Uncurable Disease Research Articles

Identification of monocyte-associated pathways participated in the pathogenesis of pulmonary arterial hypertension based on omics-data.

Pulmonary arterial hypertension (PAH) is one kind of chronic and uncurable diseases that can cause heart failure. Immune microenvironment plays a significant role in PAH. The aim of this study was to assess the role of immune cell infiltration in the pathogenesis of PAH.Differentially expressed genesbased on microarray data were enriched in several immune-related pathways. To evaluate the immune cell infiltration, based on the microarray data sets in the GEO database, we used both ssGSEA and the CIBERSORT algorithm. Additionally, single-cell RNA sequencing(scRNA-seq) data was used to further explicit the specific role and intercellular communications. Then receiver operating characteristiccurves and least absolute shrinkage and selection operatorwere used to discover and test the potential diagnostic biomarkers for PAH.Both the immune cell infiltration analyses based on the microarray data sets and the cell proportion in scRNA-seq data exhibited a significant downregulation in the infiltration of monocytes in PAH. Then, the intercellular communications showed that the interaction weighs of most immune cells, including monocytes changed between the control and PAH groups, and the ITGAL-ITGB2 and ICAM signaling pathways played critical roles in this process. In addition, ITGAM and ICAM2 displayed good diagnosis values in PAH. This study implicated that the change of monocyte was one of the key immunologic features of PAH. Monocyte-associated ICAM-1 and ITGAL-ITGB2 signaling pathways might be involved in the pathogenesis of PAH.

The applications, limitations, and future consideration of immune checkpoint inhibitors in glioblastoma

Glioblastoma multiforme is a grade IV malignant tumor found in the brain, formed by mutated astrocytes. Patients with glioblastoma may experience headaches and nausea, and several different symptoms depending on where the tumor is located. Surgery is the primary treatment used for glioblastoma to remove the tumor, but the diffusive nature of this tumor makes it hard to completely remove. The survival years after treatments had not shown improvement with surgeries along with chemo and radiotherapy. Immune checkpoint inhibitors, a type of immunotherapy, were seen to provide a foreground to the development of an effective treatment. Immune checkpoint inhibitors block pathways that create suppression of T cells to restore their function and allow them to attack cancerous cells again, stimulating an immune response. Even though immune checkpoint inhibitors had shown effectiveness and success in preclinical trials and in treating other cancers, clinical trials using these drugs failed to show improved effectiveness and prognosis of patients with immune checkpoint inhibitors. Some patients didn’t generate a response to the checkpoint inhibitors, while for others the effect of checkpoint inhibitors lasted relatively shortly. Though, several proposals for solutions to the limitations of checkpoint inhibitors were raised by scientists. Immune checkpoint inhibitor shows the potential of being a hope to enhance survival years and prognosis of this currently uncurable disease. The research targets at discussing the mechanisms of ipilimumab, nivolumab, and bevacizumab (types of immune checkpoint inhibitors) and their limitations to provide suggestions of different methods that could be applied to potentially solve the problem and enhance their effectiveness.

The cost of living.

The new biologics have great potential to help patients with hitherto uncurable diseases. But their exorbitant costs challenges the basis of health care systems based on equity.

The Discovery of Menstrual Derived Stem Cells and their Therapeutic Effects

Stem cell therapy is the future of medicine as research is ever-growing for uncurable diseases and injuries. Stem cells are difficult to obtain due to ethical conflicts, retrieval difficulties, or high expenses. However, a stem cell line referred to as menstrual derived stem cells (MenSCs) show great potential in addressing all these issues. MenSCs are a type of adult stem cell that can be found in menstrual blood. These cells are considered a valuable source of stem cells due to their ease of accessibility, abundance, and lack of ethical concerns. MenSCs have been found to possess properties similar to other adult stem cells, such as the ability to differentiate into various cell types, including nerve cells, bone cells, and cartilage cells. Research has shown that MenSCs have the potential to be used in regenerative medicine for various conditions, including cardiovascular disease, nervous system repair, spinal cord injuries, liver and lung health, as well as skin repair. Additionally, MenSCs have been found to possess anti-inflammatory and immunomodulatory properties, making them a potential candidate for the treatment of autoimmune diseases. Despite the potential benefits of MenSCs, further research is needed to fully understand their properties and potential uses. Currently, there are ongoing clinical trials to evaluate the safety and efficacy of MenSCs for various conditions.

