November 30, 2022 | A newly developed immunotherapy drug that delays the onset of type 1 diabetes in at-risk individuals by an average of almost three years; a study to engineer a human heart model, elucidate fundamental laws of muscular pumps, and apply lessons learned to a cardiovascular bio digital twin model; a highly flexible, modular patent-pending platform and wearable sensor designed to meet the specific needs of clinical trials and remote patient research; and more. 

The International Vaccine Institute (IVI) and ST Pharm exchanged a memorandum of understanding to collaborate in the clinical development of an mRNA COVID-19 vaccine in efforts to promote Korea’s WHO Global Training Hub for Biomanufacturing strategy. Specifically, IVI and ST Pharm will work together in defining a clinical development plan and regulatory process for ST Pharm’s pan-coronavirus vaccine candidate. The two organizations have also agreed to cooperate in training the biomanufacturing workforce in low- and middle-income countries. IVI has supported several companies from Korea and overseas to accelerate preclinical and clinical development—including clinical sample analysis—of various vaccine candidates in different stages, including mRNA vaccines. Press release. 

The UC San Diego Sanford Stem Cell Institute will lead its first launch of hematopoietic stem cells into space. This launch will be the third time stem cells were sent onto the International Space Station as part of UC San Diego’s Integrated Space Stem Cell Orbital Research Center, established in collaboration with the National Aeronautics and Space Administration. Researchers at the Sanford Stem Cell Institute will use space as an “aging accelerator,” first confirming that the microgravity environment accurately mimics human aging and then using that to dissect the aging process further. The study will inform scientists’ and clinicians’ understanding of stem cell aging without relying on lengthy and expensive clinical trials monitoring Earthbound humans as they age. Future launches will test additional features of stem cell biology and explore aging in other tissue types, including the liver and the brain. Press release. 

Researchers at Indiana University (IU) School of Medicine celebrated the Federal Drug Administration's approval of teplizumab. This new immunotherapy drug delays the onset of type 1 diabetes in at-risk individuals by an average of almost three years. IU School of Medicine was one of 28 sites that participated in the original teplizumab study conducted by TrialNet, the largest clinical trial network assembled to discover ways to delay and prevent type 1 diabetes. The newest findings show high-risk individuals treated with Teplizumab experienced a median delay of diagnosis by 2.7 years versus the placebo group and showed improved rates of insulin production, despite exhibiting insulin loss over time before treatment. In response to teplizumab’s approval, Riley Hospital for Children at Indiana University Health has opened a new clinic for individuals with a high risk of developing type 1 diabetes. Press release. 

The newly launched study, called the SuperAgers Family Study, is the most ambitious study ever conducted to uncover and understand the genetic and biological mysteries of exceptional longevity and healthy aging. The study will collect the DNA samples and health histories from as many as 10,000 people who celebrated their 95th birthdays and beyond, along with their children. The study will be administered at Albert Einstein College of Medicine through the support of the multi-year Super Agers Initiative led by AFAR in collaboration with Boston University’s School of Medicine and Einstein. The biobank holding the DNA records, and all the related data, will be protected and maintained at Einstein. Future researchers can apply for permission to use the specimens and information for new studies to prevent, diagnose, or treat diseases. Press release. 

NTT Research announced that its Medical & Health Informatics (MEI) Lab had entered a three-year joint research agreement with the Harvard John A. Paulson School of Engineering and Applied Sciences (SEAS). NTT Research scientists will work with the Disease Biophysics Group at SEAS to engineer a human heart model, elucidate fundamental laws of muscular pumps, and apply lessons learned to a cardiovascular (CV) bio digital twin model. The MEI Lab developed bioelectrodes as the interfaces with cells and tissues to accelerate the development of an in vitro CV bio digital twin. The MEI Lab has also undertaken joint research with the Technical University of Munich and the National Cerebral and Cardiovascular Center in Osaka, Japan, to work on multi-scale precision cardiology platforms and heart-on-a-chip technology aimed at developing the infrastructure for a digital replica of an individual’s heart. Press release. 

GlycoNet is pleased to announce that one of its five start-up companies, GlyCa Biosciences, has collaborated with Huron Innovation to advance clinical research for a new blood test to improve early detection and diagnosis of clinically significant prostate cancer in patients, with the primary goal of minimizing unnecessary invasive tissue biopsies. With support from GlycoNet, GlyCa Biosciences has been developing blood tests focused on prostate cancer detection since 2018. Huron Innovation co-developed four pieces of technology with GlyCa Biosciences, forming the foundation of the blood tests to be evaluated in the clinical trial. Press release. 

