By Matthew Bin Han Ong and Paul Goldberg
A bill moving rapidly through Congress directs NCI to spend $887.8 million over five years for pancreatic cancer research.
In addition to moving resources out of NCI’s control at a time of flat budgets, the legislation gives NCI only a minor role in deciding how the massive new program would use institute resources.
The institute would have only one vote on a critically important 13-member panel that would allocate the funds.
According to opponents, the measure amounts to a vote of no confidence in NCI’s ability to oversee pancreatic cancer research. It would catastrophically undermine the peer review system, touching off a round of what some on Capitol Hill call “disease olympics.”
The Pancreatic Cancer Action Network, the advocacy group that launched the aggressive drive that resulted in the legislation, said the old approach hasn’t produced results, citing small budgets for pancreatic cancer research and alleging that NCI has failed to respond to congressional mandates. The bill would roughly double the institute’s spending on the disease.
PanCAN hired a lobbyist, put together a glossy fact sheet and staged an “advocacy day” on Capitol Hill. Legislators were handed morgue-style toe tags with the names of constituents who died from pancreatic cancer. A child recounted a family member’s death from the disease, sources said.
The result: the bill, called the Pancreatic Cancer Research and Education Act (H.R.733 and S.362), has amassed the support of 279 House members and 58 Senate members. This means that some kind of a pancreatic cancer bill has an excellent chance of passing during the current legislative session.
The measure was introduced February 2011 by Reps. Anna Eshoo (D-Calif.), Leonard Lance (R-N.J.) and Sen. Sheldon Whitehouse (D-R.I.).
This political momentum makes critics—including many on the NIH campus in Bethesda—cringe at the prospect of interest groups competing for Congressional earmarks that would be doled out based on severity of disease and the advocates’ ability to mobilize political clout, without regard for the quality of science or the opportunity to bring about clinical advances.
The bill specifically paves the way for other patient groups to seek similar approaches to bypassing NCI authority.
“This is not a disease-specific bill,” PanCANproclaimed in documents distributed on the Hill at the advocacy day June 26. “It includes a recommendation that the NCI develop a new grant program to focus on the other deadly cancers. The program would be piloted with pancreatic cancer (the deadliest major cancer), but could be expanded to include any cancer with a five-year survival rate below 50 percent, including brain, esophageal, liver, lung, myeloma, ovarian and stomach cancers.”
To direct pancreatic cancer activities, the bill requires the HHS secretary to establish a 13-member coordinating committee including academics and a pancreatic cancer advocate. The panel will conduct evaluations and make recommendations to the HHS secretary regarding the prioritization and awarding of NIH grants. NCI will have only one vote on this panel.
“While I have tremendous sympathy for patients and families affected by pancreatic cancer, I find this bill quite disturbing,” said Charles Sawyers, cancer research physician and scientist at Memorial Sloan Kettering Cancer Center, investigator at the Howard Hughes Medical Institute, and president-elect of the American Association for Cancer Research. “As a cancer researcher, I feel strongly that the existing NIH peer review system is an effective way to identify promising science across diseases.
“Any proposal that allows a separate committee that would exist outside the existing NIH peer review system to prioritize and make funding recommendations puts the entire peer review process at risk,” said Sawyers, speaking only for himself, and not for any of the organizations with which he is affiliated. “Furthermore, this bill would establish a dangerous precedent that could balkanize our field at precisely the wrong moment.
“We are learning that different cancers share molecular features and that treatments developed for one disease can be quickly leveraged to impact others. Unfortunately, certain provisions in this bill run the risk of losing that synergy. What we really need is a commitment of sustained NIH funding across cancers.”
A Lack of “Funding, Focus, and Commitment”
The pancreatic cancer legislation criticizes NCI for a lack of “funding, focus, and commitment” on the institute’s part to implement pancreatic cancer initiatives over the past decade and for allocating only 2 percent of its federal funding to pancreatic research.
Though the bill plainly states that it would shift coordination and prioritization of pancreatic cancer research away from NCI, Julie Fleshman, CEO of PanCAN, said that the NCI peer review system would continue to function as it does.
“The Pancreatic Cancer Action Network reads the bill as creating a committee that provides recommendations to the NCI director, but does not change the NCI’s peer review process,” Fleshman said in an email exchange with The Cancer Letter.
