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Update September 2017


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Update September 30, 2017

Lab-made “mini organs” helping doctors treat cystic fibrosis

This illustration provided by the Hubrecht Institute in Utrecht, Netherlands shows organoids (mini organs), created from the intestinal tissues of a patient. (Hubrecht Institute via AP)

Maria Cheng

Utrecht, Netherlands (AP) - Els van der Heijden, who has cystic fibrosis, was finding it ever harder to breathe as her lungs filled with thick, sticky mucus. Despite taking more than a dozen pills and inhalers a day, the 53-year-old had to stop working and scale back doing the thing she loved best, horseback riding.

Doctors saw no sense in trying an expensive new drug because it hasn’t been proven to work in people with the rare type of cystic fibrosis that van der Heijden had.

Instead, they scraped a few cells from van der Heijden and used them to grow a mini version of her large intestine in a petri dish. When van der Heijden’s “mini gut” responded to treatment, doctors knew it would help her too.

“I really felt, physically, like a different person,” van der Heijden said after taking a drug - and getting back in the saddle.

This experiment to help people with rare forms of cystic fibrosis in the Netherlands aims to grow mini intestines for every Dutch patient with the disease to figure out, in part, what treatment might work for them. It’s an early application of a technique now being worked on in labs all over the world, as researchers learn to grow organs outside of the body for treatment - and maybe someday for transplants.

So far, doctors have grown mini guts - just the size of a pencil point - for 450 of the Netherlands’ roughly 1,500 cystic fibrosis patients.

“The mini guts are small, but they are complete,” said Dr. Hans Clevers of the Hubrecht Institute, who pioneered the technique. Except for muscles and blood vessels, the tiny organs “have everything you would expect to see in a real gut, only on a really small scale.”

These so-called organoids mimic features of full-size organs, but don’t function the same way. Although many of the tiny replicas are closer to undeveloped organs found in an embryo than adult ones, they are helping scientists unravel how organs mature and providing clues on how certain diseases might be treated.

In Australia, mini kidneys are being grown that could be used to test drugs. Researchers in the U.S. are experimenting with tiny bits of livers that might be used to boost failing organs. At Cambridge University in England, scientists have created hundreds of mini brains to study how neurons form and better understand disorders like autism. During the height of the Zika epidemic last year, mini brains were used to show the virus causes malformed brains in babies.

In the Netherlands, the mini guts are used as a stand-in for cystic fibrosis patients to see if those with rare mutations might benefit from a number of pricey drugs, including Orkambi. Made by Vertex Pharmaceuticals, Orkambi costs about 100,000 euros per patient every year in some parts of Europe, and it’s more than double that in the U.S., which approved the drug in 2015. Despite being initially rejected by the Dutch government for being too expensive, negotiations with Vertex were reopened in July.

Making a single mini gut and testing whether the patient would benefit from certain drugs costs a couple of thousand euros. The program is paid for by groups including health insurance companies, patient foundations and the government. The idea is to find a possible treatment for patients, and avoid putting them on expensive drugs that wouldn’t work for them.

About 50 to 60 patients across the Netherlands have been treated after drugs were tested on organoids using their cells, said Dr. Kors van der Ent, a cystic fibrosis specialist at the Wilhelmina Children’s Hospital, who leads the research.

Clevers made a discovery about a decade ago that got researchers on their way. They found pockets of stem cells, which can turn into many types of other cells, in the gut. They then homed in a growing environment in the lab that spurred these cells to reproduce rapidly and develop.

“To our surprise, the stem cells started building a mini version of the gut,” Clevers recalled.

Cystic fibrosis is caused by mutations in a single gene that produces a protein called CFTR, responsible for balancing the salt content of cells lining the lungs and other organs.

To see if certain drugs might help cystic fibrosis patients, the medicines are given to their custom-made organoids in the lab. If the mini organs puff up, it’s a sign the cells are now correctly balancing salt and water. That means the drugs are working, and could help the patient from whom the mini gut was made.

Researchers are also using the mini guts to try another approach they hope will someday work in people - using a gene editing technique to repair the faulty cystic fibrosis gene in the organoid cells.

Other experiments are underway in the Netherlands and the U.S. to test whether organoids might help pinpoint treatments for cancers involving lungs, ovaries and pancreas.

While the idea sounds promising, some scientists said there are obstacles to using mini organs to study cancer.

