Category: Science

Parents Set Up For-Profit Companies for Quicker Cures

(p. B1) Karen Aiach was working as a management consultant when she learned that her first daughter, Ornella, had Sanfilippo syndrome, a rare disease in which a missing enzyme causes toxic substances to build up in the body.

Ornella was 6 months old, and the prognosis was grim: She would develop mentally and physically to between ages 2 and 4, plateau and then lose whatever she had learned. She would become extremely hyperactive and develop sleeping disorders. Most likely she would not live past 15.
Within two years of the diagnosis, Ms. Aiach, who lives in a Paris suburb, had quit her consulting job to learn everything she could about the disease. She hired a neurobiologist to guide her in the world of medical research. And when she learned that few treatments were in the works, she founded a company called Lysogene to focus on genetic therapy.
Instead of raising money and awareness by setting up a nonprofit foundation, a more typical route, she opted to start a for-profit company to seek treatments, if not a cure. Far from common, what Ms. Aiach and other parents like her are trying is to leverage their wealth, contacts and the hope of sophisticated investors to jump-start research into rare diseases.
. . .
(p. B4) . . . with some rare diseases, where minimal research has been done, a little effort goes a long way.
Nicole Boice, who founded Global Genes, one of the leading rare-disease patient advocacy organizations, said even small investments can have meaningful impacts.
“You can start moving the needle with $3,500,” she said. “That leads you to the next $25,000, and then to innovation grants and funding at $100,000. That starts the interest from biotech.”
Gradually, parents like Matt Wilsey, a technology entrepreneur, have made headway. First, his family spent the better part of four years trying to figure out what afflicted his daughter, Grace, now 6. Even after her genome was sequenced, the first diagnosis turned out to be wrong. Grace, it finally was determined, was the second person in the world known to have a deficiency in the gene known as NGLY1.
“We went around the country,” Mr. Wilsey said. “We were just trying to find one doctor who had seen another patient with these symptoms.” After years of efforts, several dozen children have been found to have the same deficiency.
“Our goal is to find a cure,” said Mr. Wilsey, who lives in the San Francisco area.
“A lot of people in science dismiss that because cures are rare. But when I say cures, they’re not going to be astronauts. They’re going to be leading some sort of independent life. They’re going to be able to eat without choking. They’re going to be able to take a bath without drowning. They’re going to be able to communicate, whether with some assistive device or not.”
These parents also had a successful model to follow. In 1998, John Crowley left his job at Bristol-Myers Squibb to start a biotechnology company to search for a treatment for Pompe disease, a neuromuscular disorder that two of his children had. Within four years, the company, Novazyme Pharmaceuticals, had devised a treatment that he credits with saving their lives. His story was immortalized in the 2010 film “Extraordinary Measures,” starring Harrison Ford. And his company was bought by the pharmaceutical giant Genzyme for $137.5 million in 2001.

For the full story, see:
PAUL SULLIVAN. “Wealth Matters; Parents of Children With Rare Diseases Find Hope in For-Profit Companies.” The New York Times (Sat., DEC. 26, 2015): B1 & B4.
(Note: ellipsis added.)
(Note: the online version of the story has the date DEC. 25, 2015, and has the title “Wealth Matters; Building a Company to Treat a Rare Disease.”)

Affirmative Action Reduces Number of Black Scientists

Malcolm Gladwell, in chapter three of David and Goliath, persuasively argues that science students who would thrive at a solid public university, may be at the bottom of their class at Harvard, and in discouragement switch to an easier non-science major. Gladwell’s argument has implications for affirmative action, as noted by Gail Heriot in the passages quoted below.

(p. A13) . . . , numerous studies–as I explain in a recent report for the Heritage Foundation–show that the supposed beneficiaries of affirmative action are less likely to go on to high-prestige careers than otherwise-identical students who attend schools where their entering academic credentials put them in the middle of the class or higher. In other words, encouraging black students to attend schools where their entering credentials place them near the bottom of the class has resulted in fewer black physicians, engineers, scientists, lawyers and professors than would otherwise be the case.

