Vaccine Hope for Children With Neuroblastoma

Highlights:

Beau and her family help raise funds for a cancer vaccine study
Scientists are attempting to put an end to the heartbreak caused by high-risk neuroblastoma, which kills half of those afflicted
The UK has signed a ground-breaking agreement with BioNTech for cancer vaccine studies

In collaboration with the Daily Express, Solving Kids' Cancer UK is urging the UK government to commit to a long-term investment in childhood cancer research, beginning with financial support for the UK to lead Europe in a transatlantic clinical trial of the Bivalent Vaccine for children with high-risk neuroblastoma that could save the lives of many children, like brave six-year-old Beau, who has been diagnosed with a rare and deadly cancer.

‘Solving Kids' Cancer, UK joins up with the Daily Express to urge the UK government to fund pediatric cancer trials’

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'Beau’s Fight Against Cancer’- The Campaign that has Taken Over Britain

A couple of years ago, on December 23, 2020, Beau, a young child, was diagnosed with stage 4 high-risk neuroblastoma, a rare form of childhood cancer. It was the hardest year for Beau and her family. With grit and determination, Beau fought for her life, but it is known that the fight is not over. With such a high rate of relapse and a subsequent bleak survival rate, Beau’s family decided to raise the money to get Beau over to Sloan Kettering Memorial Hospital in the US for potentially life-saving treatment. They have four months to raise £300,000 while working with Solving Kids Cancer.

However impossible, they wake up every day and tell themselves and beautiful Beau that they can do it, that she will live a long, beautiful life, and that her fight was worth it.

Beau’s mom, Shirley Hepworth, said: "This could happen to anyone. You cannot do anything to 100 percent prevent cancer, and it doesn’t discriminate. Of course, I want my daughter to have the best chance at life, but this isn’t just about Beau. This campaign is about protecting future children."

Inheritable Genetic Changes Linked to Neuroblastoma Tumors
Genetic predispositions of children influence the development of MYCN-amplified neuroblastoma tumors.

Neuroblastoma Prevalence in Children

In the United Kingdom, approximately 50 children are diagnosed with high-risk neuroblastoma each year, and they face one of the most arduous treatment regimens for any malignancy. They have a 50/50 chance of survival, and one in every five people who complete their initial round of treatment experience a terrible relapse ().

Beau was diagnosed at age four after experiencing stomach pains. Shirley, 41, said: "She had stage-four high-risk neuroblastoma. It was in her bone marrow, throughout her skeletal system, and she had a soft tissue tumor around her kidney."

Beau began chemotherapy in early 2021 and underwent a 13-hour operation. When the tumor was removed, it was roughly the size of a rugby ball and had grown around her organs and spine. During her months in the hospital, she had stem cells taken, which were subsequently reintroduced to her body, and she endured more chemo and radiotherapy. The effects were sometimes so severe that she couldn't speak and had to establish her own sign language.

Shirley would have to make a choice when Beau went into remission in the autumn of 2021. An experimental vaccination has been created to lower the risk of recurrence of cancer by teaching the immune system to recognize and destroy neuroblastoma cells.

However, it was only available through research at New York's Memorial Sloan Kettering Cancer Center (MSK).

Shirley began a campaign on Christmas Eve 2021 to raise £317,000, the projected cost of numerous trips to MSK. She was astounded by her community's generosity in Batley and Spen, West Yorkshire, and has raised £691,000. Any remaining funds will be donated to other families or research.

Beau was due to travel to MSK last April, but in a cruel twist of fate, she relapsed 10 days before finishing immunotherapy, the last stage of frontline treatment. Shirley said this was why she never told her daughter that her treatment was over or held a party to celebrate her being cured. She added, "Beau is a living example of how the odds are stacked against these children."

Implications of Experimental Cancer Drug For Neuroblastoma
Scientists have identified safety concerns with a decade-old experimental drug CX-5164 for high-risk neuroblastoma patients, thus, revealing a possible risk for late effects of treatment.

Treatment Trial

The child is currently enrolled in an NHS experiment that combines radiotherapy with antibody injections. If the treatment is effective, the couple hopes to travel to New York before the end of the summer. Beau and Shirley will be joining at least 33 British families who have come to the US trial since 2016.

Although they are appreciative of the opportunity to receive the vaccine at MSK, they would experience significantly less stress and disturbance if it could be obtained here. The Express has invited Health Secretary Steve Barclay to meet with families like Beau and Shirley to learn more about how they might benefit.

Early research discovered that the vaccination elicited an immunological response, and patients with higher antibody levels looked to have a better chance of surviving. More evidence is needed, however, to verify definitively whether it is useful and, if so, to get it registered and approved for NHS usage.

Top neuroblastoma experts on both sides of the Atlantic are eager to collaborate to collect the massive amount of data required.

Taking the lead in the research would highlight America's prowess as a science superpower. It would also lay the groundwork for future collaboration in research into childhood malignancies and uncommon disorders.

If the UK helps set up a trial, British patients would be among the first to receive the vaccine. It would also ensure that our specialists can collect the robust data that regulators and the drugs watchdog require.

A Department of Health and Social Care spokesperson said: "Progress in treatment means the majority of children with cancer now survive, but tragically that is not the case with neuroblastoma, and more research is needed."

"We are working to find new and innovative ways to detect and treat cancers, and just this week we launched a partnership with BioNTech to research cancer vaccines as well as announcing more mobile breast cancer screening units."

Beau has captured the hearts of thousands of people across the country, not just in Yorkshire. In the face of her sickness, she has demonstrated amazing bravery and courage. And she always manages to keep a smile on her face.

The fact that the United Kingdom is home to some of the world's greatest scientists and medical specialists who are eager to take on the challenge and facilitate clinical backing for a homegrown vaccination study that will aid in the fight against all types of children's cancer.

Reference :

Improving Outcomes in Children With High-Risk Neuroblastoma: The Role of Randomized Trials - (https://pubmed.ncbi.nlm.nih.gov/34152837/)

Source-Medindia