Ultra-Rare Doesn’t Mean Unimportant: An MPS III Story

According to the Daily Mail, Harley Bond is a 4-year-old living in the UK. He has MPS III, or Sanfilippo Syndrome, a rare disease which has no treatment and no cure.

Recently though, the Bond family had a spark of hope. Harley was offered a spot in a clinical trial. Unfortunately, this hope quickly turned to fear, frustration, and anger. His parents were told that even if the treatment were to work, there would be no funding available for Harley to continue the treatment post-trial. In other words, even if the trial produced results that would save his life, it wouldn’t be accessible to their son.

In other words, even if the trial produced results that would save his life, the drug wouldn’t be accessible to their son.

The basic idea of the trial is to give injections of the missing enzyme, which would hopefully result in the body emulating a properly working system. While there are no guarantees, the treatment does sound promising. But it also sounds expensive. The infusion isn’t a one-time thing; it would have to happen once every single week.

Because in MPS III (as opposed to the other forms of MPS) the enzyme is missing in the brain, that would need to be the location of the injection.

The estimated cost of the treatment is €400,000, or approximately $450,000. The family is advocating against the capping system that the National Institute for Health and Care Excellence (NICE) uses. This means that NICE sets a certain price cap for specific illnesses. Harley’s parents say it should be dealt with on an individual basis.

The dilemma is that Harley’s disease is ultra-rare. There are only 40 people in the entire UK with MPS III. But does that mean those 40 children don’t deserve treatment?

The family is very excited about the upcoming trial, but they fear the future. Their son’s illness may be rare but his care is their top priority.

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