Research Models

Lrrk2 KO Mouse

Synonyms: Lrrk2 knockout mouse, LRRK2−/−

Tools

Back to the Top

Species: Mouse
Genes: Lrrk2
Modification: Lrrk2: Knock-Out
Disease Relevance: Parkinson's Disease
Strain Name: B6.129X1(FVB)-Lrrk2tm1.1Cai/J

Modification Details:

Summary

This knock-out (KO) model was generated by deleting exon 2 of the Lrrk2 gene, which results in a premature stop codon in exon 3. Brain tissue from Lrrk2 KO mice does not express full-length LRRK2 protein. KO mice are viable, fertile, and do not exhibit any developmental or gross physical abnormalities (The Jackson Laboratory; Parisiadou et al., 2009).

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Dopamine Deficiency
  • Non-Motor Impairment
  • α-synuclein Inclusions
  • Neuronal Loss

No Data

Neuronal Loss

Neuronal Loss No  differences between KO and wild-type mice up to 24 months of age in the number of tyrosine hydroxylase (TH)–positive cells in the substantia nigra pars compacta. No neurodegeneration markers observed in the striatum and cortex at 20 months. Cerebral cortex and dorsal (but not ventral) striatum volumes reduced at 12 months.

Dopamine Deficiency

Levels  of TH in the striatum are equal between genotypes in 18- to 24-month-old mice.

α-synuclein Inclusions

No abnormal accumulation of α-synuclein in the cell bodies of striatal neurons observed in 20-month-old KO mice.

Neuroinflammation

Striatal staining of GFAP, a marker of reactive astrocytosis, did not differ between control and KO mice, but cells positive for Iba1 staining, a marker of activated microglia, were moderately enlarged in the striatum of 20 -month-old KO mice. Cx3cr1 mRNA levels higher in KO mouse brains.

Mitochondrial Abnormalities

Adult (9 - to 23-week-old) Lrrk2 KO mice exhibit enhanced mitophagy in dopaminergic neurons of the substantia nigra pars compacta, as detected by an increase in the number of mitolysosomes.

Motor Impairment

Motor behavior is generally intact up to 18 months based on Rotarod and open field tests. However, some age-dependent effects are observed on the open field test: 12 -month-old mice traveled longer distances and had higher walking speeds versus controls, which was not apparent in 3- or 24-month-old mice. Older (24 months) mice had deficits in motor skill learning as measured by Rotarod.

Non-Motor Impairment

No differences were observed between KO and wild-type mice across 6 to 24 months of age on several behavioral tests, including the elevated plus maze for anxiety-like behavior, the buried treat test to measure hyposmia, the grip strength test for forelimb strength, or working memory as measured by spontaneous alternation.

Last Updated: 20 May 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Therapeutics

NT-0796

Tools

Back to the Top

Overview

Name: NT-0796
Synonyms: Propan-2-yl (2R)-2-{[(1,2,3,5,6,7-Hexahydro-s-indacen-4-yl)- carbamoyl]oxy}-3-(pyrimidin-2-yl)propanoate
Therapy Type: Small Molecule (timeline)
Target Type: Inflammation (timeline)
Condition(s): Parkinson's Disease
U.S. FDA Status: Parkinson's Disease (Phase 1/2)
Company: NodThera

Background

NT-0796 is an oral, brain-penetrant inhibitor of inflammasomes that contain NLRP3, aka nod-like receptor family pyrin domain-containing protein 3. Inflammasomes are multiprotein, cytosolic complexes that function as sensors in the innate immune system. They are found in microglia in the brain, and in monocytes and macrophages outside the brain. Their activation by pathologic proteins and other stressors triggers production and secretion of the proinflammatory cytokines IL1-β and IL-18. Inflammasomes can induce cell death.

NLRP3-containing inflammasome activation is implicated in a growing number of neurodegenerative conditions where chronic inflammation plays a role, including Alzheimer’s and Parkinson’s diseases, as well as in obesity and cardiovascular disease (reviewed in Li et al., 2023).

In Alzheimer’s mouse models, inhibition of the NLRP3 inflammasome has been shown to reduce amyloid and tau pathology (Heneka et al., 2013; Ising et al., 2020).

NT-0796 inhibits NLRP3 inflammasome-mediated cytokine production in human blood with an IC50 of 6.8 nanomolar (Harrison et al., 2023). It enters the brain of mice with a blood-to-brain ratio of 0.79. NT-0796 is a prodrug that undergoes intracellular conversion to its active form in human immune cells. This activation does not occur in mice, which lack the necessary enzyme. A humanized mouse line was produced for pharmacokinetic and pharmacodynamic profiling of the compound (Smolak et al., 2024). In diet-induced obesity in these mice, NT-0796 caused weight loss as potently as did the GLP1 inhibitor semaglutide, and improved markers of cardiovascular risk (Thornton et al., 2024). It did not cause weight loss in non-obese mice.

