STAT+: Drug metabolism AI competition results show that bigger may not always be better

In 2020, the CASP competition vaulted AlphaFold to prominence and a Nobel Prize. But the era of people being impressed by an artificial intelligence model correctly predicting the structure of a protein — once a challenge many experts didn’t think would be solved in their lifetime — is over. Now drug developers want AI that can solve their big problems, like discerning whether the body is going to attack a drug candidate and render it useless. 

One such example is the pregnane X receptor, or PXR. When activated, PXR increases the production of an enzyme that specifically breaks down foreign organic molecules — such as drug molecules — so the body can dispose of them. The specific enzyme that PXR regulates can metabolize approximately 50% of all marketed drugs. 

Most drug development campaigns only discover whether candidates trip this sensor late in the game, forcing drug developers to go back to the drawing board. But if an AI model could reliably predict whether a given drug candidate will activate the PXR receptor, it could fix a lot of problems that present hurdles for new potential drugs, including the drug exiting the body too fast or creating drug–drug interactions.

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GLP-1 Treated Patients with Diabetes Have Raised Risk for Eye Condition

A large study of people with type 2 diabetes suggests that those who started treatment with a glucagon‑like peptide (GLP)‑1 receptor agonist after diagnosis had slightly increased risk of developing a serious eye condition called ischemic optic neuropathy than people with diabetes treated with other medications.

As reported in the Annals of Internal Medicine, the risk for patients given GLP-1 drugs was about twice that of those given a sodium–glucose cotransporter (SGLT)‑2 inhibitor or a dipeptidyl peptidase (DPP)-4 inhibitor although the absolute risk was still low in all groups.

Ischemic optic neuropathy occurs when the optic nerve sustains damage caused by reduced or blocked blood flow, leading to loss of nerve tissue and vision. The main symptom is sudden, usually painless vision loss in one eye, often with missing areas of the visual field that are frequently permanent. It is a rare condition, with 4-10 cases per 100,000 people per year in the U.S., with some factors like age and conditions like type 2 diabetes increasing risk.

In this study, Chintan Dave, PhD, a researcher at Rutgers University, and colleagues included claims data from 161,489 adults aged 18 to 65 years with type 2 diabetes who were newly prescribed a GLP-1 receptor agonist, 122,114 who started a SGLT‑2 inhibitor, and 86,047 who started a DPP‑4 inhibitor.

They excluded anyone who had previously used these drugs or previously had ischemic optic neuropathy. They approximated randomization by balancing more than 80 characteristics across groups. They then collected follow up data for up to 18 months to see who was later diagnosed with ischemic optic neuropathy.

Over 18 months, about nine out of every 10,000 people in the GLP-1 group were diagnosed with ischemic optic neuropathy, compared with about six out of 10,000 in the GLT‑2 inhibitor group and about four out of 10,000 in the DPP‑4 inhibitor group. Essentially there were three to four extra cases of ischemic optic neuropathy per 10,000 patients in the GLP-1 compared with the other groups.

The researchers note that the apparent excess risk was concentrated in older adults, men, and people with more advanced diabetes or eye or cardiovascular conditions.

GLP-1 receptor agonists are now widely used, both in lower doses for treatment of type 2 diabetes and in higher doses to treat obesity.  “Despite the very low absolute risk for ischemic optic neuropathy, the rapidly expanding use of GLP-1 receptor agonists in patients with and without type 2 diabetes increases the clinical and public health importance of any potential association,” conclude the authors.

“Given that type 2 diabetes itself is a risk factor for nonarteritic anterior ischemic optic neuropathy [which constitutes approximately 75% of ischemic optic neuropathy cases] the potential for GLP-1 receptor agonists to further augment this risk has relevant implications for clinical decision making.”