“Our understanding is that authorizations are often written to the HHS secretary as a way to convey how important a priority the subject is,” Fleshman said. “But as a practical matter, the responsibility is subsequently delegated to the NIH or NCI director as the law is implemented.” The interview appears here.
PanCAN’s advocacy day documents accuse NCI of being insufficiently interested in the disease and unresponsive to concerns from legislators.
“It is time for the National Cancer Institute to develop a long-term and comprehensive research strategy that specifically addresses pancreatic cancer,” one PanCAN handout alleged. “Congress has repeatedly asked the NCI to address this issue, but the NCI has failed to respond to Congressional requests. It is time for Congress to take action, as it has done for other diseases, by requiring the NCI to develop a strategic plan for one of our nation’s leading cancer killers.”
Recently, while updating an NCI advisory committee, NCI Director Harold Varmus said the controversy over pancreatic cancer had come up at NIH appropriations hearings.
The institute has, in fact, increased funding for pancreatic cancer research, Varmus said to a joint meeting of the National Cancer Advisory Board and the Board of Scientific Advisors June 25.
“I personally had six friends who died from this disease in the last five years,” Varmus said to the two boards. “We have increased our funding of pancreatic cancer research by 300 percent in the last 10 years, despite a flat budget.
“Of course, we can’t just pour all of our money into pancreatic cancer research, but there is no doubt that there is significant concern, and I’d like to kind of meet those concerns in an equitable way,” said Varmus, whose lab at NCI is involved in pancreatic cancer research.
In a recent press release, bill sponsors Eshoo and Lance cited past earmarked funds for research on breast cancer, Alzheimer’s, autism, diabetes and HIV/AIDS as precedent for enacting a similar legislation for pancreatic cancer.
“While the strategic plan required by the bill would provide a critical tool for making progress in this terrible disease, nothing in the bill specifically requires NCI to allocate taxpayer dollars for disease-specific research,” wrote Eshoo and Lance in a letter to the Committee on Energy and Commerce. “NCI would continue to set priorities, and grants would continue to be awarded on a peer-reviewed basis.”
This is, at least in part, correct. The bill authorizes spending funds on pancreatic cancer. Appropriations would be determined through a separate legislative process. However, in the era of flat budgets, it is inconceivable that funds can come from any place other than the NCI budget, thereby lowering the amount of funds available for other research.
Effort Differs from Defense Department Program
The fact that a relatively small advocacy group like PanCAN, based in Manhattan Beach, Calif., was able to amass so much congressional support demonstrates just how vulnerable NCI is to earmarking.
PanCAN raised $13.3 million in 2010, according to tax documents, and spent $1.5 million on “government affairs and advocacy.”
The group’s lobbyist, Joel White, is a former Republican Capitol Hill staff member, whose other clients include the Pharmaceutical Research and Manufacturers of America and a coalition seeking to maintain Medicare Part D coverage.
He appears to be representing PanCAN pro bono: http://www.opensecrets.org/lobby/firmsum.php?id=F27076.
The group’s public relations campaign is handled by AK PR Group.
During this legislative session, PanCAN published an advertisement showing a pair of feet, presumed to belong to a dead pancreatic cancer patient, with a morgue toe tag attached. Penciled in on the tag is the number of U.S. pancreatic cancer-related deaths over the past five years and the disease’s low survival rate. The text of the ad reads: “Pass this bill so thousands more won’t pass away.”
A bill moving rapidly through Congress directs NCI to spend $887.8 million over five years for pancreatic cancer research.
In addition to moving resources out of NCI’s control at a time of flat budgets, the legislation gives NCI only a minor role in deciding how the massive new program would use institute resources.
The institute would have only one vote on a critically important 13-member panel that would allocate the funds.
According to opponents, the measure amounts to a vote of no confidence in NCI’s ability to oversee pancreatic cancer research. It would catastrophically undermine the peer review system, touching off a round of what some on Capitol Hill call “disease olympics.”
The Pancreatic Cancer Action Network, the advocacy group that launched the aggressive drive that resulted in the legislation, said the old approach hasn’t produced results, citing small budgets for pancreatic cancer research and alleging that NCI has failed to respond to congressional mandates. The bill would roughly double the institute’s spending on the disease.
