Marwan, aged 26, was diagnosed with stage 4 alveolar rhabdomyosarcoma. He faced his cancer diagnosis like he faced everything else in life – a soldier. Sadly, Marwan never reached remission and died after 13 months and three lines of back to back nonstop chemo. A top medical student, he believed in the power of immunotherapy and while he got very close to trying ground-breaking CAR T cell therapy, it was heartbreakingly too late.
Marwan was a thriving, respected top medical student in St John’s, Newfoundland, Canada. He was passionate about his work and loved his patients even more. In March 2020, one week before the world shut down, he was diagnosed with stage 4 fusion positive alveolar rhabdomyosarcoma in his pelvis. When he was diagnosed, the cancer had spread to his lungs, multiple vertebrae along the spine and other bones throughout his body.
Prognosis was dismal and no words of encouragement ensued. Regardless, the chances weren’t 0% and as a family we decided to pursue conventional treatment which consists of some of the most brutal chemotherapy there is. His treatment was led from the Princess Margaret Cancer Centre in Toronto, Canada. He was told that as an adult, rhabdomyosarcoma is more aggressive and less responsive but nonetheless this was the best option for him. He was told adults cannot tolerate more than 10 cycles of chemotherapy, but Marwan soldiered on to take 14 cycles of VAC, 2 cycles of Irinotecan/vincristine and 1 cycle of etoposide/ifosfamide. All 17 cycles failed to tame the relentless beast.
Fever, neutropenia, fatigue, lack of appetite, nausea, vomiting, broken bones, back pain, emotional distress, COVID isolation, lonely hospital visits, even lonelier hospitalizations, watching the world move on, his future disappear, his body fall apart. Those are just SOME of the things he experienced fighting this monster.
Immunotherapy was our only option. A brave and compassionate doctor in China, Dr. Lung-Ji Chang was willing to treat Marwan as part of a clinical trial. We were told that patients with low disease burden respond the best and while Marwan was not the best candidate, we were out of options and he wanted to try a non chemotherapy option he believed in. We managed to get a VISA to China in the middle of a worldwide pandemic which in and of itself seemed like a miracle and finally we were so close to treatment.
Four days before he was supposed to receive a groundbreaking CAR T cell therapy an MRI scan showed a shocking 5cm metastasized tumor to the cerebellum that rendered the therapy more harm than good. Sadly, Marwan died without trying the CAR T cell therapy but with the full belief in its potential to heal future patients suffering with rhabdomyosarcoma.
By the end, Marwan’s primary tumor was gone but there were metastasis to the lungs, entire spine, ribs (of which he broke himself upon examining the rib), kidneys, soft tissue (too many spots to list), femur, colon, right eye and cerebellum. He died yes, but the cancer died with him too and for that we are grateful the suffering is done.
Hell is too kind of a word to describe what this journey was like. I am his sister and so I can only imagine what my parents felt and are feeling now with this gaping void. There are no good words other than it was desperation, love, pain, exhaustion, emotional torment all mixed into one and living one minute at a time knowing tomorrow may never come. We also knew that if it came, pain was guaranteed to join us hand in hand. Wanting it to end but not end all at the same time.
Marwan was adamant and clear – immunotherapy is the way forward. Chemotherapy is brutal and should not be offered to patients in an advanced stage. There is no quality of life on chemotherapy despite what some oncologists wish to believe. We need to do better. His dream was to be cured of this and to help pave the path for children to also use immunotherapy as the solution instead of poison, burning and cutting. Less harsh treatments, a move away from killing all cells and a focus on personalized immunotherapy for each cancer patient. In Marwan’s words, you can only beat cancer by fighting smarter not harder.
Freddie, aged 4, was diagnosed with embryonal rhabdomyosarcoma in his left middle ear with parameningeal involvement, in July 2017. Sadly, Freddie passed away in October 2018, aged 5. His tumour returned aggressively to his brain, a new location. No treatments were available to stop it's growth.
Freddie was diagnosed with Rhabdomyosarcoma in late July 2017. He had started to feel unwell a few weeks earlier with an ear ache. After a few days a lump appeared in his neck. We went backwards and forwards to the GP and ENT specialist.