Shimmer Research announced the launch of its Verisense Digital Health Panel (DHP) to provide clinical researchers with access to raw, real-world, digital health ground-truth sensor data. These data will be combined with patient-reported outcomes and real-world data to provide a complete longitudinal view of participant health. Shimmer selected Schlesinger Clinical Research as its panel management partner for the Verisense DHP. Participants will initially be equipped with Verisense wearable sensors but other third-party devices that can contribute raw data to be added later. Verisense is a highly flexible, modular patent-pending platform designed from the ground up to meet the specific needs of clinical trials and remote patient research. As a result, it places a minimum burden on all the research stakeholders—sponsors, site staff, and participants. In addition, Verisense sensors are versatile and dependable, providing 24/7 access to high-quality raw data. Press release. 

The Simon Foundation is committing $250 million over the next 10 years to fund new neuroscience collaborations. The collaborations will focus on cutting-edge idea-generating research that focuses on basic principles of brain function. The foundation is particularly interested in research overlooked or deemed too risky by other funding organizations. Researchers with an idea for such an innovative collaboration should submit a vision statement by March 8, 2023. Such submissions should outline the main idea and related hypotheses the proposed partnership will address, including high-level overviews of the methods and approaches used. The foundation will prioritize cross-disciplinary collaborations integrating many levels of analysis, methodologies, ways of thinking, and scientific communities. Press release. 

The Lupus Research Alliance and its affiliate Lupus Therapeutics showed (Abstract #2245) the effectiveness of a pilot peer education program, the Patient Advocates for Lupus Studies, in increasing knowledge, perception, and willingness among people with Systemic Lupus Erythematosus to participate in clinical trials. Piloted at five academic medical centers belonging to Lupus Therapeutics’ Lupus Clinical Investigators Network, the peer-to-peer clinical trial education program was co-designed with lupus patients to improve clinical trial awareness, knowledge, and enrollment, with a focus on ensuring diverse representation in lupus clinical trials. The analysis will detail what drives the two measurement tools to produce similar and divergent results. The goal is to better understand the fundamentals underlying the endpoints so that they can be improved and employed in lupus clinical trials. Press release. 

Exscientia and The University of Texas MD Anderson Cancer Center announced a strategic collaboration to advance novel small-molecule oncology therapies. The research collaboration will use Exscientia’s precision medicine platform to identify novel anti-cancer, cell-intrinsic small-molecule compounds based on jointly identified therapeutic targets. Promising candidates will advance for further development with the team at MD Anderson’s Therapeutics Discovery division. The collaborators anticipate that successful target discovery programs may be moved into proof-of-concept clinical trials at MD Anderson. In addition, Exscientia will collaborate with the team at IACS, a core component of MD Anderson’s Therapeutics Discovery division, and an integrated team of researchers, physicians, and drug development experts working to advance impactful new therapies. Press release. 

The European Union approved the first vaccine against Respiratory Syncytial Virus (RSV), and several vaccines are in development; a solution for babies with RSV is near. The measures that protected from COVID-19 also helped to limit the spread of other viruses worldwide. Unfortunately, now that people are no longer as vigilant in using those protective measures, the RSV is back in full force. RSV is a common and highly contagious seasonal virus that infects almost all babies and young children. Every year, 150 to 200 babies in the Netherlands end up in intensive care with this virus. Globally, RSV is the second leading cause of death in infants. Press release. 

Castor announced a new offering to simplify post-marketing clinical trials. By extending global reach, integrating real-world data (RWD), and automating trial processes, Castor's latest offering can reduce trial costs by 30% and deployment timelines to only four weeks on average. Castor Real-World Evidence (RWE) addresses typical post-marketing clinical trial (PMCT) challenges by easily integrating RWD sources such as patient-reported outcomes and wearables to increase patient access and retention, data quality, and compliance. Castor RWE provides integrated technology and services to accelerate the collection of RWD for PMCT, enabling life science companies to meet regulatory and payer requirements efficiently. The offering includes pre-configured modules for patient recruitment, eligibility screening, enrollment, data capture, and participant engagement. Press release. 

Massive Bio announced a strategic partnership with Azra AI to provide early identification and precise treatment options, further improving cancer clinical care. Azra AI’s technology, used in over 200 hospitals, including HCA Healthcare, reads pathology reports in a fraction of a second, enabling clinicians to focus on the approximately 10 percent of positive pathology reports immediately to treat patients sooner and give them the best chances for survival. Where Azra AI focuses on identifying cancer, Massive Bio’s platform provides cancer patients with relevant clinical trials using AI, empowering patients to find treatment options faster and enabling life sciences companies to conduct broader, more inclusive, population-based rather than traditional site-specific recruitment. By utilizing AI technology in cancer identification and trial matching, clinical care teams can improve healthcare access and equity by eliminating unconscious bias or human errors that can prevent successful patient enrollment. Press release. 