Findings

From August 2021 to August 2022, NodThera ran a Phase 1 first-in-human study to assess safety and pharmacokinetics of NT-0796 in 76 healthy volunteers. Single ascending doses from 1 to 300 mg were given as a liquid formulation. Multiple MAD cohorts were planned based on results of the single-dose study, and included CSF sampling. Pharmacodynamic outcomes included inflammatory cytokines IL-1β, IL-18, IL-6, and TNFα in serum, and after ex vivo stimulation of blood cells from participants. In May 2022, the company reported the drug was safe after single dosing, and showed dose-proportional pharmacokinetics. Treatment lowered inflammatory cytokine levels in the ex vivo blood cell assay (press release). A September 2022 press release on the multiple-dose part of the study claimed brain penetration to levels higher than needed to achieve an anti-inflammatory effect, and a reduction in blood levels of the inflammatory biomarker C-reactive protein after treatment. No doses were specified, or data shown.

In 2023, the company began a Phase 1b/2a trial to measure the effect of NT-0796 on inflammatory and disease specific biomarkers in patients with Parkinson’s disease (press release). This trial does not appear in registries. According to NodThera, the first part of the study tested a new capsule formation in healthy volunteers, who received 150 mg twice a day for one week. A cohort with Parkinson’s disease received the same dose for four weeks. In a July 2023 press release, the company claimed significant reductions in inflammatory markers in CSF in four healthy volunteers after one week treatment. They also claimed a 13 percent reduction in CSF neurofilament light chain after one week.

Results of the completed trial were presented at the March 2024 AD/PD conference. The drug appeared safe. Adverse events were mainly mild, transient, and unrelated to drug. There were no serious adverse events. The capsule formulation extended the drug’s half-life compared to the liquid. CSF levels of drug in PD patients were two to three times higher than in elderly volunteers with the same plasma levels. After one or four weeks of treatment, patients had reductions in CSF IL-1, and IL-6, and a trend toward reduction in neurofilament light chain and soluble Trem2. Blood markers of peripheral inflammation were also reduced. Participants did not lose weight. The company is conducting long-term toxicology studies to facilitate a new trial.

In October 2023, NodThera began a Phase 1/2 trial to assess inflammation, weight loss, and safety in obese volunteers.

For details on the Phase 1 trial, see ANZCTR.org. For other trials, see clinicaltrials.gov.

Last Updated: 16 May 2024

Comments

No Available Comments

Make a Comment

To make a comment you must login or register.

Further Reading

No Available Further Reading

Therapeutics

Lixisenatide

Tools

Back to the Top

Overview

Name: Lixisenatide
Synonyms: Adlyxin, Lyxumia
Therapy Type: Other
Target Type: Other (timeline)
Condition(s): Parkinson's Disease
U.S. FDA Status: Parkinson's Disease (Phase 2)
Company: Sanofi
Approved for: Type 2 diabetes

Background

Lixisenatide is an analog of the hormone glucagon-like peptide-1. GLP-1 stimulates the pancreas to release insulin in response to food intake. GLP-1 mimetics resensitize cells to insulin signaling and are used to treat Type 2 diabetes. Several GLP-1 mimetics are also approved for weight loss. The FDA approved Adlyxin for Type 2 diabetes in 2016, but Sanofi discontinued marketing it for this disease in 2023, citing business reasons. Lixisenatide is taken as a once-daily self-injection under the skin.

Lixisenatide is one of four GLP-1 mimetics being tested for Alzheimer’s or Parkinson’s diseases. It is a 44-amino acid peptide closely related to exenatide (Leon et al., 2017). Lixisenatide appears to cross the blood-brain barrier better than liraglutide and semaglutide (Hunter and Hölscher, 2012; Salameh et al., 2020).

The rationale of using GLP-1 receptor agonists for neurodegeneration stems from observations of insulin resistance in some cases of Alzheimer's disease (e.g. Talbot et al., 2012; Talbot, 2014). Type 2 diabetes raises the risk of Parkinson’s, and in some studies, the prevalence of Parkinson’s was lower in people with diabetes who were treated with GLP-1 receptor agonists compared to those taking other diabetes medications (Brauer et al., 2020; Svenningsson et al., 2016).

GLP-1 mimetics are neuroprotective in preclinical models of Alzheimer’s and Parkinson’s (e.g. Perry et al., 2002; Bertilsson et al., 2008; Li et al., 2010). Potential mechanisms of neuroprotection by GLP-1 mimetics include reduced inflammation (reviewed in Reich and Hölscher, 2022). 

Preclinically, lixisenatide reduced Aβ-induced impairments in spatial learning and memory in rats, and was neuroprotective in APP/PS1/tau mice (Cai et al., 2014; Cai et al., 2017Cai et al., 2018). In the APPswe/PS1Δe9 mouse line, it improved object recognition and synaptic plasticity, prevented synapse loss, reduced amyloid load and microglial activation, and increased neurogenesis, all at lower doses than liraglutide (McClean and Hölscher, 2014; Hölscher, 2014). In the MPTP toxin-induced mouse model of Parkinson's lixisenatide prevented motor impairment and dopamine neuron loss (Liu et al., 2015).

For a review of the development of GLP-1 class drugs for Parkinson’s and Alzheimer’s, see Hölscher, 2024.