We were extra wary as in March 2017 Freddie’s younger brother had been diagnosed with a brain tumour and was receiving chemotherapy after a successful tumour removal. We knew the strange signs of cancer, we had trodden this path before, we had that awful feeling again. It just couldn’t be.
Freddie was suspected of having a severe infection in his neck. He started strong antibiotics. Then, trapping his finger in the art cupboard he turned around crying only half his face was not crying. He had facial palsy caused by what we now know was the tumour. Still we were sent away from A and E...... “no mum it’s not what you think it is”.... I will never forget those words.
A few days later Freddie was taken to GoSH where he started chemotherapy. A few weeks later we were splitting ourselves between boys at the were treated in separate countries! Freddie underwent proton treatment in Florida while Arthur continued his treatment in the U.K.
After months of madness the boys finished their treatment in the same day with an hour of one another. We started to recover....
However just weeks later Freddie started to have seizures. The tumour has returned, this time in his brain. He was given weeks. He fought every step of the way and died a few months later in our bed at home. Arthur remains in remission but desperately misses his brother. They had a deep bond as brothers and this reached even greater depths as they battled cancer together.
Freddie's parents are active supporters of Alice's Arc and have been fundraising for the last few years.
Freddie remained joyful throughout his cancer treatment. Always full of bounce, laughter and love. He had his brother to go through the journey with. They had their dressings changed, they talked about their wigglies, they took medicine together. They looked after each other through it all. Freddie was brave, determined and magical. You were lucky if you met him.
We would like to see quicker diagnosis and more awareness for parents. We would like to see kinder treatments. We would like more care given at home. We would like to see support for siblings of children with cancer and their friends.
Frank, now aged 6, was diagnosed in July 2016 and is currently three years clear of cancer. He was diagnosed with embryonal rhabdomyosarcoma in the nasal pharynx with optic involvement. His care was led from the Royal Marsden, Sutton.
In June 2016, Frankie, aged 2, started to wake at night screaming and saying that his nose hurt. We took him to the local GP and they said nothing appeared to be wrong with him. This continued for a few weeks and, one evening, when he was particularly upset, we took him to our local hospital, Pembury in Tunbridge Wells. They looked up his nose with an eye phone light and said there was nothing to see. He was now beginning to get streaks of fresh blood and mucus from his right nostril. We returned to our GP and they said he probably had a nasal infection and provided us with various nasal sprays. After the 6th trip to the GP when Frankie had blood coming from his right eye, we were sent to Pembury again. After a 5 hour wait they looked up his nose again and said that they could see something shiny and that they didn't want to pull it out in case it was attached something. The Consultant, we needed to see was away for a week and so we were sent home again. During that week Frankie was so ill and in so much pain that we had to go to A&E twice. However, we were sent home as the Consultant was still away. By this point there was now a visible growth coming out of the bottom of his nostril, which was very bloody. When we finally saw the Consultant, he concluded that Frankie had picked his nose and the body had over repaired itself and created granulation tissue. He said it was nothing to worry about but that they would needed to remove it Under GA. They decided this approach would be better than an MRI given that the MRI scanner was so busy and it would be a longer wait for the scan. The Consultant managed to remove part of it but told us that it was bigger than expected and was positioned deeply in the nasal passage and too risky to remove. By this point Frankie was very poorly and we were sent home. He began to bleed from his eye and nose and I demanded to have an MRI scan. Finally, the scan revealed a large mass engulfing the nasal pharynx, moving the brain out of its way, destroying a bone in the back of his nose and lurking behind his eyes.
The diagnosis was embryonal rhabdomyosarcoma, a word we had never heard before. Frankie was transferred to St Georges Hospital, Tooting where he had a bone marrow aspirate, surgery to fit a Hickman line and another MRI scan. Emergency chemo began but we noticed that his right eye had become fixed. The Moorfields eye division at St Georges confirmed that his optic nerve had been damaged by the tumour and that he had probably lost the vision in his right eye.
