cancer Foundation (PCF). Matthew Alsante, executive director of the Sarcoma Foundation ofAmerica (SFA), agrees.” We think you can do more together, and we work with other organizations to advance the cause for all cancers:' Alsante says. “Congress wants to advance cures, and the future of cancer research may be drugs for specific pathways—like the p53 gene that malfunctions in multiple cancers—rather than one cancer versus another.” Although many alliances have been formed among disease-specific organizations, each group also has a specific agenda based on their constituents' unique needs. “CancerScope” spoke with leaders of a few of these groups to learn what they are doing to improve the lives of patients with these diseases.

Lung Cancer Alliance

Lung cancer is the leading cause of cancer death in every ethnic group and will kill nearly 160,000 people nationwide in 2010. The 5-year survival rate is approximately 15.8%. Nevertheless, federal funds for research in terms of “dollars per death” are approximately $1,400 for thc disease versus nearly $29,000 for breast cancer and more than $13,000 for prostate cancer, according to statistics gathered by the Lung Cancer Alliance (LCA). The reasons for the lack of funding include both the stigma of smoking associated with the disease and the amount of money that is invested in tobacco cessation programs. “While we support tobacco cessation efforts, we know you can't solve this disease when nearly 80% of new lung cancer patients have either quit or never smoked,” says Kay Cofranccsco, LCA's director of advocacy relations.

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Lack of research funding has kept scientists from entering the field, whilc at the same time the disease has fewer advocates than other cancers. Recognizing these problems as a political issue, leaders of the organization, which was founded in 1995 in Washington State, relocated to Washington, DC in 2005.Their biggest success to date is securing the Department of Defense's congressionally directed medical research program for lung cancer, which began 2 years ago. The research is specifically directed toward providing early detection and screening options for veterans, who are at higher risk for the disease than the general population. In 2009, the program received $20 million in funding, followed by S 15 million in 2010, according to Cofrancesco.

The organization has formed 5 state chapters to raise awareness and increase state funding. They also are actively lobbying for passage of the federal Lung Cancer Mortality Reduction Act, which would authorize the departments of Health and Human Services, Defense, and Veteransbffairs to develop a comprehensive research strategy to significantly reduce lung cancer mortality by 2015, targeting prevention, diagnosis, and treatment.The bill also would provide first-year funding of $75 million for 4 National Institutes of Health (NIH) divisions to expand their lung cancer research programs. Because lung cancer is the leading cancer killer, overall cancer survival ratcs will not be reduced significantly until more money is put into research and early detection, Cofrancesco notes.

Meanwhile, the LCA is advocating for more clinical trials to be conducted in this area, just as extensive trials have occurred in determining the effcctivencss of mammography screening for women aged younger than 50 years.

In terms of awareness, “I think we're in much better shape than we were 5 years ago,” Cofrancesco says, noting that the deaths of broadcaster Peter Jennings and actress Dana Reeve have brought attention to the importance of research. “It's a testament to the scrappiness and dedication of the survivors who have talked about the disease— and it's been up to us to keep the drumbeat going.”

Sarcoma Foundation of America

“We look at ourselves as the cog in the wheel to bring new therapies to sarcoma patients,” says Alsante.” The spokes include the NCI [National Cancer Institute], NIH, FDA [Food and Drug Administration], the pharmaceutical industry, academic institutions, and sarcoma patients.” The organization was founded in 2000 by Mark Thornton, MD; his wife, Patricia; and John Brooks, MD, a pathologist. The Thorn- tons' son had sarcoma, and at the time no organizations cxisted to provide support for the disease. Sarcoma has more than 100 different subtypes, occurs in approximately 12,000 patients per year, and constitutes approximately 1% of all adult and 15% of all childhood cancers.

Since its inception, the SFA has funded 50 sarcoma research grants and partnered with the American Society of Clinical Oncology ASCO) in funding 4 young investigator awards and 2 advanced clinical research awards. “There is more research going on now than ever in the history of the disease,” notes Alsante. Nevertheless, many challenges remain, including the barriers to developing and gaining approval for new therapies for sarcoma and other rare diseases. The SFA and other rare disease organizations maintain that the FDA has been inconsistent in its approval process for drugs for many rare diseases, creating confusion and a disincentive for the pharmaceutical industry to develop new drugs.

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Alsantc and others testified at an FDA public hearing on the issue in June. After reviewing testimony, the FDA was slated to develop a guidance document by September 11,2011, to provide more detailed guidelines for developers of drugs for rare diseases. Although the Orphan Drug Act has helped somewhat in developing new drugs, it has been insufficient, Alsante says. “Again and again the FDA has demanded ‘overall survival’ as the primary endpoint for clinical trials in cases when there are not enough patients to ever complete a trial,” he says, noting that the SFA has asked the FDA for a guidance document since 2007. “We're looking for guidance that progression-free survival as an endpoint would be acceptable, which they have approved for multiple drugs.”

He cites the example of 2 sarcoma drugs—mifamurtide (Mepact) and trabectedin (Yondelis)—which have been approved for use in Europe but denied approval by the FDA. Recently a company submitted a phase 3 trial idea for drug for 2 extremely rare sarcomas. Although the European Medicines Agency accepted progression-free survival as an endpoint, the FDA did not. As a result, the company decided not to pursue the trial. “When you have maybe 200 people in the world with [a particular sarcoma], in order to get a statistically valid endpoint, you would need 100% participation,” Alsante says.” And tracking overall survival can take years. That's such a disincentive to a company when not many people will even be using the drug.” In the US, the drugs that are available for sarcoma are therapies that were approved 30 years ago for other indications and have trickled through to sarcoma with the hope that they'll be successful, he adds.

