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A scientist in a race against time to find a cure for her daughter’s rare and deadly disease has said they are “finally in the driving seat”.

Dr Michelle Teng is trying to develop the world’s first treatment for TUBB4a leukodystrophy before it is too late for her 12-year-old daughter Sofia.

The life-threatening genetic condition affects the central nervous system and can lead to an inability to walk, talk and even swallow. Most young patients die before they reach adulthood.

Sofia started showing symptoms when she was a toddler and was eventually diagnosed with the disease aged four.

“When we Googled the condition, it was a terrifying experience,” Michelle remembers.

“If you read the worst-case scenarios, the children die within 12 months.

“In [Sofia’s] case, it’s a slow, neurodegenerative disease. Obviously, I didn’t know that, and I had no idea how long she was going to live for.

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“The doctors told us there’s nothing you can do, and I think it’s probably the most devastating news for any parent to hear.”

Sofia Teng

‘It’s a ticking time bomb’

But the diagnosis spurred Dr Teng into action. At first, she raised money for research because at that point, in 2014, the disease had only just been defined.

But as Sophia’s condition began to deteriorate, to the point where she is now unable to walk or talk, Michelle realised she needed to do more.

“Anyone who’s lived with a loved one with Parkinson’s, Alzheimer’s or anything that’s degenerative will see that it’s a ticking time bomb,” she said.

“It’s a steady decline, and as the years go by, you see the people you love start losing the ability to do certain things.

“We raised something like close to £200,000 in a short space of time, which was extremely encouraging, but to really find a treatment, and to bring that treatment to clinic, will take five to 10 million, and I realised that was not going to cut the mustard.”

Sofia Teng

‘You have to be an optimist’

So in April 2021, Dr Teng set up SynaptixBio, a biotech firm based in Oxford with the sole aim of finding a treatment for TUBB4a.

The company recently signed a global licensing deal with the Children’s Hospital of Philadelphia – the world’s leading TUBB4a leukodystrophy centre – in the US to accelerate the research process.

It aims to launch clinical trials for a treatment in 2024.

Dr Teng said: “I feel like because we’ve set up this company, we’re finally in the driving seat in finding a treatment for [Sofia], within those timelines that we believe will be on time for her.

“And when you work in biotech, you have to be an optimist.”

Sofia Teng

‘A game-changer’

Research suggests there are 1,650 babies worldwide who are born with TUBB4a every year. In the UK, it is thought to affect between 60-90 newborns.

But scientists fear the number of cases could be higher still, due to many patients being misdiagnosed with other conditions, such as cerebral palsy.

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A varied combination of symptoms makes it incredibly difficult to spot. The only way to confirm the disease is by genome sequencing and an MRI scan.

Dr Dan Williams, SynaptixBio CEO and co-founder, called the treatment the company is developing a potential “game-changer”.

“One of the most horrible things is that a lot of these children don’t actually make it into late teenagers,” he said.

“Hopefully, by stopping the progression of this disease, it could offer them a better quality of life as well as an extension to their lives as well.”

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Former England rugby captain Lewis Moody reveals motor neurone disease diagnosis

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Former England rugby captain Lewis Moody reveals motor neurone disease diagnosis

England’s 2003 Rugby World Cup winner Lewis Moody has been diagnosed with motor neurone disease.

The 47-year-old former England captain said it had been “incredibly hard to process and a huge shock to me and my family”.

In a social media post, he wrote: “I’m writing to share some tough news. I have recently been diagnosed with ALS, also known as Motor Neurone Disease (MND).”

He said: “I feel fit and well in myself and I’m focused on staying positive, living life and dealing with the changes I will experience as they come.

“I am being well supported by my family, friends and medical professionals and I’m truly grateful to those who, in their time, helped progress research to support others, like me, living with this disease.

England's Lewis Moody (R) tackles Robert Sidoli from Wales during a Rugby World Cup quarter-final in 2003. Pic: Reuters
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England’s Lewis Moody (R) tackles Robert Sidoli from Wales during a Rugby World Cup quarter-final in 2003. Pic: Reuters

Lewis Moody, fourth from right in the middle row, poses with Queen Elizabeth II after winning the 2003 World Cup. Pic: Reuters
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Lewis Moody, fourth from right in the middle row, poses with Queen Elizabeth II after winning the 2003 World Cup. Pic: Reuters

Since retiring from the sport in 2012, Moody and his wife, Annie, have dedicated much of their time to fundraising for The Lewis Moody Foundation, which supports those affected by brain tumours.

“My plan is to continue with this, but to also create an opportunity to support a charity closer to my current situation,” said Moody, who was awarded an MBE for services to rugby in 2004.

“I would be so grateful for your help with this and look forward to sharing more, once I am clear on what this looks like.”

Rugby players could be prone to motor neurone disease – but causes still unknown

Fellow rugby players Doddie Weir and Rob Burrow have died from the disease in recent years.