Frankie underwent three rounds of intensive chemotherapy at the Royal Marsden in Sutton. Given that the tumour was inoperable, he was approved to have proton radiation in Oklahoma City, USA. Proton beam therapy is only suitable for a small number of people. It is used to help reduce the risk of long-term side effects that can often develop after standard radiotherapy. It can also be used to treat cancers that are close to critical structures in the body to minimise damage to healthy areas. He had 32 doses of proton with a GA each time. Alongside proton therapy, Frankie had three further rounds of intensive chemotherapy. During this time a neuro-opthalmologist confirmed that he was blind in the right eye. We spent 3 months in the US before returning to the UK for two further rounds of chemotherapy.
Frankie was scanned at the end of his treatment in February 2017 and the fantastic news was received that there was no evidence of disease! All clear! His Hickman line was removed and life returned to somewhat normality. He was been scanned 3 monthly for the last 3 years and his most recent scan in February 2020 he still remains clear. We are extremely lucky and forever grateful for the Royal Marsden and the Oklahoma proton centre for all they have done for Frankie. He is now 6 years old in Year 2 at primary school and loving life. He will have one more scan in July 2020. He requires a growth hormone injection everyday to stimulate his growth and he has also undergone surgery for cataracts in his eyes which have resulted from his treatments. Frankie and his family have been actively fundraising for Alice's Arc since they were introduced in 2016.
From a terrible misdiagnosis which resulted Frankie loosing the sight in his right eye which was a hard pill to swallow. The constant fear of infection the relentless taking of his temperature as if a spike that would lead to 3 days hospital for IV antibiotics in a hospital that didn’t have an oncology ward it was a general hospital not an ideal place for a neutropenic child. Frankie underwent 7 blood transfusions and 3 platelet transfusions. We found our time in America a positive one and were looked after very well by the hospitals there. The journey was hard and terrifying at times, people often said I don’t know how you're doing this, the answer was we have no choice in the matter we had to fight and stay as strong as we could for Frankie and his big sister Layla.
We would like to see kinder treatments, less mis-diagnosis. GP’s & hospitals should be educated more about rare children's cancers to know the signs to look for. More awareness to be raised within communities, more adverts on TV or in newspapers for fund raising and awareness to be increased in the UK.
Izzy, now aged 20, was diagnosed with orbital parameningeal rhabdomyosarcoma in 2004, a week before she turned 4. She is a survivor but suffers from extensive long-term effects as a result of her treatments.
Like any normal toddler, Isabella spent quite a lot of time scrabbling around on the floor and one day, whilst at her grandparents’ house, she bumped her head on the kitchen table. Isabella didn’t cry and the bump didn’t seem too serious so the family continued as normal. The following day however, a swelling started on the side of her head by her temple and over the course of a week, became much bigger. Naturally her parents became worried and sought medical help at their local doctors surgery, however, the GP was unsure what might have caused it and after checking Isabella over for signs of further damage, provided the family with a course of antibiotics. To be on the safe side, her parents took Isabella to the dentist and the optician for general check-ups and both were passed with nothing serious noted.
Throughout the course of treatment we felt safe at the Royal Marsden and contained in our chemotherapy world. Once treatment ended, we felt lost and alone. The anxiety about the threat of relapse was difficult to cope with. However, 15 years later and she is still here.
The transition in to adult care has been difficult and we would like to see changes in this area. There is no support and no one on the end of the phone. Multiple consultants are involved, rather than one, which is a nightmare. Unless you have a social worker you are responsible for getting them all talking about the long-term side effects and the necessary actions. For example, Izzy needed eye surgery and the eye hospital needed a letter from Great Ormond Street about pre-op and post-op management and we had to co-ordinate it.
Esther was diagnosed with stage 4 alveolar rhabdomyosarcoma in December 2014, a couple of months before her third birthday. Following first line treatment, Esther was diagnosed with disease progression in March 2016 and sadly earned her Angel wings in June 2016, aged 4 years and and 3 months old.
Esther Rosa Edwards was born in Worcester on the 29th February 2012. She was a happy and healthy baby with a lovely temperament and soon grew into a confident toddler with an outgoing personality. Esther had a very caring nature and loved helping out at nursery looking after the little babies. In December 2014 Esther developed a firm, painless swelling on her forearm and soon after she was diagnosed with high risk alveolar rhabdomyosarcoma, a rare form of childhood cancer. Sadly her diagnosis was very grim, with less than 20% survival rate. She had no other symptoms and apart for her lump she was completely fine, which made her poor prognosis hard to believe.
