Therapeutics

Etanercept

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Overview

Name: Etanercept
Synonyms: Enbrel™
Therapy Type: Immunotherapy (passive) (timeline)
Target Type: Inflammation (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 2)
Company: Amgen, Inc., Pfizer
Approved for: Rheumatoid Arthritis, Psoriatic Arthritis, Ankylosing Spondilysis

Background

Etanercept inhibits the function of a pro-inflammatory cytokine called tumor necrosis factor alpha (TNF-α). Etanercept is a fusion protein consisting of two copies of the TNF-α receptor and the Fc end of the immunoglobulin G (IgG) antibody. Also called a decoy receptor, etanercept binds both the soluble and the membrane-bound forms of TNF-α, counteracting its signaling.

Etanercept is administered by injection under the skin, and in this form is FDA-approved for the treatment of various rheumatological and inflammatory skin conditions. Etanercept is effective and widely prescribed for these conditions. Its side effects include, among others, increased risk for serious infections including tuberculosis, invasive fungal infections, and bacterial infections such as listeria (see Enbrel.com).

The rationale of evaluating peripheral administration of etanercept as a treatment for Alzheimer's disease derives from studies suggesting that low-grade, chronic, systemic inflammation releases TNF-α, which reportedly can communicate from the periphery to the brain to induce a central immune response involving adverse microglial activation. In Alzheimer's disease patients, elevated serum TNF-α has been linked to faster decline and worse psychiatric symptoms (Perry et al., 2007; Drake et al., 2001; Holmes et al., 2009; Holmes et al., 2011).

In mouse models of neurodegenerative disease, several studies have shown that microglia are primed to activation in response to systemic inflammatory stimuli (Mar 2015 conference news; Perry and Hughes, 2014; Cunningham et al., 2005).

Findings

As of spring 2015, one double-blind, randomized, placebo-controlled trial of subcutaneous etanercept in Alzheimer's disease has been conducted, at the University of Southampton, U.K. In 2011, this investigator-initiated trial began enrolling 41 people with mild to moderate Alzheimer's disease whose MMSE fell between 10 and 27 and who had no evidence of prior exposure to tuberculosis. They were randomized to 50 mg of enbrel or matching placebo injected under the skin once a week for six months. Primary outcomes were tolerability as measured by compliance, and safety as measured by the number of and type of adverse events. Secondary outcomes were cognitive, functional, and behavioral measures, as well as cytokine measures in blood.

This trial has been published in the peer-reviewed literature (Butchart et al., 2015). In brief, etanercept was reported to be well tolerated, with 18 of 20 patients in the etanercept group completing the study compared to 15 of 21 in the placebo group. No new side effects appeared in this small AD population. As expected, infections were more common in the etanercept group. They included gastroenteritis, respiratory and urinary-tract infections, pharyngitis, and cellulitis. Eleven reports of infection were captured in nine people on etanercept compared to seven infections in six people on placebo. Of 97 side effects recorded in this study, one was serious but occurred in the placebo group. 

The secondary clinical outcomes showed no statistically significant differences, but did show trends favoring the peripheral etanercept group. Of note, decline on the ADAS-cog in the placebo group was twice what had been anticipated, and the randomization results indicated slightly worse neuropsychiatric symptoms in the placebo group at baseline. Analysis of serum inflammatory markers showed no differences between the groups at baseline, but higher serum TNF-α levels at weeks 12 and 24 weeks of treatment, as well as four weeks later after washout. This is consistent with the increased half-life of the dimeric fusion protein after binding TNF-α. 

According to the authors, this study calls for independent validation in a larger, more heterogeneous AD patient population. It does not by itself support off-label use of subcutaneous etanercept for the treatment of AD dementia (Butchart et al., 2015). It also, according to the authors, is different in concept from a hypothesized rapid change of central TNF-α through a perispinal etanercept injection. This approach has not been evaluated in RCTs and remains controversial (Novella S, Science-based Medicine, accessed 11 May 2015).

Last Updated: 12 May 2015

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Therapeutics

Estrogen

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Overview

Name: Estrogen
Synonyms: Premarin™
Therapy Type: Other
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued)
Approved for: Hormone Replacement Therapy

Background

There are a number of biological mechanisms through which estrogens might exert neuroprotective effects. These include the promotion of cholinergic activity (Gibbs, 2010), increase in the morphological complexity of neurons associated with learning and memory (Brinton et al., 2000), protection from toxic insult (Brinton et al., 2000), stimulation of neuron formation (Tanapat et al., 1999), and reduction of the formation of Aβ (Pike et al., 2009). 

