Biotech innovations to spur next phase of personalized care

It’s time for hospitals to brace for the next phase of precision medicine. At least, that’s what leading investors, researchers and other experts suggest when asked what the next two decades of biotechnology innovation will bring to medicine.

Innovations in biotech—generally speaking, practices that exploit biological processes, like DNA sequencing and targeted therapies—have begun a significant shift in how medicine is practiced.

“I see a real opportunity to manipulate genes in both individuals who are affected with disease but also, in the future, potentially to prevent disease,” said Jennifer Doudna, a professor at the University of California at Berkeley, who many experts credit with developing the CRISPR gene-editing technique. “We’re not there right now, but I think that’s a direction the field is headed, and certainly in two decades I think we could be at that point.”

The targeting of biotech has been driven by continued interest from agencies like HHS’ National Institutes of Health, as well as investors who have poured millions into the space.

In February, HHS launched what it called the Foundry for American Biotechnology to spur development of innovations that would help the U.S. respond to health security threats and disasters.

The high levels of investment and interest could, in the long term, totally change how clinicians and patients interact over coming decades, with much of the innovation centered on more personalized care.

More frequent DNA analyses and always-on monitoring—maybe with implantable sensors—for example could drastically change how patient care is managed.

Amir Nashat, a managing partner at investment firm Polaris Partners who focuses on healthcare, said he envisions medicine taking a page from the automotive industry. Because of new technologies, mechanics are more frequently tasked with maintenance, rather than repairs, while sensors and warnings displayed on car dashboards help owners—in theory—proactively monitor their vehicles.

Similarly, with new inventions in healthcare, people will see “early warning signals of what’s going on that’s a departure from a healthy state,” he said.

That said, it’s difficult to predict the future—to say the least. It’s hard enough to speculate on the next two years, let alone multiple decades, Doudna said of her predictions for what biotechnology will accomplish in the coming years, adding, “I can only speculate on things that I see coming down the pike.”

But if some of these innovations are successful, it could fundamentally change how hospitals operate.

Hospitals have traditionally centered their businesses around inpatient services, but as patient care moves into new environments like the home, it changes the services they will be providing, said James Flynn, a managing partner with hedge fund Deerfield Management Co. He added that innovations like gene therapy also open the opportunity for patients to, one day, perhaps get treatment for now costly and incurable diseases.

That will require hospitals, in some ways, to “reinvent themselves over time,” Flynn said, “how they’re organized and how they function.”

DNA editing, repeated molecular analyses and continuous monitoring of different vital signs are a few areas that experts suggest hospitals watch.

In 2003, fewer than 20 years ago, scientists unveiled the completed Human Genome Project—a landmark research initiative that mapped all genes of the human body. Now, DNA sequencing is faster and cheaper, enough so that health systems like Geisinger Health and NorthShore University HealthSystem have even begun to integrate it into routine patient care.

At NorthShore, it’s part of an effort to make personalized medicine a practice that’s “no longer something specifically designated, but it just becomes the practice of medicine,” said Dr. Peter Hulick, medical director of the system’s Neaman Center for Personalized Medicine.

Other health systems will likely move in that same direction. Genetic testing is in the midst of expanding from a previously limited role in clinical medicine to affecting virtually all areas of medicine, said Dr. Matthew Taylor, director of the adult medical genetics program at UCHealth and the Anschutz Medical Campus. He anticipates that within a few years a large portion of patients will have that testing done to manage diagnoses and medications.

“In the next five to 10 years the cost of doing a genome sequence will come close to negligible, so the access will be broadly expanded,” Taylor suggested. Even “20 years might be a little bit too long of a lens, because the field is moving so quickly.”

And all that genetic data—if used in concert with researchers—could play a role in fueling scientific discoveries for the next phase of genomic medicine. “Biology is just a big puzzle,” Flynn said. As today’s research progresses, he said he envisions more gene therapies in the next 20 years, starting with those that treat single-gene disorders and progressing to more complex diseases. “We’re in a period where there’s very rapid puzzle-filling-in for all kinds of diseases.”

Gene therapy is looking particularly promising as the first few U.S. clinical trials to test CRISPR in human patients get underway.

That could be particularly effective for genetic disorders like sickle cell disease and hemophilia, many of which don’t have treatments today, said Dr. Eric Topol, director of the Scripps Research Translational Institute, which researches how to improve patient health by combining data like genomics and digital sensors. “They all should be imminently treatable, if not curable, by a decade from now,” he predicts.

Modifying genes to improve health holds significant potential, but there are many ethical concerns left to be pondered and regulated before it becomes widespread. Late last year scientist He Jiankui was sentenced to three years in jail in China after secretly using CRISPR to edit the genes of human embryos of twin girls. His research was largely condemned as unethical, given limited research to date on using CRISPR for clinical use.

