May 11, 2017- Toronto, ON- Three important steps are required when taking an idea from the research laboratory into a viable treatment. The first step is the discovery phase (also known as basic science) where researchers aim to identify new targets, such as a protein, that may be contributing to a disease. Promising results from the discovery phase are then explored further in the translational phase which requires developing a compound that can alter the behaviour of that protein and doing a series of tests that determine if the compound could act as a drug that controls the disease. This is an iterative phase that involves a lot of trial and error to assess if the drug will be safe and produce a beneficial effect in animal models of the disease. Finally, the last stage is a clinical trial, where the drug is tested in people to determine if it will truly be safe and effective. Over the last few decades, funding for translational research has declined, resulting in a critical funding gap that has caused the translation of new research discoveries into treatments to fall behind. Organizations like the MS Society of Canada and the National MS Society (U.S.) are addressing this gap by designing new programs aimed at advancing the discovery of treatments for MS. In fact, the MS Society has formed a collaboration with a non-profit subsidiary of the National Multiple Sclerosis Society called Fast Forward, and together they will co-fund a new research project that will target the neurodegenerative process that is thought to contribute to progression in MS.
The project will be led by Canadian MS researcher Dr. Fang Liu from the Centre for Addiction and Mental Health (CAMH) in Toronto, Ontario. Dr. Liu will receive over $830,000 USD from both Fast Forward and the MS Society of Canada for her top-ranked proposal based on its innovative approach and high potential for impact on the treatment of progression in MS.
About Dr. Fang Liu’s Study:
Dr. Liu’s project builds on preliminary findings from her MS Society-funded project launched in 2012. The operating grant she received from the Society enabled her research team to demonstrate the interaction between specific proteins in the brain that can lead to the destruction of nerve cells that are required for proper functioning. This form of nerve cell death, called neurodegeneration, is a hallmark of progressive MS and is an important contributor to the accumulation of neurological disability. Dr. Liu found that, by interrupting the activity of these proteins, there was a noticeable reduction in symptoms in mice with an MS-like disease. These findings were published in 2015 in Annals of Clinical and Translation Neurology.
Following this work, Dr. Liu has developed two series of drug-like compounds that could be used to halt the neurodegenerative process in MS. Her new project co-funded by Fast Forward and the MS Society of Canada involves an extensive list of translational research experiments, which plan to:
- Improve the properties of the compounds to ensure that they are potent and stable
- Determine if the compounds will effectively modify the interactions between the proteins that are causing toxicity in the central nervous system
- Determine if the compounds have drug-like properties and study them further so that they are safe and produce an effect via oral administration
- Test if the compounds are safe and potent in animals
- Determine if the compounds are effective and can improve neurological function in animals with an MS-like disease
Dr. Liu reiterates that there is a need for the development of novel therapeutics as only 55% of individuals with relapse-remitting MS have effective disease-modifying therapies. Furthermore, drugs for progressive MS are scarce with only one approved therapy for this form of the disease in the U.S. There is an important unmet need for treatments that disrupt the processes that contribute to progression in MS, specifically neurodegeneration. The MS Society has taken an important step forward by supporting Dr. Liu’s proposal to develop drugs that will target neurodegeneration in MS with the ultimate goal that these novel targets will promote neuroprotection.
Comment:
Dr. Liu’s research represents a significant step forward towards an innovative treatment for progressive forms of MS. She has identified a promising target that, with the appropriate treatment, may slow the neurodegenerative process in MS.
The steps necessary to translate scientific discoveries into therapies involve a great deal of risk, resources and expertise. By collaborating with Fast Forward and leveraging their expertise in drug development, the MS Society will enable Dr. Liu to advance her research through a critical stage of preclinical development towards a transformative treatment option.