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January 10th

Focused Ultrasound Shows Promise for Parkinson's Disease; NEJM Article Reports

A scalpel-free alternative to brain surgery has the potential to benefit people with Parkinson's disease symptoms that are much more severe on one side of the body, new research suggests. More testing is needed, but the approach, which uses a technology called focused ultrasound, could offer a new option for patients whose symptoms are poorly controlled by medications and those who cannot or do not wish to undergo traditional brain surgery. "This small brain region, the subthalamic nucleus, had a very strong and potent effect on parkinsonian symptoms when we targeted it with precise, focused ultrasound energy," said researcher Jeff Elias (photo), MD, a neurosurgeon at University of VIrginia (UVA) Health and a pioneer in the field of focused ultrasound. "The key for the ultimate adoption of this new procedure will be further refinements of the technology to ensure reliability and safety." Focused ultrasound offers a minimally invasive alternative to traditional surgery approaches. The technology focuses sound waves inside the body, much like a magnifying glass focuses light. This allows doctors to interrupt faulty brain circuits or destroy unwanted tissue. Magnetic-resonance imaging (MRI) allows doctors to monitor the procedure in real time--and to make adjustments as needed to obtain the best patient outcomes. To determine if the technology could benefit patients with "asymmetrical" Parkinson's symptoms, Dr. Elias and Binit Shah, MD, from UVA's Department of Neurology, collaborated with Spain's Centro Intregral de Neurociencias to evaluate the approach in 40 volunteers in a randomized, double-blinded study. Twenty-seven study participants received treatment with focused ultrasound, while 13 others received a simulated treatment, so that the researchers could compare the results between the real procedure and the placebo.

January 9th

Entirety of Colon Cancer Cells, Not Just Subsets, Can Coordinately Adopt Evolutionarily Conserved Embryonic Survival Program to Slow Growth & Conserve Energy Under Stress of Chemotherapy; Autophagy Inhibitor Can Disrupt This Cancer Survival Strategy

In research published in the January 7, 2020 issue of Cell, Princess Margaret Cancer Centre (Toronto) Scientist Catherine O'Brien (photo), MD, PhD, and team discovered that, when under threat, all cancer cells--rather than just a subset--have the ability to transition into this protective state, where the cells "rest" until the threat, or chemotherapy, is removed. It is the first study to identify that cancer cells hijack an evolutionary conserved program to survive chemotherapy. Furthermore, the researchers show that novel therapeutic strategies aimed at specifically targeting cancer cells in this slow-dividing state can prevent cancer regrowth. "The tumor is acting like a whole organism, able to go into a slow-dividing state, conserving energy to help it survive," says Dr. O'Brien, who is also an Associate Professor in the Department of Surgery at the University of Toronto. "There are examples of animals entering into a reversible and slow-dividing state to withstand harsh environments. It appears that cancer cells have craftily co-opted this same state for their survival benefit." The Cell article is titled “Colorectal Cancer Cells Enter a Diapause-Like DTP State to Survive Chemotherapy.” (See graphical abstract of article at end.) Dr. Aaron Schimmer, Director of the Research Institute and Senior Scientist at Princess Margaret Cancer Centre, notes that this research shows that cancer cells hibernate, like "bears in winter." He adds: "We never actually knew that cancer cells were like hibernating bears. This study also tells us how to target these sleeping bears so they don't hibernate and wake up to come back later, unexpectedly. I think this will turn out to be an important cause of drug resistance, and will explain something we did not have a good understanding of previously."

January 8th

Exosomes & Laser for Acne Scars--BENEV Announces Investigative Report on Combination Treatment with Human Adipose Tissue Stem Cell-Derived Exosomes and Fractional CO2 Laser for Acne Scars

