What is the FightMND Drug Screening Program?
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 your valuable participation, we aim to identify and validate candidate drugs as quickly as possible so that they can be fast-tracked into clinical trials.
Australian residents diagnosed with MND who are willing to travel to Melbourne to donate a skin and blood sample are invited to register for the first intake.
The FightMND Drug Screening Program consists of an enthusiastic team of stem cell scientists, 2 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. The research will be conducted at the Florey Institute of Neuroscience and Mental Health in Melbourne, the largest brain research centre in Australia, in collaboration with the Centre for Eye Research Australia, University of Melbourne, and the Australian MND Registry.
Why do we need your participation?
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.
What is involved?
We need your cells to help fight MND! 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.
How does the drug screening work?
There are millions of chemical compounds in the world. We will test compounds to find drugs that may help improve the health of motor neurones. We will begin by testing drugs that have already been shown to be safe for use in humans. This means any drugs that are shown to be effective can be put into clinical trials sooner. When we have tested all clinically approved drugs, we will test novel compounds including natural products and known bioactive molecules until we find an effective therapy.
To test the drugs, we use a large robotics platform to grow motor neurones in high-density culture plates. The state-of-the-art robotics platform allows us to treat micro samples of cells with different drugs to determine if they are effective in maintaining the health of the cells. To capture the efficacy of the drugs screened, we have installed an Australian first powerful microscope that can record the health of living cells over a period of weeks. We have equipped the microscope with a specialised automated loading system to maximise the numbers of drugs we can screen every week. We believe that drugs that improve or maintain the health of motor neurones in culture plates may also be beneficial to motor neurone health in patients.
The combination of advanced robotics and the microscope allows us to screen hundreds of potential drugs every week. First, we will screen the drugs on a representative set of patient cells selected from participant iPS cells. The effectiveness of the most promising drugs will then be validated in patient iPS cells from across the Victorian MND population.
What do I need to know?
Firstly, everyone will have a small scar at the biopsy site after the procedure. This is proof of your participation in the fight against MND. There is also a small risk of bleeding and infection following the biopsy, however all procedures are conducted by medical specialists to minimise this risk.
Also, both the generation of iPS cells and genetic mapping have important privacy implications for you and your family. The decision to participate should be carefully considered before you volunteer your cells.
Your iPS cells will have the ability to grow indefinitely in the laboratory. In theory iPS cells can be grown to form more complex systems, or even human clones of the donor (think “Mini-Me” from “Austin Powers”). We will not make human clones with your cells. We operate under strict ethical limitations to prevent misuse of the cells, and comply with all relevant Australian law. All experiments must be approved by a human ethics research committee and your cells will only be used to research MND or associated diseases.
Genetic mapping can reveal information about mutations and natural variations that contribute to your likelihood of acquiring disease. The map can also reveal information about the likelihood of your children acquiring disease. To protect your information, all the data generated in the project will be de-identified so that your personal information is always kept private. Your cells and genetic map will remain anonymous.
A video describing the use of iPS cells in research from our collaborators at the Centre for Eye Research Australia is available here (click here). If you or your family ever change your mind, you can always choose to withdraw from the Program (your cells and DNA will be destroyed).
I would like to volunteer my cells
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 [email protected] 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.
Please note – FightMND plays no role in the selection of patients for any Clinical Trials or Research Project. Please contact your doctor or neurologist to see if this Program is suitable for you.
FightMND is the largest independent financial supporter of MND research and clinical trials in Australia
We fund the best and brightest MND researchers across Australia to help develop promising new therapies in the lab to the roll into clinical trials for Australians living with MND.