Doudna stressed that there’s more research needed in modifying genes in embryos for clinical use, because “we don’t understand what the impacts of that kind of application would be, both scientifically, but also societally.”

Doudna has been a vocal proponent of the potential for modifying genes in patients’ somatic cells, or the cells that won’t be passed on to future generations, to improve health—but not in embryos, which could create heritable changes.

While exciting today, whole-genome sequencing is just the first step to precision medicine, experts predict.

DNA sequencing of a patient’s genome would likely just be done once, to map a patient’s genetics from birth. But there are other opportunities for repeated molecular analysis, said Dr. Lincoln Nadauld, chief of precision health at Intermountain Healthcare, which recently ranked eighth on Fast Company’s 2020 list of most innovative biotechnology companies.

Nadauld envisions a world where a patient’s whole genome is mapped to predict risk of various cancers and cardiovascular and neurodegenerative diseases. Then, regular analysis of tests based on other aspects of human biology—such as proteomics or proteins—“to look for the onset of disease, as predicted by their genomes.”

A patient’s microbiome, the genetic material of the bacteria, fungi, viruses and other microbiota that live within the human body, could offer another way to monitor changes to patient health.

There’s been a “first wave” of research focused on microbiome-based therapies so far, said Racquel Bracken, a partner at venture-capital firm Venrock focused on biotechnology and pharmaceuticals, which she expects to advance quickly in the next few years. She pointed to research being done at Stanford University to study the microbiome, with an ultimate goal of developing therapies that manipulate microbial communities to treat different diseases. That type of research, as well as discoveries related to how the microbiome affects conditions like autoimmune disorders, cancers and liver diseases, will pave the way for companies to move forward with actually developing products and kicking off human trials.

“Microbiome ‘bugs as drugs’ therapies are going to be much more mainstream— call it in 10 years time,” Bracken said. “There’s so much promise there.”

One of the first therapies to leverage the gut microbiome is fecal microbiota transplantation, or stool transplants, to treat Clostridium difficile infections.

While others agree we’ll see firmed up research on the microbiome in the next decade, we might still be far off from being able to use it. It’s too early to tell what will be successful and when, said Sanjay Shukla, director of the Marshfield Clinic Research Institute’s center for precision medicine research.

“Once we have enough baseline data of the microbiome—of healthy people and different disease cohorts—it’s believed … (the microbiome) could be modified to help a patient, but it’s really far out,” he said. It’s “still in an early phase of maturity in the clinical setting.”

Another component of more regular monitoring is using sensors for continuous, remote evaluation of blood pressure, breathing, body temperature and other signs, which—ideally—could allow providers to intervene when health starts to deteriorate, as opposed to after a patient starts reporting symptoms.

It’s not a new idea. But Dr. Steve Xu, medical director at Northwestern University’s Center for Bio-Integrated Electronics, said he envisions a world where patients could one day have implanted sensors that not only monitor vital signs but also provide automated health insights—transforming healthcare into a system where patients are constantly provided feedback on their health status.

He said he could see that proliferating in the next 20 years, and doesn’t think an implantable sensor would be an insurmountable privacy concern for patients. Implantable devices are already being used in healthcare, including birth-control implants and nerve stimulators. However, it would be on companies to provide evidence that these sensors are actually helpful for patient health and transparency into how the data is being used.

Xu’s research at Northwestern involves working on wearable sensors for pediatric care, such as to better monitor newborns who are born prematurely. Already, one-third of consumers report owning a wearable device to help track their health, according to a 2019 report from the Stanford Medicine Center for Digital Health and early-stage digital health venture fund Rock Health. While most of those devices track more general exercise, sleep and heart rate, and aren’t used for medical care, they could point to patient interest and comfort with monitoring health data.

And health systems have been looking at the space more closely too. To lay the groundwork for better remote monitoring of patients, such as after discharge, UCHealth in Colorado partnered with startup BioIntelliSense to help develop its health-monitoring patch, as well as to support the company as it sought regulatory clearance. About the size of a Band-Aid, the patch continuously tracks metrics like heart rate, skin temperature and respiratory rate and sends the data back to a provider.

BioIntelliSense earned U.S. Food and Drug Administration clearance for the device earlier this year.

Xu said he expects to see a “tipping point” within the next decade when almost everyone will collect data with wearable devices, sensors or patches, which can be linked with medical records.

He points to how in the 1980s, it was difficult to imagine everyone would have a cellphone. Flip phones in the mid-2000s provided a shift in perspective—“Motorola came out with the Razr flip phone, and that was a turning point, where things were really cool”—but it wasn’t until the BlackBerry and Apple’s iPhone that adoption really took off.

While wearables available today have shown promise for fitness tracking and some limited medical functions like conducting electrocardiograms, “there’s still opportunities for what the future is,” Xu said, which developers will continue to build on. “I think these wearables are probably at the flip-phone stage,” he added.