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At least 145 records · Page 8

Countermeasures against space flight related bone loss

A general review is presented of data on bone loss with references to countermeasures for use during spaceflight and bedrest. The two primary countermeasures against skeletal atrophy are skeletal loading such as centrifugation and exercise and/or the administration of drugs designed to alter the rate of bone remodeling. Bone loss is argued to be unavoidable in long-duration spaceflight in spite of countermeasures utilized, but a combination of exercise, biochemical treatments, and post-flight therapy is considered the optimal solution.

Leblanc, Adrian↗

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences

Relatively low levels of expression from naturally occurring promoters have limited the use of muscle as a gene therapy target. Myogenic restricted gene promoters display complex organization usually involving combinations of several myogenic regulatory elements. By random assembly of E-box, MEF-2, TEF-1, and SRE sites into synthetic promoter recombinant libraries, and screening of hundreds of individual clones for transcriptional activity in vitro and in vivo, several artificial promoters were isolated whose transcriptional potencies greatly exceed those of natural myogenic and viral gene promoters.

Non-NASA Center↗

Potentiating antibiotic efficacy via perturbation of non-essential gene expression

Proliferation of multidrug-resistant (MDR) bacteria poses a threat to human health, requiring new strategies. Here we propose using fitness neutral gene expression perturbations to potentiate antibiotics. We systematically explored 270 gene knockout-antibiotic combinations in Escherichia coli, identifying 90 synergistic interactions. Identified gene targets were subsequently tested for antibiotic synergy on the transcriptomic level via multiplexed CRISPR-dCas9 and showed successful sensitization of E. coli without a separate fitness cost. These fitness neutral gene perturbations worked as co-therapies in reducing a Salmonella enterica intracellular infection in HeLa. Finally, these results informed the design of four antisense peptide nucleic acid (PNA) co-therapies, csgD, fnr, recA and acrA, against four MDR, clinically isolated bacteria. PNA combined with sub-minimal inhibitory concentrations of trimethoprim against two isolates of Klebsiella pneumoniae and E. coli showed three cases of re-sensitization with minimal fitness impacts. Our results highlight a promising approach for extending the utility of current antibiotics.

60 APPLIED LIFE SCIENCES↗

Radiation Therapy Enhanced by NBTXR3 Nanoparticles Overcomes Anti-PD1 Resistance and Evokes Abscopal Effects

Radiation combined with PD1 blockade offers significant treatment benefits in several tumor types; however, anti-PD1 resistance precludes such benefits in many cases. Here we attempted to overcome anti-PD1 resistance by combining localized radiation with a radioenhancing nanoparticle (NBTXR3) and systemic anti-PD1 treatment to achieve abscopal effects in an anti-PD1–resistant mouse model of lung cancer.

62 RADIOLOGY AND NUCLEAR MEDICINE↗

Case report: Fatal Legionella infection diagnosed via by metagenomic next-generation sequencing in a patient with chronic myeloid leukemia

Legionella is an aerobic, gram-negative, intracellular pathogen and is an important cause of community-acquired pneumonia. Legionella pneumophila is the most common causative agent of Legionella pneumonia. Clinical diagnosis of Legionella pneumonia is challenging due to the lack of specific clinical manifestations and the low positive rates of conventional pathogen detection methods. In this study, we report a case of a patient with chronic myeloid leukemia who developed rigors and high fever after chemotherapy and immunotherapy. Chest computed tomography revealed consolidation in the left lower lobe of the lung and ground-glass opacities in both lower lobes. Multiple blood cultures showed Escherichia coli , Staphylococcus aureus , Bacillus licheniformis , and positive results in the β-D-glucan test (G test). The patient was treated with various sensitive antimicrobial agents, including meropenem plus fluconazole, meropenem plus carpofungin, and vancomycin. Unfortunately, the patient’s condition gradually worsened and eventually resulted in death. On the following day of death, metagenomic next-generation sequencing (mNGS) of 1whole blood revealed L. pneumophila pneumonia with concurrent bloodstream infection (blood mNGS reads 114,302). These findings suggest that when conventional empirical antimicrobial therapy proves ineffective for critically ill patients with pneumonia, the possibility of combined Legionella infection must be considered, and mNGS can provide a diagnostic tool in such cases.

