A new treatment could help children with achondroplasia, the most common form of dwarfism, grow taller and avoid health problems in later life. But there are concerns about whether this is ethical
Samuel Gray is very brave about his daily injections. At six-and-a-half, confident and happy, he was a boy who knew his own mind and made a big decision about his future. His parents had asked him if he wanted to take part in a clinical trial for a drug that could improve some of the conditions associated with achondroplasia, the most common form of dwarfism, with which Samuel was born. It would also, the researchers believed, increase his height. “Maybe children don’t know the bigger picture, but they know deep within themselves whether they want to do something or not,” says his mother, Kristina Gray, “and we would never force Samuel to do anything that he didn’t want to do.” She says she is proud of him for taking part, and that for their family it has always been about “the bigger picture, because we never knew if Samuel would benefit, but in the long run, maybe his children or grandchildren will”.
Samuel is now nine and has grown about 50% faster than would be expected of a child of his age with achondroplasia. He has no problem with tasks such as getting dressed by himself or washing his hair, which children with achondroplasia can find difficult because of their shortened arms, has more stamina and can walk for longer periods. “He doesn’t need any help from us. He is like any other child,” says Gray. She acknowledges that she does not know how much of that is due to the drug, given as a daily injection in the evening, but she suspects it has had an effect. Samuel’s legs are straighter – people with achondroplasia tend to have bowed legs, which can make walking difficult or painful, and may require surgery. “Samuel doesn’t; his legs are straight. Hopefully, lots of children will benefit from this. Some of the challenges that these children go through physically – the multiple operations they may have to have – if this puts an end to that, I think it’s incredible,” says Gray.
