The Health Thread

Nosebleeds, or epistaxis, are a common medical issue affecting approximately 60% of people in the United States at some point in their lives. Although most episodes are minor and self-limiting, around 6% of individuals experiencing nosebleeds will seek medical attention (Tunkel et al., 2020). In children, epistaxis is particularly prevalent, with 75% having at least one episode (Tunkel et al., 2020). There are two primary types of nosebleeds: anterior, which is more common, and posterior, which, although less frequent, often require medical intervention (Tabassom & Dahlstrom, 2024).

Epistaxis, despite often being seen as a mere nuisance, can occasionally pose life-threatening risks, particularly in resource-limited settings where adequate healthcare facilities are scarce. It is estimated that 60% of the global population will experience epistaxis, with about 6% requiring medical treatment due to the ineffectiveness of home remedies (Adoga et al., 2019).

Causes of Epistaxis

The most common cause of epistaxis are idiopathic (38.09%) followed by hypertension (27.38%), trauma (15.47%), and coagulopathy (8.33%) (Parajuli R, 2015)

Other Local causes are:

Anatomic deformities

Intranasal tumors

Low humidity

Vigorous nose blowing

Nose picking

In adults, medications such as non-steroidal anti-inflammatory drugs (NSAIDs) and anticoagulants like heparin and warfarin are common contributors. Hereditary bleeding disorders, including hemophilia A, hemophilia B, and von Willebrand disease, are also associated with epistaxis (Ameya et al., 2021).

Additionally, chronic vascular damage related to hypertension has been suggested as a potential mechanism linking high blood pressure to nosebleeds (Byun et al., 2021).

Management and Treatment of Epistaxis

Following steps can be used at Home. This method is also called Hippocratic method

1. Sit upright and lean slightly forward to prevent blood from running down your throat, which can cause nausea, vomiting, and diarrhea. Avoid lying flat or tilting your head back.
2. Breathe through your mouth.
3. Use a tissue or damp washcloth to catch the blood.
4. Pinch the soft part of your nose with your thumb and index finger, pressing it against the hard bony ridge that forms the bridge of your nose. Pinching above or on the bony part won’t effectively stop the bleeding.
5. Maintain pressure on your nose for at least five minutes before checking if the bleeding has stopped. If it persists, continue pinching for another 10 minutes.
6. Optionally, apply an ice pack to the bridge of your nose to help constrict blood vessels and provide comfort. This step is not essential but can be helpful (Cleveland Clinic).

Managing epistaxis requires a thorough examination and detailed patient history to identify the bleeding site and cause. Treatment methods vary depending on the location, severity, and etiology of the bleeding and can be broadly categorized into nonsurgical and surgical approaches. Simple measures include pinching the nose, while more severe cases might require ligation of vessels (Parajuli, 2015).

The majority of nosebleeds are acute, sporadic, and self-limited, typically responding to simple compression but sometimes requiring more aggressive measures like cautery (can be chemical or electric) or nasal packing. Conventional gauze pack and Merocel nasal pack are the common pack used in refractory anterior epistaxis (Shanmugam et.al, 2019)

Vasoconstrictors, such as oxymetazoline, xylometazoline can help locate the bleeding site. If simple measures fail, tranexamic acid, nasal cautery with silver nitrate, or nasal packing may be necessary. (Director, Paediatric Emergency Department, 2023). Endoscopic ligation of the sphenopalatine artery is done in case of persistent bleeding (Snyderman & Carrau, 1997).

If there is persistent bleeding then endoscopic ligation of the bleeding vessel is done.

• SPA ligation has been reported to be effective in 87-92% of cases (Kishimoto 2018, Wormald 2000).
• Bilateral SPA ligation has been shown to have lower rebleeding rates compared to unilateral ligation (Hervochon 2018).
• SPA ligation may reduce the risk of future severe epistaxis in anticoagulated patients.

Exploring effective treatment options for end-stage renal disease (ESRD) has led to the development of innovative technologies such as implantable bio artificial kidneys (BAK) and kidney regeneration. These advancements are not just impressive achievements; they are sources of hope for millions around the globe.

