Helen has good news she’s been sharing this week. It’s about blood – but don’t worry. It really is good news, not just for Helen but for millions other young people.
Transcription podcast – January 17, 2020
Her story shows how scientists are now curing illnesses by fixing people’s genes. (Genes are like bits of computer code that your body gets from mum and dad when you’re born.)
All her life – 16 years – Helen had to deal with pain that often made her cry. She had operations. And in winter, the cold kept her indoors.
Helen suffered from sickle cell anaemia, or SCA. Her blood didn’t do its job properly. The red cells in blood should be round, a bit like doughnuts, to move smoothly around in our veins. But some of Helen’s blood cells were not doughnuts but shaped like bananas – or sickles. (Any idea what a sickle is? You don’t see them much nowadays, but it’s a big curved knife that farmers used to use to harvest wheat by hand.)
The fantastic news that Helen just had, and which she spoke about to the American newspaper the New York Times this week, is that she now has brand new blood.
She can go out in the cold. She’s taken up dancing – something she used to find too painful. Sometimes she dances so much, her legs ache. But her mum is so happy to tell her that’s just what happens – to healthy people too!
Now, you might wonder – what’s the big deal about getting new blood?
Our bodies make new blood all the time. If you bleed, you just make more.
But the problem for Helen and the millions of others with SCA – the most common of all illnesses caused by genes — is that the new blood their bodies make contains more sickle cells that get jammed up inside their veins, making them unwell.
Doctors have known for many years that the disease is “inherited”. Without knowing it, parents can pass “genetic” illnesses to some of their children and not others. Helen’s big sister Haylee was also born with sickle cell but not her little brother Ryan.
Scientists are now working on various “gene therapy” cures and brave young people are a vital part of those experiments. A boy in France was just 13 when he went through an operation to replace his blood-making gene in 2014. His doctor, Professor Cavazzana, told WoW! this week that this young man is doing well.
Helen was 16 last summer when doctors in Boston took cells from her bones, changed their genetic code and injected them back into Helen’s bloodstream.
Six months later, they found that she’s just making nice round blood cells – all doughnuts, no more sticky bananas.
It will take years to be sure these gene therapies are really safe – and to make them simpler and cheaper for everyone.
But Helen’s story shows how the magic of genes is giving us new chances.
” This year has been one of the toughest years for me ” she wrote in a New Year post on Facebook. Now, Helen said, she is going to “start a new life”.
“ I’m going to live it the best I’ve ever lived life. “