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Archive - 2021 - Story


April 13th

TGen Identifies Gene That Might Be Targeted to Possibly Help Prevent or Delay Onset of Alzheimer's Disease; Boosting ABCC1 Might Lessen Production of Plaque Linked to Alzheimer's Development

Findings of a study by the Translational Genomics Research Institute (TGen), an affiliate of City of Hope, suggest that increasing expression of a gene known as ABCC1 could not only reduce the deposition of a hard plaque in the brain that leads to Alzheimer's disease, but might also prevent or delay this memory-robbing disease from developing. ABCC1, also known as MRP1, has previously been shown in laboratory models to remove a plaque-forming protein known as amyloid beta (Abeta) from specialized endothelial cells that surround and protect the brain and cerebral spinal cord. Building on previous studies, TGen conducted a series of pre-clinical genomic laboratory experiments. Results suggest that ABCC1 not only could export Abeta out of the brain, but that increasing the expression of ABCC1 could reduce Abeta production, thus preventing, or delaying, the onset of Alzheimer's. The findings were published online on January 25, 2021 in Biology Open in an open-access article titled “Adenosine Triphosphate Binding Cassette Subfamily C Member 1 (ABCC1) Overexpression Reduces APP Processing and Increases Alpha- Versus Beta-Secretase Activity, In Vitro” ( "Much work remains toward developing a drug that slows the development of or prevents Alzheimer's disease, but our findings suggest that targeting ABCC1 offers a promising path that could eventually lead to effective therapeutics," said Wayne Jepsen, PhD, a Postdoctoral Fellow in TGen's Neurogenomics Division, and the study's lead author. Alzheimer's disease is the sixth leading cause of death in the U.S., annually killing more than 120,000 people. There is no treatment that can effectively prevent or slow this disease. An estimated 5.8 million Americans age 65 or older have Alzheimer's, and that number is expected to more than double over the next 30 years.

April 13th

Novocure Announces Positive Update on Phase 3 Pivotal LUNAR Trial of Tumor Treating Fields (TTFields) in Non-Small-Cell Lung Cancer (NSCLC)

On April 13, 2021, Novocure (NASDAQ: NVCR) announced an update regarding its phase 3 pivotal LUNAR trial of Tumor Treating Fields (TTFields) in stage 4 non-small-cell lung cancer (NSCLC) following platinum failure. Following a routine review of the study by an independent data monitoring committee (DMC), Novocure was informed that the pre-specified interim analysis for the LUNAR trial would be accelerated given the length of accrual and the number of events observed, to date. The interim analysis included data from 210 patients accrued to the LUNAR trial through February 2021. After review of the interim analysis report, the DMC concluded that the LUNAR trial should continue with no evidence of increased systemic toxicity. The DMC also stated that it is likely unnecessary and possibly unethical for patients randomized to the control arm to continue accrual to 534 patients with 18 months follow-up. The DMC recommended a reduced sample size of approximately 276 patients with 12 months follow-up which it believes will provide sufficient overall power for both primary and secondary endpoints. The DMC recommended no other changes to the design of the trial. Novocure remains blinded to all data. The primary endpoint of the LUNAR trial is superior overall survival when patients are treated with TTFields plus immune checkpoint inhibitors or docetaxel versus immune checkpoint inhibitors or docetaxel alone. The final analysis will also include an analysis of overall survival in the immune checkpoint inhibitor and docetaxel treatment subgroups. Novocure has notified the U.S. Food and Drug Administration (FDA) of the DMC recommendations and of its intent to submit an Investigational Device Exemption (IDE) supplement incorporating the recommended protocol adjustments.