On January 7, 2021, BENEV Company, Inc., an FDA-registered cosmeceutical manufacturer of a globally recognized medical professional line, along with its Medical Advisory Board comprised of five global medical leaders, announced that that the 12-week Prospective, Double-Blind, Randomized, Split-Face Investigative Report on Combination Treatment with Human Adipose Tissue Stem Cell-Derived Exosomes and Fractional CO2 Laser for Acne Scars by ExoCoBio Inc., an equity-invested partner of BENEV, was published online on October 19, 2020, in Acta Dermato-Venereologica (https://www.medicaljournals.se/acta/content_files/files/pdf/100/18/5913.pdf). This open-access report outlines the investigative study that was conducted by a team of scientists and physicians including Hyuck Hoon Kwon, Steven Hoseong Yang, Joon Lee, Byung Chul Park, Kui Young Park, Jae Yoon Jung, Youin Bae, and Gyeong-Hun at Oaro Dermatology Institute (Seoul, South Korea); Guam Dermatology Institute (Guam, USA); Department of Dermatology, Dankook University, College of Medicine (Cheonan, South Korea); Department of Dermatology, Chung-Ang University, College of Medicine (Seoul, South Korea); and the Department of Dermatology, Dongtan Sacred Heart Hospital, Hallym University College of Medicine (Hwaseong, South Korea). Researchers involved in this study evaluated the clinical efficacy and safety of adipose tissue stem cell-derived exosomes as an adjuvant therapy after application of fractional CO2 laser for acne scars. 25 patients consisting of 18 men and 7 women, between ages 19 and 54, 12 with Fitzpatrick Skin Type 3 and 13 with Fitzpatrick skin type 4 and atrophic acne scars, underwent the 12-week prospective, double-blind, randomized, split-face trial.

January 7th

University of Minnesota Begins Phase I First-in-Human Clinical Trial for Glioblastoma; Novel Immune-Directed Approach, Advanced by Work on Glioblastomas Developing Naturally in Dogs, Could Be “Landmark Breakthrough” in Treatment of Rapidly Fatal Disease

Physicians and scientists at the University of Minnesota have opened a new brain cancer clinical trial and have treated their first patient. This Phase I, first-in-human trial is enrolling patients with a specific type of brain cancer, glioblastoma. The development for this innovative treatment is based on years of research by Michael Olin (photo), PhD, and Christopher Moertel, MD, researchers in the University of Minnesota Medical School’s Division of Hematology and Oncology, Department of Pediatrics, as well as a high-grade canine clinical trial conducted by G. Elizabeth Pluhar, DVM, PhD, DACVS, in the University of Minnesota College of Veterinary Medicine’s Department of Veterinary Clinical Sciences. The researchers are also members of the Masonic Cancer Center, University of Minnesota. “Our research found that the CD200 protein was acting as a protective shield inside a person’s brain tumor, effectively preventing the immune system or immune-directed therapy from attacking the tumor,” said Dr. Olin. “The CD200 checkpoint inhibitor that we developed, along with a proven vaccine, has shown amazing results in our tests and has the potential to have fewer adverse effects for patients.” Prior to being available to human patients, Dr. Olin and Dr. Moertel partnered with Dr. Pluhar at the College of Veterinary Medicine to treat pet dogs that spontaneously developed brain cancer. The CD200 checkpoint inhibitor, when added to a cancer vaccine therapy, increased their canine patients’ survival time by about 18 months after diagnosis compared to vaccine therapy alone. These tumors ultimately do recur in both dogs and people, but the time to regrowth was greatly extended in the dogs that received the experimental therapy, and their quality of life during treatment was excellent.

January 5th

Groundbreaking Trial for Treatment of Severe COVID-19 Shows Success Using Umbilical-Cord-Derived Mesenchymal Stem Cells (MSCs)

University of Miami Miller School of Medicine researchers led a unique and groundbreaking randomized controlled trial showing umbilical-cord-derived mesenchymal stem cell infusions safely reduce risk of death and quicken time to recovery for the severest COVID-19 patients, according to results published online on January 5, 2021 in STEM CELLS Translational Medicine. The open-access article is titled “Umbilical Cord Mesenchymal Stem Cells for COVID‐19 Acute Respiratory Distress Syndrome: A Double‐Blind, Phase 1/2a, Randomized Controlled Trial.” The study's senior author, Camillo Ricordi, MD, Director of the Diabetes Research Institute (DRI) and Cell Transplant Center at the University of Miami Miller School of Medicine, said treating COVID-19 with mesenchymal stem cells (image) makes sense. The paper describes findings from 24 patients hospitalized at University of Miami Tower or Jackson Memorial Hospital with COVID-19 who developed severe acute respiratory distress syndrome. Each received two infusions, given days apart, of either mesenchymal stem cells or placebo. "It was a double-blind study. Doctors and patients didn't know what was infused," Dr. Ricordi said. "Two infusions of 100 million stem cells were delivered within three days, for a total of 200 million cells in each subject in the treatment group." Researchers found the treatment was safe, with no infusion-related serious adverse events. Patient survival at one month was 91% in the stem-cell-treated group versus 42% in the control group. Among patients younger than 85 years old, 100% of those treated with mesenchymal stem cells survived at one month. Dr. Ricordi and colleagues also found time to recovery was faster among those in the treatment arm.