Findings

In June 2018, French researchers started a Phase 2 study to evaluate the effect of lixisenatide in people with early Parkinson’s disease. The LixiPark trial enrolled 156 participants, with a target dose of 20 μg daily for one year, or a matched placebo, in addition to their usual Parkinson's medications. The primary outcome was motor function, as measured by the Movement Disorders Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part 3. Conducted at 20 sites in France, the trial ended in April 2021. Results are published (see Apr 2024 news on Meissner et al., 2024). After 12 months, the lixisenatide group had improved by 0.04 points on the primary outcome, compared to a decline of 3.04 points in the placebo group, a statistically significant difference. A difference favoring treatment remained after two months off drug. Secondary outcomes including non-motor symptoms, activities of daily living, and need for levodopa were not different between the groups. One third of participants had to limit their dose to 10 μg due to side effects, mainly nausea, as well as vomiting and acid reflex. A post hoc analysis suggested greater benefit for people under 60.

For details on the LixiPark trial, see clinicaltrials.gov.

Last Updated: 14 May 2024

Comments

No Available Comments

Make a Comment

To make a comment you must login or register.

Further Reading

No Available Further Reading

Therapeutics

PhotoBioModulation

Tools

Back to the Top

Overview

Name: PhotoBioModulation
Synonyms: Low level light therapy, Low level laser therapy, Cold laser therapy, Transcranial infrared brain stimulation, Transcranial photobiomodulation, Transcranial near-infrared laser therapy (NILT), Transcranial low-level light therapy
Therapy Type: Procedural Intervention
Target Type: Inflammation (timeline), Unknown
Condition(s): Alzheimer's Disease, Mild Cognitive Impairment, Parkinson's Disease
U.S. FDA Status: Alzheimer's Disease (Not Regulated), Mild Cognitive Impairment (Not Regulated), Parkinson's Disease (Not Regulated)

Background

Photobiomodulation is a form of light therapy that exposes tissue to red or near-infrared (NIR) light. Low-energy lasers or light-emitting diodes are placed on or near the skin. Their light penetrates tissue, where endogenous chromophores absorb it, and cause physical and chemical changes in cells. This non-heating therapy employs wavelengths from ~600 to ~1100 nm. Near-infrared light, with its longer wavelengths, penetrates tissues more deeply than shorter wavelengths of red light. This treatment causes no notable side effects, and can be done at home.

Red or NIR light is claimed to promote cell health by enhancing mitochondrial energy production, and by increasing blood circulation. Absorption of NIR light by the mitochondrial enzyme cytochrome C oxidase and subsequent stimulation of the respiratory chain is the commonly claimed mechanism of action (reviewed in Hamblin, 2018). This therapy has been studied in animal models, where it is reported to increase ATP production, stimulate anti-inflammatory, anti-apoptotic, and antioxidant responses, promote neurogenesis and synaptogenesis, and improve cognition (reviewed in Salehpour et al., 2018). In healthy rats, photobiomodulation (PBM) improved the metabolic profile, spatial memory, and neuroinflammation markers in young and aged animals (Dos Santos Cardoso et al., 2021; Cardoso et al., 2022).

Literature in animal models of Alzheimer’s disease reports improved mitochondrial function, reduced Aβ plaques, tau tangles, inflammation, and oxidative stress, and lessening of cognitive deficits after transcranial PBM (e.g. De Taboada et al., 2011; reviewed in Su et al., 2023; Salehpour et al., 2021). Targeting abdomen, bone marrow, or lymph nodes with light therapy also improved outcomes in mouse models of AD and PD (Oron and Oron, 2016; Johnstone et al., 2014; Wu et al., 2022; Farfara et al., 2015). PBM is claimed to activate microglia, stimulate glymphatic clearance of Aβ, and improve gut flora (Salehpour et al., 2022; Chen et al., 2021; Liebert et al., 2019; Stepanov et al., 2022). In a negative study, transcranial PBM with 810 nm light had no benefit in the 5XFAD mouse model (Sipion et al., 2023).

For a recent review on preclinical and clinical development of PBM for AD and PD, see Shen et al., 2024.

Parameters of time, wavelength, power, and light source for PBM are under investigation (e.g. Spera et al. 2021; Joshi et al., 2024). In extracranial low-energy applications, only a tiny fraction of light penetrates the skull to reach the brain, although applying higher light intensity can increase penetration (Henderson and Morries, 2015). Human cadaver studies have shown near-infrared wavelengths can penetrate 40 mm through the scalp and skull into the brain (Tedford et al., 2015). Alternative approaches such as intracranial and intranasal delivery are being investigated to reach deeper brain structures (e.g., Salehpour et al., 2020).

Many different PBM devices are sold directly to consumers for home use. Some are FDA-approved for treating pain and inflammation, wound healing, promoting hair growth, and decreasing fat deposits; many are not. Hundreds of clinical trials are registered for a wide range of applications. A Phase 3 trial in stroke patients was terminated for futility (Hacke et al., 2014). More recently, a placebo-controlled trial found no effect on cognitive impairment in people with schizophrenia (Kheradmand et al., 2022). Evidence for cognitive improvement in healthy adults is mixed (Salehpour et al., 2019; Lee et al., 2023).

