A Drug Spurring Growth in Children With Dwarfism Causes Controversy

Not everyone in the little people community is advocating for the controversial drug that’s promising to increase the growth of people with achondroplasia, the most common type of dwarfism.
The novel formula, vosoritide, is the center of a controversial experimental study which administers daily injections until the patient reaches age 18, and decides for themselves whether or not to continue. The therapy is intended to help children with a condition known for its short stature reach standard height expectation. The study’s lead author and MCRI’s clinical geneticist Dr. Ravi Savarirayan explained that the growth would also help the children avoid other medical complications and surgeries later in life. Still, much of the community in dwarfism doesn’t like the idea of a treatment seen as cosmetic given to children. They also object to the idea of administering a therapy with the potential to undermine short stature pride.

Achondroplasia

Achondroplasia is a rare genetic bone growth disorder that stunts growth, causing dwarfism. People with the condition experience stunted bone growth in the arms and legs, spine, and base of skull, on average reaching 4 feet, 4 inches for men, and 4 feet, 1 inch for women. The condition causes patients to have an issue with their cartilage’s ability to convert to bone. The term “achondroplasia” means “without cartilage formation.” It also can cause patients to have other physical curvatures, including spinal cord curvature and compression, bowed legs, decreased muscle tone, apnea, and difficulty bending elbows.

A Small Molecule To Make Novel Drugs

In 2011, the community finally found answers when scientists discovered the small molecule CNP, a key regulator of bone growth. This led to the manifestation of the novel drug, which could potentially stimulate bone growth in children. Ravi began trials in 2013, and they found success in increasing growth and confirmed that the drug was largely well tolerated. He explains the effect as similar to overwatering a plant:
“Basically vorsoritide kinks the hose so that the plant gets the right amount of water and can resume regular growth.”
As explained in News AU, Daisy Broadway made the choice to go ahead with the treatment for her son Casper, noting that while it promises him the same height expectations as his peers, it also helps him avoid potential surgeries and complications down the road. Still, she’s careful with divulging their health care choice to too many people. Broadway said,
“It is controversial and it’s a tricky one and I sort of feel sometimes I have to filter who I talk to about it. There’s some people who are vehemently against it and that’s totally fine. Their attitude is they’re trying to fix something that doesn’t need fixing and undoing work that’s been done in short statured pride.”

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