What does 2016 have in store in the realm of science and clinical innovation? Vector asked clinical, digital and business leaders from around Boston Children’s Hospital to offer their forecasts.
Our quantified brains
2016 will see more work on brain differences from individual to individual, and even from day to day in a single person. For example, Russ Poldrack at Stanford University, a well-known researcher in the field, scanned his own brain twice a week for one year and also looked at his gene expression, diet, mood, life events and other parameters. The data, published last month, are openly available so that more people can use existing tools and develop new ones to analyze data sets like this. There are several similar projects underway, along with a lively debate about the advantages, disadvantages and ethical implications of this kind of work. It will, for sure, advance our knowledge about the brain, its development and the effects of genes, mood, nutrition, medications, environmental factors and more over time. — Nadine Gaab, PhD, Developmental Medicine Research
Growth of the ‘internet of health care’
The convergence of consumers’ increasing awareness of their health and their ever-connected devices has given rise to a more portable, proactive form of health care. Families will increasingly look to perform front-line health care triage with diagnostic mobile applications and devices like Oto and iThermometer and decision support apps like Thermia. Home connectivity hubs like Amazon’s Echo, Microsoft’s Xbox/HoloLens and social robots like Pepper and Jibo will begin to be used for intuitive health care delivery. (At Boston Children’s Hospital, we are developing a health app for Amazon Echo, a wireless speaker that responds to voice commands and delivers information via a personal digital assistant, Alexa.)
Prescription of mobile apps and devices to patients to manage their care will become the norm in 2016. Improving upon traditional electronic health record (EHR) applications and patient portals, connected health care will reach consumers at home, play or work, allowing real-time tracking of their health information, better medication adherence and ready access to advice and care from any location. The personalized insights gathered from connected health care will improve our health by putting more focus on preventive care. Applications curated by health care providers will engage patients further and improve communications with care teams throughout patients’ medical journey. — Nitin Gujral, BS, Software Development Manager, Innovation and Digital Health Accelerator
Patient-enabled record sharing, through apps
The ability to tap one’s own medical data is crucial to being an engaged, empowered patient and to having better health outcomes. This year we will get closer to the vision of patients authorizing their own apps to connect to their electronic health record (EHR) on their behalf. With the upcoming Application Programming Interface (API) standard FHIR (pronounced “fire”), patients can enable any authorized third-party health app to connect to their EHRs and provide a value-added service. Through HIPAA, patients will have the right to access their health records and to be the center/mediator for sharing these records for medical and research purposes.
In October, the Centers for Medicare and Medicaid Services and the Office of the National Coordinator (ONC) released the final rules for the third stage of the Meaningful Use incentive program for EHRs and the corresponding certification requirements for EHR vendors. One requirement allows patients (or authorized representatives) to access their health records using third-party applications or devices with ONC-certified APIs. As this requirement rolls out in 2016, we will begin to see more interoperability between different EHR systems. This will translate into better health outcomes: for example, patients with chronic conditions will be able to share data from records scattered in multiple health systems and enjoy more coordinated care. — Gajen Sunthara, BSc, MS, director, Innovation R&D, Innovation and Digital Health Accelerator
Bringing meaning to user-generated Big Data
Through social media, search engine queries, wearable technologies and mobile devices, we generate an enormous amount of health-related data. Our digital phenotypes can provide deep insight into our personal health and, when scaled up, population health. While we’ve made some progress analyzing social media data for acute and chronic conditions, much work remains to find meaning in the large amount of data collected from wearable and mobile devices. To have a true impact, the data need to be engaging and actionable, not simply presented in a dashboard (as in a step count). As technology companies actively develop sophisticated machine learning algorithms that can link information and flag patterns, we should expect to see a bigger emphasis on making the most out of user-generated data in 2016. — Jared Hawkins, MMSc, PhD, director of informatics, Innovation and Digital Health Accelerator; faculty, Computational Health Informatics Program (CHIP)
The microbiome arrives
