Parkinson’s UK funds project to ID new therapeutic targets

Effort will involve simulating candidates' mode of action against GPNMB

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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A hand holds up a coin as paper money and dollar signs float downward around it.

Scientists at the Imperial College London in England have been awarded nearly £100,000, or about $125,000, by Parkinson’s UK to identify new therapeutic targets for Parkinson’s disease.

The grant was awarded to Michael Johnson, PhD, and his team, and will fund efforts to harness the power of computers to simulate how best to target glycoprotein nonmetastatic melanoma protein B (GPNMB), which has been identified as a possible biomarker of Parkinson’s.

“It is a great honor to be awarded this grant from Parkinson’s UK. This award will fund us to work with the U.K.’s Nucleic Acid Therapy Accelerator (NATA) to develop a new type of drug against GPNMB as a potential treatment for Parkinson’s,” Johnson said in a Parkinson’s UK press release.

While candidate therapies sometimes show promise in slowing the progression of Parkinson’s in early lab tests, several fail to be effective when they’re tested in clinical trials.

“A major problem with drug development is that half of all drugs will fail during clinical trials due to lack of efficacy — i.e. the drugs don’t work despite extensive testing prior to clinical trials in people,” Johnson said.

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Exploring what makes a candidate therapy effective

Understanding how new candidate therapies actually recognize and target a specific protein may increase their chances of success in clinical trials.

Johnson’s research team will generate computer-based simulations of a candidate’s mode of action to determine how best to target GPNMB, which is elevated in blood levels of people with Parkinson’s. GPNMB also promotes the toxic accumulation of alpha-synuclein, a hallmark feature of Parkinson’s that causes the death of nerve cells and contributes to the neurodegeneration seen in the disease. Reducing GPNMB may prevent or slow the disease’s progression.

Johnson and his team are designing specialized antisense oligonucleotides (ASOs) to reduce GPNMB’s activity. ASOs are short, synthetic nucleic acid sequences that can bind to target RNA molecules, causing them to degrade or interfere with protein production. RNA acts as the bridge between DNA and proteins; it’s an intermediate molecule that transmits genetic information during protein production.

By optimizing these ASOs to target GPNMB, the researchers seek to mitigate the toxic effects of alpha-synuclein aggregation.

The outcomes of this project will help choose the best candidates for clinical trials. The grant was awarded under Parkinson UK’s drug accelerator award grant scheme, which funds early research that helps bridge the gaps in developing new therapies.