Health – Page 100 – artdiamondblog.com

FDA Hurdles Block Widespread Use of Baby-Saving Drug

(p. A1) BOSTON — Like thousands of children in the U.S., Maggie Leaver has short bowel syndrome. These children can’t absorb enough nutrients from food, and some need intravenous feedings to survive.

A baby’s digestive system can adapt over time, but that may take months or years. Many of these babies can’t wait. For reasons not fully understood, children put on intravenous nutrition may suffer liver damage. Some require liver and small bowel transplants, risky procedures that don’t always work. Others die waiting for a transplant.

In July, in a paper in the scientific journal Pediatrics, researchers at Children’s Hospital Boston reported on a small study that suggested a promising treatment. They found that by switching from the standard intravenous formula to a different kind — called Omegaven — babies weren’t progressing to liver failure. Omegaven, used in Europe for adults, isn’t approved in the U.S. and is considered experimental treatment.

"The kids aren’t dying anymore," says Mark Puder, a pediatric surgeon who was lead investigator on the study. "We think we have a good treatment."

But Dr. Puder’s effort to get Omegaven widely used in babies has put him in an unusual conflict with the German company that developed the drug. Fresenius Kabi AG, which makes Omegaven, says it isn’t interested in bringing the drug to the U.S. market. The company says it doesn’t agree that Omegaven is the best drug for these babies and has a new product that it believes is better.

In 28 of 29 babies treated with Omegaven so far at Children’s Hospital, Dr. Puder says they were able to stop further liver damage — and damage that children already incurred seemed to improve. Some babies who were switched to Omegaven rebounded enough that they were taken off the waiting list for an organ transplant. At one point, Maggie Leaver’s condition deteriorated so much that her surgeon thought she was going to die. Now the 18-month-old is thriving at home in Hingham, Mass.

. . .

Mr. Ducker says the company’s new product, called SMOFlipid, "presents a better option for pediatric feeding." The company believes the new product does contain all the essential fatty acids babies need and can be used on its own.

Fresenius Kabi says it doesn’t want to invest the resources required to test both products for approval by the U.S. Food and Drug Administration. It hopes to eventually sell the new product in the U.S., Mr. Ducker says, although no timetable has been set and no trials are under way.

. . .

Because Omegaven is considered experimental in the U.S., if hospitals want to try it, they have to ask permission from the FDA for each individual patient. The FDA has regulations that enable doctors to use experimental drugs in certain (p. A15) emergency situations.

If hospitals obtain the required permissions, they must then find a way to buy the drug on their own, since insurers typically won’t cover Omegaven because it’s experimental. The cost can run from $50 to $100 a day per patient. At Children’s Hospital Boston, the surgical department has already spent close to $100,000 to buy Omegaven for babies.

. . .

Dr. Mooney says he wrestled almost from the beginning about whether to put Maggie on Omegaven. He knew about Dr. Puder’s results, which he calls "amazingly great," but the number of children treated was still small. He worried about adverse effects. "It is so easy to get caught in the hype of new things," Dr. Mooney says. Maggie was already fragile. What if he put her on Omegaven, he says, "and there was a horrible side effect that could tip her over the edge?"

But when standard therapies failed, he felt "there was nothing else to do." Given that the treatment is experimental, Dr. Mooney says he believes it was right to wait. But he also feels Omegaven has made a difference. "Five years ago, every single one of the kids taking Omegaven would be dead by now, Maggie included," he says.

For the full story, see:

MARCUS, AMY DOCKSER. "Different Rx; A Doctor’s Push For Drug Pits Him Against Its Maker; Dr. Puder Thinks Omegaven Is Best Option for Sick Babies; Company Prefers New Product Turnaround for Little Maggie." Wall Street Journal (Mon., November 13, 2006): A1 & A15.

People Want to Live (So There, Leon Kass)

Source of edited screen capture: http://www.nytimes.com/gst/mostemailed.html

In an earlier post, Leon Kass was quoted as opining that life is better when it is short.