The sport – and in particular England skills coach Kevin Sinfield – have worked on high-profile fundraising campaigns to tackle it.

Athletes appear to be disproportionately likely to contract MND, which causes muscle weakness to the point it can eventually be hard to eat or breathe.

Credit: Action Images / Paul Harding Livepic via Reuters
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Credit: Action Images / Paul Harding Livepic via Reuters

Research from Durham University found rugby players could be especially prone, as those who have suffered multiple concussions have higher levels of certain proteins in their blood that are linked to the disease.

But the exact cause of it remains unknown.

Read more: ‘Game changer’ test could spot motor neurone disease

“For now, please know I feel your love and support,” Moody added.

“All I ask is that I am given some space to navigate this with my wife and sons, and those closest to us – but without doubt, I will continue to embrace life and grasp opportunities in the same way I always have.”

What is motor neurone disease?

Motor neurone disease (MND) causes muscle weakness that gets worse over the course of months or years.

It’s usually life-shortening and there’s currently no cure, but treatments have been developmed to help manage the symptoms.

Initial symptoms can be: stiff or weak hands, weak legs and feet and twitches, spasms or muscle cramps.

They can worsen into problems breathing, swallowing and speaking, changes to personality and mood and being unable to walk or move.

It affects around one in 300 people.

A handful of studies have shown a correlation between professional sports to MND, but do not prove a cause.

A ‘glittering career’

Moody won 71 caps for England and also five for the British and Irish Lions, and was a seven-time title winner with Leicester.

He is perhaps best known for winning the final lineout during the 2003 World Cup final, which led to Jonny Wilkinson’s historic match-winning drop goal.

Lions Rugby Chair Ieuan Evans MBE said: “We are all greatly saddened to hear the news of Lewis Moody’s diagnosis.”

He added: “As a player, Lewis inspired so many fans during a glittering career.”

Moody joined Leicester Tigers from Oakham School and made 223 appearances between 1996 and 2010.

The club said everyone there was “deeply saddened to learn that one of our greatest players” had been diagnosed with MND.

“The figures, trophies and awards tell you what an incredible player Lewis was, but that is only half the story,” said Tigers chief executive Andrea Pinchen.

“As an individual, his commitment to his club along with his warmth and passion shone through, which endeared him to teammates, staff and supporters alike.”

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Major shake-up of home buying aims to make process cheaper, quicker and easier

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Major shake-up of home buying aims to make process cheaper, quicker and easier

A shake-up to the house-buying system which could cut a month off the time it takes – and slash around £700 from the moving bill – is on the table.

Changes could include requiring property sellers and estate agents to provide more information when a home is listed for sale, reducing the need for buyers to carry out searches and surveys.

Binding contracts could also be introduced at an earlier stage, reducing the risk of a chain collapsing.

The proposals could also deliver clearer information to consumers about estate agents and conveyancers, including their track record and expertise, along with new mandatory qualifications and a code of practice to drive up standards.

Housing Secretary Steve Reed said the proposals, which are the subject of a consultation, would help make “a simple dream, a simple reality”.

The government says it will set out a full roadmap in the new year after consulting on its proposals.

Housing Secretary Steve Reed. Pic: PA
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Housing Secretary Steve Reed. Pic: PA

Mr Reed said: “Buying a home should be a dream, not a nightmare.

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“Our reforms will fix the broken system so hardworking people can focus on the next chapter of their lives.”

Officials believe the proposed package of reforms could cut around a month off the time it takes to buy a new home and save first-time buyers an average of £710.

People selling a home could face increased costs of around £310 due to the inclusion of upfront assessments and surveys.

Those in the middle of a chain would potentially gain a net saving of £400 as a result of the increased costs from selling being outweighed by lower buying expenses.

Wider use of online processes, including digital ID, could help make transactions smoother, the government argued, pointing to the Finnish digital real estate system which can see the process completed in around two weeks.

The consultation also draws on other jurisdictions, including the Scottish system where there is more upfront information and earlier binding contracts.

‘Process the same as for our grandparents’

The planned shake-up was welcomed by property websites and lenders.

Rightmove chief executive Johan Svanstrom said: “The home-moving process involves many fragmented parts, and there’s simply too much uncertainty and costs along the way.

Looking for the perfect home on Rightmove. File pic: PA
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Looking for the perfect home on Rightmove. File pic: PA

“Speed, connected data and stakeholder simplicity should be key goals. We believe it’s important to listen to agents as the experts for what practical changes will be most effective, and we look forward to working with the government on this effort to improve the buying and selling process.”

Santander’s head of homes David Morris said: “At a time when technology has changed many processes in our lives, it is incredible that the process of buying a home – an activity that is a cornerstone of our economy – remains much the same for today’s buyers as it did for their grandparents.”