Abstract 566: Perivascular Accumulation Of CD45+ Cells In The Lungs Of Female BMPR2 +/R899X Mutant Mice May Be A Factor To Sex-linked Pulmonary Arterial Hypertension

Introduction: Pulmonary arterial hypertension (PAH) is an uncurable disease characterized by pulmonary vasoconstriction and remodeling, elevated right ventricle systolic pressure (RVSP), and right ventricle hypertrophy (RVH). Recently, our group showed that BMPR2 +/R899X loss-of-function mutation per se significantly increased vasoconstrictive Endothelin-1 (ET-1) in the lungs of female animals, who also exhibited higher RVSP compared to males. Why females have higher PAH incidence remains unclear. Hypothesis: We hypothesize that higher PAH incidence in female mice carrying the BMPR2 +/R899X mutation is associated with the spontaneous development of pulmonary vascular inflammation and ET-1-mediated vasoconstriction. Methods: To test this hypothesis, we used 12-month-old male and female wild-type and BMPR2 +/R899X mutant mice for quantification of RVSP and RVH and for analysis of the ET-1 and CD45+ pulmonary expression by Western Blot and immunohistochemistry. Results: In opposition to BMPR2 +/R899X mutant animals, data indicated no significant difference between sexes regarding ET-1 expression and RVSP in wild-type animals. When investigating the source of ET-1 in the lungs of BMPR2 +/R899X female mice, our preliminary data suggested it accumulates in perivascular cells. Moreover, double-blind immunohistochemistry examination of the pan-leukocyte marker CD45 revealed a massive perivascular infiltration of CD45+ cells in the lungs of female mutant animals compared to males (4.09 +/- 0.68 and 10.46 +/- 1.15 CD45+ cells/microvessel, male and female, respectively; 8 micrographs/animal; 3 mice/group), supporting our hypothesis that BMPR2 +/R899X mutation is an important factor for sex-linked vascular inflammation in PAH. Conclusion: In conclusion, elevated perivascular CD45+ cells in the lungs of female BMPR2 +/R899X mutant mice may contribute to PAH sex bias.

Case Study of Co-morbidities --- HIV/AIDS and Blood Cancer

HIV/AIDS is considered an uncurable disease today, and patients have to take medicine uninterrupted for their whole life to control the viral load. However, there have been five special cases possibly cured --- the Berlin patient (HIV-1 and AML), the London patient (HIV-1 and Hodgkin’s Lymphoma), the Düsseldorf patient (HIV-1 and AML), the New York patient (HIV-1 and high-risk AML monosomy 7), and the City of Hope patient (HIV-1 and AML). They were infected with HIV and blood cancer concurrently and received allogeneic hematopoietic stem cell transplant (allo-HSCT). By comparing the Düsseldorf patient and the New York patient who are in relatively similar conditions, umbilical cord blood (UCB) is very likely to be the better source for transplant based on the severity of graft versus host disease (GvHD) after transplant. A possible future treatment which is to combine the haematopoietic stem cell transplant (HSCT) and gene therapy with the help of CRISPR-Cas9, is suggested.

Blocking Caspase-1/Gsdmd and Caspase-3/-8/Gsdme pyroptotic pathways rescues silicosis in mice.

Millions of patients suffer from silicosis, but it remains an uncurable disease due to its unclear pathogenic mechanisms. Though the Nlrp3 inflammasome is involved in silicosis pathogenesis, inhibition of its classic downstream factors, Caspase-1 and Gsdmd, fails to block pyroptosis and cytokine release. To clarify the molecular mechanism of silicosis pathogenesis for new therapy, we examined samples from silicosis patients and genetic mouse models. We discovered an alternative pyroptotic pathway which requires cleavage of Gsdme by Caspases-3/8 in addition to Caspase-1/Gsdmd. Consistently, Gsdmd-/-Gsdme-/- mice showed markedly attenuated silicosis pathology, and Gsdmd-/-Gsdme-/- macrophages were resistant to silica-induced pyroptosis. Furthermore, we found that in addition to Caspase 1, Caspase-8 cleaved IL-1β in silicosis, explaining why Caspase-1-/- mice also suffered from silicosis. Finally, we found that inhibitors of Caspase-1, -3, -8 or an FDA approved drug, dimethyl fumarate, could dramatically alleviate silicosis pathology through blocking cleavage of Gsdmd and Gsdme. This study highlights that Caspase-1/Gsdmd and Caspase-3/8/Gsdme-dependent pyroptosis is essential for the development of silicosis, implicating new potential targets and drug for silicosis treatment.

