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Parkinson’s drug shown to slow ALS progression in clinical trial
An early clinical trial has shown that the Parkinson’s drug ropinirole is safe to use in patients with the fatal motor neurone disease amyotrophic lateral sclerosis, and delayed disease progression by an average of 27.9 weeks.
ALS causes people to gradually lose control of their muscles and while the disease can strike at any time of life, symptoms most commonly develop between the ages of 55 and 75. Men are more likely to develop ALS than women.
There is currently no cure for ALS – which is also known as Lou Gehrig’s disease – with available treatments focussing on reducing symptoms and providing supportive care.
Researchers at Keio University School of Medicine in Japan have reported their findings in the journal Cell Stem Cell.
Senior author and physiologist Hideyuki Okano of the Keio University School of Medicine in Tokyo, said: “ALS is totally incurable, and it’s a very difficult disease to treat.
“We previously identified ropinirole as a potential anti-ALS drug in vitro by iPSC (Induced pluripotent stem cells) drug discovery, and with this trial, we have shown that it is safe to use in ALS patients and that it potentially has some therapeutic effect, but to confirm its effectiveness we need more studies, and we are now planning a phase 3 trial for the near future.”
To test ropinirole’s safety and effectiveness in sufferers with sporadic (non-familial) ALS, the team recruited 20 patients receiving care at Keio University Hospital. None of the patients carried genes predisposing them to the disease, and, on average, they had been living with ALS for 20 months.
The trial was double blinded for the first 24 weeks, meaning that the patients and doctors did not know who was receiving ropinirole and who was being given a placebo.
Then, for the following 24 weeks, all patients who wished to continue were knowingly administered ropinirole.
Many patients dropped out along the way – partially due to the Covid-19 pandemic. This meant only seven out of 13 ropinirole-treated and one out of seven placebo-followed-by-ropinirole-treated patients were monitored for the full year.
However, no patients dropped out due to safety reasons.
To determine whether the drug was effective at slowing the progression of ALS, the team monitored a variety of different measures throughout the trial and for four weeks after treatment finished. 
These included changes in the patients’ self-reported physical activity and ability to eat and drink independently, and data from wearable devices and physician-measured changes in mobility, muscle strength, and lung function.
First author Satoru Morimoto, a neurologist at the Keio University School of Medicine, said: “We found that ropinirole is safe and tolerable for ALS patients and shows therapeutic promise at helping them sustain daily activity and muscle strength.”
Patients who received ropinirole during both phases of the trial were more physically active than patients in the placebo group. They also showed slower rates of decline in mobility, muscle strength, and lung function, and they were more likely to survive.
The benefits of ropinirole relative to the placebo became increasingly pronounced as the trial progressed.
However, placebo group patients who began taking ropinirole halfway through the trial did not experience these improvements. The researchers said it suggests that ropinirole treatment may only be useful if it’s started earlier and administered over a longer duration.
Dr Morimoto said: “We found a very striking correlation between a patient’s clinical response and the response of their motor neurons in vitro. Patients whose motor neurons responded robustly to ropinirole in vitro had a much slower clinical disease progression with ropinirole treatment, while suboptimal responders showed much more rapid disease progression despite taking ropinirole.”
It’s unclear why some patients are more responsive to ropinirole than others, but the researchers think this is probably due to genetic differences it’s hoped will be pinpointed in future studies.
The researchers findings have met with a mixed response from neurological experts and others working in the field of motor neurone disease.
Michael Swash, professor of Neurology at Barts and the London School of Medicine in the UK, said the Keio report was of interest. “There has long been an effort to find an ‘off the shelf medication’ applicable to ALS management. Ropinirole may be one such drug.
“But there needs to be a clearer understanding of its mechanism of action in order to apply such knowledge more widely. In addition, a larger study is required to understand who might benefit and what might be the limits of practical therapy in using ropinirole in ALS.
“More data on possible unwanted effects are also required. There are interesting parallels in proteinaceous accumulation in the neuronal cytosol between PD and ALS that should be explored.”
Dr Brian Dickie, director of research at the MND (Motor Neurone Disease) Association, added: “Whilst these results may be of some interest to the research community, the clinical trial is far too small and the findings too preliminary to draw any valid conclusions.”