Implantable Bio artificial Kidney (BAK): A Game Changer

Imagine a kidney replacement that’s like having a tiny, high-tech sidekick doing all the hard work for you. That’s the dream behind the implantable BAK. Dr. William H. Fissell and Shuvo Roy, Ph.D., are the masterminds behind this marvel. Picture a device no bigger than a soda can, but with the power to mimic your kidney’s functions. It hooks up to your blood vessels, acting like a natural kidney without the hassle of dialysis or meds.

The Kidney Project: Making Sci-Fi a Reality

The Kidney Project, a tag team effort between Vanderbilt University Medical Center and the University of California San Francisco, has been the driving force behind the BAK’s evolution. Their latest prototype is a real showstopper. It’s proven it can keep kidney cells alive inside a bioreactor, essentially acting as a mini kidney. The silicon membranes protect these cells like armor, ensuring they keep ticking away. (Kim et al., 2023)

Preclinical Success and What’s Next

Recent trials have been a roaring success. The BAK operates silently in the background, much like a superhero, without setting off alarms in the recipient’s immune system. This means it could be the ticket to freedom from dialysis and the endless wait for donor kidneys. . (Kim et al., 2023)

Kidney Regeneration Tech: Healing Magic

But wait, there’s more! While the BAK steals the spotlight, kidney regeneration tech is quietly making waves. Scientists have stumbled upon a magic trick: block a pesky protein called interleukin-11 (IL-11), and damaged kidney cells start to regrow. It’s like hitting the rewind button on kidney damage caused by diseases or injuries. (Widjaja et al., 2022)

The Future’s Bright for Kidney Care

Combine the power of BAK with regeneration tech, and you’ve got a winning combo. The BAK offers immediate relief for those in dire need, while regenerative therapies work their magic over time, restoring natural kidney function.

Challenges and the Big Picture

Sure, these innovations are thrilling, but there are hurdles to jump. We need to make sure the BAK is safe and effective for humans and fine-tune regeneration therapies. Plus, we can’t forget about making these treatments accessible and affordable for everyone.

In Conclusion: Hope on the Horizon

The birth of the BAK and kidney regeneration tech is like finding a pot of gold at the end of the rainbow for kidney disease sufferers. These breakthroughs promise a brighter future, where kidney failure isn’t a life sentence. It’s a journey filled with obstacles, but the destination—a world free from the grip of kidney disease—is within reach.

Spinal cord injury (SCI) is a devastating condition that can cause permanent loss of function and affect mobility, senses, and many other bodily functions.Globally 15 million people are living with SCI with the majority of cases due to preventable trauma ( WHO,2024). Beyond the physical limitations, SCI also has a profound impact on the psychological well- being of individuals. Adults living with SCI have a significantly raising risk of depression and anxiety (Peterson et al., 2022). Furthermore, SCI imposes a substantial financial burden on society. The estimated lifetime burden of  per individual with SCI ranges from 1.5 to 3.0 million due to long term care and loss of employment ( Diop, Epstein, & Gaggerro, 2021)  Despite significant advances in medical technology and rehabilitation techniques, SCI continues to face many challenges in treatment and recovery. However, recent research has revealed new approaches and treatments that may improve outcomes for patients with SCI.

Stem Cell Therapy: Building New Pathways

One of the most promising areas of SCI research is the use of stem cells. Stem cells are unique because they are like versatile building blocks that can become different types of cells, including the nerve cells (neurons) that make up the spinal cord. Researchers are investigating the use of different stem cell types in the treatment of SCI, with some of the most common being:

• Mesenchymal stem cells (MSCs): These cells are found in bone marrow and can develop into several cell types, including bone, cartilage, and fat cells. In SCI research, MSCs have shown promise in promoting nerve regeneration and reducing inflammation.
• Neural stem cells (NSCs): These stem cells are already on the path toward becoming cells of the nervous system. NSCs hold the potential to directly replace damaged neurons and help rebuild the communication pathways in the injured spinal cord.

