Pacesetters in Discovery and Care

Thousands of basic, translational and clinical studies are undertaken each year by Harvard Medical School’s world-class affiliates and research institutions, which lead the world in biomedical discovery. Just a few of the remarkable discoveries made in 2015-2016 include the following:

Beth Israel Deaconess Medical Center

Evolutionary relic of the genome causes cancer

Investigators at Beth Israel Deaconess Medical Center found evidence that a pseudogene—a subclass of noncoding RNA that developed from the genome’s 20,000 protein-coding genes but has lost the ability to produce proteins—has a role in causing cancer. Published in Cell, the findings indicate that this “evolutionary relic” led to an aggressive lymphoma-like illness in a mouse model. The existence of pseudogenes means the functional genome may be much larger than previously thought, perhaps triple or quadruple its currently known size.

Boston Children’s Hospital

Protein-based scaffolding to help repair torn ACL

Orthopedic surgeons at Boston Children’s Hospital combined basic and clinical research to develop Bridge-Enhanced ACL Repair (BEAR), a procedure to repair torn anterior cruciate ligaments (ACLs). They received approval in 2014 to begin the first safety trials in humans, which compares control surgeries, or typical repairs, with experimental surgeries that use a bridge of protein-based scaffolding to help the ACL repair itself, avoiding the need to take tendon grafts from elsewhere in the body. The new method could accelerate healing, which would allow patients to return to normal activity sooner. Read about BEAR research

Brigham and Women’s Hospital

New gene implicated in multiple sclerosis

In a multiple sclerosis (MS) study, researchers at Brigham and Women’s Hospital discovered a genetic variant associated with a patient’s likelihood of responding to interferon-beta, one of the medications used to treat MS. Published in the Annals of Neurology, the BWH study presented evidence that the affected gene, SLC9A9, may play a broader role in regulating the development and activity of immune cells involved in inflammatory diseases such as MS. The findings are key to early identification of the most effective drug for a given individual, which is critical for long-term outcomes.

Cambridge Health Alliance

Top performer in reducing hospital readmission rates

Cambridge Health Alliance (CHA), in collaboration with several community partners, is among top performers in a national pilot program aimed at reducing hospital readmission rates. It succeeded in lowering 30-day readmission rates in high-risk patients by nearly 9 percent over a two-year period. Of 104 original U.S. community-based programs, CHA is one of just 16 surviving programs participating in the Centers for Medicare and Medicaid Services Community-based Care Transitions Project, which aims to improve transitions from the hospital to the home in order to enhance quality of care, reduce readmissions for high-risk patients and show measurable savings to Medicare. This is accomplished, in part, by giving patients appropriate support and services after their hospital stays.

Dana-Farber Cancer Institute

New immunotherapy vaccines show promise in treating brain tumors

At Dana-Farber Cancer Institute, early results of a clinical immunotherapy trial showed that a cancer vaccine called rindopepimut, combined with the antiangiogenic drug Avastin, improved survival in certain patients with relapsed glioblastomas. The patients’ tumor cells carried a genetic mutation known as EGFRvIII, found in about one-third of glioblastoma tumors. The rindopepimut vaccine contains EGFRvIII peptide molecules, which triggered an immune system response against the tumors carrying that mutation. Read more about the new rindopepimut vaccine.

Harvard Pilgrim Health Care Institute

Awarded $150 million FDA sentinel contract

The U.S. Food and Drug Administration (FDA) awarded a $150 million contract to Harvard Pilgrim Health Care Institute to lead the Sentinel System, a program using electronic health care data to monitor the safety of FDA-regulated drugs and other medical products. The Institute will partner with more than 50 health care and academic organizations on this undertaking. The institute previously led the development of Mini-Sentinel, a five-year pilot project that built an innovative data network and analytic tools. The transition to the full-fledged Sentinel System will expand resources to track and understand more about drugs, biologics and medical devices used by patients nationwide.

Hebrew SeniorLife

Improving balance and gait in the elderly

Research conducted by scientists at the Institute for Aging Research (IFAR) at Hebrew SeniorLife, which celebrated its 50th anniversary in 2015, has been instrumental in understanding the mechanisms and treatments of common syndromes associated with aging, including delirium, dementia, falls, fractures, and other cognitive and mobility problems. IFAR leaders believe that genetic research and collaboration between clinical and bioengineering researchers represent great opportunities for future investigation. On the bioengineering front, findings published in the Archives of Physical Medicine and Rehabilitation showed that a shoe insole that applies imperceptible vibratory sensations to the soles of the feet of healthy elderly people improved balance and gait. In the study, IFAR researchers collaborated with colleagues at HMS and the Wyss Institute for Biologically Inspired Engineering. Read about a shoe insole delivering subsensory vibratory noise improves balance and gait in healthy elderly people

Joslin Diabetes Center

Roles of gut bacteria in diabetes and obesity

The trillions of bacteria in the human digestive system play a major role in metabolism. They are linked to a person’s risks of Type 2 diabetes, obesity and related conditions that make up metabolic syndrome. Humans and animals with diabetes and obesity have different gut bacteria than those who don’t, and when scientists transfer microbiota from obese humans or animals to germ-free animals, the recipients are more likely to become obese or diabetic. As reported in Cell Metabolism, researchers at Joslin Diabetes Center found that one strain of mice genetically prone to become obese became resistant to excess weight gain after their populations of gut microbiota were transformed simply by sharing an environment with other mice.

