How the U.S. can beat hepatitis C: Don’t buy the pills, buy the company

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Pharma veteran and MIT visiting scientist Mark Trusheim advances a provocative solution to overcome treatment costs.

 

In a recent article in Forbes, MIT Sloan School of Management visiting scientist Mark Trusheim and co-author Peter B. Bach, a physician at Memorial Sloan Kettering Cancer Center in New York, propose a startling response to the prevalence of hepatitis C and the high cost of the leading therapy: The U.S. government should not just pay for the pills — they should buy their maker.

The pharmaceutical company Gilead sells hepatitis C drugs that have 80 percent market share at an estimated average cost per patient as high as $42,000 per curative treatment course. In clinical studies it’s shown to halt, and in some cases reverse, liver damage caused by the disease. “In this unique case … there is an intersection of a clear winning therapy, large public health need, slow adoption, high product pricing,” and a solid business case, say the authors.

By taking the unorthodox approach of buying Gilead and divesting the parts not related to U.S. hepatitis C treatment, the United States “will make [the therapy] affordable to rapidly treat the 2.7 million Americans that the CDC estimates still have hepatitis C,” and save money in the long run, compared to the traditional approach of reimbursing for treatments, Trusheim and Bach say.

This unprecedented solution, backed by a break-up analysis and financial cost-benefit calculations, fulfills another public aim: “Improving the health and wellbeing of the U.S. is the very objective of public investment in research, medical care, and in this one case, the stock market,” the authors assert.

“In a desire to cure more patients faster, we discovered that private equity financial tools could provide a more effective route than what the current drug purchasing approaches are achieving,” said Trusheim, who is also a strategic director of the MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative. NEWDIGS convenes a broad sample of healthcare stakeholders — including global leaders in research, development, insurance, regulation, clinical care, and patient advocacy — to develop and pilot innovative biomedical innovation practices that benefit all.

Current hepatitis C treatment costs are limiting their impact on patient care. “Science is making remarkable treatments possible,” said Gigi Hirsch, executive director of NEWDIGS and the MIT Center for Biomedical Innovation. “The creativity and analytic rigor that Peter and Mark demonstrate in this proposal stimulate the kinds of collaborative discussions we need to overcome the financial barriers between patients and the medicines they need.”

 

Source: http://news.mit.edu/2017/how-us-can-beat-hepatitis-c-dont-buy-pills-buy-company-0120

New prospects for growing human replacement organs in animals

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For the first time, biologists have succeeded in growing human stem cells in animal embryos, shifting from science fiction to the realm of the possible the idea of developing human organs in animals for later transplant.
The approach involves generating stem cells from a patient’s skin, growing the desired new organ in a large animal, and then harvesting it for transplant into the patient’s body. Since the organ would be made of a patient’s own cells, there would be little risk of immune rejection.
The human-organ-growing animals would be examples of chimeras, animals composed of two different genomes. They would be generated by implanting human stem cells into an early embryo, resulting in an animal composed of mixed animal and human cells.
One team of biologists, led by Jun Wu and Juan Carlos Izpisua Belmonte at the Salk Institute, has shown for the first time that human stem cells can contribute to forming the tissues of an animal, despite the 90 million years of evolution between the two species.
Another group, headed by Tomoyuki Yamaguchi and Hideyuki Sato of the University of Tokyo, and Hiromitsu Nakauchi of Stanford, has reversed diabetes in mice by inserting pancreas glands composed of mouse cells that were grown in a rat. The Salk team’s report was published in Cell and the Stanford-Tokyo team’s in Nature.
The two reports together establish the feasibility of trying to grow replacement human organs in animals, though such a goal is still far off.
“I think this is very promising work in principle,” said Rudolf Jaenisch, a stem cell expert at the Whitehead Institute in Cambridge, Masschusetts.
Many technical and ethical barriers have yet to be overcome, but the research is advancing alongside the acute need for organs; some 76,000 people in the United States alone are awaiting transplants.
Creating chimeras, especially those with human cells, may prove controversial, given the possibility that test animals could be humanised in undesirable ways. One would be if human cells should be incorporated into an animal’s brain, endowing it with human qualities. Almost no one wants a talking animal.
Another untoward outcome would be if human cells should come to compose the animal’s reproductive tissues. Few people want to see what might result from the union between an animal with human sperm and an animal with human eggs.
Izpisua Belmonte’s and Nakauchi’s teams have both pursued a strategy of directing the human donor cells to generate specific organs in the recipient species. This is desirable for both technical and ethical reasons.
Nakauchi has disabled the master gene in rats for making a pancreas so that when mouse stem cells are injected into the early embryo of such a rat, the growing embryo has no choice but to construct its pancreas of pure mouse cells, instead of the usual mixture of rat and mouse cells.
The result provides proof of principle that Type 1 diabetes can be treated by growing a pancreas from an individual’s cells in another animal, Nakauchi and colleagues conclude.
The next step is to repeat the experiment in bigger animals, which produce organs of a more suitable size for use in humans. Izpisua Belmonte’s team has now shown that human stem cells do survive in the animal’s embryos and help form their organs, although not very efficiently.
“The human cell doesn’t contribute much. To the brain we observed little or no contribution at all,” said his colleague Wu.”This is good news because we can guide the human cells to the organ we want.”
Both Izpisua Belmonte and Nakauchi said there was a long way to go before human organs could successfully be grown in animals. Chimeras will be more immediately useful in studying human embryogenesis, testing drugs and following the progress of disease.
Both scientists expressed confidence that ethical concerns about chimera research could be addressed. Chimeras are typically mosaics in which each organ is a mixture of the host and donor cells. But new techniques like the Crispr-Cas gene editing system should allow the human cells in an animal embryo both to be channelled into organs of interest and to be excluded from tissues of concern like the brain and reproductive tissues.
“This isn’t dangerous research. We’re not creating monsters,” Nakauchi said.
“There isn’t a need to get into a debate about moral humanisation if scientists target the organs where the human cells will go,” said Insoo Hyun, a medical ethicist at Case Western Reserve University.”Scientists are not making chimeras just for fun ” it’s to relieve the dire shortage of transplantable organs.”

