Genetic Cutting and Pasting

By Alyssa
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“In the future, CRISPR will be a common and useful tool to modify plants and agricultural animals and breed new varieties.”

-Caixia Gao, researcher at the Institute of Genetics and Developmental Biology at the Chinese Academy of Sciences


Is CRISPR the greatest medical advance since vaccines and antibiotics?  Many experts think so.  Since its discovery less than a decade ago, CRISPR – an acronym for “clustered regularly interspaced short palindromic repeats” –  has scientists imagining sweeping uses in all plants and animals…including humans.

So, what exactly is CRISPR?  Put simply, it’s a type of genetic engineering that allows scientists to target not only a specific gene, but one part of a specific gene.  It’s based on the revelation that many single-celled bacteria have immune systems that contain repeating bits of DNA.  Between these identical repetitions are short segments of “spacer DNA,” which match virus DNA from previous exposures, ensuring that the bacterium can recognize and ward off further attacks. Whenever a previously exposed bacterium encounters the virus, the sequence acts like tiny scissors to chop it up.

So, what does this mean?  Its uses can range from extending the shelf life of fruit to the human lifespan.  It could edit out hereditary diseases such as cystic fibrosis and hemophilia, or attack certain cancers. It could fight bacteria that is resistant to antibiotics. Or it could be used to create drought-resistant wheat or crops that can’t be bothered by pests.

So, what could be bad?  What’s unknown is if CRISPR techniques could create new problems because any changes in the genome will be passed down to future generations.  Could those tiny alterations cause profound but unknown mutations decades from now? Scientists and regulators alike will be watching closely.

To learn more about CRISPR – including interviews with scientists and videos that provide a basic overview of the technique and a deeper look into the associated controversies – check out the latest issue of Compass.



The Promise of Precision Medicine

By Catherine
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By Catherine Bolgar

Medicine is moving away from a one-size-fits-all model.

Precision medicine, sometimes called personalized medicine, holds so much promise that the U.S., China, and France have announced massive investments in this field over the past year.

“Precision medicine, we contend, has the potential to result in systemic savings,” says Christopher J. Wells, communications director at the Personalized Medicine Coalition (PMC), a Washington nonprofit organization representing scientists, patients, providers and insurers. “Now, medicine is by trial and error. You try one treatment and if doesn’t work, you try another.

The power of precision medicine is that if you get it right the first time, everybody benefits.”

IV solution in a patient hand and IVS machinePrecision medicine has made the biggest strides in oncology, where time is of the essence and chemotherapy drugs have strong side effects. For example, some breast cancers may be resistant to treatments such as trastuzumab. But that chemotherapy drug is very effective for breast cancers caused by the HER2 mutation.

No two cancers are the same,” says Susanne Haga, associate professor of medicine at Duke University in Durham, N.C. “They may be the same with respect to their tissue origin. But each person has a unique set of mutations that give rise to uncontrollable cell-division cycle, or cancer. With that in mind, each person would have some commonalities with other patients but also some unique qualities.”

However, the majority of patients still don’t receive personalized care, notes Mr. Wells. The PMC notes that a previous study has demonstrated chemotherapy use would drop 34% in women with breast cancer if they had a genetic test of their tumor before treatment.

With cystic fibrosis, “we thought it was one disease,” says Euan Ashley, associate professor of medicine at Stanford University in Stanford, California. “But as we dig deeper with genetic sequencing, we find it’s many diseases. If you can subcategorize patients, you can treat a disease much more effectively.”

Dr. Ashley’s specialty, cardiovascular disease, is the leading cause of death globally. “So we tended to do very large population studies, giving the same drug to everyone,” he says. “But if you study closely afterward, you see that a small number of people drive the effect—they get a significant benefit. Many get no benefit. And a few get significant harm.”

Precision medicine is prompting different ways of thinking about populations and individuals. “The answer has to be to measure people in as high a resolution as we can and work out who is responding and why,” he says.

With the cost of developing a prescription drug at about $2.6 billion, pharmaceutical companies have a big interest in seeing that the drugs they make get tested on the right segment of patients. A drug could appear to have no effect, when in fact it’s highly effective but only for a smaller number of patients.

Scientist woman“Drug companies now are building precision medicine into their research-and-development strategies,” says Mr. Wells. Personalized medicines accounted for 28% of novel new drugs approved by the U.S. Food and Drug Administration in 2015, and for 35% of novel new oncology drugs. Novel new drugs go beyond improved formulations or new dosages to deliver truly innovative advances.

Meanwhile, pharmacogenomics looks at how different people metabolize drugs. “There are a number of genes that are particularly active in the liver,” says Dr. Haga of Duke, adding that there are many variations of these genes among people. “We can test whether a patient metabolizes fast or slow and, if necessary, can prescribe a different drug that goes through different pathways so the affected genes aren’t involved.”

This test is valid for life, because one’s genes don’t change. Some institutions are trying to incorporate the information into electronic medical records, so all the different doctors and specialists one might see—as well as pharmacists—would be more knowledgeable, for prescribing drugs.