The table above is from the New York Times list of most emailed articles within the last 24 hour period. The listing above was for the period ending at 1:00 PM CST on Thursday, November 2, 2006.

Notice that of the top four articles, three of them have to do with the study showing that reversatrol may lengthen life.

Resveratrol May Slow Aging

Harvard Medical School antiaging researcher Dr. David Sinclair. Source of image: http://webweekly.hms.harvard.edu/archive/2003/8_25/index.html

If our institutions sufficiently allow and reward entrepreneurial innovation in health care, substantial gains in the length and quality of life are possible. (In the WSJ article passages that follow, "CR" stands for "calorie restriction.")

(p. A1) Now a coterie of scientists and biotech ventures are rekindling interest in CR as they try to mimic its antiaging effects with medicines. It is still a highly speculative quest, and many researchers fret that it hasn’t completely shaken its association with centuries of dubious nostrums to slow aging, from inhaling virgins’ breath to eating gold to implanting monkey glands.

Much of the new focus is on a substance in red wine called resveratrol. The interest in it started three years ago when a group led by Harvard Medical School biologist David Sinclair reported that it boosted yeast cells’ life span by 70% via a mechanism resembling CR. He later co-authored a study showing that it also boosts life span in fruit flies and roundworms. But his tendency to make bold leaps based on tentative data has also sparked intense controversy. One big question: Does he really understand the workings of CR well enough to mimic them in a drug?

Last spring, Italian scientists reported that resveratrol boosted life span more than 50% in a kind of short-lived fish. Intriguingly, fish on resveratrol had much faster swimming speeds as they aged, and spent far more time moving around, than did undosed control fish.

At least two groups of researchers are now testing whether resveratrol can extend life span in mice — the first such studies in mammals. At a meeting of the American Aging Association in June, Dr. Sinclair and colleagues presented preliminary results from a study showing that resveratrol had "CR-like protective effects" against the buildup of fatty deposits in the livers of mice on high-calorie diets. That suggests that resveratrol could lead to new drugs for diseases of aging associated with rich diets, such as adult-onset diabetes.

A company that Dr. Sinclair co-founded in 2004, Sirtris Pharmaceuticals Inc., of Cambridge, Mass., has begun testing a resveratrol-based drug in diabetic patients. It has raised $82 million from venture capitalists, a hefty sum for an early-stage biotech. . . .

. . .

(p. A11) Dr. Guarente recalls that Dr. Sinclair, who came to MIT in 1995 to do post-doctoral studies, breezed into his lab as if out of a Crocodile Dundee movie, greeting everyone with a cheery, "Hello, mate." The eldest son of parents who both worked in medical diagnostics, he was known in high school as a talented class clown and risk-taker, a kid who aced science classes but got in trouble for setting off minor explosions in chemistry lab. The idea of taking part in unorthodox, high-risk studies on aging suited him.

. . .

. . . , Dr. Sinclair joined forces with a researcher at the National Institute on Aging, Rafael de Cabo, to plan one of the ongoing studies of resveratrol in mice. But he had a problem: He lacked the $20,000 needed to buy mice. Then he got a call out of the blue from Tom LoGiudice, foreman at the U4EA ("euphoria") Ranch near Thousand Oaks, Calif. Mr. LoGiudice had phoned on behalf of the ranch’s owner, Harman Rasnow, who was considering taking resveratrol pills and wanted to know more about them. When Mr. LoGiudice heard about Dr. Sinclair’s problem, he arranged for his boss to talk directly to the researcher. "I have an 85-year-old passion for longevity," says Mr. Rasnow, pinpointing his age. "David sounded like he was really onto something. So I told him, ‘I’ll send you a check for $20,000.’ "

Dr. Sinclair later got another call from Mr. LoGiudice, this time inviting him to make a pitch for funding to one of Mr. Rasnow’s wealthy acquaintances, Paul Glenn, a venture capitalist and a longtime supporter of research on aging. After Dr. Sinclair did so, the Glenn Foundation for Medical Research in Santa Barbara, Calif., awarded $5 million to Harvard Medical School to launch a center on the basic mechanisms of aging with Dr. Sinclair as its founding director. Now plans are afoot to expand the center into a leading institute on aging, says Mr. Glenn, with start-up funding of $75 million to $100 million.