Estrogen is a steroid hormone important in the development and maintenance of the female reproductive system and secondary sex characteristics. In addition, in vitro experiments have demonstrated that estrogen can protect cultured neurons from Aβ-related toxicity (Behl et al., 1995Goodman et al., 1996Mook-Jung et al., 1997), as well as oxidative stress-related damage (e.g., Behl, 2002). Animal models based on these mechanisms also support potential benefits of estrogen in the CNS. Estrogen is capable of decreasing amyloid accumulation and improving memory performance in ovariectomized rats (Carroll et al., 2007Shang et al., 2010), and has been shown to enhance long-term potentiation in the hippocampus in these rats (Foy et al., 1999).

Last Updated: 26 Oct 2015

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Therapeutics

Eptastigmine

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Overview

Name: Eptastigmine
Synonyms: MF 201
Therapy Type: Small Molecule (timeline)
Target Type: Cholinergic System (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued)
Company: Mediolanum
Approved for: None

Background

Eptastigmine is a centrally acting cholinesterase inhibitor.

Last Updated: 16 Oct 2013

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Therapeutics

ELND005

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Overview

Name: ELND005
Synonyms: AZD-103, Scyllo-inositol, cyclohexane-1,2,3,4,5,6-hexol
Therapy Type: Small Molecule (timeline)
Target Type: Amyloid-Related (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Inactive)
Company: Elan Corporation, Opko Health, Speranza Therapeutics, Transition Therapeutics, Inc.
Approved for: None

Background

Originally developed by Transition Therapeutics, ELND005 was temporarily taken over by Elan Corporation, Speranza's parent company. In 2014, ELND005 reverted to Transition Therapeutics, which was acquired by Opko Health in 2016.

ELND005 is scyllo-inositol, an oral inositol stereoisomer that is thought to neutralize toxic, low-N Aβ oligomers and prevent them from aggregating. Scyllo-inositol has been reported to lead to dose-dependent decreases in amyloid pathology, insoluble Aβ40 and Aβ42, and subsequent plaque accumulation in TgCRND8 mice. The compound was also reported to rescue Aβ-induced toxicity to synaptic transmission in mouse hippocampi and to erase learning deficits in transgenic mice. The treatment appeared to work both when it was begun before symptoms appeared, and after the disease process was established. Beneficial effects via neuronal autophagy and preservation of choline acetyltransferase have also been proposed. Preclinical and some clinical data were published (e.g. Townsend et al., 2006Fenilli et al., 2007Ma et al., 2012).

Findings

In Phase 1, a first study in eight healthy volunteers and a second, ascending-dose study in 13 healthy volunteers, done in 2005 and 2006, indicated that the treatment was safe and well-tolerated. Subsequent studies raised further questions, however, and Phase 1 characterization of this compound's metabolism continued. In 2015, a renal clearance study concluded (company release). Some pharmacokinetic data were formally published (Liang et al., 2013).

A Phase 2 trial in 353 patients with mild to moderate Alzheimer’s disease tested doses of 500, 2,000, and 4,000 mg/day taken for 18 months. Primary endpoints were the Neuropsychological Test Battery (NTB) and Alzheimer's Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) scores. In June 2009, a long-term extension began, enrolling 103 participants who had completed the study's randomized portion. Primary endpoints of the extension were safety, tolerability, and clinically important changes in laboratory assessments; this study completed in mid-2011. After an interim analysis, in December 2009 investigators dropped the 2,000 or 4,000 mg/day doses in both the randomized and the extension study due to a higher number of serious adverse events in patients on these doses. Besides an increased rate of infections, the adverse events included nine deaths, though they were never definitively linked to ELND005 (see Dec 2009 news). The trial continued with 500 mg/day. Falls, depression, and confusion were the most common side effects at this dose, and the data safety monitoring committee deemed its tolerability to be acceptable. This Phase 2 study was negative on its primary endpoints of cognitive or functional improvement. At 500 mg/day, ELND005 did appear to reduce Aβ levels in a CSF substudy (see Salloway et al., 2011).

