DESIGNER IMMUNOTHERAPY
The Institute for Protein Design team designed a new method to create an artificial interleukin-2 that acts more powerfully than the natural protein to stimulate an immune response against cancer, but lacks a receptor — a cellular doorway — that's associated with IL-2's often-deadly side effects. A company has been launched to develop this into a product to compete with other modified versions of IL-2.
VELCRO CANCER THERAPY
Cytokines such as interkeukins are immune system signaling proteins that have anti-tumor properties but are also highly toxic to healthy cells. Injecting cytokines into solid tumors has failed because they quickly leak into the body's circulation. Koch Institute researchers fused cytokines to a protein that binds to collagen, a molecule tumors have in abundance. This sharply reduced the leakage out of tumors and in mice, the fused cytokines enhanced the effectiveness of other cancer immunotherapies.
SIMPLER MEMORY TEST
Some people balk at taking written and verbal tests used to diagnose Alzheimer's and mild cognitive impairment (MCI). Emory researchers, building on studies linking eye movement with memory impairment, devised a method to precisely measure eye movements of people taking a simple test — they were presented an image, and then later with something taken out. Most people noticed the missing item but those with dementia or MCI had a different eye pattern when looking at it. The team is developing an iPad version of the test.
PREDICTING MEMORY IMPAIRMENT
A kind of brain wave that occurs when the hippocampus replays certain memories could be used to identify people at risk of Alzheimer's disease, Gladstone Institutes researchers suggest. Changes in "sharp-wave ripples" at a young age in a mouse model of Alzheimer's predicted memory impairment 10 months later — the equivalent of 30 human years. Recent advances have made it possible to detect these brain waves noninvasively, and the authors suggest they could be useful in research and drug development.
LEAKY BRAINS
Research by UC Berkeley suggests the mental decline that accompanies aging may be caused by breakdown of the blood brain barrier. Building on earlier work that showed albumin in blood triggers inflammation that kills brain cells, the team found increased albumin and inflammation in autopsied brains, and using MRI, they showed greater leakage in the blood brain barrier of older people with cognitive dysfunction. Young mice showed similar inflammation and cognitive decline after their brains were injected with albumin, but senile mice given an albumin-blocking drug had fewer signs of inflammation and were better able to learn new tasks.
CELL THERAPY FOR BRAIN INJURY
In mice with a traumatic brain injury, transplanted embryonic progenitor cells, capable of generating a type of inhibitory neuron that tamps down excessive brain activity, migrated into the hippocampus, the brain region responsible for learning and memory, forming new connections and surviving long term. Within a month, the mice showed signs of improved memory, performing as well on tests as uninjured mice, UC Irvine researchers reported.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
CAR-T THERAPY FOR HEART DISEASE
In most forms of heart disease, fibroblasts cause the heart muscle to stiffen, contributing to heart failure. Penn Medicine researchers engineered CAR-T cells, a form of immunotherapy that has successfully treated some cancers, to target a surface protein they found was expressed at much higher levels in fibroblasts from diseased hearts. After this CAR-T therapy, mice with heart disease showed reduced levels of fibroblasts compared with controls, and they partially regained heart function.
PHAGE THERAPY FOR LIVER DISEASE
As many as 75% of patients die within 90 days of a diagnosis of severe alcoholic hepatitis, and a liver transplant is the only cure. UC San Diego researchers found that cytolysin — a toxin secreted by Enterococcus faecalis — kills liver cells, and people with alcoholic liver disease have more of these bacteria in their gut than healthy people. Mice colonized with bacteria from patients with alcoholic hepatitis developed the disease, but treatment with bacteriophages that target E. faecalis eliminated the bacteria and the disease.
BYPASSING LIVER BIOPSY
Up to 30% of the population has non-alcoholic fatty liver disease, which can progress to NASH, fibrosis, and cirrhosis. A liver biopsy is now the standard way these conditions are diagnosed, but Beth Israel Deaconess researchers developed a non-invasive alternative: They used machine learning to analyze blood levels of lipids, glycans, and hormones and devise algorithms that can diagnose these liver conditions. Further testing must be done to validate their accuracy.
ARE 10,000 STEPS NECESSARY?
While 10,000 daily steps is a popular goal, there's little research on just how many steps are needed to improve health. This Brigham and Women's Hospital study of older women found that those who took just 4,400 steps per day had markedly lower mortality rates after four years than the least active women, who took 2,700 steps per day. More steps were associated with even lower mortality rates up to 7,500 steps per day, though as an observational study, it couldn't prove causation.
"DUAL BURDEN" BIRTHS
A quarter of women who have serious maternal complications during childbirth also have premature births, posing a “dual burden” on families. The NYU College of Nursing study was the first to focus specifically on these doubly difficult births, finding they accounted for 1 in 270 births in California over a six-year period — and were twice as likely to affect Black mothers.
GENE SILENCING FOR OSTEOPOROSIS
Researchers showed in mice that an RNAi-based gene therapy that targets a gene called SHN3, which has been associated with bone loss in osteoporosis, could be delivered to bone using an AAV9 viral vector. The therapy reversed bone loss and enhanced bone mechanical properties. Such vectors target many tissues and can cross the blood-brain barrier, and heart side effects have been seen with other SHN3-targeted therapies, so the UMass Medical team modified the protein coat of the viral vector and showed it preferentially targeted bone.
BUBBLE BOY DISEASE
Using a novel gene therapy, a St. Jude Children's Research Hospital team appears to have cured eight babies born with X-linked severe combined immunodeficiency, a disease that leaves infants without a functional immune system and is usually fatal by age 2 unless they qualify for a bone marrow transplant. After an average followup of 16 months, the treatment restored infants' immune function and wiped out existing infections.
BREAST CANCER THERAPY
Adding a targeted drug called ribociclib to standard hormone therapy for younger women with an aggressive form of breast cancer lowered their risk of death. After 3.5 years, 70% of premenopausal women with metastatic cancer who got the combination were alive, compared to 46% of those on hormone therapy alone. This long-running trial led by MD Anderson Cancer Center already changed the standard of care, and the FDA approved ribociclib for this population in 2018. The new results demonstrated the durability of the treatment's benefits.
