Advancing Science Empowering Families Defeating Neuroblastoma · Contents About uS 1 We are working...

Annual Review April 2016–March 2017

Advancing Science

Empowering Families

Defeating Neuroblastoma

Contents About uS 1We are working for and on behalf of children and families affected by neuroblastoma. Find out what we aim to achieve.

About NEuroblAStomA 2Neuroblastoma is an aggressive, complex childhood cancer. Read about its impact and learn the facts.

mESSAgE From our CEo 3This was a busy year for Solving Kids’ Cancer. Find out what our CEO, Stephen Richards, said about our achievements and impact during this period.

FAmily Support 5When a parent finds out that their child has neuroblastoma, life is never the same again. Find out how we support families throughout their neuroblastoma journey.

ACCESS to trEAtmENt 10Treatment as part of a clinical trial often represents a child’s best hope of beating neuroblastoma. Find out how we have supported families to access treatment abroad.

rESEArCh 12Advancing neuroblastoma research is imperative to improve outcomes for children. Find out more about our research work for children fighting neuroblastoma, and those yet to be diagnosed.

ADVoCACy 15Read about our successful attempt to challenge the National Institute for Health and Care Excellence (NICE) on behalf of children with neuroblastoma in the UK.

FuNDrAiSiNg 16Find out about some of the fundraising activities that contributed to our general funds this year.

WhAt WE WANt to AChiEVE NExt yEAr 18See how we plan to increase our impact in the coming year.

our moNEy 20Find out how much we raised and spent this year, and how we manage our funds.

About Us

We are a highly ambitious charity, working for and on behalf of all children and their families affected by neuroblastoma; an aggressive childhood cancer that takes a child’s life every 10 days in the uK.

We are passionate about working tirelessly to achieve significant improvements in the survival of, and care for, all children diagnosed with this horrendous disease. Through our activities, working ambitiously and collaboratively, we aim to improve access to promising and low-toxicity treatments for children in the UK whilst advancing science to improve overall survival. In addition, through our support, information and guidance, we aim to ease the burden on families dealing with the multitude of challenges neuroblastoma presents.

Jamie, diagnosed with neuroblastoma, age three

Annual Review April 2016 – March 2017 1

About Neuroblastoma Neuroblastoma is a particularly aggressive childhood cancer mostly diagnosed in babies and toddlers. it has one of the lowest survival rates of all childhood cancers.

Many children are diagnosed with neuroblastoma only once their disease has progressed to a high-risk stage, which makes it particularly challenging to treat. These children face some of the most intensive medical intervention of any type of cancer – chemotherapy, surgery, high-dose therapy with stem cell rescue, retinoid therapy and immunotherapy – and despite all of this, and enduring the horrific side effects, their chances of surviving the disease remain poor.

Those families affected by the disease often describe it as relentless. For around 10% of children with high-risk neuroblastoma, the disease does not respond to chemotherapy. For another 40-50%, although they may successfully respond to treatment and have no evidence of

disease at some point, they will subsequently go on to experience a recurrence, or relapse. For these children, additional treatment is often accessed through clinical trials in the UK or overseas; but few exist on a global scale. Many children will simply run out of options; one mum said, ‘My son didn’t die because he had cancer; he died because we ran out of options to treat it’.

There remains an urgent need for more effective and less damaging treatments for children, to save lives as well as improve quality of life post-neuroblastoma. Families also require comprehensive support to manage the tough challenges they face throughout the neuroblastoma journey; these are the driving forces behind our work.

• Neuroblastoma is an embryonal tumour that develops from specialised nerve cells (neuroblasts) left behind from a baby’s development in the womb.

• It most commonly occurs in one of the adrenal glands above the kidneys, or in the tissues of the sympathetic nervous system (SNS) that run alongside the spinal cord in the neck, chest, abdomen or pelvis.

• Around 90% of neuroblastoma cases occur in children younger than 5 years of age, with a median age at diagnosis of 19 months.

• It is the most common cancer in infants, accounting for 20–30% of all cancer diagnoses in the first year of life.

• It is the second most common solid tumour in childhood, affecting just under 100 children in the UK each year.

• Neuroblastoma accounts for around 7% of all childhood cancer diagnoses, and approximately 15% of all paediatric oncology deaths.