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Targeting Microglia Could Help Extend the Recovery Window After Stroke

Stroke care has advanced most clearly in the acute phase: rapid reperfusion, prevention of edema, secondary prevention, and early rehabilitation. Yet for many patients, the most difficult clinical reality begins after stabilization. Neurological recovery often improves over weeks to months, then plateaus. Once that spontaneous recovery window closes, residual motor, language, or cognitive deficits may become permanent.

A study published in Nature now identifies a molecular mechanism that may help explain why this window narrows. Researchers led by Jun Tsuyama and Takashi Shichita, PhD, at the Institute of Science Tokyo found that microglia, the brain’s resident immune cells, can remain in the post-stroke brain after losing their reparative function. The team identified ZFP384 as a transcriptional regulator that suppresses the microglial repair program and showed that blocking it with an antisense oligonucleotide improved long-term recovery in mouse models of ischemic stroke.

A repair program that fades too soon

Microglia are often discussed in the context of neuroinflammation, but their role after stroke is not uniformly harmful. In the acute phase, activated myeloid cells contribute to inflammatory injury. During recovery, however, microglia can shift toward a reparative state, producing neurotrophic and tissue-supportive factors that contribute to remyelination, synaptic remodeling, and functional improvement.

One of the key markers in this study was insulin-like growth factor 1, or IGF1, a neurotrophic factor produced by reparative microglia. IGF1 has known roles in synaptogenesis, oligodendrocyte function, and myelin repair. The researchers used IGF1 expression to track microglia involved in the recovery phase after ischemic stroke.

“We aimed to identify the molecular mechanism responsible for diminishing microglial reparative functions,” Tsuyama said in the press release.

The central observation was clinically relevant: reparative microglia did not simply disappear. Instead, lineage-tracing experiments showed that cells which had once expressed repair-associated genes persisted in the peri-infarct region but later lost that gene-expression program. In mice, recovery-associated gene expression rose after stroke and then declined toward baseline by around day 28. The cells remained, but their reparative identity faded.

ZFP384 as a brake on microglial repair

To identify what shuts down this repair state, the researchers combined RNA sequencing, single-cell RNA sequencing, ATAC-seq, and transcription-factor analysis. A protein known as ZFP384 emerged as a candidate regulator whose expression increased as the microglial repair program declined.

Functionally, ZFP384 acted as a brake. Overexpression of Zfp384 reduced Igf1 expression in microglial cells, while genetic deletion of Zfp384 in microglia sustained recovery-associated gene expression after stroke. Mice lacking Zfp384 specifically in microglia showed better long-term neurological outcomes on behavioral tests, without significant differences in infarct volume, cerebral blood flow, or survival.

That distinction is important. The intervention did not appear to reduce the initial ischemic injury. Instead, it improved the recovery phase, suggesting a therapeutic concept distinct from acute neuroprotection.

Mechanistically, the study links ZFP384 to disruption of YY1-mediated chromatin interactions. YY1 helped maintain enhancer–promoter contacts required for recovery-associated gene expression, including at the Igf1 locus. As ZFP384 increased, it displaced this repair-permissive chromatin organization, shifting microglia toward a dysfunctional state.

The authors describe this as a strategy to “prevent the loss of reparative immunity,” preserving beneficial immune-cell functions rather than broadly suppressing inflammation.

Antisense therapy improved recovery in mice

The translational component of the study used antisense oligonucleotides (ASO) designed to reduce Zfp384 expression. After intracerebroventricular administration, the ASO was taken up by microglia and reduced Zfp384 mRNA expression.

When ASO-Zfp384 was administered on days 8 and 22 after stroke onset, mice showed improved neurological recovery compared with controls. Notably, benefit was also observed when treatment began on day 29, suggesting that the approach may influence the chronic recovery phase in this mouse model rather than only early repair.

The biological readouts supported the behavioral findings. ASO-Zfp384 sustained microglial recovery-associated gene expression, increased IGF1-positive microglia in peri-infarct tissue, and promoted broader neural repair signatures in oligodendrocyte precursor cells, excitatory neurons, and astrocytes. Treated mice showed improved myelination, enhanced white matter conduction, and increased synaptic markers including synaptophysin and PSD95.