PanCAN hired a lobbyist, put together a glossy fact sheet and staged an “advocacy day” on Capitol Hill. Legislators were handed morgue-style toe tags with the names of constituents who died from pancreatic cancer. A child recounted a family member’s death from the disease, sources said.
The result: the bill, called the Pancreatic Cancer Research and Education Act (H.R.733 and S.362), has amassed the support of 279 House members and 58 Senate members. This means that some kind of a pancreatic cancer bill has an excellent chance of passing during the current legislative session.
The measure was introduced February 2011 by Reps. Anna Eshoo (D-Calif.), Leonard Lance (R-N.J.) and Sen. Sheldon Whitehouse (D-R.I.).
This political momentum makes critics—including many on the NIH campus in Bethesda—cringe at the prospect of interest groups competing for Congressional earmarks that would be doled out based on severity of disease and the advocates’ ability to mobilize political clout, without regard for the quality of science or the opportunity to bring about clinical advances.
The bill specifically paves the way for other patient groups to seek similar approaches to bypassing NCI authority.
“This is not a disease-specific bill,” PanCANproclaimed in documents distributed on the Hill at the advocacy day June 26. “It includes a recommendation that the NCI develop a new grant program to focus on the other deadly cancers. The program would be piloted with pancreatic cancer (the deadliest major cancer), but could be expanded to include any cancer with a five-year survival rate below 50 percent, including brain, esophageal, liver, lung, myeloma, ovarian and stomach cancers.”
To direct pancreatic cancer activities, the bill requires the HHS secretary to establish a 13-member coordinating committee including academics and a pancreatic cancer advocate. The panel will conduct evaluations and make recommendations to the HHS secretary regarding the prioritization and awarding of NIH grants. NCI will have only one vote on this panel.
“While I have tremendous sympathy for patients and families affected by pancreatic cancer, I find this bill quite disturbing,” said Charles Sawyers, cancer research physician and scientist at Memorial Sloan Kettering Cancer Center, investigator at the Howard Hughes Medical Institute, and president-elect of the American Association for Cancer Research. “As a cancer researcher, I feel strongly that the existing NIH peer review system is an effective way to identify promising science across diseases.
“Any proposal that allows a separate committee that would exist outside the existing NIH peer review system to prioritize and make funding recommendations puts the entire peer review process at risk,” said Sawyers, speaking only for himself, and not for any of the organizations with which he is affiliated. “Furthermore, this bill would establish a dangerous precedent that could balkanize our field at precisely the wrong moment.
“We are learning that different cancers share molecular features and that treatments developed for one disease can be quickly leveraged to impact others. Unfortunately, certain provisions in this bill run the risk of losing that synergy. What we really need is a commitment of sustained NIH funding across cancers.”
A Lack of “Funding, Focus, and Commitment”
The pancreatic cancer legislation criticizes NCI for a lack of “funding, focus, and commitment” on the institute’s part to implement pancreatic cancer initiatives over the past decade and for allocating only 2 percent of its federal funding to pancreatic research.
Though the bill plainly states that it would shift coordination and prioritization of pancreatic cancer research away from NCI, Julie Fleshman, CEO of PanCAN, said that the NCI peer review system would continue to function as it does.
“The Pancreatic Cancer Action Network reads the bill as creating a committee that provides recommendations to the NCI director, but does not change the NCI’s peer review process,” Fleshman said in an email exchange with The Cancer Letter.
“Our understanding is that authorizations are often written to the HHS secretary as a way to convey how important a priority the subject is,” Fleshman said. “But as a practical matter, the responsibility is subsequently delegated to the NIH or NCI director as the law is implemented.” The interview appears here.
PanCAN’s advocacy day documents accuse NCI of being insufficiently interested in the disease and unresponsive to concerns from legislators.
“It is time for the National Cancer Institute to develop a long-term and comprehensive research strategy that specifically addresses pancreatic cancer,” one PanCAN handout alleged. “Congress has repeatedly asked the NCI to address this issue, but the NCI has failed to respond to Congressional requests. It is time for Congress to take action, as it has done for other diseases, by requiring the NCI to develop a strategic plan for one of our nation’s leading cancer killers.”
Recently, while updating an NCI advisory committee, NCI Director Harold Varmus said the controversy over pancreatic cancer had come up at NIH appropriations hearings.
The institute has, in fact, increased funding for pancreatic cancer research, Varmus said to a joint meeting of the National Cancer Advisory Board and the Board of Scientific Advisors June 25.