ARTIFICIAL PANCREAS
An artificial pancreas system — which automatically monitors and regulates blood sugar levels — was more effective than existing treatments at controlling blood sugar levels in people with type 1 diabetes. The artificial pancreas uses a continuous glucose monitor and automatically delivers insulin. Based on the results of this randomized trial led by University of Virginia researchers, the FDA approved this system in December 2019.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
THE SUGAR PAPERS
A University of California, San Francisco researcher went through old files to show how the sugar industry undertook a campaign to downplay sweeteners’ harmful effects — using similar tactics as Big Tobacco to influence scientific research for years. Big Sugar's efforts changed government policies and led to a decades-long emphasis on lowering fat to reduce obesity, diabetes, and heart disease.
SWEETENED DRINKS AND CANCER
Consumption of drinks sweetened with high fructose corn syrup has been associated with colon cancer risk, and their growing use has coincided with a rise of colorectal cancer in young and middle-aged adults. Weill Cornell researchers showed that ingestion of HFCS, even in moderate amounts, promotes the growth of colon cancer in mice. An enzyme in tumors converts fructose to a metabolite that promotes cell growth. Whether a similar process occurs in humans remains to be seen, but the authors urge people with colon cancer or at high risk to avoid sugary drinks.
DRUGGABLE DIABETES TARGET
University of Utah and Merck researchers made genetically engineered mice lacking an enzyme called dihydroceramide desaturase 1 (DES1), which prevented insulin resistance and fatty liver disease in animals fed fat-laden diets and reversed prediabetes in obese mice. The findings suggest this enzyme may be a possible target for human treatments for obesity, diabetes, and related conditions.
DISRUPTED BODY CLOCKS
High-fat diets disrupt the body's circadian clocks, which regulate the timing of cellular functions, in turn raising inflammation and the risk of disease. A Texas A&M team, working with cells in the lab, figured out which message-carrying chemicals called cytokines are involved in this disruption, suggesting it might be possible to use anti-inflammatory drugs to treat people with metabolic disorders due to a high-fat diet.
ENGINEERED IMMUNITY
Nearly every vaccine works by coaxing B cells — a key component of the immune system — to make antibodies, but sometimes vaccines provide only partial protection, or none at all. Fred Hutchinson cancer center researchers demonstrated an alternative approach: Using CRISPR genome-editing, they inserted DNA blueprints for specific antibodies into B cells from mice and humans and showed they could produce antibodies against a range of viruses. B cells engineered to produce antibodies against respiratory syncytial virus provided potent, long-lasting protection in mice.
PRECISION ANTIBIOTICS
Broad Institute and Massachusetts General Hospital researchers have developed a diagnostic test that identifies the right antibiotic within hours rather than days. The test, called GoPhAST-R, could one day help patients get the most effective treatment faster, while slowing the spread of antibiotic-resistant superbugs. It looks for genetic sequences in bacterial RNA that are associated with antibiotic resistance.
PATIENT AS RESEARCHER
A UPenn doctor resesarched his own rare disorder, called Castleman disease, and after molecular analysis to decipher the underlying biology, identified an existing immunosuppressive drug called sirolimus, which is used to prevent rejection of kidney transplants, that might work on the disease pathway. He'd previously suffered five relapses that nearly killed him, but has been in remission for more than five years since starting on the drug. Two other patients given the drug have been in remission for 19 months.
REPURPOSED DRUG
University of Iowa researchers and colleagues identified a drug widely prescribed for an enlarged prostate as a possible therapy for Parkinson's disease, for which there is no progression-slowing treatment. Impaired energy metabolism is a common feature of Parkinson's, and when the team tested terazosin in rodents, fruit flies, and lab-grown human neurons, they found it enhanced energy production, slowed neuron death, and partially restored motor function. A search of patient databases revealed decreased disease progression in Parkinson's patients who happened to be taking the drug for an enlarged prostate.
THE SOCIAL BRAIN
UCLA researchers found that socially interacting mice exhibit striking correlations of brain activity during ongoing social interaction. This interbrain synchronization emerges from two sets of neurons that separately encode one’s own social behaviors and those of the interacting partner. Synchronization during the initial phase of the social encounter predicts future social interactions as well as the dominance relationship the animals develop over time. These findings may help study disorders that affect social functioning, including depression and autism.
BRAIN DOGMA CHALLENGED
Cellular activity was restored in pigs' brains four hours after death using a special pump to infuse the brain with souped-up artificial blood. Yale scientists observed the return of many basic cellular and metabolic functions previously thought to irreversibly cease seconds or minutes after oxygen and blood flow stop. There saw no organized, cortex-wide electrical activity associated with consciousness, pain, or other higher-order functions, but the method might be used to preserve whole brains for research.
SYNTHETIC HEART ARTERIES
Lab-made arterial grafts for heart surgery have so far not worked well, and they can take two months to fabricate. Johns Hopkins researchers engineered a small-diameter vascular graft that could be fabricated in less than a week and, in a six-month mouse study, withstood the high pressure arterial environment, degraded as new tissue grew, and maintained mechanical properties similar to healthy vessels. The authors envision its use to treat children with congenital heart defects and adults with coronary artery disease.
3D-PRINTED ORGANS
Wyss Institute researchers developed a new technique to bioprint blood-vessel-like channels into mini-organs grown from induced pluripotent stem cells. They created batches of organ-specific tissues with high levels of oxygenation and function that survived for multiple days, and they hope to refine the technique to develop fully 3D-printed organs for transplant.
CHEMO-RESISTANT TUMORS
University of Michigan researchers demonstrated how pancreatic tumors neutralize the effects of gemcitabine chemotherapy, by attracting and manipulating macrophages. The mechanism may underlie resistance in other tumors, as well, and might help guide the choice of treatments for patients based on the levels of these susceptible macrophages in tumors.
UNBOUNDED DNA
Genetic mutations that give cancer resistance to chemotherapy are frequently found on circular DNA outside the chromosomes, called extrachromosomal DNA. Working on cancer cell lines in lab dishes, Boundless Bio and UC San Diego researchers found the physical location of these mutant genes helped tumors evade chemo and immune therapy. Boundless is trying to come up with ways to target this free-floating DNA.