• Neuroblastoma has one of the lowest survival rates of all childhood cancers, with only 68% of children surviving for five years, versus 83% for all childhood cancers combined.

• Long-term survival for children diagnosed with high-risk neuroblastoma has remained steadfastly at or below 40%.

• On average, 35–40 children age 0–14 die of neuroblastoma in the UK each year, making it one of the leading causes of death from cancer in children.

• The causes of neuroblastoma are not known.

Facts and figures

2 Annual Review April 2016 – March 2017

A message from Our Chief Executive The past year at Solving Kids’ Cancer (SKC) has been exciting, challenging and rewarding in equal measure.

Supporting the families of children with neuroblastoma is central to our work. Throughout the year our Family Support team has continued to act as a trusted guide, helping families to navigate their way through the health system both in the UK and abroad.

We have worked hard to consolidate excellent relationships with the clinical teams in the principal treatment centres across the UK; informing, supporting and empowering families to ask the right questions in order to make informed decisions about their child’s care. This will continue to be at the heart of our work going forward.

Our annual Neuroblastoma Parent Education Conference 2016 was a great success. Families, clinicians and researchers came from across the world to discuss some of the latest clinical research in the world of neuroblastoma.

As always, it was a humbling experience. We were so impressed by the knowledge, passion and tenacity of the parents together with the commitment, expertise and humility of our speakers. It was also wonderful to see so many of our children enjoying each other’s company and, in particular, a visit from Father Christmas!

The months of July to November were dominated by our appeal against the National Institute for Health and Care Excellence’s decision not to approve the anti-GD2 monoclonal antibody dinutuximab for use on the NHS in patients with high-risk neuroblastoma; at that time the only drug ever specifically developed against the disease.

With the combined support of international clinical and ‘patient’ experts and the pro-bono support of Covington & Burling LLP, we successfully overturned the decision. This is the first occasion a charity has done so; challenging the scientific basis underpinning NICE’s ruling, and using the UN Convention on the Rights of the Child to support our appeal case.

Whilst it was incredibly disappointing to hear in early 2017 that the company marketing and producing the drug had underestimated its world-wide demand and was, therefore, restricting availability to solely the North American market, we learnt much from the experience. We will not hesitate to act again, standing alone if needs be, to secure the rights of children to access cutting edge, world-class cancer treatment.

During November to March, a strategic review of the charity’s direction and activities was a key priority. We consulted our main stakeholders to help determine our way forward, all of whom we look forward to working with to deliver our new 5-year-plan.

Neuroblastoma is a deadly cancer, and we must once again sadly acknowledge the many beautiful young children who lost their lives to the disease during the course of the year. They strengthen our determination to bring about change. We also acknowledge the long-standing contribution of Allison Hyde, our formar Chair, who stood down as a trustee early in 2017.

Lastly, we’d like to thank the families, volunteers, supporters, staff and trustees for their commitment to the work of SKC.

Stephen richards

Chief Executive


• Supported 54 families, either emotionally or practically.

• Helped 4 children to access potentially life-saving treatment abroad.

• Facilitated 20 fundraising campaigns for children to access treatment abroad, which raised a combined total of £517,448.

• Approved pastoral grants to 11 families facing financial hardship, totalling £48,815.

• Successfully organised and delivered our 5th annual Neuroblastoma Parent Education Conference, a weekend event bringing together leading neuroblastoma experts from around the globe and families affected by the disease.

• Transferred £41,500 to pay for research to look at methods of identifying children who are more likely to respond to a certain treatment, thus enabling patient selection.

• Committed £60,000 of funding to allow The Royal Marsden Hospital to participate in a US-led clinical trial, that will enable children with a particular genetically driven type of neuroblastoma to be treated with the newest and most promising targeted agent here in the UK.

• Committed £94,000 of funding for University Hospital Southampton to lead the first ever transatlantic neuroblastoma study, investigating the use of targeted radiation therapy and two different immunotherapies, to treat children with neuroblastoma that is resistant to conventional therapy or that has come back after treatment.

• Became the 1st ever charity to unilaterally challenge a decision by the National Institute for Health and Care Excellence, successfully appealing their decision not to recommend dinutuximab - a drug shown to increase survival for children with high-risk neuroblastoma, for use on the NHS.