But university administrators don’t want to hear that their support for affirmative action has left many intended beneficiaries worse off, and they refuse to take the evidence seriously.
The mainstream media support them on this. The Washington Post, for instance, recently featured a story lamenting that black students are less likely to major in science and engineering than their Asian or white counterparts. Left unstated was why. As my report shows, while black students tend to be a little more interested in majoring in science and engineering than whites when they first enter college, they transfer into softer majors in much larger numbers and so end up with fewer science or engineering degrees.
This is not because they don’t have the right stuff. Many do–as demonstrated by the fact that students with identical entering academic credentials attending somewhat less competitive schools persevere in their quest for a science or engineering degree and ultimately succeed. Rather, for many, it is because they took on too much, too soon given their level of academic preparation.

For the full commentary, see:
GAIL HERIOT. “Why Aren’t There More Black Scientists? The evidence suggests that one reason is the perverse impact of university racial preferences.” The Wall Street Journal (Thurs., Oct. 22, 2015): A13.
(Note: ellipsis added.)
(Note: the online version of the commentary was updated on Oct. 21, 2015.)

Heriot’s report for the Heritage Foundation, is:
Heriot, Gail. “A “Dubious Expediency”: How Race-Preferential Admissions Policies on Campus Hurt Minority Students.” Heritage Foundation Special Report #167, Aug. 31, 2015.

Gladwell’s book, mentioned above, is:
Gladwell, Malcolm. David and Goliath: Underdogs, Misfits, and the Art of Battling Giants. New York, NY: Little, Brown and Company, 2013.

Spontaneous Mummification in San Bernardo Is Unexplained

Some claim that science has gone about as far as it can go. The claim is often a step in an argument for pessimism on the future of technological progress. But that claim has been made many times in the past, and so far has always proven wrong. There’s plenty of phenomena for which we have no scientific explanation, implying that there is plenty of room for the advance of science. Mostly we ignore or forget these phenomena, because it causes cognitive dissonance for us to carry around facts that do not fit into our current theories. Add spontaneous mummification in San Bernardo to the list.

(p. A14) Locals and mummification experts agree San Bernardo is a somewhat unlikely place for what’s known as spontaneous mummification, a phenomenon that occurs naturally, without embalming fluids and other techniques. The climate here is neither excessively dry, like in Northern Africa, nor freezing, like the Alpine environment that preserved Otzi the Iceman, a prehistoric body found in 1991.
San Bernardo’s temperature hovers around 70 degrees during the day, with enough rainfall to support crops like corn, onions, and green beans.
Cemetery workers here began noticing the mummification phenomenon in the mid-1960s, after a new graveyard was built. In Colombia, due to both tradition and earth that is often too soggy for proper burial, it is typical to inter loved ones in aboveground cement vaults, called bovedas. The bodies are generally removed after about five years because of space constraints and regulations.
Bodies in such vaults usually deteriorate significantly after a year or two, but that hasn’t been the case in San Bernardo–where it is believed that most of those buried in vaults are at least partially mummified.
“Hmmm,” said Ronn Wade, a member of the World Congress on Mummy Studies, an international organization, when asked about San Bernardo’s spontaneous mummification. “It could be dietary, environmental, or even the concrete of the vaults where they are stored.”
“It would be nice to have an explanation,” added Mr. Wade, who directs the anatomical services department of the University of Maryland.
. . .
“Whatever it is, it’s very local,” said Gonzalo Correal, a professor at Bogota’s Academy of Natural Sciences, who has studied San Bernardo’s mummies.

For the full story, see:
SARA SCHAEFER MUÑOZ. “In Small Colombian Town, People Love Their Mummies; Preserved bodies attract tourists, but remain a mystery; something in the diet?” The Wall Street Journal (Thurs., Oct. 1, 2015): A1 & A14.
(Note: ellipsis added.)
(Note: the online version of the story was updated on Sept. 30, 2015, and has the title “In This Small Colombian Town, People Love Their Mummies; Preserved bodies of people born in roughly the last hundred years become tourist attraction.”)

