MJFF is tracking 15 alpha-synuclein – targeted therapies in various stages of clinical testing— seven of which we are funding— including some in later Phase III testing( typically, the last stage of clinical testing before regulators like the FDA decide whether to make the drug available for public use). This includes prasinezumab, which has the potential to be the first drug approved to slow or stop Parkinson’ s( see sidebar, page 13). It is important to note that while the progress is hopeful, Phase III trials take years and are not guaranteed to be successful.
Other drugs in clinical trials target biology associated with some of the more common gene changes linked to PD development, particularly in LRRK2 and GBA1 genes. Research, including trials funded by MJFF, into how the biology behind these genetic differences may lead to Parkinson’ s disease has fueled a robust pipeline of potentially promising treatments. This pipeline is being further expanded by a new MJFF-led program— the LRRK2
A world without Parkinson’ s disease means developing and delivering critical treatment options.
Symptom-directed therapies are treatments, including the gold-standard levodopa, that reduce the impact of the disease, as well as medicines that manage sleep problems and wearable devices that improve walking and balance.
Disease-modifying therapies are treatments that slow, delay or prevent the progression of disease biology— such as drugs that prevent alphasynuclein from clumping in the brain and damaging dopamine neurons.
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