In numbers, this year we:


the moment a family learns of their child’s neuroblastoma diagnosis, life as it was ceases to exist. they are plunged into an unfamiliar, frightening new world where they struggle to understand their child’s disease and come to terms with its impact.Having a child with cancer reverberates far and wide – across professional and home lives, siblings, grandparents, extended family and close friends. Personal relationships and marriages can suffer; siblings can develop behavioural problems due to a changing dynamic that they are too young to understand; and parents witnessing their very sick child being diagnosed with a life-threatening illness can experience significant emotional trauma, on top of the logistical and practical challenges that arise. These demands can lead to one or both parents being unable to work, causing financial hardship.

Furthermore, neuroblastoma presents a unique set of challenges determined by the highly aggressive nature of the disease. The standard treatment plan offered to children upon diagnosis is extraordinarily intensive; requiring long periods in hospital separated from other family members. Notwithstanding an extensive treatment regime, high-risk neuroblastoma has a high rate of relapse, causing families to live in fear of their child’s cancer returning if they do achieve remission; the survival rate for relapsed neuroblastoma is less than 10%.

For children that do relapse or whose disease does not respond to frontline treatment, there is no proven or standard treatment pathway. This means that families must navigate the clinical trial landscape to find additional treatment options for their child, which can be an incredibly complex and overwhelming process.

And as few clinical trials exist globally, many families will seek for their child to access a clinical trial that is not available in the UK. This requires tens, if not hundreds of thousands of pounds, requiring families to launch public fundraising campaigns to raise the necessary costs – a huge undertaking and pressure at an already very difficult time. If they succeed, one or both parents must make repeated trips overseas with their child, causing further emotional and practical challenges for all the family.

Families need support to deal with all of the demanding challenges that they face as a result of this terrible disease.

Family Support


How we help: Providing information, support and guidance

How we support families varies depending on their needs, but can include:

• Helping families to identify and understand available treatment options post-NHS standard treatment, or when their child is not responding to standard treatment.

• Helping families to identify what questions they need to ask their oncologist to enable them to make informed decisions.

• Appropriately liaising between a child’s family, healthcare team and relevant third parties to help achieve best outcomes.

• Helping families to deal with some of the emotional and practical challenges caused by neuroblastoma.

• Facilitating fundraising campaigns for families to help raise the necessary costs of accessing treatment not available through the NHS.

• Helping families to fully prepare for accessing treatment abroad, from cost negotiations with the relevant overseas hospital to organising flights and accommodation.

• Hosting families at our annual Neuroblastoma Parent Education Conference where they can learn about the global research landscape from world-leading experts.

• Providing a pastoral grant to families who are struggling with the financial repercussions of having a child diagnosed with neuroblastoma.

this year, we supported

54 families affected by neuroblastoma and in excess

of 200 families overall.

We are on hand to support families affected by neuroblastoma with the challenges they face throughout their journey with the disease. our frontline Family Support team has personal and professional experience of dealing with neuroblastoma, and the knowledge and expertise to support families in the most effective way.


‘When we first found out that Zakky had cancer, it frightened the life out of us. There’s a lot to take in, but to have SKC there supporting you, and taking over everything, is absolutely fantastic.

‘Vicky (Family Coordinator) said: “We’re here for you, if there’s anything you need to ask, or if you want to go for a coffee to just talk”. Vicky understands the parent perspective and the highs and lows that you go through. Her experience really makes a difference.’

gareth brennan, father of Zakky who was diagnosed with high-risk neuroblastoma the day before his third birthday.

‘Solving Kids’ Cancer’s Family Coordinator (Vicky) has been through the same situation, so she knows and understands. If I had questions about treatment that I wasn’t sure about, or wanted information about a new clinical trial, I talked to her. Or if I was upset and just needed someone to speak to, she was there.

‘It makes such a difference when you’ve got someone who knows what they’re doing, because it takes that pressure off. There’s no way I would have been able to do this myself.

‘Even now Eileidh’s gone, I still speak to Vicky. She’s always still there if I need her for anything – if I have any questions, or just need someone to rant at.’

gail paterson, mother of Eileidh who died from neuroblastoma in July 2017, age 5 years old.

