By Sonalee Ghelani
12-year-old Meesha went from near death and needing a double lung transplant to dancing and playing sport, all thanks to the new CF wonder drug.
The day your child is born is supposed to be one of the best days of your life, and it was definitely like that for me. I had my second child, Meesha, just 14 months after my first. I knew it would be tough with two little ones, but I was ready for it.
What I wasn’t ready for was a call four weeks later from my obstetrician, a call that changed the course of my life forever. My daughter was diagnosed with cystic fibrosis.
I had NO idea what CF was and immediately googled it. I was confronted by phrases like ‘life expectancy of 35’, ‘transplant’, ‘diabetes’ and ‘death’. I was in absolute, utter shock, hyperventilating while trying to relay the news to my family. The next few days were full of meetings with genetic counsellors, CF consultants, dieticians and physiotherapists. All of a sudden, the weight of the world was on my shoulders.
I found myself grieving for the healthy child I thought I had, simultaneously overwhelmed with the responsibility of having to care for a child with a complex multi-system disease.
As days and months went on, I became an expert on cystic fibrosis. I came to realise it’s a cruel, relentless invisible disease. No matter how careful I was, or how hard I tried, my baby was constantly ill and in hospital. At four, she had reoccurring chest infections lasting six weeks at a time with only a week or two between her next infection. She spent many weeks in hospital and would continue IV treatment at home. She went to kinder, and eventually primary school with PICC lines (a catheter inserted in the upper arm) to pump strong IV antibiotics 24-hours-a-day into her fragile veins.
By six, she was drastically underweight and had a feeding tube (Peg) inserted in her stomach, which she still has today.
Our days were filled with hours upon hours of medical therapy and my remaining hours were looking for any advancements within the CF world. My husband and I came across a pharmaceutical company researching new drug therapies − gene modulators! This brought a glimmer of hope that maybe, in our lifetime, my daughter would be fortunate enough to have a drug that would prolong her life. However, as months and years rolled on, Meesha’s health deteriorated. I didn’t want to think about it at the time, but her future looked grim and all those statistics on young children with cystic fibrosis dying were becoming a close reality.
I tried to hold onto hope, after all, we were adhering to a strict regime of treatments and physiotherapy to preserve what was left of her precious lungs. But Meesha wasn’t so lucky – she ended up with advanced lung disease by the age of eight and she was further diagnosed with CF-related diabetes. She had dangerously low oxygen levels, extremely low lung function and was on oxygen for a period. She could no longer run, dance, or even laugh without choking and vomiting. All the things she loved to do were ripped away.
She was now at the end of the road for treatment. She needed a double lung transplant to survive.
A few months later, we were given an opportunity to access a new drug only available in America. A magic pink pill called Orkambi, which our CF clinic managed to obtain on compassionate grounds, as there were no other alternatives to keep Meesha alive than to use this new, unapproved drug. She was one of only a few on this medication in Australia at the time. These pills took a little while to work, but she eventually went from needing a double lung transplant to going back to school part-time. Her lung function increased slightly, and she was able to put her transplant on hold.
As fortunate and as happy as we were to receive this medication, her CF was so severe, and her body was so compromised, that she started declining once again. By age 11, she could barely walk 150m, she was still 100 per cent tube fed, and her diabetes was wreaking havoc on her body. She was on over 40 pills a day, including strong antibiotics with very scary side effects like hearing loss. I was once again losing my daughter to CF. At this stage a miracle drug called Trikafta was developed. It had just passed through FDA approval in America for children 12 years and up. Every story we read was about “life-changing” results − people claiming they didn’t feel like they even had CF anymore. Trikafta was adding years to people’s lives. We NEEDED this drug!! My husband and I decided to sell everything we had to purchase Trikafta. The cost would be $US300,000 for just a year’s supply as at the time there was no PBS support for the medication. Amazingly, with a little luck and the phenomenal CF clinic at the Monash Children’s Hospital on our side, we were granted access to Trikafta on compassionate grounds, which meant we were given it for free.
March 26, 2021, was the best day of my life. In the midst of a global pandemic, I was the happiest I have ever been. Meesha started Trikafta!! Aside from her body going through a purge for a few weeks, the effects were almost instantaneous. Meesha gained a kilogram in a week, she stopped coughing, her lung function increased, and she started eating food. It was incredible.
Meesha’s transformation has been nothing short of miraculous − the difference between life and death. I am truly thankful and appreciative for researchers, scientists and doctors who have all played their role in CF. Without them, I would not have my baby girl.
If you would like to follow Meesha’s story, you can join her Facebook Group – My journey on Trikafta.
Read more on Cystic Fibrosis here.