Bu, Chunhong↗

Thermoeconomic cost optimization of superconducting magnets for proton therapy gantries

A compact gantry delivering 70-220 MeV protons with fixed field in the superconducting magnets could reduce the cost and improve the adoption of proton therapy. While a number of magnet and cryogenics designs have been proposed, the combined capital and operating costs of state-of-the-art superconducting materials have not been analyzed. In response, we develop a thermoeconomic model of a multi-stage, conduction cooled gantry lattice and analyze the cryocooler operating cost, cryocooler capital cost and conductor capital cost for Nb-Ti, Nb 3 Sn, REBCO and Bi-2223 over a continuous range of magnet temperatures, and a differential evolution algorithm is used to identify the optimal combination of thermal intercept temperatures. Although Nb3Sn yields the lowest Net Present Value (NPV) of $111.7k at a magnet temperature of 9.4 K, the optimized Bi-2223 design at 12.8 K approaches the realm of commercial feasibility by offering improved thermal stability and forgoing the need for costly conductor heat treatment and magnet quench training. Furthermore, it was found that Nb3Sn was more cost effective than Nb-Ti and that REBCO was not economically viable for the parameters of this investigation. Overall, the thermoeconomic model developed herein can optimize conductor choices, magnet temperatures and thermal staging which has value for any conduction-cooled superconducting magnet.

BSCCO↗

Microcapsules and Methods for Making

This invention relates to methods for forming multi-lamellar microcapsules of both hydrophilic and hydrophobic immiscible liquid phases using several polymer/solvent systems. Liquid-Liquid diffusion and spontaneous emulsification are controlled by properly timed sequence exposures of immiscible phases in aqueous vehicles dispersed in hydrocarbon solvents containing small quantities of oil, co-surfactants, and glycerides. Water-in-oil and oil-in-water microcapsules are formed containing selected combinations of several types of drugs, co-encapsulated within fluid compartments inside the microcapsule. Commercial applications of the process and the resultant product relate to drug therapy for treating medical conditions such as cancer, circulatory conditions, and other conditions in which pharmaceuticals are advantageously targeted to specific organs, or delivered in combination with other pharmaceuticals. Small microcapsules may be delivered intravenously to diseased organs or clotted vessels. The use of multiple drugs within the same microcapsule structure provides advantages for applications such as chemoembolization treatments and may be used to deliver both chemotherapeutic drugs, against tumor cells, and an immuno-adjuvant or immunological stimulant to enhance the patient's immune response. Active forms of urokinase and other enzymes may be delivered without dilution to the local site of an embolism for dissolving the embolism. Thus, the invention has several potentially valuable commercial applications related to pharmaceutical and medical applications.

Morrison, Dennis R.↗

State and Regional Variation in Prescription- and Payment-Related Promoters of Adherence to Blood Pressure Medication

Introduction: Medication adherence can improve hypertension management. How blood pressure medications are prescribed and purchased can promote or impede adherence. Methods: We used comprehensive dispensing data on prescription blood pressure medication from Symphony Health’s 2017 Integrated Dataverse to assess how prescription- and payment-related factors that promote medication adherence (ie, fixed-dose combinations, generic formulations, mail order, low-cost or no-copay medications) vary across US states and census regions and across the market segments (grouped by patient age, prescriber type, and payer type) responsible for the greatest number of blood pressure medication fills. Results: In 2017, 706.5 million prescriptions for blood pressure medication were filled, accounting for $29.0 billion in total spending (17.0% incurred by patients). As a proportion of all fills, factors that promoted adherence varied by state: fixed-dose combinations (from 5.8% in Maine to 17.9% in Mississippi); generic formulations (from 95.2% in New Jersey to 98.4% in Minnesota); mail order (from 4.7% in Rhode Island to 14.5% in Delaware); and lower or no copayment (from 56.6% in Utah to 72.8% in California). Furthermore, mean days’ supply per fill (from 43.1 in Arkansas to 63.8 in Maine) and patient spending per therapy year (from $38 in Hawaii to $76 in Georgia) varied. Concentration of adherence factors differed by market segment. Patients aged 18 to 64 with a primary care physician prescriber and Medicaid coverage had the lowest concentration of fixed-dose combination fills, mean days’ supply per fill, and patient spending per therapy year. Patients aged 65 years or older with a primary care physician prescriber and commercial insurance had the highest concentration of fixeddose combinations fills and mail order fills. Conclusion: Addressing regional and market segment variation in factors promoting blood pressure medication adherence may increase adherence and improve hypertension management.