Growing a mini cancer tumor, for example, would be far more challenging because scientists have found it difficult to make tumors in the lab that behave like in real life, said Mathew Garnett of the Wellcome Trust Sanger Institute, who has studied cancer in mini organs but is not connected to Clevers’ research.

Also, growing the cells and testing them must happen faster for cancer patients who might not have much time to live, he said.

Meanwhile, Clevers wants to one day make organs that are not so mini.

“My dream would be to be able to custom-make organs,” he said, imagining a future where doctors might have a “freezer full of livers” to choose from when sick patients arrive.

Others said while such a vision is theoretically possible, huge hurdles remain.

“There are still enormous challenges in tissue engineering with regards to the size of the structure we’re able to grow,” said Jim Wells, a pediatrics professor at the Cincinnati Children’s Hospital Medical Center. He said the mini organs are far smaller than what would be needed to transplant into people and it’s unclear if scientists can make a working, life-sized organ in the lab.

There are other limitations to growing miniature organs in a dish, said Madeline Lancaster at Cambridge University.

“We can study physical changes and try to generate drugs that could prevent detrimental effects of disease, but we can’t look at the complex interplay between organs and the body,” she said.

For patients like van der Heijden, who was diagnosed with cystic fibrosis as a toddler, the research has helped her regain her strength. Vertex agreed to supply her with the drug.

“It was like somebody opened the curtains and said, ‘Sunshine, here I am, please come out and play’,” she said. “It’s strange to think this is all linked to some of my cells in a lab.”


Update September 23, 2017

New drug reduces heart attacks, but is that enough?

A new type of cholesterol drug meant to prevent heart attacks and other complications clearly did so, in an unusually large study whose results were announced Tuesday, Aug. 29, 2017, at a conference of heart specialists. (AP Photo/Mel Evans, File)

Linda A. Johnson

Trenton, N.J. (AP) - So-so results for a new type of cholesterol drug have left Merck in a quandary: Does the company try to bring it to market or scrap it?

A large, long-term study of the drug showed that it prevents heart attacks and reduces the need for heart procedures, while three similar drugs developed by rivals failed. But the drug, anacetrapib, only reduced those complications by 9 percent.

Now Merck, which has spent 13 years and likely hundreds of millions of dollars testing the drug, has to decide whether to spend even more to seek approval from regulators and convince people to buy it in a market full of cholesterol drugs.

The results of the 30,450-patient study were announced Tuesday, Aug. 29, 2017, at a conference of heart specialists in Barcelona, Spain and published in the New England Journal of Medicine.  The study found that anacetrapib is safe and somewhat effective.

That kind of result is normally enough to seek approval to market a new medicine, especially for heart disease, which is the top killer in many developed countries. Yet even after seeing the results weeks ago, Merck says its executives are still consulting with medical experts and regulators on whether to go through the costly process of applying for approval.

Analyst Steve Brozak, president of WBB Securities, predicts Merck will do so, given anacetrapib’s safety, the huge pool of potential patients and all the resources Merck has poured into the drug.

“This will get used,” Brozak said.

Merck would likely price the bill somewhere between the two extremes that now define the market for cholesterol drugs.

Generic versions of brand-name statin cholesterol pills including Lipitor, Crestor and Merck’s own Zocor now cost $10 to $20 a month. Repatha and Praluent, two new injected medicines in a different drug category that have been shown to dramatically reduce cholesterol, cost $14,000 a year.

Georgetown University cardiologist Dr. Allen J. Taylor said he thinks the drug would be approved by the Food and Drug Administration despite its “relatively weak benefit.”

“If you were discussing this with patients,” Taylor said, “you would have to tell them that when you start this, you’ll have to take it for four years to have a 1 percent chance of preventing an event,” meaning a heart attack or a procedure such as bypass surgery or implanting a stent to keep an artery open.

Taylor said it’s still unclear how anacetrapib controls cholesterol, which would make it hard for doctors to determine which patients would benefit much from it. But he and Brozak praised the company for doing such an exhaustive, expensive study in an era when many studies are quick and relatively small, sometimes producing unclear results.

In the study, patients getting anacetrapib plus a statin for four years had, on average, lower levels of bad cholesterol and other fats, and higher levels of good cholesterol, compared to a group getting a statin and a dummy pill. But Merck’s drug didn’t prevent any deaths from heart attacks or other cardiac problems.