Pre-specified subgroup analysis indicated that some patients on the low dose had improved on some neuropsychiatric measures, and Elan decided to continue to explore those. A new mechanism of action was proposed whereby ELND005, i.e. scylloinositol, lowers brain levels of its endogenous isoform myoinositol, and exerts effects similar to lithium in the treatment of bipolar disorder (see Jun 2012 conference news).

In November 2012, Elan started a Phase 2 trial, evaluating a single dose of ELND005 in 350 people with moderate to advanced Alzheimer's for its effect on the agitation and aggression subscores in the Neuropsychiatric Inventory-Clinician rating scale (NPI-C). Called HARMONY, this was a 12-week trial with an 18-week safety extension, which enrolled 296 participants. The FDA fast-tracked ELND005 for this indication in July 2013.

In 2012, Elan started a Phase 2 study of AZD-103 as an add-on therapy in 400 patients with bipolar disorder; this program was discontinued in 2014.

In 2013, a four-week Phase 2 trial began evaluating 250 and 500 mg daily of AZD-103 in 23 young adults with Down’s syndrome. This trial was completed in November 2014, and reported at the CTAD conference to have had an acceptable safety profile and exposure levels at the 500 mg dose that support further development (see Dec 2014 conference news). For all trials of this drug, see clinicaltrials.gov.

In June 2015, Transition Therapeutics announced that the HARMONY trial had missed its primary efficacy endpoint, noting a greater-than-expected decline in agitation in the placebo group on the NPI-C scale used in this trial. In July, the company terminated the long-term extension of this study (see company release). In October 2015, the company announced that, based on posthoc analysis, the trial results numerically favored the most severely symptomatic treatment group on 20 of 21 symptoms that comprise the primary outcome measurement (see company press release).

For all trials with this drug, see clinicaltrials.gov.

Clinical Trial Timeline

  • Phase 2
  • Study completed / Planned end date
  • Planned end date unavailable
  • Study aborted
Sponsor Clinical Trial 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034
Elan Corporation, Transition Therapeutics, Inc. NCT00568776
N=353RESULTS
Elan Corporation, Transition Therapeutics, Inc. NCT00934050
N=150RESULTS

Last Updated: 14 Jun 2019

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Therapeutics

EHT 0202

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Overview

Name: EHT 0202
Synonyms: Etazolate
Chemical Name: ethyl 1-ethyl-4-[2-(propan-2-ylidene)hydrazinyl]-1H-pyrazolo[3,4-b]pyridine-5-carboxylate
Therapy Type: Small Molecule (timeline)
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 2)
Company: ExonHit Therapeutics
Approved for: None

Background

Last Updated: 22 Nov 2013

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Therapeutics

Donepezil

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Overview

Name: Donepezil
Synonyms: Aricept™, Donepezil hydrochloride, Eranz®, E 2020
Chemical Name: 2-[(1-benzylpiperidin-4-yl)methyl]-5,6-dimethoxy-2,3-dihydroinden-1-one
Therapy Type: Small Molecule (timeline)
Target Type: Cholinergic System (timeline)
Condition(s): Alzheimer's Disease, Dementia with Lewy Bodies, Down's Syndrome, Parkinson's Disease Dementia
U.S. FDA Status: Alzheimer's Disease (Approved), Dementia with Lewy Bodies (Inactive), Down's Syndrome (Inactive), Parkinson's Disease Dementia (Inactive)
Company: Corium, Inc., Eisai Co., Ltd., Pfizer
Approved for: Alzheimer's Disease, Dementia with Lewy Bodies (Japan)

Background

Donepezil is approved in more than 90 countries around the world for the treatment of mild to moderate Alzheimer's disease, and is approved for the treatment of severe AD in the United States, Japan, Canada, and several other countries. The effect size of donepezil's benefit is small, though one patient-level analysis has suggested greater benefit in early responders than in non-responders (Levine et al., 2022). Donepezil does not modify the underlying pathophysiology of the disease. Even so, this symptomatic treatment has become a mainstay of Alzheimer's therapy in North America. Donepezil is used off-label for other conditions including dementia with Lewy bodies, traumatic brain injury, vascular dementia, Parkinson’s disease dementia, and mild cognitive impairment.