Findings

Case reports and pilot studies claim benefits of PBM in people with mild cognitive impairment or dementia. For example, one patient with dementia reversed cognitive and olfactory dysfunction after daily PBM therapy to the head, lower back, and intranasally (Salehpour et al., 2019). In MCI, a trend was claimed toward improved blood flow and cognition after eight weeks of red light to the main arteries supplying the brain (Baik et al., 2021). A single session of light therapy was claimed to acutely improve memory in adults with MCI (Chan et al., 2021). Among 42 women with MCI, five sessions of transcranial PBM reportedly improved MMSE scores and measures of attention over sham treatment (Papi et al, 2022). 

From 2010-2012, the Quietmind Foundation sponsored a pilot trial in 11 people with early and mid-stage dementia, to assess whether repeated scalp exposure to six minutes of 1,072 nm infrared stimulation daily for 28 days improves cognitive and behavioral functioning as indicated by normalization of EEG activity, increased cerebral oxygenation and performance on standardized neuropsychological measures. The sham-controlled study lists a sole outcome of ADAS-Cog. Results are published, claiming trends toward a benefit in some parts of the ADAS-Cog related to executive functioning, and improvements in EEG parameters and functional connectivity (Berman et al., 2017). A follow-up study with 100 participants began in October 2018. This trial combined PBM with neurofeedback, and ran for eight weeks. Sixty people were enrolled, and the results are published (Nizamutdinov et al., 2021). Treatment was associated with improvements in the MMSE and several parts of the neuropsychological test battery, sleep, anxiety, mood, and daily routine.

Several device makers have sponsored trials for treatment of mild cognitive impairment or dementia. The Vielight Neuro Gamma device consists of five LEDs placed on the scalp to target default mode network nodes, plus an intranasal LED to target the hippocampus. All LEDS deliver 810 nm light, at either 10 or 40 Hz frequency. Both frequencies have been shown to modulate EEG alpha, beta, and gamma waves in healthy adults (e.g. Zomorrodi et al., 2019). An early study in five people with AD used 10 Hz and reported significant improvement on the MMSE and the ADAS-Cog after 12 weeks of treatment. Caregivers reported that patients slept better, had fewer angry outbursts, and less anxiety and wandering (Saltmarche et al., 2017).

In May 2017, a single-center trial at University of California, San Francisco, began testing the Vielight device in 20 dementia patients. It compared 12 weeks of 40 Hz therapy, delivered for 20 minutes three days a week at home, to usual care, reporting improvements in the ADAS-Cog and neuropsychiatric symptoms, increased cerebral blood flow, and increased connectivity within the default mode network in the first eight treated patients (Chao, 2019). A follow-up trial, completed in January 2021, tested active versus sham 40 Hz treatment for 16 weeks in 14 AD patients, against a primary outcome of change in ADAS-Cog. Results posted on clinicaltrials.gov indicate no changes with treatment in cognition or biomarkers of Aβ42, tau, and neurofilament light.

In December 2017, Vielight began a study in 60 people with moderate to severe AD at two sites in Ontario, comparing 12 weeks of active to sham home treatment with 40 Hz therapy for 20 minutes daily, six days a week. The primary outcome was change in the Severe Impairment Battery score. The company claims sham treatment is indistinguishable from active, as their device produces no heat and no visible light. This study was completed in February 2022. No results have been made public.

In June 2019, Vielight began a pivotal trial of the 40 Hz device. This trial planned to enroll 228 participants with severe dementia, for active versus sham treatment against endpoints of SIB and ADCS-ADL. This trial was supposed to finish in May 2023, but was suspended due to slow recruitment.

In June 2019, an academic study in Florida and Arizona began to study whether PBM could improve age-related cognitive and mood changes in older healthy adults and those with PD. The study plans to enroll 135 participants to be randomized to 12 weeks of photobiomodulation combining lab-based cranial stimulation with the MedX health Console plus intranasal stimulation at home with the Vielight device. The primary outcome is change in performance on the Arena task, a virtual reality version of the Morris water maze (Parslow et al., 2004). Completion is anticipated in October 2024.

The same university groups began a preventive trial in August 2020, involving 168 older adults at risk for AD, defined by subjective cognitive complaints and a first-degree family history of Alzheimer's disease. Participants are randomized to 12 weeks of in-lab cranial plus at-home intranasal treatment, against the primary outcome of the ARENA score. Completion was expected in April 2024.

In April 2021, the TRAP-AD trial began testing transcranial PBM in 125 people with MCI or mild AD dementia (Iosifescu et al., 2023). This academic, multisite, sham-controlled study is assessing the effects of continuous 808 nm NIR light delivered bilaterally to the forehead for 11 minutes, in 24 sessions over eight weeks. The device is an investigational helmet made by LiteCure medical laser company. Target engagement is to be confirmed by changes in fMRI blood oxygen-level dependent (BOLD) signal after one treatment (Gaggi et al., 2024). RBANS total score after eight weeks serves as the primary outcome; secondary outcomes include RBANS and other cognitive tests out to three months. This study also uses MRS imaging to assess change in mitochondrial function, and tau-PET to relate treatment effects to baseline tau burden. Completion is expected in November 2025.

