Wednesday, August 30, 2017

Pioneering cancer drug will cost $475,000. Analysts call it a bargain

The Food and Drug Administration on Wednesday approved a futuristic new approach to treating cancer, clearing a Novartis therapy that has produced unprecedented results in patients with a rare and deadly cancer. The price tag: $475,000 for a course of treatment.

That sounds staggering to many patients — but it's far less than analysts expected.

The therapy, called a CAR-T, is made by harvesting patients' white blood cells and rewiring them to home in on tumors. Novartis's product is the first CAR-T therapy to come before the FDA, leading a pack of novel treatments that promise to change the standard of care for certain aggressive blood cancers.

Novartis's therapy is approved to treat children and young adults with relapsed acute lymphoblastic leukemia. It will be marketed as Kymriah.

The treatment's approval has looked a foregone conclusion for months, but its potential price has been the subject of speculation and debate. Novartis picked the $475,000 price tag in an effort to balance patient access to Kymriah while giving the company a return on its investment, said Bruno Strigini, Novartis's head of oncology, in a conference call Wednesday. The cost is below Wall Street analyst expectations, which reached as high as $750,000 for a dose. And it's considerably cheaper than the roughly $700,000 price tag that U.K. regulators said would be fair considering Kymriah's potential benefits.

Novartis also said it is working with Medicare on a system in which the government would only pay for CAR-T treatment if patients respond within a month.

In a clinical trial, a single dose of Kymriah left 83 percent of participants cancer-free after three months, results oncologists have hailed as a major advance for patients with few other options. The most frequent side effect was an inflammatory storm called cytokine release syndrome, a reaction to CAR-T that can prove fatal in some patients but is commonly controlled with immunosuppressant drugs.

"I think this is most exciting thing I've seen in my lifetime," said Dr. Tim Cripe, an oncologist with Nationwide Children's Hospital, at an FDA meeting on Kymriah in July.

More ...

https://www.statnews.com/2017/08/30/novartis-car-t-cancer-approved/?

Could Your Next Doctor Be Your Dentist? - Slate

Tooth enamel is the strongest substance in the human body. It's harder than steel. Which helps explain why the three words "root canal treatment" often strike such terror into patients sitting in the dental chair. It starts by boring a hole through enamel as effortlessly as if it were rice paper. Ninety-nine percent of the time, that gaping hole is filled and sealed immediately after treatment. A few months ago, I met a patient I'll call Janet, to protect her privacy. She was one of the 1 percent.

I carefully peered my head into her mouth. The remaining pulp, or core, of the treated tooth—an amalgam of nerves, blood vessels, and immune cells—had blossomed out of the fractured crown into a twisting, intersecting polyp with the color and consistency of bubble gum. She had chronic hyperplastic pulpitis, a rare inflammatory condition that triggers pulp tissue to irreversibly expand above the walls of the enamel shell. I pulled my stethoscope off the shelf and checked what I had been trained to with every new patient: her blood pressure. 174 over 104, I whispered to myself, having expected only a slight deviation from the normal 120 over 80. This seemed impossible. I checked the other arm; 172/104. I waited 15 minutes and checked again. 164/100.

Hypertensive emergency, which can cause patients to spontaneously suffer a severe stroke, heart attack, or kidney damage, occurs when blood pressure reaches 180/110 or higher. While her blood pressure was trending downward, Janet was dangerously close to that threshold. I asked her if any physician in the past had ever told her that she had high blood pressure. A recent Dominican immigrant unsure of her past medical history, she told me she couldn't remember. Her expanding pulp, a rarity for me, was only a distraction from a bigger concern—her heart. I immediately called her primary care physician, discussed the situation, and told her to go see her doctor immediately. Janet, who had showed up for some basic dental work, had been inches away from a medical emergency.

A 2016 Association of American Medical Colleges report projects that over the next 10 years, the U.S. will face a serious physician shortage, especially among primary care physicians in rural geographic areas. Despite increased health insurance coverage for millions of Americans over the last few years, affordable health care is still difficult to access in rural areas. Certain states, such as Tennessee, Iowa, and my home state of Arizona, are seeing insurance companies drop out of individual markets due to political uncertainty, making access to affordable care harder for a significant fraction of the U.S. population, including many of those I grew up with.

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http://www.slate.com/articles/health_and_science/medical_examiner/2017/08/why_your_next_doctor_could_be_your_dentist.html

Tuesday, August 29, 2017

‘Dying: A Memoir’ Is a Bracing Illumination of Terminal Illness - The New York Times

DYING
A Memoir
By Cory Taylor
141 pages. Tin House Books. $18.95.