On the 2nd January 2015, when she was just under 3 years old, Esther started treatment at Birmingham Children’s Hospital. She underwent 9 cycles of intensive chemotherapy followed by 7 weeks of radical radiotherapy to her arm. The cancer responded well to the drugs and Esther seemed to sail through the treatment without many side effects. In between the hospital stays, that she hated, she was able to keep doing all the things she loved and she never lost her beautiful smile.
Esther was incredibly brave and always kept a positive attitude, making everything look fairly easy. In February 2016 Esther started to complain of headaches, being sick and generally poorly and then devastatingly at the beginning of March 2016, a MRI scan showed extensive disease in her head, and she was pronounced terminal. Esther’s family decided to spend whatever time was left with her to make precious memories, far from hospitals. The disease was even more aggressive now and her health declined very rapidly. Her medications mainly kept her pain at bay, and Esther spent the last three months of her short life on palliative care in her beloved home: colouring, reading her favourite books, going for walks in her pushchair, eating ice creams and ice lollies. She even went on holiday with her family to the seaside and to the woods.
Esther died peacefully at home, surrounded by her family on the 7th June 2016, aged 4 years old. Esther left behind her little sister, Alice, who was only 15 months old at the time, and her mummy and daddy, who miss her every day more than words can say.
The first few months were very very hard on Esther's little body, with hard chemo treatment and all its side effects. Esther had temperature spikes and had to be repeatedly admitted to hospital with low blood count. Following this harder period and the birth of her little sister Alice, things improved considerably and Esther sailed though the rest of her treatment. She didn't like like the hospital stays but we never made a big thing of it, trying to make her life as normal as possible. Esther was still having play-dates with her friends, went to nursery when she felt like it, and spent the most precious time at home with her family. Esther was very brave, enjoyed been surrounded by the people she loved and never lost her happy and bubbly personality until the very end.
Definitely less harsh, more targeted treatment and a better chance for survival. A cure. The support around cancer families in the U.K. is good, our children community nurses were amazing: they were always there for us until the end and I can only praise how the medical team guided us through palliative care, the hardest time of all. The problem in my view is the lack of research and funding in new treatments that could save our precious children's life. Children diagnosed with some type of cancers, like ARMS, don't stand a real chance because there is no proven protocol able to cure them. They have to go through months of gruelling treatment and even if the cancer seems gone often it comes back. Even if if doesn't come back the therapies and procedures when underwent were often so harsh that the children never fully recover and are scarred for life.
We must do better. We must give our precious children a better chance. That is why charities like Alice's Arc are so important.
Jessica, aged 8, was diagnosed with stage 4 alveolar rhabdomyosarcoma, in June 2019. She sadly died 3.5 months after her diagnosis. The cancer had unexpectedly spread to her brain and spine, in spite of scans a few days before showing she was free of disease.
Jessica was diagnosed with stage 4 alveolar rhabdomyosarcoma in June 2019. Sadly, by the time she was diagnosed she was very ill. The primary source was thought to be her right foot, where a lump had appeared some months earlier, but had been wrongly diagnosed by a soft tumour specialist at Spire Bushey as a haemangioma (a collection of blood vessels that swell up). Jessica first started to feel ill two weeks before her diagnosis and rapidly deteriorated. She had seen the GP twice in the first week and visited A&E three times in the following week, but despite (what we now know to be) warning signs, Jess was wrongly sent home each time. We were eventually referred to an oncologist at Watford General Hospital who acted very quickly as soon as she saw Jessica and admitted her to hospital for further tests that same day. Two days later we were delivered the news that a tumour had been found on her spine and it was clear there was bone marrow involvement too and Jess was transferred by blue light ambulance to Great Ormond Street Hospital (GOSH), where later that week she was diagnosed with ARMS. Further tests soon revealed the extent of her disease, which by this point was wide spread. Jess needed daily blood and platelet transfusions and started chemotherapy straight away. After a month in GOSH she was well enough to return home in between her chemotherapy treatments, of which there were to be nine rounds. After Jess’ sixth round of chemotherapy (in October) Jess was sent for scans to show how effectively the chemotherapy was working and the results showed that she had responded well, with all the tumours presenting as ‘dead’ and the bone marrow had also cleared. Unfortunately, two days before getting these results, Jess had started to feel poorly again and was having bouts of dizziness. Several trips back into Watford Hospital and she was again blue lighted to GOSH where she was placed in intensive care. An MRI showed that the cancer was present in her spinal and brain fluid. This is something never seen before at GOSH. Despite attempts to deliver chemotherapy directly into Jess’ spinal fluid, she died suddenly, after a week in ICU, on Friday 18th October 2019 following a cardiac arrest. Jess' death has had a huge impact on her family. She has a brother Finlay, who was only 6 when she died. She was also very close to her cousins and in particular, Shihung, aged 12, who was like a sister to her. Jess' parents Laura and Simon are in the process of setting up a foundation, in memory of their amazing Jess, that will raise funds for Alice's Arc.