A recent study published in Stem Cell Reports showed that transplanting stem cells called mesenchymal stem cells (MSCs) improved the ability to move and promoted nerve regeneration in rats with SCI (Wang et al., 2021). The researchers found that MSCs helped new nerve cells grow, improved the overall health of the spinal cord, and even contributed to forming new connections across the injury site.

Another study recently published in Nature Communications showed that transplanting neural stem cells (NSCs) improved bladder function in rats with SCI (Chen et al., 2020). The researchers found that the NSCs transformed into neurons that became part of the spinal cord circuitry, improving signaling between the bladder and the brain.

Boosting Nerve Growth with Neurotrophic Factors

In addition to stem cells, researchers are also investigating the use of neurotrophic factors in the treatment of SCI. Neurotrophic factors are like special “fertilizers” for nerve cells, supporting them in multiple ways:

• Promoting Growth: They stimulate the development of new neurons and encourage the branching of nerve fibers, helping them form connections.
• Supporting Survival: Neurotrophic factors help existing neurons stay healthy and function optimally.
• Reducing Inflammation: Some neurotrophic factors can help calm the excessive inflammation that occurs after a spinal cord injury.

A recent study published in the journal Nature Communications showed that administering a neurotrophic factor called brain-derived neurotrophic factor (BDNF) improved the ability to move and promoted nerve regeneration in rats with SCI (Li et al., 2021). BDNF helped new neurons grow, encouraged connections within the injured spinal cord, and improved the overall health of nerve tissue.

Electrical Stimulation: Re-wiring the Connection

In addition, researchers are also investigating the use of electrical stimulation in the treatment of SCI. Electrical stimulation involves the use of electrical currents to stimulate nerves and muscles. This type of stimulation is already used in other areas of medicine, such as pacemakers for the heart, and researchers are exploring how it could be adapted to help in the recovery from spinal cord injury.

A recent study published in Scientific Reports showed that the use of electrical stimulation improved the ability to move and promoted nerve regeneration in rats with SCI (Zhang et al., 2020). The researchers believe that electrical stimulation works by encouraging surviving nerve fibers to sprout new branches, facilitating the formation of alternative signal pathways around the damaged area.

Calming the Immune Response for Better Healing

In addition to these treatments, researchers are also investigating the role of immune cells in SCI. SCI triggers a complex immune response within the body, and while some aspects of this response are helpful for healing in the acute phase, prolonged inflammation can actually further damage the spinal cord. Researchers are investigating ways to modulate the immune response (adjust its activity) to improve healing and reduce long-term damage.

A recent study published in the journal Nature Neuroscience showed that targeting a type of immune cell called microglia improved motor function and nerve regeneration in mice with SCI (Zhou et al., 2021). Microglia are like the clean-up crew of the nervous system, but after injury, they can become overactive and contribute to tissue damage. This study suggests that finding ways to calm microglia activity could be a beneficial treatment strategy.

Hope for the Future

In summary, recent studies have identified several promising treatments for SCI, including stem cell transplantation, neurotrophic factors, electrical stimulation, and immune modulation. Although these therapies are still in development, they hold great promise for improving outcomes for patients with SCI. Further research is needed to fully understand these treatment options, optimize their delivery, and develop safe and effective treatments for SCI. 

Pancreatic cancer is a tough and dangerous type of cancer that’s hard to treat and often doesn’t have a good outcome. But there’s good news: a group of experts from around the world is working hard to create a new program that will help doctors find this cancer early, which could save many lives.