Years ago, a palliative care doctor told me that what he knew of a patient's personality often had little to do with how he or she coped with dying. Generous people could become ungenerous, and brave people could become frightened. Angry people could become gentle, and controlling people could become Zen. Dying, in other words — like combat, like becoming a parent, like any transformative life event — doesn't always reveal or intensify aspects of our character. It sometimes coaxes out new ones.

For a long time, the writer Cory Taylor took, by her own admission, "a fairly leisurely approach to life." That changed in 2005, just before her 50th birthday, when doctors removed a mole on the back of her leg. Melanoma, Stage 4. She wrote the novel she'd always meant to write, then another. Then she wrote "Dying: A Memoir."

The book rings louder in my imagination the more time I spend apart from it, a kind of reverse Doppler effect. "Dying" is bracing and beautiful, possessed of an extraordinary intellectual and moral rigor. Every medical student should read it. Every human should read it. My own copy is so aggressively underlined it looks like a composition notebook.

"Dying" is short, but as dense as dark matter. There is an electrifying matter-of-factness to it, one that normalizes death, which is part of Taylor's goal. She deplores the "monstrous silence" surrounding the subject of mortality. "If cancer teaches you one thing," she writes, "it is that we are dying in our droves, all the time. Just go into the oncology department of any major hospital and sit in the packed waiting room."

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F.D.A. Panel Recommends Approval for Gene-Altering Leukemia Treatment - The New York Times

A Food and Drug Administration panel opened a new era in medicine on Wednesday, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient's own cells to fight cancer, transforming them into what scientists call "a living drug" that powerfully bolsters the immune system to shut down the disease.

If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market in the United States. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.

To use the technique, a separate treatment must be created for each patient — their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

One of those patients, Emily Whitehead, now 12 and the first child ever given the altered cells, was at the meeting of the panel with her parents to advocate for approval of the drug that saved her life. In 2012, as a 6-year-old, she was treated in a study at the Children's Hospital of Philadelphia. Severe side effects — raging fever, crashing blood pressure, lung congestion — nearly killed her. But she emerged cancer free, and has remained so.

"We believe that when this treatment is approved it will save thousands of children's lives around the world," Emily's father, Tom Whitehead, told the panel. "I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive."

The main evidence that Novartis presented to the F.D.A. came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission — a high rate for such a severe disease. Eleven others died.

"It's a new world, an exciting therapy," said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment.

The next step, she said, will be to determine "what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight." She added, "This is the beginning of something big."

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https://www.nytimes.com/2017/07/12/health/fda-novartis-leukemia-gene-medicine.html?

When Your Doctor Is Fitter Than You Are - The New York Times

"I enjoy working out at the gym," declares one profile. "To keep myself fit, I like to hike, bike and exercise," says another.

These comments aren't part of a dating site. Rather, they come from physicians' online profiles that prospective patients view when they are looking for a new doctor.

There are good reasons doctors might strive to lead by example. "I practice what I preach by living healthy every day," declares one physician on Kaiser Permanente's online doctor search portal. Patients may trust or be inspired by such a doctor, the thinking goes. And if health care professionals fail to follow their own advice, they may be accused of hypocrisy.

But for some patients, particularly those battling weight issues, a doctor's declarations of personal fitness may not have the intended effect of attracting new patients. Instead, rather than inspiring them, it can drive them away.

Recently, my colleague Benoît Monin and I studied doctors who advertise their fitness online. Past research has shown that people worry that those who claim the moral high ground will look down on others whose behavior seems unfavorable by comparison. For example, meat-eaters worry that vegetarians will judge them because of their diet. We wondered: Could emphasizing fitness make doctors seem "healthier than thou" and turn off patients?

We thought that people who are overweight and obese might be particularly sensitive to judgment from doctors. Unlike unhealthy habits such as smoking, weight can't be hidden. Research shows that negative attitudes toward people who are overweight are surprisingly prevalent among health professionals. So potential patients who are overweight might be especially turned off by doctors who show off healthy habits.

To test this idea, in research recently published in the Journal of Personality and Social Psychology, we turned to the real-world examples of physicians practicing what they preach on the website for Kaiser Permanente, the largest managed care organization in the United States. Here, patients choose among dozens of doctors from self-descriptions only a few sentences long, making any information provided consequential. We asked adults who were overweight or obese to rate a sample of these profiles. Some physicians emphasized their fitness in these profiles, while others did not.

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https://www.nytimes.com/2017/07/13/well/family/when-your-doctor-is-fitter-than-you-are.html

The Conversation Placebo - The New York Times

In my daily work as a primary care internist, I see no letup from pain. Every single patient, it seems, has an aching shoulder or a bum knee or a painful back. "Our bodies evolved to live about 40 years," I always explain, "and then be finished off by a mammoth or a microbe." Thanks to a century of staggering medical progress, we now live past 80, but evolution hasn't caught up; the cartilage in our joints still wears down in our 40s, and we are more obese and more sedentary than we used to be, which doesn't help.