Eileidh

Zakky


Neuroblastoma Parent Education Conference

Navigating the clinical trial landscape can be an overwhelming experience for parents of children with neuroblastoma. one parent said: ‘Trying to find information about clinical trials is like looking for a needle in a haystack. And when you do find it, you can’t follow most of it.’Our annual Neuroblastoma Parent Education Conference, offered as part of our Family Support service, is an opportunity for parents to better understand the global research landscape and some of the options which might be available to their child. The Conference:

• Shares information on some of the most innovative and up-to-date neuroblastoma research from world-leading experts.

• Provides an opportunity for families to engage with the experts in an open forum.

• Brings families together to share experiences and support.

• Connects experts from across the world; creating valuable opportunities to share information and collaborate for the benefit of children with neuroblastoma.

This year we delivered our fifth conference; a two-day residential event. Thanks to sponsorship from United Therapeutics Corporation, we were once again able to provide the event free to families, including the provision of accommodation, food and refreshments, and childcare for children. Watch our conference video to find out more:

www.solvingkidscancer.org.uk/neuroblastoma-parent-education-conference/

96 parent delegates and 32 children

attended our 2016 Conference.

13 world-renowned clinicians and

researchers presented information on their specialist areas. 100% of delegates said they would return again.


‘Excellent conference – so informative and well run; thank you for organising a fabulous weekend. It was great to hear updates on treatments/trials, have our questions answered by the experts and meet so many wonderful families on the same journey.’

Emma Khanna, auntie to a child diagnosed with neuroblastoma.

‘It’s been an incredible weekend, truly fascinating! We learnt a lot about new treatments including relapse and refractory trials. Being in the same room as so many incredible families is one of the most wonderful aspects of this weekend.’

Elsbeth hallam, mum to a child diagnosed with neuroblastoma.

‘Thanks so much for having me at the conference, it was a big success. Not only did I enjoy talking to families, but it was also great to network with my British and European colleagues. I learnt a lot and it was great to see that there is so much more research going on in Britain. I think Solving Kids’ Cancer has facilitated a lot of that. Congratulations.’

Dr. Shakeel modak, pediatric oncologist.

‘I found the conference very interesting and I was struck by the warmth of support for each other. I think other families with children with rare conditions could learn a lot from you. Once again thank you for inviting me.’

Dr. Anne Karenia Anderson, Consultant in paediatric palliative medicine.

testimonials from our 2016 Conference


Access to treatment

many families of a child diagnosed with high-risk neuroblastoma will seek for their child to access treatment through a clinical trial in the hope that it will prevent relapse from occurring or be effective against existing disease. A family of a child that has finished frontline treatment and whose end-of-treatment scans reflect ‘no evidence of disease’ may choose to continue their child’s treatment through participation in a clinical trial, in the hope that it will prevent relapse from occurring. This is in

the knowledge that even when a child is in full remission the chance of relapse is high and long-term survival after relapse is very low.

A family of a child with relapsed neuroblastoma or disease which has not responded to frontline treatment – for which there is no proven or standard treatment pathway – must look to clinical trials for any additional treatment for their child; trials aimed at either slowing down or stopping disease growth, or in the ideal scenario, eradicating it altogether.

As not all clinical trials are available across the globe, sometimes the best option for a child may exist outside the UK. In these instances, families must pay thousands of pounds to access the treatment, which forces many to appeal to the public for help to raise the necessary funds. This is an incredibly onerous and stressful undertaking, particularly on top of having to deal with their child’s poor health and the other challenges that it brings.

How we help: Facilitating fundraising campaigns and supporting access to treatment abroad We support families to raise the funds they need to access clinical trials aboard. We establish the infrastructure, proper administration and governance around campaigns; give families and fundraisers tools and guidance; and enable campaigns to benefit from corporate donations and Gift Aid, which increases personal donations by 25%, helping families to raise funds more quickly.

this year we supported

20 families to raise a total of £517,448 in order to potentially access treatment abroad and 4 families to go on to access treatment abroad, fully supporting them throughout the process.