Abstract 5384: Cell growth of sunitinib-resistant renal cell carcinoma was inhibited by Shikonin through necroptosis induction and Akt/mTOR signaling inhibition

Abstract Despite the establishment of new therapy options during the last decades, advanced renal cell carcinoma (RCC) remains an uncurable disease, due to evolving drug-resistance. New, more efficient treatment strategies are still in urgent demand. Shikonin (SHI) from Traditional Chinese Medicine has exhibited anti-tumor properties in several tumor entities. Thus, in the current investigation we evaluated the impact of SHI on progressive growth and metastatic behavior, not only in therapy-sensitive (parental) but also in therapy-resistant RCC cells. Parental and sunitinib-resistant RCC cells, Caki-1, 786-O, KTCTL-26, and A498, were treated with SHI. Tumor cell growth, proliferation, clonogenic capacity, cell cycle phase distribution, induction of cell death (apoptosis and necroptosis) and the expression and activity of regulating and signaling proteins were evaluated. Moreover, the adhesion and chemotactic activity of the RCC cells after exposure to SHI were investigated. SHI did not affect cell motility but led to significant inhibition of progressive tumor cell growth in both therapy-sensitive and therapy-resistant RCC cells. Thereby, SHI significantly inhibited the growth, proliferation and clone formation in parental and sunitinib-resistant RCC cells by G2/M phase arrest through down-regulation of cell cycle activating proteins, blocking cell division. Furthermore, SHI induced apoptosis and necroptosis by activating necrosome complex proteins. Concomitantly, SHI impaired the Akt/mTOR pathway, pivotal for cell survival and growth. Thus, SHI may hold promise as an additive option in treating patients with advanced and therapy-resistant RCC. Citation Format: Eva Juengel, Sascha D. Markowitsch, Olesya Vakhrusheva, Yasminn Akele, Jovanna Kitanovic, Patricia Schupp, Kimberly S. Slade, Anita Thomas, Igor Tsaur, Rene Mager, Thomas Efferth, Axel Haferkamp. Cell growth of sunitinib-resistant renal cell carcinoma was inhibited by Shikonin through necroptosis induction and Akt/mTOR signaling inhibition [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2022; 2022 Apr 8-13. Philadelphia (PA): AACR; Cancer Res 2022;82(12_Suppl):Abstract nr 5384.

Direct Cell-Cell Communication via Membrane Pores, Gap Junction Channels, and Tunneling Nanotubes: Medical Relevance of Mitochondrial Exchange.

The history of direct cell-cell communication has evolved in several small steps. First discovered in the 1930s in invertebrate nervous systems, it was thought at first to be an exception to the “cell theory”, restricted to invertebrates. Surprisingly, however, in the 1950s, electrical cell-cell communication was also reported in vertebrates. Once more, it was thought to be an exception restricted to excitable cells. In contrast, in the mid-1960s, two startling publications proved that virtually all cells freely exchange small neutral and charged molecules. Soon after, cell-cell communication by gap junction channels was reported. While gap junctions are the major means of cell-cell communication, in the early 1980s, evidence surfaced that some cells might also communicate via membrane pores. Questions were raised about the possible artifactual nature of the pores. However, early in this century, we learned that communication via membrane pores exists and plays a major role in medicine, as the structures involved, “tunneling nanotubes”, can rescue diseased cells by directly transferring healthy mitochondria into compromised cells and tissues. On the other hand, pathogens/cancer could also use these communication systems to amplify pathogenesis. Here, we describe the evolution of the discovery of these new communication systems and the potential therapeutic impact on several uncurable diseases.

Towards gene therapy for IPEX syndrome.

Immune dysregulation polyendocrinopathy enteropathy X linked (IPEX) syndrome is an uncurable disease of the immune system, with immune dysregulation that is caused by mutations in FOXP3. Current treatment options, such as pharmacological immune suppression and allogeneic hematopoietic stem cell transplantation, have been beneficial but present limitations, and their life‐long consequences are ill‐defined. Other similar blood monogenic diseases have been successfully treated using gene transfer in autologous patient cells, thus providing an effective and less invasive therapeutic. Development of gene therapy for patients with IPEX is particularly challenging because successful strategies must restore the complex expression profile of the transcription factor FOXP3, ensuring it is tightly regulated and its cell subset‐specific roles are maintained. This review summarizes current efforts toward achieving gene therapy to treat immune dysregulation in IPEX patients.