So it's no surprise that chronic arthritis and back pain are the second and third most common non-acute reasons that people go to the doctor and that pain costs America up to $635 billion annually. The pain remedies developed by the pharmaceutical industry are only modestly effective, and they have side effects that range from nausea and constipation to addiction and death.

What's often overlooked is that the simple conversation between doctor and patient can be as potent an analgesic as many treatments we prescribe.

In 2014, researchers in Canada did an interesting study about the role of communication in the treatment of chronic back pain. Half the patients in the study received mild electrical stimulation from physical therapists, and half received sham stimulation (all the equipment is set up, but the electrical current is never activated). Sham treatment — placebo — worked reasonably well: These patients experienced a 25 percent reduction in their levels of pain. The patients who got the real stimulation did even better, though; their pain levels decreased by 46 percent. So the treatment itself does work.

Each of these groups was further divided in half. One half experienced only limited conversation from the physical therapist. With the other half, the therapists asked open-ended questions and listened attentively to the answers. They expressed empathy about the patients' situation and offered words of encouragement about getting better.

Patients who underwent sham treatment but had therapists who actively communicated reported a 55 percent decrease in their pain. This is a finding that should give all medical professionals pause: Communication alone was more effective than treatment alone. The patients who got electrical stimulation from engaged physical therapists were the clear winners, with a 77 percent reduction in pain.

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https://www.nytimes.com/2017/01/19/opinion/sunday/the-conversation-placebo.html

‘No Apparent Distress’ Tackles the Distress of the Sick, Poor and Uninsured - Book review - The New York Times

NO APPARENT DISTRESS 
A Doctor's Coming-of-Age on the Front Lines of American Medicine
By Rachel Pearson 
260 pp. W. W. Norton & Company. $26.95.


Just after residency at Bellevue Hospital in New York City, I worked briefly in a private practice in rural Florida. One afternoon, the E.R. called about a man with very high blood pressure — not high enough to be admitted to the hospital, but high enough to need prompt treatment. "Send him over to our office," I said.

When the Mexican farmworker arrived, the office manager hissed at me: "You can't just bring these patients here." Initially I was perplexed; I was preventing an admission to the hospital, saving thousands of dollars. But then I realized — this office would not treat him because he lacked insurance and means.

As the only person in the office who spoke Spanish, I had to break the news. This was the lowest moment in my medical career and I vowed never to have to utter such words to a patient again. I scrambled back to Bellevue and never looked back.

Rachel Pearson repeatedly found herself in the same miserable situation during her medical school training in Galveston, Tex. The island city had been devastated by Hurricane Ike in September 2008, just before Pearson arrived. There's no doubt that the University of Texas Medical Branch (U.T.M.B.) took a financial hit from the natural disaster, but many suspected that the draconian cuts to charity care were already in the works; the hurricane was merely convenient cover.

"It was January when Susan's patients began to die," Pearson writes in her engrossing book, part med-school memoir, part probing moral inquiry. Susan, a cancer surgeon at U.T.M.B., was unable to treat her patients after the medical center — without her knowledge — sent her patients a letter saying the doctor would be "discontinuing her professional relationship" with the people in her care. Without an operating room (and access to chemotherapy and radiation), the surgeon could only visit her patients at home and hold their hands as the disease killed them.

Pearson, who comes from a working-class family herself, finds her element at St. Vincent's House, a social-services center in one of Galveston's poorer neighborhoods, which were disproportionately devastated by Hurricane Ikeand then disproportionately ignored during the rebuilding. At first, she is "under the impression that there was a safety net." With the bare bones of donated supplies, the students diagnose cancer, heart disease and other standard medical fare. But they quickly learn that U.T.M.B. and other hospitals would not be stepping in to deliver medical treatment. A fellow medical student concludes bitterly, "I didn't realize that we were the safety net."

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Monday, August 28, 2017

Etiquette and the Cancer Patient – Season of the Witch – Ted Rheingold - Medium

I've failed to complete this post a number of times. It's laden with caveats and nuances and limited to just my learning, but it's important that everyone becomes familiar and adept at how to support the cancer patient. But I think it's applicable to any patient whose life is threatened.

Thanks to science and medicine, many Americans get well into middle age before a friend becomes gravely ill. We know how to talk about in hushed tones amongst ourselves but barring previous misfortune have no idea how to properly respond. But even more importantly I found many adults flounder to respond because they haven't had to grapple with overwhelming senses of despair and helplessness and the secret they dare not verbalize that they and their family are just as vulnerable. The last thing a patient needs is for their friends and acquaintances to respond from a crazed, fearful place. For some it's just too much and they can't even show up. For others it's becomes about them, offering to help just to avoid facing what I describe above. Plenty of others want to help simply because they don't know what to do. Some people figure it out quickly and get with the program. A small few have the life experience to approach it the right way, while some other expert humans are simply so comfortable accepting that life is change and emotions are life that they're naturals.