Anacetrapib’s only worrisome side effect was a long-term accumulation of the drug in patients’ fatty tissue. It’s unknown if that will cause problems, so Merck plans to study that by following some participants for two years after they stop taking the drug.


Update September 16, 2017

US clears breakthrough gene therapy for childhood leukemia

In this July 9, 2015, photo, provided by Novartis Pharmaceuticals Corp., human T cells belonging to cancer patients arrive at Novartis Pharmaceuticals Corp.’s Morris Plains, N.J., facility. (Brent Stirton/Novartis Pharmaceuticals Corp. via AP)

Lauran Neergaard

Washington (AP) - Opening a new era in cancer care, U.S. health officials have approved a breakthrough treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia.

The Food and Drug Administration said the approval was historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it’s one of a wave of “living drugs” under development to fight additional blood cancers and other tumors, too.

Novartis Pharmaceuticals set the price for its one-time infusion of so-called “CAR-T cells” at $475,000, but said there would be no charge for patients who didn’t show a response within a month.

“This is a brand new way of treating cancer,” said Dr. Stephan Grupp of Children’s Hospital of Philadelphia, who treated the first child with CAR-T cell therapy - a girl who’d been near death but now is cancer-free for five years and counting. “That’s enormously exciting.”

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient’s blood, reprogram them to harbor a “chimeric antigen receptor” or CAR that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

It’s a completely different way to harness the immune system than popular immunotherapy drugs called “checkpoint inhibitors” that treat a variety of cancers by helping the body’s natural T cells better spot tumors. CAR-T cell therapy gives patients stronger T cells to do that job.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb.

The first CAR-T version, developed by Novartis and the University of Pennsylvania, is approved for use by several hundred patients a year who are desperately ill with acute lymphoblastic leukemia, or ALL. It strikes more than 3,000 children and young adults in the U.S. each year and while most survive, about 15 percent relapse despite today’s best treatments.

In a key study of 63 advanced patients, 83 percent went into remission soon after receiving the CAR-T cells. Importantly, it’s not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare long-term.

Still, “a far higher percentage of patients go into remission with this therapy than anything else we’ve seen to date with relapsed leukemia,” said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites. “I wouldn’t say we know for sure how many will be cured yet by this therapy. There certainly is a hope” that some will be.

Most patients suffered side effects that can be grueling, even life-threatening. An immune overreaction called “cytokine release syndrome” can trigger high fevers, plummeting blood pressure and in severe cases organ damage, side effects that require sophisticated care to help patients without blocking the cancer attack. The FDA designated a treatment for those side effects Wednesday.

“This is remarkable technology,” said Dr. Mikkael Sekeres of the American Society of Hematology. But, he cautioned that CAR-T “isn’t a panacea.”

Among concerns, sometimes leukemia can develop resistance, and sometimes patients worsen while waiting for their new cells, said Sekeres, who directs the Cleveland Clinic’s leukemia program and wasn’t involved with CAR-T testing.

“Unfortunately leukemia grows so rapidly that it can evade even the smartest of our technologies,” he added.

To better ensure patient safety, the FDA is requiring Novartis to offer CAR-T therapy only through medical centers specially trained and certified to handle the complicated treatment. Novartis expects to have 32 centers around the country, mostly in large cities, running by year’s end, with the first 20 offering care within the next month.

Patients’ collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks. The $475,000 price tag doesn’t include the cost of needed hospitalizations, travel to a certified hospital and other expenses.

On a conference call Wednesday, Novartis executives said the company is working with the Medicaid program and private insurers and expects broad coverage, and will offer some financial assistance with such things as copay and travel costs. But they didn’t promise all patients would be able to get the therapy.

For some patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study.

“I don’t want to be an apologist for high drug prices in the U.S.,” Grupp stressed. But if it’s the last treatment they need, “that’s a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them.”

“This is a turning point in the fight” against ALL, said Penn’s Dr. Carl June, who pioneered the therapy.

But he and other researchers say thousands more patients eventually may benefit. Kite Pharma’s similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma.

Scientists around the country also are trying to make CAR-T therapies that could fight more common solid tumors such as brain, breast or pancreatic cancers - a harder next step.

“Although narrow in scope, today’s FDA ruling is a milestone,” said Dr. David Maloney of the Fred Hutchinson Cancer Research Center in Seattle, whose team has worked with Juno and is researching CAR-T in a variety of cancers. “Approvals are an important step, but they’re just the beginning.”