Mia Chambers’ story A strong willed, determined and loving little girl, Mia was diagnosed with high-risk neuroblastoma in May 2016 at 4 years old. She went through frontline treatment which came to an end over a year later, following which her parents wanted her to access a clinical trial in the hope that it would help prevent cancer returning, in the knowledge of the high relapse rates for Mia’s type of cancer.

Together, we launched a public fundraising campaign to raise the funds. The campaign received incredible local support, enabling Mia to start the clinical trial at Helen De Vos Children’s Hospital in Michigan in August 2017.

Zakky Brennan’s story Zakky was diagnosed with high-risk neuroblastoma a day before his third birthday. He fell ill quickly and had to start frontline treatment as a matter of urgency. This came to an end mid-December 2016, after which there were no additional treatment options available to him in the UK.

His family were keen for him to participate in a clinical trial in America, aimed at helping to prevent relapse occurring; and therefore started fundraising with us to raise the costs. We identified that they needed approximately £134,000 for the treatment and the associated costs of accessing it, which became the campaign’s fundraising target.

Zakky’s campaign received incredible support from the community, raising in excess of what was needed, enabling Zakky to start the first round of a clinical trial at Helen De Vos Children’s Hospital in Michigan in January 2017.

Zakky’s dad, gareth said: ‘When we heard that we had to raise £134,000, we thought: “We can’t do this – where will we find the money?” We just panicked.

‘I can’t speak highly enough about Solving Kids’ Cancer and the value of their support. All the behind the scenes things for the fundraising were just done. And for the clinical trial they said to us: “Here are the dates to go and the flights – does it suit? Here are your tickets, here’s where you’re staying.”

‘When you don’t know what’s around the corner, you just want to spend every single minute with your child. SKC just took care of everything. SKC are brilliant – I wish there was more and more of them.’

mia’s dad, Josh said:

‘When we were first told we needed to raise over £100,000 to get Mia on a clinical trial, we were quite frightened. We live on a council estate, not in a wealthy area. It seemed so far out of our grasp to get the money for the trial.

‘So I phoned up Solving Kids’ Cancer and spoke to Vicky and said “My daughter has neuroblastoma, we’ve been looking at these treatments and we’ve been told to contact you – what do we need to do?” And Vicky said “We can do everything for you”.

‘All this time I’d been thinking “How can I contact the doctors in America? How do I correspond with all the different people?” SKC said they do everything, and told us what we could get access to.

‘I can’t say enough good things about SKC. They do everything for us, all our flights, all our accommodation. We don’t have to worry about these things now, which means we can enjoy being with Mia.’


Research

Accessing treatment abroad undoubtedly places additional demands on families; firstly to raise the necessary funds, and then to access the treatment in a different country and unfamiliar environment, away from family and vital support networks. We work hard to find and fund promising clinical research projects which will provide real benefit to children affected by neuroblastoma in the UK, now and in the future; always advocating for these to be more effective and less damaging. Through focussing our research work on bringing potential new treatments into UK clinics, we

can increase the options available to children fighting the disease today, and, contribute to the development of new and innovative treatments for children diagnosed in the future. The clinical trial projects that we fund are often international in scope because the rarity of neuroblastoma requires collaboration across geographical boundaries, to speed up how quickly trials can be completed and to increase options for children closer to home.

This work is incredibly challenging. In 2016, our trustee Nick Bird became a member of the National Cancer Research Institute Children’s Cancer & Leukaemia Clinical Studies Group (NCRI CCL CSG), and a member of the specialist sub-group for neuroblastoma. He said: ‘Supporting and funding clinical research is no simple task. It takes time, money, and continuous effort. For a variety of reasons, progress in neuroblastoma, and children’s cancers in general, is painfully slow and ponderous. Solving Kids’ Cancer aims to make a difference to children both now and in the future, through the research projects we select to fund.’


in october 2016, we gave a grant of £41,500 to professor Sue burchill, of the university of leeds. professor burchill is working to evaluate neuroblastoma mrNAs in children enrolled in the European bEACoN-neuroblastoma trial.

When combinations of drugs are given to children, we don’t always know which ones are providing benefit and which ones are contributing to toxicity alone. To validate the biomarkers (from blood tests) predicting which children will respond to particular therapies is a giant step forward as a proof of concept for treating children with cancer.