Methylation of RNA May Play Key Role in Autosomal Dominant Polycystic Kidney Disease (ADPKD); New Findings Suggest Dietary Modification & Targeting Methylating Enzyme May Be Two Ways to Control Common, Potentially Fatal Genetic Disorder

A chemical modification of RNA that can be influenced by diet appears to play a key role in autosomal dominant polycystic kidney disease (ADPKD), an inherited disorder that is the fourth leading cause of kidney failure in the U.S., University of Texas Southwestern (UTSW) researchers report in a new study. The findings, published online on April 13, 2021 in Cell Metabolism, suggest possible new ways to treat this incurable condition. The article is titled “A Methionine-Mettl3-N6-Methyladenosine Axis Promotes Polycystic Kidney Disease” ( Approximately 600,000 Americans and 12.5 million people worldwide have ADPKD, a condition caused by mutations in either of two genes, PKD1 or PKD2. These mutations cause kidney tubules (small tubes that filter blood and generate urine) to dilate, forming cysts that grossly enlarge the kidneys. In approximately 50 percent of patients, these cysts eventually cause kidney failure, necessitating dialysis or a kidney transplant. (Image at left shows enormous polycystic kidney versus normal kidney). Although one FDA-approved drug exists to treat ADPKD, it merely slows the decline in kidney function, explain study leaders Vishal Patel, MD, Associate Professor of Internal Medicine at UTSW, and Harini Ramalingam, PhD, a postdoctoral fellow in Dr. Patel's lab. More treatments for this condition are urgently needed, they say, but the molecular mechanisms that cause ADPKD to develop and progress are still not fully known. To better understand this condition, Dr. Patel, Dr. Ramalingam, and their colleagues investigated whether chemical modifications to the genetic molecule RNA, which translates instructions from DNA to produce proteins in the body, could play a part.

How “Imprinting” on Some Smells by Newborn Mice Affects Adult Social Behaviors; Study Sheds Light on Neuro-Developmental Disorders Such As Autism Spectrum Disorders, Suggests More Effective Use of Oxytocin Therapy for Such Disorders at Early Age

The smells that newborn mice are exposed to (or “imprint” on to use the academic term) affect many social behaviors later in life, but how this happens is still a mystery. Scientists from Japan have now discovered three molecules necessary for imprinting. Their new study sheds light on the decision-making process and neurodevelopmental disorders such as autism spectrum disorders (ASDs). It also proposes more effective use of oxytocin therapy for such disorders at an early age. Imprinting is a popularly known phenomenon, wherein certain animals and birds become fixated on sights and smells they experience immediately after being born. In ducklings, this can be the first moving object, usually the mother duck. In migrating fish like salmon and trout, it is the smells they knew as neonates that guides them back to their home river as adults. How does this happen? Exposure to environmental input during a critical period early in life is important for forming sensory maps and neural circuits in the brain. In mammals, early exposure to environmental inputs, as in the case of imprinting, is known to affect perception and social behavior later in life. Visual imprinting has been widely studied, but the neurological workings of smell-based or “olfactory” imprinting remain a mystery. To find out more, scientists from Japan, including Drs. Nobuko Inoue, Hirofumi Nishizumi, and Hitoshi Sakano at the University of Fukui and Drs. Kazutaka Mogi and Takefumi Kikusui at Azabu University, worked on understanding the mechanism of olfactory imprinting during the critical period in mice. Their study, published online on March 5, 2021 in eLife, offers fascinating results. The article is titled “The Olfactory Critical Period is Determined by Activity-Dependent Sema7A/PlxnC1 Signaling within Glomeruli” (

Study Suggests That Difficulty of Pecking Hole in Parasitic Egg for Gripping Purposes May Explain Why Most Host Birds Fail to Eject Foreign Eggs from Nest