BLOCKING CANCER STEM CELLS
Researchers from Stony Brook University discovered an enzyme driving cancer stem cell growth that underlies tumor recurrence, drug resistance, and progression in triple negative breast cancer, suggesting it could be a new drug target. They showed how the enzyme regulates cancer stem cells and that inhibiting the enzyme suppressed tumor growth and improved sensitivity to chemotherapy in mice. Enzyme activity was also increased in breast tumors from patients with lymph node metastasis.
FLAWED CANCER TARGET
Studies with the first-in-class inhibitors of MTH1, an enzyme that protects tumor cells from oxidative DNA damage, suggested MTH1 was a potent target for treatment of a variety of cancers. Interest in MTH1 as a therapeutic target has skyrocketed, but it’s become controversial because MTH1 inhibitors don’t always work in lab tests. A University of Miami team showed that's because cancer cells have redundant mechanisms that bypass the effects of these experimental drugs, meaning more research is needed to turn them into effective cancer therapies.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
MAGNETIC STIMULATION FOR SCHIZOPHRENIA
There are no effective treatments for the "negative symptoms" of schizophrenia, including inability to feel pleasure and lack of motivation. Using MRI, Beth Israel Deaconess researchers showed that a breakdown in a network connecting the frontal cortex to the cerebellum correlated with severity of negative symptoms in 44 patients. In a separate group of 11 patients, twice-daily treatment with transcranial magnetic stimulation for five days restored the network and reduced negative symptoms, establishing a causal connection and suggesting TMS might be an effective therapy.
LIGHT AND SOUND THERAPY FOR ALZHEIMER'S
Brain wave stimulation using pulses of light and sound were associated with improved learning and memory in mouse models of Alzheimer’s disease, and prolonged visual stimulation prevented neuron death and preserved synaptic connections, researchers at MIT's Picower Institute showed. Based on these findings, human clinical trials of this non-invasive therapy began in 2019.
AI FOR EMBRYO SELECTION
A new artificial intelligence approach can identify high-quality embryos for use in IVF by analyzing time-lapse images, a technique that might improve on the subjective process now used to select embryos for implantation. The algorithm, dubbed STORK, was able to analyze the quality of the embryo with over 97% accuracy, outperforming five experienced embryologists in a study by Weill Cornell researchers. By itself, the algorithm can't yet predict whether an embryo will lead to a live birth.
SMART BREATHING MONITOR
University of Washington researchers demonstrated that smartphones or smart speakers could be programmed to accurately identify the distinctive breathing sounds of a person experiencing cardiac arrest or an overdose. They developed the software using recordings of 911 calls, and audio from a supervised injection facility and an operating room during administration of anesthesia. The goal is to alert emergency responders and others in time to begin life-saving treatment.
RNA MEDICINE
Small interfering RNAs (siRNAs) can block expression of disease-causing genes. Two such drugs have been approved for liver diseases, but delivery into the brain is a challenge. UMass scientists linked together two modified siRNAs that target the Huntington's disease gene and injected this treatment into mice and macaques, achieving broad distribution in the brain. It significantly cut production of the huntingtin protein for up to six months with limited off-target effects. A similar approach could eventually be applied to other neurological disorders.
GULF WAR ILLNESS
Tests on mice suggest a protein called HMGB1 might underlie many of the troubling symptoms of Gulf War Illness — a syndrome affecting American troops who served in the first Gulf War in the early 1990s. These mouse models of the syndrome had elevated levels of HMGB1 and other inflammatory chemicals in the brain. Texas A&M researchers also found a non-invasive way to measure these chemicals in blood. Monoclonal antibody drugs that inhibit HMGB1 are already available and the authors suggest testing them in people with the illness.
ENCYCLOPEDIA OF SEX DIFFERENCES
A comprehensive survey of sex differences in gene expression in human, macaque, mouse, rat, and dog, across 12 tissues, identified hundreds of genes associated with sex differences. They explain 12% of the variation in human height and show that sex differences evolved as mammals did. The Whitehead Institute-led team created an extensive catalog of which genes have sex-biased expression in which organs for use in future research.
CRISPR ADVANCE
The discovery of "prime editing" expands the scope of the revolutionary CRISPR genome editing technology, giving it the capability to correct up to 89% ofl known genetic variants associated with human diseases. Prime editing can efficiently and precisely make targeted insertions, deletions, and all possible single-letter changes in DNA, and Broad Institute researchers demonstrated its utility by repairing the primary genetic causes of sickle cell and Tay-Sachs diseases.
ENHANCING CHECKPOINT INHIBITORS
A MD Anderson team analyzed bone marrow from patients with metastatic prostate cancer treated with checkpoint inhibitors to learn why the immunotherapy fails to stop lethal bone metasteses. They found when tumors damage bone, the body produces huge amounts of a growth factor that steers the immune system away from making the kind of cells required to trigger an anti-tumor response, thwarting checkpoint blockade therapy. Based on mouse studies, they are ready to start a clinical trial combining checkpoint inhibitors with a growth-factor inhibitor.
NEW CANCER IMMUNOTHERAPY
A type of cancer immunotherapy called tumor-associated antigen-specific cytotoxic T cells was shown by Children's National Hospital researchers to be safe in a first-in-human clinical trial in patients — mostly children — with solid organ tumors that didn't respond to previous treatments; 11 of 15 patients had stable disease or better 45 days after infusion, including 6 with progression-free survival after a median of 13.9 months.
ROBOTIC FLOSSING?
UPenn researchers designed antimicrobial, microscopic robots that precisely and efficiently killed, degraded, and removed biofilms from biological surfaces. Iron oxide nanoparticles were precisely controlled with a magnetic field, and the team demonstrated how they could be used to clean hard-to-reach surfaces on teeth. The technology could one day be used to remove plaque from teeth, as well as remove stubborn biofilms from catheters and medical devices.