In this study, blood and bone marrow samples from 120 children treated in the BEACON-neuroblastoma trial will be collected in order to analyse levels of neuroblastoma mRNAs – molecules that carry codes to make proteins – and the results will be evaluated looking at which children responded to therapy. If the biomarkers are validated in this research study, they can be used in the future to predict which children are likely to respond to a therapy before they are treated.

This project has significant potential to help the children of tomorrow, by allowing the incorporation of mRNAs into future neuroblastoma clinical trials and helping with patient selection and stratification.

biomarker evaluation of neuroblastoma mrNAs

How we help: Investing in research

Children who are unlikely to respond could be saved from undergoing significantly toxic therapy unnecessarily, and perhaps have an opportunity to begin other experimental treatments earlier.

professor Sue burchill, director of the Children’s Cancer research group at the university of leeds said: ‘We have developed a simple blood test that can identify, at the time of diagnosis, children with neuroblastoma who are unlikely to respond well to current treatment. Being able to spot these children early means they could be offered alternative treatments that might be more effective.

It is now important that this is validated and we define how best to use the test in the clinic for the benefit of these children. With funding from Solving Kids’ Cancer we are currently investigating how this blood test can be used in children with drug refractory or relapse disease, to improve outcome by adapting treatment in real-time depending on patient-specific characteristics and response.

This important research would not be possible without funding, and the hard work, support and commitment of Solving Kids’ Cancer supporters and fundraisers. Thank you.’


MiNivAn In December 2015, together with Solving Kids’ Cancer US, we awarded neuroblastoma experts in England, America, and Germany joint funding for the first ever transatlantic neuroblastoma study. They put forward a collaborative proposal for an innovative trial combining targeted radiation therapy and two different immunotherapies, aimed at treating patients with neuroblastoma that is resistant to other therapies (refractory) or that has come back after a period of remission (relapsed). Band of Parents and J-A-C-K subsequently became co-funders, and together the charity partners each committed one quarter of the total $500,000 project cost. If this trial is successful it will result in a ground-breaking and much needed new therapy becoming available internationally for children with neuroblastoma.

Significant progress has been made with the preparations for this trial. It is hoped that the first patient will be enrolled in the UK in early 2018, and the study also aims to be open in the US and Germany soon after.

Third generation ALK-inhibitor LorlatinibIn 2015, we advocated for the opening of a clinical trial site in the UK for a third-generation targeted drug aimed at a particular mutation found in neuroblastoma (ALK). We successfully engaged researchers in the US and at the Royal Marsden to work together to open the site, to ensure that this important new treatment was available to children in the UK. We subsequently committed £60,000 for the London-based site, co-funding the clinical trial with several US charities. This high profile clinical trial is the first single-arm phase 2 registration trial in children with cancer, and, if successful, will likely result in rapid European Medicine Agency and Federal Drug Agency approval for children with neuroblastoma.

While it was hoped this trial would open sometime in 2017, it is currently planned to open early in 2018. This is the first international trial of its kind, and hopefully the challenges that have needed to be overcome for it to open will mean there is now a blueprint for future collaborative studies, to allow faster implementation from grant award to enrollment of the first patient.

update on funds previously committed:

‘We are delighted that the MiNivAN study is soon to open. Sponsored by the University of Southampton NHS Foundation Trust, the study is investigating combining 131-I mIBG therapy with two different antibodies and will be the first ever transatlantic neuroblastoma study. We were very fortunate to secure the collaboration and support of Bristol Myers Squibb and EUSA Pharma, who will provide Nivolumab and Dinutuximab beta respectively for the study. Whilst the new international collaborations have been very exciting, there have been a number of regulatory challenges to overcome, but together we have been able to move steadily forward with this exciting project. The funding from SKC and other charity partners has driven and inspired the study, and without this initial commitment we would not have been able to secure the investment of the pharmaceutical companies.’ Dr. Juliet gray, Associate professor and Consultant in paediatric oncology at Southampton general hospital.

yael mosse, mD, one of the principal investigators on the trial said: ‘If this trial proves successful, we expect the drug to quickly become part of frontline therapy in children with ALK-driven neuroblastoma – which is a tremendous accomplishment in cancer research, overall’.