Judge Baker Children’s Center

Improving children’s mental health care

Judge Baker Children’s Center is in the forefront of translational research through its expertise in implementation science. Currently, Judge Baker staff are working on multiple initiatives with the Commonwealth of Massachusetts and the provider community to improve the quality of mental health care for children. An example is their ongoing collaboration with the Executive Office of Health and Human Services to improve the quality of in-home treatment through the statewide Children’s Behavioral Health Initiative. Experts at Judge Baker also are working with provider organizations to implement the Modular Approach to Therapy for Children with Anxiety, Depression, Trauma or Conduct Problems (MATCH). Read about the Quality Care Initiative

Massachusetts Eye and Ear | Schepens Eye Research Institute

Resurrecting ancient viruses to improve gene therapy

Researchers at Massachusetts Eye and Ear | Schepens Eye Research Institute have computationally reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle and retina. This discovery, published in Cell Reports, could potentially be used to design gene therapies that are safer and more potent than therapies that are currently available. In the lab, the scientists recreated synthetic ancestors of adeno-associated viruses, which naturally circulate in human populations. If patients have been previously exposed to a virus, their bodies will likely recognize and destroy it before it can deliver the therapy. Engineering new, benign viruses could render the viruses unrecognizable and increase the number of people for whom gene therapy will work.

Massachusetts General Hospital

Transplantable bioengineered forelimb developed in an animal model

Massachusetts General Hospital investigators took the first steps toward developing bioartificial replacement forelimbs suitable for transplantation. Adapting an experimental approach previously used in animal models to build bioartificial organs, they worked to engineer rodent forelimbs with functioning vascular and muscle tissue. More than 1.5 million people in the U.S. have lost limbs, and although prosthetic technology has greatly advanced, the ability to regrow limbs from a patient’s own tissues would be revolutionary.

McLean Hospital

Anti-malarial drugs a potential treatment for Parkinson’s

The findings of a multi-institutional research project, led by McLean Hospital, has shown that existing anti-malaria drugs are a potential treatment for Parkinson's disease. Available treatments can help relieve symptoms, but there are no treatments that can slow or stop the progress of the disease. After screening more than 1,000 FDA-approved drugs, the researchers discovered that two common anti-malaria treatments, chloroquine and amodiaquine, could activate a protein in the brain that is vital to fighting Parkinson's. This protein is essential for the development and maintenance of dopamine neurons, which are needed to move muscles and maintain motor control. Read about the potential Parkinson's treatment

Mount Auburn Hospital

New lung nodule and lung cancer program

Aiming to identify potentially cancerous lung lesions early on, Mount Auburn Hospital has launched a multidisciplinary screening program designed to identify, diagnose and treat patients with lung nodules, or small tissue masses. The program principally monitors people over age 55 who currently smoke or quit smoking within the past 15 years and have a long history of smoking for suspicious lung nodules found on any CT scan or x-ray of the chest. Led by specialists in pulmonary medicine, and supported by specialists in eight disciplines, the lung nodule multidisciplinary care team has developed an urgent access pathway to rapidly evaluate and treat suspicious lesions detected in chest x-rays and scans. Read about the Lung Cancer & Lung Nodule Care Program

Spaulding Rehabilitation Hospital

ADHD drug also may help patients with PTSD

A drug used to treat attention deficit hyperactivity disorder may be a better treatment for post-traumatic stress disorder than current therapies, according to a study published Neuropsychopharmacology, which involved scientists at Spaulding Rehabilitation Hospital. In 32 patients diagnosed with PTSD, researchers found that treatment with methylphenidate, used to treat ADHD, was associated with significant declines in PTSD symptoms and post-concussion symptoms as well as improvements in tests of attention and the ability to process information quickly. Trials with larger numbers are the next step to further validate these findings. Read "Better Treatment"

VA Boston Healthcare System

Brains of veterans near bomb blasts appear to age faster

U.S. veterans who were near bomb blasts appear to experience faster brain aging, according to a study in Brain: A Journal of Neurology. The study, which included scientists from the Translational Research Center for Traumatic Brain Injuries and Stress Disorders at VA Boston Healthcare System, looked at exposure to blasts in which veterans thought they were just fine, not necessarily blasts that led to concussion, a dizzy feeling or getting knocked out. Veterans who were exposed to bomb blasts, even up to a football field away, showed "leakiness" and fraying of white matter in the brain. The damage was similar to that associated with the normal aging process, only at a much faster rate. Read a CBS News report