Update September 9, 2017

Is it really Lyme? Researchers developing a new test to tell

In this undated photo provided by the U.S. Centers for Disease Control and Prevention (CDC), a blacklegged tick - also known as a deer tick. Diagnosing if a tick bite caused Lyme or something else can be difficult but scientists are developing a new way to catch the disease early, using a “signature” of molecules in patients’ blood. (CDC via AP)

Lauran Neergaard

Washington (AP) - Diagnosing if a tick bite caused Lyme or another disease can be difficult but scientists are developing a new way to do it early - using a “signature” of molecules in patients’ blood.

It’s still highly experimental, but initial studies suggest the novel tool just might uncover early-stage Lyme disease more accurately than today’s standard test, researchers reported Wednesday. And it could tell the difference between two tick-borne diseases with nearly identical early symptoms.

“Think about it as looking at a fingerprint,” said microbiology professor John Belisle of Colorado State University, who helped lead the research.

Lyme disease is estimated to infect 300,000 people in the U.S. every year. Lyme-causing bacteria are spread by blacklegged ticks - also called deer ticks - primarily in the Northeast and Midwest, although their range is spreading. Lyme typically starts as a fever, fatigue and flu-like symptoms - often but not always with a hallmark bulls-eye rash - and people usually recover quickly with prompt antibiotics. But untreated, Lyme causes more serious complications, including swollen joints and arthritis, memory and concentration problems, even irregular heartbeat.

Yet today’s best available test often misses early Lyme. It’s considered no more than 40 percent accurate in the first few weeks of infection. It measures infection-fighting antibodies the immune system produces. Those take a while to form, making the test more useful a month or more after infection sets in than when people first start feeling ill.

“We are trying our best to come up with something to help the diagnosis in the very early stages of this infection,” said microbiologist Claudia Molins of the Centers for Disease Control and Prevention, who teamed with Belisle to develop a new test. “Our goal really is to try to fill that gap.”

The new approach essentially looks for a biochemical fingerprint that shows the body is beginning to respond to an infection, long before antibodies mobilize. It’s based on cellular metabolism, subtle changes in the kind and amount of small molecules that cells produce, such as sugars and amino acids and fats.

First, Belisle and Molins found a signature - specific changes in those metabolites - that enabled them to distinguish between blood from Lyme patients and from healthy people.

The tougher hurdle: Could the tool also tell the difference between Lyme and a disease with very similar symptoms? To tell, they compared a mysterious Lyme look-alike called Southern Tick-Associated Rash Illness, or STARI.

STARI is spread by a different tick, the Lone Star tick that is found widely throughout the East and Southeast, areas that overlap with the Lyme-carrying blacklegged ticks. STARI involves a round rash and other symptoms similar to early Lyme, and is treated with the same antibiotic - but it’s not caused by the same bacteria. In fact, scientists don’t yet know the cause of STARI, and there’s no test for it. The only way to identify STARI is to definitively rule out other ailments.

Using carefully stored blood samples from people determined to have either Lyme or STARI, Belisle and Molins found biomarkers that could tell the two disease apart. Using those markers to study additional blood samples, they concluded their tool was 82 percent accurate in determining early Lyme - far better than today’s standard, Molins said.

The research was published in the journal Science Translational Medicine.

“It is a very novel way of looking at diagnosis,” said Dr. John Aucott, who directs Johns Hopkins University’s Lyme Disease Clinical Research Center. He was not involved in the new study, and notes that other test approaches are being explored, too.

Creating a test will take several more years of research, cautioned Molins. First, the team is turning its sophisticated metabolic-measuring techniques into a test that standard laboratories could use. Then, with next spring’s tick season, researchers will start a new round of testing.

Hopkins’ Aucott cautions that other infections unrelated to ticks can be confused with Lyme, too, so any new test also would have to rule out those possibilities.

But better early diagnosis isn’t the only need, he stressed. Today’s Lyme test also can’t prove if antibiotic treatment was successful, because it can detect antibodies even years after people recovered. He wonders if tracking cellular metabolism could solve that problem.

“If you can show the host metabolic signature goes back to normal, that could be a great test of cure,” he said.