Besides the standard 5- and 10-mg tablets of donepezil, a rapidly disintegrating tablet, as well as liquid, oral jelly, and transdermal formulations have been developed, but thus far approved only in select countries. In July 2010, the FDA approved a once-daily, sustained-release 23-mg tablet for the treatment of moderate to severe AD. This formulation sparked controversy. The advocacy group Public Citizen in 2011 unsuccessfully petitioned the FDA to withdraw it, charging that the formulation was more toxic than the 10-mg tablets without clearly showing greater benefit (Public Citizens Petition). In March 2022, a transdermal patch delivering 5 or 10 mg daily for one week was approved in the United States (press release). Generic versions of donepezil tablets are available in the United States and many European countries.

Donepezil reversibly inhibits acetylcholinesterase (AChE), the enzyme that degrades the neurotransmitter acetylcholine after its release from the presynapse. Donepezil and other acetyl cholinesterase inhibitors increase the availability of the acetylcholine in cholinergic synapses, enhancing cholinergic transmission. Donepezil delays the progressive worsening of cognitive symptoms of Alzheimer's disease. It has also been reported to mediate a reduction in delirium induced by hospital admission in critically ill patients with dementia (Lieberman et al., 2023).

Donepezil was first approved to be marketed in the United States in 1996, with registration following suit in Canada and the European Union in 1997 and in many other countries shortly thereafter. In 2006/2007, regulatory approval was granted in the United States and Canada, but not in Europe, for its use in the treatment of severe Alzheimer's. Eisai's applications in the United States and Europe for regulatory approval of donepezil for vascular dementia in 2002 and 2003 were rejected and development for this indication stopped, though the drug is being marketed for vascular dementia in India and several other countries. Donepezil was approved in Japan in 2014 for Lewy body dementia.

Donepezil's most common side effects are gastrointestinal, i.e., diarrhea, nausea, and vomiting; dizziness, sleeplessness/fatigue, and urinary incontinence also have been reported. These are known class effects of cholinergic therapies, and often occur primarily at the beginning of treatment. One post-hoc analysis reported an inverse correlation between body weight and side effects, recommending stepwise titration in people with low body mass index (Hong et al., 2021). With long-term use, a decrease in heart rate and arrhythmia have been reported (e.g. Koh et al., 2020Koh, et al., 2021Morris et al., 2021Kobayashi et al., 2023).

Findings

More than 250 clinical trials have been conducted thus far with donepezil, a large majority involving Alzheimer's disease. For example, in early stage AD categorized by MMSE between 21 and 26, 10 mg/day of donepezil modestly improved cognition as measured by the ADAS-cog in the first trial exclusively in patients at that stage. Similarly, a separate trial of 296 patients with mild AD showed modest benefit on cognition, particularly on various types of memory (see Seltzer et al., 2004; Seltzer et al., 2002).

In mild to moderate AD, donepezil given at 5 and 10 mg/day modestly improved cognition and functional measures in separate six-month trials of 473 patients and of 818 patients. These and other trials quantified cognition with the ADAS-cog and clinical and global function with the Clinician's Interview-Based Assessment of Change-Plus (CIBIC-plus) and the Clinical Dementia Rating-Sum of the Boxes scale (CDR-SB), as well as other measures. A one-year study of the same doses in 286 patients showed similar modest benefits on cognitive and global function, as well as on reducing the burden on caregivers (see, e.g., Rogers et al., 1998Burns et al., 1999).

The Phase 3, 817-patient AWARE (Aricept WAshout and REchallenge) study looked at continued treatment in patients who did not respond to 5 or 10 mg/day of donepezil for the first six months of treatment. In this study, donepezil was generally well-tolerated and had a benefit on memory, as well as a small benefit on activities of daily living and behavioral symptoms in the majority of patients at nine months, including those who did not initially show those benefits (see, e.g., Johannsen et al. 2006). Additional trials in various countries typically evaluated donepezil in 100 to 200 patients and lasted three to six months. In general, most donepezil AD trials have consistently shown modest benefits on cognition, whereas benefits on behavioral symptoms and activities of daily living have been more mixed (see, e.g., Oct 2007 newsGauthier et al., 2002; Cummings et al., 2006).