January 5th

Glioblastoma Could Be Linked to Combination of Mutational Change in Key Cells and Healing Process After Brain Injury, New Study Suggests; Findings May Lay Foundation for Precision Medicine Efforts to Treat Deadly Disease

The healing process that follows a brain injury--from trauma to infection and stroke--could spur growth of glioblastoma. This finding, published online on January4, 2021 in Nature Cancer (https://www.nature.com/articles/s43018-020-00154-9), was made by an interdisciplinary team of researchers from the University of Toronto, The Hospital for Sick Children (SickKids), and the Princess Margaret Cancer Centre. The researchers are part of the pan-Canadian Stand Up to Cancer Canada Dream Team (https://standuptocancer.ca/su2c-canada-cancer-stem-cell-dream-team/) that focuses on the common brain cancer known as glioblastoma. “Our data suggest that the right mutational change in particular cells in the brain could be modified by injury to give rise to a tumor,” says Peter Dirks, MD, PhD, a Professor in the Temerty Faculty of Medicine at the University of Toronto, and Dream Team leader, who is the Head of the Division of Neurosurgery and also a Senior Scientist in the Developmental and Stem Cell Biology Program at Sickkids. Gary Bader, PhD, a Professor of Molecular Genetics in the Temerty Faculty of Medicine and the Donnelly Centre for Cellular and Biomolecular Research at the University of Toronto, and Trevor Pugh, PhD, an Associate Professor of Medical Biophysics in the Temerty Faculty of Medicine and senior scientist at Princess Margaret, also led the research. The findings could lead to new therapy for glioblastoma patients who currently have limited treatment options with an average lifespan of 15 months after diagnosis.

Brown Fat May Protect Against Numerous Chronic Diseases, Rockefeller Study of 50,000 People Suggests; May Also Hold Clues to New Obesity Treatments

Brown fat may be an almost magical tissue you might want more of. Unlike white fat, which stores calories, brown fat burns energy and scientists hope it may hold the key to new obesity treatments. But it has long been unclear whether people with ample brown fat truly enjoy better health. For one thing, it has been hard to even identify such individuals because brown fat is hidden deep inside the body. Now, a new study in Nature Medicine (https://www.nature.com/articles/s41591-020-1126-7), published online on January 4, 2021, and conducted by Rockefeller University scientists and collaborators, offers strong evidence. The research team found that, among over 52,000 participants, those who had detectable brown fat were less likely than their peers to suffer cardiac and metabolic conditions ranging from type 2 diabetes to coronary artery disease, which is the leading cause of death in the United States. The Nature Medicine article is titled “Brown Adipose Tissue Is Associated with Cardiometabolic Health.” This study, by far the largest of its kind in humans, confirms and expands the health benefits of brown fat suggested by previous studies. "For the first time, it reveals a link to lower risk of certain conditions," says Paul Cohen (https://www.rockefeller.edu/our-scientists/heads-of-laboratories/1112-pa...), MD, PhD, the Albert Resnick, Assistant Professor and Senior Attending Physician at The Rockefeller University Hospital. "These findings make us more confident about the potential of targeting brown fat for therapeutic benefit." Although brown fat has been studied for decades in newborns and animals, it was only in 2009 that scientists appreciated it can also be also found in some adults, typically around the neck and shoulders.

January 3rd

DNA from Seabiscuit’s Hooves Offers Clues to Famous Race Horse’s Gifts of Speed & Stamina

