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June 5th, 2020

New Study Identifies Approved Anti-Cancer Drug (BTK-Inhibitor Acalabrutinib) As Having Potential in Treatment of Patients with Severe COVID-19; Drug Reduces Respiratory Distress and Overactive Immune Response

Early data from a clinical study suggest that blocking the Bruton tyrosine kinase (BTK) enzyme (image) provided clinical benefit to a small group of patients with severe COVID-19. Researchers observed that the off-label use of the cancer drug acalabrutinib, a BTK inhibitor that is approved to treat several blood cancers, was associated with reduced respiratory distress and a reduction in the overactive immune response in most of the treated patients. The findings were published in the June 5, 2020 issue of Science Immunology. The open-access article is titled “Inhibition of Bruton Tyrosine Kinase in Patients with Severe COVID-19.” The study was led by researchers in the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), in collaboration with researchers from the National Institute of Allergy and Infectious Diseases (NIAID), both part of the National Institutes of Health (NIH), as well as from the U.S. Department of Defense's Walter Reed National Military Medical Center, and four other hospitals nationally. These findings should not be considered clinical advice, but are being shared to assist the public health response to COVID-19. While BTK inhibitors are approved to treat certain cancers, they are not approved as a treatment for COVID-19. This strategy must be tested in a randomized, controlled clinical trial in order to understand the best and safest treatment options for patients with severe COVID-19. The BTK enzyme ( plays an important role in the normal immune system, including in macrophages, a type of innate immune cell that can cause inflammation by producing proteins known as cytokines. Cytokines act as chemical messengers that help to stimulate and direct the immune response.

June 4th

Incyte Announces Initiation, with Novartis, of Phase 3 RUXCOVID Study Evaluating JAK1/JAK2 Inhibitor Ruxolitinib (Jakafi®/Jakavi®) As a Treatment for Patients with COVID-19-Associated Cytokine Storm

On April 17, 2020, Incyte (Nasdaq:INCY) announced the initiation of RUXCOVID, a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of ruxolitinib (Jakafi®) plus standard-of-care (SoC) in patients aged ≥12 years with COVID-19-associated cytokine storm. The collaborative study is sponsored by Incyte in the United States and Novartis outside of the United States. The composite primary endpoint is the proportion of patients who die, develop respiratory failure (require mechanical ventilation), or require intensive care unit (ICU) care by Day 29. Secondary endpoints comprise various efficacy assessments, including evaluation of clinical status using a 9-point ordinal scale; in-hospital outcomes (mortality rate; proportion of patients requiring mechanical ventilation; duration of hospitalization, ICU stay, supplemental oxygen, invasive mechanical ventilation); change in the National Early Warning Score (NEWS2); change in SpO2/FiO2 ratio; proportion of patients with no oxygen therapy (oxygen saturation of ≥94% on room air); and safety. RUXCOVID will enroll approximately 400 patients globally. Additionally, given the urgent nature of the COVID-19 pandemic, Incyte is also initiating a separate emergency Expanded Access Program (EAP) in the United States. The protocol will allow eligible patients with severe COVID-19-associated cytokine storm to receive ruxolitinib while it is being investigated for this indication. “There is an unprecedented unmet medical need for treatments that prevent or reduce severe COVID-19-related complications to improve outcomes for patients and alleviate the overwhelming pressure on the global healthcare system,” said Steven Stein, MD, Chief Medical Officer, Incyte.

Immune Disease (HLH) Research at Cincinnati Children's Hospital Points to Possible Treatment for COVID-19 "Cytokine Storms"-- Anti-Inflammatory Drug (Ruxolitinib) Used in HLH, Shows Possible Effectiveness in Calming “Cytokine Storms” in COVID-19