With 20 chapters nationwide, the SFA has been very successful in raising money and has created a national sarcoma patient registry, which should aid research, Alsante says. Each year, the organization holds a national gala in NewYork City as well as an educational conference for patients and one for professionals as part ofASCO's annual meeting.

Prostate Cancer Foundation and Us TOO

The fight against prostate cancer includes an effort by 13 organizations that make up the Prostate Cancer Round- table, a group that collaborates to support patients and their families and to find new diagnostics and treatments for a disease that will strike approximately 218,000 men this ycar. Among those in the roundtable are 2 well-known organizations: the PCF, which supports research, and the Us TOO International Prostate Cancer Education and Support Network, which addresses education and awareness.

The PCF, founded by Michael Milken in 1993, is the leading philanthropic organization for prostate cancer research, funding more than $400 million in projects since its inccption.That amount encompasses more than 1500 programs at nearly 200 research centers in 20 countries. “We started a new approach called venture philanthropy;' says Zenka, who was recently diagnosed with prostate cancet “We shortened the application to 5 pages, and we reviewed in 60 days and funded in 30. If a project takes a left turn and changes direction, grantecs don't have to reapply—they just have to teli us where their work is taking them and why:' Every ycar,fundcd scientists share their data at a PCF retreat.

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Despite the economic downturn, the past 2 years have been some of the PFC's best in terms of fundraising, mainly because it is a small organization that has been able to react and adjust more easily than larger organizations, Zenka notes.The organization raises approximately $37 million each year through a variety of programs, with approximately 85% of those funds directly supporting research. Events range from the Home Run Challenge—in which money is pledged for home runs in designated baseball games—to Movcmbcr, a worldwide fundraiser for men's diseases in which men receive pledges for growing mustaches (“Mo” is an Australian term for moustache).

“We've seen more progress this year in prostate cancer research than probably in the last decade,” Zenka notes. He points to the discovery of 24 gene fusions related to prostate cancer that create new targets and will help to better direct research toward new, promising personalized medicine efforts. Several of the Prostate Cancer Rouud table organizations worked together to organize an “Advance on Washington” event during the week of September 13, 2010. The groups hope to build awareness and support on a federal level. “We know we spend about $3 billion a year in over treating the disease, yet more than 32,000 men are dying each year—we'd love to see that $3 billion put into research,” Zenka says.

Meanwhile, Us TOO is celebrating its 20th anniversary of helping men and their families to make informed decisions about prostate cancer through support, education, and advocacy. The Chicago-based organization founded by 5 prostate cancer survivors now has 325 affiliated support groups and chapters worldwide, primarily in the United States. “Our name comes from the realization that cancer is important for us too—just as breast cancer is important to women,” says Us TOO president and CEO Tom Kirk.” Like the breast cancer movement was 30 years ago when people didn't use the word ‘breast’ in public, we're gaining awareness and have seen a big turnaround in the last 5 to 10 years.”

Unlike when the organization first was founded, when there was a lack of information concerning prostate cancer diagnosis and treatment, people who contact Us TOO now are often confused by the overwhelming amount of information available,Kirk notes.The group's main challenges are getting men to speak openly about the disease, to understand the risks, and to take action rather than trying to maintain a heroic attitude that their needs come last. “Doctors now don't have as much time to spend with us as they did in the past, so we need to turn to each other for information, and men need to understand that they have to be the quarterback or CEO of their own health care,” Kirk says.

The Leukemia and Lymphoma Society

The Leukemia and Lymphoma Society (LLS) is the world's largest voluntary health organization dedicated to funding blood cancer research, education, and patient services. It has 59 chapters across the United States and Canada and raises approximately $275 million each year for leukemia, lymphoma, and myeloma cancers. Since its inception some 61 years ago, it has funded more than $750 million in research, notes CEO John Walter. “Other than extreme exposure to benzene, there are no known causes or opportunities for prevention, so research is very important to us:' Walter says. We're focused on funding cures for these diseases:'

Recognizing that pathways to new treatments take time, LLS has chosen to continue a multifaceted approach that also focuses on patients' existing needs. Those include services such as family and peer support groups, a back-to- school program for kids, and direct financial aid to help patients afford their insurance co-pays.

In the legislative arena, the organization is focused on ensuring sufficient funding of the Cures Acceleration Network, which was part of the Patient Protection and Affordable Care Act signed into law by President Obama in March 2010.The provision creates a new office within the NIH, under the director's leadership, which is charged with speeding translation of basic scientific discoveries into treatments for patients. The goal is to encourage partnerships within the NIH, the pharmaceutical industry, academia, and other organizations to develop new collaborations in this area. LLS already has such a program on a smaller scale, in which they mine work from their academic scientists to generate clinical trials. One example is work by a Canadian researcher that led to development of a promising treatment for acute myeloid leukemia, which has a poor outcome and few new treatments. LLS partnered with Princess Margaret Hospital in Toronto, Ontario, Canada, and the University of Kansas in Lawrence to support the project, and within 9 months helped launch a phase 1 clinical trial for the compound. The organization also canvases the biotech industry to determine who might have compounds that could be translated into clinical trials.

Because the blood cancer patient population is relatively small (an estimated 137,260 people will be diagnosed in 2010) the opportunity for profit is not large. That why LLS funding combined with federal government support of new treatments is vital, Walter notes. The organization is primarily funding phase 1 trials, with the hope that promising results will encourage partnerships with companies that can advance the research and eventually bring a drug to market. “In 3 to 5 years, one of these treatments will be successful, and we'll have a new standard of for our patients in the near tcrm” Walter says.