Our mission is to raise £500K+ to help children and their families impacted by rhabdomyosarcoma.
We want these funds to help advance the research in to rhabdomyosarcoma in order to find better treatments to lengthen and improve the quality of life of these children and their families. Ultimately, we would like to see treatments in place that can cure rhabdomyosarcoma.
Alice’s Arc was inspired by the ongoing journey of a little girl named Alice, diagnosed with stage 4 alveolar rhabdomyosarcoma, at the age of 3, in March 2015. She had 20 months of chemotherapy and proton radiation in the US. Alice spent one year in remission before her cancer returned in February 2018. She underwent 6 months of relapse chemotherapy and a specialist surgical procedure with brachytherapy, known as AMORE, in Amsterdam.
She was given a 50/50 chance of survival over 5 years. However, relapse made these odds significantly worse. The first symptom Alice’s family noticed was a small lump on her neck, she was completely healthy otherwise. It took around 6 weeks to diagnose due to lack of knowledge available to local GPs and local hospitals about rhabdomyosarcoma. Once the ideas of various infections and cystic structures were dismissed an MRI revealed a solid tumour and Alice received a referral to the oncology department at Great Ormond Street Hospital.
GOSH provided Alice's probable diagnosis within a day and commenced the diagnostic process involving a biopsy, lumbar puncture, blood tests and PET scans. Full diagnosis revealed that Alice had stage 4 alveolar rhabdomyosarcoma with the primary tumour attached to the submandibular gland in her neck with positive lymph nodes and a tiny tumour in her lung. Her treatment begun immediately as the tumour was compromising her airway.
Alice was treated under the care of GOSH and Jacksonville, University of Florida Health Proton Therapy Institute. She received a combination of intensive in-patient chemotherapy, out-patient maintenance chemotherapy and radiation over a period of almost two years. The treatment involved spending 11 weeks in Florida receiving proton radiation which was unavailable in the UK. Proton radiation is a highly targeted form of radiotherapy - the proton beams kill the cancer cells and cause less damage to surrounding healthy tissues. This is particularly relevant to young children so that long term damage can be limited.
When Alice relapsed she had chemotherapy at GOSH and travelled to the Emma Children's Hospital in Amsterdam for AMORE which involved surgical removal of the tumour, brachytherapy and reconstruction.
Throughout the journey Alice’s parents met a worldwide network of medical professionals and families with children undergoing cancer treatments. They became increasingly aware of the lack of funding and clinical research in to the treatment of children’s cancers. And so Alice’s Arc was born with a commitment to help find a cure with less toxic treatment options.
How will we achieve our goal?
- By collaborating with leading institutions like the Institute of Cancer Research, Great Ormond Street Hospital and the Royal Marsden we can help to realise this goal.
We have pledged to support the clinical research that they undertake both in the laboratory and in the hospital. This involves providing funds for a new rhabdomyosarcoma clinical trial due to launch in 2019 which is being led out of the UK and co-ordinated with other European Centres.
For the Institute of Cancer Research, we will provide funds for the salaries of key staff required to undertake the necessary research to help make the clinical trial a success - this involves a Clinical Fellow in the laboratory and a Higher Scientific Officer. These individuals coordinate and analyse samples of tumours and look at gene sequencing data to determine how the tumour is wired. They then experiment in the laboratory with drugs that target these molecular defects and could be used to treat rhabdomyosarcoma patients. We will also fund consumables/lab reagents that help the team with cell culture, drug sensitivity screening and preclinical testing including animal models.
For GOSH we have pledged to fund the role of a Research Nurse working on the clinical trial. This role will help support the collection and processing of blood and other samples for the study including molecular pathology, circulating tumour DNA and PK (pharmacokinetics).
- By supporting patients and families currently being treated for rhabdomyosarcoma by improving the quality of hospital stays and creating opportunities for patients to use drugs that can potentially ease the treatment process.
For example, by providing funds to acquire drugs such as neulasta, which involves a single injection, to help prevent infections as blood counts drop as a result of treatment which can result in long stays in isolation in hospital receiving antibiotics. Neulasta is a drug which boosts white blood cell recovery and involves a single injection of GcSF (granulocyte colonies stimulating factor).
- By raising awareness of childhood cancers like rhabdomyosarcoma.
It is surprising to learn how little investment is made in to research for childhood cancers compared to adult cancers. Research and development budgets in pharmaceutical companies covers 60% for adult cancer drugs and close to zero for children.
This is a complex area involving issues relating to politics, legislation and profitability. We aim to work with other childhood cancer charities and influential cancer research centres in order to lobby for changes that can help to maximise the opportunities to make progress understanding and treating rhabdomyosarcoma.
Please support us to help these children and stop this happening to families in the future.