Kiwi scientists 'want to find a treatment' for group of rare diseases

Three top scientists - from left, Professor Gary Evans, Dr Phillip Rendle and Professor Bradley Williams - have each been awarded million dollar grants for major studies. Photo / Supplied

A team of Kiwi scientists has set its sights on finding a safe and effective treatment for a group of rare but severe diseases.

The nine known polyglutamine diseases, which affect about one in 10,000 people worldwide, are neuro-degenerative conditions caused by the abnormal interaction of inherited mutated proteins.

Best known is Huntington's disease, a brain disorder which causes cells in specific parts of the brain to die, resulting in impairment of mental capability and physical control.

Perhaps its most famous victim was legendary folk singer Woody Guthrie, whose symptoms were at first confused with alcoholism and schizophrenia.

Dr Phillip Rendle, of the Victoria University-based Ferrier Research Institute, said there was no cure for the nine diseases, just treatments for the symptoms, which typically emerged in mid-life and get progressively worse with age.

"I want to find a treatment using dendrimers, which are molecules that look like a heavily branched tree," he said.

"Our aim is to use dendrimers to disrupt this interaction to delay the onset age, which would effectively be a cure."

Dr Rendle will be working alongside Auckland University Huntington's disease expert Professor Russell Snell, who will conduct biological testing of the materials being developed in the project.

The study is one of three million-dollar projects funded through the Ministry of Business, Innovation and Employment's Smart Ideas science investment round for 2015.

Another, to be led by Professor Gary Evans of the Ferrier Research Institute, aims to develop materials which could reduce infections that sometimes require orthopaedic implants, such as hip or other joint replacements, to be removed.

These infections occur when micro-organisms are able to produce biofilms that form a protective layer adhering to different surfaces.

The majority of infections that follow orthopaedic implant surgery are caused by bacteria growing within a biofilm, which develops on the implant.

Where a biofilm is allowed to form, these bacteria are protected from the patient's immune system and antibiotic treatments.

In the study, Professor Evans will team up with researchers from Callaghan Innovation and University of Otago to engineer entirely new materials through coating titanium -- used in the majority of modern orthopaedic implants -- with molecules that stop biofilm formation.

"While removal of a joint is only necessary in 1 to 2 per cent of cases, when that's applied to 3.7 million such surgeries a year in the United States alone, preventing those infections would have a big impact and save a lot of money," he said.

"As the population ages and expects to be more mobile with the help of joint replacements, the issue is only going to become more pronounced."

A third Ferrier study will investigate a new kind of paint that would stop barnacles and other organisms from accumulating on the hulls of marine vessels.

Professor Bradley Williams, who is heading the work, said the growth of seaweed, barnacles and the like on ships was a major problem for marine industries.

"It creates lots of resistance, which means slower ships, and requires as much as 30 per cent more fuel to maintain shipping speeds for on-time delivery -- that's hard on both the financial bottom line and the environment."

Professor Williams said his research was based on an "extremely simple idea" that pulled together existing scientific principles in a new way.

"But the way this idea intends to solve the problem, and the fact it won't have any impact on the environment, means it has the potential to be a game changer for paint manufacturers in the shipping industry."