Conservative shadow housing minister Paul Holmes said that while Labour welcomed steps to digitise and speed up the process, the party risked “reinventing the last Labour government’s failed Home Information Packs – which reduced the number of homes put on sale, and duplicated costs across buyers and sellers”.

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Shadow chancellor to pledge further cuts to foreign aid – as Tories vow to ‘stand up for fiscal responsibility’

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Shadow chancellor to pledge further cuts to foreign aid - as Tories vow to 'stand up for fiscal responsibility'

The Tories will pledge to make even further cuts to the foreign aid budget, as the party attempts to regain its reputation for fiscal responsibility in the wake of the Truss mini-budget.

Shadow chancellor Sir Mel Stride will unveil plans to cut overseas development aid to 0.1% of Gross National Income (GNI), down from the current 0.3%, cementing a sea change in the Conservative Party’s position on international aid.

In his keynote speech to the party conference in Manchester, Sir Mel will claim that his plans can save £47bn over the next parliament, which include cuts to welfare, the civil service, and green subsidies.

In the wake of the Truss mini-budget that saw the pound fall and interest rates soar, the senior MP will say that his party will “never, ever make fiscal commitments without spelling out exactly how they will be paid for”, and commit to fiscal responsibility.

A key part of Sir Mel’s plans to demonstrate that is to reduce foreign aid to 0.1% of GNI, or around £3bn per year – down from spending of an estimated £9.4bn in 2028-29.

The Boris Johnson government reduced aid spending to 0.5% of GNI in 2021, in order to pay for the vast public spending during the pandemic. Sir Keir Starmer announced a further cut to 0.3% of GNI earlier this year to pay for the increase in defence spending.

Bond, the network for organisations working in international development and humanitarian assistance, has hit out at the announcement, describing it as “reckless, short-sighted, and morally indefensible”.

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Its chief executive, Romilly Greenhill, told Conservatives at a fringe event at the Tory Party conference on Sunday: “Let’s just be really clear, such a policy would negatively impact millions of people around the world.

“It would harm deeply vital programmes being made in reducing, eradicating, killer diseases, and it would also severely undermine our ability to respond to devastating global crises.”

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Does it matter that foreign aid has been cut in the UK?

But the Tories say they “cannot justify taxing people in this country to pay for billions of spending abroad”, and it marks the death-knell of Tory former prime minister David Cameron’s target of spending 0.7% of GNI on aid, announced in 2011.

Welfare, green subsidies, and asylum hotels to face the chop

Another key area where Sir Mel will pledge to make savings will be the welfare system, where they claim £23bn can be cut.

He will say that narrowing the eligibility for sickness benefits, stopping claims from people with “low-level mental health problems” who could be treated instead, limiting the VAT subsidy for Motability, and reforming job-seeking obligations are key areas where savings can be made.

But a major change will be restricting welfare to British citizens – bringing Tory party policy in line with Reform UK.

But he will vow to reverse any decision from the current Labour government to lift the two-child benefit cap, which stands in contrast with Nigel Farage’s party that wants to lift it.

Sir Mel is expected to say the reforms are essential not just for balancing the books, but for tackling the deeper social damage caused by long-term dependency.

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Tories would quit European Convention on Human Rights

Another key target of the shadow chancellor is the civil service, where he will argue that £8bn in savings can be made by reducing the headcount from 517,000 down to 2016 levels of 384,000.

Scrapping the Climate Change Act and “costly and ineffective green subsidies being pushed by Ed Miliband” is also on Sir Mel’s agenda. The Tories say there are savings of £1.6bn a year to be made in this area.

And closing all asylum hotels will save at least £3.5bn, the Tories say – at least £1.6bn of which they have already allocated to their new ICE-style “removals force”, to detain and remove 150,000 illegal migrants per year.

In his speech, Sir Mel Stride MP is expected to say: “The Conservative Party will never, ever make fiscal commitments without spelling out exactly how they will be paid for.

“We’re the only party that gets it. The only party that will stand up for fiscal responsibility. We must get on top of government spending.

“We cannot deliver stability unless we live within our means. No more pretending we can keep spending money we simply do not have.”

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Is the Tory party a ‘distressed asset’?

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‘Trussonomics is still in full swing’

But Labour Party chair Anna Turley said: “The Tories let welfare bills, civil service numbers and asylum hotel use skyrocket on their watch – and they’ve never apologised. Now they want to rehash failed promises from their failed manifesto to try to solve the problems they caused.

“This is the same old Tories, with the same old policies. They didn’t work then and you can’t trust them now.”

And Liberal Democrat Treasury spokeswoman Daisy Cooper said it was “clear the Conservative Party learnt absolutely nothing from their disastrous handling of the economy, which left families struggling with a cost-of-living crisis and public services on their knees”.

She added: “Cutting vital support to bring household bills down, trying to balance the books on the backs of people with mental health conditions and slashing the UK’s soft power abroad through aid budget cuts shows Trussonomics is still in full swing.”

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