When Autism Speaks the NIH obeys?

Two articles from the journal Nature. It's sad to think of children dying of childhood leukemia because some funding was siphoned off leukemia research to satisfy some screaming antivax politician who wants more research done on autism and vaccines. Many of the "autism advocate" parents are more than happy to tell politicians and others that autism is worse than cancer. Autism Diva has seen kids on chemotherapy in hospitals, in person. Autism is not worse than cancer. Having an autistic child is not worse than having a dead child.

Special report
Autism Speaks: the United States pays up

Abstract
In recent years, autism has become the golden child of the fund-raising circuit. Meredith Wadman looks at a US public-relations success that is driving research funds and expertise towards this childhood condition, and asks who is missing out.

Toni Braxton, Matthew Broderick, Bill Cosby -- perhaps not names you would associate with social or communication difficulties, and yet in one night, they helped raise $1.45 million at a fund-raiser for research into one of the least-understood disorders affecting children: autism.

Star-studded events such as that fund-raiser at New York's swanky Lincoln Center in April are the latest front of a public-relations battle to raise money for research into childhood diseases. In the United States, it's a battle that autism seems to be winning.

"I was sitting in the nosebleed section and it was $1,500 a ticket up there," recalls neurologist Gary Goldstein, president of the Kennedy Krieger Institute in Baltimore and chair of the scientific advisor committee for Autism Speaks, the group that organized the event. Goldstein recalls a 2005 Hollywood fund-raising concert featuring Jerry Seinfeld and Paul Simon."Every four months there's something like that, and that doesn't count all the golf classics," Goldstein says.

In less than three years, autism has emerged from relative obscurity on the fund-raising circuit to become the disease du jour. The 1,000 biggest US private foundations nearly doubled their autism-related giving to $2.7 million between 1998 and 2005, according to the New York-based Foundation Center. The Autism Society of America, the founding father of autism groups, has also seen its budget grow steadily to $20 million. More striking are the contributions of two newcomers: Autism Speaks alone raised more than $33 million in 2006,
its first full year of operation, and it is well on target to meet its goal of $55 million in 2007. And the lower-profile Simons Foundation, which began funding autism research in 2003, has spent more than $40 million and plans to spend another $100 million over the next five years.

The commonly held belief that the prevalence of autism is rising may be partly responsible for the cash flow. ...

But in the past two years, two people have had perhaps a greater impact on autism's visibility -- and coffers -- than any other single force, essentially rewriting the book on how to raise money for disease-specific research: Bob and Suzanne Wright, who founded Autism Speaks in 2005 after learning that their grandson had the poorly-understood condition.

Bob Wright, then chairman and chief executive of media and entertainment conglomerate NBC Universal, provided his business savvy and his extensive connections to a Who's Who of American stardom. He also brought urgency. "I know how devastating this problem is to families. I am reminded of it daily," says Wright.

In 2004, Goldstein presented the Wrights with a graph contrasting charitable autism spending, then at about $14 million, with the $100-million-plus raised by disease groups for juvenile diabetes, muscular dystrophy, childhood cancer and cystic fibrosis -- all of which are significantly less prevalent. "To move this field forward, we need that kind of money," Goldstein told them. Less than a year later, in February 2005, the Wrights launched Autism Speaks, which merged with the National Alliance for Autism Research and Cure Autism Now, in 2006 and 2007 respectively.

Today, the organization has 141 employees and thousands of volunteers in 41 states. "At the rate the group is growing, I'd be shocked if five years from now it's not raising $100 million a year," observes Goldstein.

Although there are other private groups focused on research and treatment, in the past the public's awareness of autism has not been as high as it could have been. "We were really out there by ourselves on awareness," says Wright. A public-relations coup changed that: last year, the group won a three-year campaign of free, slick publicity from the Advertising Council.

The New York-based Simons Foundation is a very different player. Formed by Jim Simons, a mathematician and hedge-fund billionaire who has an autistic daughter, the organization is spending tens of millions funding individual researchers. It's also financing a $12-million collection of genetic information and cell lines from 2,000 affected families that will be made freely available to other scientists. The database is called the Simons Simplex collection, and is to be stored at Rutgers University, New Jersey.

In 2006, Simons recruited Gerald Fischbach, the former director of the National Institute of Neurological Disorders and Stroke, as his scientific director. Taking the job, says Fischbach, was "a chance to focus on just the very best science".