The group called PRECEDE is leading a project that shows how finding pancreatic cancer early could help more people survive it. Right now, not many people survive this cancer worldwide—only about 12 out of 100 do (. Rawla, Sunkara, & Gaduputi, 2019). But if it’s found early, more than 80 out of 100 could survive, especially if they can have surgery. Sadly, most people find out they have this cancer too late when it’s already spread too much. ((MUHC News, 2024)

PRECEDE is working on a better way to keep an eye on people who are more likely to get pancreatic cancer because of their personal or family health history. Dr. George Zogopoulos and his team are focusing on how to check these high-risk people more effectively, especially if they have relatives who had pancreatic cancer or they have genes that could make them get cancer. (fortner, 2024 )

The study shows that people who have a high chance of getting pancreatic cancer are really good at following advice on getting checked. These checks can be done well in places that specialize in health care. This proves that PRECEDE can use this method of checking for cancer all over the world and gather information to learn more and get better at watching for signs of cancer in patients. (MUHC News, 2024)

 Based on their findings, the researchers suggest putting people who might get pancreatic cancer into three groups. These groups are for people who have a family history of the disease, those who have a genetic mutation that could cause cancer, or those who have both these risk factors. If someone is worried they might be at risk for pancreatic cancer, they can join the PRECEDE program and go to one of its centers in North America or Europe to get checked and learn more about their risk.

The study found that people who are at high risk for pancreatic cancer just because of their family history are more likely to have cysts in their pancreas than those who have a genetic change known to cause cancer but no family history. These cysts might mean that these individuals could be more likely to develop pancreatic cancer as time goes by. This could happen either because the cysts themselves change or because the cysts are a sign that the pancreas is more likely to develop problems that could turn into cancer. Zogopoulos et al., 2024)

We need more time to watch and see if having family members with pancreatic cancer means a person is more likely to get it themselves, compared to just having a gene change that can cause cancer Zogopoulos et al., 2024). The study points out that even though it’s been hard to set up big screening programs for people at high risk of pancreatic cancer, it’s possible to do this kind of research with many centers working together across different countries. The first results from the scans in this study show that we need to keep researching how to find pancreatic cancer early. (Fortner, 2024).

Besides other methods, artificial intelligence tools are helping a lot in the battle against pancreatic cancer. One of these programs was able to pick out the people who were most likely to get pancreatic cancer, up to three years before they were actually diagnosed, just by looking at their health records Pesheva, 2023).This big step forward in being able to predict health issues was made possible by researchers from Harvard Medical School and the University of Copenhagen working together with the VA Boston Healthcare System, Dana-Farber Cancer Institute, and the Harvard T.H. Chan School of Public Health.

Using AI to check for pancreatic cancer could really change how we find and treat this illness. It’s a way that doesn’t hurt, doesn’t cost much, and is really good at spotting people who might have it (Huang et al., 2022). For example, AI can look very closely at CT scans and MRIs to find tiny signs of cancer that people might not notice (Katta et al., 2023). It can also help figure out if cysts in the pancreas might turn into cancer later on. (Jiang, Chao, Culp, & Krishna, 2023)

At the same time, AI is also changing the way we look for signs of pancreatic cancer in blood tests. It can find special markers in the blood that might mean someone has pancreatic cancer and understand complicated genetic information to figure out who might be more likely to get the disease (Tripathi et al., 2024). Another thing AI does well is look through lots of health records to find hidden patterns that show who might be at risk. This helps doctors decide who really needs to be checked for pancreatic cancer. (Tripathi et al., 2024)

The important parts of using AI in checking for pancreatic cancer—like looking at images, finding markers in the blood, and studying health records—are all connected. They’re part of a big plan that uses AI to make sure we find pancreatic cancer early and accurately. This could help patients get better treatment sooner and have a better chance of surviving.

The work that PRECEDE is doing, together with the use of AI, gives us a lot of hope for how we’ll be able to handle pancreatic cancer in the future. Creating a strong program to watch for this cancer isn’t just about science; it’s also a sign of hope for people who might get pancreatic cancer. It shows how working together across countries and never giving up on finding new solutions can make a big difference, even when things are tough.

The ongoing research is bringing us closer to the goal of making pancreatic cancer something we can treat instead of something that can’t be cured. The hard work and commitment of the scientists, doctors, nurses, and patients involved in this research are what’s making this progress possible. If we keep supporting and funding research that helps us detect pancreatic cancer early, we might reach a time when this disease isn’t so scary anymore.

