FightMND Drug Screening Program

This Program aims to find new potential treatments for motor neurone disease (MND). Researchers based at the Florey Institute of Neuroscience and Mental Health will test the effects of thousands of experimental and clinically approved drugs on motor neurones derived from the skin cells of people with MND. They hope to accelerate the process of identifying candidate drugs, which can then be tested on people in clinical trials.
The investigators will convert skin cells from people with MND into "induced pluripotent stem cells" or “iPS cells” in the laboratory. iPS cells can grow indefinitely and be turned into any type of cell in the body. In this research, iPS cells will be converted to motor neurones for drug screening. These motor neurones share the same DNA as the person who has donated the skin sample. A drug that appears to slow or prevent the progression of MND in the laboratory will be identified as a potential MND treatment for further testing in animal models or clinical trials in the case of prescription medications already approved for other conditions.

What is involved? 

People with MND who are willing to travel to Melbourne to donate a skin biopsy and blood sample are invited to participate in this project. This includes people diagnosed with amyotrophic lateral sclerosis (ALS), progressive muscular atrophy (PMA), progressive bulbar palsy (PBP) and primary lateral sclerosis (PLS).

The investigators will collect samples from 155 people with MND and 30 control samples from people without MND in the first phase of the project. Sample collection will take place at the Calvary Health Care Bethlehem Hospital in Melbourne to enable processing within 24 hours. Samples from 25 people will be collected each month from February to September 2018. 

Participants will be asked to donate a blood sample and a 3mm skin biopsy from the upper arm (collected with a local anaesthetic by a physician or nurse). The procedure is quick and simple and takes 15-30 minutes. The researchers will also ask participants about their symptoms, and retrieve information from the Australian MND Register if participants are enrolled.

Is this related to sporadic or familial MND?

The researchers are searching for drugs to treat both sporadic and familial MND. They require samples from a wide range of people with sporadic MND for testing and validation of the drugs. People with a family history of MND or a known genetic mutation are especially valuable to the study, especially those with rare mutations.

Is this a treatment? 

The Program does not involve treating people with MND. The investigators are using state-of-the-art robotics and microscopes to test drugs on motor neurones grown in culture in a research laboratory. This allows them to screen hundreds of potential drugs every week. If they find any drugs that are effective in this environment they plan to do further testing and take promising drugs to clinical trials.

Where can I get more information? 

For more information or to register your interest in participating, visit the FightMND Drug Screening website.

This project has been approved by the University of Melbourne Ethics Committee and all information collected will remain confidential to the Florey researchers working on this project.  For any further enquiries, contact the team at the Florey Institute of Neuroscience and Mental Health at directly or call (03) 8344 5272.

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