In March 2023, a pilot trial began at Unity Health Toronto testing the Vielight device in 20 people with mild cognitive impairment due to AD. The treatment regimen involves 20 minutes per day, six days a week, for six weeks, and includes a sham control. The primary outcomes are MMSE, and memory and executive function tests. The trial will assess quality of life, blood and MRI biomarkers, sleep, and neuropsychiatric and depressive symptoms. Completion was expected in March 2024.

From September 2018 to May 2020, the French company REGEnLIFE ran a feasibility trial of its RGn530 device, which features a helmet and abdominal belt that deliver 10 Hz pulses of NIR and red light from a laser and LEDs. The study randomized 53 people with mild to moderate AD to active or sham treatment, before being terminated due to COVID. Results are published (Blivet et al., 2022). No serious adverse events were recorded. Patients complied, with most completing the prescribed 40 treatment sessions of 25 minutes each over eight weeks. The investigators claimed trends toward improvement on some cognitive measures.

In July 2023, REGEnLIFE began recruiting for the pivotal LIGHT4Life study. This sham-controlled trial plans to enroll 108 people with mild to moderate AD in multiple centers in France. Treatment consists of six months of 20 minute in-clinic sessions, beginning at five per week, and gradually ramping down to two per week (Blivet et al., 2024). The primary outcome is ADAS-Cog; 34 secondary and exploratory outcomes include other cognitive and functional tests, safety, blood biomarkers of amyloid, inflammation, tau, and neurodegeneration, as well as analysis of fecal microbiota, and metabolomics. Primary completion is expected in May 2025.

REGEnLIFE is also running a pilot in 50 people with concussion. This follows on clinical studies of transcranial NIR in people with traumatic brain injury that claim improvements in executive function, learning, and memory (Naeser et al., 2014).

A pilot study of an NIR device in Iran reported safety and benefits in people with AD (Razzaghi et al., 2024); studies on other devices began in late 2023 in China and Japan.

PBM is also being tested for Parkinson’s disease (for review, see Bicknell et al., 2024). A sham-controlled study in people with PD suggested that red light targeting the substantia nigra could speed walking (Santos et al., 2019). In an open-label study of 12 people, Australian investigators claimed better mobility, cognition, dynamic balance, and fine motor skills after 12 weeks to one year of in-clinic and at-home PBM to the abdomen, neck, head, and nose (Liebert et al., 2021). The treatment induced changes in the gut microbiome (Bicknell et al., 2022). Some patients improved with at-home PBM to just the abdomen and neck (Liebert et al., 2022). A sham controlled pilot subsequently tested an Australian company’s transcranial device in 20 people with PD, finding a 12-week course of treatment to be safe and feasible (Herkes et al., 2023). Another study in Australia reported that four weeks of transcranial plus intraoral treatment did not change cognition measured by the MoCA, although some improvement was measured in writing and walking. A placebo effect was seen (Bullock-Saxton et al., 2021). An ongoing trial in France is testing endoventricular PBM using an implanted light source (see Darlot et al., 2016).

For more on photobiomodulation trials for AD, see clinicaltrials.gov.

For more on photobiomodulation trials for PD, see clinicaltrials.gov.

Last Updated: 09 May 2024

Comments

No Available Comments

Make a Comment

To make a comment you must login or register.

Further Reading

No Available Further Reading

Therapeutics

UCB0022

Tools

Back to the Top

Overview

Name: UCB0022
Therapy Type: Small Molecule (timeline)
Target Type: Other Neurotransmitters (timeline)
Condition(s): Parkinson's Disease
U.S. FDA Status: Parkinson's Disease (Phase 2)
Company: UCB S.A.

Background

This oral small molecule is a positive allosteric modulator of dopamine receptor type 1, which enhances the receptor's signaling in the presence of endogenous dopamine. It binds outside the dopamine site on the receptor, and has no intrinsic receptor-activating potential. UCB0022 is intended to reduce the need for escalating dopamine doses in people with Parkinson’s disease, and to prevent side effects like dyskinesis that arise due to excess signaling through the dopamine type 2 receptor.

The company has shown preclinical data at meetings (e.g. Vermeiren et al., 2022), claiming nanomolar affinity and selectivity and allosteric effects of UCB0022 in cell-based and in vitro assays. The compound reportedly enhances the potency of dopamine to activate D1Rs up to 10-fold, with no effect on other dopamine receptor subtypes. In an MTPT toxin model of Parkinson’s in nonhuman primates, UCB0022 had a longer duration of benefit on motor symptoms, less dyskinesia, and extended ON time compared to levodopa.

UCB has patented a series of tetrahydroisoquinoline derivative D1 positive allosteric modulators. Lilly has taken its D1 PAM, Mevidalen, through Phase 2.