In the end, the work being done by a global team to watch for pancreatic cancer is a huge leap in fighting this illness. The PRECEDE research and the use of AI show us what the future could look like, where we can find and stop pancreatic cancer early. We still have a long way to go in this fight, but these new tools make us more ready than ever to face it. The research that keeps going on is very important, and everyone is watching and hoping as we head into a new time of dealing with pancreatic cancer.

What is CHD?

Coronary Heart Disease (CHD) is a health condition that involves the constriction or obstruction of the coronary arteries, which are responsible for delivering oxygenated blood to the heart muscle. This constriction or obstruction is caused by the accumulation of fatty substances known as plaques on the inner lining of the arteries, a condition referred to as atherosclerosis. There are several types of plaques and some of the plaques are less likely to rupture because they have a thick fibrous cap with a small lipid core (LC) area . While unstable and vulnerable plaques have been characterized by several studies which indicate that they have a thin fibrous cap (< 65 µm) and its LC is substantial . If plaque ruptures in the carotid artery, it will either block the oxygenated blood from reaching the brain or bleed, which will lead the brain cells to die.[11] Over time, these plaques can limit the flow of blood to the heart, resulting in various symptoms such as angina (chest pain), breathlessness, and in severe instances, myocardial infarction (heart attack) [1].

CHD is a progressive disease that develops over several years, often due to a combination of factors, including genetic predisposition, lifestyle habits, and pre-existing health conditions. Risk factors for CHD encompass age, gender (men are generally at a higher risk, particularly before menopause), family history of heart disease, smoking, hypertension, hypercholesterolemia, diabetes, obesity, sedentary lifestyle, unhealthy diet, and chronic stress [2].

Why is CHD a Pandemic?CHD has escalated to pandemic levels due to a multitude of interconnected factors, ranging from lifestyle habits to global health disparities. Here’s an explanation of why CHD has attained pandemic status, supported by citations:

Global Prevalence: As individuals age, their arteries tend to stiffen and become more susceptible to damage, thereby increasing the risk of CHD [2].

Increasing Burden in Developing Countries: Low- and middle-income countries (LMICs) shoulder a significant burden of CHD, with over three-quarters of cardiovascular disease (CVD) deaths occurring in these regions [2]. Rapid urbanization, the adoption of unhealthy western lifestyles, and limited healthcare access contribute to the rising prevalence of CHD in LMICs.

Common Risk Factors: Shared risk factors such as tobacco use, unhealthy diet, physical inactivity, obesity, hypertension, and diabetes contribute to the global spread of CHD [3]. These risk factors are common across various populations and contribute to the worldwide proliferation of CHD.

Economic and Healthcare Systems Impact: CHD imposes a substantial economic burden on societies, encompassing direct medical costs, lost productivity, and social welfare losses. The World Heart Federation estimates the annual cost of CHD to be in the billions of dollars, with projections indicating a further increase in costs over time [4].

Health Inequities: Disparities in access to healthcare and preventive services exacerbate the impact of CHD, particularly in underserved communities and marginalized populations. Limited access to affordable healthcare, preventive interventions, and treatment modalities perpetuates the cycle of CHD burden in vulnerable populations [5].

Environmental and Social Determinants: Environmental factors such as air pollution, noise pollution ,inadequate urban planning, and exposure to toxins contribute to the development of CHD. Social determinants of health, including poverty, education level, and social support networks, also play a significant role in shaping CHD risk [6].

Globalization of Unhealthy Lifestyles: Globalization has facilitated the spread of unhealthy lifestyles characterized by sedentary behavior, poor dietary choices, and increased stress levels. These lifestyle factors contribute to the rising incidence of CHD worldwide, transcending geographical boundaries [7].