The Age-Related Macular Degeneration (AMD)-Preventing Mechanism of Natural Products

Age-related macular degeneration (AMD) is related to central visual loss in elderly people and, based on the increment in the percentage of the aging population, the number of people suffering from AMD could increase. AMD is initiated by retinal pigment epithelium (RPE) cell death, finally leading to neovascularization in the macula lutea. AMD is an uncurable disease, but the symptom can be suppressed. The current therapy of AMD can be classified into four types: device-based treatment, anti-inflammatory drug treatment, anti-vascular endothelial growth factor treatment, and natural product treatment. All these therapies have adverse effects, however early AMD therapy used with products has several advantages, as it can prevent RPE cell apoptosis in safe doses. Cell death (apoptosis) is caused by various factors, such as oxidative stress, inflammation, carbonyl stress, and a deficiency in essential components for cells, and RPE cell death is related to oxidative stress, inflammation, and carbonyl stress. Some natural products have anti-oxidative effects, anti-inflammation effects, and/or anti-carbonylation effects. The AMD preventive mechanism of natural products varies, with some natural products activating one or more anti-apoptotic pathways, such as the Nrf2/HO-1 anti-oxidative pathway, the anti-inflammasome pathway, and the anti-carbonyl pathway. As AMD drug candidates from natural products effectively inhibit RPE cell death, they have the potential to be developed as drugs for preventing early (dry) AMD.

Chronic unilateral leg warty lesion: clinical image and literature review

54 years old diabetic male presented with history of with painless multiple progressive warty lesions on the right lower two third of the left leg remain undiagnosed for twelve years. It was spreading and growing relentlessly, not responding to medication prescribed by multiple doctors and patient thought of a uncurable disease. The biopsy came confirmatory. Antifungal therapy along with surgical excision hasten up the healing. An adequate biopsy is a must in undiagnosed skin lesion. Specific medical therapy and adjuvant surgery give early relief.

Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy

Background. New pathogenetic treatment options, such as gene therapy, are now used to treat previously uncurable diseases. However, price of such treatment is high, especially in the case of orphan diseases, where costs may many-fold exceed the prices for other types of medication. This raises a question of optimal way of financing gene therapy in Russia.Objective: to evaluate economic consequences of centralizing procurement of gene therapy in 2021–2030 in the case of the drug indicated for treatment of biallelic RPE65 mutation-associated retinal dystrophy.Material and methods. Voretigene neparvovec is a new gene therapy that is used to treat RPE65 mutation-associated Leber congenital amaurosis and isolated retinitis pigmentosa. We estimated the number of patients (children and adults) that could be treated with voretigene neparvovec in 2021–2030 in Russia using demographic forecasting method, literature and expert data. Budget costs of treatment were estimated for two scenarios: status-quo, where gene therapy is purchased by regions for higher price, and centralized scenario with federal procurements and lower price for the drug.Results. Up to 100 children and 56 adults could be treated with voretigene neparvovec in 2021–2030 in Russia. Centralizing procurements at the expense of federal budget may save up to 20.7% or 1.8 billion rub. (1.13 billion rub. for children and 0.67 billion rub. for adults), compared to regional procurements.Conclusion. Centralizing procurements of expensive drugs intended for gene therapy of orphan diseases may save budget costs of the Russian Federation, compared to status-quo decentralized purchases.

Dissection of Laboratory Animal and Sample Collection for Histology

The starting point for the laboratory investigation of a dissection of laboratory animal for experiment is the taking of samples. This review considers some general principles involved in sample collection for histology (Liu et al., 2016). For disease diagnosis, the tissues sampled should be representative of the condition being investigated and the lesions observed. Samples should be taken with care, to avoid undue stress or injury to the animal or danger to the operator. Where appropriate, samples should be collected aseptically, and care should be taken to avoid crosscontamination between samples (Lapage, 1958). Mice and rats are the most used animals in experimental researches, the anatomical, histological and genetic differences between species should be carefully evaluated, to better apply the study model and avoid unnecessary waste avoid (Corte et al., 2021). Currently, the implantation of defined genetically and sanitarily laboratory animals has aided in new discoveries, through experimental models, contributing to the prevention of uncured diseases such as cancers, AIDS, and multiple sclerosis, and also for the development of new surgical treatment techniques. Other applications correspond to the vaccines development, monoclonal antibodies, evaluation and control of biological products, pharmacology, toxicology, bacteriology, virology and parasitology, and basic immunology studies, immunopathology, organ transplants and immunosuppressive drugs development. However, with technological advances, it is now possible to obtain satisfactory results through alternative methods in vitro, using cell culture and other methods, allowing the 84 replacement of laboratory animals (Gunatilake, 2018).