Next-generation sequencing has revolutionized our ability to characterize the microorganisms that live within us, as exemplified by the 2008 launch of the NIH-sponsored Human Microbiome Project and the 2015 formation of the Unified Microbiome Initiative Consortium. Researchers from a variety of fields are jumping on this bandwagon, because it’s headed somewhere fascinating, with implications for health and a huge cool factor. It’s as though we’ve discovered a new organ critical to life that was right under our noses (and in our gut, lungs, reproductive organs and everywhere else) all along. The microbiome gives us a new lens for examining disease associations, such as last month’s finding that skin-to-skin contact may lower the risk of sepsis and death in premature infants. (Could the skin microbiome be involved?) Medical treatments are emerging, like fecal transplants to engineer the gut microbiome in patients with recurring C-diff infections. In 2016, this wave of interest will continue to escalate as startups, established big pharma companies like Novartis and academic institutes vie for microbiome scientists. — Jane Amara, PhD, associate director, Technology and Innovation Development Office
Clinical trials in a dish
The initial excitement of stem cell medicine was predominantly based on the promise of regenerating healthy, stem-cell-derived tissues or cells. This year, I predict more use of stem cells as an aid to therapeutic discovery. Tissue samples from patients can be transformed into induced pluripotent stem cells (iPSCs) that can then be differentiated into specific cell types. For example, in amyotrophic lateral sclerosis (ALS), patient-specific motor neurons can be made and phenotyped for disease characteristics in parallel with phenotyping of the patient. The cells can then be tested for their response to different treatments, which may then predict the patient’s responses. This opens a whole new form of precision medicine, one with transformative diagnostic and therapeutic implications. The first ALS clinical trial where patients will have stem cell lines made for their disease and treatment response characterized is already underway. — Clifford Woolf, MB, BCh, PhD, Director, F.M. Kirby Neurobiology Center
Deep brain stimulation moves into pediatrics
The ability to normalize malfunctioning brain circuits through targeted electrical stimulation has advanced tremendously since chronic deep brain stimulation (DBS) systems emerged nearly 30 years ago. Surgeons can implant electrodes into precise locations deep within the brain, together with a neurostimulator (a device akin to a pacemaker), and alter the function of specific brain circuits by delivering reversible, adjustable electrical impulses. Most commonly used for movement disorders, such as Parkinson’s disease and dystonia, DBS therapy has grown exponentially in recent years both in the number of patients treated and in the range of diseases addressed.
DBS has largely been limited to adults, but 2016 will see greater application in pediatrics as advances emerge. Next-generation devices are coming, including implants with longer lifespans, greater adjustability to better “fine tune” stimulation and smarter “closed loop” electronic systems that both sense and respond to brain activity. Medical imaging and our understanding of brain development and function are continually improving, allowing clinicians to better target faulty brain circuits for existing and expanding disease indications. The prospect of altering the course of disease early on — enabling children to achieve greater benefits and reach their highest potential — will continue to drive expansion of DBS in the pediatric setting. — Scellig Stone, MD, PhD, director, Deep Brain Stimulation program
Noninvasive brain stimulation in epilepsy, autism
Discoveries will continue in the basic mechanisms of neurostimulation, particularly in applications related to epilepsy. The effects of electrical (and perhaps even magnetic) brain stimulation can be studied at the level of channels, cells and small circuits. Through this “bottom up” understanding, discoveries can be generalized to clinical application quickly, and this will lead to new clinical modalities in epilepsy. Similar advances in understanding the neurological disorders on the autism spectrum will eventually (not in 2016, but in the next decade or so) lead to a neuromodulatory treatment for autism that will dramatically improve clinical care. — Joseph Madsen, MD, director, Epilepsy Surgery
Genetics informing therapy
The Precision Medicine Initiative, initiated in 2015 by President Obama, has helped fuel efforts to use genetic and other individual-specific test results to guide medical decision making. Nowhere is precision medicine more likely to have a significant impact as in pediatrics. A number of studies have already demonstrated the value of using genetic approaches to identify disease etiology. These studies are only the tip of the iceberg: Many medical specialties are gaining genetic insight into patients’ disorders, in many cases leading us to identify new therapeutic avenues. In hematology, rare blood disorders like Diamond-Blackfan anemia are one example. In the coming year, it is likely that many more examples will emerge. — Vijay Sankaran, MD, PhD, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center