Terrific scientists

According to Fischbach, the swell of interest in the disease has been boosted by new genetic techniques and imaging tools. He notes that 11 of the 18 scientists to whom the foundation will soon make three-year grants of up to $3 million are new to the field. "You couldn't attract terrific scientists if there were no good ways to approach the disease," Fischbach says.

There is a dark side to the surge in autism funding.

The upward trend in money and interest has been mirrored at the National Institutes of Health (NIH), where funding for autism research has grown from $22 million in 1997 to $108 million this year -- with 16% of that growth happening since 2003, while the rest of the NIH has been essentially flat-funded.

At the CDC, which in 2000 spent just over $1 million on epidemiological research on autism, spending has soared to $15.1 million in 2007. Even the Department of Defense is getting in on the act, spending $7.5 million on autism research this year, largely thanks to Autism Speaks, which successfully lobbied Congress for the money on the grounds that autism in military families costs more than $200 million to treat annually.

The most recent legislative achievement of the autism-research advocacy groups is the Combating Autism Act, which was passed in December 2006 after intense lobbying. It guarantees that the spotlight will stay on the disease, requiring for the first time that the government draw up an annual strategic plan for autism research, identifying gaps and opportunities, all with the input of affected families.

"The advocacy groups here have had an enormous impact," says Tom Insel, the director of the National Institute of Mental Health, which with its $62-million spending in 2006 made the largest financial contribution of the five NIH institutes that fund autism research. Insel, who also chairs an inter-agency committee that coordinates autism research for the federal government, says of the advocates: "They hold our feet to the fire, and make sure we are relevant and accountable."

But something distinguishes autism advocates, especially Autism Speaks. "They are actually raising very substantial amounts of funds to do a lot of the science themselves," adds Insel.

Bigger share

That fund-raising prowess has generated both admiration and envy from disease groups that could be considered competitors. For example, since the doubling of the NIH budget ended in 2003, NIH's cystic fibrosis funding has fallen from $117 million to $85 million; funding for childhood leukaemia from $70 million to $53 million; and funding for Down's syndrome, which is about one-fifth as prevalent as autism, from $23 million to $13 million.

"Why is Down's syndrome funding low? Autism I think is a big contributor," says Jon Colman, the chief operating officer of the National Down Syndrome Society. "It's dominating priorities."

Fischbach concedes that what's good for autism has to be bad for others that rely on NIH funding. As long as the biomedical agency's budget continues to be frozen year after year, he argues, pouring new NIH millions into autism necessarily means funding less research on some other diseases. "There is a dark side" to the surge in autism funding, he admits.

But other disease groups say that they have benefited from the rise in autism's profile -- some by learning from Autism Speaks' example, and others because autism can coexist with other, lesser-known diseases such as tuberous sclerosis and fragile X syndrome, which causes mental retardation. "People are beginning to talk about the probable genetic basis for most autism. And then often, if not always, they mention fragile X as an example," says Robert Miller, the executive director of the National Fragile X Foundation in Walnut Creek, California, who was pleased when a recent cover story on autism in Newsweek made a passing mention of fragile X.

But despite its new visibility and funding, autism remains a little-understood and untreatable disease, and it is unclear how much this well-managed public-relations exercise will change that. Insel is optimistic, though. "Autism has attracted the attention of the most stellar geneticists and neuroscientists," he points out. "So I'm hopeful."

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Clinical Precision
Disease advocates should influence, but not dictate, research priorities.
Nature.com
August 8, 2007
Disease lobby groups have always made energetic efforts to ensure that the formidable resources of the US National Institutes of Health (NIH) are brought to bear on the health issues that interest them; and that is as it should be. When these resources are expanding, there has been room for most of the participants in the process to feel that they are winning. Now that the budget of the largest research agency in the world is effectively frozen, there are likely to be more losers.

In the case of research into autism - a common but poorly understood developmental disorder - effective advocacy has seen a generous increase in NIH support specifically targeted at the disease, even since the real growth in the agency's total budget came to an end in 2003. Unfortunately, some of autism's success may have come at the expense of research into other children's diseases (see 'Autism Speaks: the United States pays up').

Autism research also garners an unusually strong level of support from private sources, including funds raised by the disease group Autism Speaks and contributed by philanthropists such as Jim Simons, a mathematician and hedge-fund billionaire whose daughter suffers from the condition. This year, these sources will spend some $50 million - roughly half what the NIH is spending on autism research. Typically, disease groups can muster charitable funding that is only a small fraction of what the NIH spends (see Nature 447, 248-254; 2007). That leaves them scrambling for slices of a pie that is no longer growing.