To find out more about the research projects we fund, visit: www.solvingkidscancer.org.uk/research-we-fund/projects

14 Annual Review April 2016 – March 2017 Annual Review April 2016 – March 2017 15

Advocacy

in 2016 we robustly challenged the National institute for health and Care Excellence (NiCE) in an attempt to ensure that children’s access to life-saving drugs wasn’t hindered. After NICE had taken the decision not to recommend that dinutuximab (UnituxinTM / ch.14.18/SP2/0) be made available on the NHS to treat children with high-risk neuroblastoma, we lodged an appeal to challenge this.

The appeal took months of intense and painstaking work, including engaging an Associate Professor of Paediatrics from Harvard Medical School and Lead Statistician for the Neuroblastoma Committee of the American Children’s Oncology Group, to evaluate and provide expert commentary on sensitive and confidential clinical trial data, released to us under a non-disclosure agreement (NDA).

We secured pro bono legal support from a leading international law firm specialising in regulatory law, and appeared before the NICE Appeals Committee in September 2016.

Speaking before the hearing, Stephen Richards, our CEO, said:

‘This is the first time that a charity has made a unilateral appeal against a NICE decision, but we cannot stand by and allow NICE to condemn UK children diagnosed with this devastating illness to a reduced chance of survival. If this decision stands, parents will have to raise in excess of £500,000, as well as enormous reserves of emotional resilience and determination, to take their sick child abroad for the greater chance of survival that dinutuximab therapy provides. We are committed to doing everything in our power to ensure that this flawed decision is reversed.’

We robustly challenged the decision and were commended for our emotive but also objective and scientific arguments. Our appeal was successfully upheld on two separate grounds, which resulted in the original decision being referred back to the Appraisal Committee for reconsideration.

Unfortunately, shortly afterwards the drug company withdrew their application for NICE approval because of supply issues, essentially bringing the process to an unsatisfactory and premature close. We were bitterly disappointed by this outcome; and despite our best efforts the uncertainty surrounding availability of this potentially life-saving treatment continues.

Our CEO presented our work at the International Society of Paediatric Oncology (SIOP) conference in Washington, D.C. recently, to demonstrate the important role charities like SKC have in advocating on behalf of children for access to the best available treatment.

Case study: appeal against NiCE decision

Fundraising

in addition to raising funds for children to access treatment abroad through clinical trials, which are designated for this specific purpose, we raise funds for research, advocacy, family support and to cover our operational functions. these are just some of the fantastic fundraising activities that have helped fund our work this year.

Tough Mudder

A 20-strong team from a leading banking and financial recruitment agency – marks Sattin, took on london West tough mudder in April 2016. they raised over £12,000!

British 10K London Run

in our biggest third-party challenge event, over 50 runners took to the capital in July 2016, raising over £6,000.

GamesAid

in September 2016, we received £95,400 from gamesAid, a uK games industry charity supporting disadvantaged and disabled children and young people. this remains our single biggest donation!


Charles River

through their ‘make your mark’ charity initiative, Charles river raised £20,000! the initiative involved inviting exhibition attendees to add their hand print to a specially designed wall in their exhibition booth and donating 5€ for each one.

In-memory Fundraising

Donations and fundraising activities in memory of robyn higgins, Felix White, ben lee, oscar Knox and many other children who are sadly no longer with us, raised £37,864 to support our work.

United Therapeutics Corporation

We are very grateful to united therapeutics for sponsoring £50,000 to fully fund our annual parent conference 2016. their funding meant that we could deliver a full programme free to families affected by neuroblastoma from across the uK.

Christmas Raffle

our annual Christmas raffle raised over £5,000! thank you to everyone who donated a prize, and bought and sold tickets on our behalf.

our sincerest thanks to everyone who has helped to support children and families affected by neuroblastoma.


our visionA future where no child dies of neuroblastoma and no child suffers due to its treatment.

our mission helping those affected by neuroblastoma, by:

•Facilitating access to treatment in partnership with clinicians and researchers

•Advancing science through investment in clinical research

• Advocating for more effective and less toxic treatments• providing hope, information, and support to families

throughout their journeys.