Climate Change Likely to Be Slower and Less Harmful than Feared

(p. A11) . . . , we are often told by journalists that the science is “settled” and there is no debate. But scientists disagree: They say there is great uncertainty, and they reflected this uncertainty in their fifth and latest assessment for the United Nations Intergovernmental Panel on Climate Change (IPCC). It projects that temperatures are likely to be anything from 1.5 to 4.5 degrees Celsius (2.7 to 8.1 degrees Fahrenheit) warmer by the latter part of the century–that is, anything from mildly beneficial to significantly harmful.
As for the impact of that future warming, a new study by a leading climate economist, Richard Tol of the University of Sussex, concludes that warming may well bring gains, because carbon dioxide causes crops and wild ecosystems to grow greener and more drought-resistant. In the long run, the negatives may outweigh these benefits, says Mr. Tol, but “the impact of climate change does not significantly deviate from zero until 3.5°C warming.”
Mr. Tol’s study summarizes the effect we are to expect during this century: “The welfare change caused by climate change is equivalent to the welfare change caused by an income change of a few percent. That is, a century of climate change is about as good/bad for welfare as a year of economic growth. Statements that climate change is the biggest problem of humankind are unfounded: We can readily think of bigger problems.” No justification for prioritizing climate change over terrorism there.
. . .
To put it bluntly, climate change and its likely impact are proving slower and less harmful than we feared, while decarbonization of the economy is proving more painful and costly than we hoped. The mood in Paris will be one of furious pessimism among the well-funded NGOs that will attend the summit in large numbers: Decarbonization, on which they have set their hearts, is not happening, and they dare not mention the reassuring news from science lest it threaten their budgets.
Casting around for somebody to blame, they have fastened on foot-dragging fossil-fuel companies and those who make skeptical observations, however well-founded, about the likelihood of dangerous climate change. Scientific skeptics are now routinely censored, or threatened with prosecution. One recent survey by Rasmussen Reports shows that 27% of Democrats in the U.S. are in favor of prosecuting climate skeptics. This is the mentality of religious fanaticism, not scientific debate.

For the full commentary, see:
MATT RIDLEY And BENNY PEISER. “Your Complete Guide to the Climate Debate; At the Paris conference, expect an agreement that is sufficiently vague and noncommittal for all countries to claim victory.” The Wall Street Journal (Sat., Nov. 28, 2015): A11.
(Note: ellipses added.)
(Note: the online version of the commentary has the date Nov. 27, 2015.)

The Tol working paper mentioned above, is:
Tol, Richard S. J. “Economic Impacts of Climate Change.” University of Sussex Economics Working Paper No. 75-2015.

Scientific Insight Requires Hard Work More than Easy Epiphany

(p. A21) The myth of the finches obscures the qualities that were really responsible for Darwin’s success: the grit to formulate his theory and gather evidence for it; the creativity to seek signs of evolution in existing animals, rather than, as others did, in the fossil record; and the open-mindedness to drop his belief in creationism when the evidence against it piled up.
The mythical stories we tell about our heroes are always more romantic and often more palatable than the truth. But in science, at least, they are destructive, in that they promote false conceptions of the evolution of scientific thought.
Of the tale of Newton and the apple, the historian Richard S. Westfall wrote, “The story vulgarizes universal gravitation by treating it as a bright idea … A bright idea cannot shape a scientific tradition.” Science is just not that simple and it is not that easy.
. . .
Even if we are not scientists, every day we are challenged to make judgments and decisions about technical matters like vaccinations, financial investments, diet supplements and, of course, global warming. If our discourse on such topics is to be intelligent and productive, we need to dip below the surface and grapple with the complex underlying issues. The myths can seduce one into believing there is an easier path, one that doesn’t require such hard work.
But even beyond issues of science, there is a broader lesson to learn, . . . . We all run into difficult problems in life, and we will be happier and more successful if we appreciate that the answers often aren’t quick, or easy.

For the full commentary, see:
LEONARD MLODINOW. “It Is, in Fact, Rocket Science.” The Wall Street Journal (Sat., MAY 16, 2015): A21.
(Note: ellipsis internal to third quoted paragraph, in original; other ellipses, added.)
(Note: the online version of the commentary was updated on MAY 15, 2015.)

Mlodinow’s book, related to the commentary quoted above, is:
Mlodinow, Leonard. The Upright Thinkers: The Human Journey from Living in Trees to Understanding the Cosmos. New York: Pantheon Books, 2015.

Inflation of the Co-Authorship Bubble

CoauthorInflationGraph2015-10-30.jpg Source of graphic: online version of the WSJ article quoted and cited below.

