In mid-July 2021, biotechnology company Alterity Therapeutics (“Alterity”) shared the publication of new data regarding ATH434 for patients with multiple system atrophy (MSA). The data comes from a preclinical study evaluating the treatment in mice models. During the study, performed at the Medical University of Innsbruck, researchers determined that ATH434 treatment lowered neurodegeneration caused by alpha-synuclein (a-synuclein), thereby offering protective benefits. Interested in seeing the full results? Take a look at the data published in Movement Disorders.
So what exactly is ATH434? According to Alterity, ATH434 is a small-molecule treatment meant to inhibit proteins associated with neurodegeneration, such as a-synuclein, from accumulating in the brain. Through restoring iron balance in the brain, ATH434 lowered a-synuclein buildup in mice models of MSA. Alterity continues that ATH434:
has excellent potential to treat various forms of atypical Parkinsonism [in which] patients experience parkinsonism (slowness of movement, stiffness and tremor) as well as other debilitating symptoms. Atypical forms of Parkinsonism [such as MSA, Progressive Supranuclear Palsy, and dementia with Lewy bodies] have additional, associated symptoms and often respond poorly to drugs for treating the motor symptoms of Parkinson’s disease.
It is important to note here that MSA and Parkinson’s disease are not the same. Although symptoms are similar, and patients with MSA may experience Parkinsonism symptoms, the conditions are two separate entities. In the case of MSA, there are currently no available approved treatments. Thus, ATH434 has the opportunity to fulfill a huge unmet need. So far, ATH434 received Orphan Drug designation in both the EU and US.
The newly published data centers around a preclinical study evaluating ATH434 and its neuroprotective abilities. In addition to reducing a-synuclein aggregations and brain iron, ATH434 also improved motor function. Additionally, the treatment preserved and protective striatum neurons. Normally, the striatum is impacted in human patients with MSA. Thus, this treatment already shows specific, targeted promise. Moving forward, Alterity hopes to begin a Phase 2 clinical trial by the end of 2021.
Multiple System Atrophy (MSA)
Multiple system atrophy (MSA) is a rare and progressive neurodegenerative disorder affecting the autonomic nervous system. While doctors are unsure of the exact cause of MSA, they do know that a-synuclein accumulates in glia; this differs from Parkinson’s disease, in which a-synuclein accumulates in nerve cells. Additionally, patients with MSA may experience shrinking or degenerating areas of the brain. MSA is divided into two categories: Parkinsonian or cerebellar. Symptoms often manifest when patients are in their 50s and progress quickly over the next 5-10 years. These include:
- Lightheadedness and/or dizziness
- Heartbeat irregularities
- Sleep disruptions
- Sexual dysfunction
- Lack of balance
- Muscle rigidity
- Slowed movement
- Postural orthostatic hypotension
- Difficulty bending arms or legs, moving, swallowing, or balancing
- Slowed or slurred speech
- Visual impairment