Charitable Choice: Research Grants–Juvenile Diabetes Research Foundation – National Journal
Tue, 8 Mar 2005
The Washington Post reports that conservatives have crafted a “bioethics agenda” on cloning and stem cell research. Such an agendaq determines what research the government will and will not fund. http://www.washingtonpost.com/wp-dyn/articles/A15569-2005Mar7.html
Below is an excerpt of a fascinating article in the National Journal about how competing scientists’ interests can confound the best laid efforts. The case example is a highly successful, well organized research advocacy foundation, the Juvenile Diabetes Research Foundation (JDRF), whose mission is to find a cure for juvenile (type 1) diabetes. JDRF attempts to achieve a balance of power on its board of directors, between science professionals and parents of children who suffer from diabetes. Their reviewing process for awarding researh grants is professional, and reaches out to seemingly independent outside scientists from top academic institutions. But an innovative research approach that might result in a breakrthough treatment for diabetes may be impeded because of underlying competing financial interests, and politics.
The catalyst for the National Journal article was the denial of a grant to Dr. Denise Faustman of Harvard whose divergent research approach is unnerving the scientific community. She wants to conduct a clinical trial using an approach that was successful in mice: she proposes to use organ-regenerative therapies to cure diabetes rather than introducing embryonic stem cells which are an arduous, painful and, so far, unsuccessful approach. But her research, using patients’ own stem cells, conflicts with the work of most other scientists who are locked into the promotion of embryonic stem cell research – for which JDRF has lobbied aggressively.
Dr. Faustman says: “Four years ago, nobody believed that adult organs [could] regrow.” She calls her results “a major paradigm shift.” This discovery, she argued, affects other scientists’ research and business plans, especially those who are racing to create vats of embryo stem cells for transplantation into diabetes patients. The stakes are high:
“If Faustman’s work is proved valid, it could lead to a cure for juvenile diabetes at modest cost, generating incalculable benefits for millions of people around the world. It could greatly lower the demand and the revenue for other therapies, such as the Edmonton Protocol, or for diabetes-related amelioration and maintenance products. The technique could also cut revenues for pharmaceutical companies, which take in roughly $1.3 billion a year in the United States from the sale of insulin-related products. Much of the revenue from the new treatment would likely go to Faustman’s own company, although hospitals and doctors would make some money from her treatment. And such a breakthrough would likely affect funding for rival researchers in the $500 million-a-year diabetes-research sector, including those active in the JDRF. They would likely see cuts in their research funding, and would perhaps migrate into new areas, where they would face uphill competition from researchers already established in those disciplines. This shift would be painful, partly because scientists are understandably reluctant to discard years of work and hopes. “If you have a belief, you pursue it, until it becomes totally and completely, undeniably wrong,” said Joel Habener, a JDRF-funded researcher at Harvard Medical School.”
“Faustman’s therapy could also have implications for research beyond diabetes, because it seemingly works by allowing the body to use its own stem cells to repair damaged organs once the disease is blocked. Numerous other autoimmune diseases — multiple sclerosis, lupus erythematosus, Lou Gehrig’s disease (or ALS), muscular dystrophy — might also be treated in the same fashion, said Larry Raff, president of the Newton, Mass.-based Autoimmune Disease Research Foundation, which supports Faustman’s approach.
Her approach clashes with that of advocates of embryo-stem-cell research, who already face opposition from a political coalition maintaining that adults’ stem cells are a faster and more ethical alternative to embryo stem cells. Proponents of embryo-stem-cell research frequently cite diabetes — and diabetic children — as a primary reason for using embryo stem cells. But backers of adult stem cells note that Faustman’s research could make that political argument moot if an adult’s own stem cells can indeed repair diseased organs.”
The article notes that other researchers who have reported breakthroughs using adult stem cells have similarly faced a funding drought. The examples cited: Israeli researchers at the Technion Institute of Technology have been injecting BCG (the compound Faustman uses) into a person who was expected to develop diabetes. That patient has remained free of diabetes for seven years. But the researchers have been unable to gain additional funding for tests on more people. In Los Angeles, Dr. Michel Levesque, the chief of neurosurgery at Cedars Sinai Medical Center, used one patient’s stem cells in 1999 to nearly eliminate his symptoms of Parkinson’s disease, but has since failed to win financial backing for a formal clinical trial of the therapy. And Norman Ende, at the University of Medicine and Dentistry of New Jersey, says he has successfully treated diabetic mice with stem cells from umbilical cords, but has yet to win any support even in his university — not even permission to issue a press release.
