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“Spleen-on-a-Chip” Yields Insight into Sickle Cell Disease
Every day, billions of red blood cells pass through the spleen, an organ that is responsible for filtering out old or damaged blood cells. This task is made more difficult when the blood cells are misshapen, as they are in patients with sickle cell disease, which affects millions of people throughout the world. Sickled blood cells can clog the spleen’s filters, leading to a potentially life-threatening situation. Researchers at MIT, Nanyang Technological University in Singapore, the Pasteur Institute in Paris, and other institutions have now designed a microfluidic device, or “spleen-on-a-chip,” that can model how this phenomenon, known as acute splenic sequestration, arises. The researchers found that low oxygen levels make it more likely that the spleen’s filters will become clogged. They also showed that boosting oxygen levels can unclog the filters, which may help to explain how blood transfusions help patients suffering from this condition.
Preliminary Success in Gene Therapy for Inherited Blistering Skin Disease (Dystrophic Epidermolysis Bullosa) Described at Precision Medicine World Conference (PMWC 2023)
The closing presentation of Day 1, Track1, Gene & Cell Therapies, on Wednesday, January 25, at the Precision Medicine World Conference (PMWC 2023) in Santa Clara, California, featured the description of a possibly major breakthrough in gene therapy treatment for the genetic blistering skin disease known as dystrophic epidermolysis bullosa. The presentation was delivered by M. Peter Marinkovich, MD, an Associate Professor of Dermatology at Stanford, where he directs the Stanford Blistering Disease Clinic. Dr. Marinkovich’s laboratory has had a longstanding focus on the development of molecular therapy for various subtypes of epidermolysis bullosa. In a major advance, Dr. Marinkovich and colleagues published the following article in the December 15, 2022 issue of the New England Journal of Medicine: “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa.” The conclusion of this work was that complete wound healing at 3 and 6 months in patients with dystrophic epidermolysis bullosa was more likely with topical administration of B-VEC than with placebo. Pruritus and mild systemic side effects were observed in patients treated with B-VEC. Dystrophic epidermolysis bullosa is a rare genetic blistering skin disease caused by mutations in COL7A1, which encodes a protein involved in assembling type VII collagen (C7). Beremagene geperpavec (B-VEC) is a topical investigational herpes simplex virus type 1 (HSV-1)–based gene therapy designed to restore C7 protein by delivering normal COL7A1. Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease, the authors advised.
OncoHost Launches PROphet® NSCLC Cancer Test in the United States; Predictive Blood Test Guides First-Line Treatment Decisions for Advanced Unresectable Non-Small Cell Lung Cancer (NSCLC) Patients
On February 1, 2023, OncoHost, a precision diagnostics company centered on predictive biomarker development for improved patient care, announced the official launch of its PROphet® non-small cell lung cancer (NSCLC) Test in the United States. OncoHost’s first test, PROphet® NSCLC, guides first-line treatment decisions for advanced unresectable NSCLC patients. The PROphet® NSCLC Test provides clinicians with actionable clinical insights into optimal first-line therapeutic choices, and a better understanding of their patients’ personalized cancer dynamics. Requiring just one pre-treatment blood test, PROphet® scans approximately 7,000 proteins in a patient’s blood plasma and delivers a report that predicts the patient’s clinical benefit from anti-PD-1/PD-L1 immunotherapy-based treatment plans versus clinical benefit from immunotherapy plus chemotherapy.
Pangaea, Provider of AI-Driven Product for Patient Characterization, Named “Digital Solution of the Year” by the UK Government’s Department for International Trade (DIT)
On January 31, 2023, Pangaea Data, (Pangaea), provider of a novel AI-driven product for characterizing patients by mapping their journeys and disease trajectories in a privacy-preserving and scalable manner, today announced that it has been named “Digital Solution of the Year” by the UK Government’s Department for International Trade (UK DIT), as part of its 2023 UK Life Science Innovator Showcase. UK DIT selected Pangaea for this award from thousands of UK companies across the life science industry as a direct result of its international growth and innovative approach to tackling healthcare challenges. This award follows Pangaea’s selection by the UK DIT as a Leading Digital Health Innovator, and inclusion in the UK government’s Digital Health Playbook for the second consecutive year. The UK Digital Health Playbook showcases the top UK innovators and exporters, providing solutions for current challenges in digital health, including scaling the power of AI for improved diagnoses and more accurate predictions.1
Supplementation with Amino Acid Serine Eases Neuropathy in Diabetic Mice
Approximately half of people with type 1 or type 2 diabetes experience peripheral neuropathy—weakness, numbness, and pain, primarily in the hands and feet. The condition occurs when high levels of sugar circulating in the blood damage peripheral nerves. Now, working with mice, Salk Institute researchers and collaborators have identified another factor contributing to diabetes-associated peripheral neuropathy: altered amino acid metabolism. The team found that diabetic mice with low levels of two related amino acids, serine and glycine, are at higher risk for peripheral neuropathy. What’s more, the researchers were able to alleviate neuropathy symptoms in diabetic mice by supplementing their diets with serine. The study, published January 25, 2023 in Nature, adds to growing evidence that some often-underappreciated, “non-essential” amino acids play important roles in the nervous system. The findings may provide a new way to identify people at high risk for peripheral neuropathy, as well as a potential treatment option. The open-access Nature article is titled “Insulin-Regulated Serine and Lipid Metabolism Drive Peripheral Neuropathy.”
