Research offers hope for Huntington's patients

The Health Research Council has granted $1.2 million to Waipapa Taumata Rau, University of Auckland Professor Bronwen Connor for a four-year project aiming to develop a treatment for Huntington's Disease.

Connor is testing innovative new techniques that could be used to replace brain cells that are lost due to the effects of Huntington’s Disease.

The genetic condition causes people to gradually lose brain cells called medium spiny striatal neurons, says Connor, who heads the Department of Pharmacology.

Connor’s lab at the University’s Centre for Brain Research has developed a groundbreaking method of reprogramming human skin cells so they become precursors that grow into medium spiny striatal neurons.

Huntington’s Disease affects about 15 in 100,000 people in New Zealand, and Māori have significantly higher rates than other New Zealanders.

The inherited condition typically causes jerky, involuntary movements of the arms, legs, face, and tongue; difficulty walking, speaking and swallowing; and can cause mental health problems, such as depression.

Late last year, Connor and Dr Amy McCaughey-Chapman published groundbreaking research showing that transplants of brain cell precursors helped alleviate movement problems in rats with Huntington’s.

Now, the team is trialling whether placing the precursor brain cells in a supramolecular fibre composite hydrogel that resembles brain tissue will help new brain cells grow and survive.

Connor hopes the gel developed by Associate Professor Jenny Malmstrom might allow the new brain cells to generate more connections between each other and within the brain.

“We’re hoping to see more replacement cells growing and bigger improvements in the symptoms of Huntington’s by using this new gel.

“It gives the cells a beautiful environment to grow and extend their branches and make connections,” says Connor.

The study will involve transplanting precursor brain cells into rats. The researchers will be able to see whether the new brain cells are firing.

Movement tests will be used to assess whether movement problems in rats with Huntington’s are alleviated after the transplants.

The transplanted cells will then be 'turned off', to verify whether any improvements are related to the transplants.

Connor says a new treatment for Huntington’s is needed, because no treatments are available at present to slow the degenerative disease or stop it from causing increasingly severe movement difficulties.

“We really want to try to find something that might help these patients – and we believe cell-replacement therapy holds a lot of promise,” she says.

If the research demonstrates the treatment works, a first trial on a person with Huntington’s might be possible within five years. From there, bigger clinical trials could be carried out within a decade.

The exact gene responsible for Huntington’s was only discovered in the early 1990s and a genetic test followed shortly after.

“Before this, there was a huge stigma associated with Huntington’s.

“It was often undiagnosed or misdiagnosed.

“They used to get sent to mental hospitals and a lot of people with Huntington’s took their own lives,” she says.

Connor has spent decades studying Huntington’s and Parkinson’s diseases.

In 2018, she was made a Member of the New Zealand Order of Merit for her contribution to the treatment of neurological diseases.

Her research has resulted in three patents – these cover her techniques for growing precursor brain cells from skin cells for people with Huntington’s, growing dopamine precursor cells for people with Parkinson’s, and growing oligodendrocyte precursor cells for people with multiple sclerosis – a process developed with McCaughey-Chapman.

• Learn more about the use of animals in research and teaching at the University of Auckland.