The launch of clinical trials in Abu Dhabi to find new treatments for an inherited blood disorder has been described as an opportunity to greatly reduce the burden of regular transfusions and improve the quality of life of patients.
Burjeel Medical City will assess the effectiveness and safety of Mitapivat, a drug that has shown promise as a treatment for thalassaemia, one of the most common genetic illnesses in the UAE.
It occurs when the body doesn't make enough of a protein called haemoglobin. Severe cases require frequent blood transfusions.
Stem cell or bone marrow transplants are the only current treatments to cure thalassaemia.
This will have great implications ... because not only will it decrease the transfusion demand but it could improve quality of life
Dr Khaled Musallam
Abu Dhabi will take part in the phase 3 trials, called Energise and Energise-T, which will test Mitapivat's effectiveness on adults with thalassemia.
The Energise study aims to evaluate whether Mitapivat can effectively and safely improve haemoglobin levels in patients who do not require regular transfusions. It aims to evaluate whether the same drug can reduce the need for transfusions in patients who regularly receive new blood.
“The Energise-T study is looking at patients who we call transfusion-dependent anaemic,” said Dr Khaled Musallam, group chief research officer of Burjeel Holdings.
“This means that they have a severe form of anaemia that requires them to take blood from external sources almost on a monthly basis for the rest of their lives.”
The primary goal of the Energise-T trial is to reduce the need for transfusions by at least 50 per cent.
“This will have great implications, if we're able to do that, because not only will it decrease the transfusion demand but it could improve quality of life,” he said.
“Also the transfusions that patients get are from outside sources, so they have a lot of extra iron that the body cannot get rid of, which increases the levels of toxic iron that accumulates in the heart and the liver and can cause severe organ damage and death.
“So, we are not only saving them from transfusions but also from the side effects of long life transfusions.”
Preventive measures remain key
Thalassaemia tends to be more common in people of Middle Eastern, African, Mediterranean and South, South-East or East Asian descent.
Consanguineous marriages often contribute to higher rates of thalassaemia in parts of the Middle East as the couple are more likely to have similar genes, increasing the likelihood of them passing it on to their children.
There are more than 2,000 patients with thalassaemia in the UAE.
It is mandatory for couples to be screened for the condition before marriage, although there is no law that bars them from marrying if they are found to be carriers.
Early intervention is required, said Saeed Al Awadhi, a thalassaemia patient and a member of the board of directors of the Thalassaemia International Federation and the Emirates Thalassaemia Society.
“It is right before a couple’s wedding date and after they have made all the preparations for marriage — by then they would have fallen in love, made commitments and plans, so nothing would stop them from getting married,” he said.
UAE currency: the story behind the money in your pockets
COMPANY%20PROFILE
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Founder: Ahmed Wadi
Launched: 2016
Employees: 76
Financing stage: Series A ($4 million)
Investors: Partech, Sawari Ventures, 500 Startups, Dubai Angel Investors, Phoenician Fund
PROFILE OF INVYGO
Started: 2018
Founders: Eslam Hussein and Pulkit Ganjoo
Based: Dubai
Sector: Transport
Size: 9 employees
Investment: $1,275,000
Investors: Class 5 Global, Equitrust, Gulf Islamic Investments, Kairos K50 and William Zeqiri
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Springsteen: Deliver Me from Nowhere
Director: Scott Cooper
Starring: Jeremy Allen White, Odessa Young, Jeremy Strong
Rating: 4/5
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Al Ain: Caio (5', 73'), El Shahat (10'), Berg (65'), Khalil (83'), Al Ahbabi (90' 2)
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Name: Mariam Ketait
Emirate: Dubai
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Favourite quote: "Be the change you wish to see" - unknown
Favourite activity: Connecting with different cultures
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Favourite Quote: “Real victories are those that protect human life, not those that result from its destruction emerge from its ashes,” by The late king Hussain of Jordan.
Favourite Hobby: Writing and cooking
Favourite Book: The Prophet by Gibran Khalil Gibran
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Engine: 2-litre 4-cylinder and 3.6-litre 6-cylinder
Power: 220 and 280 horsepower
Torque: 350 and 360Nm
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Favourite place in UAE: Al Rams pearling village
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How to avoid crypto fraud
- Use unique usernames and passwords while enabling multi-factor authentication.
- Use an offline private key, a physical device that requires manual activation, whenever you access your wallet.
- Avoid suspicious social media ads promoting fraudulent schemes.
- Only invest in crypto projects that you fully understand.
- Critically assess whether a project’s promises or returns seem too good to be true.
- Only use reputable platforms that have a track record of strong regulatory compliance.
- Store funds in hardware wallets as opposed to online exchanges.
'The worst thing you can eat'
Trans fat is typically found in fried and baked goods, but you may be consuming more than you think.
Powdered coffee creamer, microwave popcorn and virtually anything processed with a crust is likely to contain it, as this guide from Mayo Clinic outlines:
Baked goods - Most cakes, cookies, pie crusts and crackers contain shortening, which is usually made from partially hydrogenated vegetable oil. Ready-made frosting is another source of trans fat.
Snacks - Potato, corn and tortilla chips often contain trans fat. And while popcorn can be a healthy snack, many types of packaged or microwave popcorn use trans fat to help cook or flavour the popcorn.
Fried food - Foods that require deep frying — french fries, doughnuts and fried chicken — can contain trans fat from the oil used in the cooking process.
Refrigerator dough - Products such as canned biscuits and cinnamon rolls often contain trans fat, as do frozen pizza crusts.
Creamer and margarine - Nondairy coffee creamer and stick margarines also may contain partially hydrogenated vegetable oils.
Past winners of the Abu Dhabi Grand Prix
2016 Lewis Hamilton (Mercedes-GP)
2015 Nico Rosberg (Mercedes-GP)
2014 Lewis Hamilton (Mercedes-GP)
2013 Sebastian Vettel (Red Bull Racing)
2012 Kimi Raikkonen (Lotus)
2011 Lewis Hamilton (McLaren)
2010 Sebastian Vettel (Red Bull Racing)
2009 Sebastian Vettel (Red Bull Racing)
In-demand jobs and monthly salaries
- Technology expert in robotics and automation: Dh20,000 to Dh40,000
- Energy engineer: Dh25,000 to Dh30,000
- Production engineer: Dh30,000 to Dh40,000
- Data-driven supply chain management professional: Dh30,000 to Dh50,000
- HR leader: Dh40,000 to Dh60,000
- Engineering leader: Dh30,000 to Dh55,000
- Project manager: Dh55,000 to Dh65,000
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- Senior process engineer: Dh28,000 to Dh38,000
- Senior maintenance engineer: Dh22,000 to Dh34,000
- Field engineer: Dh6,500 to Dh7,500
- Field supervisor: Dh9,000 to Dh12,000
- Field operator: Dh5,000 to Dh7,000