Current Status of CHD in Nepal

Coronary Heart Disease (CHD) in Nepal currently poses a significant public health challenge, underscored by increasing prevalence rates and associated risk factors. As per data from the World Health Organization (WHO), CHD contributes to a considerable portion of Nepal’s disease burden, accounting for approximately 12.26% of total deaths, with an age-adjusted death rate of 102.19 per 100,000 population [1].

Emerging epidemiological data suggests a worrying trend of rising CHD prevalence in Nepal. A study conducted in urban Kathmandu revealed a prevalence of 5.9%, indicating a significant burden of cardiovascular diseases in urban areas [2]. Moreover, recent findings indicate that the prevalence of smoking, hypertension, diabetes, and dyslipidemia among the population aged 40 to 80 years in rural Nepal was 27.8%, 34.4%, 6.9%, and 38.5%, respectively, highlighting the multifactorial nature of CHD risk factors in the Nepalese population [3].

Furthermore, the shift towards sedentary lifestyles, urbanization, and dietary changes exacerbates the prevalence of CHD. These factors, compounded by limited access to healthcare services, especially in rural regions, pose significant challenges to effective CHD management [4].

Addressing the Burden of CHD in Nepal

Addressing the burden of Coronary Heart Disease (CHD) in Nepal necessitates a comprehensive approach, drawing insights from strategies employed in both developed and developing nations. Here are some potential strategies for improvement, incorporating insights from global initiatives and interventions tailored for Nepal:

Health Education and Awareness Campaigns: The implementation of public health campaigns to raise awareness about CHD risk factors and promote healthy lifestyle choices is crucial. This strategy has proven effective in various contexts, including developed countries like the United States and Europe [1]. In Nepal, community-based health education programs can target both rural and urban populations, emphasizing the importance of quitting smoking, adopting healthy dietary habits, engaging in regular physical activity, and managing hypertension [2].

Access to Healthcare Services:  Due to fragmented health care systems in many LMICs(Low and Middle Income Countries), many patients are unaware of the disease and disease symptoms resulting in the delay of the care-seeking behavior. People from remote areas and limited access to advanced technology are more prone to suffer. The limited ambulance services in these parts also play a major role in the delay.Apart from the delay in reaching the primary care centers or hospitals, the lack of specialists and inadequate medical facilities hinder the delivery of proper and timely care. [10]Enhancing access to healthcare services, especially in rural areas, is vital for effective CHD management. Telemedicine facilities and mobile clinics can help bridge the gap in healthcare access, as demonstrated in countries like India and Bangladesh [3]. In Nepal, initiatives to establish primary healthcare clinics in remote regions and promote telemedicine consultations can enhance CHD diagnosis, treatment, and follow-up care [4].

Risk Factor Modification:  Early identification of pre-diabetic and pre-hypertensive condition and applying appropriate dietary and behavioural measures will be crucial .Encouraging lifestyle modifications to reduce CHD risk factors is paramount. Developed countries have implemented policies to regulate the availability of unhealthy foods and promote smoke-free environments [5]. In Nepal, advocating for tobacco control measures, promoting healthy dietary patterns rich in fruits, vegetables, and whole grains, and facilitating access to affordable medications for hypertension and dyslipidemia can help mitigate CHD risk [6].

Policy Interventions: Implementing policies to regulate unhealthy behaviors and strengthen healthcare infrastructure is vital. Examples include taxation on tobacco products, legislation on trans-fat content in foods, and investments in healthcare workforce training and facility development [7]. In Nepal, aligning with the Multisectoral Action Plan for the Prevention and Control of Noncommunicable Diseases (2014–2020) and integrating CHD prevention strategies into primary healthcare systems can drive sustainable improvements [8].

Early Detection and Management: Enhancing screening programs for early detection of CHD risk factors and ensuring timely management of the condition are critical. Evidence-based treatment protocols and risk-based management approaches, as outlined in global initiatives like the HEARTS Technical Package, can guide healthcare providers in Nepal [9]. Strengthening data collection systems and integrating cardiovascular risk assessment tools into routine clinical practice are also essential steps.