Avian brood parasites lay their eggs in the nests of other bird species, forcing the hosts to do the hard work of raising the unrelated young. A team of scientists wanted to simulate the task of piercing an egg – a tactic that only a minority of host birds use to help grasp and eject the foreign eggs. Their study offers insight into some of the physical challenges the discriminating host birds face. The new findings were published on March 5, 2021 in the Journal of Experimental Biology. The article is titled “Nest Substrate and Tool Shape Significantly Affect The Mechanics and Energy Requirements of Avian Eggshell Puncture” ( Take cowbirds, for example. Their eggs look nothing like the host birds’ eggs, “yet most of their hosts do not reject the parasite eggs,” said study co-author Mark Hauber (, PhD, Professor of Host-Parasite Interaction in the Department of Evolution, Ecology, and Behavior at the University of Indiana and a brood parasitism expert. “One explanation is that the cowbird eggshell is too thick and strong for a small host’s beak to pierce.” To determine whether the difficulty of piercing a brood parasite’s egg played a role in whether the host bird tried to eject it, Daniel Clark, an undergraduate student working in Hauber’s laboratory, teamed up with an Assistant Professor in the same department, Philip Anderson, PhD, an expert in the biomechanics of piercing, slashing, and stabbing. Dr. Anderson has previously studied the characteristics that contribute to the cutting and crushing ability of teeth and the piercing power of viper fangs ( and cactus spines (

Researchers Develop Promising Blood Test for Depression & Bipolar Disorder; May Open Door to Precise, Personalized Matching with Medications, and Objective Monitoring of Response to Treatment

Worldwide, 1 in 4 people will suffer from a depressive episode in their lifetime. While current diagnosis and treatment approaches are largely trial and error, a breakthrough study by Indiana University (IU) School of Medicine researchers sheds new light on the biological basis of mood disorders, and offers a promising blood test aimed at a precision medicine approach to treatment. Led by Alexander B. Niculescu, MD, PhD, Professor of Psychiatry at IU School of Medicine, the study was published online on April 8, 2021 in Molecular Psychiatry. The work builds on previous research conducted by Dr. Niculescu and his colleagues into blood biomarkers (focused on gene expression from immune cells) that track suicidality, as well as pain, post-traumatic stress disorder, and Alzheimer's disease. The ability to identify peripheral gene expression changes that reflect brain activities is likely due to the fact that the brain and immune system have developmental commonalities, marked by shared reactivity and ensuing gene expression patterns, the authors stated. The open-access Molecular Psychiatry article is titled “Precision Medicine for Mood Disorders: Objective Assessment, Risk Prediction, Pharmacogenomics, and Repurposed Drugs” ( "We have pioneered the area of precision medicine in psychiatry over the last two decades, particularly over the last 10 years. This study represents a current state-of-the-art outcome of our efforts," said Dr. Niculescu. "This is part of our effort to bring psychiatry from the 19th century into the 21st century; to help it become like other contemporary fields such as oncology.

April 12th

Cancer-Killing Virus Therapy Shows Promise Against Inoperable Advanced Melanoma

Early results show that a new combination drug therapy is safe and effective against advanced melanoma in patients who were not able to have their tumors surgically removed. The drug combination is among the first, researchers say, to demonstrate the potential value of a live common cold virus, a coxsackievirus, to infect and kill cancer cells. The Phase I study, led by a researcher at New York University (NYU) Langone Health and its Perlmutter Cancer Center, is also among the first to show how such oncolytic viruses can safely boost the action of widely used cancer therapies that help the body's immune defense system detect and kill cancer cells. Currently, such immunotherapies are only effective in shrinking melanoma tumors in just over a third of patients who receive them. The new study results showed that injections of experimental coxsackievirus drug V937, along with pembrolizumab, an immunotherapy drug known as pembro or Keytruda, were well tolerated. Moreover, the combined treatment shrank melanoma tumors in nearly half (47 percent) of 36 men and women who received the therapy every few weeks for at least two years. Researchers say most side effects, such as rash and fatigue, were minimal, while 13 patients (36 percent) had serious immune reactions in the liver, stomach, or lungs, not unlike side effects that are known to happen with pembrolizumab alone. Presented on April 10 at the virtual annual meeting of the American Association for Cancer Research(AACR) 2021 (Week 1: April 9-14; Week 2: May 17-21) (, the study also showed that eight patients who received both drugs (22 percent) experienced complete remission, with no remaining signs of skin cancer.