What we want to achieve next year

this has been an exciting, challenging and rewarding year for the charity. going forward, we want to build on our achievements and strengthen the impact of our work, which will be aided by our 5-year plan – a result of our strategic review – and the revision of our vision and mission.


here are some of the key things we aim to achieve in the future:

Support and enable clinical research, accessible in the uK We will play a leading role – working in partnership with prominent researchers and clinical experts, and bringing insight from those impacted by neuroblastoma – to increase, shape, and accelerate UK-based neuroblastoma research.

By influencing the clinical trial environment – and by increasing our own research funding – we will increase the UK’s clinical trial footprint and make

sure more children can access innovative therapies closer to home. To help achieve this we will:

• Assemble a Scientific Advisory Board of international standing, to develop and oversee a proactive Research Strategy.

• Ensure, with the help of our Scientific Advisory Board, that areas of unmet need are identified and addressed.

Strengthen our governance It is vital that we have strong governance to achieve our strategic objectives for families affected by neuroblastoma; ensuring we meet our legal requirements, delivering best practice in terms of data protection and information governance; and developing the talent and ability of our staff and volunteers.

To help achieve this we will:

• Strengthen and develop our Board of Trustees.

• Develop a best practice staff and volunteer journey.

• Continue to strengthen our systems of financial control, risk management, service delivery and impact measurement.

• Ensure we have clear policies in place, especially in data protection, information governance and health and safety.

build our fundraising capability In order to secure our long-term sustainability and achieve our vision and mission, we must significantly increase our net income. We plan to do this by investing in our fundraising team and

evolving our fundraising approach to one that is focused on engaging, motivating and retaining long-term supporters, and using a broader range of funding sources.

Collaboration is key to beating neuroblastoma. We need to seek opportunities to maximise the collective impact of the neuroblastoma community on research, treatment and support, by building meaningful partnerships with all key stakeholders – domestically and internationally.

We will therefore:

• Work with the specialist clinical teams in the

NHS to identify how we can support them to better support children and families affected by neuroblastoma.

• Develop strategic partnerships with healthcare institutions of international repute.

• Identify opportunities to collaborate with other charities.

build strategic partnerships with key stakeholders


Total Income: £1,102,188

Our money

We’re very grateful to everyone who supports us financially. We want you to feel confident that we’re spending your money wisely and also holding funds raised for individual children to potentially access treatment abroad. that’s why we are committed to being open about how we’re funded and how we manage our money.

A further £117,000 of our income was restricted (as per the conditions upon which it was received) and £92,000 reserved for future commitments.

3%

11%

9%

9% 3% 5%6%

51%

2%1%

• Governance and Administration: £145,814• Access to Treatment: £420,664• Neuroblastoma Research: £137,300• Family Support: £188,913

Total Expenditure: £892,691

21%

16%

47%

16%

• Challenge Events: £37,266• Event Sponsorship: £50,000• Community Fundraising: £65,473• Children’s Campaigns: £558,040• 7 & 10% Allocations (from Children’s Campaigns): £35,758• FAN Transfers: £117,932• Charity: £95,500• Corporate: £25,926• Business Development: £13,034• Merchandise: £1,110• Investment: £102,149

0%


74%

22%

Our funds

At the end of the year, our funds totalled £5,412,774. 74% of funds were designated for use for specific children should they need to access treatment abroad. 22% of funds were restricted for use by specific children according to the conditions attached to the receipt of funds. the remaining 4% of funds totalled our free reserves, not sufficient to support our growth and long-term sustainability, nor our continuation should income fall. our essential work currently relies on a diverse and balanced income portfolio of events, community and corporate fundraising.

• General Funds: £208,419• Restricted Funds: £1,207,842• Designated Funds: £3,996,513

Total Funds: £5,412,774

4%


SolViNg KiDS’ CANCEr

CAN Mezzanine, 49–51 East Road, London, N1 6AH

Tel: 020 7284 0800

www.SolvingKidsCancer.org.uk

Solving Kids’ Cancer Europe is a registered charity in England and Wales (1135601), and in Scotland (SCO45094). It is a company limited by guarantee in England and Wales (7208648). The charity and company is currently operating as Solving Kids’ Cancer.

has your child been affected by neuroblastoma? Call 020 7284 0800 for help and information.

Advancing Science Empowering Families Defeating Neuroblastoma · Contents About uS 1 We are working...

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