Initially, the small effect size of donepezil and other cholinesterase inhibitors created controversy about the cost-effectiveness of this therapy (see Jul 2004 news and extensive commentary). Since then, several long-term studies—some open-label, some initially blinded but followed by open-label extensions—have shown that donepezil is modestly effective in the long-term treatment of AD. For example, patients who received donepezil for three years had less cognitive decline than patients who took placebo for one year, then donepezil for two (see Winblad et al., 2006). Discontinuation of treatment was associated with increased risk of nursing home placement (Howard et al., 2015). A study analyzing five-year follow up data from the Swedish Dementia Registry reported Class 3 evidence of reduced cognitive decline with cholinesterase inhibitors (Xu et al., 2021).

Pharmacoeconomic studies in the United States and European countries have generally found that donepezil treatment reduces the cost of care. In the United Kingdom, where this debate had called into question coverage of donepezil by this country's universal health care system, the National Institute for Health and Clinical Excellence (NICE) in 2011 issued a guidance recommending the use of donepezil in the treatment of mild and moderate AD (see, e.g., Fillit et al., 1999; Wimo et al., 2003; NICE guidanceFeldman et al., 2004). Additional pharmacoeconomics studies have since confirmed the cost-effectiveness of donepezil treatment (e.g., Lopez-Bastida et al., 2009). In 2018, the French government stopped paying for including donepezil and other acetylcholinesterase inhibitors for the treatment of dementia symptoms, citing concerns of limited effectiveness (e.g. see Walsh et al., 2019).

For severe AD, donepezil has regulatory approval in the United States, Canada, and Japan. In general, studies report modest benefits on cognition, global function, and activities of daily living (see, e.g., Aug 2007 news; Winblad et al., 2009).

Two Phase 4 trials and one Phase 3 trial evaluated 5 to 10 mg/day of donepezil in more than 1,800 patients with mild cognitive impairment (MCI) in the United States and Canada. Donepezil was reported to slow the progression to AD dementia for about a year; however, a recent meta-analysis of the trials literature found no slowing of progression, and donepezil does not appear to be under development for an MCI indication (Petersen et al., 2005; Apr 2005 news and extensive commentary; Zhang et al., 2022). Subsequent trials detected small and mixed effects on cognitive and functional endpoints (Salloway et al., 2004; Doody et al., 2009). Donepezil had no benefit in a trial in people with depression and MCI (Aug 2018 news).

Donepezil was approved in Japan for dementia with Lewy bodies (DLB), based on a Phase 3 trial evaluating 5 or 10 mg/day of donepezil in 142 patients and a prior Phase 2 trial in 167 patients with DLB (Ikeda et al., 2015; Ikeda et al., 2013).

A trial of donepezil in 550 patients with Parkinson's disease dementia (PDD), in which dementia develops at least a year after parkinsonian symptoms, missed primary endpoints but showed signals in secondary analyses (Dubois et al., 2012). A study is planned to start in Spain in 2023 in 120 people with Parkinson’s disease and mild cognitive impairment.

Among the indications where donepezil failed to be effective are attention deficit hyperactivity disorders, migraine, Down's syndrome, and Fragile X syndrome (see, e.g., Wilens et al., 2005, Kishnani et al., 2010; Bruno et al., 2019). Trials in vascular dementia reported mixed results, some finding no clear benefit in the hereditary form of the disease called CADASIL, but others finding some benefits in sporadic, stroke-related dementia (see, e.g., Salloway et al., 2008Roman et al., 2010Sep 2003 news story). Perfusion imaging has been reported to pick up dose-dependent differences in cerebral perfusion between people on AChEIs including donepezil and untreated patients with AD, DLB, and PD (Moyaert et al., 2023).

Between 2016 and 2018, Corium conducted four Phase 1 studies comparing donepezil patches to pills in a total of 599 healthy adults. The trials tested pharmacokinetics, placement on different areas of the body, and effects of heat on drug delivery. Based on these bioequivalence studies, the FDA granted marketing approval in March 2022. The patches last for one week, delivering 5 or 10 mg drug daily. The most common side effects in the trials were headache, itching or irritation at the application site, muscle spasms, and expected cholinergic side effects such as constipation, diarrhea, and dizziness. Results of one study were published, showing the patch caused fewer GI and nervous system side effects than pills (Tariot et al., 2022). A different patch formulation was approved in Korea in 2021(see Han et al., 2022); its maker, Icure Pharmaceuticals, reportedly planned to begin testing in the United States in 2022, but no trial has been registered to date. The Japanese company Teikoku Seiyaku tested their own donepezil patch in that country, and found efficacy equal to a 5 mg daily pill (Nakamura et al., 2023).

