Category: Health

Regulations Reduce Biotech Innovation

(p. A15) Modern genetic engineering, also called genetic modification or GM, has been around since the 1970s. Yet with the notable exception of biopharmaceuticals–beginning with the marketing of human insulin in 1982 and now accounting for more than 20% of U.S. drug expenditures–genetic engineering has failed to realize anything approaching its potential for vertical progress.
The reason is plain: In the non-pharmaceutical sectors, federal regulators for years seemingly have done everything they can to prevent U.S. researchers and companies from employing genetic engineering to create the “next big thing.”
. . .
Regulatory disincentives are potent. It costs about $136 million to bring a genetically engineered crop plant to market. This is the primary reason more than 99% of such crop plants are those that are grown at huge scale: . . .
. . .
“Biopharming”–the once-promising biotechnology area that uses genetic engineering techniques to induce crops such as corn, tomatoes and tobacco to produce high concentrations of high-value pharmaceuticals (one of which is the Ebola drug, ZMapp)–is moribund because of the Agriculture Department’s extraordinary regulatory burdens. Thanks to EPA’s policies, which discriminate against organisms modified with the most precise and predictable techniques, the high hopes for genetically engineered “biorational” microbial pesticides and microorganisms to clean up toxic wastes have evaporated.

For the full commentary, see:
HENRY I. MILLER. “Regulators Put the Brakes On Biotech; Thanks to EPA, hopes have evaporated for genetically engineered microorganisms to clean up toxic wastes.” The Wall Street Journal (Weds., Jan. 14, 2015): A15.
(Note: ellipses added.)
(Note: the online version of the commentary has the date Jan. 13, 2015.)

Serendipitous Discovery that Titanium Fuses with Bone, Leads to Implants

(p. 24) Implants have been a major advance in dentistry, liberating millions of elderly people from painful, ill-fitting dentures, a diet of soft foods and the ignominy of a sneeze that sends false teeth flying out of the mouth. But addressing those problems was not Dr. Branemark’s initial intent.
At the start of his career, he was studying how blood flow affects bone healing.
In 1952, he and his team put optical devices encased in titanium into the lower legs of rabbits in order to study the healing process. When the research period ended and they went to remove the devices, they discovered to their surprise that the titanium had fused into the bone and could not be removed.
Dr. Branemark called the process “osseointegration,” and his research took a whole new direction as he realized that if the body could tolerate the long-term presence of titanium, the metal could be used to create an anchor for artificial teeth.
. . .
. . . , Dr. Branemark’s innovation was poorly received. After Dr. Branemark gave a lecture on his work in 1969, Dr. Albrektsson recalled, one of the senior academics of Swedish dentistry rose and referred to an article in Reader’s Digest describing Dr. Branemark’s research, adding, “This may prove to be a popular article, but I simply do not trust people who publish themselves in Reader’s Digest.”
As it happened, that senior academic was well known to the Swedish public for recommending a particular brand of toothpick. So Dr. Branemark immediately rose and struck back, saying, “And I don’t trust people who advertise themselves on the back of boxes of toothpicks.”

For the full story, see:
TAMAR LEWIN. “Per-Ingvar Branemark, Dental Innovator, Dies at 85.” The New York Times, First Section (Sun., DEC. 28, 2014): 24.
(Note: ellipses are added.)
(Note: the online version of the story has the date JAN. 27, 2015.)

Innovation and Jobs Destroyed by Tax

(p. 7A) I was humbled to receive in November the National Medal of Technology and Innovation at the White House for the development of life-changing medical devices. Traveling to our nation’s capital, I couldn’t help but think: There is no way I could have had the same impact if the tax on medical devices was in place when I got started over 50 years ago.
Simply put, the medical device tax is destroying job creation and innovation, and as a result, patient care is suffering.
. . .
Every day, I see firsthand the difficult choices innovators must make as a result of this ill-conceived tax. Perhaps worst of all, the medical device tax is helping cause a steep drop of investments in promising therapies.
. . .
It’s time to put an end to this disastrous policy so that medical device entrepreneurs can do what America does best — innovate.

For the full commentary, see:
Tom Fogarty. “Opposing View: Tax Destroys Jobs and Innovation.” USA Today (Mon., January 5, 2015): 7A.
(Note: ellipses added.)
(Note: the online version of the commentary has the date January 4, 2015, and has the title “Tax Destroys Jobs and Innovation: Opposing View.”)

“It’s My Life, and I Want the Chance to Save It”

(p. 18) LYONS, Colo. — Since May [2014], a string of states have passed laws that give critically ill patients the right to try medications that have not been approved by the Food and Drug Administration.
Deemed “Right to Try” laws, they have passed quickly and often unanimously in Colorado, Michigan, Missouri, Louisiana and Arizona, bringing hope to patients like Larry Kutt, who lives in this small town at the edge of the Rocky Mountains. Mr. Kutt, 65, has an advanced blood cancer and says his state’s law could help him gain access to a therapy that several pharmaceutical companies are testing. “It’s my life,” he said, “and I want the chance to save it.”
The laws do not seem to have helped anyone obtain experimental medicine, as the drug companies are not interested in supplying unapproved medications outside the supervision of the F.D.A. But that seems almost beside the point to the Goldwater Institute, the libertarian group behind legislative efforts to pass Right to Try laws. “The goal is for terminally ill patients to have choice when it comes to end-stage disease,” said Craig Handzlik, state policy coordinator for the Goldwater Institute, based in Arizona. “Right to Try is something that will help terminally ill people all over the country.”