The article describes how even well informed parents on the JDRF board are at a disadvantage vis a vis scientists whose multiple financial interests and research agendas may conflict with theirs. For all its lobbying and fundraising successes, “the JDRF can claim few clear achievements in curing or even treating juvenile diabetes.” One former JDRF chairman of the board (from 1992 and 1993) J. Richard Munro (a former top Time Warner executive) grasped this inherent conflict: “For 25 years, ever since my two sons became diabetic, JDRF people have said, ‘We’re making real progress,’ and my answer is, B.S. There’s nothing that’s changed for my sons since they became diabetic.” He realized that “‘Blind faith’ is an awful thing to say, but that’s pretty much how I thought [JDRF’s grant-giving] worked. You hoped it was being spread to the right people, but there was no way you could be sure.” He left JDRF.
(Similar reasons–‘blind faith’ in biological psychiatry and its drug-centered agenda in clinical practice and research – led me to leave the National Alliance for the Mentally Ill. NAMI’s leaders avoid taking a hard look at the dismal record of failed theories, failed treatments, worsened recovery rates, and increased costs.. Like JDRF, NAMI lobbying efforts are influenced by researchers whose own careers and income have been greatly enhanced by those efforts. But the billions of dollars spent on psychiatric research and psychotropic drugs have failed to improve the quality of life mentally disabled people).
Parents at JDRF ostensibly control the organization and decide which research to fund, but a tell-tale sign of the inordinate influence that JDRF affiliated scientists exert on grant awards, is the fact that the chair of the research committee, who is on the board, did not even meet with Dr. Faustman or invite her to present her proposal at a 2004 workshop. Instead, scientists who are hostile to regenerative research, and have competing financial ties to biotech companies that favor embryonic stem cell research, were invited presenters. JDRF does not disclose the financial interests of scientists.
This is an intelligent, and provocative article providing insight into the politics and economics of research grant awards. (entire article available in pdf)
Contact: Vera Hassner Sharav
© National Journal
Science – Charitable Choices
In 2001, Denise Faustman, an associate professor at Harvard Medical School and the director of the immunobiology lab at Massachusetts General Hospital, announced a surprising laboratory discovery in her research on diabetes. Faustman had injected a compound called BCG into diabetic mice, expecting that it would help repair their haywire immune systems. Diabetes occurs when the immune system goes awry and gradually destroys the insulin-producing islet cells in the pancreas. The BCG injection worked as Faustman had expected. But upon dissecting the animals, she also found that, in most of the mice, the pancreas was restored to health.
Faustman reported her news-making results in a top-tier publication, The Journal of Clinical Investigation. In 2003, she published an update in the magazine Science, describing evidence that in mice, stem cells migrated from their spleens and repaired their pancreases. Faustman next won the backing of some top diabetes researchers; boosted the cure rate in diabetic mice; got approval from the Food and Drug Administration for limited testing of BCG on people; and applied for grants to further her research. She also got three rejection notices in the mail from the Juvenile Diabetes Research Foundation. “We don’t fund work that does not meet the standards of our lay and peer reviewers,” Peter Van Etten, president and CEO of the JDRF, told National Journal. “The fact that this [proposed treatment] is popular, and there is great support for it, is not justification to violate the policies we have established over many years.”
As the world’s leading charitable fundraiser and advocate for research on juvenile diabetes, the New York City-based JDRF doled out $93 million in 2004 to help researchers find a cure for a disease that afflicts 1.3 million American children. An accomplished researcher like Faustman would seem to be an ideal candidate to receive some of that financial support.
But there is a complicated back story to the rejection letters that Faustman received from the foundation — a story that involves scientists’ collective and individual ambitions; political disputes over stem-cell research; $3 billion in California taxpayer money; and the balance of power within the JDRF between the science professionals on the one hand and, on the other, the lay members whose children suffer from diabetes.
As is true elsewhere in the medical-research community, JDRF’s grant-making process fosters furious competition among research advocates for money. JDRF board member Margery Perry, a nonscientist and the chair of the research committee, calls the process “a mirror of human nature. You see some scientists that are extremely collaborative and very sharing, and you see the exact opposite.”
The Face of a Child
By Washington standards, the JDRF is a highly successful operation. The organization began with only a few parents when it was launched in 1970, and now boasts thousands of volunteers in 100 locations worldwide. The 39-person board of directors and several advisory committees oversee the sophisticated lobbying efforts and fundraising programs that allowed the JDRF in fiscal 2003 to award 500 grants to researchers in 19 countries, and put $30 million into communications and education efforts. Most of that research funding went to university-affiliated scientists in the United States; a remarkably low 10 percent was spent on operating costs and fundraising expenses. And the JDRF gets high marks for visibility. Actress Mary Tyler Moore is the group’s international chairman, and other celebrities have also helped with fundraising and raising public awareness of juvenile diabetes.