SMART MATERIALS
In a novel use of CRISPR, resesarchers from Harvard's Wyss Institute and MIT engineered programmable materials that exploit the genome-editor's DNA-cutting properties to trigger the release of whole cells and biofluorescent markers from hydrogels made with DNA. In response to specific stimuli, CRISPR can precisely snip open these hydrogels to deliver a drug at the site of a tumor or infection, or build an electronic sensor that could signal the presence of a pathogen such as Ebola virus.
STOPPING MOSQUITO BITES
Appetite suppressants may work to keep disease-carrying mosquitoes from biting people. A Rockefeller University team identified the signaling molecule that tells mosquitoes they've had enough blood. They modified it and found it could stop them from feeding. It's a version of neuropeptide Y, which affects hunger in many species. They suggest developing this compound for use in bait traps, or breeding genetically engineered males so that their offspring won't be hungry for blood.
PREVENTING PEANUT ALLERGIES
A team led by Notre Dame scientists devised a possible new way to treat peanut allergies by stopping immune system antibodies (IgE) from attaching to the allergens in peanuts. The researchers identified the binding sites and designed compounds to stop this binding. In tests using samples from 16 allergic people, the compounds prevented an allergic response in 14 of them, indicating they might be an effective allergy therapy.
STRESS TEST
This study suggests that the first year of medical residency causes young doctors’ DNA to age six times faster than normal, with the largest effect among those whose training programs demand the longest hours. The study, by researchers at Michigan and Brown University, is the first to measure people's telomere length before and after a prolonged, intense experience. Telomeres are seen as an indicator of aging and disease risk, and the work suggests telomere length can serve as a biomarker for the effects of stress.
VAPING'S HEALTH EFFECTS
Vaping can disrupt lung function and cause abnormal accumulation of lipids in the lungs, a Baylor College of Medicine team found. In tests on rats, the researchers showed they were also more susceptible to influenza after exposure to e-cigarette vapor. The findings support other studies that found lipid-laden immune cells called macrophages in the lungs of patients with a vaping-associated pneumonia called EVALI.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
THREE MUTATIONS, ONE DISEASE
Many diseases are thought to result from inheriting multiple rare mutations that are benign on their own but harmful in combination, but this hypothesis has been hard to prove. Gladstone Institutes researchers demonstrated that a form of childhood-onset cardiomyopathy is caused by mutations in three genes; they sequenced the genomes of three siblings with the heart disease and their parents, who were asymptomatic, then used CRISPR genome editing to insert the mutations into mice, which went on to develop heart disease.
PERSONALIZED MEDICINE
Doctors at Children's Hospital of Philadelphia identified a genetic mutation causing a 12-year-old’s rare illness, in which lymphatic vessels proliferate and leak fluid into the lungs and other organs, and then engineered the mutation into zebrafish. The fish developed a piscine version of the boy’s disease, and after the team tested multiple drugs on these fish, they found one — an existing melanoma drug called trametinib — that stopped the vessels’ growth. When they tried it on the boy, he recovered and has since returned to a normal life.
ATTACKING C. DIFF TOXIN
Researchers uncovered the three-dimensional structure of a toxin primarily responsible for Clostridium difficile infection and revealed how antibodies could neutralize the toxin, providing a blueprint for developing new drugs and vaccines. The UC Irvine-led team is now developing a novel vaccine based on the findings.
CLUE TO STOPPING GONORRHEA
Gonorrhea cases in the U.S. rose 67% from 2014-17, and drug-resistant strains are an urgent concern. Georgia State researchers found that blocking the ability of Neisseria gonorrhoeae to take up the mineral zinc can stop its growth. The findings could be used to propel the development of a vaccine that would starve the microbe of the nutrients it needs to infect people and maybe help control gonorrhea.
MICROBIOME HEALTH
Aiming to analyze how the makeup of the gut microbiome correlates with health and obesity, Institute for Systems Biology researchers found that certain metabolites in blood could predict the diversity of the microbiome, suggesting a blood test might be used to monitor the microbiome. Their early findings indicate that more diversity of gut bacteria may not always be the best and that there is a "Goldilocks zone" of gut flora diversity.
PREDICTING MORTALITY WITH BLOOD TEST
A retrospective study of 30,000 patients found that a a patient’s white blood count, which is collected as part of routine blood work performed during standard health exams, can be a powerful predictor of mortality in the general population. Researchers from Cleveland Clinic and University Hospitals found that lymphopenia — abnormally low levels of lymphocytes — is associated with increased risk of death from cardiovascular disease, cancer, and respiratory illnesses, especially if there is also inflammation.
BIASED AI
An algorithm widely used by hospitals to identify patients needing extra medical help was shown to have significant racial bias because it uses health costs as a proxy for illness. The algorithm affects health care decisions for tens of millions of patients, and removing the bias would more than double the number of Black patients eligible for additional care, according to the UC Berkeley-led study. Retraining the algorithm using patient health data instead of insurance claims reduced bias by 84%.
HURRICANES' HARM
Natural disasters have far-reaching health effects. American Cancer Society researchers showed lung cancer patients whose radiation treatment was interrupted by a hurricane were less likely to survive than patients who underwent treatment at the same facility when no disaster occurred. Mean survival time was 29 months for those whose care was disrupted vs. 31 months for the others. The longer the disaster declaration, the worse the patients’ survival.
DNA MICROSCOPE
Researchers at the Broad and McGovern institutes invented a new way of imaging the inside of cells that simultaneously shows not only the locations of DNA and RNA in a cell but also the precise nucleotide-by-nucleotide sequences of each molecule. The technique can be used to image interactions within a single cell and might be used to guide cancer treatments, as well as to look at cell structures at the molecular and genetic level at the same time.
LIQUID BIOPSY
A University of Michigan team designed a microfluidic chip to efficiently capture circulating tumor cells and isolate their RNA for single-cell gene expression analysis — a non-invasive way to comprehensively profile tumor genetics without a biopsy. The system was validated by isolating 666 tumor cells from the blood of 21 breast cancer patients and showing it could detect intrapatient heterogeneity in biomarkers critical for metastasis and to identify drug targets.
NEW CANCER APPROACH
Close genetic analysis using CRISPR editing shows how 19 different mutations in a gene involved in processing DNA instructions into proteins led to faulty splicing and drove cancer growth in a variety of cancer types, including leukemia and solid tumors. Researchers from Fred Hutchinson and Memorial Sloan Kettering cancer centers developed a targeted antisense oligonucleotide that corrected the fault and caused tumors in mice to die off. The work shows the potential for using antisense therapy against cancer.