60 APPLIED LIFE SCIENCES↗

Promethazine and its use as a treatment for space motion sickness

Until Mar. 1989, no effective treatment--either prophylactic or symptomatic--for space motion sickness (SMS) had been discovered. Since Mar. 1989, intramuscular (IM) promethazine (PMZ) has been used in the treatment of SMS with extremely favorably results reported by the crew. A retrospective study was undertaken to quantify the efficacy of IM PMZ since its institution and the incidence of its major anticipated side-effect drowsiness and sedation. The results from a standardized crew medical debriefing conducted immediately after landing and follow-up interviews with the crews were used in establishing the efficacy and incidence of side effects from treatment. Only crews from the first 44 Shuttle flights on their first mission were considered. For a total of 132 crewmembers, 96 exhibited symptoms of SMS; and, of these, 20 were treated with IM PMZ. Ninety percent of those receiving IM PMZ 25-50mg received nearly immediate (less than 2 hours) relief of symptoms and 75 percent required no further treatment through the first 2 days of spaceflight. Those not receiving this treatment did not have any near-term resolution of their symptoms, and 50 percent were still ill through the second day of flight. This represents a significant difference at the p = 0.46 level. In stark contrast to the 60 percent to 73 percent incidence of sedation or drowsiness reported in individuals treated with PMZ in terrestrial environment at the doses used here, less than 5 percent reported these symptoms during spaceflight. IM PMZ is an effective therapy for SMS and is associated with minimal incidence of sedation or drowsiness. This combination of efficacy that is absent of significant side effects represents a substantial improvement in the operational situation of crewmembers afflicted with SMS. Studies to understand the mechanisms underlying these observations will be undertaken in the future.

Bagian, James P.↗

HIV-1 protease inhibitors with a P1 phosphonate modification maintain potency against drug-resistant variants by increased interactions with flap residues

Protease inhibitors are the most potent antivirals against HIV-1, but they still lose efficacy against resistant variants. Improving the resistance profile is key to developing more robust inhibitors, which may be promising candidates for simplified next-generation antiretroviral therapies. Here, in this study, we explored analogs of darunavir with a P1 phosphonate modification in combination with increasing size of the P1′ hydrophobic group and various P2′ moieties to improve potency against resistant variants. The phosphonate moiety substantially improved potency against highly mutated and resistant HIV-1 protease variants, but only when combined with more hydrophobic moieties at the P1′ and P2′ positions. Phosphonate analogs with a larger hydrophobic P1′ moiety maintained excellent antiviral potency against a panel of highly resistant HIV-1 variants, with significantly improved resistance profiles. The cocrystal structures indicate that the phosphonate moiety makes extensive hydrophobic interactions with the protease, especially with the flap residues. Many residues involved in these protease-inhibitor interactions are conserved, enabling the inhibitors to maintain potency against highly resistant variants. These results highlight the need to balance inhibitor physicochemical properties by simultaneous modification of chemical groups to further improve resistance profiles.

60 APPLIED LIFE SCIENCES↗

Exercise support for therapy

Constant-value weight-relieving apparatus, which moves on rollers on overhead track, supports weight of walking, stooping, squatting, or standing patient with combination of multiple pulleys and spring clusters. Individually preselected support force is constant for all movements.

Long, M. J.↗

High-throughput methods leveraging robotics and computer vision for the development of therapeutic phage cocktails

We present the high-throughput automated screening techniques that are being used to develop bacteriophage-based therapeutic products currently under investigation in human clinical trials to combat urinary tract infections. By integrating modern liquid handling robotics, standardized phenotypic assays, and computer vision-based enumeration, we established a platform capable of reproducibly screening large collections of phages against clinically derived bacterial strain panels. This approach enabled systematic assessment of phage-bacteria interactions at scale, facilitating the identification and optimization of phage cocktails with broad in vitro activity. Although bacteriophage therapy has long been investigated as a strategy for treating bacterial infections, few frameworks exist for developing phage combinations in a reproducible and scalable manner. The methods outlined here address this gap and aim to support the broader development of therapeutic assets available to combat antibiotic resistance.