Concern about human cells’ incorporation into a reduce animal’s mind is not with out basis. Dr. Steven Goldman of the College of Rochester Clinical Center discovered in 2013 that mice injected with a particular style of human mind mobile experienced enhanced understanding talents. But other kinds of humanized mice, this sort of as mice engineered to have a human immune system, are schedule laboratory animals that appear to be to event very little angst.

Dr. Izpisua Belmonte’s insertion of human stem cells into pig embryos was not influenced by the N.I.H. moratorium on this sort of chimeras mainly because he utilized personal cash. His experiment was accredited by the authorities in Spain and in California, and following their guidance, the improvement of the pig chimeras was stopped following four weeks in the womb.

Dr. Nakauchi moved his lab to Stanford from Tokyo in 2014 mainly because Japanese rules do not permit chimera exploration, only to be strike with the N.I.H. moratorium a year afterwards, which prevented him from generating chimeras with human cells. His mouse pancreas experiment has taken eight or nine many years to finish. “I have been in a very aggravating problem,” he said.

Source: https://www.nytimes.com/2017/01/26/science/chimera-stemcells-organs.html?rref=collection%2Fsectioncollection%2Fhealth&action=click&contentCollection=health&region=rank&module=package&version=highlights&contentPlacement=9&pgtype=sectionfront

 

Chinese Investors Have Money, Want Biotech and Don’t Fear Trump

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Despite tough talk by President-elect Donald Trump about a possible trade war, Chinese investors are hungry for U.S. biotech and they’ve got money to spend.

In the latest purchase following a record 2016 for Chinese health-care takeovers, Nanjing-based Sanpower Group Co., said Tuesday it would buy a cancer treatment business from Valeant Pharmaceuticals International Inc. for $820 million.

Sanpower’s interest is far from unique: Chinese venture capitalists and individual investors are looking as well. Last Sunday morning, in a hotel ballroom a short hop from the San Francisco airport, about 250 jet-lagged potential dealmakers and American biotech executives could be heard chatting in Mandarin and English. They were attending a get-together hosted by boutique investment firm CTIC Capital, hoping to forge alliances before the start of the industry’s biggest annual gathering, the J.P. Morgan Healthcare Conference, at the Westin St. Francis in San Francisco, which runs this week.

China has a “huge pile of money’’ and a lot of new venture capital firms, but a limited number of homegrown drug startups to invest in, said Kevin Chen, a partner at the China Fund of Menlo Park, California-based Sequoia Capital. The fund, which spends about a quarter of its $3 billion under management on health care, last year started founding U.S.-based startups with Chinese ties, helping bridge the gap for investors.

Buying Spree

U.S. investors are beginning to notice. The J.P. Morgan conference has an entire track dedicated to China-based health-care companies at the conference. And last year, Chinese firms announced or completed about $8 billion in cross-border acquisitions, according to data compiled by Bloomberg.