(p. A1) . . . , there has been a notable spike since 2009 in the number of technical reports whose author (p. A10) counts exceeded 1,000 people, according to the Thomson Reuters Web of Science, which analyzed citation data. In the ever-expanding universe of credit where credit is apparently due, the practice has become so widespread that some scientists now joke that they measure their collaborators in bulk–by the “kilo-author.”

Earlier this year, a paper on rare particle decay published in Nature listed so many co-authors–about 2,700–that the journal announced it wouldn’t have room for them all in its print editions. And it isn’t just physics. In 2003, it took 272 scientists to write up the findings of the first complete human genome–a milestone in biology–but this past June, it took 1,014 co-authors to document a minor gene sequence called the Muller F element in the fruit fly.
. . .
More than vanity is at stake. Credit on a peer-reviewed research article weighs heavily in hiring, promotion and tenure decisions. “Authorship has become such a big issue because evaluations are performed based on the number of papers people have authored,” said Dr. Larivière.
. . .
Michigan State University mathematician Jack Hetherington published a paper in 1975 on low temperature physics in Physical Review Letters with F.D.C. Willard. His colleagues only discovered that his co-author was a siamese cat several years later when Dr. Hetherington started handing out copies of the paper signed with a paw print.
In the same spirit, Shalosh B. Ekhad at Rutgers University so far has published 32 peer-reviewed papers in scientific journals with his co-author Doron Zeilberger. It turns out that Shalosh B. Ekhad is Hebrew for the model number of a personal computer used by Dr. Zeilberger. “The computer helps so much and so often,” Dr. Zeilberger said.
Not everyone takes such pranks lightly.
Immunologist Polly Matzinger at the National Institute of Allergy and Infectious Diseases named her dog, Galadriel Mirkwood, as a co-author on a paper she submitted to the Journal of Experimental Medicine. “What amazed me was that the paper went through the entire editorial process and nobody noticed,” Dr. Matzinger said. When the journal editor realized he had published work crediting an Afghan hound, he was furious, she recalled.
Physicists may be more open-minded. Sir Andre Geim, winner of the 2010 Nobel Prize in Physics, credited H.A.M.S. ter Tisha as his co-author of a 2001 paper published in the journal Physica B. Those journal editors didn’t bat an eye when his co-author was unmasked as a pet hamster. “Not a harmful joke,” said Physica editor Reyer Jochemsen at the Leiden University in the Netherlands.
“Physicists apparently, even journal editors, have a better sense of humor than the life sciences,” said Dr. Geim at the U.K.’s University of Manchester.

For the full story, see:
ROBERT LEE HOTZ. “Scientists Observe Odd Phenomenon of Multiplying Co-Authors.”The Wall Street Journal (Mon., Aug. 10, 2015): A1 & A10.
(Note: ellipses added.)
(Note: the online version of the story has the title “How Many Scientists Does It Take to Write a Paper? Apparently, Thousands.”)

Lives Lost Due to Peer Review Delays

(p. A25) In this age of instant information, medicine remains anchored in the practice of releasing new knowledge at a deliberate pace. It’s time for medical scientists to think differently about how quickly they alert the public to breakthrough findings.
Last week the National Institutes of Health announced that it had prematurely ended a large national study of how best to treat people with high blood pressure because of its exceptional results.
In this trial of more than 9,000 people age 50 and older with high blood pressure, an aggressive treatment strategy to keep systolic blood pressure below 120 was compared with a conventional one aimed at keeping it below 140. The subjects all had a high risk of heart attacks, stroke and heart failure. The N.I.H. concluded, six years into a planned eight-year study, that for these patients, pushing blood pressure down far below currently recommended levels was very beneficial.
. . .

The new information may justify a more vigorous strategy for treating blood pressure, but for now doctors and patients have been left with incomplete results, some headlines and considerable uncertainty about whether to modify current treatments.
Medicine needs to change its approach to releasing new, important information. Throughout science we are seeing more rapid modes of communication. The traditional approach was not to publish until everything was finalized and ready to be chiseled in stone. But these sorts of delays are unnecessary with the Internet. Moreover, although all the trial data has yet to be tabulated, an analysis was considered sufficiently definitive to lead independent experts to stop the multimillion-dollar study.
We believe that when there is such strong evidence for a major public health condition, there should be rapid release of the information that led to the decision to stop the trial. This approach could easily be accomplished by placing the data on the N.I.H. website or publishing the data on such platforms as bioRxiv.org, which enables fast, open review by the medical community.
. . .
Kudos to the scientists who conducted such a large, complex and important study with what will be likely to have lifesaving consequences for a condition that can be treated easily in most patients. Now the medical community needs to adopt a new approach in situations like this one to disseminate lifesaving results in a timely, comprehensive and transparent way. Lives depend on it.