For the full story, see:
JULIE TURKEWITZ. “Patients Seek ‘Right to Try’ New Drugs.” The New York Times, First Section (Sun., JAN. 11, 2015): 18.
(Note: the bracketed year is added.)
(Note: the online version of the story has the date JAN. 10, 2015.)

Mandated Health Treatment Regulations Are Often Reversed

(p. A25) After spending nearly two decades in medicine, I am still amazed by how spare the evidence is on which we doctors base our medical decisions. Treatment guidelines, often accompanied by a de facto mandate, are frequently reversed.
Only a few years ago, for example, beta-blocker drugs were routinely recommended for almost all patients undergoing noncardiac surgery. Since then, research has shown that these drugs may significantly increase the risk of stroke at the time of surgery. I remember colleagues questioning the beta-blocker recommendation for certain patients and being admonished for not being “evidence-based.” I shudder to think how many patients were left disabled by strokes because of the blanket adoption of this standard.
What is in vogue today is often discarded tomorrow. Hormone replacement therapy for women after menopause is an example of a once widely implemented treatment that we have now largely abandoned. In September, in response to new research, the American College of Cardiology revoked a major recommendation on heart-attack treatment. “Science is not static but rather constantly evolving,” said its president, Patrick T. O’Gara, in explaining the decision.
. . .
Instead of being allowed to deliver “patient-centered” care, many physicians feel they are being co-opted by regulations. Some feel pressured to prescribe “mandated” treatment, even to frail older adults who may not benefit. Guidelines are supposed to assist and advise. But all too often, recommended care in certain situations becomes mandated care in all situations.

For the full commentary, see:
SANDEEP JAUHAR. “Don’t Homogenize Health Care.” The New York Times (Thurs., DEC. 11, 2014): A25.
(Note: the online version of the commentary has the date DEC. 10, 2014.)

Smart Phones Bring Power to the Patient

(p. A11) We instinctively reach for our smartphones when we need to take pictures, get directions, deposit checks or reserve a table. Eric Topol, a cardiologist and digital pioneer, thinks that they are ready to perform at least one more task: revolutionize health care. In “The Patient Will See You Now,” he argues that smartphones will democratize medicine by bringing data and control directly to the people.
The power of doctors, says Dr. Topol, “can be likened to that of religious leaders and nobility” in centuries past, when knowledge and authority belonged to a small elite. He notes that we’ve never seen “a discrete challenge to the medical profession” akin to Luther ‘s challenge to the Roman Catholic Church or democracy’s challenge to monarchy and despotism. “But we’ve not had the platform or landscape for that to be accomplished. Until now.” Smartphones, he says, enable a range of medical applications to move from the hospital to the home, and they shift medicine’s locus of control from doctor to patient.

For the full review, see:
DAVID A. SHAYWITZ. “BOOKSHELF; Doctor Android; In the same way that Luther challenged the Catholic Church, smartphones are poised to upend the medical profession.” The Wall Street Journal (Tues., Jan. 13, 2015): A11.
(Note: the online version of the review has the date Jan. 12, 2015.)

The book under review is:
Topol, Eric. The Patient Will See You Now: The Future of Medicine Is in Your Hands. New York: Basic Books, 2015.

Double-Blind Clinical Trials Are NOT the Only Source of Good Evidence

(p. 16) Back in her office, . . . [rheumatologist Jennifer Frankovich] found that the scientific literature had no studies on patients like this to guide her. So she did something unusual: She searched a database of all the lupus patients the hospital had seen over the previous five years, singling out those whose symptoms matched her patient’s, and ran an analysis to see whether they had developed blood clots. “I did some very simple statistics and brought the data to everybody that I had met with that morning,” she says. The change in attitude was striking. “It was very clear, based on the database, that she could be at an increased risk for a clot.”
The girl was given the drug, and she did not develop a clot. “At the end of the day, we don’t know whether it was the right decision,” says Chris Longhurst, a pediatrician and the chief medical information officer at Stanford Children’s Health, who is a colleague of Frankovich’s. But they felt that it was the best they could do with the limited information they had.
A large, costly and time-consuming clinical trial with proper controls might someday prove Frankovich’s hypothesis correct. But large, costly and time-consuming clinical trials are rarely carried out for uncommon complications of this sort. In the absence of such focused research, doctors and scientists are increasingly dipping into enormous troves of data that already exist — namely the aggregated medical records of thousands or even millions of patients to uncover patterns that might help steer care.
. . .
(p. 17) . . . , developing a “learning health system” — one that can incorporate lessons from its own activities in real time — remains tantalizing to researchers. Stefan Thurner, a professor of complexity studies at the Medical University of Vienna, and his researcher, Peter Klimek, are working with a database of millions of people’s health-insurance claims, building networks of relationships among diseases. As they fill in the network with known connections and new ones mined from the data, Thurner and Klimek hope to be able to predict the health of individuals or of a population over time. On the clinical side, Longhurst has been advocating for a button in electronic medical-record software that would allow doctors to run automated searches for patients like theirs when no other sources of information are available.
With time, and with some crucial refinements, this kind of medicine may eventually become mainstream. Frankovich recalls a conversation with an older colleague. “She told me, ‘Research this decade benefits the next decade,’ ” Frankovich says. “That was how it was. But I feel like it doesn’t have to be that way anymore.”