“The gate is open and it won’t be closed again,” said Lan Huang, chief executive officer of BeyondSpring Pharmaceuticals Inc., a maker of cancer drugs with headquarters in New York and offices in Dalian, China. Her company, which plans to go public, has been meeting with investors from both countries.

The conference comes at a pivotal moment for the two nations. Trump’s rhetoric toward China has some investors worrying about a chilling effect on business. He has promised tariffs on Chinese goods, and China has said it’s prepared to step up its scrutiny of U.S. companies.

“If there’s no clear sign that it’s safe to invest in the U.S. and that innovation will import to China, that’s going to hurt investors,’’ said Jennifer Hu, a partner at China-based Qiming Ventures Partners. “They could go to Australia or Canada for that innovation instead.’’

Qiming, which spends about 40 percent of its $2.7 billion in funds on health care, is currently raising money for its first fund to be dedicated to U.S. health investments. Up to now, Qiming had focused its investments on Chinese companies.

Yet unless there’s “dramatic change” in U.S. policy, Sequoia’s Chen sees Chinese investors continuing to push money toward U.S. biotechs.

Out of Reach

Reasons include a shortage of Chinese companies whose financials pencil out. Many that have gone public are “unicorns” worth more than $1 billion — valuations that may be out of reach, said Yanhong Lin, founder and managing partner of CTIC, which is based in Palo Alto, California, and has offices in Shanghai and Nanjing.

While Chinese regulators are becoming more amenable to approving homegrown drugs, the U.S. is still perceived as the place to go for growth.

“The U.S. still represents the highest technology,” said Huang, the BeyondSpring CEO. As Chinese investors “increasingly are trying to think globally, this meeting is their opportunity.”

 

Source: https://www.bloomberg.com/news/articles/2017-01-10/china-investors-have-money-want-biotech-and-don-t-fear-trump

Digital Addiction Therapy Could Be First FDA-Approved App

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LAS VEGAS — Digital therapy could soon be prescribed and reimbursed like a pharmaceutical drug for patients with chronic substance use disorder.

“This could be a watershed moment for digital health,” said Corey McCann, MD, PhD, chief executive officer of Pear Therapeutics, the developer of the software. The product, known as reSET, is currently under review by the US Food and Drug Administration (FDA), and there have been “a lot of positive conversations back and forth with the FDA,” he reported.

“We have a good deal of clarity around the label and timelines, so I think it’s fair to say that we’ll see this happen in 2017,” Dr McCann told Medscape Medical News.

Dr McCann took part in a discussion on the opioid epidemic here at the Consumer Technology Association 2017 Digital Health Summit, where leaders in digital health technologies talked about various strategies to manage the crisis.

Pear Therapeutics is working toward a reimbursement model for digital therapy that mirrors the steps a pharmaceutical company takes to approve a new drug. “Based on our conversations, we are eager to pursue a label for our product for the treatment of substance use disorder, to enhance abstinence and to enhance retention in treatment,” he explained.

This could be a watershed moment for digital health.

With rates of opiate addiction soaring in the United States, there is a need for new and innovative ways to deal with the problem. A digital solution would radically disrupt the traditional management of chronic disease, and has given investors reason to see big potential in the digital therapeutics market. “Software as a drug” has the potential to grow to a $6 billion market in the next 5 years, according to a recent research report by Goldman Sachs, which looked at an analysis by Psilos.

The basis of the digital therapy software, which delivers a comprehensive behavioral program, is the belief that environmental contingencies can play a powerful role in encouraging or discouraging drinking or drug use. It provides patients with a set of rewards for abstinent behavior and requires them to complete a set of on-screen modules that leverage the community-reinforcement approach.

The reSET software, which is also available as an app, has been studied in randomized clinical trials of patients with substance use disorders related to opiates, stimulants, cannabis, cocaine, and alcohol.

The company’s pivotal clinical trial involved 507 patients from 10 outpatient addiction treatment programs, as previously reported by Medscape Medical News (Am J Psychiatry. 2014;171:683-690). The 252 patients in the control group received usual care for 12 weeks, and the 255 in the intervention group received usual care, but four internet-based modules, completed on site or remotely each week, replaced 2 hours of counseling.

The 62 modules teach basic cognitive behavioral skills for relapse prevention, such as refusing drugs, managing thoughts about using, conducting functional analyses, and improving psychosocial functioning (communication, mood management, family and social relations, time management). They also cover the prevention of HIV, hepatitis, and other sexually transmitted infections.