Because the cost of genetic sequencing has fallen dramatically, to about $1,000 today for a genome from $100 million in 2001, some are asking why everybody doesn’t get tested. It could speed up treatment for cancer patients or could allow for early intervention to arrest development of certain other diseases.

The U.S. National Institutes of Health devoted $25 million to four projects of genetic sequencing in newborns over five years with the goal of diagnosing conditions at the start of life. China just launched a project to do genetic tests on 100,000 newborns over the next five years, to improve treatment strategies and patients’ quality of life.

“The technology is going to continue to improve,” Mr. Wells says. “But already we’re at a point where the scientific advances are incredible.”



Catherine Bolgar is a former managing editor of The Wall Street Journal Europe, now working as a freelance writer and editor with WSJ. Custom Studios in EMEA. For more from Catherine Bolgar, along with other industry experts, join the Future Realities discussion on LinkedIn.

Photos courtesy of iStock

Power to the Patient

By Catherine
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By Catherine Bolgar

Senior Patient Having Consultation With Doctor In Office

The explosion of chronic diseases threatens to be a major health issue in coming years, especially with the baby boomers entering old age. Getting patients to participate in, and assume responsibility for their own care, is key to keeping health care costs in line. In 2050, the number of people older than 60 is expected to reach 2 billion.

Indeed, health budgets are under pressure in all countries, causing a rethink of how to structure the way it’s delivered and paid for. For example, both the U.S. and U.K. are moving toward value-based or outcome-based care, with incentives for providers to coordinate care and improve quality of care, rather than payment based on the number of procedures done.

“The key word is going to be patient engagement,” says Felipe Lobelo, associate professor of global health at Emory University in Atlanta. “That means not just taking care of someone who’s sick, but also preventing someone from getting sick in the first place. The health-care system is going to be more proactive in working with people to make healthy choices.”

Doctor and patientOne study found that having a voice in decision-making led patients to better adhere to treatment, with better outcomes. Another found that patients who used online systems to see test results, manage their medication list and exchange secure messages with their providers felt more in control of their own care and were more satisfied.

“The bigger challenge is what to do to keep patients well,” says Phil Koczan, chief clinical information officer at UCL Partners, a health-science partnership linking higher education and National Health Service (NHS) members in the U.K.

The difficulty is how to identify those patients, many of whom don’t see a doctor on a regular basis, and how to change their behavior.”

Five key behaviors are related to avoiding chronic disease—never smoking, regular physical activity, no or moderate alcohol consumption, normal weight and enough sleep. In a recent study, only 6% of Americans do all five.

While physicians try to offer advice and support to people with risky behaviors, “it’s quite difficult and time-consuming, and resources are limited to offer that sort of support,” Dr. Koczan says. “But there’s a lot of benefit if we can get it right.”

Wearable devices and mobile applications can help—if patients stick with them. A device “doesn’t say how to change eating habits or how to change exercise habits. It’s not personalized enough,” says Vibhanshu Abhishek, assistant professor of information systems at Carnegie Mellon University in Pittsburgh. “Devices need to get more personalized and give specific recommendations based on current behavior. Just walking 10,000 steps isn’t enough. It has to give goals and specific instructions to individuals—if I ate a big lunch, then here’s how much more I need to work out today.”

Devices are most useful “when the intervention is tailored to the patient,” agrees Dr. Lobelo. For each patient, “it needs to be tweaked. It’s a never-ending series of projects and applications, not one universal solution.”

Another aspect of prevention involves keeping patients with diseases or chronic conditions from becoming sicker. Most patients with chronic conditions are at home, not hospitalized, so no doctor or health professional regularly observes whether they follow the recommendations they’ve been given, Dr. Abhishek says. “Mobile apps provide an opportunity to collect this information in a cost-effective manner on a continuous basis. Using algorithms or health-identification tools, a doctor can figure out whether a treatment is working. It hasn’t been possible to do this in a generalized way because data collection has been so expensive. In the future we can say treatment A works for this type of patient, and treatment B works for this other type of patient, based on the data from mobile devices.”

Measuring the pulseA number of online platforms offer information and support for self-care by patients with different diseases. The University of Pittsburgh developed iMHere, a mobile health platform to empower chronic-disease patients for self-care under a clinician’s guidance. For example, iMHere aims to help spina bifida patients avoid secondary complications, such as skin problems and urinary tract infections, through remote monitoring, with clinicians sending patients customized treatment plans. Other programs aim to help cancer patients manage their care, such as managing the accumulation of lymph fluid after breast cancer treatment.

Health systems are going to be more proactive in working with people to make healthy choices,” Dr. Lobelo says. “Including patients—that’s the center of the whole thing. We want to encourage people to self-measure and use the data to improve their health. An active dialog needs to happen.”


Catherine Bolgar is a former managing editor of The Wall Street Journal Europe, now working as a freelance writer and editor with WSJ. Custom Studios in EMEA. For more from Catherine Bolgar, along with other industry experts, join the Future Realities discussion on LinkedIn.

Photos courtesy of iStock

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