For the full story, see:

DAVID STIPP. "Youthful Pursuit; Researchers Seek Key to Antiaging In Calorie Cutback A Controversial Hypothesis Draws Scientists, Investors; Will It Work in Humans? Fighting Fat in Lab Mice." Wall Street Journal (Mon., October 30, 2006): A1 & A11.

(Note: ellipses added.)

The NBC nightly news on Weds, Nov. 1, 2006 ran a brief, but nice story, reported by Robert Bazell, on the basics of this: http://www.msnbc.msn.com/id/15511128/

“Come With Me, If You Want to Live”

Schumpeter famously stated that creative destruction is "the essential fact" about capitalism. Was he right?

To determine what is "the essential fact" you need to first answer the question "essential for what purpose?" If the purpose is "life, liberty, and the pursuit of happiness" then I think you can show that creative detruction is indeed the essential fact about capitalism; in the key sense that with creative destruction you have a form of capitalism that is best able to enhance "live, liberty, and the pursuit of happiness."

The Terminator famously said "Come with me, if you want to live!" ("Terminator 2: Judgment Day," 1991). Life is a choice. You can choose death instead. Most people, most of the time, choose life. But there are examples of choosing death. E.g., Leon Kass, an oft-quoted "expert" on medical ethics issues, is against current efforts to lengthen the human life span:

(p. D4) While an anti-aging pill may be the next big blockbuster, some ethicists believe that the all-out determination to extend life span is veined with arrogance. As appointments with death are postponed, says Dr. Leon R. Kass, former chairman of the President’s Council on Bioethics, human lives may become less engaging, less meaningful, even less beautiful.

“Mortality makes life matter,” Dr. Kass recently wrote. “Immortality is a kind of oblivion — like death itself.”

That man’s time on this planet is limited, and rightfully so, is a cultural belief deeply held by many. But whether an increasing life span affords greater opportunity to find meaning or distracts from the pursuit, the prospect has become too great a temptation to ignore — least of all, for scientists.

“It’s a just big waste of talent and wisdom to have people die in their 60s and 70s,” said Dr. Sinclair of Harvard.

(And there’s the occasional hermit, like the unibomber, who chooses to live a brutish life without electricity and indoor plumbing.) So long as I, Arnold, and our compatriots, are allowed an island somewhere to peacefully pursue life, I do not much care what Leon and his friends do. My argument, and the book I am writing on creative destruction, are not written for Leon. They are written for all those who choose life, liberty, and the pursuit of happiness.

The NYT quote related to Leon Kass’s praise of mortality, is from p. D4 of:

MICHAEL MASON. "One for the Ages: A Prescription That May Extend Life." The New York Times (Tues., October 31, 2006): D1 & D4.

To FDA, Death is Not a Disease, So FDA Won’t Approve Drugs to Lengthen Life

Source of graphic: online version of the NYT article cited below.

(p. A1) Can you have your cake and eat it? Is there a free lunch after all, red wine included? Researchers at the Harvard Medical School and the National Institute on Aging report that a natural substance found in red wine, known as resveratrol, offsets the bad effects of a high-calorie diet in mice and significantly extends their lifespan.

Their report, published electronically yesterday in Nature, implies that very large daily doses of resveratrol could offset the unhealthy, high-calorie diet thought to underlie the rising toll of obesity in the United States and elsewhere, if people respond to the drug as mice do.

Resveratrol is found in the skin of grapes and in red wine and is conjectured to be a partial explanation for the French paradox, the puzzling fact that people in France enjoy a high-fat diet yet suffer less heart disease than Americans.

