According to a story from the St. Helens Star, Claire Riley, the mother of Kaycee Bradshaw, said in a recent statement that she would not hesitate to move out of her home country of England to save her daughter’s life. Kaycee is six years old and has a rare disorder called Batten disease, and unfortunately her access to treatment could be taken away in a instant. The only reason Kaycee is still getting treatment is because she was fortunate enough to participate in a clinical trial.
Batten disease is a nervous system disorder that tends to appear between the ages of five and ten years. This neurodegenerative disease is caused by mutations which affect the CLN3 gene. Girls tend to display symptoms later than boys, but their disease tends to progress more rapidly. Diagnosis is often difficult. Symptoms include seizures, vision problems, repetitive speech, learning regression or delays, scoliosis, decreased muscle and body fat, changes to personality and behavior, poor coordination, and speech loss. Symptoms generally progress over time. There is only one drug for Batten disease that can slow progression, but it is only effective in certain cases. Most treatment is supportive, and the disease is ultimately lethal. There is a dire need for more effective therapies to improve survival times and outcomes for patients with Batten disease. To learn more about Batten disease, click here.
Kaycee is receiving an enyzme replacement therapy called cerliponase alfa (marketed at Brineura), and is one of just five patients in the country that has access to the treatment. The therapy has halted the progression of her disease. Unfortunately, the NHS has deemed the drug to be too expensive and does not cover the therapy. Claire has been involved in the push to get Brineura covered; two children with Batten disease have died because they could not get treated.
Some parents have had to resort to just what Claire had previously suggested and have had to move to other countries to receive the drug. Claire also leveled criticism at BioMarin, the manufacturer of the drug, for not being willing to compromise on the cost:
“Companies like BioMarin are not putting children’s lives, and the lives of their families first, it’s all about money.”