[Editor’s Note: The following is a reprint of an article authored by Steven Tammariello (https://www.binghamton.edu/biology/people/profile.html?id=tammarie), PhD, that appeared in The Conversation (https://theconversation.com/can-seabiscuits-dna-explain-his-elite-racing...) on October 29, 2018 and was reprinted in Smithsonian Magazine (https://www.smithsonianmag.com/history/scientists-extract-dna-from-seabi...). It is reprinted here in BioQuick News in accordance with the pertinent Creative Commons License. Dr. Tammriello is Associate Professor of Biological Sciences and Director of the Institute for Equine Genomics, Binghamton University, State University of New York.]--Seabiscuit (http://www.americanclassicpedigrees.com/seabiscuit.html) (https://en.wikipedia.org/wiki/Seabiscuit) was not an impressive-looking horse. He was considered quite lazy, preferring to eat and sleep in his stall rather than exercise. He’d been written off by most of the racing industry after losing his first 17 races. But Seabiscuit eventually became one of the most beloved thoroughbred champions of all time – voted 1938 Horse of the Year after winning his legendary match race (https://www.theguardian.com/sport/2013/nov/01/seabiscuit-war-admiral-hor...) as an underdog against Triple Crown winner War Admiral in 1938 [photo shows Seabiscuit in the lead over War Admiral in their match race at Baltimore’s Pimlico Race Track on November 1, 1938 (Credit: AP)]. As a molecular physiologist, the concept of understanding how specific gene variants can affect performance--whether in athletics, learning, or even how an organism develops--has always intrigued me.

January 2nd

Adaptation of Forward Genetics Technique Leads to ID of Gene (KDM5A) Newly Associated with Autism Spectrum Disorder (ASD); Approach May Open Door to ID of More ASD-Associated Mutations and Advance Future Treatments; Article Co-Author Is Nobelist

University of Texas (UT) Southwestern (UTSW) scientists have adapted a classic research technique called forward genetics to identify new genes involved in autism spectrum disorder (ASD). In a study published online on December 22, 2020 in eLife (https://elifesciences.org/articles/56883), the researchers used this approach in mice to find one such gene called KDM5A. The open-access eLife article is titled “KDM5A Mutations Identified in Autism Spectrum Disorder Using Forward Genetics.” Approximately 1 in 54 children in the U.S. is diagnosed with ASD, a neurodevelopmental disorder that causes disrupted communication, difficulties with social skills, and repetitive behaviors. As a disease with a strong genetic component, it is hypothesized that thousands of genetic mutations may contribute to ASD. But to date, only about 30 percent of cases can be explained by known genetic mutations. For decades, forward genetics (https://www.google.com/search?client=firefox-b-1-d&q=reverse+genetics+an... ) has been used to find mutations that cause disease. It involves inducing genetic mutations in mice, screening for certain phenotypes, and then identifying the causative mutation through sequencing all of the organism’s genome. “The difficult part in the beginning was finding the mutations. It had to be done by laborious cloning,” says Nobel Laureate Bruce Beutler (https://profiles.utsouthwestern.edu/profile/10593/bruce-beutler.html) (https://www.nobelprize.org/prizes/medicine/2011/beutler/biographical/), MD, Director of the Center for the Genetics of Host Defense (https://www.utsouthwestern.edu/education/medical-school/departments/gene...) at UTSW and study co-author.

Nanoparticle Drug-Delivery System Developed to Aid Treatment of Brain Disorders/Diseases; Use in Mouse Models Results In Unprecedented Penetration of siRNA Across Intact Blood-Brain Barrier; System Could Be “Game-Changer” in Treatment of CNS Diseases

In the past few decades, researchers have identified biological pathways leading to neurodegenerative diseases and developed promising molecular agents to target them. However, the translation of these findings into clinically approved treatments has progressed at a much slower rate, in part because of the challenges scientists face in delivering therapeutics across the blood-brain barrier (BBB) and into the brain. To facilitate successful delivery of therapeutic agents to the brain, a team of bioengineers, physicians, and collaborators at Brigham and Women's Hospital and Boston Children's Hospital created a nanoparticle platform, which can facilitate therapeutically effective delivery of encapsulated agents in mice with a physically breached or intact BBB. In a mouse model of traumatic brain injury (TBI), the scientists observed that the delivery system showed three times more accumulation in brain than conventional methods of delivery and was therapeutically effective as well, which could open possibilities for the treatment of numerous neurological disorders. Findings were published online on January 1, 2021 in Science Advances. The article is titled “BBB Pathophysiology Independent Delivery of siRNA in Traumatic Brain Injury.” Previously developed approaches for delivering therapeutics into the brain after TBI rely on the short window of time after a physical injury to the head, when the BBB is temporarily breached. However, after the BBB is repaired within a few weeks, physicians lack tools for effective drug delivery. "It's very difficult to get both small and large molecule therapeutic agents delivered across the BBB," said corresponding author Nitin Joshi, PhD, an associate bioengineer at the Center for Nanomedicine in the Brigham's Department of Anesthesiology, Perioperative and Pain Medicine.