As the new  year starts, 2024 marked significant strides in Parkinson’s disease research, therapies, and policy initiatives. Here’s a quick look at the year’s advancements and challenges. 

Game-Changing Therapies

Vyalev (brand name in the US) Produodopa (brand name in Europe, Canada and Japan): Approved by the FDA in October 2024 and Available in the UK and the EU since February 2024, this infusion-based therapy provides a continuous source of levodopa/carbidopa, enhancing “on” time and reducing symptom fluctuations.

Crexont: Approved by the FDA in August 2024, this reformulated, longer-acting levodopa/carbidopa pill provides extended symptom relief with fewer doses. Crexont is available through medical prescriptions in the US.

In 2024, anticipation grows for the results of the ASPro-PD Phase 3 trial on ambroxol, led by Professor Anthony Schapira at UCL. This trial examines whether the widely available cough medicine can slow Parkinson’s progression by enhancing the GCase enzyme, which helps break down toxic protein clumps linked to neurodegeneration. Earlier Phase 2 results showed promising increases in GCase levels and reductions in harmful protein buildup. The study aims to determine ambroxol’s effectiveness in altering the disease course, potentially paving the way for a groundbreaking and accessible Parkinson’s treatment.

HER-096: Based on the naturally occurring brain growth factor CDNF, this innovative drug shows potential to protect and restore dopamine-producing cells. The trial is taking place in Finland where 24 people with Parkinson’s will be given HER-096 or placebo twice a week for 4 weeks and monitored for any unwanted side effects.

Mitochondria-Targeted Treatments: Compounds like Coenzyme Q10, Creatine, Mitoquinone (MitoQ), and MTX325 are being studied for their ability to repair mitochondrial dysfunction and protect neurons.

Exercise and Brain Health: Prior research has shown that many forms of exercise are linked to improved symptoms of Parkinson’s disease. But there has been no evidence that hitting the gym could create changes at the brain level. Now, a small proof-of-concept study by Yale School of Medicine involving 10 patients showed that high-intensity aerobic exercise preserved dopamine-producing neurons, the brain cells that are most vulnerable to destruction in patients with the disease.

Scientific Breakthroughs

Early Detection: A blood test has emerged as a tool for detecting Parkinson’s up to seven years before symptoms appear, promising earlier interventions and better management strategies.

Gene Therapies: Therapies are exploring methods to reprogram brain cells, enhancing their function and longevity. These techniques aim to slow disease progression and protect against neurodegeneration.

Advances in stem cell therapies for Parkinson’s disease aim to repair or replace damaged dopamine-producing cells. Companies like Bluerock Therapeutics and Aspen Neuroscience are making progress, with Bluerock’s treatment showing improvements in “on” times and reduced symptom variability in Phase 1 trials. Aspen Neuroscience is focused on personalized therapies, while Lund University in Sweden successfully implanted 7 million lab-grown brain cells into patient Thomas Matsson. Following this treatment, Matsson regained his sense of smell, reduced his medication, and returned to an active lifestyle, including sports like skating, skiing, and golf.

Growth Factors: Substances like glial cell-derived neurotrophic factor (GDNF) continue to show promise in supporting the survival and function of neurons, potentially delaying disease progression.

Mitochondrial Dysfunction: Targeting mitochondria — cellular “battery packs” that malfunction in PD — remains a focus. Therapies aim to repair these structures and protect brain cells from energy deficits.

Alpha-Synuclein Clumps: Misfolded alpha-synuclein proteins, implicated in PD, are being targeted in research to prevent their accumulation and reduce cell damage.

Adaptive Deep Brain Stimulation (DBS): Research is advancing adaptive DBS technology, enabling real-time symptom tracking and personalized stimulation adjustments for better outcomes. Medtronic introduced Asleep DBS, approved by the FDA, allowing surgery under general anesthesia. Percept RC DBS system captures real-time brain activity, enabling personalized therapy with a battery lifespan exceeding 15 years. $1 billion investments in brain-computer interfaces (BCIs) and graphene-based electrodes aim to further improve precision and minimize side effects.

Several biopharmaceutical companies, including Halia Therapeutics, NodThera, and Gain Therapeutics, are developing treatments targeting brain inflammation in Parkinson’s disease. NodThera and Halia focus on blocking NLRP3, a protein complex that drives harmful inflammation, while Gain Therapeutics is working on a therapy aimed at correcting the GBA1 gene, which increases Parkinson’s risk and may slow disease progression.

Treatment focus on the gut, with promising advances in probiotic therapies and mitochondrial-targeted drugs. Researchers have developed a probiotic that synthesizes L-DOPA in the gut, reducing the need for frequent doses and minimizing side effects, while animal trials show improvements in motor and cognitive functions. Additionally, drugs targeting gut bacteria like Enterococcus faecalis, which degrade levodopa, have shown success in ensuring more levodopa reaches the brain, enhancing its effectiveness and reducing side effects. These developments signal a shift towards more personalized, gut-focused therapies.

Changes in Parkinson’s Care

Food as Medicine: Natural compounds like turmeric, green tea, and probiotics are being explored for their potential to reduce brain inflammation and protect against Parkinson’s. Diets such as gluten- and dairy-free, Mediterranean, keto, and intermittent fasting have shown benefits for managing symptoms and supporting brain health.

Telemedicine and Smartwatches: Advances in telemedicine and wearable technology like smartwatches are enhancing patient care by enabling remote monitoring. This allows for better tracking of symptoms, empowering patients to make more informed decisions about their treatment.

Holistic Patient Approach: Patients are increasingly adopting a holistic care approach that combines medication, exercise, diet, and meditation. This shift toward a more informed and empowered patient is helping improve quality of life and overall care management.

Legislative Wins

The Parky Charter (UK): Advocated for five essential needs for Parkinson’s patients, pushing policymakers toward tangible support measures.

National Plan to End Parkinson’s Act (US): The first federal legislation aimed at addressing Parkinson’s comprehensively was enacted, marking a milestone in government commitment.

The Not-So-Good News

Exenatide’s Phase 3 Trial: Hopes for this diabetes drug were tempered when it failed to meet its motor symptom improvement targets.

Annovis Bio’s Buntanetap: While showing promise for cognitive improvement, the drug fell short in addressing motor symptoms.

Limited Focus on Disease Modification: Of 14 ongoing Phase 3 trials, only two target modifying the disease’s progression, highlighting a critical research gap.

Gratitude time!

A huge shoutout to all the amazing individuals and organizations that made 2024 a phenomenal year for the Parkinson’s community! From the trailblazing efforts of Cure Parkinson’s, Parkinson’s UK, the Michael J. Fox Foundation, The Parkinson’s Foundation and NINDS (and many others) to the relentless passion of researchers, activists, and, most importantly, people living with Parkinson’s and their families — this year wouldn’t have been the same without your dedication. You’ve made waves, sparked hope, and shown the power of teamwork. Here’s to more breakthroughs and joy ahead! ?