Neutralizing IGF1 or SPP1 reduced the recovery benefit, strengthening the conclusion that microglial neurotrophic factors were functionally involved.

Human tissue supports the pathway

The researchers also examined human post-mortem brain tissue from patients who had experienced ischemic stroke. In peri-infarct regions, IGF1-positive IBA1-positive cells were more abundant early after stroke and declined later. ZNF384, the human orthologue of mouse ZFP384, showed the opposite pattern, increasing later in the recovery period.

The inverse relationship between IGF1 and ZNF384 supports the relevance of the pathway in human stroke biology, although it remains correlative. The human samples do not show that ZNF384 inhibition would improve patient outcomes, but they indicate that the same molecular pattern observed in mice may also occur in the human post-stroke brain.

Implications for rehabilitation medicine

The findings point to a therapeutic space that remains underdeveloped: enhancing recovery after the acute phase. Rehabilitation depends on plasticity, remyelination, and circuit remodeling, but current pharmacological options to extend or potentiate this biology are limited.

A therapy that sustains reparative microglia could, in principle, complement rehabilitation by keeping the peri-infarct environment more permissive for repair. That would represent a different clinical goal from thrombolysis, thrombectomy, or anti-inflammatory intervention. Rather than rescuing threatened tissue in the first hours, the aim would be to improve the quality and duration of recovery over subsequent weeks.

The study remains preclinical. Delivery route, dose, safety, timing, durability, and patient selection all require further work. The mouse model cannot capture the full heterogeneity of human stroke, including lesion location, age, comorbidities, vascular risk, rehabilitation intensity, and medication use. Sustaining immune-mediated repair also needs careful safety evaluation, because prolonged activation of tissue-resident immune cells could have context-dependent risks.

Still, the concept is compelling. Microglia are not merely inflammatory cells to inhibit; they can be repair partners whose beneficial state may be actively preserved. If ZFP384-targeted approaches prove safe and effective in larger models, they could open a new class of post-stroke recovery therapies focused on extending the brain’s own repair window.

As Tsuyama put it, “sustaining the brain’s endogenous repair program” may create opportunities to reduce permanent neurological symptoms during rehabilitation.

The post Targeting Microglia Could Help Extend the Recovery Window After Stroke appeared first on Inside Precision Medicine.

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Nucleus accumbens DRD2 receptor agonism attenuates escape behavior

Animals learn to approach and escape stimuli in their environment, in part through the representation of rewarding or aversive outcomes in the nucleus accumbens (NAc). The regulation of reward motivation in the NAc by dopamine signaling at DRD1 and DRD2 receptors has been the subject of extensive study. However, the process by which aversive stimuli are signaled within this system to promote motivated escape behavior is less well characterized. Conventional wisdom posits that rewarding and aversive stimuli ultimately affect DRD1 or DRD2-receptor expressing medium spiny neurons (MSNs) in an opposing manner to differentially modulate motivated behavior. However, recent studies have challenged this view and demonstrate the need to better characterize the processes that mediate aversion learning. To determine if DRD2 dopamine receptor activation disrupts escape behavior, 21 male and female Sprague Dawley rats were treated with an intra-NAc core DRD2 receptor agonist, quinpirole, while escape behavior was negatively reinforced by the termination of aversive white noise. This treatment attenuated escape, a result that is consistent with the view that aversion-induced reductions in dopamine promote escape behavior through decreased DRD2 receptor signaling in the NAc, and potential disinhibition of an aversion-sensitive striatal output circuit.

