“HAPPY and smiley” Melody Aggett was diagnosed with aggressive blood cancer in April last year when she was just four months old, after developing a “cold”.
Now, her devastated parents Rachel-Elizabeth Hewitt and Kevin Aggett have been told there are no treatment options left in the UK for their 14-month-old baby girl.
Their only “glimmer of hope” lies in a drug called Revuforj, which has been approved in the US but not in the UK.
The couple found out about the drug – a type of menin inhibitor that works by blocking proteins that keep cancer cells growing – by doing their own research.
Their consultant at University Hospital Southampton (UHS) wrote to the Medicines and Healthcare products Regulatory Agency (MHRA) to get approval for it to be imported, which was granted.
It means Melody would likely be the first person in the UK – and one of the first in Europe – to receive the treatment.
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With time against them, they hope to get Melody started on it as soon as possible.
But Revuforj would cost Rachel-Elizabeth and Kevin £20,500 per month – amounting to £500,000 for the two years Melody would need to be on it.
And the parents can’t order the drug until they have a lot of the funds in place.
Rachel-Elizabeth told the BBC: “The horrible reality is that Melody could become very poorly at any point in time.”
“We need to order the drug as soon as possible.
“We asked our consultant last week if we had the drug available in England now when would she start it – and she said she would have started it yesterday.”
It all started when Melody, from Poole in Dorset, developed what her parents thought was a cold last spring.
Rachel-Elizabeth told Sun Health: “By day 10, she had reduced her oral intake and had fewer wet nappies; that’s when we knew it was time to get some advice.
“We were taken into Poole Hospital by ambulance.
“At first, the doctors thought it was bronchiolitis.
“The next day Melody hadn’t improved and so the doctor decided to do a blood test.
“As soon as the results came back from this, they knew something was drastically wrong.”
Rachel and Kevin got “the worst news that any parents can imagine” – that four-month-old Melody had a very rare and aggressive form of acute myeloid leukaemia (AML).
AML is a fast-growing type of blood cancer that is rare in children and usually needs urgent treatment.
About 100 children and teens are diagnosed with AML every year in the UK, according to Blood Cancer UK.
It was heart-breaking to watch her go through treatment, she suffered such a lot and we felt so helpless. Then to be told we are out of options, just broke our hearts all over again
Rachel-Elizabeth Hewitt
The disease affects blood cells produced in the bone marrow, causing them to multiply too quickly.
Symptoms can include tiredness and pale skin, unexplained bruising and frequent infections.
“It didn’t feel real,” Rachel-Elizabeth said.
“I had to ask the doctor to repeat herself because I couldn’t believe the words that she had said, then everything happened so quickly that there wasn’t really the time to process it.”
‘Shattered hope’
Four-month-old Melody had two rounds of intensive chemotherapy, which involved multiple blood and platelet transfusions.
She also had a prolonged stay in intensive care and spent several weeks on a ventilator.
Doctors told the parents that Melody’s only chance of survival was a bone marrow transplant to replace cancerous cells with healthy ones, which she underwent at Great Ormond Street Hospital.
“For a brief moment, we dared to hope,” Rachel and Kevin wrote in their GoFundMe.
“But that hope was soon shattered.”
Melody suffered a series of complications from the procedure, including severe acute Graft-versus-host disease – a condition where bone marrow cells from the graft attack the body’s own cells.
At the end of last year – just 30 days after receiving the transplant – Melody’s cancer returned.
Being able to access this treatment will save Melody’s life. It will mean she gets the chance at childhood and life that she truly deserves
The parents were told Melody needed another transplant, but wouldn’t be able to have it for another two years.
She’d need treatment to survive that long, but no other options were available in the UK.
Rachel-Elizabeth and Kevin were told to consider palliative care for Melody and to cherish their remaining time together – possibly weeks.
“It was heart-breaking to watch her go through treatment, she suffered such a lot and we felt so helpless,” the mum said.
“Then to be told we are out of options, just broke our hearts all over again. It didn’t and still doesn’t feel real.
“But again, there hasn’t been time to process it because we can’t waste one moment.
“Instead, we are fighting for our little girl and we won’t give up until she is better and can live the happy life she deserves to live.”
Fighting chance
Revuforj is Melody’s only remaining hope of achieving remission and getting another bone marrow transplant in two years’ time.
Rachel-Elizabeth told Sun Health: “Being able to access this treatment will save Melody’s life.
“It will mean she gets the chance at childhood and life that she truly deserves.
“It would mean absolutely everything to us and our family.”
So far, Rachel-Elizabeth and Kevin have been able to raise over £70,000 of their £500,000 goal.
“We are able to order the drug as early as this week, but only if the money is raised in time,” they said.
“Every day we lose risks the leukaemia progressing beyond the point where this treatment can help her.”
Rachel-Elizabeth explained: “We don’t need the full funds before we can order. But we need a substantial amount as the drug is very difficult to get.
WHAT IS ACUTE MYELOID LEUKAEMIA?
ACUTE myeloid leukaemia is a type of blood cancer that affects the immune system’s white blood cells.
The causes of the disease are not well understood and it usually cannot be prevented. Previous chemotherapy, exposure to radiation or smoking could raise the risk.
It is a rare type of cancer and affects around 2,900 people each year in the UK.
AML is an aggressive form of cancer and often needs urgent treatment with chemotherapy and possibly a bone marrow transplant.
Symptoms of the disease include:
- Paleness
- Concerning tiredness or weakness
- Often feeling breathless for no reason
- Getting regular infections
- Unexplained weight loss
- Unexplained bleeding and/or bruising
Source: NHS
“Each time we order, we will have to use a specialist company in Manchester to import the drug, which also comes with very expensive fees, and it takes 21 days to get here.
“We need to order a big enough supply and have funds readily available to then order the next supply.
“Putting Melody in a position where we can’t afford to buy the next supply would be detrimental, and so that cannot be an option.”
The tot is in hospital with her parents almost every day undergoing tests and receiving blood and platelet transfusions.
“Melody is doing really well day to day,” Rachel-Elizabeth said.
“She is the happiest girl and when she smiles, her whole face lights up.
“She loves to play, to crawl, to giggle, she is just amazing.”
The mum told the BBC the tot’s smile had kept her and Kevin going.
“She obviously can’t talk and she can’t tell us how she’s feeling – but she does show us how she’s feeling,” she said.
“We can’t give up on her – she wants to keep fighting.”
You can donated to Melody’s GoFundMe here.