BioAge Labs is expanding its lead drug candidate into diabetic macular oedema, with plans to start a phase 1b/2a trial in mid-2026.
The clinical-stage biotechnology company will test BGE-102, an oral therapy, in patients with the condition, which is one of the most common causes of vision impairment among people with diabetes.
Diabetic macular oedema occurs when persistently high blood sugar damages the small blood vessels in the retina, the light-sensitive tissue at the back of the eye, leading to fluid leakage and distorted vision.
While the condition is linked to diabetes, its progression is tied to chronic inflammation.
Current treatments focus on managing damage after it has begun. Patients often receive regular injections directly into the eye, sometimes monthly, to control swelling and preserve sight.
These therapies can be effective, but they are invasive, time-intensive and difficult to sustain over years.
Kristen Fortney is chief executive and co-founder of BioAge.
She said: “NLRP3 sits at the apex of this cascade, and BGE-102 offers the potential to deliver broader anti-inflammatory benefit in an oral formulation, which could meaningfully reduce treatment burden for patients with serious, sight-threatening conditions who currently require frequent intravitreal injections.”
BGE-102 is an oral NLRP3 inhibitor, designed to dampen inflammation at its source.
NLRP3 is a protein that drives inflammatory signalling and becomes increasingly active with age and metabolic stress.
When overactivated, it triggers signals that damage tissues throughout the body, including the retina.
What BioAge says makes BGE-102 notable in ophthalmology is its potential to reach the retina via oral dosing, a barrier many drugs struggle to cross.
If successful, this could reduce the treatment burden for patients who currently rely on frequent eye injections.
In early laboratory studies designed to mimic diabetic eye disease, BGE-102 helped keep the retina’s tiny blood vessels intact.
In studies examining ageing in the retina more broadly, blocking NLRP3 reduced the build-up of lipofuscin, a toxic waste material that accumulates in eye cells over time and is linked to degenerative vision loss, by roughly 80 per cent.
In an ongoing phase 1 trial, the drug has been well tolerated and reduced inflammatory signals in the body, including markers linked to cardiovascular and metabolic ageing.
The phase 1b/2a trial will test BGE-102 on its own and alongside existing treatments, aiming to show whether the drug calms the inflammation that damages vision over time.
Researchers will track changes in IL-6, a key inflammatory signal, within the eye, alongside measures of vision and retinal swelling. Results are expected in mid-2027.
The eye study will run alongside BioAge’s ongoing cardiovascular trial.
The company describes BGE-102 as a potential “pipeline in a pill”, targeting NLRP3-driven inflammation across cardiovascular, central nervous system and ocular diseases.
The USC Clinical Trial Recruitment Lab will fund four projects testing how AI can strengthen recruitment for Alzheimer’s trials.
The initiative, dedicated to accelerating and improving Alzheimer’s clinical trials, selected the projects from more than 30 applicants to explore digital approaches.
Alzheimer’s clinical trials are more complex, costlier and take longer than those in other therapeutic areas, despite the pressing need for new treatments.
The lab evaluates innovative recruitment strategies to improve access and representation in trials, with the goal of identifying scalable evidence-based recruitment practices.
The USC Clinical Trial Recruitment Lab is a collaboration between the USC Schaeffer Center for Health Policy and Economics and the USC Epstein Family Alzheimer’s Therapeutic Research Institute.
The four projects will explore the following strategies.
- Miriam Ashford at University of California, San Francisco will develop and test a generative AI voice agent to support remote informed consent and assess patient capacity for Alzheimer’s clinical trials.
- Erika Cottrell at OCHIN, a national network of community health centres, and Vijaya Kolachalama at Cognimark will integrate an AI-enabled diagnostic platform into primary care electronic health record workflows to support earlier identification and referral of patients.
- Andrew Kiselica at University of Georgia will establish a digitally enabled, trial-ready cohort of rural older adults to improve recruitment, participant selection and engagement.
- Raeanne Moore at University of California, San Diego will leverage electronic health record portals and digital cognitive assessments to accelerate prescreening and better match potential participants.