“I personally had six friends who died from this disease in the last five years,” Varmus said to the two boards. “We have increased our funding of pancreatic cancer research by 300 percent in the last 10 years, despite a flat budget.
“Of course, we can’t just pour all of our money into pancreatic cancer research, but there is no doubt that there is significant concern, and I’d like to kind of meet those concerns in an equitable way,” said Varmus, whose lab at NCI is involved in pancreatic cancer research.
In a recent press release, bill sponsors Eshoo and Lance cited past earmarked funds for research on breast cancer, Alzheimer’s, autism, diabetes and HIV/AIDS as precedent for enacting a similar legislation for pancreatic cancer.
“While the strategic plan required by the bill would provide a critical tool for making progress in this terrible disease, nothing in the bill specifically requires NCI to allocate taxpayer dollars for disease-specific research,” wrote Eshoo and Lance in a letter to the Committee on Energy and Commerce. “NCI would continue to set priorities, and grants would continue to be awarded on a peer-reviewed basis.”
This is, at least in part, correct. The bill authorizes spending funds on pancreatic cancer. Appropriations would be determined through a separate legislative process. However, in the era of flat budgets, it is inconceivable that funds can come from any place other than the NCI budget, thereby lowering the amount of funds available for other research.
Effort Differs from Defense Department Program
The fact that a relatively small advocacy group like PanCAN, based in Manhattan Beach, Calif., was able to amass so much congressional support demonstrates just how vulnerable NCI is to earmarking.
PanCAN raised $13.3 million in 2010, according to tax documents, and spent $1.5 million on “government affairs and advocacy.”
The group’s lobbyist, Joel White, is a former Republican Capitol Hill staff member, whose other clients include the Pharmaceutical Research and Manufacturers of America and a coalition seeking to maintain Medicare Part D coverage.
He appears to be representing PanCAN pro bono: http://www.opensecrets.org/lobby/firmsum.php?id=F27076.
The group’s public relations campaign is handled by AK PR Group.
During this legislative session, PanCAN published an advertisement showing a pair of feet, presumed to belong to a dead pancreatic cancer patient, with a morgue toe tag attached. Penciled in on the tag is the number of U.S. pancreatic cancer-related deaths over the past five years and the disease’s low survival rate. The text of the ad reads: “Pass this bill so thousands more won’t pass away.”
A packet of PanCAN's advocacy materials can be downloaded at http://www.cancerletter.com/categories/documents.
PanCAN provided similar toe tags to the 650 participants of the June 26 advocacy day. Each of these tags contained a blank that could be filled in with the name of a patient who had died of pancreatic cancer.
The participants were instructed to leave the tags with the congressional offices after their meetings with all 100 Senate members and 371 of 435 House members.
“I feel strongly, as does the Pancreatic Cancer Action Network, that we must be aggressive and even shocking to get our message heard above all of the others that legislators hear each day,” said Richard
Orr, PanCAN advocate and pancreatic cancer survivor, in a letter published on PanCAN’s website: http://bit.ly/QnnPxr.
“We expect members of the Congress and their staff to be shocked by this tactic and believe that this is a good thing as it will be impossible to ignore,” Orr wrote. “As a result, they will not be able to ignore our message that they must pass the bill this year.”
PanCAN isn’t the only cancer organization to employ in-your-face tactics.
The Lung CancerAlliance has been advocating changing current practices to include screening for early-stage lung cancer, working closely with the International Early Lung Cancer Action Program. LCA’s “No One Deserves To Die” campaign of public service announcements, is similarly designed to shock: http://www.noonedeservestodie.org/.
Arguments over earmarking funds for specific diseases have been a part of the cancer funding process for at least two decades. However, no group has ever tried to bypass the NIH peer review system while keeping the money within HHS.
Why didn’t PanCAN try to take pancreatic cancer research outside NCI, to the Department of Defense? At DOD, such research programs are rigorously reviewed.
Fleshman said the idea of building a program within DOD was discarded to allow maximum collaboration with institutions receiving NCI funding.
“Unlike many other cancers, the only federal institute that is truly looking at pancreatic cancer in any real way is the NIH and the NCI,” she said.
At NIH, the institutes set their own spending priorities.