COMBINATION CANCER THERAPIES
UC San Diego researchers used cocktails of cancer therapies molecularly targeted to the tumors of stage 4 patients. There was no control group and the number of patients was small, but the approach was safe and the authors said the findings suggest this combination approach to personalized cancer treatment is superior to trying one agent at a time. The therapies customized for patients appeared to increase progression-free survival by 30 percent.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
OPIOID ALTERNATIVE
NYU researchers and colleagues showed that nanoparticles could be used to deliver a pain drug — one that previously failed in clinical trials — into specific compartments of nerve cells, dramatically increasing its ability to treat pain in mice and rats. This approach could offer a non-opioid treatment option for chronic pain, said the authors. The treatment, however, involves a spinal injection.
ARTERY DISEASE
Yale researchers identified a new mechanism underlying artery disease, showing in mice that activation of TGF-beta signaling in the walls of blood vessels brings on inflammation, which in turn causes atherosclerosis. Interrupting this positive feedback loop that keeps vascular inflammation going led to a 70% regression of atherosclerosis lesions, something never seen before. The work suggests atherosclerosis is driven more by a population of susceptible endothelial cells in arteries than by traditional risk factors including high cholesterol and hypertension.
TRANSPLANTING INFECTED HEARTS
Hoping to reduce the shortage of organ donors, UPenn researchers transplanted hearts from deceased donors infected with hepatitis C — organs that have long been discarded as unsafe — and treated the recipients with new curative antiviral drugs to prevent infection. Hearts were transplanted into 10 uninfected recipients, and there was a 100% cure rate among the 9 completing antiviral therapy. One recipient died from organ rejection, and another required dialysis, but these were unrelated to the infection and antiviral therapy. The results pave the way for routine use of hep C hearts.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
VELCRO CANCER THERAPY
Cytokines such as interkeukins are immune system signaling proteins that have anti-tumor properties but are also highly toxic to healthy cells. Injecting cytokines into solid tumors has failed because they quickly leak into the body's circulation. Koch Institute researchers fused cytokines to a protein that binds to collagen, a molecule tumors have in abundance. This sharply reduced the leakage out of tumors and in mice, the fused cytokines enhanced the effectiveness of other cancer immunotherapies.
SIMPLER MEMORY TEST
Some people balk at taking written and verbal tests used to diagnose Alzheimer's and mild cognitive impairment (MCI). Emory researchers, building on studies linking eye movement with memory impairment, devised a method to precisely measure eye movements of people taking a simple test — they were presented an image, and then later with something taken out. Most people noticed the missing item but those with dementia or MCI had a different eye pattern when looking at it. The team is developing an iPad version of the test.
CELL THERAPY FOR BRAIN INJURY
In mice with a traumatic brain injury, transplanted embryonic progenitor cells, capable of generating a type of inhibitory neuron that tamps down excessive brain activity, migrated into the hippocampus, the brain region responsible for learning and memory, forming new connections and surviving long term. Within a month, the mice showed signs of improved memory, performing as well on tests as uninjured mice, UC Irvine researchers reported.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
PHAGE THERAPY FOR LIVER DISEASE
As many as 75% of patients die within 90 days of a diagnosis of severe alcoholic hepatitis, and a liver transplant is the only cure. UC San Diego researchers found that cytolysin — a toxin secreted by Enterococcus faecalis — kills liver cells, and people with alcoholic liver disease have more of these bacteria in their gut than healthy people. Mice colonized with bacteria from patients with alcoholic hepatitis developed the disease, but treatment with bacteriophages that target E. faecalis eliminated the bacteria and the disease.
ARE 10,000 STEPS NECESSARY?
While 10,000 daily steps is a popular goal, there's little research on just how many steps are needed to improve health. This Brigham and Women's Hospital study of older women found that those who took just 4,400 steps per day had markedly lower mortality rates after four years than the least active women, who took 2,700 steps per day. More steps were associated with even lower mortality rates up to 7,500 steps per day, though as an observational study, it couldn't prove causation.
BUBBLE BOY DISEASE
Using a novel gene therapy, a St. Jude Children's Research Hospital team appears to have cured eight babies born with X-linked severe combined immunodeficiency, a disease that leaves infants without a functional immune system and is usually fatal by age 2 unless they qualify for a bone marrow transplant. After an average followup of 16 months, the treatment restored infants' immune function and wiped out existing infections.
BREAST CANCER THERAPY
Adding a targeted drug called ribociclib to standard hormone therapy for younger women with an aggressive form of breast cancer lowered their risk of death. After 3.5 years, 70% of premenopausal women with metastatic cancer who got the combination were alive, compared to 46% of those on hormone therapy alone. This long-running trial led by MD Anderson Cancer Center already changed the standard of care, and the FDA approved ribociclib for this population in 2018. The new results demonstrated the durability of the treatment's benefits.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
SWEETENED DRINKS AND CANCER
Consumption of drinks sweetened with high fructose corn syrup has been associated with colon cancer risk, and their growing use has coincided with a rise of colorectal cancer in young and middle-aged adults. Weill Cornell researchers showed that ingestion of HFCS, even in moderate amounts, promotes the growth of colon cancer in mice. An enzyme in tumors converts fructose to a metabolite that promotes cell growth. Whether a similar process occurs in humans remains to be seen, but the authors urge people with colon cancer or at high risk to avoid sugary drinks.
DRUGGABLE DIABETES TARGET
University of Utah and Merck researchers made genetically engineered mice lacking an enzyme called dihydroceramide desaturase 1 (DES1), which prevented insulin resistance and fatty liver disease in animals fed fat-laden diets and reversed prediabetes in obese mice. The findings suggest this enzyme may be a possible target for human treatments for obesity, diabetes, and related conditions.