By integrating these strategies into a comprehensive national CHD prevention and control program, Nepal can make significant strides in reducing the burden of CHD and improving cardiovascular health outcomes across diverse populations.

Glioblastoma (GBM), the most common primary malignant brain tumor, presents a daunting challenge in clinical management, with a meager 5-year survival rate of merely 5% [1]. Despite advancements in treatment modalities such as surgery, radiation, and chemotherapy, the prognosis remains dismal due to tumor heterogeneity and the intricate interplay between tumor cells and normal brain tissue, compounded by the impermeable blood-brain barrier.

Conventional therapies, including surgical resection, radiotherapy, and chemotherapy, have shown limited efficacy, resulting in high relapse rates and poor patient outcomes [2]. Therefore, there is an urgent need for innovative therapeutic approaches to combat GBM effectively.

Chimeric Antigen Receptor T (CAR-T) cell therapy, heralded for its success in hematological malignancies, has emerged as a promising avenue for solid tumors like GBM [3]. However, its application faces formidable challenges posed by the unique anatomical features of GBM, including the blood-brain barrier and the immunosuppressive tumor microenvironment, alongside tumor heterogeneity.

CAR-T therapy involves the extraction of T lymphocytes from the patient’s peripheral blood, genetic modification to express chimeric antigen receptors targeting specific tumor antigens, and reinfusion into the patient, thereby harnessing the immune system to target and destroy cancer cells [4].

The manufacturing process of CAR-T cells entails several meticulous steps, including T cell isolation, genetic engineering to introduce CAR genes, in vitro activation and expansion, and rigorous quality assessment before clinical administration [5].

While CAR-T therapies have demonstrated remarkable efficacy in hematologic malignancies, their application in solid tumors has been limited. The complexity of solid tumors, characterized by diverse cell populations, presents challenges in achieving sustained therapeutic responses [6].

Innovative strategies combining CAR-T therapy with bispecific antibodies, such as T-cell Engaging Antibody Molecules (TEAMs), hold promise in overcoming the hurdles posed by tumor heterogeneity [7]. These approaches aim to enhance the specificity and potency of CAR-T cells against solid tumors like GBM.

Recent clinical studies have reported encouraging outcomes with CAR-T therapy in GBM patients, demonstrating reduced tumor sizes and prolonged survival [8]. Notably, early-phase trials employing dual-target CAR-T cells, engineered to recognize multiple tumor-associated antigens, have shown promising results in shrinking tumors and extending patient survival [9].

Despite the progress, CAR-T cell therapy in GBM is not without limitations, with treatment-associated toxicities, including cytokine release syndrome and central nervous system complications, warranting careful monitoring and management [10].

In conclusion, the innovative field of CAR-T cell therapy is at the forefront of transforming the treatment paradigm for glioblastoma multiforme (GBM). The journey from preclinical studies to clinical trials has been fraught with challenges, yet these hurdles have been instrumental in uncovering and addressing the complexities of successful cancer treatment. The advent of CAR T cells equipped with multivalent receptors, combined with cutting-edge therapies, is tackling the problem of antigen escape [11]. Furthermore, the synergy of CAR T cell therapy with other treatments, such as immunotherapies, chemotherapies, or mechanical tumor ablation, is poised to foster a more inflammatory microenvironment conducive to better patient outcomes [11]. As research continues to refine these therapeutic strategies, there is a growing hope for significant advancements in the battle against this formidable disease.

Rare kidney diseases pose significant challenges to patients worldwide, often leading to chronic debilitation, morbidity, or even death. These conditions, characterized by low prevalence and genetic or metabolic origins, demand specialized care and concerted efforts to enhance awareness, diagnosis, and treatment accessibility (Iyengar et al., 2023). This article explores the landscape of rare kidney diseases, focusing on the challenges faced by children, common elements in these conditions, and the imperative need for advocacy and research initiatives.

