They say the mind is our strongest muscle, and while it’s not exactly a muscle, delvin...
They say the mind is our strongest muscle, and while it’s not exactly a muscle, delving into its complexities alongside the nervous system reveals profound insights into human cognition, behavior and movement.
Neurology, the study of this intricate field, untangles the differences in how we think, feel and move. For those navigating neurological disorders, there are often roadblocks that disrupt the smooth functioning we all strive for, meaning they’re presented with significant challenges.
Fortunately, researchers are forging ahead with new tools and treatments aimed at not just managing symptoms but reshaping the landscape of care. These advancements offer hope to millions by potentially slowing or even preventing the progression of these debilitating conditions.
In this article, we’ll explore five groundbreaking advancements in neurology, spotlighting pivotal developments in the treatment of Alzheimer’s, Parkinson’s, Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), and epilepsy.
From Xenon Pharmaceuticals’ promising XEN1101 for epilepsy and Teva’s Austedo combating HD chorea and tardive dyskinesia, to Vigil Neuroscience’s innovative VG-3927 for Alzheimer’s and Prilenia’s pridopidine addressing both HD and ALS, we’ll delve into the latest clinical trials reshaping the sector. Plus, we look at how researchers at the University of Rochester Medical Centre are leveraging Apple Watches to detect early signs of Parkinson’s disease.
So, settle in and buckle up as we dive into five recent breakthroughs in neurology, where scientific progress meets human resilience in the search for better treatments and brighter futures…
Xenon Pharmaceuticals, a Vancouver-based biotech specializing in therapies targeting neurological disorders, has announced a significant breakthrough in epilepsy treatment. The news comes after late last year, results from their Phase II trial revealed that their novel potassium channel opener, XEN1101, can safely reduce seizure frequency in adults with focal epilepsy by over 50%.
Published in the prestigious JAMA Neurology journal, the mid-stage study involved 325 patients suffering from focal-onset seizures, the most common type of epileptic seizures that occur when specific brain regions send out sudden, excessive bursts of electrical signals.
Prior to the study, all participants had previously tried and stopped an average of six different drugs that failed to control their seizures and were experiencing at least four seizures per month despite ongoing treatment. Remarkably, the trial showed that just one daily dose of XEN1101 significantly reduced seizure burden.
Throughout the trial, various doses of XEN1101 were tested. At 25mg, seizure frequency dropped by an average of 53% after eight weeks of treatment. The 20mg dose reduced seizures by a median of 46%, while even the lowest dose of 10mg resulted in a significant 33% decrease in seizure frequency compared to the placebo, which only achieved an 18% reduction.
These findings are particularly pivotal considering that around 50 million people around the globe suffer from epilepsy, marking it as one of the most common neurological conditions. Current epileptic therapies fail to stop seizures in around a third of patients, making the results from XEN1101 a potential game-changer.
In fact, encouraged by the positive outcomes, many participants opted to extend their trial beyond the initial eight weeks. Impressively, 18% of the group remained seizure-free after six months of treatment, and 11% stayed seizure-free for over a year.
The next phase of development, already in progress, will advance XEN1101 through Phase III trials. These trials will not only continue to focus on focal-onset seizures but will also evaluate the drug’s effectiveness on tonic-clonic seizures too.
It’s thought that the novel drug could help prevent seizures from happening altogether by enhancing the opening of the potassium channels associated with them. Given the promising results thus far, Xenon could be on the cusp of a critical new therapy for epilepsy, offering hope to millions worldwide who continue to struggle with this challenging condition.
Teva Pharmaceuticals has achieved a major milestone with the FDA’s approval of Austedo, their flagship drug, now available as a convenient, once-daily pill for adults suffering from Huntington’s disease (HD) chorea and tardive dyskinesia (TD).
For context, Huntington’s disease is a fatal neurodegenerative disorder characterized by cognitive decline, psychological issues, and uncontrollable movements known as chorea, affecting 90% of patients. On the other hand, tardive dyskinesia impacts up to one in four individuals on mental health medications, leading to chronic movement disorders that affect physical, emotional and psychological wellbeing. Both conditions severely impact daily activities such as eating, speaking and walking.