Donepezil continues to be studied for a range of indications. Trials are ongoing for traumatic brain injury, glycemic control, and wound healing in Type 2 diabetes, and cognitive problems due to chemotherapy, radiation, or surgery, as well as chemotherapy-induced peripheral neuropathy and alcohol use disorder. A trial started in February 2022 in France, comparing donepezil to that country’s current standard non-drug approach of cognitive remediation and stimulation.

Besides its widespread use in clinical practice, donepezil is frequently used as standard of care in trials testing various investigational drugs as add-on therapy to donepezil.

For a listing of donepezil trials, see clinicaltrials.gov.

Last Updated: 16 May 2023

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Therapeutics

Docosahexaenoic acid (DHA)

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Overview

Name: Docosahexaenoic acid (DHA)
Synonyms: Omega-3 fatty acid
Therapy Type: Supplement, Dietary (timeline)
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 4)
Company: Martek Biosciences Corporation, NeuroBioPharm, Inc.

Background

Docosahexaenoic acid (DHA) is one of the most abundant polyunsaturated fatty acids in the human brain. Epidemiological research has linked high DHA consumption with a lower risk of Alzheimer's disease (e.g., Morris et al., 2003, Zhang et al., 2016). People ingest DHA from foods such as fatty fish, walnuts, or flax seeds, or from dietary supplements. These are being marketed in different formulations of DHA, or DHA mixed with other omega-3 fatty acids such as eicosapentaenoic acid (EPA).

In early epidemiology work, elevated blood levels of DHA correlated with reduced dementia risk in some but not all studies (Schaefer et al., 2006; Laurin et al., 2003). Newer studies support the link between DHA intake or blood levels and a reduced risk of cognitive decline or AD (e.g. Wei et al., 2023; He et al., 2023; Shang et al., 2018). In people with AD, higher serum DHA was associated with slower cognitive decline (Chu et al., 2022). An analysis of ADNI data found associations between low omega-3 in red blood cells and greater Aβ accumulation and memory decline (Rouch et al., 2022). Data from the large Framingham Heart study correlated higher DHA or EPA in red blood cells with larger hippocampal volume and better abstract reasoning at midlife, and up to half the risk of incident AD in older adults (Satizabal et al., 2022; Sala-Vila et al., 2022). This and other studies (e.g., Sala-Vila et al., 2021; Coughlan et al., 2021) indicate that the relationship of DHA status to cognition and neurodegeneration biomarkers is affected by ApoE4 genotype (for review see Zhange et al., 2023).

In contrast, U.K. Biobank data indicate fish oil supplementation in older people reduced risk of all-cause dementia, but not specifically of AD (Huang et al., 2022; Liu et al., 2022). In the same population, total omega-3 status, but not DHA alone, correlated with lowered risk of dementia (Sala-Vila et al., 2023). A smaller study found no association of circulating DHA with incident dementia over 13 years (de Oliveira Otto et al., 2023).

Multiple autopsy studies have described deficits of DHA-containing phospholipids in brain tissue from people with Alzheimer's, which correlates with markers of the disease (reviewed in Heath and Wood, 2021). In samples from dementia with Lewy body cases, low levels of DHA-containing phospholipids were associated with lower soluble Aβ42, higher phosphorylated α-synuclein, and diminished synaptic proteins (Chong et al., 2023).

In animal studies, oral intake of DHA leads to a reduction of amyloid, tau, and neuritic pathology (Lim et al., 2005; Green et al., 2007; Calon et al., 2004). This appears to stem from beneficial effects on amyloid processing, inflammation, oxidative stress, and metabolic function (for review see Mett 2021). In APP/PS mice, omega-3 supplementation reversed some effects of a high-diet on Aβ peptide expression, insulin resistance, and inflammation (Ma et al., 2022). DHA reduced retinal amyloid pathology in 5XFAD mice, when delivered as a lysophosphatidylcholine form uniquely bioavailable to retinal cells (Sugasini et al., 2023). Nasal delivery of DHA reduced tau phosphorylation and improved cognitive function in J20 transgenic mice, or after brain injection of Aβ peptides in normal mice (Zussy et al., 2022). DHA supplementation also reversed cognitive and olfactory deficits due to ApoE4 expression in transgenic mice (González et al., 2023; Lessard-Beaudoin et al., 2021).