An estimated 5.6 million Americans are living with Alzheimer’s and related dementias, a number expected to increase dramatically in the coming decades as the population ages.
An extensive therapeutic development pipeline and new early-detection approaches, such as diagnostic blood tests and advanced digital tools, have the potential to reduce the burden of the disease.
However, fewer than one per cent of eligible individuals participate in Alzheimer’s therapeutic trials due to barriers that include limited patient awareness, health system resource constraints and lack of access to diagnostics, according to research from USC Schaeffer.
Certain populations at higher risk for the disease, including Black and Hispanic patients, remain underrepresented.
“We can only innovate as quickly as we can test new therapies,” said Dana Goldman, founding director of the USC Schaeffer Institute.
“That’s why it’s crucial we keep expanding the toolkit of evidence-based recruitment strategies for running faster, better trials.”
The lab previously funded six pilots, some of which have already yielded insights.
For example, one found remote blood collection could help identify potential participants, while another showed that offering a small gift card significantly increased enrolment in an online memory concerns registry.
“Faster and more effective recruitment is essential, and we’re excited to incorporate these solutions in an integrated way as part of our clinical trials,” said Paul Aisen, founding director of the USC Epstein Family Alzheimer’s Therapeutic Research Institute.
“As studies move earlier into pre-symptomatic disease, this opens the door to new recruitment paradigms, and continuing to push forward the science of recruitment will be critical to what comes next in Alzheimer’s research.”
Insilico Medicine has agreed a US$66m deal with a Chinese biotech for half of the rights to a brain-penetrant Parkinson’s drug.
Under the agreement, the artificial intelligence-powered drug developer will take the lead on bringing the preclinical asset, dubbed ISM8969, into a phase 1 trial.
Hygtia Therapeutics will then take over for further studies and onward toward regulators and commercialisation.
Ren Feng is co-chief executive and chief scientific officer at Insilico.
Feng said: “Targeting neuroinflammation via NLRP3 represents a scientifically sound and high-potential approach to treating neurodegenerative and age-related diseases.
“However, developing a safe molecule with good blood-brain barrier penetration remains a formidable obstacle for drug developers.
“Through our generative AI platform, we have designed a molecule specifically engineered to overcome this barrier.
“We are pleased to partner with Hygtia Therapeutics.
“We believe that through our combined efforts, we can accelerate its clinical progress to address significant unmet medical needs.”
ISM8969 is an NLRP3 inhibitor.
NLRP3 is a protein involved in inflammation, and blocking it is being explored as a way to address a range of neuroinflammatory and cardiometabolic diseases. Interest in NLRP3 inhibition has increased in the past year.
Earlier this month, Eli Lilly paid US$1.2bn to acquire Ventyx Biosciences in the wake of a phase 2 study tying Ventyx’s lead NLRP3 inhibitor to improvements in Parkinson’s symptoms.
That same NLRP3 inhibitor had also been shown to cut levels of a biomarker for stroke and other serious risks by almost 80 per cent within a week in a separate mid-stage study.
Preclinical data for ISM8969 have “demonstrated the molecule’s robust efficacy, favourable safety profile and marked anti-inflammatory activity in various disease models,” said Insilico, which also noted the therapy’s “desirable blood-brain barrier penetration.”
The blood-brain barrier is the protective barrier around the brain that many drugs struggle to cross.
Insilico, which went public on the Hong Kong Stock Exchange last month, said it had discovered ISM8969 via its generative chemistry engine Chemistry42.
As well as an upfront fee of US$10m, Insilico is in line for up to US$56m in milestone payments from Hygtia.
Insilico has attracted interest from pharmaceutical companies for its AI-enabled drug discovery technology, including Sanofi, Pfizer, Menarini Group and Boehringer Ingelheim.
Most recently, Lilly agreed a deal worth more than US$100m in November.
Hygtia, which was founded last August after being incubated by Fosun Pharma, said the agreement with Insilico “marks a pivotal step in our global strategy.
This partnership aligns with our strategy to expand our innovative neuroscience pipeline through high-quality assets.”
BioAge expands drug into diabetic macular oedema
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