A transgenic mouse developed at Cincinnati Children's Hospital Medical Center to model the deadly childhood immune disease HLH (hemophagocytic lymphohistiocytosis) ( ) ( ) may play a key role in saving lives during the COVID-19 virus pandemic. One of the genetically engineered mouse strain's inventors--Cincinnati Children's cancer pathologist Gang Huang, PhD-- is co-investigator on a small clinical trial that successfully tested a drug (ruxolitinib), already used to treat HLH, that dramatically reversed respiratory and multi-system inflammation in severely ill COVID-19 patients. Data from the Phase II clinical study was published online on May 26, 2020 in the Journal of Allergy and Clinical Immunology ( The articl is titled "Ruxolitinib in Treatment of Severe Coronavirus Disease 2019 (COVID-19): A Multicenter, Single-Blind, Randomized Controlled trial." The study involved 43 hospitalized patients diagnosed with severe COVID-19 between February 9 and February 28 in Wuhan, China, believed to be ground zero for the pandemic. The multi-center study was led by Jianfeng Zhou, MD, PhD, Department of Hematology at Tongji Hospital, Tongji Medical College, Huazhong University of Science in Wuhan. Dr. Zhou is a longtime collaborator of Dr. Huang and colleagues at the Cincinnati Children's HLH Center of Excellence, part of the hospital’s Cancer and Blood Diseases Institute.

June 3rd

“Outstanding” Phase III Results for New Immunotherapeutic Agent (Rituximab) for Children and Adolescents with Advanced Non-Hodgkin Lymphoma Published in NEJM

The excellent results of the Phase III international pediatric study, Inter-B-NHL ritux 2010, have been published in the June 4, 2020 issue of the New England Journal of Medicine. The article is titled” Rituximab for High-Risk, Mature B-Cell Non-Hodgkin’s Lymphoma in Children.” This academic trial involved two international cooperative groups: the European Intergroup for Childhood Non-Hodgkin Lymphoma (EICNHL) and the Children's Oncology Group (COG). The trial sponsors were the Comprehensive Cancer Centre Gustave Roussy (for countries in Europe and Asia) and COG (for Australia, Canada, and the United States) and included a partnership with Roche. The trial establishes a new standard treatment with an improved cure rate for children with advanced non-Hodgkin lymphoma, mainly Burkitt lymphoma. It supports the value of an immunotherapeutic agent, which was authorized in March 2020 by the European Commission for the treatment of a rare childhood cancer. "With a three-year survival rate exceeding 95%, these results are outstanding. This study changes the international treatment bench-mark in young patients with advanced B-cell non-Hodgkin lymphoma,” declared Dr. Véronique Minard-Colin, pediatrician at the Gustave Roussy Department of Child and Adolescent Oncology in France, who coordinated this major international trial with Dr. Thomas G. Gross currently at Children's Hospital Colorado in the United States. The management of children with Burkitt lymphoma has improved considerably over recent decades. Cure rates have risen from 30% in the 1980s to higher than 85% with chemotherapy alone (LMB/Laboratory of Molecular Biology protocol) with no major late sequelae associated with the medication or the disease. This conventional LMB treatment was established more than 30 years ago by Dr.

Bruton’s Tyrosine Kinase (BTK) Inhibitors Could Prevent Anaphylaxis in People With Food, Drug Allergies; Drug Would Be First Known Treatment to Prevent Anaphylaxis

For someone with a food or drug allergy, the risk of life-threatening anaphylactic shock lurks around every corner. A new Northwestern Medicine study shows there might be a pill that can be taken proactively to prevent mild to life-threatening anaphylaxis, no matter the cause. Results of the new study were published online on June 2, 2020 in the Journal of Clinical Investigation. The article is titled “Bruton’s Tyrosine Kinase Inhibition Effectively Protects Against Human IgE-Mediated Anaphylaxis.” Anaphylaxis ( is a severe, potentially life-threatening systemic allergic reaction that can occur within seconds or minutes of exposure to an allergen. It occurs in about 1 in 50 Americans, though many believe the rate is higher (closer to 1 in 20), according to the Asthma and Allergy Foundation of America. If a person's blood pressure drops so low during anaphylaxis or their airway closes up enough that they can't get enough oxygen to their organs, they enter anaphylactic shock. The drugs used in this study are known as BTK inhibitors. BTK stands for an enzyme called Bruton's tyrosine kinase (image), which is found inside cells, including mast cells. The reason BTK inhibitors work to block allergic reactions is that by inhibiting, or blocking, the BTK enzyme, the mast cells cannot be triggered by allergens and allergic antibody to release histamine and other allergic mediators. The study used three different BTK inhibitors, which blocked allergic reactions when tested on human mast cells in a test tube. Additionally, the study used one U.S.