Findings

In April 2021, Phase 1 began with a safety and pharmacokinetic study of single and multiple ascending doses in healthy people and people with Parkinson’s. A total of 100 volunteers were exposed to drug, against a primary endpoint of treatment-emergent adverse events. According to data presented at the March 2024 AD/PD conference, all side effects were mild or moderate. The most common were insomnia, dizziness, and decreased appetite. Higher doses transiently increased heart rate and blood pressure in healthy participants. All adverse events were reduced by dose titration. In Parkinson’s patients, blood pressure did not increase. There were no new neurological findings, deaths, or severe treatment-emergent adverse events.

In November 2023, a Phase 2 study called ATLANTIS began evaluating UCB0022 as an adjunct to levodopa in people with advanced PD. The trial is recruiting 189 patients, to be randomized to one of two doses or placebo. The primary outcome is fluctuation in motor function, measured as the average number of patient-reported OFF hours per day. Secondary outcomes are incidence of side effects, trial withdrawal, and drug levels in blood. The trial, at 40 sites in the U.S., is expected to finish in December 2024.

For details on UCB0022 trials, see clinicaltrials.gov.

Last Updated: 03 May 2024

Comments

No Available Comments

Make a Comment

To make a comment you must login or register.

Further Reading

No Available Further Reading

Research Models

Trem2-H157Y x 5xFAD

Tools

Back to the Top

Species: Mouse
Genes: Trem2, APP, PSEN1
Modification: Trem2: Knock-In; APP: Transgenic; PSEN1: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: N/A

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

  • Synaptic Loss

No Data

  • Tangles
  • Neuronal Loss
  • Changes in LTP/LTD
  • Cognitive Impairment

Plaques

Decreased plaque burdens and densities in 5xFAD;Trem2H157Y/H157Y compared with 5xFAD;Trem2+/+ at 8.5 months, but genotypes similar at 4 months.

Tangles

No data.

Neuronal Loss

No data.

Gliosis

Decreased microgliosis and astrogliosis in 5xFAD;Trem2H157Y/H157Y compared with 5xFAD;Trem2+/+ at 8.5 months, but genotypes similar at 4 months.

Synaptic Loss

Cortical levels of synaptophysin and PSD95 did not differ between genotypes at 8.5 months.

Changes in LTP/LTD

No data.

Cognitive Impairment

No data.

Last Updated: 20 May 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Research Models

Trem2-IPDxAPP23xPS45

Synonyms: TREM2-IPDxAPP23xPS45, APP23xPS45xIPD

Tools

Back to the Top

Species: Mouse
Genes: Trem2, APP, PSEN1
Modification: Trem2: Knock-In; APP: Transgenic; PSEN1: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: N/A

Summary

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

No Data

  • Tangles
  • Neuronal Loss
  • Changes in LTP/LTD
  • Cognitive Impairment

Plaques

Greater numbers of plaques, particularly small plaques, and larger areas occupied by plaques in 3-month-old Trem2-IPDxAPP23xPS45 mice, compared with APP23xPS45 animals. These genotype-dependent differences disappeared by 7 months.

Tangles

No data.

Neuronal Loss

No data.

Gliosis

Increased microgliosis in the vicinity of plaques in 3-month-old Trem2-IPDxAPP23xPS45 mice, compared with APP23xPS45 mice.

Synaptic Loss

Compared with APP23xPS45 mice, 3-month-old Trem2-IPDxAPP23xPS45 mice had fewer and smaller puncta stained for the presynaptic marker Sv2a (synaptic vesicle glycoprotein 2A) in the vicinity of plaques. These genotype-dependent differences disappeared  by 7 months.

Changes in LTP/LTD

No data.

Cognitive Impairment

No data.

Last Updated: 22 Apr 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Research Models

AAV-sTREM2 PS19

Synonyms: AAV-sTREM2 Tau P301S

Tools

Back to the Top

Species: Mouse
Genes: TREM2, MAPT
Modification: TREM2: Virus; MAPT: Transgenic
Disease Relevance: Frontotemporal Dementia, Alzheimer's Disease
Strain Name: N/A

Summary

Triggering Receptor Expressed on Myeloid Cells 2 (TREM2) is a transmembrane receptor found on microglia, where it modulates cell activity and survival. In addition to membrane-associated TREM2, there are soluble forms of the protein—generated by protease cleavage of the extracellular domain or expression of alternative transcripts that lack a transmembrane domain.

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

No Data

  • Plaques
  • Tangles
  • Neuronal Loss
  • Gliosis

Plaques

No data.

Tangles

No data.

Neuronal Loss

No data.

Gliosis

No data.

Synaptic Loss

AAV-mediated expression of sTREM2 protected against hippocampal synapse loss in PS19 mice.

Changes in LTP/LTD

LTP at Shaeffer collateral-CA1 synapses was slightly enhanced in hippocampal slices from 7-month-old AAV-sTREM2 PS19 mice, compared with PS19 mice who had received control vector.

Cognitive Impairment

AAV-mediated expression of sTREM2 improved performance of PS19 mice in the Morris water maze and Y-maze.

