In support of Brain Awareness Week 2024, the global initiative that began almost 30 years ago, TFS HealthScience supports efforts to promote public interest and support for brain science by exploring the complexity of the human brain. To spark interest in the discoveries that can potentially improve the quality of life of millions living with brain-related disorders, let’s look at recent innovative, patient-centric discoveries in neuroscience, including halting disease progression at the genetic level, repurposing a vaccine for treating brain cancer, new treatment for Alzheimer’s Disease (AD), 24-hour subcutaneous infusion for Parkinson’s Disease (PD), and digital therapeutics (DTx) for migraine management.

1. Halting the Progression of Alzheimer’s Disease

According to the Centers for Disease Control and Prevention (CDC), AD is the most common type of dementia and affected as many as 5.8 million Americans in 2020. A recent study published findings on potentially halting the progression of AD. AD is defined by the appearance of pathological lesions, amyloid plaques (extracellular aggregates of Aβ peptides), and neurofibrillary tangles (NFTs); therefore, understanding the underlying molecular mechanisms will help find genes and pathways during the early stages of the disease.

The researchers identified a gene that may lead to the degeneration of neutrons most vulnerable to AD. Working with computational genomic experts from Rice University, researchers from Boston University (BU) School of Medicine and Karolinska Institute used machine learning (ML) to identify the gene DEK. According to the researchers, targeting DEK (or proteins that collaborate with DEK) would prevent memory loss and inhibit the spread of the disease.

2. Repurposing Zika Virus Live-Attenuated Vaccine for Brain Cancer

Glioblastoma multiforme (GBM) is the most common malignant brain cancer and one of the most aggressive malignancies, accounting for 14.5% of all central nervous system (CNS) tumors and 48.6% of malignant CNS tumors. Survival rates are poor (only 15 months). A study by Victorio et al. on repurposing the Zika virus live-attenuated vaccine (ZIKV-LAV) found a new approach to destroying human GBM cells in vitro and inhibiting tumor growth while avoiding healthy cells. Scientists from Duke-NUS Medical School studied how oncolytic ZIKV infects and induces death in tumor cells and discovered that Zika virus vaccine candidates they developed target rapidly growing cells over mature cells.

The team found that ZIKV-LAV strains caused death in 65 – 90% of GBM tumor cells. While still in early development, their findings suggest a new treatment alternative for brain cancer patients who currently have a poor prognosis. Scientists are improving the Zika virus strains to increase their effectiveness in killing brain cancer cells and other types of cancer, making them safer for patients, and modifying the virus to be imaged non-invasively after being administered in patients.

3. New Treatment for Early AD

In 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to lecanemab, a new treatment that moderately slows cognitive and functional decline in early AD. Of note, this is the first time the FDA has granted full approval to a drug for AD in two decades. However, there is a “black box” warning due to safety concerns. A study in the New England Journal of Medicine (NEJM) published the results of a Phase III clinical trial of lecanemab, which selectively targets the amyloid protein thought to be most toxic to brain cells. The drug is delivered intravenously every two weeks. While the drug’s price was set at $26,500 per year, the Centers for Medicare & Medicaid Services (CMS) announced broader Medicare coverage for people living with AD in July 2023.

4. Parkinson’s Disease Subcutaneous Infusion

Parkinson’s Disease (PD) is a chronic, progressive neurodegenerative condition that affects movement, mental health, and sleep and causes pain and other health issues. Affecting approximately 6.1 million people across the globe, disability and death due to PD are rapidly increasing worldwide. In January 2024, AbbVie announced the launch of its treatment for advanced PD, PRODUODOPA, in the European Union (EU). In February, the U.K. NHS started rolling out PRODUODOPA in England. The therapy is indicated for patients with PD with severe motor fluctuations and hyperkinesia (excessive movement) or dyskinesia (involuntary movement) when available PD treatment has not given satisfactory results.

According to AbbVie, it is the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of severe motor fluctuations in people living with advanced PD whose symptoms are inadequately controlled by other treatment options. The easy-to-use infusion is delivered through a cannula under the skin and controlled by a small automatic pump to help manage PD symptoms 24 hours a day. Three studies supported the EU launch, the most recent being a Phase III, 12-month open-label study, which evaluated the long-term safety, tolerability, and efficacy of continuous subcutaneous infusion of PRODUODOPA with results published in 2023.

5. Digital Therapeutics (DTx) & Migraine

Migraine is a chronic disease that affects over one billion people worldwide. A common and potentially disabling disorder, migraines can cause severe throbbing head pain, nausea, vomiting, and extreme sensitivity to light and sound. There are acute treatments to stop symptoms of a migraine attack and long-term preventive therapies to decrease the frequency and severity of migraines.

DTx is an emerging approach for remote migraine management. Clinical research in this area is growing, and the number of publications on “DTx in migraine management” has revealed an initial upward trend. A recent review of the latest research on DTx and migraines found its application in many aspects of migraine management, including digitizing migraine management tools, patient education and wearable devices, and non-pharmaceutical migraine prophylaxis. DTx ideally enables a link between practitioners and patients in their day-to-day care.

Recent studies examined the usefulness of digital diaries and digital self-management tools in diagnosing and tracking migraine attacks, the efficacy and safety of digital cognitive behavioral therapy (CBT), and Internet/smartphone app-based surveys. The authors found that most studies support the feasibility or effectiveness of DTx, however, they suggest that further large-sample studies be conducted to confirm the effectiveness of DTx in migraine management.

TFS HealthScience: Your Neuroscience CRO Partner

TFS HealthScience (TFS) is a premier and innovative global mid-size contract research organization (CRO) that partners with biotech and pharmaceutical companies throughout their clinical development. TFS Neuroscience CRO has broad experience across a range of neuroscience indications in neurology (including AD, amyotrophic lateral sclerosis (ALS), multiple sclerosis) and non-neurology, psychiatry, and pain (including addiction, schizophrenia, neuropathy). TFS Neuroscience has conducted nearly 150 studies on neurology, psychiatry, and pain, with over 50 ongoing projects. In the last five years, there have been more than 12,000 patients and more than 1,000 specialized sites.

Visit our Neuroscience CRO website to learn more about TFS’s expertise for your next neuroscience clinical trial, or connect with a TFS representative today!