Brave little Angel with extremely rare CLOVES syndrome given new hope with experimental pill
Heartbreaking moment brave little girl with extremely rare disease says she ‘just wants to be normal like other kids’ – new experimental pill offers hope
- Angel, 11, suffers from an incredibly rare syndrome
- She has been put on an experimental new drug
A brave young girl battling an incredibly rare disease has been given a glimmer of hope in the form of a tiny pill that could change her life.
Angel, 11, was born in Pakistan with CLOVES syndrome, a disease that affects fewer than 200 people worldwide, and first came to Australia for treatment in 2017.
Her condition causes overgrowth of body tissue – so much so that her feet are permanently swollen to the size of watermelons and she needs a wheelchair to get around.
Her cheerful and bubbly personality – she loves to play board games – is a miracle in itself, considering that she struggles with pain almost daily as her organs are compressed by the overgrown tissue, leading to a full hysterectomy at just 10 years old last year. was needed.
But thanks to Moira Kelly of the Creating Hope Foundation, who became Angel’s guardian while she’s in Australia, she’s been given access to a trial drug that has produced remarkable preliminary results.
Angel (pictured) suffers from CLOVES syndrome and said she just wants to be like ‘other normal kids’
The rare disease that affects fewer than 200 people worldwide causes overgrowth in her body tissue. She is cared for by Moira Kelly (pictured together) who runs the Creating Hope Foundation
“All I really want right now is to get better and just be normal like other kids,” Angel told A Current Affair in a segment that aired Tuesday as he fought back tears.
“I just want to be able to walk and do other things without pain and struggle.”
After seeing more than 100 doctors in three countries and undergoing multiple major surgeries, a double foot amputation was canceled last year because it was too risky. Kelly began a deep dive into her own research.
At 3 a.m. one morning while on one of her research trips, she found gold—a drug originally created by Novartis for breast cancer that has produced remarkable results with Angel’s condition.
Ms. Kelly and Angel raised funds and traveled to France to meet with a specialist who is one of the few in the world with experience with CLOVES and he offered to trial her on the medication.
The little pink pill costs $100,000 a year, but after hearing Angel’s story, Novartis is providing the drug for free on compassionate grounds.
After lengthy fundraising, Angel flew Ms. Kelly to Paris, where French specialist Professor Guillaume Canaud (pictured together) recommended her for the trial of an experimental new drug called Vijoice
“We really hope as a team that this drug will shrink her feet down and give her back her ability to walk,” said Dr Luke Sammartino, Angel’s pediatrician in Melbourne.
He is part of a larger team of radiographers, pathologists, oncologists, doctors and surgeons in Australia who are working together to help Angel.
“She’s amazing, everything she’s been through and she never complains.”
“I would like Angel to achieve two things: she once told me she wants to dance and second, I would like her to outlive me for many, many years,” he said.
She will receive her first dose of the drug next week.