Attention-deficit/hyperactivity disorder and chronic pain: a scoping review of epidemiology, clinical phenotypes, mechanisms, and treatment

IntroductionEmerging evidence suggests a potential association between attention-deficit/hyperactivity disorder (ADHD) and chronic pain; however, extant findings are dispersed across disciplines and have not yet been comprehensively synthesized. This scoping review aimed to systematically map the epidemiological evidence, clinical phenotypes, proposed neurobiological mechanisms, and reported therapeutic interventions related to comorbid ADHD and chronic pain.MethodsA comprehensive literature search was conducted in PubMed, PsycINFO, and the Cochrane Library from database inception to December 28, 2025. Human studies examining the association between ADHD (diagnosed or symptom-defined) and chronic or recurrent pain were also included. Fifty studies met the eligibility criteria, including observational studies (cross-sectional and longitudinal), case reports and series, and one interventional study.ResultsEpidemiological studies have consistently reported significant associations between ADHD symptoms or diagnoses and chronic pain in both the general population and clinical samples. Higher ADHD symptom burden was associated with greater pain severity and pain-related functional impairment. Comorbidity was observed not only in widespread pain syndromes, such as fibromyalgia, but also in site-specific conditions, including chronic low back pain, orofacial pain, and migraine. Proposed mechanisms involve dopaminergic and noradrenergic dysfunction affecting motor regulation, sensory processing, and descending pain modulation systems. Several case-based reports have described improvements in pain outcomes after ADHD-targeted pharmacotherapy. However, controlled trials remain scarce.DiscussionCurrent evidence suggests that ADHD traits may be relevant in a subset of individuals with chronic pain, particularly those with treatment-resistant presentations. Although causality and treatment efficacy remain unconfirmed, consideration of neurodevelopmental characteristics may enhance clinical assessments and inform future research on individualized mechanism-based treatment strategies.

Family caregivers’ involvement in home-based recovery for patients with schizophrenia: a qualitative study in Beijing, China

BackgroundFamily caregivers play critical roles in supporting the home-based recovery of patients with schizophrenia, but they often encounter substantial challenges and receive insufficient systemic support. Understanding caregivers’ involvement in home-based recovery is essential for aligning community mental health services with families’ capacities and needs. This study aimed to explore family caregivers’ involvement and adaptive processes in supporting home-based recovery of patients with schizophrenia in China.MethodsA qualitative study using interpretative phenomenological analysis was conducted through semi-structured interviews. Family caregivers were purposively recruited from four community health service centers (CHSCs) across urban and rural areas of Beijing. All interviews were audio-recorded, transcribed verbatim, anonymized, and analyzed iteratively to identify themes and subthemes.ResultsA total of 20 family caregivers were recruited, including 11 from two urban districts and 9 from a rural district in Beijing. Five themes were identified: Caregivers’ redefinition of recovery as stability rather than cure; Routine recovery involvement in medication management and symptom monitoring; Experienced tensions between the patient’s independence and relapse prevention; Bearing family obligation and personal strain in sustained caregiving involvement; and Uncertainty in sustaining caregiving and the patients’ future stability. Caregivers reported persistent challenges in supporting patients’ independent living, participation in family activities, communication, and social interaction.ConclusionFamily caregivers gradually develop their capacity to support home-based recovery, but continue to encounter complex challenges while receiving limited support from CHSCs. Strengthening recovery-oriented family support within community mental health services, particularly through accessible psychoeducation and rehabilitation guidance, may enhance caregivers’ capacity to support patients’ independence and social participation, thereby promoting sustainable home-based care and long-term functional recovery.

STAT+: A breakthrough in pancreatic cancer has experts excited — and braced for what’s to come

LONDON — When researchers started planning a pancreatic cancer conference here, the aim was to simply offer a counterpart to a regular U.S. meeting, where international scientists and doctors could discuss the latest developments in the specialty. 

Then came the biggest shake-up in pancreatic cancer care in decades. 

“It’s one of those moments,” Talia Golan, an oncologist at Israel’s Sheba Medical Center, said on a panel that conference organizers hastily added to the schedule centered on a new pancreatic cancer drug. She likened the medicine’s recent clinical trial performance to other milestones in cancer treatment such as the arrival of the first checkpoint inhibitors. 

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