Juvenile, or Type 1, diabetes is a gene-related disease in which the immune system attacks the insulin-producing cells in the pancreas. Without a well-regulated supply of insulin, other organs begin to fail, eventually causing blindness, kidney failure, and death. Although there’s no cure, many scientists, as well as pharmaceutical, biotech, and medical companies, have developed knowledge and products that help patients regulate or slow the disease through a regimen of diet, exercise, pills, insulin injections, and blood-sugar monitoring. Adult, or Type 2, diabetes, which afflicts some 16 million Americans, manifests later in life and is often related to poor diet and obesity. The direct financial cost of all diabetes care in the United States was $92 billion in 2002, according to a study by the Lewin Group.
One of the JDRF’s most visible weapons in the fight against juvenile diabetes is the afflicted children. They’re young and often outwardly healthy. Many of their parents are educated and wealthy, and they know how to attract media attention, solicit donations, and build political influence. “You would be hard-pressed to look into the face of a child — as legislators do — and tell them you do not support their hope for a cure for diabetes,” said JDRF spokesman Peter Cleary. The JDRF organizes a “Children’s Congress” and a “Promise to Remember Me” campaign, in which kids and their families visit Capitol Hill lawmakers. These visits have been “enormously successful,” said JDRF board member Maureen Barunas, whose teenage son lobbied Congress in 2001. “The point is to bring their personal stories, to put a face on the story of diabetes.”
In recent years, the JDRF, which employs three staff lobbyists in Washington, has helped persuade Congress to give the scientists at the National Institutes of Health an additional $750 million over five years for investigating juvenile diabetes. And JDRF’s adult volunteers are now raising almost $150 million per year in charitable donations; in 2004, JDRF’s executives set a higher fundraising goal of $200 million a year. Moreover, Congress passed a law in 2004 intended to promote Medicare funding for a new form of diabetes therapy — the Edmonton Protocol — that several medical centers around the country now provide. The JDRF also works with other Washington-based advocacy organizations. In the campaign for embryo-stem-cell research, for example, the foundation has played the leading role alongside scientists, universities, and biotech companies that try to persuade others of their belief that embryo stem cells can be used to understand the workings of the human body. Rep. Michael Castle, R-Del., a supporter of embryo- stem-cell research, told National Journal that the effort is “the best lobbying campaign I’ve ever seen.” The scientists rallied by the JDRF are particularly influential: “Their words are my words,” said Castle.
So far, the broad campaign has persuaded President Bush — despite strong opposition from his social-conservative base — to allow federal funding for a limited kind of embryo research; has won over a slew of legislators who have impeccable anti-abortion credentials; and has gained near-uniform backing from Democrats and socially liberal Republicans. The push for embryo research has generated a wave of media coverage, and in November it helped pass a California ballot initiative that will pump $3 billion into the state’s universities and biotech centers for stem-cell research.
For all its lobbying and fundraising successes, however, the JDRF can claim few clear achievements in curing or even treating juvenile diabetes. In interviews for this article, JDRF officials repeatedly pointed to their support of the recently developed Edmonton Protocol, in which pancreas cells from cadavers are transplanted into patients with a severe form of diabetes. But the protocol’s co-developer, James Shapiro, a researcher at the University of Alberta in Canada, said that the JDRF had played no direct role in its development. The protocol, moreover, is of modest benefit because of several problems: Not many cadaver cells are available; the operation is very expensive; the patients suffer debilitating side effects from anti-rejection drugs; and after a few years, the transplanted cells die. About 300 patients have received the treatment so far.
Former top Time Warner executive J. Richard Munro (no relation to the author) chaired the JDRF board in 1992 and 1993 but is no longer involved with the foundation. For 25 years, ever since his two sons became diabetic, he complains, JDRF people “have said, ‘We’re making real progress,’ and my answer is, B.S. There’s nothing that’s changed for my sons since they became diabetic.” Still, in science, lack of success is not conclusive evidence of failure. Diabetes is a difficult disease, and lots of funding, much trial-and-error, the generous use of partial therapies, many disappointments, and periodic false hopes are to be expected before a cure appears. Even when a major therapy is first developed, there’s likely to be much professional disagreement before the therapy has proved effective on many patients. That reality gives JDRF’s nonscientist lay leaders the difficult task of navigating a path guided by advice from competing advocates.
Lay members repeatedly say that their goal is a cure — not the scientific knowledge sought by university-affiliated scientists, and not the revenues sought by companies selling products that ameliorate the effects of diabetes. This goal, however, cannot be accomplished by JDRF’s laypeople alone, because they are dependent on the corps of diabetes scientists. When a new scientific claim is raised, “we can only vet it by asking other independent scientists [and] our staff, who don’t have biases,” said JDRF board member Roy Smith, a former international banker at Goldman Sachs and a finance professor at New York University who headed the JDRF planning committee in 2002. Said former board member Munro: “There’s no way that I could second-guess a scientist — it would be foolhardy…. ‘Blind faith’ is an awful thing to say, but that’s pretty much how I thought [JDRF’s grant-giving] worked. You hoped it was being spread to the right people, but there was no way you could be sure.”
