The right chemistry

In the first of a new series about high-achieving Kiwis, Dame Margaret Brimble tells CAITLIN SYKES how she discovered a breakthrough drug that is changing many people's lives.

- CAITLIN SYKES

Dame Margaret Brimble is already well known as one of New Zealand's most distinguished scientists. But of all her professional achievements so far, the latest tops them all, she says. "This is the biggest," the organic chemist says of the announcement last month that a drug based on a molecule she discovered more than two decades ago has received US Food and Drug Administration (FDA) approval.

The drug, trofinetide, is a synthetic version of a molecule essential for cognitive and motor function. In some people, a gene mutation inhibits formation of the molecule, causing a condition called Rett syndrome, which has symptoms that vary in severity, but are similar to cerebral palsy and autism. It almost exclusively affects females, emerging at around six to 18 months of age in an estimated one in 12,000 girls.

Brimble describes news of the FDA approval - a first for a neurological drug discovered in New Zealand - as "surreal".

"It's like a marathon where you've reached the end and can hand it over to someone else. Now it can go somewhere useful," says the Distinguished Professor and director of medicinal chemistry at the University of Auckland.

"As a female scientist, it's also really lovely to know it's a drug for females. With all the female scientists I've worked with over the years, I see this as a legacy for them."

Brimble's work on trofinetide has connected her to members of the Rett syndrome community for many years. She talks about receiving handcrafted gifts from US-based woman Katelin Lancaster, who has Rett syndrome and whose mother, Melinda, made necklaces featuring the trofinetide molecule for patients taking part in clinical trials for the drug.