Regenerative Medicine in South Korea: Bridging the Gap Between Authorization and Reimbursement

Regenerative medicine (RM) has considerable potential to address the needs of aging-related and uncurable diseases. However, its incorporation into reimbursement of health insurance benefits poses many challenges, including uncertain evidence and insufficient investment. This paper examines the wide gap between manufacturers, regulatory bodies, and health technology bodies regarding reimbursements for RMs focused cell therapy products. In this mixed-methods study, we first analyzed the sales of RMs approved in South Korea. In addition to exploring beliefs related to the market value of RMs, in-depth interviews were conducted with 24 experts (17 from bio-industries, two from the regulatory body, three from a health technology assessment (HTA) body, and two from the Pharmaceutical Benefit Coverage Assessment Committee [PBCAC]). Lastly, we surveyed PBCAC members about the market value of RMs. In total, 15 of the 20 developed cell therapy products are on the market in South Korea, and amounted to 0.24% of total pharmaceutical expenditures in 2018. We identified a wide gap between stakeholders and regulators regarding the market value and pricing of RMs. The interviewees from the pharmaceutical manufacturer association raised the issue of rising manufacturing costs and proposed a specific pricing policy for RMs. To bridge the gap between approval and reimbursement, stakeholders demand an alternative framework of value-based pricing. Conditional health insurance reimbursement may be an alternative to the traditional process in order to generate evidence of the effects of RMs using “risk-based” or “outcome-based” approaches.

Immunotherapy in the treatment of lymphoma.

Relapsed or refractory non-Hodgkin’s lymphomas, especially diffuse large B-cell lymphoma as well as relapsed or refractory Hodgkin lymphomas are hard-to-treat diseases. Patients who do not respond to initial therapy or experience relapse are treated with salvage regimens, and if eligible for aggressive therapy, treatment is continued with high-dose chemotherapy and autologous stem cell transplantation. Current therapy options can cure substantial numbers of patients, however for some it is still an uncurable disease. Numerous new drugs and cell therapies are being investigated for the treatment of relapsed or refractory lymphomas. Different types of immunotherapy options have shown promising results, and some have already become the standard of care. Here, we review immunotherapy options for the treatment of lymphoma and discuss the results, positions, practical aspects, and future directions of different drugs and cellular therapies for the treatment of this disease.

Hsp90-mediated regulation of DYRK3 couples stress granule disassembly and growth via mTORC1 signaling.

Stress granules (SGs) are dynamic condensates associated with protein misfolding diseases. They sequester stalled mRNAs and signaling factors, such as the mTORC1 subunit raptor, suggesting that SGs coordinate cell growth during and after stress. However, the molecular mechanisms linking SG dynamics and signaling remain undefined. We report that the chaperone Hsp90 is required for SG dissolution. Hsp90 binds and stabilizes the dual‐specificity tyrosine‐phosphorylation‐regulated kinase 3 (DYRK3) in the cytosol. Upon Hsp90 inhibition, DYRK3 dissociates from Hsp90 and becomes inactive. Inactive DYRK3 is subjected to two different fates: it either partitions into SGs, where it is protected from irreversible aggregation, or it is degraded. In the presence of Hsp90, DYRK3 is active and promotes SG disassembly, restoring mTORC1 signaling and translation. Thus, Hsp90 links stress adaptation and cell growth by regulating the activity of a key kinase involved in condensate disassembly and translation restoration.

Abstract PS11-22: Lipophilic statins for prolonging dormancy and overall survival: A real-world retrospective investigation