Now back to Teva Pharmaceuticals… Originally launched in 2017, Austedo has been continuously refined by Teva to enhance patient lives by streamlining its regimen and improving dosing flexibility. The latest approval for Austedo, dubbed Austedo XR, maintains the drug’s proven efficacy while offering the added convenience of a once-daily pill available in 6-, 12-, and 24-mg doses.
For patients with TD, Austedo provides symptom improvements within two weeks, while those with HD chorea can expect a significant reduction in their total maximal chorea (TMC) score. This comes after real-world Phase IV data presented at the 2024 American Academy of Neurology Annual Meeting highlighted that nearly 80% of HD chorea patients achieved optimal dosing within four weeks using the four-week Titration Kit for Austedo.
The study showed that 76% of HD patients completed the titration kit with over 90% adherence and a 33% average reduction in TMC score from baseline. Both patients and providers reported 100% satisfaction with the kit’s ease of use and its effectiveness in ensuring adherence to the titration schedule. This data underscores Austedo’s tailored treatment approach, allowing patients to adjust their medication based on their unique chorea symptoms.
One of the most significant advantages of Austedo XR is its suitability for patients with concurrent conditions who take multiple medications. This can often complicate adherence to medication schedules, but Austedo XR alleviates this burden. In fact, it’s the only approved VMAT2 inhibitor treatment without use restrictions, making it an important option for patients managing other underlying conditions.
Additionally, with financial assistance programs, approximately 90% of insured patients will pay just $10 or less for their prescription, ensuring broad accessibility across different socioeconomic backgrounds, and ultimately enhancing the quality of life for thousands of HD chorea and TD patients.
Prilenia is gearing up to file for approval of its drug, pridopidine, for Huntington’s disease (HD) in the EU, while simultaneously advancing into Phase III trials for treating amyotrophic lateral sclerosis (ALS).
Pridopidine is an oral agonist of the Sigma-1 receptor protein, which is crucial in regulating key processes impaired in neurodegenerative diseases like HD and ALS. This protein, found in the brain and spinal cord, promotes nerve cell function and survival.
Despite not meeting the primary endpoint in Phase III trials for HD, Prilenia is moving forward with its approval filing. The trial aimed to slow functional decline in adults with HD over 65 weeks, measured using the Unified Huntington Disease Rating Scale-Total Functional Capacity score (TFC). While the drug didn’t achieve this, it did show benefits in a subgroup of patients who were not on other treatments for symptom management.
Prilenia believes that the drug’s effects were potentially reduced by neuroleptic medications and VMAT2 inhibitors, used to control chorea – the involuntary, jerky movements associated with HD – both of which affect the dopamine pathway.
It’s key to point out that current HD therapies only address symptoms, while pridopidine aims to be the first drug approved to slow the disease’s progression, offering hope to approximately 100,000 HD patients worldwide.
But that’s not all pridopidine has to offer. It’s also being developed to treat ALS, with Phase III trials set to begin later this year. For reference, ALS is a progressive neurodegenerative disease that leads to muscle weakness and paralysis, with few approved treatments to ease symptoms and no known cure.
Similar to the HD trial, pridopidine did not meet its primary endpoint in the Phase II ALS trial. However, Prilenia is pushing ahead with Phase III after finding encouraging results. While the Phase II trial did not show significant differences in primary or secondary endpoints compared to the placebo, further analysis revealed that patients with definite ALS and less than 18 months duration on pridopidine experienced slower disease progression.
Moreover, findings indicated a 57% improvement in survival benefit for patients with definite or probable ALS early in the disease course. Pridopidine was also well-tolerated with no serious adverse effects, maintaining a safety profile consistent with the placebo group and previous studies.
Overall, despite not hitting the primary endpoints in initial trials, pridopidine has shown significant potential for patients with both ALS and HD, diseases that currently have no known cure.
Vigil Neuroscience has received the FDA’s green light for a Phase I trial of VG-3927, marking a significant step forward in the fight against Alzheimer’s disease. The placebo-controlled, double-blind study has already dosed its first participant, aiming to explore the potential of VG-3927, the first and only small molecule TREM2 agonist, in treating Alzheimer’s.