The Institute of Molecular Genetics and Genetic Engineering (IMGGE) at the University of Belgrade introduced its new research interests. Collaboration between IMGGE and BGI began with building two permanent Huoyan laboratories, one of which became a National Laboratory for Molecular Diagnostics of Infectious Agents. IMGGE collaborated with BGI again to create the Serbian Center for Genome Sequencing and Bioinformatics. Subsequently, the staff underwent extensive onsite training in COVID-19, whole exome sequencing, whole genome sequencing, and noninvasive prenatal testing. Following IMGGE’s plans, the Government of the Republic of Serbia plans to build the BIO4 campus in Belgrade, a 20-hectare facility focusing on four key areas: biomedicine, biotechnology, bioinformatics, and biodiversity. Press release. 

Bio-Rad Laboratories and NuProbe USA signed a licensing and product development agreement where NuProbe USA will exclusively license its allele enrichment technologies to Bio-Rad to develop multiplexed digital PCR assays. This technology will help to advance Bio-Rad’s menu of products in oncology, where highly sensitive and multiplexed mutation detection assays aid translational research, therapy selection, and disease monitoring. NuProbe USA developed and optimized the Blocker Displacement Amplification technology as a PCR method in which variant DNA alleles are enriched over 1000-fold over wild-type alleles. BDA has been experimentally validated on hundreds of clinical samples across multiple platforms, including quantitative PCR, digital PCR, Sanger sequencing, sequencing-by-synthesis, and nanopore sequencing. Press release. 

OneStudyTeam and Antidote Technologies announced a partnership to provide sites and sponsors with a connected, end-to-end patient referral management experience to give them control in clinical trial recruitment efforts. OneStudyTeam works directly with research sites, sponsors, and technology partners to reduce site staff burden by integrating patient recruitment solutions with its cloud-based technology platform, StudyTeam. Antidote’s data-driven outreach focuses on ensuring the right mix of patients is connected with each clinical trial opportunity. At the same time, OneStudyTeam’s solutions provide insights into minority representation further down the funnel. When combined, this data provides sponsors with a complete picture of who is enrolling in their trials—and who is not—and allows recruitment adjustments to ensure the real-world patient population is reflected in every clinical trial. Press release. 

In a study called AWARE II, researchers at NYU Grossman School of Medicine and elsewhere studied 567 men and women whose hearts stopped beating while hospitalized and who received CPR between May 2017 and March 2020 in the United States and the United Kingdom. Survivors reported having unique lucid experiences, including a perception of separation from the body, observing events without pain or distress, and a meaningful evaluation of life, including their actions, intentions, and thoughts toward others. The researchers found these death experiences to be different from hallucinations, delusions, illusions, dreams, or CPR-induced consciousness. They concluded that further research is needed to more precisely define biomarkers of what is considered clinical consciousness, the human-recalled experience of death, and to monitor the long-term psychological effects of resuscitation after cardiac arrest. Press release. 

Researchers attending the American Society of Nephrology Kidney Week 2022 presented algorithms that predict patients at risk for acute kidney injury (AKI) to help clinicians take appropriate preventive measures. A Taichung Veterans General Hospital team used their artificial intelligence-based model to accurately predict AKI 24 hours ahead of time (Study: “Machine learning for development of a real-time AKI risk prediction model in ICU with external validation and federated learning at five medical centers: From model development to clinical application”). A University of Chicago team accurately predicted adverse kidney events within 90 days of discharge (i.e., acute kidney injury, chronic kidney disease, need for dialysis, or kidney-related death) (Study: “Development of a Machine Learning Algorithm to Predict Major Adverse Kidney Events After Hospitalization”). A third group recently developed a strategy that combines information from the treatment effects on urinary albumin:creatinine ratio changes and glomerular filtration rate (GFR) slope to improve the prediction of treatments’ effects on patient outcomes. The analysis revealed that combining these two measures improves predictions of the treatments’ impact on clinical endpoints (Study: “Change in albuminuria and GFR slope as joint surrogate endpoints for kidney failure - Implications for phase 2 trials”). Press release one and two. 

Medable announced the availability of its Total Consent offering, a fully enabled SaaS electronic consent management solution compatible with every clinical trial in more than 120 locales around the globe. With Total Consent, Medable is transforming the consenting process into one simple-to-use platform that enables trial sponsors and CROs to customize eConsent in compliance with local regulatory requirements. The solution allows patients to consent onsite or remotely, with eSignature or wet ink signature. It also includes print-to-sign templates, template management, and streamlined workflows. Enhanced authoring tools also allow sponsors and CROs to include customized instructional videos and knowledge checks, further enhancing patient understanding and reducing site burden. Press release. 