ENGINEERED IMMUNITY
Nearly every vaccine works by coaxing B cells — a key component of the immune system — to make antibodies, but sometimes vaccines provide only partial protection, or none at all. Fred Hutchinson cancer center researchers demonstrated an alternative approach: Using CRISPR genome-editing, they inserted DNA blueprints for specific antibodies into B cells from mice and humans and showed they could produce antibodies against a range of viruses. B cells engineered to produce antibodies against respiratory syncytial virus provided potent, long-lasting protection in mice.
REPURPOSED DRUG
University of Iowa researchers and colleagues identified a drug widely prescribed for an enlarged prostate as a possible therapy for Parkinson's disease, for which there is no progression-slowing treatment. Impaired energy metabolism is a common feature of Parkinson's, and when the team tested terazosin in rodents, fruit flies, and lab-grown human neurons, they found it enhanced energy production, slowed neuron death, and partially restored motor function. A search of patient databases revealed decreased disease progression in Parkinson's patients who happened to be taking the drug for an enlarged prostate.
THE SOCIAL BRAIN
UCLA researchers found that socially interacting mice exhibit striking correlations of brain activity during ongoing social interaction. This interbrain synchronization emerges from two sets of neurons that separately encode one’s own social behaviors and those of the interacting partner. Synchronization during the initial phase of the social encounter predicts future social interactions as well as the dominance relationship the animals develop over time. These findings may help study disorders that affect social functioning, including depression and autism.
3D-PRINTED ORGANS
Wyss Institute researchers developed a new technique to bioprint blood-vessel-like channels into mini-organs grown from induced pluripotent stem cells. They created batches of organ-specific tissues with high levels of oxygenation and function that survived for multiple days, and they hope to refine the technique to develop fully 3D-printed organs for transplant.
CHEMO-RESISTANT TUMORS
University of Michigan researchers demonstrated how pancreatic tumors neutralize the effects of gemcitabine chemotherapy, by attracting and manipulating macrophages. The mechanism may underlie resistance in other tumors, as well, and might help guide the choice of treatments for patients based on the levels of these susceptible macrophages in tumors.
BLOCKING CANCER STEM CELLS
Researchers from Stony Brook University discovered an enzyme driving cancer stem cell growth that underlies tumor recurrence, drug resistance, and progression in triple negative breast cancer, suggesting it could be a new drug target. They showed how the enzyme regulates cancer stem cells and that inhibiting the enzyme suppressed tumor growth and improved sensitivity to chemotherapy in mice. Enzyme activity was also increased in breast tumors from patients with lymph node metastasis.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
LIGHT AND SOUND THERAPY FOR ALZHEIMER'S
Brain wave stimulation using pulses of light and sound were associated with improved learning and memory in mouse models of Alzheimer’s disease, and prolonged visual stimulation prevented neuron death and preserved synaptic connections, researchers at MIT's Picower Institute showed. Based on these findings, human clinical trials of this non-invasive therapy began in 2019.
SMART BREATHING MONITOR
University of Washington researchers demonstrated that smartphones or smart speakers could be programmed to accurately identify the distinctive breathing sounds of a person experiencing cardiac arrest or an overdose. They developed the software using recordings of 911 calls, and audio from a supervised injection facility and an operating room during administration of anesthesia. The goal is to alert emergency responders and others in time to begin life-saving treatment.
RNA MEDICINE
Small interfering RNAs (siRNAs) can block expression of disease-causing genes. Two such drugs have been approved for liver diseases, but delivery into the brain is a challenge. UMass scientists linked together two modified siRNAs that target the Huntington's disease gene and injected this treatment into mice and macaques, achieving broad distribution in the brain. It significantly cut production of the huntingtin protein for up to six months with limited off-target effects. A similar approach could eventually be applied to other neurological disorders.
CRISPR ADVANCE
The discovery of "prime editing" expands the scope of the revolutionary CRISPR genome editing technology, giving it the capability to correct up to 89% ofl known genetic variants associated with human diseases. Prime editing can efficiently and precisely make targeted insertions, deletions, and all possible single-letter changes in DNA, and Broad Institute researchers demonstrated its utility by repairing the primary genetic causes of sickle cell and Tay-Sachs diseases.
NEW CANCER IMMUNOTHERAPY
A type of cancer immunotherapy called tumor-associated antigen-specific cytotoxic T cells was shown by Children's National Hospital researchers to be safe in a first-in-human clinical trial in patients — mostly children — with solid organ tumors that didn't respond to previous treatments; 11 of 15 patients had stable disease or better 45 days after infusion, including 6 with progression-free survival after a median of 13.9 months.
SMART MATERIALS
In a novel use of CRISPR, resesarchers from Harvard's Wyss Institute and MIT engineered programmable materials that exploit the genome-editor's DNA-cutting properties to trigger the release of whole cells and biofluorescent markers from hydrogels made with DNA. In response to specific stimuli, CRISPR can precisely snip open these hydrogels to deliver a drug at the site of a tumor or infection, or build an electronic sensor that could signal the presence of a pathogen such as Ebola virus.
PREVENTING PEANUT ALLERGIES
A team led by Notre Dame scientists devised a possible new way to treat peanut allergies by stopping immune system antibodies (IgE) from attaching to the allergens in peanuts. The researchers identified the binding sites and designed compounds to stop this binding. In tests using samples from 16 allergic people, the compounds prevented an allergic response in 14 of them, indicating they might be an effective allergy therapy.
STRESS TEST
This study suggests that the first year of medical residency causes young doctors’ DNA to age six times faster than normal, with the largest effect among those whose training programs demand the longest hours. The study, by researchers at Michigan and Brown University, is the first to measure people's telomere length before and after a prolonged, intense experience. Telomeres are seen as an indicator of aging and disease risk, and the work suggests telomere length can serve as a biomarker for the effects of stress.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
PERSONALIZED MEDICINE
Doctors at Children's Hospital of Philadelphia identified a genetic mutation causing a 12-year-old’s rare illness, in which lymphatic vessels proliferate and leak fluid into the lungs and other organs, and then engineered the mutation into zebrafish. The fish developed a piscine version of the boy’s disease, and after the team tested multiple drugs on these fish, they found one — an existing melanoma drug called trametinib — that stopped the vessels’ growth. When they tried it on the boy, he recovered and has since returned to a normal life.