For the full commentary, see:
ERIC J. TOPOL and HARLAN M. KRUMHOLZ. “Don’t Sit on Medical Breakthroughs.” The New York Times (Fri., SEPT. 17, 2015): A25.
(Note: ellipses added.)
(Note: the online version of the commentary has the date SEPT. 17, 2015, and the title “Don’t Delay News of Medical Breakthroughs.”)

Newly Found, Early Human Species, Respected Their Dead

(p. A1) [A] . . . new hominin species was announced on Thursday, [September 10, 2015] by an international team of more than 60 scientists led by Lee R. Berger, an American paleoanthropologist who is a professor of human evolution studies at the University of the Witwatersrand in Johannesburg. The species name, H. naledi, refers to the cave where the bones lay undisturbed for so long; “naledi” means “star” in the local Sesotho language.
In two papers published this week in the open-access journal eLife, the researchers said that the more than 1,550 fossil elements documenting the discovery constituted the largest sample for any hominin species in a single African site, and one of the largest anywhere in the world.
. . .
The finding, like so many others in science, was the result of pure luck followed by considerable effort.
Two local cavers, Rick Hunter and Steven Tucker, found the narrow entrance to the chamber, measuring no more than seven and a half inches wide. They were skinny enough to squeeze through, and in the light of their headlamps they saw the bones all around them. When they showed the fossil pictures to Pedro Boshoff, a caver who is also a geologist, he alerted Dr. Berger, who organized an investigation.
. . .
(p. A3) Besides introducing a new member of the prehuman family, the discovery suggests that some early hominins intentionally deposited bodies of their dead in a remote and largely inaccessible cave chamber, a behavior previously considered limited to modern humans. Some of the scientists referred to the practice as a ritualized treatment of their dead, but by “ritual” they said they meant a deliberate and repeated practice, not necessarily a kind of religious rite.
. . .
At the news conference in South Africa on Thursday, [September 10, 2015] announcing the findings, Dr. Berger said: “I do believe that the field of paleoanthropology had convinced itself, as much as 15 years ago, that we had found everything, that we were not going to make major discoveries and had this story of our origins figured out. I think many people quit exploring, thought it was safer to conduct science inside a lab or behind a computer.” What the new species Naledi says, Dr. Berger concluded, “is that there is no substitute for exploration.”

For the full story, see:
JOHN NOBLE WILFORD. “Cave Yields Addition to Human Family Tree.”The New York Times (Fri., SEPT. 11, 2015): A1 & A3.
(Note: ellipses, and bracketed word and date, added.)
(Note: the online version of the story has the date SEPT. 10, 2015, and has the title “Homo Naledi, New Species in Human Lineage, Is Found in South African Cave,”)

Those Who Use “Consensus” Argument on Global Warming, Should Endorse Genetically Modified Food

(p. B3) NAIROBI, Kenya — Mohammed Rahman doesn’t know it yet, but his small farm in central Bangladesh is globally significant. Mr. Rahman, a smallholder farmer in Krishnapur, about 60 miles northwest of the capital, Dhaka, grows eggplant on his meager acre of waterlogged land.
As we squatted in the muddy field, examining the lush green foliage and shiny purple fruits, he explained how, for the first time this season, he had been able to stop using pesticides. This was thanks to a new pest-resistant variety of eggplant supplied by the government-run Bangladesh Agricultural Research Institute.
Despite a recent hailstorm, the weather had been kind, and the new crop flourished. Productivity nearly doubled. Mr. Rahman had already harvested the small plot 10 times, he said, and sold the brinjal (eggplant’s name in the region) labeled “insecticide free” at a small premium in the local market. Now, with increased profits, he looked forward to being able to lift his family further out of poverty. I could see why this was so urgent: Half a dozen shirtless kids gathered around, clamoring for attention. They all looked stunted by malnutrition.
. . .
I, . . . , was once in [the] . . . activist camp. A lifelong environmentalist, I opposed genetically modified foods in the past. Fifteen years ago, I even participated in vandalizing field trials in Britain. Then I changed my mind.
After writing two books on the science of climate change, I decided I could no longer continue taking a pro-science position on global warming and an anti-science position on G.M.O.s.
There is an equivalent level of scientific consensus on both issues, I realized, that climate change is real and genetically modified foods are safe. I could not defend the expert consensus on one issue while opposing it on the other.