When it comes to their professional culture and economy, diabetes scientists are no different from other scientists: They are simultaneously allies and rivals. They compete for prestige and for the resources that generate prestige — grants, publishing opportunities, and patents — even as they cooperate to lobby for greater federal funding and for freedom from federal regulation.
Professional and personal rivalries among researchers can be intense, but are also moderated by their shared dependence on each other for funding, which is typically awarded by peer-review panels of fellow scientists. Diane Mathis, a researcher at Harvard’s Joslin Diabetes Center whose work is funded partly by the JDRF, said, “We do depend [on each other], but we like to think … [that] we’re judged objectively on the science, not on whether our colleagues like us or not.”
Within this community, scientists hold overlapping affiliations with rival universities, companies, cliques, specialties, and preferences. These splits create conflicts, as well as a confluence of interests. To prosper in this professional economy, scientists need a keen eye for peer politics, business, and science. “I think I learned not too long ago that scientists are just about as political as any other group. They have their own agendas,” Munro said.
Faustman is no exception. In her work at Massachusetts General Hospital and at Harvard Medical School, she has written or contributed to more than 100 published papers, has reviewed grant requests for the JDRF, and chairs the board of the Society for Women’s Health Research. In 1989, she helped form a company that was sold in 2003 for $40 million. Also in 2003, she helped create Keel Pharmaceuticals, to commercialize her diabetes-related technology. Faustman promotes her work to the parents of diabetic children and the media.
The parents and lay members of JDRF, however, have a much narrower focus than the scientists do. “We have one agenda,” Perry said. “We want a cure now for our kids and loved ones.”
Balancing scientists’ multiple priorities with lay members’ single-minded goal of finding a cure would be a challenge for any organization dedicated to fighting a disease. “All you can do is to try to minimize all those things that creep in,” said Moira Murphy, a science adviser to Diabetes UK, a British patient-advocacy group akin to the JDRF. “You can never get rid of them.”
The JDRF seems well prepared to manage such a challenge. “In every step of the process, we have an awful lot of committed volunteers that are connected to the disease and have no axes to grind, other than funding the best research possible,” said lawyer Robert German, the father of a diabetic son and the chairman of the JDRF board. “I would not tolerate anything going on, other than what’s contributing to a cure.”
Far more than nearly all other disease-advocacy groups, the JDRF board and its committees are steered by laypeople, including top executives, lawyers, and other professionals, plus some executives from the health care sector. Many of these lay members have educated themselves about diabetes through their own work at the JDRF or through outside study, and this knowledge puts them in a position to spar with scientists over various issues. JDRF panels of laypeople review grant requests from scientists after panels of other scientists have ranked them in quality.
All laypeople and scientists must disclose possible conflicts of interest, and must remove themselves from any decision that could affect their personal interests, Van Etten said. For example, at the JDRF’s twice-yearly review sessions, perhaps as many as 20 scientists exclude themselves from reviews of particular grants because they have a potential conflict of interest, according to Van Etten. Such potential conflicts are to be expected, he said, because the JDRF seeks out expertise wherever it can be found. “There is self-policing that takes place by the scientists.”
Sometimes, the lay review committee does override the scientists’ funding recommendations, according to Van Etten, who said he could not offer specific examples because of the desire to protect the confidentiality of the process. Perry, however, offered one example from a decade ago, in which lay members backed a proposal calling for the nation’s small supply of insulin-producing cadaver cells to be shared by a wide range of researchers. The scientists on the peer-review panel had given the proposal a very low rating, but the project is now established, she said. Laypeople and scientists also differ on policy priorities. Board member Smith said that the organization is “moving away” from university-based research efforts because “we want to get close to the clinical-trials phase of this stuff. We are tired of doing it to mice. We want to do it to humans.” Board members also say that the JDRF needs to cast a wider net than the scientists’ conventional wisdom. “We need to look beyond what people are telling us is possible and take some risk,” Barunas said. But, according to a recent article in Forbes that prasied the JDRF, the foundation’s policy is to “achieve more by moving toward fewer but bigger fixed-term collaborations with brand-name research institutions like Columbia and Harvard.” JDRF spokesman Cleary said that the foundation began to develop such relationships several years ago, “and they continue to expand today.”
To minimize conflicts of interest among board members, JDRF’s directors also disclose their various interests. For example, the agenda-setting portfolio committee is chaired by Leo Mullin, the father of a diabetic child. Mullin, the former head of Delta Airlines, is on the board of pharmaceutical company Johnson & Johnson. Another JDRF board member, Charles J. Queenan III, is the No. 2 executive at Amaranth Bio, a diabetes-research firm. He joined the firm last year after serving as the chair of the JDRF research committee, a role filled by Perry since 2004.
The Faustman Challenge xxxxx cut xxxx
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