Penke, Taylor J. R. [Locus Biosciences, Morrisvill↗

Combinatorial Multidomain Mesoporous Chips and a Method for Fractionation, Stabilization, and Storage of Biomolecules

A new fractionation device shows desirable features for exploratory screening and biomarker discovery. The constituent MSCs may be tailored for desired pore sizes and surface properties and for the sequestration and enrichment of extremely low abundant protein and peptides in desired ranges of the mass/charge spectrum. The MSCs are effective in yielding reproducible extracts from complex biological samples as small as 10 microliter in a time as short as 30 minutes. They are inexpensive to manufacture, and allow for scaled up production to attain the simultaneous processing of a large number of samples. The MSCs are multiplexed, label-free diagnostic tools with the potential of biological recognition moiety modification for enhanced specificity. The MSCs may store, protect and stabilize biological fluids, enabling the simplified and cost-effective collection and transportation of clinical samples. The MSC-based device may serve as a diagnostic tool to complement histopathology, imaging, and other conventional clinical techniques. The MSCs mediated identification of disease-specific protein signatures may help in the selection of personalized therapeutic combinations, in the real-time assessment of therapeutic efficacy and toxicity, and in the rational modulation of therapy based on the changes in the protein networks associated with the prognosis and the drug resistance of the disease.

Ferrari, Mauro↗

A multi-specific, multi-affinity antibody platform neutralizes sarbecoviruses and confers protection against SARS-CoV-2 in vivo

Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the causative agent of coronavirus disease 2019 (COVID-19), has been responsible for a global pandemic. Monoclonal antibodies (mAbs) have been used as antiviral therapeutics; however, these therapeutics have been limited in efficacy by viral sequence variability in emerging variants of concern (VOCs) and in deployment by the need for high doses. In this study, we leveraged the multi-specific, multi-affinity antibody (Multabody, MB) platform, derived from the human apoferritin protomer, to enable the multimerization of antibody fragments. MBs were shown to be highly potent, neutralizing SARS-CoV-2 at lower concentrations than their corresponding mAb counterparts. In mice infected with SARS-CoV-2, a tri-specific MB targeting three regions within the SARS-CoV-2 receptor binding domain was protective at a 30-fold lower dose than a cocktail of the corresponding mAbs. Furthermore, we showed in vitro that mono-specific MBs potently neutralize SARS-CoV-2 VOCs by leveraging augmented avidity, even when corresponding mAbs lose their ability to neutralize potently, and that tri-specific MBs expanded the neutralization breadth beyond SARS-CoV-2 to other sarbecoviruses. Our work demonstrates how avidity and multi-specificity combined can be leveraged to confer protection and resilience against viral diversity that exceeds that of traditional monoclonal antibody therapies.

60 APPLIED LIFE SCIENCES↗

Use of Longitudinal Serum Analysis and Machine Learning to Develop a Classifier for Cancer Early Detection

Early detection of solid tumors through a simple screening process, such as the proteomic analysis of biofluids, has the potential to significantly alter the management and outcomes of cancers. The application of advanced targeted proteomics measurements and data analysis strategies to uniformly collected serum or plasma samples would enable longitudinal studies of cancer risk, progression, and response to therapy that have the potential to significantly reduce cancer burden in general. In this article, we describe a generalizable workflow combining robust, multiplexed targeted proteomics measurements applied to longitudinal samples from the Department of Defense Serum Repository with a Random Forest machine learning method for developing and initially evaluating the performance of candidate biomarker panels for early detection of cancers. The effectiveness of this approach was demonstrated in a cohort of 175 head and neck squamous cell carcinoma patients. The outlined protocols include methods for sample preparation, instrument analysis, and data analysis and interpretation using this workflow.

Longitudinal analysis, machine learning, cancer, e↗

Science &Technology Review (October/November 2022)

Livermore researchers seek to understand the disease mechanism of amyotrophic lateral sclerosis (ALS) and, with that knowledge, identify therapies to cure this always-fatal disease. As the article beginning on p. 4 describes, the ALS research effort combines Laboratory core competencies in bioscience, engineering, computational modeling, and data science.

36 MATERIALS SCIENCE↗

CD8 Lymphocyte Depletion Enhances the Latency Reversal Activity of the SMAC Mimetic AZD5582 in ART-Suppressed Simian Immunodeficiency Virus-Infected Rhesus Macaques