For the full commentary, see:
MARK LYNAS. “How I Got Converted to G.M.O. Food.” The New York Times, SundayReview Section (Sun., APRIL 26, 2015): 5.
(Note: ellipses, and bracketed word, added.)
(Note: the online version of the commentary has the date APRIL 24, 2015.)

“Strong-Willed Scientists Overstated the Significance of Their Studies”

The New York Times seems open to the idea that strong-willed scientists might overstate their results in science food studies. I wonder if The New York Times would be open to the same possibility in science climate studies?

(p. A19) For two generations, Americans ate fewer eggs and other animal products because policy makers told them that fat and cholesterol were bad for their health. Now both dogmas have been debunked in quick succession.
. . .
Epidemiological data can be used to suggest hypotheses but not to prove them.
Instead of accepting that this evidence was inadequate to give sound advice, strong-willed scientists overstated the significance of their studies.
Much of the epidemiological data underpinning the government’s dietary advice comes from studies run by Harvard’s school of public health. In 2011, directors of the National Institute of Statistical Sciences analyzed many of Harvard’s most important findings and found that they could not be reproduced in clinical trials.
It’s no surprise that longstanding nutritional guidelines are now being challenged.
In 2013, government advice to reduce salt intake (which remains in the current report) was contradicted by an authoritative Institute of Medicine study. And several recent meta-analyses have cast serious doubt on whether saturated fats are linked to heart disease, as the dietary guidelines continue to assert.
Uncertain science should no longer guide our nutrition policy. Indeed, cutting fat and cholesterol, as Americans have conscientiously done, may have even worsened our health.

For the full commentary, see:
NINA TEICHOLZ. “The Government’s Bad Diet Advice.” The New York Times (Sat., FEB. 21, 2015): A19.
(Note: ellipsis added.)
(Note: the online version of the commentary has the date FEB. 20, 2015.)

Entrepreneurs Creating Healthy, Tasty Meat, Without Killing Animals

(p. B2) “The next couple of years will be exciting ones,” says Joseph D. Puglisi, a Stanford University professor of structural biology who is working on meat alternatives. “We can use a broad range of plant protein sources and create a palette of textures and tastes — for example, jerky, cured meats, sausage, pork.”
“The true challenge will be to recreate more complex pieces of meat that are the pinnacle of the meat industry,” he added. “I believe that plausible, good-tasting steaks and pork loins are only a matter of time.”
Puglisi is advising Beyond Meat, a start-up that is a leader in the field, with investments from Bill Gates and both Biz Stone and Ev Williams of Twitter fame, not to mention Kleiner Perkins Caufield & Byers, the venture capital firm that backed Google and Amazon. Beyond Meat says its sales are doubling each year.
“We’re really focused on the mainstream,” said Ethan Brown, the founder of Beyond Meat, over a lunch of fake chili, meatballs and hamburgers.
. . .
“We want to create the next great American meat company,” Brown says. “That’s the dream.”
. . .
The mainstream food industry isn’t saying much publicly. But recently released documents from the American Egg Board, a quasi-governmental body, show it regarded Hampton Creek’s egg-free “Just Mayo” spread as a “major threat.” In one internal email, an Egg Board executive jokingly suggests hiring a hit man to deal with Hampton Creek.
. . .
. . . if I can still enjoy a juicy burger now and then, while boosting my health, helping the environment and avoiding the brutalizing of farm animals, hey, I’m in!

For the full commentary, see:
Nicholas Kristof. “The (Fake) Meat Revolution.” The New York Times, SundayReview Section (Sun., SEPT. 20, 2015): 11.
(Note: the online version of the commentary has the date SEPT. 19, 2015.)