If the bill passes, pancreatic cancer would become the largest earmarked cancer program within the federal government. With $177.6 million a year to spend, it would overtake the DOD breast cancer program, which received $120 million during fiscal 2012. According to PanCAN, pancreatic cancer currently receives approximately $99 million a year from NCI.
The fundamental question is whether scientists have the leads that would make it possible to put additional funds to good use.
“The notion that there are only a few leads on the etiology of pancreatic cancer is erroneous,” Fleshman said. “Over the past decade, scientists have gained a much better understanding of pancreatic cancer and what makes it unique biologically.
“For instance, scientists now know that there is a dense microenvironment that surrounds the pancreatic cancer tumor and that understanding this microenvironment will help aid in drug delivery. Further, scientists now know about the genetics of pancreatic cancer, including the genes that need to be targeted, like the KRAS gene. KRAS is present in 90 percent of pancreatic cancer cases. Additionally, there is a genetically engineered mouse model for pancreatic cancer that allows scientists to test drugs. Scientists believe that these mice mimic how the test drugs would react in humans.
“And due to increased efforts of the NCI and private foundations, there are a growing number of scientists—senior and junior—focusing on pancreatic cancer. This was perhaps best evidenced by the first ever American Association of Cancer Research special conference for pancreatic cancer, which occurred a month ago. The conference had 450 attendees and was considered a great success.
“However, there is still more progress that needs to be made. One area that is particularly problematic is the number of researchers. While there is a growing field of researchers dedicated to pancreatic cancer, it is still very small compared to the amount that we need to truly make progress. For example, the number of training grants and significant grants of $500,000 or more, which sustain laboratories, are generally much less than those funded for the other leading cancer killers. There aren’t enough scientists in the field directly related to the resources available.”
While this information has been discovered—mostly as a result of NCI funding—hardly any of it is ready to be translated into clinical advances, critics say.
David Tuveson, a pancreatic cancer researcher, said the federal government has the potential to advance pancreatic cancer research if it proclaims that clinical trials are the standard of care for the disease and if it funds a large number of proof of concept studies.
“I am not advocating bypassing peer review; I never would advocate that,” said Tuveson, a professor at the Cold Spring Harbor Laboratory and co-chair of a recent PanCAN-AACR meeting on pancreatic cancer research. “I am advocating increased concentration by regulatory agencies and by funding bodies on pancreas cancer.”
Tuveson was not involved in work on the PanCAN bill and was not familiar with its provisions, he said. “We have to be an evidence-based effort, and I would not advocate loosening peer review,” Tuveson said. “I would advocate increasing the spending on hypothesis-driven research conducted in the clinic and in the laboratory for pancreas cancer patients.
“If NCI is willing to fund proof of concept trials that physician scientists direct, where they can determine in small and very organized clinical studies, then I would support continuing the process,” Tuveson said.
“If they are going to continue to ignore this serious need we have in the field, then we have to find an alternative funding arrangement. They have to do it in the clinical realm. If they take it back from CTEP, take it back from PhRMA, they are not going to do it properly, if they are going to allow the academics drive that discovery process, then I would remain a full supporter of the peer review system we have now.”
Tuveson, who recently came to Cold Spring Harbor from Cancer Research UK and Cambridge Research Institute, said the U.S. government should sponsor innovative clinical trials, “where we have multiple companies giving their drugs and you are able to test them in combination, just like during the HIV days, when arm-twisting by activists and a little intervention by the government allowed combination trials to get off the ground.
It’s possible to move research forward faster, Tuveson said.
“The trials that I want are ones that are science-driven proof of concept—prove your principle—in patients, and those are not trials supported by the private sector, by and large, not supported by our government, by and large,” Tuveson said. “We are not funding the essential first step in the fight against this disease.”
Bill to Create Two SPOREs
Under the pancreatic cancer legislation, $733 million will be allocated over five years for the creation of the coordinating committee, which will develop a plan to review pancreatic cancer grant applications—including grants awarded through NCI—and to enlist additional review personnel.
The committee will supervise the budget for an additional incubator pilot project as well as two Specialized Programs of Research Excellence, which would be designed to focus solely on pancreatic cancer research.
This aspect of the proposal is particularly noteworthy at a time when NCI officials are talking about cutting SPOREs as part of reprogramming the institute’s resources.