Ionis and Roche Announce Enrollment Completion of Global Phase 3 Trial of Anti-Sense Drug (Tominersen) for Treatment of Huntington's Disease

On April 20, 2020, Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced that its partner Roche, also known as Genentech in the United States, has completed enrollment for GENERATION HD1, a global Phase 3 study evaluating the efficacy and safety of tominersen (previously IONIS-HTTRx or RG6042), an investigational anti-sense therapy for people living with Huntington's disease (HD). "Completion of the enrollment of this Phase 3 study is an important landmark for the clinical development of tominersen and for families affected by Huntington's disease. While there is much work ahead of us, we are now closer to potentially providing a treatment for people living with this devastating disease. We are grateful to Huntington's disease patients, their families, and healthcare providers for their courage and resilience, particularly in the current challenging environment," said Brett P. Monia, PhD, Ionis' Chief Executive Officer. "At Ionis, knowing that sick people depend on us fuels our passion for discovering and delivering novel anti-sense medicines like tominersen, the first and only therapy in pivotal trials targeting the underlying cause of HD." GENERATION HD1 is evaluating the efficacy and safety of tominersen treatment administered once every two months (eight weeks) or every four months (16 weeks) over a period of 25 months, compared to placebo. The study has completed enrollment with 791 patients across approximately 100 sites around the world. HD is a devastating, and ultimately fatal, hereditary disease resulting in deterioration in mental abilities and physical control. Currently, there is no approved disease-modifying treatment for HD. There are approximately 3 to 10 per 100,000 people worldwide affected by HD. In the U.S.

First Results from Rockefeller Study of Convalescent Plasma from 149 COVID-19 Survivors Reveal 40 Neutralizing Antibodies from the 1% "Elite" Responders; Of These, Three Most Highly Potent Are Selected, Cloned, and Now Being Prepared for Clinical Use

In a May 25, 2020 release, The Rockefeller University announced that the first round of results from an immunological study of 149 people who have recovered from COVID-19 show that, although the amount of antibodies they generated varies widely, most individuals had generated at least some antibodies that were intrinsically capable of neutralizing the SARS-CoV-2 virus. Antibodies vary widely in their efficacy. While many may latch on to the virus, only some are truly “neutralizing,” meaning that they bind to the virus at a position that blocks it from entering the cells. Since April 1, 2020, a team of immunologists, medical scientists, and virologists, has been collecting blood samples from volunteers who have recovered from COVID-19. The majority of the samples the scientist have studied showed poor to modest “neutralizing activity,” indicating a weak antibody response. However, a closer look revealed everyone’s immune system is capable of generating effective antibodies—just not necessarily enough of them. Even when neutralizing antibodies were not present in an individual’s serum in large quantities, researchers could find some rare immune cells that make them. “This suggests just about everybody can do this, which is very good news for vaccines,” says Michel C. Nussenzweig, MD, PhD, Head of the Laboratory of Molecular Immunology at The Rockefeller University. “It means if you were able to create a vaccine that elicits these particular antibodies, then the vaccine is likely to be effective and work for a lot of people.” Moreover, the researchers identified three distinct antibodies that were shown to be the most potent, of all those tested, in neutralizing the virus. The scientists are working to develop these three antibodies further into therapeutic and preventive drugs.

