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Gene therapy has potential to cure thalassemia patients from blood disorder

Thalassemia is a blood disorder that affects the body’s ability to produce red blood cells, and hemoglobin, the protein that carries oxygen in red blood cells. Even with blood transfusion and appropriate iron chelation therapy; patients can develop iron overload, with potential to damage the liver, heart, and endocrine system.

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Gene therapy could pave the way for patients with the inherited blood disorder thalassemia to stop or significantly reduce blood transfusions and transform their daily lives, says an expert at a top American hospital, Cleveland Clinic.

Dr. Rabi Hanna, a pediatric hematologist-oncologist, said: “Thalassemia is a disease that can be cured, with gene therapy as a novel step that uses the patients’ own hematopoietic stem cells to produce healthier red blood cells and fix their blood disorder. Thalassemia patients who have received gene therapy have either eliminated or significantly reduced the amount of blood transfusions needed to manage their condition. With gene therapy, we can remove the challenges that thalassemia patients face to give them the courage to pursue their goals and dreams, whether in education, careers, or families.”

Thalassemia is a blood disorder that affects the body’s ability to produce red blood cells, and hemoglobin, the protein that carries oxygen in red blood cells. Even with blood transfusion and appropriate iron chelation therapy; patients can develop iron overload, with potential to damage the liver, heart, and endocrine system.

There are two type of thalassemia, alpha and beta, depending on the defects that can occur in the protein chains that make up hemoglobin. Patients with alpha thalassemia tend to be silent carriers without symptoms, while patients with beta thalassemia major will have major symptoms early after birth and require frequent red blood transfusions. Moderate and severe thalassemia cases are usually diagnosed with early childhood blood tests. Married couples can also have genetic tests that can predict the risk of thalassemia and related blood disorders.

Worldwide, there are 270 million carriers with abnormal hemoglobin and thalassemia, with 300,000-400,000 babies born with serious hemoglobin disorders annually, according to the US National Institute of Health. It is estimated that 90 percent of those births are in low- or middle-income countries, especially in the Middle East, South and Southeast Asia, the Mediterranean, Africa, and the South Pacific. Recognizing this, International Thalassemia Day’s theme for 2021 is “Addressing Health Inequalities Across the Global Thalassemia Community.”

In contrast to time-consuming, life-long blood transfusions, gene therapy could be a one-time therapy and provide a potential cure. Despite that, the allogeneic bone marrow transplant is currently the only available option with the potential to correct the genetic deficiency in Transfusion-dependent Thalassemia (TDT), but it has possible complications such as graft failure, graft-versus-host disease (GvHD), and opportunistic infections, particularly in patients who undergo non-sibling matched allogeneic HSCT. Gene therapy, in contrast, uses the patient’s own cells and eliminates the risk of GVHD.

The challenge in Dr. Hann’s opinion is how we can make this therapy available worldwide, especially in developing countries, where most of the patients are, globally.

The European Medicines Agency has given conditional marketing authorization to one form of genetically modified products for beta-thalassemia. In the United States, there are currently clinical trials for gene therapy, and this therapy is under review by Food and Drug Administration – which experts hope could lead to authorization later in 2021.

In recent phase 1-2 studies of gene therapy for 22 patients, all of them had reduced or eliminated the need for long-term red blood cell transfusions. Of the 13 alpha-thalassemia patients, 12 stopped receiving red blood cell transfusions. For the nine beta-thalassemia patients, three stopped red blood cell transfusions, and the remaining six patients saw their median annualized transfusion volume decrease by 73 percent.

“While gene therapy is a promising cure for transfusion-dependent thalassemia, many patients do not know the whole process – including chemotherapy to get rid of the old bone marrow and create space for the new modified stem cells. This will currently require admission to hospital for four to 6 weeks until new the stem cells are working and able to produce white blood cells, platelets and healthier red blood cells,” added Dr. Hanna. “We are hopeful that in the future we can target the bone marrow more selectively using reduced intensity chemotherapy or other medication to avoid the acute and long-term toxicity associated with high doses of chemotherapy.”

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Men with sensory loss are more likely to be obese

The association between physical activity and obesity was higher in men with hearing loss, who were 2.319 times more likely to be obese than women who reported difficulty hearing. Obesity in those with sight loss was 1.556 times higher in inactive men than women.

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Men who suffer sensory loss, particularly hearing loss, are more likely to be physically inactive and obese than women, according to a new study published in the European Journal of Public Health.

Researchers analysed data from more than 23,000 Spanish adults, and examined associations with physical inactivity and obesity in people with vision and hearing loss, and explored differences between men and women.

Results suggest inactive people with hearing loss were 1.78 times more likely to be obese compared to those who did not have any hearing loss. In people who had difficulty seeing, the odds ratio is slightly smaller, with a likelihood of obesity being 1.375 times higher than those who did not report vision loss.

The association between physical activity and obesity was higher in men with hearing loss, who were 2.319 times more likely to be obese than women who reported difficulty hearing. Obesity in those with sight loss was 1.556 times higher in inactive men than women.

Those with combined seeing and hearing difficulties had the highest prevalence of physical inactivity (44.8%) and obesity (26.1%). Analysis showed a significant association between physical inactivity and obesity in men with vision or hearing loss, but not in women.

Around 62% of adults in Spain are overweight, with 26% reporting as obese. In the UK, the figures are broadly similar at around 64% and 28% respectively, suggesting strong similarities between the countries.