Patients who completed the required software modules each week and proved abstinence through negative urine tests or alcohol breathalyzer screens earned contingency-management vouchers, or low-cost rewards, which were distributed by the research staff.

Retention in treatment was better in the intervention group than in the control group. In patients abstinent at study entry, rates of abstinence were equally high in the two groups; in patients not abstinent at study entry, the odds of abstinence was twice as high in the intervention group as in the control group.

The rate of abstinence during study weeks 9 to 12 was higher in the intervention group than in the control group (58.1% vs 29.8%; P < .01). In addition, patients in the intervention group had significantly more half-weeks of abstinence than those in the control group (11.1 vs 8.8; P = .008), and more consecutive half-weeks of abstinence (8.0 vs 5.1; P = .001).

The authors of that study noted that it was impossible to disentangle the unique effects of the computerized community-reinforcement approach and contingency management. That limitation was addressed in a second study looking at digital therapy for opiate addiction.

Second Study on Digital Therapy to Augment Methadone Program

Clinically relevant results with reSET-O, which addressed opiate addiction, were shown in another study by Pear Therapeutics (J Subst Abuse Treat. 2014;46:43-51).

In that trial, 160 people receiving a daily dose of methadone were randomly assigned to standard treatment with 1 hour of counseling each week or to 30 minutes of counseling plus 30 minutes of digital therapy.

All participants received contingency management in this study, which consisted of $50 for completing baseline and monthly clinical assessments and $10 for each urine sample provided.

Contingency-management incentives, often used as part of a packaged intervention, were shown to make an independent contribution to a community-reinforcement approach delivered by highly trained therapists (Arch Gen Psychiatry. 2003;60:1043-1052). However, the reSET-O study is the first to demonstrate the effectiveness of a computerized community-reinforcement approach “in the absence of contingency-management incentives,” the study investigators report.

Abstinence was better in the intervention group than in the usual-care group, and the drop-out rate was lower.

Other digital therapies from Pear Therapeutics used in combination with pharmaceuticals have been shown to be effective for people with schizophrenia or PTSD.

Reimbursement for Digital Therapy

Digital therapy “is not a panacea,” Dr McCann cautioned. “It’s not the sort of thing that you just download and it will produce a result.” When used with a medical support, he said, the software shows good efficacy. “We have remarkably good retention data. There’s a certain degree of power that comes with a physician prescription,” Dr McCann explained. “We are working toward a reimbursement model.”

The idea of seeking FDA approval for the software as a treatment intervention is supported by Maxine Stitzer, PhD, professor of psychiatry and behavioral sciences at the Hopkins Bayview Medical Center in Baltimore, who was involved in the first reSET study.

“We could now prescribe this mobile therapy to our patients. I think that’s good,” she said.

“This is the modern age; why should we be dragging our feet?” she asked. The fact that all patients receive the same material is a bonus. “It’s the same for everyone, but patients can go over it at their own pace.” In fact, she said, a lot of the material is standard cognitive behavior therapy that would be delivered by a counselor, but in this case it is delivered online.

She pointed out that some groups of people had difficulty with the software, especially those with a lower level of education. The concept of reimbursement for digital therapy is good, she said, “but we do want to make sure that it is efficacious in the way it is being delivered.”

Others said they agree.

“For the right patient, I would be willing to try an app,” said session moderator Arshya Vahabzadeh, MD, chief medical officer for Brain Power. He said he applauds Pear Therapeutics for going through clinical trials to test their approach. “It’s a high bar, and it would suggest robustness. Not all companies are doing that; it’s too expensive for most digital companies.”

“There is research showing that people with mental illness are willing and able to use devices,” Dr Vahabzadeh reported. “But I have to say that some psychiatrists, in particular those focusing on psychotherapy instead of medication management, may be reluctant to prescribe an app because of the effect the technology might have on the doctor–patient therapeutic relationship.”

“It could put a wedge between them,” he pointed out.

Dr McCann is chief executive officer of Pear Therapeutics. Dr Stitzer and Dr Vahabzadeh have disclosed no relevant financial relationships.

Consumer Technology Association 2017 Digital Health Summit. Presented January 6, 2017.

 

Source: http://www.medscape.com/viewarticle/874490

The Stem-Cell Revolution Is Coming — Slowly

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In 2001, President George W. Bush issued an executive order banning federal funding for new sources of stem cells developed from preimplantation human embryos. The action stalled research and discouraged scientists.