For the full story, see:
VERONIQUE GREENWOOD. “Eureka; Dr. DATA; Can Statistical Analysis Tell Us What Clinical Trials Cannot?” The New York Times Magazine (Sun., OCT. 5, 2014): 16-17.
(Note: ellipses, and bracketed name, added.)
(Note: the online version of the story has the date OCT. 3, 2014, and has the title “Eureka; Can Big Data Tell Us What Clinical Trials Don’t?”)

26 Different Drugs Lengthen Healthy Life Span in Mice

(p. F5) For thousands of years, people have sought to escape or outrun their mortality with potions, pills and elixirs, often blended with heavy doses of hope and will.
In the “Epic of Gilgamesh,” a Mesopotamian king searched for the secret of immortality after the death of his best friend. At least three Chinese emperors in the Tang dynasty died after consuming treatments containing lead and mercury that they hoped would make them immortal. In the late 19th century, a French-American physiologist seemed to have found the elixir of life by injecting the elderly and himself with extracts from animal testicles.
. . .
“By targeting fundamental aging processes, we might be able to delay the major age-related chronic diseases instead of picking them off one at time,” said Dr. James Kirkland, a professor of aging research and head of the Robert and Arlene Kogod Center on Aging at the Mayo Clinic. “For example, we don’t want to have situation where we, say, cure cancer and then people die six months later of Alzheimer’s disease or a stroke. It would be better to delay all of these things together.”
This is where the field known as the biology of aging is moving — to develop drugs that will increase life span and what researchers refer to as health span, the period of life when people are able to live independently and free from disease.
Dr. Kirkland said that at least six drugs had been written up in peer-reviewed journals and that he knew of about 20 others that appear to affect life span or health span in mice. The goal is to see if those benefits can be translated into humans to increase their longevity, “to find interventions that we can use in people that might, say, make a person who’s 90 feel like they’re 60 or a person who’s 70 feel like they’re 40 or 50.”
Other researchers are studying centenarians, seeking to understand whether certain genes have carried them past 100 years old and kept them in good health.

For the full story, see:
TRACEY SAMUELSON. “Science (and Quacks) vs. the Aging Process.” The New York Times (Weds., Nov. 19, 2014): F5.
(Note: ellipsis added.)
(Note: the online version of the story has the date NOV. 18, 2014.)

Obamacare Advisor Says Obscure Law Passed Due to “Stupidity of the American Voter”

(p. A4) Jonathan Gruber, the economist at the heart of a fresh debate about the Affordable Care Act, has had more than a dozen appointments to visit the White House since Democrats began drafting the health law in 2009, records show.
The visits included at least one group meeting with President Barack Obama , as well as appointments with senior administration officials who helped shape the 2010 law that expanded health insurance to millions of Americans.
The White House in recent days has tried to distance itself from Mr. Gruber, a 49-year-old Massachusetts Institute of Technology economist, since a 2013 video surfaced last week in which he said the law passed because of the “huge political advantage” of the legislation’s lacking transparency. He also referred to the “stupidity of the American voter.”
Republicans have seized on the comments as evidence that supporters of the law purposely misled the public about its costs.
“It is amusing to watch Washington liberals discount Mr. Gruber’s truth-telling as a gaffe and disown” his involvement in the law, said Sen. Orrin Hatch (R., Utah).

For the full story, see:
STEPHANIE ARMOUR and COLLEEN MCCAIN NELSON. “Health Adviser Gruber Logged Regular White House Visits.” The Wall Street Journal (Tues., Nov. 18, 2014): A4.
(Note: the online version of the story has the date Nov. 17, 2014.)

Outsiders Persevere to Pursue Breakthroughs

(p. 315) Despite all the examples given, mainstream medical research stubbornly continues to assume that new drugs and other advances will follow exclusively from a predetermined research path. Many, in fact, will. Others, if history is any indication, will not. They will come not from a committee or a research team but from an individual, a maverick who views a problem with fresh eyes. Serendipity will strike and be seized upon by a well-trained scientist or clinician who also dares to rely upon intuition, imagination, and creativity. Unbound by traditional theory, willing to suspend the usual set of beliefs, unconstrained by the requirement to obtain approval or funding for his or her pursuits, this outsider will persevere and lead the way to a dazzling breakthrough. Eventually, once the breakthrough becomes part of accepted medical wisdom, the insiders will pretend that the outsider was one of them all along.

Source:
Meyers, Morton A. Happy Accidents: Serendipity in Modern Medical Breakthroughs. New York: Arcade Publishing, 2007.