Her experience was horrific. We made the decision early on not to tell Jess she had cancer as she was an anxious child and we did not want her to worry. We felt shocked by the lack of treatment options available and it was heart-breaking watching her.
Jess was initially seen by a GP who told us we needed to wait three weeks for a scan, so we decided to seek private advice and Jess saw a ‘sarcoma expert’ at Spire Bushey, who misdiagnosed her. She was seen by multiple doctors during the two weeks of illness leading up to her diagnosis and not one of them picked up on the seriousness of her condition. We feel extremely let down. We were told that a GP may only ever see one child in their career that has cancer. Better diagnosis is essential.
Elsa, aged 6, was diagnosed in April 2016, with embryonal rhabdomyosarcoma in her right nostril. The cancer returned in October 2018 with a large tumour found growing in her right jaw bone. In March 2020, a second relapse was discovered with tumours growing on her liver and stomach. There were no treatment options left. Elsa died on the 14th April 2020.
Elsa, forever 6, diagnosed in April 2016, age 2, with embryonal rhabdomyosarcoma presenting as a small tumour below her right nostril. Thought to be a cyst but post removal biopsy revealed cancer. An unexpected relapse in October 2018 identified a large tumour growing in her right jaw bone. Later identified through biopsy and MRI as spread of the initial cancer. Treatment yet again brutal but successful. In March 2020 stomach pains and a push for ultrasound testing, identified a large tumour on her liver and small growths in her stomach. CT revealing tumours also growing on her lungs. Elsa was almost 10 months into maintenance chemotherapy at this point. With no further treatment available, Elsa passed away 14th April 2020. Elsa's family have been great supporters of Alice's Arc throughout her journey.
We sailed through the initial treatment and naively believed we were the fortunate ones. Elsa hated everything about chemotherapy, hair loss, lack of taste but most of all the lack of control. Her strong, sassy and sometimes aggressive attitude is what kept us all from falling. She was determined not to let this get her down. Our beautiful girl had no desire to be a girly girl but jut to be accepted, hair or no hair. With a personality that always left a huge Elsa sized impression on the hearts she touched, she will be missed by many.
More involvement from the government with better funding for children’s cancer research. New, less aggressive treatments and most importantly, a cure.
Mollie, now aged 5, was diagnosed with embryonal rhabdomyosarcoma, in August 2018. Her cancer is currently stable following the completion of her treatment in January 2020. Mollie's treatment was led from the Royal Victoria Hospital for Sick Children, Belfast.
Mollie was diagnosed, at 2 years old, with embryonal rhabdomyosarcoma in August 2018. The tumour was located between her eye and sinus causing her to lose the sight in her left eye. The only visual symptoms she had was a slightly droopy eyelid which developed over a period of 24-36 hours. Other than that she was very well and a typical 2 year old! The GP initially thought she may have an allergy however the next day I was not happy with her eye and took her to A&E. The nurse who triaged her recognised the warning signs having had an eye cancer as a young child. A CT scan later and a 2cm x 3cm mass was found deep inside Mollie's face. After a series of invasive tests we received the diagnosis of a cancer I couldn't even pronounce. Mollie started intense chemotherapy which involved a hospital admission every 3 weeks for 3 days. In October 2018, we travelled to Jacksonville, Florida so Mollie could receive Proton Radiation. Radiation is an important part of treating rhabdomyosarcoma and despite knowing the awful long term side effects this treatment was needed to try and save Mollie's life. Despite the awful reasons we were in Florida we had a truly wonderful time as a family. Mollie and her big sister Grace still talk about their 'holiday!' On return from Florida, Mollie finished intense chemo and then completed 12 months of maintenance chemotherapy. Treatment finished in January 2020 and Mollie now has regular MRI scans to monitor the residual tumour. Alice's Arc was one of the first rhabdomyosarcoma pages I found and Sara has been supporting me from the very early days!