The researchers fed one group of mice a diet in which 60 percent of calories came from fat. The diet started when the mice, all males, were a year old, which is middle-aged in mouse terms. As expected, the mice soon developed signs of impending diabetes, with grossly enlarged livers, and started to die much sooner than mice fed a standard diet.

Another group of mice was fed the identical high-fat diet but with a (p. A18) large daily dose of resveratrol (far larger than a human could get from drinking wine). The resveratrol did not stop them from putting on weight and growing as tubby as the other fat-eating mice. But it averted the high levels of glucose and insulin in the bloodstream, which are warning signs of diabetes, and it kept the mice’s livers at normal size.

Even more striking, the substance sharply extended the mice’s lifetimes. Those fed resveratrol along with the high-fat diet died many months later than the mice on high fat alone, and at the same rate as mice on a standard healthy diet. They had all the pleasures of gluttony but paid none of the price.

. . .

For the Food and Drug Administration, if for no one else, aging is not a disease and death is not an end-point. The F.D.A. will approve only drugs that treat diseases in measurable ways, so Dr. Westphal hopes to show that his sirtuin activators will improve the indicators of specific diseases, starting with diabetes.

“We think that if we can harness the benefits of caloric restriction, we wouldn’t simply have ways of making people live longer, but an entirely new therapeutic strategy to address the diseases of aging,” Dr. Guarente said.

For the full story, see:

NICHOLAS WADE. ‘Yes, Red Wine Holds Answer. Check Dosage." The New York Times (Thurs., November 2, 2006): A1 & A18.

(Note: ellipsis added.)

Here is a link to an abstract of the research report in Nature (which, by the way, is usually considered one of the top journals in science):

http://www.nature.com/nature/journal/vaop/ncurrent/abs/nature05354.html

Pill Mimicking Calorie Restriction Would Be Highly Cost-Effective

Source of graphic: online version of the NYT article cited below.

(p. D1) Recent tests show that the animals on restricted diets, including Canto and Eeyore, two other rhesus monkeys at the primate research center, are in indis-(p. D4)putably better health as they near old age than Matthias and other normally fed lab mates like Owen and Johann. The average lifespan for laboratory monkeys is 27.

The findings cast doubt on long-held scientific and cultural beliefs regarding the inevitability of the body’s decline. They also suggest that other interventions, which include new drugs, may retard aging even if the diet itself should prove ineffective in humans. One leading candidate, a newly synthesized form of resveratrol — an antioxidant present in large amounts in red wine — is already being tested in patients. It may eventually be the first of a new class of anti-aging drugs. Extrapolating from recent animal findings, Dr. Richard A. Miller, a pathologist at the University of Michigan, estimated that a pill mimicking the effects of calorie restriction might increase human life span to about 112 healthy years, with the occasional senior living until 140, though some experts view that projection as overly optimistic.

According to a report by the Rand Corporation, such a drug would be among the most cost-effective breakthroughs possible in medicine, providing Americans more healthy years at less expense (an estimated $8,800 a year) than new cancer vaccines or stroke treatments.

“The effects are global, so calorie restriction has the potential to help us identify anti-aging mechanisms throughout the body,” said Richard Weindruch, a gerontologist at the University of Wisconsin who directs research on the monkeys.

. . .

While an anti-aging pill may be the next big blockbuster, some ethicists believe that the all-out determination to extend life span is veined with arrogance. As appointments with death are postponed, says Dr. Leon R. Kass, former chairman of the President’s Council on Bioethics, human lives may become less engaging, less meaningful, even less beautiful.

“Mortality makes life matter,” Dr. Kass recently wrote. “Immortality is a kind of oblivion — like death itself.”

That man’s time on this planet is limited, and rightfully so, is a cultural belief deeply held by many. But whether an increasing life span affords greater opportunity to find meaning or distracts from the pursuit, the prospect has become too great a temptation to ignore — least of all, for scientists.

