By Eric S. Lander
IS THE PROMISE of genomic medicine overhyped?
This might seem a strange question coming from one of the leaders of the Human Genome Project, and the director of the Broad Institute, which brings together researchers from Harvard, MIT, and Harvard-affiliated hospitals to accelerate the understanding and treatment of disease.
I think the answer is a clear yes — and a resounding no. The contradiction highlights a thorny challenge in the ongoing conversation between scientists and the public.
This summer, I gave a talk at the Aspen Ideas Festival in which I discussed the need to accelerate medical progress through data-sharing and expressed the hope that, within the next 30 to 40 years, we might have enough knowledge to be able to turn cancer, for the majority of patients, into a treatable chronic condition rather than a lethal disease. Read more
by Jess Vilvestre
CORRECTING A MUTATION
Gene editing shows promise as a new treatment for sickle cell disease, according to a study published in the online journal Science Translational Medicine.
Experts from the University of California, Berkeley, UCSF Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine have found success in correcting the blood cell mutation in tests of the blood of both mice and human sickle cell patients using CRISPR-Cas9, a genome “scissor” that can cut out and edit a DNA sequence.
After CRISPR was used to correct the mutated hematopoietic stem cells — precursor cells that mature into the hook-shaped hemoglobin characteristic of sickle cell disease, the corrected blood stem cells produced healthy hemoglobin. Following reintroduction into the mice, the genetically engineered stem cells remained in circulation for at least four months — a significant indication that any potential therapy would be lasting. Read more
By Virginia Morell
All great mind reading begins with chocolate. That’s the basis for a classic experiment that tests whether children have something called theory of mind—the ability to attribute desires, intentions, and knowledge to others. When they see someone hide a chocolate bar in a box, then leave the room while a second person sneaks in and hides it elsewhere, they have to guess where the first person will look for the bar. If they guess “in the original box,” they pass the test, and show they understand what’s going on in the first person’s mind—even when it doesn’t match reality.
For years, only humans were thought to have this key cognitive skill of attributing “false belief,” which is believed to underlie deception, empathy, teaching, and perhaps even language. But three species of great apes—chimpanzees, bonobos, and orangutans—also know when someone holds a false belief, according to a new study published today in Science. The groundbreaking study suggests that this skill likely can be traced back to the last common ancestor of great apes and humans, and may be found in other species. Read more
By Andrea Morabito
Three to four days a week, Philadelphia veterinarian Clint Kuban drops off his 3-year-old German shepherd, Tsunami, at the Penn Vet Working Dog Center for her eight-hour shift.
But the pooch isn’t just participating in agility, obedience and search training — she’s also on the front lines of cancer research.
Kuban is one of six fourth-year students at the University of Pennsylvania School of Veterinary Medicine followed in the new Animal Planet series “Life at Vet U,” premiering Saturday at 10 p.m. Tsunami has worked on the center’s ovarian-cancer-detection research project since it launched in 2013 to study whether odors emanating from ovarian tissue can provide a reliable method for early detection. Read more
The 2016 Lasker Awards have highlighted some great discoveries and the scientists behind them. This guest post by Samuel Henager, a graduate student at Johns Hopkins University, investigates how animal studies contributed to the discoveries celebrated by this years’ Lasker Awards.
Basic Medical Research Award
The 2016 Albert Lasker Basic Medical Research Award was awarded to William G. Kaelin, Jr. of Dana-Farber Cancer Institute, Harvard Medical School, Peter J. Ratcliffe of University of Oxford, Francis Crick Institute, and Gregg L. Semenza of Johns Hopkins University School of Medicine for their work in discovering how cells sense and respond to changes in oxygen levels. Read more
By Jennifer Abbasi
As the first local mosquito-borne transmissions of the Zika virus are being reported in the continental United States, an investigational vaccine developed by the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH) is entering phase 1 clinical trials.
At least 80 people between the ages of 18 and 35 years will be enrolled in the trial, which will take place at 3 study sites in the United States including the NIH Clinical Center in Bethesda, Maryland. The trial will test vaccine safety and immunogenicity.
Scientists at NIAID’s Vaccine Research Center developed the DNA vaccine, which does not contain Zika virus and therefore cannot cause an infection. The vaccine contains a genetically engineered plasmid—a small, circular piece of DNA—that encodes Zika virus proteins. These proteins assemble into viruslike particles that provoke an immune response against the virus. This response is composed of both neutralizing antibodies and T cells.
Researchers from the University of Maryland Fischell Department of Bioengineering and the University of Maryland School of Medicine report a new way to “turn off” the harmful immune attack that occurs during autoimmune diseases such as multiple sclerosis (MS), while keeping healthy functions of the immune system intact.
“Our lab is combining immunology and nanotechnology to reprogram how the immune system responds to self-cells in the brain that are mistakenly attacked during MS,” said BIOE Assistant Professor Christopher Jewell, corresponding author on the new report. “The finding, conducted in cells and pre-clinical animal models of MS, could lead to new approaches for reversing paralysis in MS, or better therapies for other autoimmune diseases.” Read more
Letter to The Guardian
Nonhuman primates have long played a key role in life-changing medical advances. A recent white paper by nine scientific societies in the US produced a list of 50 medical advances from the last 50 years made possible through studies on nonhuman primates. These included: treatments for leprosy, HIV and Parkinson’s; the MMR and hepatitis B vaccines; and earlier diagnosis and better treatment for polycystic ovary syndrome and breast cancer.
The biological similarities between humans and other primates mean that they are sometimes the only effective model for complex neurodegenerative diseases such as Parkinson’s. More than 10 million people suffer from Parkinson’s worldwide, and a recent study estimated that one in three people born in 2015 will develop dementia in their lifetime. Primate research offers treatments, and hope for future treatments, to patients and their families. Already over 200,000 Parkinson’s patients have had their life dramatically improved thanks to deep brain stimulation surgery, which reduces the tremors of sufferers. This treatment was developed from research carried out in a few hundred monkeys in the 1980s and 1990s. Read more
By Wendy Rigby
San Antonio scientists are part of a push to develop laboratory animal models to study the Zika virus. Baboons and monkeys may be key to unlocking new treatments and vaccines.
Marmosets are a New World monkey that lives in Brazil, where it’s been infected with Zika in the wild. Now, scientists at Texas Biomedical Research Institute are using the small primate, about the size of a guinea pig, to test possible vaccines and treatments for Zika.
“When you infect marmoset with a human virus, the disease looks very much like what you see in humans,” explained Jean L. Patterson, Ph.D., scientist in the virology department. Read more
By David Grimm
Depending on whom you ask, yesterday’s U.S. government workshop on the state of nonhuman primate research was either a raging success or a complete fiasco. The event, held at the National Institutes of Health (NIH) in Bethesda, Maryland, brought together dozens of scientists, veterinarians, and bioethicists to discuss how research on monkeys and related animals is contributing to human medicine and to review the welfare policies that surround this work. But observers differed widely on whether it accomplished what Congress had in mind when it told NIH to hold the event.
“It was a great showcase of the importance nonhuman primates have played and continue to play in human health,” says Anne Deschamps, a senior science policy analyst at the Federation of American Societies for Experimental Biology in Bethesda, one of several scientific organizations that signed onto a white paper released in advance of the meeting that promoted the use of these animals in biomedical research. She contends that research on these animals has been critical for our understanding of HIV and the human brain. Read more