Awful. Watching your child go through gruelling cancer treatment knowing they might not survive is simply horrific. I don't think I will ever get over the trauma of the last two years. Mollie was strong and just got on with it like it was just normal. She was so young, she didn't know any better. I find following completion of treatment very difficult. Most people assume Mollie has 'beat cancer' (a term I HATE. This is not a competition, race or battle) and we can put this 'all behind us.' What people don't realise is rhabdomyosarcoma is sneaky and unpredictable. It can come back whenever it likes and sadly I've seen this happen far too many times. I live life not knowing what the next day will bring, not knowing if Mollie will see her next birthday or christmas but at the same time I am so grateful she is here and doing well because so many children are not.
I've become a little obsessed about childhood cancer over the last two years and what is clear is treatment options for children with rhabdomyosarcoma is limited and dated but more importantly for some types of rhabdomyosarcoma doe not have satisfactory outcomes. These little children have an incredibly tough time and many do not survive. This is not good enough. We owe it to them to fight for kinder more successful treatment options. Awareness also needs to increase. Doctors need to be made aware of sarcomas in general and always think 'could it be a sarcoma.' The work of Alice's Arc is incredible and a charity I will be a life long supporter of. In memory of Alice and all those other little children who have lost their lives. WE CAN make a difference.
Gaspard, aged 4, was diagnosed in December 2018 and is currently receiving maintenance chemotherapy for stage 3 prostate embryonal rhabdomyosarcoma at Great Ormond Street Hospital. He has responded well to treatment and his tumour has shrunk substantially.
Gaspard was diagnosed at age 3, with stage 3 prostate rhabdomyosarcoma with a large 10x11cm tumour, involving bladder and lymph nodes. The tumor was so large that it was blocking most of his vital organs. Before the diagnosis, Gaspard experienced severe constipation, tummy pain and leg pains. Gaspard was rushed to Great Ormond Street Hospital in December 2018 as he could not urinate and his parents were given the diagnosis after tests and scans. He had to have 2 nefrostomies placed to drain his kidneys, a catheter to drain his bladder and a stoma bag to drain his bowel. Gaspard's treatment involved 9 cycles of Ifosofamide, Vincristine, Actinomycin (IVA) chemotherapy and 28 days of Proton Therapy in USA. Currently he is doing 6-12 months maintenance chemotherapy treatment. His response has been positive and the tumour has shrunk from 10cm to less than 2cm. Gaspard is French and is part of a family of 5, with two sisters. His parents Benjamin and Caroline have lived in London for over 15 years and work in investment banking and financial services. They have set up a foundation called Hope With Gaspard to raise funds for Alice's Arc and to promote awareness of Gaspard's journey and childhood cancer.
Gaspard kept his smile and innocence through out his cancer journey. His strength and courage have made his parents very proud of him.
We need to see more awareness for GP's around rhabdomyosarcoma and sarcoma cancers in general. There needs to be more communication between families affected by cancer and more communication around treatment options and drugs so families can make well informed decisions. There also need to be better treatments and clinical trials available for rhabdomyosarcoma.
Ruby, aged 4, was diagnosed with stage 4 alveolar rhabdomyosarcoma in September 2017. At diagnosis, Ruby had a primary tumour in her left lower calf and metastases in her thoracic spine, pelvic area and lungs. Following successful 1st line treatment and maintenance chemotherapy, Ruby relapsed in February 2019 and sadly passed away on 3rd January 2020, 2 weeks before her 7th birthday.
In July 2017, we noticed that there was a slight swelling at the bottom of Ruby’s left calf, just above her ankle. It was very subtle, but there was a definite difference compared with her right. She had no symptoms of anything. She was a perfectly happy, apparently healthy, very active 4 year old, about to start primary school.