Besides the $733 million earmarked for the main initiative, the bill authorizes:
• $107.5 million for two SPOREs focusing solely on pancreatic cancer research. The secretary may choose to designate one or more satellite centers that augment the work of a previously designated SPORE.
• $26.3 million for a new incubator project, allowing the HHS secretary to award five-year grants to research institutions for use in pancreatic cancer research. Recipients of these grants will be encouraged to use the funds for research activities that may serve as a “springboard” for the receipt of other grants, including SPOREs grants, from the NIH.
• $10.5 million for a primary care provider education program, which the secretary will develop in consultation with NIH, CDC and patient advocates. Accredited continuing medical education programs and activities may be included as the secretary deems appropriate.
• $10.5 million for the development of a communication toolkit for patients and families, which requires NCI and CDC directors to work with patient advocacy organizations such as PanCAN to focus on specific pancreatic cancer issues.
“We hope to be included in that discussion as well,” Fleshman said. “The Pancreatic Cancer Action
Network has a good relationship with NCI, so we would welcome the opportunity to work closely with them as they implement the bill.”
Collaboration With AACR
In a related development, PanCAN has been working closely with AACR in designing an agenda for accelerating progress against pancreatic cancer.
The two organizations held a meeting on the “progress and challenges” in pancreatic cancer at Lake Tahoe June 18-21, forming a joint Scientific Review Committee. The meeting’s co-chairs included Daniel
Von Hoff, of Translational Genomics Research Institute, Dafna Bar-Sagi, of New York University School of Medicine, Chi Van Dang, director of the University of Pennsylvania Cancer Center, and Cold Spring Harbor’s Tuveson.
The review committee formed by PanCAN and AACR “will oversee a new grant opportunity that provides a 1-3 year grant of $1,000,000 to an existing, multi-institutional, clinical or translational research project within the pancreatic cancer research community,” said AACR CEO Margaret Foti in a recent letter to a scientist who agreed to serve on the committee.
The text of Foti’s letter follows:
The current grant review and selection process is as follows: Letters of Intent (LOI) will be accepted between late August and early October. Once all submitted LOIs are reviewed by AACR staff for eligibility and compliance, committee members are directed to access proposal CENTRAL to view the LOIs and select those most suitable to review based on their scientific knowledge.
Committee members must also indicate whether any LOI constitutes a conflict of interest. Each LOI will be assigned to at least three committee member reviewers that will have approximately three weeks to complete their critiques. Once the LOI reviews are completed, AACR staff will compile the scores and comments, and provide the results to the committee.
A conference call with the committee will then be held to select the applicants worthy of proceeding to the full application phase (25% of the LOIs). We anticipate this call to be held in late November/early December. Once all full applications are received, the application review period will begin in January of 2013. A second teleconference will then be held in February to select the finalists as well as primary and alternate grantees.
For the remainder of your two-year term, based on confirmation from the award supporter, this grant may be launched once more with the review and selection process occurring between October 2013 and February 2014. Throughout this time, AACR’s Scientific Review and Grants Administration staff will provide full support and guidance to both you and the committee.
Please note that committee members may be called upon in the future to review selected grantees’ annual and final progress reports and possibly to provide feedback to the grantees on his/her project. Also, selection committee members are prohibited from applying for or participating on any applications for this grant throughout the course of their term.
The text of the bill: http://thomas.loc.gov/cgi-bin/query/z?c112:H.R.733:
The House and Senate versions of the bill, H.R.733 and S.362, are identical.
PanCAN provided similar toe tags to the 650 participants of the June 26 advocacy day. Each of these tags contained a blank that could be filled in with the name of a patient who had died of pancreatic cancer.
The participants were instructed to leave the tags with the congressional offices after their meetings with all 100 Senate members and 371 of 435 House members.
“I feel strongly, as does the Pancreatic Cancer Action Network, that we must be aggressive and even shocking to get our message heard above all of the others that legislators hear each day,” said Richard
Orr, PanCAN advocate and pancreatic cancer survivor, in a letter published on PanCAN’s website: http://bit.ly/QnnPxr.
“We expect members of the Congress and their staff to be shocked by this tactic and believe that this is a good thing as it will be impossible to ignore,” Orr wrote. “As a result, they will not be able to ignore our message that they must pass the bill this year.”
PanCAN isn’t the only cancer organization to employ in-your-face tactics.