Rare Kidney Diseases in Children: A Call for Action

Rare kidney diseases affect a substantial number of children globally, with a prevalence of 60–80 cases per 100,000 population in Europe and the United States. These diseases include over 150 different conditions, many of which manifest in childhood (Iyengar et al., 2023). Children with rare kidney diseases often face serious morbidity and require lifelong treatment. However, inequitable access to disease-modifying therapies remains a significant challenge, particularly for children from low- and middle-income countries (LMICs) (Iyengar et al., 2023).

Notable Rare Kidney Diseases:

1. Cystinosis: A rare genetic disease causing cystine accumulation in cells, affecting various organs including the kidneys. Treatment involves cysteamine therapy to reduce cystine levels.
2. Primary Hyperoxaluria Type 1: A genetic disorder causing oxalate buildup in the kidneys, often necessitating combined liver and kidney transplantation.
3. Alport Syndrome: A genetic condition affecting the kidneys, ears, and eyes due to defects in specific genes. It can lead to kidney failure and other complications.
4. Amyloidosis: Characterized by abnormal protein accumulation in organs like the kidneys, heart, brain, liver, and intestines.
5. Polycystic Kidney Disease (PKD): Genetic mutations leading to fluid-filled cysts in the kidneys. Treatment varies based on the type of PKD.

Diagnosis of Rare Kidney Diseases:

Children suspected of having rare kidney diseases are given a thorough clinical assessment by pediatric nephrologists. Symptoms such as swelling, changes in urine output, blood in urine, fatigue, and growth issues are evaluated to determine the need for further testing (National Institute of Diabetes and Digestive and Kidney Diseases, n.d.).

Genetic testing plays a crucial role in diagnosing rare kidney diseases with a genetic basis. Tests like the Genetic Renal Panel screen complement genes to identify genetic mutations associated with conditions like Alport syndrome, cystinosis, or polycystic kidney disease (University of Iowa Hospitals & Clinics, n.d.).

Imaging techniques such as ultrasound are used to visualize the kidneys and detect abnormalities like cysts or structural defects that may indicate specific rare kidney diseases like autosomal recessive polycystic kidney disease (ARPKD) (National Institute of Diabetes and Digestive and Kidney Diseases, n.d.).

Laboratory tests are conducted to assess kidney function, protein levels in urine (proteinuria), blood in urine (hematuria), electrolyte imbalances, and other markers that can provide insights into the underlying condition causing the kidney disease (National Institute of Diabetes and Digestive and Kidney Diseases, n.d.).

Specialized clinics like the Rare Renal Disease Clinic and Renal Genetics Clinic offer comprehensive evaluation and management for children with rare kidney diseases. These clinics provide state-of-the-art genetic testing and access to clinical trials for new therapies (University of Iowa Hospitals & Clinics, n.d.).

By combining these diagnostic approaches under the care of experienced specialists like pediatric nephrologists and geneticists, children with rare kidney diseases can receive accurate diagnoses leading to tailored treatment plans and improved outcomes.

Common Elements in Rare Kidney Diseases

Rare kidney diseases share common challenges such as small patient populations, unidentified disease causes, lack of biomarkers for disease monitoring, and complex care requirements (Aymé et al., 2017). Diagnostic hurdles often delay accurate identification of these conditions, necessitating advanced techniques like genetic testing for precise diagnosis (Aymé et al., 2017).

Advocacy Efforts and Research Initiatives

Enhancing access to diagnostic testing through low-cost genetic testing initiatives and training clinicians in interpreting genetic analyses are crucial steps towards improving outcomes for individuals with rare kidney diseases (Iyengar et al., 2023). Collaborative efforts involving patient support organizations, healthcare providers, researchers, governments, and pharmaceutical industries are essential to drive advancements in diagnosis, treatment accessibility, and research innovation.

In Conclusion

Uniting stakeholders globally and prioritizing the needs of individuals with rare kidney diseases will lead to better outcomes and quality of life. Addressing the challenges posed by rare kidney diseases requires a multifaceted approach encompassing advocacy for equitable access to care, research into innovative treatments, and enhanced awareness initiatives (Rare Kidney Disease Foundation, n.d.).