A total of 11.04% of the people surveyed self-reported vision loss, 6.96% reported hearing loss, and 3.93% reported suffering both vision and hearing loss.

Lead author Professor Shahina Pardhan, Director of the Vision and Eye Research Institute at Anglia Ruskin University, said: “It is clear from our study that there are significant differences between genders.

“Although women were overall less physically active than men, we found an association between physical inactivity and obesity in men, but not in women. This indicates that, especially in people with vision and hearing losses, exercise and being active has a very important role in preventing obesity for men.

“Adults, especially those with sensory losses, should be encouraged to be as physically active as possible but there are obviously challenges, strongly suggesting that intervention and encouragement would play a very important role.

“An effective strategy to increase the levels of physical activity in this population group would be through targeted intervention programmes based on health awareness on the importance of physical activity.”

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Dad tips we take for granted but go a long way

With all the pressure that comes with responsibilities, dads also need to look after their own wellbeing too.

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There is no one-size-fits all way to being a father, but most working dads want to do the best to support their loved ones. With all the pressure that comes with these responsibilities, dads also need to look after their own wellbeing too. 

Donato Avellana is the Health and Wellbeing Lead for the Vibe Team at Canva Philippines, and as a father himself, he acknowledges the importance of work-life balance. He has helped build programs at Canva that empower employees to have a healthy work-life balance, with holistic programs for all domains of wellness. Taking his learnings from this, he shares some helpful and practical tips to help dads to develop healthy habits that can boost their happiness at work and at home. 

1. Rest and recover well

Rest and relaxation is crucial to improve our overall health. Try your best to get at least 7 hours of sleep, go to bed an hour before you go to sleep, and stay away from your TV or mobile phone, as several studies show that the blue light from screens can disrupt sleep. Sleep is often a neglected component of overall well-being, but this is where the body repairs itself to get ready for another day. Remember that you deserve to rest.

2. Start your day with a cold shower 

Showering can also offer more benefits than many realise.  By using water that is a little cold, you can improve blood circulation, with the lower temperature causing blood to run to the skin’s surface. Good blood circulation plays an important role in our health for proper nutrient distribution throughout our body.

This is also a good time for dads to have their ‘me’ time. Breathing exercises are a great way to start the day, by offering the opportunity to regroup and reflect. 

3. Move more, make it a habit

The health benefits of movement have been scientifically proven time and time again. Your body is connected to your brain and your movement habits can positively impact how you think and feel. Inactivity can make you feel sluggish and tired, making it hard to find motivation and deliver on responsibilities.

Add regular walks, stand often, or follow stretching routines that you can commit to during the day. Taking time for a short 10-15-minute walk is a great way to have a mental and physical break.

4. Eat smart: practice a mindful eating habit. 

We have been educated about what healthy eating is since we were kids. Eating intuitively on a regular schedule is very important. Eating with no structure creates mindless eating that can lead to poor nutrition choices. Think of nourishing your body with healthy fuel for efficient performance instead of just eating. Eat a variety of whole foods, have fruits and vegetables, and hydrate smartly.

5. It’s okay to ask for help

This shortlist might not be easy for everyone, so I encourage dads to seek help when they need it. You might want to start at home with your spouse, ask for help from co-workers, peers, fellow fathers, professionals such as your doctor.

Showing appreciation to your father

If you’re still looking for heart-felt gift ideas for your dad, you can browse through Canva for free design ideas and inspiration. 

Make your dad, grandfather or any other special figure feel loved and appreciated by designing personalized cards. A wide variety of photos, customizable templates, and full-color professional layouts are available. You can also find Filipino templates by changing the language settings to Tagalog and searching for the word ‘tatay’.

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Sun Life Philippines holds ‘Tiny Habits’ workshop

The interactive event carried a fitness theme and was conducted by certified Tiny Habits Coaches TJ Agulto and Claire Limof AHA! Behavorial Design.They shared tips on creating tiny habits in a continuous period of two weeks and also emphasized the importance of celebrating small wins.

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Members of Sun Life Philippines’ Bright Habit Starters community were recently treated to an exclusive workshop on Tiny Habits, the breakthrough method for building habits by world-renowned Behavior Scientist Dr. BJ Fogg.

The interactive event carried a fitness theme and was conducted by certified Tiny Habits Coaches TJ Agulto and Claire Limof AHA! Behavorial Design.They shared tips on creating tiny habits in a continuous period of two weeks and also emphasized the importance of celebrating small wins.

The event was followed by one-on-one coaching sessions to help the participants commit to their habits. This was supervised through the Bright Habits Chatbot, which prompted them to perform daily check-ins for two weeks. This led to the participants achieving 93% success rate in practicing their Bright Tiny Habits.

Following the launch, a second batch of participants is now undergoing the same program, this time with the goal of creating habits to improve their relationships.

The Tiny Habits workshop is just one of the many perks enjoyed by the Bright Habit Starter community. Members are also provided with helpful tips, exclusive promos, and other activities designed to create a sustained behavior change in the present so they can reach their goals in the future. Launched just last March as part of Sun Life’s Ito Ang Araw Mo campaign, the community now has over 3,000 members.

Those interested to be a part of the Bright Habit Starters Community simply have to join via Facebook https://www.facebook.com/groups/itoangarawmo.

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