The Lung CancerAlliance has been advocating changing current practices to include screening for early-stage lung cancer, working closely with the International Early Lung Cancer Action Program. LCA’s “No One Deserves To Die” campaign of public service announcements, is similarly designed to shock: http://www.noonedeservestodie.org/.
Arguments over earmarking funds for specific diseases have been a part of the cancer funding process for at least two decades. However, no group has ever tried to bypass the NIH peer review system while keeping the money within HHS.
Why didn’t PanCAN try to take pancreatic cancer research outside NCI, to the Department of Defense? At DOD, such research programs are rigorously reviewed.
Fleshman said the idea of building a program within DOD was discarded to allow maximum collaboration with institutions receiving NCI funding.
“Unlike many other cancers, the only federal institute that is truly looking at pancreatic cancer in any real way is the NIH and the NCI,” she said.
At NIH, the institutes set their own spending priorities.
If the bill passes, pancreatic cancer would become the largest earmarked cancer program within the federal government. With $177.6 million a year to spend, it would overtake the DOD breast cancer program, which received $120 million during fiscal 2012. According to PanCAN, pancreatic cancer currently receives approximately $99 million a year from NCI.
The fundamental question is whether scientists have the leads that would make it possible to put additional funds to good use.
“The notion that there are only a few leads on the etiology of pancreatic cancer is erroneous,” Fleshman said. “Over the past decade, scientists have gained a much better understanding of pancreatic cancer and what makes it unique biologically.
“For instance, scientists now know that there is a dense microenvironment that surrounds the pancreatic cancer tumor and that understanding this microenvironment will help aid in drug delivery. Further, scientists now know about the genetics of pancreatic cancer, including the genes that need to be targeted, like the KRAS gene. KRAS is present in 90 percent of pancreatic cancer cases. Additionally, there is a genetically engineered mouse model for pancreatic cancer that allows scientists to test drugs. Scientists believe that these mice mimic how the test drugs would react in humans.
“And due to increased efforts of the NCI and private foundations, there are a growing number of scientists—senior and junior—focusing on pancreatic cancer. This was perhaps best evidenced by the first ever American Association of Cancer Research special conference for pancreatic cancer, which occurred a month ago. The conference had 450 attendees and was considered a great success.
“However, there is still more progress that needs to be made. One area that is particularly problematic is the number of researchers. While there is a growing field of researchers dedicated to pancreatic cancer, it is still very small compared to the amount that we need to truly make progress. For example, the number of training grants and significant grants of $500,000 or more, which sustain laboratories, are generally much less than those funded for the other leading cancer killers. There aren’t enough scientists in the field directly related to the resources available.”
While this information has been discovered—mostly as a result of NCI funding—hardly any of it is ready to be translated into clinical advances, critics say.
David Tuveson, a pancreatic cancer researcher, said the federal government has the potential to advance pancreatic cancer research if it proclaims that clinical trials are the standard of care for the disease and if it funds a large number of proof of concept studies.
“I am not advocating bypassing peer review; I never would advocate that,” said Tuveson, a professor at the Cold Spring Harbor Laboratory and co-chair of a recent PanCAN-AACR meeting on pancreatic cancer research. “I am advocating increased concentration by regulatory agencies and by funding bodies on pancreas cancer.”
Tuveson was not involved in work on the PanCAN bill and was not familiar with its provisions, he said. “We have to be an evidence-based effort, and I would not advocate loosening peer review,” Tuveson said. “I would advocate increasing the spending on hypothesis-driven research conducted in the clinic and in the laboratory for pancreas cancer patients.
“If NCI is willing to fund proof of concept trials that physician scientists direct, where they can determine in small and very organized clinical studies, then I would support continuing the process,” Tuveson said.
“If they are going to continue to ignore this serious need we have in the field, then we have to find an alternative funding arrangement. They have to do it in the clinical realm. If they take it back from CTEP, take it back from PhRMA, they are not going to do it properly, if they are going to allow the academics drive that discovery process, then I would remain a full supporter of the peer review system we have now.”
Tuveson, who recently came to Cold Spring Harbor from Cancer Research UK and Cambridge Research Institute, said the U.S. government should sponsor innovative clinical trials, “where we have multiple companies giving their drugs and you are able to test them in combination, just like during the HIV days, when arm-twisting by activists and a little intervention by the government allowed combination trials to get off the ground.
