Emerging Medications & Therapies for Cerebellar Ataxia

Ataxia is characterised by movement patterns that are unsteady, imbalanced, uncoordinated, action tremors, difficulty speaking and swallowing, disorientation/dizziness, involuntary movements, and double vision. This condition is usually brought on by cerebellar dysfunction due to genetic, acquired, or degenerative causes. The illness process may also include neuronal circuits outside the cerebellum, such as intrinsic brainstem nuclei, spinal long tracts, and supratentorial pathways, even if these symptoms may originate primarily from the cerebellum.

Even though an ataxic patient's multifaceted presentation may seem overly complicated, using the many therapy approaches available for the specific symptom areas can always be helpful. To stop or delay the progression of the disease, nevertheless, for any type of ataxia, it is necessary to identify the precise underlying processes and develop disease-modifying medications that target them. For hereditary or degenerative cerebellar illnesses, there are currently no disease-modifying medications available. The acquired ataxias are still those that can be treated. Here are some of the emerging therapies and medications listed for cerebellar ataxia:

Symptomatic Medications

Several symptomatic medications, all aimed at enhancing Purkinje cell function, are being developed for cerebellar ataxia. Riluzole, a glutamate transporter promoter and calcium-activated potassium channel opener, attenuated impairments in a rat model of ataxia by lowering Purkinje neuron overexcitability. Symptomatic medications are designed to reduce various symptoms of ataxia in patients.

Supplementary Medicines

Vitamins and dietary supplements are frequently advised when nutrition or weight loss is a problem (with regard to caloric intake or micronutrient deficiencies caused by difficulty swallowing or food intolerances). Under the supervision and for a fair amount of time, patients can use naturopathic, homoeopathic, herbal, and other popular alternative therapy, but it's crucial to learn about any potential adverse effects before letting them try these methods.

Neuroprotective Approaches

It is currently unclear how the various neurodegenerative disorders cause nerve cell death by a chain of interrelated molecular events, and the crucial factors driving this process have not been pinpointed in a way that may lead to viable targets for therapeutic development. In the ageing process, genetic, and environmental variables that affect neuronal homeostasis (such as neuroinflammation) trigger the pro-degenerative pathways that eventually lead to neuronal death.

Neurostimulation

A consensus screen determines that cerebro-cerebellar pathways can impact cerebellar motor movements, visual tracking, motor control and adaptation, as well as emotional and cognitive processes, effectively with both transcranial magnetic stimulation and transcranial direct current stimulation. They also concur that greater controlled studies are required to confirm the safety and long-term effectiveness of these non-invasive technologies before they may be used to treat a wide range of symptoms.

Anti-Inflammatory

By impairing the structure and function of neurons, inflammatory mediators generated by the brain's innate immune cells are key players in the development of neurodegenerative disorders. As a potential moderator of neuroinflammation and caspase activity in neurodegenerative disorders, minocycline has been investigated in preclinical and clinical trials.

Genetic Modalities

It is being attempted to use epigenetic methods to "make the aberrant gene act like a normal gene.  Protein replacement therapy or other methods to influence the metabolic pathway engaged are examples of disease-modifying therapies for hereditary diseases where there is a drop in protein levels (often common genetic disorders).

Anti-Apoptotic Approaches

The extensive examination of neuroprotective compounds that have been modelled after ALS research has only resulted in the FDA approval of two ALS medications, the first of which, riluzole, has a negligible impact on longevity. By preventing glutamatergic transmission and reducing glutamate concentration, riluzole guards against excitotoxic motor neuron degeneration. According to European studies, it might have a comparable impact on spinocerebellar ataxia degeneration.

Rehabilitative Approaches

Physical therapy, occupational therapy, speech therapy, orthotic devices, assistive equipment assessment, safety training evaluations, and driving assessments are just a few examples of therapies that can enhance patient and family quality of life and safety, decrease falls, and monitor patient individuality.

Stem Cell Therapy

It has been proposed that nerve growth factor augmentation or neural replacement treatment are two possible methods for how stem cell therapies for cerebellar ataxia and other neurological diseases could work. Six individuals with spinocerebellar ataxia type 3 participated in a phase 1/2a open-label study using intravenous adipose-derived mesenchymal stem cells.

Viewing the Future

It is improbable that one "miracle cure" will be able to regulate all the aforementioned processes and be recognised as "the solution" for ataxia. It is anticipated that a "cocktail" of medications, some particular to the disease and some particular to the ataxia, would eventually transform the neurodegenerative cerebellar disorders into curable diseases. However, it's crucial to keep in mind there's always something you can do while interacting with ataxic patients and their families, even if it's just providing information, listening, and having a discussion.