ATTACKING C. DIFF TOXIN
Researchers uncovered the three-dimensional structure of a toxin primarily responsible for Clostridium difficile infection and revealed how antibodies could neutralize the toxin, providing a blueprint for developing new drugs and vaccines. The UC Irvine-led team is now developing a novel vaccine based on the findings.
MICROBIOME HEALTH
Aiming to analyze how the makeup of the gut microbiome correlates with health and obesity, Institute for Systems Biology researchers found that certain metabolites in blood could predict the diversity of the microbiome, suggesting a blood test might be used to monitor the microbiome. Their early findings indicate that more diversity of gut bacteria may not always be the best and that there is a "Goldilocks zone" of gut flora diversity.
BIASED AI
An algorithm widely used by hospitals to identify patients needing extra medical help was shown to have significant racial bias because it uses health costs as a proxy for illness. The algorithm affects health care decisions for tens of millions of patients, and removing the bias would more than double the number of Black patients eligible for additional care, according to the UC Berkeley-led study. Retraining the algorithm using patient health data instead of insurance claims reduced bias by 84%.
LIQUID BIOPSY
A University of Michigan team designed a microfluidic chip to efficiently capture circulating tumor cells and isolate their RNA for single-cell gene expression analysis — a non-invasive way to comprehensively profile tumor genetics without a biopsy. The system was validated by isolating 666 tumor cells from the blood of 21 breast cancer patients and showing it could detect intrapatient heterogeneity in biomarkers critical for metastasis and to identify drug targets.
NEW CANCER APPROACH
Close genetic analysis using CRISPR editing shows how 19 different mutations in a gene involved in processing DNA instructions into proteins led to faulty splicing and drove cancer growth in a variety of cancer types, including leukemia and solid tumors. Researchers from Fred Hutchinson and Memorial Sloan Kettering cancer centers developed a targeted antisense oligonucleotide that corrected the fault and caused tumors in mice to die off. The work shows the potential for using antisense therapy against cancer.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
ARTERY DISEASE
Yale researchers identified a new mechanism underlying artery disease, showing in mice that activation of TGF-beta signaling in the walls of blood vessels brings on inflammation, which in turn causes atherosclerosis. Interrupting this positive feedback loop that keeps vascular inflammation going led to a 70% regression of atherosclerosis lesions, something never seen before. The work suggests atherosclerosis is driven more by a population of susceptible endothelial cells in arteries than by traditional risk factors including high cholesterol and hypertension.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
VELCRO CANCER THERAPY
Cytokines such as interkeukins are immune system signaling proteins that have anti-tumor properties but are also highly toxic to healthy cells. Injecting cytokines into solid tumors has failed because they quickly leak into the body's circulation. Koch Institute researchers fused cytokines to a protein that binds to collagen, a molecule tumors have in abundance. This sharply reduced the leakage out of tumors and in mice, the fused cytokines enhanced the effectiveness of other cancer immunotherapies.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
PHAGE THERAPY FOR LIVER DISEASE
As many as 75% of patients die within 90 days of a diagnosis of severe alcoholic hepatitis, and a liver transplant is the only cure. UC San Diego researchers found that cytolysin — a toxin secreted by Enterococcus faecalis — kills liver cells, and people with alcoholic liver disease have more of these bacteria in their gut than healthy people. Mice colonized with bacteria from patients with alcoholic hepatitis developed the disease, but treatment with bacteriophages that target E. faecalis eliminated the bacteria and the disease.
BUBBLE BOY DISEASE
Using a novel gene therapy, a St. Jude Children's Research Hospital team appears to have cured eight babies born with X-linked severe combined immunodeficiency, a disease that leaves infants without a functional immune system and is usually fatal by age 2 unless they qualify for a bone marrow transplant. After an average followup of 16 months, the treatment restored infants' immune function and wiped out existing infections.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
DRUGGABLE DIABETES TARGET
University of Utah and Merck researchers made genetically engineered mice lacking an enzyme called dihydroceramide desaturase 1 (DES1), which prevented insulin resistance and fatty liver disease in animals fed fat-laden diets and reversed prediabetes in obese mice. The findings suggest this enzyme may be a possible target for human treatments for obesity, diabetes, and related conditions.
ENGINEERED IMMUNITY
Nearly every vaccine works by coaxing B cells — a key component of the immune system — to make antibodies, but sometimes vaccines provide only partial protection, or none at all. Fred Hutchinson cancer center researchers demonstrated an alternative approach: Using CRISPR genome-editing, they inserted DNA blueprints for specific antibodies into B cells from mice and humans and showed they could produce antibodies against a range of viruses. B cells engineered to produce antibodies against respiratory syncytial virus provided potent, long-lasting protection in mice.
3D-PRINTED ORGANS
Wyss Institute researchers developed a new technique to bioprint blood-vessel-like channels into mini-organs grown from induced pluripotent stem cells. They created batches of organ-specific tissues with high levels of oxygenation and function that survived for multiple days, and they hope to refine the technique to develop fully 3D-printed organs for transplant.
CHEMO-RESISTANT TUMORS
University of Michigan researchers demonstrated how pancreatic tumors neutralize the effects of gemcitabine chemotherapy, by attracting and manipulating macrophages. The mechanism may underlie resistance in other tumors, as well, and might help guide the choice of treatments for patients based on the levels of these susceptible macrophages in tumors.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
LIGHT AND SOUND THERAPY FOR ALZHEIMER'S
Brain wave stimulation using pulses of light and sound were associated with improved learning and memory in mouse models of Alzheimer’s disease, and prolonged visual stimulation prevented neuron death and preserved synaptic connections, researchers at MIT's Picower Institute showed. Based on these findings, human clinical trials of this non-invasive therapy began in 2019.
CRISPR ADVANCE
The discovery of "prime editing" expands the scope of the revolutionary CRISPR genome editing technology, giving it the capability to correct up to 89% ofl known genetic variants associated with human diseases. Prime editing can efficiently and precisely make targeted insertions, deletions, and all possible single-letter changes in DNA, and Broad Institute researchers demonstrated its utility by repairing the primary genetic causes of sickle cell and Tay-Sachs diseases.