Abstract Background: Micrometastases from breast cancer (BC) might undergo quiescence following adjuvant therapies. Oftentimes they reactivate, prompting metastatic relapse and aggressive, uncurable disease. It is necessary to find alternative, safe therapies that could be used as adjuvants for delaying the dormancy process and reduce metastatic breast cancer (MBC) progression. Prior epidemiological studies found prolonged overall survival from chronic statin use. However, the effectiveness of statin use through different stages of progression, including advanced MBC, has not yet been defined. Methods: A real-world, retrospective analysis of 1,016 deceased patients with MBC treated from 1999-2019 at the University of Pittsburgh Medical Center (UPMC). Lipophilic statin (atorvastatin and simvastatin) use and dosing were retrieved from medical records through an IRB-approved R3 request and merged with already available clinical variables. A total of 122 patients were included in the analysis as the statin use group. This included patients who were prescribed statins through the course of their breast cancer. For measuring time to relapse, only patients with statin prescription before being diagnosed with MBC were selected. Log-ranks were used to compute survival. Uni- and multi-variate Cox hazard models were also applied. Statistical analysis was computed using the package ggpubr on R v3.6.3.Results: Women who used statins were older at the time of detection of primary (60 vs 52 years of age, respectively) and MBC (66 vs 56 years of age, respectively). An almost 2-fold reduction was found in the onset of liver metastasis as first relapse in the statin group, compared to general population (5.7% vs 10.4%, respectively). Increased overall survival (OS) was found among statin users who had ER (+) (p<0.05), and HER2 (-) (p<0.05) BC. Statins were associated with an increase in time from diagnosis until relapse, in ER (+) (p=0.03) and HER2(-) (p=0.02) patients with BC. Patients with ER (-), HER2 (-) MBC also seemed to partially benefit from statin use (p=0.09). Multivariate analysis adjusted for age, ER-status, statin use, and adjuvant chemotherapy also predicted increased OS for patients using statins. No differences in survival were seen when measuring time from metastatic diagnosis to death, suggesting that statins may be effective in prolonging dormancy but lack benefit against relapsing MBC.Conclusion: Lipophilic statins may prolong dormancy and overall survival but not slow the progression of relapsing metastatic breast cancer. Citation Format: Juan Luis Gomez Marti, Alan Wells. Lipophilic statins for prolonging dormancy and overall survival: A real-world retrospective investigation [abstract]. In: Proceedings of the 2020 San Antonio Breast Cancer Virtual Symposium; 2020 Dec 8-11; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2021;81(4 Suppl):Abstract nr PS11-22.

Cancer Stem Cells-Key Players in Tumor Relapse.

Simple SummaryCancer is one of the hardest pathologies to fight, being one of the main causes of death worldwide despite the constant development of novel therapeutic strategies. Therapeutic failure, followed by tumor relapse, might be explained by the existence of a subpopulation of cancer cells called cancer stem cells (CSCs). The survival advantage of CSCs relies on their ability to shape their phenotype against harmful conditions. This Review will summarize the molecular mechanisms exploited by CSCs in order to escape from different kind of therapies, shedding light on the potential novel CSC-specific targets for the development of innovative therapeutic approaches.Tumor relapse and treatment failure are unfortunately common events for cancer patients, thus often rendering cancer an uncurable disease. Cancer stem cells (CSCs) are a subset of cancer cells endowed with tumor-initiating and self-renewal capacity, as well as with high adaptive abilities. Altogether, these features contribute to CSC survival after one or multiple therapeutic approaches, thus leading to treatment failure and tumor progression/relapse. Thus, elucidating the molecular mechanisms associated with stemness-driven resistance is crucial for the development of more effective drugs and durable responses. This review will highlight the mechanisms exploited by CSCs to overcome different therapeutic strategies, from chemo- and radiotherapies to targeted therapies and immunotherapies, shedding light on their plasticity as an insidious trait responsible for their adaptation/escape. Finally, novel CSC-specific approaches will be described, providing evidence of their preclinical and clinical applications.

Solution to support informal caregivers of patients with dementia

Abstract The dementia is a degenerative uncurable disease. Giving its high prevalence worldwide, it is considered a major global public health concern. Patients with dementia need long-term care and support at home. Current evidence demonstrates that their informal caregivers are prone to psychiatric and cardiovascular diseases and lack formal support that can help them in their exhausting daily routine. We intend to report the creation of an ICT solution to support informal caregivers of patients with dementia, addressing identified needs. The academic engineering project launched was divided in two phases: 1) requirements analysis and software design and 2) software development and deployment. The user-friendly and smooth functioning application developed, called OneCare, has a set of functionalities that allows the free access to information about the disease, useful resources, medication, outpatient consultation management and communication with peers. We consider that this application may improve effectiveness in care provided by informal caregivers of patients with dementia, while having a positive impact in their health outcomes and quality of life. We advocate that future policies in this area should consider the use of this kind of technology as a way to reduce disease burden, institutionalization needs and increase the chances of living well at home.