Complementary Models

The AAV-sTREM2 PS19 model employed AAV-mediated over expression of an extracellular fragment of TREM2 in the brains of PS19 mice to study the effects of chronically elevated soluble TREM2 (sTREM2) in the context of tauopathy (Zhang et al., 2023).

Another approach to investigating the role of sTREM2 is the direct application of the protein to animals or cells, but these studies generally look at short-term effects. sTREM2-Fc is a recombinant, chimeric protein intended to mimic sTREM2, consisting of the human TREM2 extracellular domain (amino acids 1-171) fused to human IgG-Fc to aid in purification of the recombinant protein (Zhong et al., 2017). When applied to primary neurons from PS19 mice or HEK293 cells stably overexpressing GFP-Tau, sTREM2-Fc led to a dose-dependent decrease in levels of tau phosphorylated at sites targeted by GSK3β (Zhang et al., 2023). Conditioned medium from BV2 cells infected with the same AAV-sTREM2 vector used in the PS19 mice also reduced tau phosphorylation in the GFP-Tau-expressing HEK293 cells (Zhang et al., 2023).

Last Updated: 21 May 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Research Models

AAV-sTREM2 5xFAD

Synonyms: 5xFAD-sTREM2

Tools

Back to the Top

Species: Mouse
Genes: TREM2, APP, PSEN1
Modification: TREM2: Virus; APP: Transgenic; PSEN1: Transgenic
Disease Relevance: Alzheimer's Disease
Strain Name: N/A

Summary

Triggering Receptor Expressed on Myeloid Cells 2 (TREM2) is a transmembrane receptor found on microglia, where it modulates cell activity and survival. In addition to membrane-associated TREM2, there are soluble forms of the protein—generated by protease cleavage of the extracellular domain or expression of alternative transcripts that lack a transmembrane domain.

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

No Data

  • Tangles
  • Neuronal Loss
  • Synaptic Loss

Plaques

When examined at 7 months of age, amyloid plaque burdens in the hippocampus and cortex of AAV-sTREM2 5xFAD mice were about half those of 5xFAD mice injected with control vector.

Tangles

No data.

Neuronal Loss

No data.

Gliosis

The number of plaque-associated microglia was increased in AAV-sTREM2 5xFAD mice examined at 7 months of age.

Synaptic Loss

No data.

Changes in LTP/LTD

Long-term potentiation at Shaeffer collateral-CA1 synapses is impaired in 5xFAD mice. AAV-mediated over expression of sTREM2 rescued LTP in AAV-sTREM2 5xFAD mice.

Cognitive Impairment

5xFAD mice show impaired learning and memory in the Morris water maze, but AAV-sTREM2 5xFAD mice performed as well as non-transgenic controls.

Complementary Models

The AAV-sTREM2 5xFAD model employed AAV-mediated over expression of an extracellular fragment of TREM2 in the brains of 5xFAD mice to study the effects of chronically elevated soluble TREM2 (sTREM2) in the context of amyloidosis.

Another approach to investigating the role of sTREM2 is the direct application of the protein to animals or cells, but these studies generally look at short-term effects. sTREM2-Fc is a recombinant, chimeric protein intended to mimic sTREM2, consisting of the human TREM2 extracellular domain (amino acids 1-171) fused to human IgG-Fc to aid in purification of the recombinant protein (Zhong et al., 2017). Guojun Bu, Xiao-Fen Chen and colleagues injected sTREM2-Fc into the right hippocampi of 7-month-old 5xFAD mice; the left hippocampi were injected with buffer (Zhong et al., 2019). One day post-injection, sTREM2-Fc was found throughout the right hippocampus, but the injected protein was barely visible via immunohistochemistry at three-days post-injection. When examined one week after injection, the hippocampi injected with sTREM2-Fc had reduced plaque loads compared with the hippocampi injected with buffer. This finding is congruent with the decreased plaque loads seen in AAV-sTREM2 5xFAD mice. Exposure to sTREM2-Fc also resulted in an increased number of plaque-associated microglia—like the finding in AAV-sTREM2 5xFAD—increased microglial phagocytosis of Aβ, and increased expression of inflammatory cytokines. sTREM2-Fc did not appear to affect astrogliosis, as assessed by GFAP immunoreactivity. Neuritic pathology was ameliorated by exposure to sTREM2-Fc.

The protease-cleavage site in TREM2 had not yet been identified at the time Bu, Chen and colleagues began the study described above (i.e., it was not known that the physiological form of sTREM2 ended at amino acid 157). These investigators subsequently made a chimeric protein consisting of amino acids 1-157 of human TREM2 fused to IgG-Fc. One week following injection of sTREM2(1-157)-Fc into the right hippocampi of 5xFAD mice, a reduced plaque load and an increased number of plaque-associated microglia were seen in the right side, compared with the left hippocampi, which received buffer injections.