Our friend and next door neighbour is a GP so we asked if she would have a look at Ruby’s leg, which she did, the same day as we noticed the swelling. She said it was probably nothing to worry about but to make an appointment with our GP for when we returned from holiday. We then went away for 2 weeks and saw the GP on our return. Following that appointment a referral was made to our local hospital, Ninewells, in Dundee.
We were fortunate that one of us works in Ninewells with good contacts in paediatrics and orthopaedics. We managed to find out that an orthopaedic referral had been made but this had then been changed to paediatric haematology. We chased the referral directly with senior clinicians in Ninewells and Ruby was eventually seen within a few weeks of the referral being made.
Following the first review in Ninewells, an MRI was ordered and we found out that it was highly likely to be cancer. We were then referred to the nearest specialist centre, Edinburgh Sick Children’s hospital, for further tests. By the time a diagnosis was made and treatment started, Ruby couldn’t stand or walk because the tumour in her spine was wrapping around her spinal cord. She was waking up during the night with a sore back.
Ruby received 9 cycles of IVADo chemotherapy and 6 weeks of radiotherapy in Edinburgh. A couple of months into treatment she could walk again; the chemo was working. We had regular admissions to Ninewells during this time due to febrile neutropenia. The most difficult period was during radiotherapy; it really affected her, especially her oesophagus. For the first time during her treatment, a nasogastric tube was inserted. She hated it. Her weight plummeted, she didn‘t eat, refused to have feed down the tube and only drank iced water. She spent a week in isolation in Ninewells with shingles.
Once the radiotherapy was complete there was a short break before starting maintenance chemotherapy which she tolerated really well. At this point she was like a normal, healthy child, doing everything a young child does and loving life. The maintenance finished in November 2018 and her scans were clear in December.
In February 2019 Ruby complained of a sore back. Scans revealed relapse and soon after that she commenced 2nd line treatment – VIT chemotherapy. A PEG tube was placed into her stomach for the oral medication to be given, to make her quality of life better. Unfortunately, after about 5 cycles, scans revealed progression in her spine so our consultant started looking for the next option, as did we. There was no clear pathway. She started a new protocol in October 2019 but it didn’t work and she didn’t tolerate it well. The tumour in her lumbar spine quickly took over. She was paralysed from the waist down. She was incontinent. She was in severe pain. The rhabdo was spreading. The next few months were extremely difficult, with a short reprieve following emergency radiotherapy which gave her significant pain relief, but we were losing her and we knew it. She now had tumours behind both of her eyes. She developed pneumonia and had to be transferred by ScotSTAR to PICU in Edinburgh following a left lung collapse. They saved her life and she managed to make the most of Christmas while in Edinburgh Sick Children’s hospital. She was then transferred back to Ninewells where she remained until she left us, peacefully, in the early hours of 3rd January, the day after her Daddy’s birthday.
Ruby's family have set up a foundation called Be More Ruby and have raised more than £60K to date. These funds will go towards research in to rhabdomyosarcoma.
At times it was difficult but for most of it Ruby was happy, active, resilient and led a pretty normal life. She didn’t enjoy getting chemo but she never really complained and always made the most of the situation, having plenty of fun while she was in hospital, when she was well enough. At the start she saw it all as a big adventure, excited about having a sleep over in the hospital. The last few months of her life were horrible but even then, we managed to have some fun times and made some brilliant memories.
We need a cure. If we can’t find a cure we need a treatment that successfully manages the disease. The current treatment, chemotherapy that’s been around for decades, isn’t a cure. It treats the symptoms – the tumours. It doesn’t cure the disease, not the type of rhabdomyosarcoma that Ruby had. The treatment just isn’t good enough. There need to be more, better options and more, better research is needed to achieve this. There also needs to be better sharing of outcomes by clinicians globally to improve understanding and awareness of options for 2nd & 3rd line treatment. Lastly, medicines that could provide benefit need to be more available to children especially when there are limited treatment options. Due to patient numbers there will not always be a lot of evidence, but when you run out of options anything that might work is worth trying, as long as it’s safe.