Proteome Systems touts Huntington's biomarker find

Six months from completing an 18-month collaboration with the US-based High Q Foundation, Sydney's Proteome Systems (ASX:PXL) has statistically validated some of the multiple biomarkers for the inherited neurodegenerative disorder Huntington's disease which it identified in an earlier smaller study.

The High Q Foundation has also agreed to fund a two-year human study to validate these biomarkers with hopes of developing an assay to monitor the progression of the disease and the effectiveness of treatments in clinical trials.

"We are currently mapping and planning with High Q what that study will be," said Proteome Systems' deputy CEO Dr Jenny Harry.

Using its proprietary platforms ProteomIQ (a mass spectrometry and 2D gel electrophoresis platform technology) and BioinformatIQ (a bioinformatics platform) for the collaborative program, Proteome has found proteins in human blood samples which may constitute biomarkers for detecting the onset and progression of Huntington's disease.

Huntington's disease is caused by a defect in the huntingtin gene which genetically programs degeneration of neurons in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. People with the defective gene will develop the fatal neurodegenerative disease at some point in their lives, usually between the ages of 30 and 50.

"At the moment the disease can be diagnosed [using DNA testing] at birth," said Harry, who is also Proteome Systems' head of diagnostics, "but we can't tell when it's going to impact on the person -- when it's going to manifest.

"People don't want to be tested because they don't know when it's going to happen or how severe it's going to be when it does," she said. "If however they knew there was a way of determining when that would happen, and the upside of that would be that they could immediately treat the disease, then it would become very useful."

There are currently no drugs available to treat Huntington's disease and rapid drug development requires diagnostic biomarkers to determine drug efficacy.

"The identification of biomarkers that can be used to monitor the pathological changes that occur during Huntington's disease is essential for the development of effective drug treatments," said High Q's scientific advisor Dr Allan Tobin in a statement. "At present, we have no way of knowing if a therapeutic compound is having a desired effect until physical signs appear. This can take months or even years."

Proteome Systems and High Q are considering patenting several of the proteins as candidate biomarkers for the onset and progression of neurodegenerative diseases, including Huntington's.

"We will be doing some longitudinal studies, starting at the very early stages and determining which of these biomarkers present in very early stages and what is their profile during the course of the disease," said Harry. "In this way we should be able to know a panel of biomarkers that are used for measurement at different stages of the disease."

As part of the agreement, High Q will own the Huntington's disease applications of the biomarkers, but Proteome will own the rights to the biomarkers for assays for other neurodegenerative diseases.

"We also believe that the biomarkers we have found are proteins that are common to pathways that are involved in other neurological diseases such as Parkinson's and Alzheimer's and as a result of that we believe they have the potential as biomarkers -- diagnostic/prognostic drug trial markers -- for those other conditions," said Harry.

"We really want to be able to help understand which of these biomarkers might be generic to neurological disorder and which of them might be specific to a disease state."

While Harry could not say how much the collaboration with High Q would be worth because the contract terms had not yet been determined, she said that "it's an agreement to fund the program, there are work effort payments and there are milestone payments."

The initial 18-month collaboration with High Q was potentially worth US$3 million in milestone payments to Proteome. "There are still some outstanding payments," said Harry. "But the majority have been finished and we will now be rolling that into the program."

Harry said that High Q and Proteome Systems will probably work together on developing assays based on the biomarkers and have agreed to contract out the antibody component of the assay development. The work on Huntington's disease and other neurodegenerative diseases will be conducted in parallel.

Proteome Systems' shares were trading at AUD$0.28 at the time of writing, up $0.02 since the market opened.