Update September 2, 2017

Science Says: DNA test results may not change health habits

National Institutes of Health (NIH) Director Francis Collins poses for a portrait at the NIH headquarters in Bethesda, Md., Friday, July 28, 2017. After DNA testing showed he was predisposed to Type 2 diabetes, which is more likely to develop if a person is overweight or obese, Collins shed 35 pounds (16 kilograms). (AP Photo/Sait Serkan Gurbuz)

Malcolm Ritter

New York (AP) - If you learned your DNA made you more susceptible to getting a disease, wouldn’t you work to stay healthy?

You’d quit smoking, eat better, ramp up your exercise, or do whatever else it took to improve your odds of avoiding maladies like obesity, diabetes, heart disease or cancer, right?

The scientific evidence says: Don’t bet on it.

DNA testing for disease risk has recently expanded in the U.S. The company 23andMe recently started selling the nation’s first approved direct-to-consumer DNA tests that evaluate the buyer’s genetic risk for certain disease or conditions. That go-ahead came in April, about three years after it was told to stop selling such kits until it got the OK from regulators.

The field also gained a new entrant in July, when a company called Helix launched an online marketplace for DNA tests, including some for genetic health risk. Helix decodes a consumer’s DNA and passes the results along to another company for analysis. A request for the currently available health tests must be approved by a physician’s group that reviews the customer’s medical history.

DNA tests for diseases typically assess genetic predisposition to getting sick. They don’t provide absolute predictions about whether or not a disease will strike. Genetic risk is only part of a person’s overall risk, which includes influence from other things like a person’s lifestyle.

While some disease are caused by a single malfunctioning gene, more common illnesses are influenced by multiple genes, and often each gene nudges a person’s risk only slightly.

A 23andMe test that includes ancestry and other information goes for $199. Helix’s decoding costs $80, while the currently available health-risk analyses cost $150 and $125. Both companies use a saliva sample for the test.

Last year, researchers published an analysis that combined 18 studies of people who got doctor-ordered DNA test results about disease risks. None involved direct-to-consumer tests; participants were drawn mostly from medical clinics or elsewhere. Eight of the 18 studies were done in the United States.

The result? Getting the DNA information produced no significant effect on diet, physical activity, drinking alcohol, quitting smoking, sun protection or attendance at disease-screening programs.

That fits with other results showing that, on balance, getting the information “has little if any impact on changing routine or habitual behaviors,” said psychologist Theresa Marteau of Britain’s Cambridge University, a study author.

In an interview, Dr. James Lu, a co-founder of Helix, agreed that the evidence on whether people change their lifestyles in response to DNA information is mixed. But he said it’s more likely if they get the right information, education and support.

“We’re learning a lot as the field evolves,” Lu said.

Marteau is not claiming that testing never changes behavior. She notes the example of Dr. Francis Collins, director of the U.S. National Institutes of Health. After DNA testing showed he was predisposed to Type 2 diabetes, which is more likely to develop if a person is overweight or obese, Collins shed 35 pounds (16 kilograms).

“It was a kick in the pants,” Collins explained. “It was an opportunity to wake up and say, maybe I’m not going to be immortal and maybe there are things I am doing to myself that aren’t healthy that I ought to change.”

Dr. Robert C. Green of Brigham and Women’s Hospital in Boston, whose research indicates DNA test results can change health behavior, said cases like Collins are just the point.

It’s very hard to get people to improve health habits, and even when they do, it’s hard for researchers to prove that DNA test results were responsible, he said.  So it’s no surprise that evidence favoring an effect is limited, he said.

“It doesn’t necessarily mean that it doesn’t help some people,” said Green, who’s also a scientific adviser to several companies involved in genetic testing.

He and co-authors in May reported evidence that simply going through the process of DNA testing may slightly improve diet and exercise, regardless of what the results reveal. Maybe the experience serves to remind and motivate people about beneficial health behaviors, the authors said.

Green also said that people seek such results for a number of reasons, including simple curiosity, so the value of DNA testing should not be judged simply by whether it changes health behavior.

“I think people have a right to this information,” he said.
 


DAILY UPDATEE

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HEADLINES [click on headline to view story]

Lab-made “mini organs” helping doctors treat cystic fibrosis


New drug reduces heart attacks, but is that enough?


US clears breakthrough gene therapy for childhood leukemia


Is it really Lyme? Researchers developing a new test to tell


Science Says: DNA test results may not change health habits


 



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