The power of some advocacy groups must be tempered to some degree. Scientists, especially those involved in the basic biomedical sciences, are also scrabbling for a share of these funds. The NIH's success has been built on a tacit accommodation between scientists, who run its 27 institutes and centres on a day-to-day basis, and the public, as represented by Congress. Institutes have been established - often against the scientists' advice - to deal with specific conditions or diseases of particular organ systems. Then Congress has, in large part, left the researchers and physicians in the agencies to pursue their work as they see fit, without indulging in too much micromanagement.

This has enabled the NIH to maintain a formidable reputation for scientific integrity and excellence, while also appearing to be reasonably responsive to patient needs. It has produced an agency that does a great deal of basic scientific research - with unknown and largely serendipitous benefits for the development of drugs and devices - as well as plenty of laboratory and clinical work devoted to particular ailments.

The public is paying for the NIH's annual budget of $29 billion, and it is entirely appropriate that it should energetically articulate its demands of the agency. That is one of the roles of the disease-advocacy groups, and their input, as any NIH institute director will attest, provides invaluable assistance in assigning research directions and priorities. That said, the power of some advocacy groups must be tempered to some degree - especially in today's difficult funding environment. Otherwise, these groups' ability to influence budgets is likely to dominate, to the detriment of both basic laboratory science and of research targeted at diseases that have weaker constituencies.

It falls to the scientist-administrators who run the NIH to work closely with members of the congressional appropriations committees that fund the agency to make sure that this does not happen. Both groups understand the careful political balance that has allowed the agency to thrive; they must act as moderators whenever the more energetic lobby groups are pushing the agency's agenda too far in the direction of one public-health issue at the expense of others.

Regarding Jim Simons autistic daughter, a Wall Street Journal article reported:

Audrey, who is now 19, has a mild form of autism. She started college classes in Manhattan this year...

The ever-so-effective, torch brandishing "advocacy groups" have not only been effective in demanding research into autism's causes they have also been very effective in trying to silence the voices of autistic adults who would like to have a say in how autism is researched and how it is described by researchers. Up until now there have been no autistic adults consulted by the various funding agencies (unless you consider Mr. Simons himself to be one).

It's important to note that key "autism" research findings could come out of research into an unrelated disorder, so demanding that "autism" research be funded is not necessarily going to be the most productive for "autism." As what the NIH/NIMH is funding; there's a big problem at the NIMH where a couple or three politicians have demanded that research be done on what THEY want researched. This seems to have resulted in the truly unethical NIMH funded chelation study that was to be led by Susan Swedo. Swedo's description of why the study was a valid one was hysterical, no make that painful, to read. She wrote that since parents could buy one chelator (illegally) over the counter (via Internet) that that meant they should go ahead and use it for the study it. There was no scientific evidence offered for how the kids in the study would be likely to benefit, and in fact only kids shown not to have toxic levels of mercury were to be enrolled. This study is now basically in the dumpster, having entered a kind of bureaucratic purgatory (a special IRB committee review) when it shall not likely leave, given how awful it is.

Then Swedo is the principal investigator of another hideous NIMH study involving an antibiotic that is supposedly going to reduce some suspected inflammation that no one understands really...but she's going to try to reduce it anyway. That study requires two spinal taps (lumbar punctures). The protocol for that one requires that the child agree that he or she wants to have the spinal tap. For some reason someone decided to put this same frighteningly ignorant woman as the head of the group that is working on Pervasive Developmental Disorders for the next version of the DSM. Autism Diva hopes that this means that Swedo will be kept so busy with the DSM revising that Dr. Swedo won't have time to lay her hands on an actual autistic child in a clinic.

On a related topic, a refreshing read: Scientifically Unsupported Therapies in the Treatment of Young Children with Autism Spectrum Disorders. Free article go to this page click on the red and black Adobe Acrobat logo. One point Autism Diva disagrees with. They need to look at Facilitated Communication again, as it was over-hyped in the 1980's and some very serious abuse arose from it's use by unscrupulous or mentally unbalanced people, but rational people are using various forms of it now, and there doesn't seem to be a continuing pattern of "facilitators" abusing it now, and repeatedly, kids who start out needing obvious help are taught to type more and more independently, which has always been the goal of facilitated communication. There's a good description of it's use in "Reasonable People," the recent book about DJ Savarese by his father.



Autism Diva
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