Breakthrough in Understanding of Mitochondrial Regulation
A process known as “mitophagy” is responsible for the removal of mitochondria, the energy-producing parts of a cell. This occurs if the mitochondria are defective, or to regulate their numbers. A protein anchored in the mitochondrial surface, called Atg32, promotes this process when it interacts with another protein, Atg11. Modification of Atg32 by “phosphorylation”–the attachment …
Precision Medicine World Conference (PMWC 2023) Concludes in Silicon Valley; Conference Ends with Riveting Presentation by BioTech Giant Leroy Hood
On Friday, January 27, the three-day Precision Medicine World Conference 2023 (PMWC 2023) (Jan 25-27) “Celebrating 14 Years of Precision Medicine Innovation” concluded in Santa Clara, California, the heart of Silicon Valley, with day-long sessions on six different topic tracks. These included Pharmacogenomics, AI & Data Sciences in Drug Discovery & Clinical Research, Population Scale Omics, Imaging Applications; and two showcase sessions—one on AI & Data Sciences, Emerging Therapeutics, and Genomic & Microbial Profiling, and the other on Clinical Diagnostics and Clinical & Research Tools. The day also included presentation of four PMWC Pioneer Awards and two PMWC Luminary Awards. Pioneer Awards were presented to David Bently, PhD, Dan M. Roden, MD, Lea T. Grinberg, MD, PhD, and Daniela Ushizima, PhD . Luminary Awards were presented to Gad Getz, PhD, and Kelly Caudle, Pharm.D, PhD. The meeting ended with a powerful futuristic presentation by biotech legend and visionary Leroy Hood, MD, PhD.
Second Day of Precision Medicine World Conference (PMWC 2023) in Silicon Valley
On Thursday, January 26, the three-day Precision Medicine World Conference 2023 (PMWC 2023) (Jan 25-27) “Celebrating 14 Years of Precision Medicine Innovation” continued in Santa Clara, California, the heart of Silicon Valley, with day-long sessions on six different topic tracks. These included Gene & Cell Therapies, Real World Evidence Data, Women’s Health, Clinical Utility of Liquid Biopsies; and two showcase sessions—one on AI & Data Sciences, PGx, and Health Monitoring, and the other on Clinical Diagnostics and Clinical & Research Tools. The day also included presentation of three PMWC Pioneer Awards and one PMWC Luminary Award. Pioneer Awards were presented to Marcia Stepanick, PhD, Razelle Kurzrock, MD, and Luis Diaz, MD. A Luminary Award was presented to Bruce Levine, PhD.
Biological Dynamics to Present Data Demonstrating Clinical Utility of ExoVerita™ Exosome-Isolation Platform for Detection of Early-Stage Cancers at Precision Medicine World Conference (PMWC 2023)
Biological Dynamics, a company developing and commercializing its ExoVeritaTM platform for early disease diagnostics, announced that Harmeet Dhani, MD, MSc, Medical Director will present at the Precision Medicine World Conference (PMWC 2023) in Santa Clara, California. Dr. Dhani’s PMWC presentation on Thursday, January 26th is part of the Clinical Utility of Liquid Biopsies track and will be given at 10:15 AM PST. The track focuses on how liquid biopsy technologies facilitate detection of early-stage cancer, which could help contribute to improving patient outcomes rates and reduce the need for invasive diagnostic procedures. Biological Dynamics’ ExoVerita platform targets extracellular vesicles (EVs) in blood, isolating exosomes to detect biomarkers of early-stage cancers. Its first product is an assay for pancreatic ductal adenocarcinoma (PDAC) assay that identifies exosomal PDAC-associated protein biomarkers, detecting Stage 1 and 2 pancreatic cancers with high sensitivity, a key advantage of the technology. Dr. Dhani’s presentation will discuss the implication of exosomes, the ExoVerita technology platform, review a subject case study, and provide an overview of the preliminary data in multi-cancer early detection and the ExoLuminate registry trial (NCT05625529) which enrolled its first subject in December.