ABSTRACT Inducing latency reversal to reveal infected cells to the host immune system represents a potential strategy to cure human immunodeficiency virus (HIV) infection. In separate studies, we have shown previously that CD8 + T cells may contribute to the maintenance of viral latency and have identified a novel SMAC (second mitochondrial activator of caspases) mimetic/IAP (inhibitor of apoptosis protein) inhibitor (AZD5582) capable of reversing HIV/simian immunodeficiency virus (SIV) latency in vivo by activating the noncanonical (nc) NF-κB pathway. Here, we use AZD5582 in combination with antibody (Ab)-mediated depletion of CD8α + cells to further evaluate the role of CD8 + T cells in the maintenance of viral latency. Six rhesus macaques (RMs) were infected with SIV mac239 and treated with antiretroviral therapy (ART) starting at week 8 postinfection. After 84 to 85 weeks of ART, all animals received a single dose of the anti-CD8α depleting Ab MT807R1 (50 mg/kg of body weight, administered subcutaneously [s.c.]), followed by five weekly doses of AZD5582 (0.1 mg/kg, administered intravenously [i.v.]). Following combined treatment with CD8α depletion and AZD5582, 100% of RMs experienced on-ART viremia levels above 60 copies per ml of plasma. In comparator groups of SIV-infected, ART-suppressed RMs treated with AZD5582 only or CD8α depletion only, on-ART viremia was experienced by 56% and 57% of the animals, respectively. Furthermore, the increased frequency of viremic episodes during the treatment period was greater in the group treated with CD8α depletion plus AZD5582 than in the other groups. Mathematical modeling of virus reactivation suggested that in addition to viral dynamics during acute infection, CD8α depletion influenced the response to AZD5582. This work suggests that the latency reversal induced by activation of the ncNF-κB signaling pathway with AZD5582 can be enhanced by CD8α + cell depletion. IMPORTANCE A favored approach to curing HIV infection aims at inducing viral expression using latency-reversing agents (LRAs) to allow the elimination of infected cells. Here, we tested a combination of two recently identified LRAs, the SMAC mimetic/IAP inhibitor AZD5582, which activates the noncanonical NF-κB pathway, and the antibody (Ab) MT807R1, which depletes CD8α + cells, in SIV-infected rhesus macaques (RMs) on ART. Latency reversal, as defined by increased on-ART viremia, was observed in all six SIV-infected, ART-treated RMs that received this combined treatment. Furthermore, comparison of viral reactivation between these animals and groups of SIV-infected, ART-treated RMs treated with AZD5582 only or CD8α + cell depletion only showed more frequent increases in viremic episodes when the two treatments were combined. This study provides additional evidence that CD8 + T cells may contribute to the maintenance of HIV/SIV latency on ART and potentially inhibit latency reversal during HIV cure approaches.

59 BASIC BIOLOGICAL SCIENCES↗

Lasofoxifene as a potential treatment for therapy-resistant ER-positive metastatic breast cancer

Background: Endocrine therapy remains the mainstay of treatment for estrogen receptor-positive (ER+) breast cancer. Constitutively active mutations in the ligand binding domain of ERα render tumors resistant to endocrine agents. Breast cancers with the two most common ERα mutations, Y537S and D538G, have low sensitivity to fulvestrant inhibition, a typical second-line endocrine therapy. Lasofoxifene is a selective estrogen receptor modulator with benefits on bone health and breast cancer prevention potential. This study investigated the anti-tumor activity of lasofoxifene in breast cancer xenografts expressing Y537S and D538G ERα mutants. The combination of lasofoxifene with palbociclib, a CDK4/6 inhibitor, was also evaluated. Methods: Luciferase-GFP tagged MCF7 cells bearing wild-type, Y537S, or D538G ERα were injected into the mammary ducts of NSG mice (MIND model), which were subsequently treated with lasofoxifene or fulvestrant as single agents or in combination with palbociclib. Tumor growth and metastasis were monitored with in vivo and ex vivo luminescence imaging, terminal tumor weight measurements, and histological analysis. Results: As a monotherapy, lasofoxifene was more effective than fulvestrant at inhibiting primary tumor growth and reducing metastases. Adding palbociclib improved the effectiveness of both lasofoxifene and fulvestrant for tumor suppression and metastasis prevention at four distal sites (lung, liver, bone, and brain), with the combination of lasofoxifene/palbociclib being generally more potent than that of fulvestrant/palbociclib. X-ray crystallography of the ERα ligand binding domain (LBD) shows that lasofoxifene stabilizes an antagonist conformation of both wild-type and Y537S LBD. The ability of lasofoxifene to promote an antagonist conformation of Y537S, combined with its long half-life and bioavailability, likely contributes to the observed potent inhibition of primary tumor growth and metastasis of MCF7 Y537S cells. Conclusions: We report for the first time the anti-tumor activity of lasofoxifene in mouse models of endocrine therapy-resistant breast cancer. The results demonstrate the potential of using lasofoxifene as an effective therapy for women with advanced or metastatic ER+ breast cancers expressing the most common constitutively active ERα mutations.

60 APPLIED LIFE SCIENCES↗