Vigil Neuroscience, a clinical-stage biotech, focuses on developing innovative therapeutics for rare and common neurodegenerative diseases by restoring the sentinel immune cells of the brain. Their latest effort centres on VG-3927, which targets the triggering receptor expressed on myeloid cells 2 (TREM2). This receptor plays a crucial role in the brain’s immune response, making it a promising target for Alzheimer’s treatment.
Alzheimer’s disease is the most common cause of dementia, responsible for 60-80% of cases, and currently affects approximately 6.7 million Americans age 65 and older. Despite the availability of treatments like Eisai and Biogen’s Leqembi, which helps reduce cognitive and functional decline by removing the beta-amyloid, there is still no cure for Alzheimer’s. As such, researchers worldwide are striving to develop better treatments, delay onset and prevent the disease altogether.
VG-3927 aims to enhance the function of TREM2, a receptor that plays a key role in regulating the brain’s immune cells, known as microglia. By activating TREM2, VG-3927 is expected to promote the clearance of toxic proteins and debris from the brain, reduce inflammation and support neuronal health. This mechanism of action could potentially address multiple pathological features of Alzheimer’s, offering a more comprehensive approach to treatment.
The Phase I trial of VG-3927 will assess its pharmacodynamics, pharmacokinetics, tolerability, and safety in healthy volunteers. This initial study is crucial for understanding how the drug behaves in the body and how it affects the brain’s immune system. The results, expected to be published in the coming months, could pave the way for new therapies that offer improved safety and efficacy, addressing multiple aspects of Alzheimer’s pathophysiology.
If successful, VG-3927 could fulfil a critical unmet patient need, offering hope for improved therapeutic options for Alzheimer’s patients and opening up new avenues for treating other neurodegenerative disorders, reshaping our understanding and approach to these complex conditions.
Neurologists at the University of Rochester Medical Center have discovered that Apple Watches could play a vital role in detecting early symptoms of Parkinson’s disease.
Parkinson’s, the second most common neurodegenerative disease after Alzheimer’s, affects nearly 90,000 Americans each year and over 10 million people worldwide. Yet, despite its rapid growth as a neurological condition, Parkinson’s remains incurable.
The exact cause of Parkinson’s is still unknown, but researchers believe it results from a combination of ageing, genetics, and environmental factors leading to the death of dopamine-producing nerve cells. The disease’s hallmark symptoms – tremors, rigidity, and slowness of movement – emerge when the brain can no longer produce enough dopamine to control movement effectively.
In a groundbreaking study, the team at the University of Rochester Medical Center utilized Apple Watches and iPhones to monitor individuals with early-stage Parkinson’s over a year. These digital tools allowed the researchers to passively track symptoms such as gait disturbances and tremors while also capturing speech-related issues through voice recordings.
The data revealed that patients with early Parkinson’s experienced significant declines in gait, an increase in tremors, and modest changes in speech. These findings underscore the potential of widely accessible digital devices to remotely monitor and detect changes in multiple domains of the disease.
One of the most compelling aspects of this research is the accessibility of these digital measures. With the widespread availability of smartwatches and smartphones, this technology could revolutionize how we monitor Parkinson’s disease. By leveraging the data generated by these devices, it is possible to remotely track the progression of symptoms, which could significantly accelerate therapeutic development and bring treatment options to Parkinson’s patients much quicker.
But that’s not all… The implications of this study extend beyond just monitoring. The continuous data collection enabled by smartwatches and smartphones could lead to more personalized treatment plans, ensuring that patients receive the right interventions at the right time. This approach aligns with the broader trend towards personalized healthcare, where treatments are tailored to the individual needs of each patient.
Furthermore, the ability to monitor symptoms remotely could ease the burden on patients who currently need to visit clinics frequently for evaluations. This convenience can lead to better patient adherence to treatment plans and improved overall quality of life.
As the fastest-growing neurological condition in the world, Parkinson’s demands innovative approaches to detection and treatment. The University of Rochester’s study highlights a promising avenue, demonstrating that the technology we use every day could become a critical tool in the fight against this debilitating disease.