Collection of Key Lung Cancer Articles from the Pioneering TRACERx Study Provides Insights; Surrounding Environment & Immune System Are Keys to Understanding Cancer Evolution

A collection of nine papers ( published in 2020 and 2019 in various Nature journals and presented as a single “TRACERx” collection online on May 27, 2020 in a special section of Nature has transformed our understanding of how lung cancer evolves over time, in particular how the surrounding environment and immune system drives changes. The collective findings from the pioneering TRACERx (Tracking Cancer Evolution Through Therapy) study ( have already changed the way researchers and clinicians view lung cancer, leading to new clinical trials and research projects aimed at tackling these hard-to-treat tumors. The process of cancer evolution closely mirrors the way that species evolve through natural selection. As lung cancer cells multiply, mutations take place in their DNA, which can help, harm, or have no effect. A helpful mutation may make the cell resistant to certain treatments or able to more quickly digest the nutrients it needs to divide. But observing the product of this evolution only provides part of the picture. For example, in order to understand the variety of different finches on the Galapagos Islands, Charles Darwin needed to consider the different environments in which each of the finches lived. The same is true for studying cancer evolution. One needs to understand the surrounding environment, i.e., the body, to understand why and how a tumor is going to evolve. This complex challenge is being tackled by the TRACERx consortium, led by researchers at the Francis Crick Institute and the University College London (UCL), and funded by Cancer Research UK. Findings from the first 100 patients studied have now been brought together in this special Nature collection.

Optimal Time to Treat Huntington’s Disease ID'd; Abnormally High Levels of Neurofilament Light (NfL) Biomarker Detectable 24 Years Before Onset of Clinical Symptoms; Marker Could Be Used to Monitor & Gauge Effectiveness of Future Treatments

The earliest brain changes due to Huntington’s disease can be detected 24 years before clinical symptoms show, according to a new University College London (UCL)-led study. The researchers say their findings, published in the June 1, 2020 issue of The Lancet Neurology (, could help with clinical trials by pinpointing the optimal time to begin treating the disease. The open-access article is titled “Biological and Clinical Characteristics of Gene Carriers Far from Predicted Onset in the Huntington's Disease Young Adult Study (HD-YAS): A Cross-Sectional Analysis.” There is currently no cure for Huntington’s, a hereditary neurodegenerative disease, but recent advances in genetic therapies hold great promise. Researchers would ultimately like to treat people before the genetic mutation has caused any functional impairment. However, until now, it was unknown when the first signs of damage emerge--but because there is a genetic test for Huntington’s susceptibility, researchers have a unique opportunity to study the disease before symptoms appear. Professor Sarah Tabrizi (photo), MD, PhD, ( (UCL Huntington’s Disease Centre, UCL Queen Square Institute of Neurology), the study lead, said: “Ultimately, our goal is to deliver the right drug at the right time to effectively treat this disease – ideally we would like to delay or prevent neurodegeneration while function is still intact, giving gene carriers many more years of life without impairment.” “As the field makes great strides with the drug development, these findings provide vital new insights informing the best time to initiate treatments in the future, and represent a significant advance in our understanding of early Huntington’s.”

June 2nd

Cancer Cells Cause Inflammation to Protect Themselves from Oncolytic Viruses

Researchers at the Francis Crick Institute in the UK have uncovered how cancer cells protect themselves from oncolytic viruses that are harmful to tumors, but not to healthy cells. These findings could lead to improved viral treatments for cancers. In their study, published online on June 1, 2020 in Nature Cell Biology (, the researchers identified a mechanism that protects cancer cells from oncolytic viruses, which preferentially infect and kill cancer cells. The article is titled “Cancer Cells Cause Inflammation to Protect Themselves from Viruses.” These oncolytic viruses are sometimes used as a treatment to destroy cancer cells and stimulate an immune response against the tumor. However, they only work in a minority of patients and the reasons why they are effective or not are not yet fully understood. The research team examined the environment surrounding a tumor and how cancer cells interact with their neighbors, in particular, with cancer-associated fibroblasts (CAFs) (, which researchers know play a significant role in cancer protection, growth, and spread. The researchers found that when cancer cells are in direct contact with CAFs, this leads to inflammation that can alert the surrounding tissue, making it harder for viruses to invade and replicate within the cancer cell. This protective inflammatory response occurs when cancer cells pass small amounts of their cytoplasm through to the CAFs. This triggers the fibroblasts to signal to nearby cells to release cytokines, molecules that cause inflammation.