A concern with these experiments is the amount of recombinant sTREM2-Fc injected into the mouse brains. Bu and colleagues immunoprecipitated TREM2 from the brains of 5xFAD mice and measured the amount of sTREM2 by mass spectrometry, finding around 15 fmol sTREM2/mg brain tissue (Qiao et al., 2023). They then retrospectively estimated the concentration of sTREM2-Fc in the hippocampi of the mice in their 2017 study, in which they injected sTREM2-Fc into the hippocampi of wild-type mice (Zhong et al., 2017). Assuming that the injected sTREM2-Fc spread throughout the entire hippocampus but did not spread much beyond this structure, they calculated that the concentration of sTREM2-Fc was about 3.3 pmol sTREM2-Fc/mg brain tissue—more than 200-fold that of endogenous sTREM2 in 5xFAD. (It should be noted that this comparison also depends upon the assumption that there was nearly complete recovery of endogenous sTREM2 from brain lysates.) Twice the amount of sTREM2-Fc was injected into the hippocampi of the 5xFAD mice in the 2019 study (Zhong et al., 2019) as was injected into the wild-type mice in the 2017 study.

When applied to hippocampal slices from 5xFAD mice, sTREM2-Fc (50 nM) rescued deficits in long-term potentiation, as did AAV-mediated over expression of sTREM2 in 5xFAD brains (Zhong et al., 2019).

Last Updated: 21 May 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Research Models

TREM2-IPD

Synonyms: Trem2-IPD

Tools

Back to the Top

Species: Mouse
Genes: Trem2
Modification: Trem2: Knock-In
Disease Relevance: Alzheimer's Disease
Strain Name: B6-Trem2em2Npa

Summary

Triggering Receptor Expressed on Myeloid Cells 2 (TREM2) is a transmembrane receptor found on microglia, where it modulates cell activity and survival. In addition to membrane-associated TREM2, there are soluble forms of the protein—generated by protease cleavage of the extracellular domain or expression of alternative transcripts that lack a transmembrane domain.

Phenotype Characterization

When visualized, these models will distributed over a 18 month timeline demarcated at the following intervals: 1mo, 3mo, 6mo, 9mo, 12mo, 15mo, 18mo+.

Absent

No Data

  • Plaques
  • Tangles
  • Neuronal Loss
  • Synaptic Loss
  • Changes in LTP/LTD
  • Cognitive Impairment

Plaques

No data.

Tangles

No data.

Neuronal Loss

No data.

Gliosis

At 3 months of age, TREM2-IPD mice had more Tmem119-positive microglia and a greater percentage of proliferating microglia than mice expressing wild-type Trem2.

Synaptic Loss

No data.

Changes in LTP/LTD

No data.

Cognitive Impairment

No data.

Complementary Models

The TREM2-IPD model employs a genetic manipulation to disrupt the ADAM cleavage site on TREM2—reducing the generation of sTREM2 and increasing signaling-competent TREM2 on the cell surface. A team led by Christian Haass in Munich and Joseph Lewcock at Denali used an alternate strategy to block ADAM cleavage of TREM2, employing a monoclonal antibody (4D9) that binds near the cleavage site and impedes access of the enzyme to the receptor (Schlepckow et al., 2020; see 10 Mar 2020 news). Like the IPD mutation, antibody 4D9 reduces levels of sTREM2 and increases cell-surface TREM2, but the models differ in an important aspect: Antibody 4D9 activates TREM2 signaling, likely through cross-linking cell-surface TREM2.

The IPD mutation and 4D9 treatment had similar effects on the physiology of myeloid cells in vitro. Both manipulations increased the survival of bone-marrow-derived macrophages after withdrawal of macrophage colony-stimulating factor, and both increased phagocytic activity of microglia.

The effects of 4D9 treatment and of the IPD mutation have also been studied in mouse models of amyloidosis. As the effects of these manipulations were studied in two different mouse models, direct comparisons are difficult. Nonetheless, both manipulations appeared to accelerate the transition of microglia from a homeostatic to a disease-associated phenotype. In Trem2-IPDxAPP23xPS45 mice, the IPD mutation led to higher plaque burdens and more severe plaque-associated pathology at an early stage of plaque deposition, but these effects disappeared as the mice aged (Dhandapani et al. 2022). Administration of the antibody over a 10-day period to 6-month-old APP NL-G-F knock-in mice—a late stage of plaque deposition in this line—led to decreased cortical plaque loads.

While the agonist (i.e., TREM2-activating) effect of 4D9 limits its usefulness as a tool to study the consequences of reduced TREM2 shedding, agonist antibodies are being investigated as potential therapeutics for AD (Price et al., 2020; Wang et al., 2020; 26 Jun 2020 news), and at least one such antibody is in clinical trials.

Reduced generation of sTREM2—in this case, by HEK293 cells overexpressing TREM2 and its adaptor DAP12—has also been achieved using single-chain variable fragments (scFvs) that bind the extracellular domain of TREM2 (Szykowska et al., 2021; 29 Jul 2021 news). However, in this case, decreased production of sTREM2 was likely caused by internalization of the receptors rather than blockage of the ADAM cleavage site.

Last Updated: 21 May 2024

COMMENTS / QUESTIONS

No Available Comments

Make a comment or submit a question

To make a comment you must login or register.

Further Reading

No Available Further Reading

Subscribe to ALZFORUM RSS