“It’s a just big waste of talent and wisdom to have people die in their 60s and 70s,” said Dr. Sinclair of Harvard.

For the full story, see:

MICHAEL MASON. "One for the Ages: A Prescription That May Extend Life." The New York Times (Tues., October 31, 2006): D1 & D4.

(Note: ellipsis added.)

Mike Linksvayer is eating a calorie restricted diet. Source of photo: online version of the NYT article cited above.

Medical Cures Going First to the Dogs

One of the dogs cured of melanoma by a new vaccine. Source of photo: screen capture from NBC news report.

Melanoma has taken many human lives, including my father’s on April 15, 2000. Government licensing and regulations reduce competition in medicine and slow the pace of medical innovation. Animal health care is less regulated. Is it an accident that dogs are being cured for melanoma before humans?

Vet Philip Bergman remembers the first time he tried the vaccine in a dog.

"That was a dog that thankfully underwent complete disappearance of his tumor," says Bergman. "It was remarkable, obviously, to us."

Since then, more than 100 dogs have been treated, including Lawana Hart’s Lucky, who last June appeared to have only a few months to live.

For the full report, see:

Robert Bazell. "Treatment for canines with cancer raises hopes; Researchers encouraged by melanoma vaccine’s success on dogs." NBC Evening News Report; online print version updated: 6:36 p.m. CT Oct 26, 2006.

For the video version, go to:

http://video.msn.com/v/us/msnbc.htm?g=d7f603e0-86bb-44db-bad0-524ec79b02c8&f=00&fg=copy

300,000,000 Strong, and Free

Source of graph: online version of the WSJ article cited below.

The Census Bureau tells us that some time in the weeks ahead the U.S. population will reach 300 million. . . .

This demographic milestone is not cause for alarm — as some prophets of doom would have it. Rather, it is cause for celebration. We 300 million Americans are on balance healthier and wealthier and freer than any population ever: We breathe cleaner air, drink cleaner water, earn higher incomes, have more leisure time, and live in less crowded housing. Every natural resource we depend on — water, food, copper and, yes, even oil — is far more abundant today measured by affordability than when our population was 100 million or even 30 million.

Thanks to the rapid pace of technological progress, there’s every reason to believe these resources will be still more abundant when our population reaches 400 million — which should happen about 40 years from now. As the late economist Julian Simon reminded us, thanks to our free market capitalist system, the history of America is one of leaving the storehouse for every successive generation more endowed with wealth, knowledge and natural resources.

For the full commentary, see:

STEPHEN MOORE. "Supply Side; 300,000,000." Wall Street Journal (Tues., October 3, 2006): A26.

(Note: ellipsis added.)

The Missing Pillow: A Lack of Incentives Leaves an Obvious ‘Job’ Undone

In late July, I had an appointment for a treadmill stress-test at Omaha’s Methodist Hospital. They told me the process would be over in an hour, but it took about two hours, due to another patient having some sort of crisis during their stress-test.

They had me put on a gown, they stuck an I-V "dye" drip in back of my hand, and they pasted about six electrodes to my chest, after shaving and applying something like sand paper to the parts of the chest where the electrodes were attached. Then they had me lie on my side on a hard table, to wait. It was very uncomfortable. The first nurse said that there was supposed to be a pillow on the table, but did nothing to obtain one. Every several minutes some technician or nurse would stop in to ask if I was ready for them. (I was always ready.) But it turned out that someone needed to do something to me first, and that person was, I guess, taking care of the crisis next door. At least one of these visitors also mentioned that I was supposed to have a pillow, but did nothing to acquire one. If memory serves, the first nurse came back in, and again mentioned that I was supposed to have a pillow, but again did nothing to obtain one.

These people were all pleasant and friendly. For example, they had a lot of friendly chats amongst themselves, that I could not help but over-hear. (One of them was pregnant with twins, but did not know the genders of the babes-to-be, and so had not yet spent the time to come up with names.)

