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High throughput drug screening for motor neurone disease

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.

We require patient iPS cells to test the drugs on, and to verify their effectiveness when we find promising candidates. Participants will be asked to donate a blood sample, and a 3mm skin biopsy from the upper arm (with a local anaesthetic). From these samples, we will generate a genetic map of your DNA, and iPS cells genetically identical to your own for testing of drugs. The procedure is quick and simple, and will only take 15-30 minutes. We will also ask you questions about your symptoms, and retrieve your information from the Australian MND Register if you are enrolled.

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.

We also encourage you to discuss participation in the Program with your clinician if you have any concerns. We will send you a more detailed information pack about the Program and associated risks for your consideration before you enrol.

For people looking for more information on the ongoing CuATSM trial please visit MND Australia.

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