It’s possible to move research forward faster, Tuveson said.
“The trials that I want are ones that are science-driven proof of concept—prove your principle—in patients, and those are not trials supported by the private sector, by and large, not supported by our government, by and large,” Tuveson said. “We are not funding the essential first step in the fight against this disease.”
Bill to Create Two SPOREs
Under the pancreatic cancer legislation, $733 million will be allocated over five years for the creation of the coordinating committee, which will develop a plan to review pancreatic cancer grant applications—including grants awarded through NCI—and to enlist additional review personnel.
The committee will supervise the budget for an additional incubator pilot project as well as two Specialized Programs of Research Excellence, which would be designed to focus solely on pancreatic cancer research.
This aspect of the proposal is particularly noteworthy at a time when NCI officials are talking about cutting SPOREs as part of reprogramming the institute’s resources.
Besides the $733 million earmarked for the main initiative, the bill authorizes:
• $107.5 million for two SPOREs focusing solely on pancreatic cancer research. The secretary may choose to designate one or more satellite centers that augment the work of a previously designated SPORE.
• $26.3 million for a new incubator project, allowing the HHS secretary to award five-year grants to research institutions for use in pancreatic cancer research. Recipients of these grants will be encouraged to use the funds for research activities that may serve as a “springboard” for the receipt of other grants, including SPOREs grants, from the NIH.
• $10.5 million for a primary care provider education program, which the secretary will develop in consultation with NIH, CDC and patient advocates. Accredited continuing medical education programs and activities may be included as the secretary deems appropriate.
• $10.5 million for the development of a communication toolkit for patients and families, which requires NCI and CDC directors to work with patient advocacy organizations such as PanCAN to focus on specific pancreatic cancer issues.
“We hope to be included in that discussion as well,” Fleshman said. “The Pancreatic Cancer Action
Network has a good relationship with NCI, so we would welcome the opportunity to work closely with them as they implement the bill.”
Collaboration With AACR
In a related development, PanCAN has been working closely with AACR in designing an agenda for accelerating progress against pancreatic cancer.
The two organizations held a meeting on the “progress and challenges” in pancreatic cancer at Lake Tahoe June 18-21, forming a joint Scientific Review Committee. The meeting’s co-chairs included Daniel
Von Hoff, of Translational Genomics Research Institute, Dafna Bar-Sagi, of New York University School of Medicine, Chi Van Dang, director of the University of Pennsylvania Cancer Center, and Cold Spring Harbor’s Tuveson.
The review committee formed by PanCAN and AACR “will oversee a new grant opportunity that provides a 1-3 year grant of $1,000,000 to an existing, multi-institutional, clinical or translational research project within the pancreatic cancer research community,” said AACR CEO Margaret Foti in a recent letter to a scientist who agreed to serve on the committee.
The text of Foti’s letter follows:
The current grant review and selection process is as follows: Letters of Intent (LOI) will be accepted between late August and early October. Once all submitted LOIs are reviewed by AACR staff for eligibility and compliance, committee members are directed to access proposal CENTRAL to view the LOIs and select those most suitable to review based on their scientific knowledge.
Committee members must also indicate whether any LOI constitutes a conflict of interest. Each LOI will be assigned to at least three committee member reviewers that will have approximately three weeks to complete their critiques. Once the LOI reviews are completed, AACR staff will compile the scores and comments, and provide the results to the committee.
A conference call with the committee will then be held to select the applicants worthy of proceeding to the full application phase (25% of the LOIs). We anticipate this call to be held in late November/early December. Once all full applications are received, the application review period will begin in January of 2013. A second teleconference will then be held in February to select the finalists as well as primary and alternate grantees.
For the remainder of your two-year term, based on confirmation from the award supporter, this grant may be launched once more with the review and selection process occurring between October 2013 and February 2014. Throughout this time, AACR’s Scientific Review and Grants Administration staff will provide full support and guidance to both you and the committee.
Please note that committee members may be called upon in the future to review selected grantees’ annual and final progress reports and possibly to provide feedback to the grantees on his/her project. Also, selection committee members are prohibited from applying for or participating on any applications for this grant throughout the course of their term.
The text of the bill: http://thomas.loc.gov/cgi-bin/query/z?c112:H.R.733:
The House and Senate versions of the bill, H.R.733 and S.362, are identical.