Five years later, a Kyoto University scientist, Shinya Yamanaka, and his graduate student, Kazutoshi Takahashi, re-energized the field by devising a technique to “reprogram” any adult cell, such as a skin cell, and coax it back to its earliest “pluripotent” stage. From there it can become any type of cell, from a heart muscle cell to a neuron.

The breakthrough sidestepped the embryo controversy, offering researchers an unlimited supply of stem cells. Dr. Yamanaka shared the 2012 Nobel Prize in Physiology or Medicine for reprogramming mature cells into what are now called induced pluripotent stem cells, or iPS cells. Still, the march toward new treatments has been halting.

Dr. Yamanaka directs Kyoto University’s Center for iPS Cell Research and Application. He also leads a small research lab at the Gladstone Institutes,affiliated with the University of California, San Francisco, where his group studies the molecular mechanisms that underlie pluripotency and the factors that induce reprogramming.

Source: https://www.nytimes.com/2017/01/16/science/shinya-yamanaka-stem-cells.html

Internet of Things in healthcare: Information technology in health

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The Internet of Things is about to transform the way we live and work. And if it reaches its fullest potential, it will fundamentally change every aspect of our lives.

That sort of disruption is evident in the healthcare sector, where the pen and paper has been the primary means of recording patient information for decades. But now, healthcare technology is changing in major ways.

Healthcare applications now let patients schedule their appointments without the need to call a doctor’s office and wait for a receptionist. Healthcare information technology lets doctors carry information with them anywhere they go through apps on their smartphones.

And this increasing connectivity shows no signs of slowing down. In fact, it’s only accelerating.

BI Intelligence, Business Insider’s premium research service, forecasts that the installed base of healthcare IoT devices (not includingwearable devices such as fitness trackers) will grow from approximately 95 million in 2015 to 646 million in 2020.

In short, more connection means more accessible data and better healthcare for patients. Below, we’ve laid out a roadmap of healthcare’s past, present, and future thanks to the IoT.

The Evolution of Healthcare Technology

Arguably the greatest technological leap forward in the last several decades has been the growth of electronic health records, or EHRs. In 2009, a mere 16% of U.S. hospitals were using an EHR, but that figure soared to approximately 80% in 2013, according to Becker’s Hospital Review. Previously, hospitals had multiple systems that handled different functions, but EHRs roll all of those into a single system.

Then we have portal technology, which lets patients take a more active role in their own health and well being. Portals let users log on to the healthcare provider’s websites to access their medical records, download forms, and prepare for appointments.

Finally, home monitoring systems allow patients and doctors to keep track of an individual’s health when not in the doctor’s office to prevent unnecessary and costly trips to sit down with a physician.

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How IoT Will Revolutionize the Healthcare Industry

The Internet of Things is slowly starting to weave into healthcare on both the doctor and patient fronts. Ultrasounds, thermometers, glucose monitors, electrocardiograms, and more are all starting to become connected and letting patients track their health. This is crucial for those situations that require follow-up appointments with doctors.

Multiple hospitals have started to utilize smart beds, which can sense the presence of a patient and automatically adjust itself to the correct angle and pressure to provide proper support without the need for a nurse to intervene.

The IoT could also help transform patient care at home. Sadly, some patients don’t take their medication in appropriate doses or at the correct times. Smart medication dispensers in the home could automatically upload information to the cloud and alert doctors when patients don’t take their medicine. More broadly, this type of technology could let doctors know of any potentially dangerous patient behavior.

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Healthcare IoT Companies Leading The Way

Several well-known companies are leading the pack when it comes to the IoT and healthcare. Grand View Research recently released a report on this sector and noted that a handful of key players dominate the industry. These companies are clamoring to gain a major slice of the pie by developing products for specific medical applications, increasing collaborative research and development, and acquiring new startups.

Microsoft, for example, has built its Microsoft Azure cloud platform to facilitate cloud-based delivery of multiple healthcare services. IBM, meanwhile, has announced it would invest approximately $3 billion in the IoT, and undoubtedly some of that money would go toward the healthcare sector.

But dozens of startups are breaking into this space, such as:

ConnectedHealth EarlySense Proteus Digital Health Qardio Whoop ThalmicLabs
Kinsa Neurovigil Athos Atlas Beddit Pristine
Sproutling Sensoria Lark Thync NeuroSky AliveCor
Awarepoint BioSerenity iRhythm BL Healthcare Sotera Wireless Voluntis

Source: http://www.businessinsider.com/internet-of-things-in-healthcare-2016-8