But two hours later, when the whole process was over, I still did not have a pillow.

A week or two after the test, I received a several page survey from Methodist Hospital asking a bunch of questions about how I thought they had done during the test. You see they really "care" about my opinion. (They also run frequent, slick TV ads about how much they "care.")

Marketers, and management gurus, say that organizations need to invest in surveys and the like to figure out what the customer wants and needs. And Clayton Christensen advocates spending resources to figure out what "job" the customer needs to have done. And maybe, sometimes, it does take surveys and research.

But sometimes it is obvious that the customer needs a pillow.

What is missing is not a survey, or statistical analysis.

What is missing is the incentive for someone to go get the pillow.

P.S. You may wonder, then, if it is simply a mistake for the hospital to send out the survey? I suspect that those who send out the survey are not making a mistake, but are trying to get a different job done than the one that appears to be intended. It appears that they are trying to find out what customers want and need. But maybe they already know that. Maybe they are mainly sending out the survey so that if anyone asks if they are "customer-oriented" they can whip out the survey to prove that yes-indeed, they sure are. In other words, the point of the survey is not to learn about customers; it is to cover rear-ends.

United States Cardiologists Fail to Prescribe Fish Oil, Despite Low Cost, Safety, and Evidence of Efficacy

Source of graphic: online verison of the NYT article quoted and cited below.

United States cardiologists are reluctant to prescribe fish oil, wanting more definitive data on efficacy. But a lack of definitive data on efficacy doesn’t stop them from performing costly and risky procedures such as the application of stents. Possibly relevant: installing stents is much more lucrative for cardiologists, than prescribing fish oil. Doctors are not bad people, but like most of us, they respond to financial incentives.

(p. D5) ROME — Every patient in the cardiac care unit at the San Filippo Neri Hospital who survives a heart attack goes home with a prescription for purified fish oil, or omega-3 fatty acids.

“It is clearly recommended in international guidelines,” said Dr. Massimo Santini, the hospital’s chief of cardiology, who added that it would be considered tantamount to malpractice in Italy to omit the drug.

In a large number of studies, prescription fish oil has been shown to improve survival after heart attacks and to reduce fatal heart rhythms. The American College of Cardiology recently strengthened its position on the medical benefit of fish oil, although some critics say that studies have not defined the magnitude of the effect.

But in the United States, heart attack victims are not generally given omega-3 fatty acids, even as they are routinely offered more expensive and invasive treatments, like pills to lower cholesterol or implantable defibrillators. Prescription fish oil, sold under the brand name Omacor, is not even approved by the Food and Drug Administration for use in heart patients.

“Most cardiologists here are not giving omega-3’s even though the data supports it — there’s a real disconnect,” said Dr. Terry Jacobson, a preventive cardiologist at Emory University in Atlanta. “They have been very slow to incorporate the therapy.”

For the full story, see:

ELISABETH ROSENTHAL "In Europe It’ s Fish Oil After Heart Attacks, but Not in U.S." The New York Times (Tues., October 3, 2006): D5.

Technology Liberates the Paralyzed

Paralyzed from a stabbing, Matthew Nagle can move computer cursor by means of a sensor implanted in his brain. Source of image: online version of NYT article cited below.

(p. A1) A paralyzed man with a small sensor implanted in his brain was able to control a computer, a television set and a robot using only his thoughts, scientists reported yesterday.

Those results offer hope that in the future, people with spinal cord injuries, Lou Gehrig’s disease or other conditions that impair movement may be able to communicate or better control their world.

“If your brain can do it, we can tap into it,” said John P. Donoghue, a professor of neuroscience at Brown University who has led development of the system and was the senior author of a report on it being published in today’s issue of the journal Nature.

For the full story, see:

ANDREW POLLACK. "Paralyzed Man Uses Thoughts to Move a Cursor." The New York Times (Thurs., July 13, 2006): A1 & A21.