A computational study led by researchers with the SWOG Cancer Research Network has found that for modestly sized phase 2 trials, stratified analysis with more than one or two stratification factors can significantly reduce the power of a trial to detect a positive result. For example, the researchers found that for the small phase 2 trial design they worked from, which had a total sample size of 84 participants (42 per arm), a stratified analysis that included one or two stratification factors did not significantly decrease the statistical power from that of an unstratified design. However, when four or six stratification factors were used, statistical power decreased from around 88 percent in the unstratified setting to 75 percent or 55 percent, respectively. Generally, clinical trial designs aim to have at least 80 percent power to detect a difference between treatment arms. Press release. 

Saint Louis University announced the launch of the Institute for Translational Neuroscience (ITN). The new Institute brings together experts in biochemistry, chemistry, pharmacology, social justice, and community outreach under a shared research umbrella to study the physiology and diseases of the central nervous system and ease human suffering. ITN aims to improve human health through the pre-clinical and clinical research in cognition, Alzheimer’s disease, chronic neuropathic pain, opioids, metabolism, and traumatic brain injury, among others. The institute will provide neuroscience training for undergraduate and graduate students at the University, engage the neuroscience community at large, and promote neuroscience through advocacy and outreach efforts. Press release. 

The American College of Rheumatology (ACR) has released Continuing Medical Education (CME) for dermatologists and nephrologists to help them learn more about clinical trials for lupus patients. The condition disproportionately affects African/Black Americans, Hispanics, and Native Americans. This new CME is part of the ACR’s “Materials to Increase Minority Involvement in Clinical Trials” initiative, aimed at educating these providers on the importance of increasing minority participation in lupus clinical trials. The new CME provides nephrologists and dermatologists with specific information on racial disparities in lupus clinical trials, increasing minority participation, and the barriers providers face when encouraging patients to participate. More information about the ACR’s work can be found on the Lupus Initiative website. Press release. 

The GOG Foundation (GOG-F), the Society for Gynecologic Oncology (SGO), and the Foundation for Women’s Cancer (FWC) are elevating their shared dedication to improving patient outcomes and accelerating cancer research through a strategic partnership in three critical areas: research and career advancement; diversity, inclusion, and health equity; and industry relations. GOG-F, SGO, and FWC representatives developed a dynamic and diverse GOG/SGO/FWC steering committee to provide continual partnership oversight and guidance. Future collaborative efforts across organizations will continue to be informed by a shared commitment to foster relationships and identify strategic opportunities for the mutual benefit of patients, members, and the broader community. GOG-F, SGO, and FWC will share the resulting impact of collaborative efforts on internal and external communications platforms and in future joint press releases. Press release. 

Medidata, a Dassault Systèmes company, and Boehringer Ingelheim announced a five-year renewal of their collaboration to extend the use of Rave EDC for Boehringer Ingelheim's clinical trials worldwide and include myMedidata, Medidata’s suite of innovative patient-facing technologies focused on enhancing patient centricity and diversity in decentralized clinical trials. The myMedidata patient portal is a single-destination web-based platform that provides access to electronic consent, electronic Clinical Outcome Assessment, and myMedidata LIVE video investigator/patient visits. The Medidata Decentralized Clinical Trials Program—the industry’s only scalable, end-to-end offering—creates opportunities to improve patient access, inclusion, and engagement across geographies and improve patient diversity in clinical trials. Press release. 

Gladstone Institutes and the Chan Zuckerberg Biohub signed an affiliation agreement to increase their research collaboration. The two San Francisco organizations will partner to uncover the fundamental mechanisms of disease to find new prevention strategies, treatments, and cures. The new affiliation agreement enables Gladstone investigators appointed as CZ Biohub Investigators to access resources to participate in research at both organizations. These investigators can also hire postdoctoral fellows and staff at Biohub facilities to support their science. Press release. 

Paradigm4’s REVEAL Integrative Analytics platform enables Alnylam Pharmaceuticals to discover novel, genetically validated drug targets from population-scale biobank genotype-phenotype datasets. It underlies the extraordinary pace and productivity of Alnylam’s RNAi therapeutics pipeline. Fundamental to Paradigm4’s REVEAL platform is a unique technology stack for working with high-dimension, content-diverse, genotypic, and longitudinal phenotypic data at human population scales, soon to be counted in millions. By pairing Paradigm4’s unique technology with a platform for rationally designed, sequence-based precision medicines, Alnylam can go from target discovery to a Phase 1 clinical trial in as little as 18 months. Using REVEAL, Alnylam scientists can systematically calculate single-variant and gene-level tests on a dynamic burst cluster that scales to 200 or more spot workers in a time- and cost-efficient manner. Press release.