NEW CANCER IMMUNOTHERAPY
A type of cancer immunotherapy called tumor-associated antigen-specific cytotoxic T cells was shown by Children's National Hospital researchers to be safe in a first-in-human clinical trial in patients — mostly children — with solid organ tumors that didn't respond to previous treatments; 11 of 15 patients had stable disease or better 45 days after infusion, including 6 with progression-free survival after a median of 13.9 months.
STRESS TEST
This study suggests that the first year of medical residency causes young doctors’ DNA to age six times faster than normal, with the largest effect among those whose training programs demand the longest hours. The study, by researchers at Michigan and Brown University, is the first to measure people's telomere length before and after a prolonged, intense experience. Telomeres are seen as an indicator of aging and disease risk, and the work suggests telomere length can serve as a biomarker for the effects of stress.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
ATTACKING C. DIFF TOXIN
Researchers uncovered the three-dimensional structure of a toxin primarily responsible for Clostridium difficile infection and revealed how antibodies could neutralize the toxin, providing a blueprint for developing new drugs and vaccines. The UC Irvine-led team is now developing a novel vaccine based on the findings.
MICROBIOME HEALTH
Aiming to analyze how the makeup of the gut microbiome correlates with health and obesity, Institute for Systems Biology researchers found that certain metabolites in blood could predict the diversity of the microbiome, suggesting a blood test might be used to monitor the microbiome. Their early findings indicate that more diversity of gut bacteria may not always be the best and that there is a "Goldilocks zone" of gut flora diversity.
NEW CANCER APPROACH
Close genetic analysis using CRISPR editing shows how 19 different mutations in a gene involved in processing DNA instructions into proteins led to faulty splicing and drove cancer growth in a variety of cancer types, including leukemia and solid tumors. Researchers from Fred Hutchinson and Memorial Sloan Kettering cancer centers developed a targeted antisense oligonucleotide that corrected the fault and caused tumors in mice to die off. The work shows the potential for using antisense therapy against cancer.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
BUBBLE BOY DISEASE
Using a novel gene therapy, a St. Jude Children's Research Hospital team appears to have cured eight babies born with X-linked severe combined immunodeficiency, a disease that leaves infants without a functional immune system and is usually fatal by age 2 unless they qualify for a bone marrow transplant. After an average followup of 16 months, the treatment restored infants' immune function and wiped out existing infections.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
ENGINEERED IMMUNITY
Nearly every vaccine works by coaxing B cells — a key component of the immune system — to make antibodies, but sometimes vaccines provide only partial protection, or none at all. Fred Hutchinson cancer center researchers demonstrated an alternative approach: Using CRISPR genome-editing, they inserted DNA blueprints for specific antibodies into B cells from mice and humans and showed they could produce antibodies against a range of viruses. B cells engineered to produce antibodies against respiratory syncytial virus provided potent, long-lasting protection in mice.
CHEMO-RESISTANT TUMORS
University of Michigan researchers demonstrated how pancreatic tumors neutralize the effects of gemcitabine chemotherapy, by attracting and manipulating macrophages. The mechanism may underlie resistance in other tumors, as well, and might help guide the choice of treatments for patients based on the levels of these susceptible macrophages in tumors.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
LIGHT AND SOUND THERAPY FOR ALZHEIMER'S
Brain wave stimulation using pulses of light and sound were associated with improved learning and memory in mouse models of Alzheimer’s disease, and prolonged visual stimulation prevented neuron death and preserved synaptic connections, researchers at MIT's Picower Institute showed. Based on these findings, human clinical trials of this non-invasive therapy began in 2019.
NEW CANCER IMMUNOTHERAPY
A type of cancer immunotherapy called tumor-associated antigen-specific cytotoxic T cells was shown by Children's National Hospital researchers to be safe in a first-in-human clinical trial in patients — mostly children — with solid organ tumors that didn't respond to previous treatments; 11 of 15 patients had stable disease or better 45 days after infusion, including 6 with progression-free survival after a median of 13.9 months.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
ATTACKING C. DIFF TOXIN
Researchers uncovered the three-dimensional structure of a toxin primarily responsible for Clostridium difficile infection and revealed how antibodies could neutralize the toxin, providing a blueprint for developing new drugs and vaccines. The UC Irvine-led team is now developing a novel vaccine based on the findings.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
ENGINEERED IMMUNITY
Nearly every vaccine works by coaxing B cells — a key component of the immune system — to make antibodies, but sometimes vaccines provide only partial protection, or none at all. Fred Hutchinson cancer center researchers demonstrated an alternative approach: Using CRISPR genome-editing, they inserted DNA blueprints for specific antibodies into B cells from mice and humans and showed they could produce antibodies against a range of viruses. B cells engineered to produce antibodies against respiratory syncytial virus provided potent, long-lasting protection in mice.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
NEW CANCER IMMUNOTHERAPY
A type of cancer immunotherapy called tumor-associated antigen-specific cytotoxic T cells was shown by Children's National Hospital researchers to be safe in a first-in-human clinical trial in patients — mostly children — with solid organ tumors that didn't respond to previous treatments; 11 of 15 patients had stable disease or better 45 days after infusion, including 6 with progression-free survival after a median of 13.9 months.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
RESTORING HEART FUNCTION
After a heart attack, patients often develop scar tissue that can lead to dangerous heart rhythm abnormalities. Researchers at Texas Heart Institute and Rice University used thread-like carbon nanotube fibers to bridge across scar tissue in an effort to restore normal electrical activity. They sewed these fibers into the hearts of sheep with scarring like that from a heart attack, and the animals' electrical function improved, suggesting this approach might improve treatment of cardiac arrhythmias in people.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.
SEEING TINY TUMORS
Ovarian cancer patients' survival depends on how much tumor can be removed, but tiny tumors often have spread so abundantly it’s difficult for surgeons to find and remove them. MIT and Massachusetts General Hospital researchers developed an imaging system using a fluorescing nanotubule that binds a protein abundant in ovarian cancer cells, making it possible to see tumors smaller than a poppy seed in real-time so that they could be removed. Mice undergoing surgery with the imaging system survived longer than those receiving standard surgery. Human prototypes are under development.