Yale Cancer Center Study Demonstrates Promise for Novel Immunotherapy Approach to Fight Melanoma

In a new study led by the Yale Cancer Center, researchers have advanced a tumor-targeting and cell-penetrating antibody that can deliver payloads to stimulate an immune response to help treat melanoma. The study was presented on April 11, 2021 at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 (Week 1: April 9-14; Week 2: May 17-21) ( "Most approaches rely on direct injection into tumors of ribonucleic acids (RNAs) or other molecules to boost the immune response, but this is not practical in the clinic, especially for patients with advanced cancer," said Peter M. Glazer, MD, PhD, Chair of the Department of Therapeutic Radiology at Yale, Chief of Radiation Oncology at Smilow Cancer Hospital, and senior author of the study. "In this study, we can deliver immune stimulatory RNA to tumors in vivo following systemic administration." RNA is a nucleic acid present in all living cells. Its principal role is to act as a messenger to carry instructions from DNA to control the synthesis of proteins, although, in some viruses, RNA rather than DNA carries the genetic information. In this study, using mice with melanoma tumors, members of the Glazer lab at Yale achieved almost complete tumor suppression upon intravenous injection of antibody/RNA complexes. "These results are very encouraging," added Dr. Glazer.

Codiak BioSciences Presents Data at AACR 2021 Demonstrating Potential of Engineered Exosomes to Enhance the Therapeutic Index of Well-Validated Cancer Immunotherapy Pathways

On April 10, 2021, Codiak BioSciences, Inc. (Nasdaq: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, today reported new preclinical evidence from Codiak’s exoASO-STAT6 program and clinical results from the healthy volunteer portion of the ongoing Phase 1 trial of Codiak’s exoIL-12 program at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 (Week 1: April 9-14; Week 2: May 17-21) ( These data illustrate the potential of engineered exosomes to target previously undruggable, but well-validated, pathways in cancer immunotherapy and generate potent single-agent activity. “We now have a growing body of preclinical and clinical evidence across our pipeline programs demonstrating that engineering exosomes to deliver potent drug molecules enhances the therapeutic index of pathways known to drive the immune response to fighting tumors,” said Douglas E. Williams, PhD, President and Chief Executive Officer of Codiak. “In particular, data from multiple in vitro and in vivo studies of engineered exosomes incorporating an antisense oligonucleotide demonstrate potent single-agent and highly selective genetic reprogramming of tumor associated macrophages, which is unique among macrophage targeting strategies.

April 6th

Exosome-Coated Stent Heals Vascular Injury, Repairs Damaged Tissue

Researchers from North Carolina State (NC State) University have developed an exosome-coated stent with a “smart-release” trigger that could both prevent reopened blood vessels from narrowing and deliver regenerative stem cell-derived therapy to blood-starved, or ischemic, tissue. Angioplasty--a procedure that opens blocked arteries--often involves placing a metal stent to reinforce arterial walls and prevent them from collapsing once the blockage is removed. However, the stent’s placement usually causes some injury to the blood vessel wall, which stimulates smooth muscle cells to proliferate and migrate to the site in an attempt to repair the injury. The result is restenosis: a re-narrowing of the blood vessel previously opened by angioplasty. “The inflammatory response that stents cause can decrease their benefit,” says Ke Cheng, PhD, corresponding author of the research. “Ideally, if we could stop smooth muscle cells from over-reacting and proliferating, but recruit endothelial cells to cover the stent, it would mitigate the inflammatory response and prevent restenosis.” Dr. Cheng is the Randall B. Terry Jr. Distinguished Professor in Regenerative Medicine at NC State and a Professor in the NC State/UNC-Chapel Hill Joint Department of Biomedical Engineering. There are drug-eluting stents currently in use coated with drugs that discourage cell proliferation, but these anti-proliferative drugs also delay stent coverage by endothelial cells--which are the cells healthcare providers want to coat the stent. To solve this problem, Dr. Cheng and his team developed a stent coating composed of exosomes derived from mesenchymal stem cells (MSCs). Exosomes are tiny nano-sized vesicles secreted by all cell types that have been studied.