Gene therapy for patients with a severe form of the blood disorder beta-thalassemia could be safe and effective as well as end the need for transfusions, results from the clinical trials show.
Beta-thalassemia is a genetic disorder that impairs the body’s ability to produce a key component of hemoglobin — a critical protein in red blood cells that carries oxygen to organs and tissue.
People with the most severe forms of beta-thalassemia require monthly blood transfusions, right from their toddler age, to replenish their red blood cell supplies along with iron chelation to remove extra iron from the body.
Frequent transfusions, however, can cause serious complications due to iron toxicity and viral infections.
The results, published in New England Journal of Medicine, showed that a one-time treatment with the gene therapy known as LentiGlobin BB305 vector reduced or eliminated the need for blood transfusions in 22 patients with severe beta-thalassemia.
“We saw remarkable outcomes using LentiGlobin gene therapy, with most patients no longer needing monthly transfusions,” said lead author Alexis Thompson, from Ann & Robert H. Lurie Children’s Hospital of Chicago.
“These study results exceeded our expectations with clinical benefit for nearly all patients and suggest that gene therapy may be an effective treatment for thalassemia in the future,” Thompson added.
Of the 22 patients aged between 12 and 35 years, 15 patients achieved transfusion-free status, while others needed transfusions less often.
All the patient underwent chemotherapy, after which they were infused with their own stem cells that were treated in the lab with a modified virus to replace the gene that is defective in thalassemia. Patients typically reached peak production of hemoglobin in 9-12 months after infusion.
“While these gene therapy trials were the largest for thalassemia to date, we need to evaluate effectiveness in a much larger population,” Thompson said, adding that they “are now enrolling patients as young as five-years-old on a Phase 3 trial”.
Never ignore the common signs of A Heart Attack in Women
Every person knows the common signs of Heart attack is chest pain. It’s not like how it is shown in movies where a man is shown gasping for breath, clutches his chest and falls on the ground. When it comes to real life, the symptoms of heart attack are more than just pain in the chest. Yes, chest pain is a symptom of heart attack, but there are other subtle signs of cardiovascular problems, which are important to know. As per studies, women do mostly feel chest pain when they suffer from a cardinal problem, there are few other signs you should be cautious about. If these signs are overlooked then it can even turn fatal.
The common signs of a heart attack one should not ignore in women
- Do you feel uncomfortable pressure in your chest?
One of the most common signs of a Heart attack in women. If you are feeling pressure and tightness around your chest, then ask for help. Pain can happen anywhere in the chest, it is not necessary to be the middle of the heart. Do not brush off the situation just because the pain is on the left side.
- Breathing Difficulty
Uneasiness and difficulty in breathing is another sign of heart attack in Women. If you are not able to catch your breath and move around even a little bit, then it is an indicator that something is not right with your heart.
Sweating on a sunny day or due to intense workout is normal, but if it is random then you should immediately call someone for help. Profuse and sudden sweating can be a sign of a cardiovascular problem. This sign is easily confused with night sweats or hot flashes, which is common with age Overlooking this can be dangerous for you.
- pain experienced in both the arms
It is not necessary that pain be experienced only in the chest or in the middle of the heart. At times it can even be on the left or right arm, or even in the upper abdomen. It is important to note that any type of pain above the waist could be due to a heart problem. The pain could be irregular or intense
- The most common sign Dizziness
Nausea and vomiting are common symptoms in women. These signs of a heart attack are mostly confused with food poisioned or gastrointestinal issues. But if you are experiencing nausea and vomiting along with pain in the upper part of the body, then it is time immediately rush to the hospital.
One feels very exhausted, but just like other signs of heart attack, if you feel excessively tired than usual then you get yourself checked. You would actually feel overwhelmed and would not be able to perform any other activity. This sign is often mistaken for anxiety. If you suddenly feel fatigued and uneasy then speak to your practitioner.
Cutting screen time may reverse sleep problems in teens
Limiting exposure to blue-light emitting devices such as phones and laptops in the evening for just a week can help teenagers improve their sleep quality and reduce symptoms of fatigue, lack of concentration and bad mood, a study has found.
Recent studies have indicated that exposure to too much evening light, particularly the blue light emitted from screens on smartphones, tablets and computers can affect the brain’s clock and the production of the sleep hormone melatonin, resulting in disrupted sleep time and quality.
The lack of sleep does not just cause immediate symptoms of tiredness and poor concentration but can also increase the risk of more serious long-term health issues such as obesity, diabetes and heart disease.
Other studies have suggested that sleep deprivation related to screen time may affect children and adolescents more than adults, but no studies have fully investigated how real-life exposure is affecting sleep in adolescents at home and whether it can be reversed.
Researchers from Netherlands Institute of Neuroscience, the Amsterdam UMC and the Dutch National Institute for Public Health and the Environment, investigated the effects of blue light exposure on adolescents at home.
Those who had more than four hours per day of screen time had on average 30 minutes later sleep onset and wake up times than those who recorded less than one hour per day of screen time, as well as more symptoms of sleep loss.
The team conducted a randomised controlled trial to assess the effects of blocking blue light with glasses and no screen time during the evening on the sleep pattern of 25 frequent users.
Both blocking blue light with glasses and screen abstinence resulted in sleep onset and wake up times occurring 20 minutes earlier, and a reduction in reported symptoms of sleep loss in participants, after just one week.
“Adolescents increasingly spend more time on devices with screens and sleep complaints are frequent in this age group,” said Dirk Jan Stenvers from the Amsterdam UMC.
“Here we show very simply that these sleep complaints can be easily reversed by minimising evening screen use or exposure to blue light,” Stenvers said.
“Based on our data, it is likely that adolescent sleep complaints and delayed sleep onset are at least partly mediated by blue light from screens,” he said.
Common chemicals can increase risk of metabolic disorders
Do you know that your everyday exposure to everyday harmful chemicals can land you into serious trouble?
A recent study has found that people exposed to chemicals called Phthalates, increasing the risk of metabolic disorders. The study was discussed in the meeting, ‘ECE 2019’. Researchers found a correlation between levels of phthalate exposure and markers of impaired liver function, which are indicators of increased risk of obesity, diabetes and heart disease.
These findings suggest that more actions may need to be taken to reduce people’s exposure to these potentially harmful, yet commonly used chemicals. Phthalates are common additives used in manufacturing to produce plastics and they can be found in numerous everyday items including milk, bottled water, instant coffee, perfume, makeup, shampoo, toys and food packaging.
Exposure to endocrine-disrupting chemicals has previously been implicated in causing serious harm to fertility and development, as well as increased obesity risk in rodents and people.
However, no studies have directly investigated how Phthalate exposure is associated with obesity and metabolism. In this study, Professor Milica Medi Stojanoska, one of the researchers correlated the levels of Phthalate absorbed by people with their body weight, type 2 diabetes incidence and markers of impaired liver and metabolic function.
Higher exposure to the chemical was associated with increased markers of liver damage, insulin resistance and cholesterol in people with obesity and diabetes.
Prof Stojanoska says, “Although a small association study, these findings suggest that not only do phthalates alter metabolism to increase the risk of obesity and diabetes but that they are also causing toxic damage to the liver.”
Prof Stojanoska’s research is now looking at the effects of endocrine-disrupting chemicals on human health in adults, adolescents and babies.
“We need to inform people about the potential adverse effects of endocrine disruptors on their health and look at ways to minimise our contact with these harmful chemicals,” adds the professor.