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Motor Neurone Disease Drug Screening Laboratory

The aim of this world-first program is to rapidly find potential treatments for Motor Neurone Disease.

We are using Nobel Prize winning technology that allows us to grow motor neurones from patient cells called “iPS cells” in the dish. We will use these cells in combination with next-generation robotics and powerful microscopes to screen thousands of clinically approved and experimental drugs until we find one that can slow or prevent the progression of MND. With the valuable participation of MND patients, we aim to identify and validate candidate drugs as quickly as possible so that they can be fast-tracked into clinical trials.

The Florey's high throughput drug screening program consists of an enthusiastic team of stem cell scientists, two robots, and a state-of-the-art facility dedicated solely to rapidly finding effective treatments for MND. This Program has been made possible by the generosity of the Victoria State Government, FightMND, and the Balcon Group Pty Ltd. 

We are using living cells from volunteers so we can test drugs directly on motor neurones derived from MND patients. One of the main issues in identifying drugs that may help MND patients is finding a relevant model to test the drugs on. We do not know why most MND patients get sick, which means we cannot easily replicate this process in the laboratory. The recent discovery that skin cells can be turned into iPS cells means that we can now take skin cells directly from a patient, turn them into iPS cells, and use them in the laboratory to test drugs.

What is an iPS cell?

An iPS cell is a normal adult cell that has been returned to an “embryonic” state. This means that it can replicate indefinitely, and can grow into any type of cell in the body. From the iPS cells we can grow motor neurones for the drug screening. These motor neurones share the same DNA as the donating patient, and give us the best possible chance of finding effective drugs for patients.

Who can take part?

All Australian residents diagnosed with Motor Neurone Disease, including Amyotrophic Lateral Sclerosis (ALS), Progressive Muscular Atrophy (PMA), Progressive Bulbar Palsy (PBP) and Primary Lateral Sclerosis (PLS), are invited to participate in the project.

To register to participate, or for further information please email FightMND-DSP@florey.edu.au directly, or call 03 8344 5272.

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