So, the first part of being good to the patient is to get your own head together. Be mindful of how this is making you feel. Channel your fear of this happening to your family into a celebration of being alive. Accept that feeling helpless isn't a shortcoming. Recognize despair and grief are part of the pantheon of life experiences. You do this because you don't want to make any of this about you. Support and love must flow to the patient and fear and discomfort must flow away. If you are not good at expressing love and gratefulness to your friend, learn how to do so quickly.

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https://medium.com/season-of-the-witch/etiquette-and-the-cancer-patient-630a50047448

Sunday, August 27, 2017

Ecstasy could be ‘breakthrough’ therapy for soldiers, others suffering from PTSD - The Washington Post

For Jon Lubecky, the scars on his wrists are a reminder of the years he spent in mental purgatory. 

He returned from an Army deployment in Iraq a broken man. He heard mortar shells and helicopters where there were none. He couldn't sleep and drank until he passed out. He got every treatment offered by Veterans Affairs for post-traumatic stress disorder. But they didn't stop him from trying to kill himself — five times.

Finally, he signed up for an experimental therapy and was given a little green capsule. The anguish stopped.

Inside that pill was a compound named MDMA, better known by dealers and rave partygoers as ecstasy. That street drug is emerging as the most promising tool to come along in years for the military's escalating PTSD epidemic.

The MDMA program was created by a small group of psychedelic researchers who had toiled for years in the face of ridicule, funding shortages and skepticism. But the results have been so positive that this month the Food and Drug Administration deemed it a "breakthrough therapy" — setting it on a fast track for review and potential approval.

The prospect of a government-sanctioned psychedelic drug has generated both excitement and concern. And it has opened the door to scientists studying new uses for other illegal psychedelics like LSD and psilocybin (commonly known as magic mushrooms).

"We're in this odd situation where one of the most promising therapies also happens to be a Schedule 1 substance banned by the [Drug Enforcement Administration]," said retired Brig. Gen. Loree Sutton, who until 2010 was the highest ranking psychiatrist in the U.S. Army. 

Because of the stigma attached to psychedelics since the trippy 1960s, many military and government leaders still hesitate to embrace them. Some scientists are also wary of the nonprofit spearheading ecstasy therapy, a group with the stated goal of making the banned drugs part of mainstream culture.

But the scope and severity of PTSD makes it all irrelevant, said Sutton, who now works as New York City's commissioner of veteran services. "If this is something that could really save lives, we need to run and not walk toward it. We need to follow the data."

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https://www.washingtonpost.com/national/health-science/ecstasy-could-be-breakthrough-therapy-for-soldiers-others-suffering-from-ptsd/2017/08/26/009314ca-842f-11e7-b359-15a3617c767b_story.html?

Major drug study opens up vast new opportunities in combating heart disease - The Washington Post

A landmark drug study has opened up a potent way to lower the risk of heart attacks — beyond the now standard advice of reducing cholesterol — promising new avenues of treatment of Americans' number one killer.

The findings, more than two decades after the discovery of powerful cholesterol-lowering drugs, called statins, taken by tens of millions, were announced Sunday at a medical conference in Barcelona and published in two leading medical journals.

Physicians not involved in the study described the results as a scientific triumph, calling the implications for drug treatment of heart disease "huge."

The findings provide validation of an idea that has been tantalizing cardiologists for years: that reducing inflammation could be a way to treat artery-clogging heart disease.

"It's a new paradigm: a new opportunity to further reduce death and disability," said Mark Creager, a past president of the American Heart Association, who was not involved in the study. "We've made such tremendous inroads in treating heart disease over the last couple of decades, and it's hard to imagine we could confer additional benefits, but here you go."

But the implications and timing of any benefit for patients remain to be seen. The drug company that sponsored the trial, Novartis, plans to meet with regulators this fall and file for approval by the end of the year. The drug, an injection given once every three months, would then be reviewed by the Food and Drug Administration.

A key question is which patients will benefit; the study showed its effect — a 15 percent drop in a combined measure of heart attacks, stroke and cardiovascular death — in a select, high-risk population of people who had suffered a previous heart attack and had high levels of a marker of inflammation in their blood. But a subset of patients appeared to get greater benefit from the drug, called canakinumab.

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https://www.washingtonpost.com/news/wonk/wp/2017/08/27/major-drug-study-opens-up-vast-new-opportunities-in-combating-heart-disease/?