Findings

DHA has been tested in clinical trials by itself and as part of other food-supplement formulations.

In 2000, Karolinska University Hospital began the OmegAD trial, which evaluated a six-month course of treatment with a DHA-containing fish oil formulation called EPAX 1050 TG in 204 people with mild to moderate AD, of whom 174 completed the study. The treatment and placebo groups did not differ on either of the main outcome measures, decline on the MMSE and ADAS-Cog, or on neuropsychiatric symptoms overall. However, analysis of a small subgroup of the 32 mildest cases did suggest less decline on the MMSE, though not ADAS-Cog; a similar slowing of decline appeared to occur in the placebo group once switched to DHA after six months (Freund-Levi et al., 2006; see news and commentaryFreund-Levi et al., 2008). Substudies suggested that DHA treatment increased CSF levels of DHA and other fatty acids and decreased levels of tau, as well as changing expression of inflammation-related genes, and release of certain cytokines and inflammatory mediators in white blood cells (Freund-Levi et al., 2014; Vedin et al., 2012; Vedin et al., 2010Vedin et al., 2008; Wang et al., 2015). Other substudies reported no effect on CSF biomarkers of inflammation or amyloidosis, or urine biomarkers of oxidative stress (Freund-Levi et al., 2009; Freund-Levi et al., 2014). Supplementation appeared to increase plasma, but not CSF transthyretin, a cofactor for Aβ clearance (Faxen-Irving et al., 2013). The treatment caused a global DNA hypomethylation measured in blood cells, but the effect correlated more strongly with EPA blood concentrations than DHA (Karimi et al., 2017).

Additional post hoc analyses of this trial showed a dose response between the increase in omega-3 plasma levels after supplementation, and slowing of decline on the ADAS-Cog (Eriksdottir et al., 2015). Patients with better Vitamin B status showed more improvement after supplementation on the MMSE and CDR-SB, but not the global CDR or ADAS-Cog (Jerneren et al., 2019). Most recently, analysis of a panel of CSF biomarkers revealed an increase in YKL-40 and NfL after six months of supplementation, while other markers of Aβ, tau, and inflammation were stable (Tofiq et al., 2021).

A Dutch study conducted in 2006 reported no effect of six months of fish oil supplements in 302 cognitively normal older adults. The participants had to have an MMSE greater than 22, and took 1,800 mg or 400 mg EPA-DHA daily, or placebo. Treatment caused no change in a neuropsychiatric test battery of attention, sensorimotor speed, memory and executive function, in quality of life, or in mental well-being (van de Rest et al., 2008; van de Rest et al., 2008; van de Rest et al., 2009).

From 2007 to 2009, the Alzheimer's Disease Cooperative Study conducted a study at 51 centers in North America to evaluate an 18-month course of 2 grams per day of DHA in 402 patients with mild to moderate AD, of whom 295 completed the trial. DHA had no effect, relative to placebo, on the rate of decline on either the ADAS-Cog or the CDR-SOB clinical/functional assessment. Analysis by participants' ApoE genotype indicated a slower cognitive decline in ApoE4 noncarriers, who may have been relatively less advanced in their disease (Quinn et al., 2010Nov 2010 news). Subsequently, ApoE4 carriers were found to have smaller increases in plasma DHA and EPA, and less delivery to CSF after supplementation, than noncarriers (Tomaszewski et al., 2020; Yassine et al., 2016).

This pharmacogenetic hint of a differential effect prompted several bioavailability studies at the Université de Sherbrooke, Quebec, Canada, between 2009 and 2011, which analyzed the percentage of DHA in lipoproteins, incorporation into plasma lipids, and pharmacokinetics by ApoE genotype in healthy young adults and people with MCI (e.g., Chouinard-Watkins et al., 2013Plourde et al., 2014). The study confirmed lower plasma levels and higher oxidation rates, contributing to a shorter half-life of DHA in ApoE4 carriers.

The largest clinical trial of DHA is MAPT, conducted in four cities in France. This three-year, secondary prevention study in 1,680 participants began in 2008 and enrolled people 70 and older who reported a subjective memory complaint and a mild functional loss, were frail and walked slowly, but did not meet an Alzheimer's diagnosis (Carrié et al., 2012). MAPT compared three interventions—800 mg DHA and 225 mg EPA daily alone, DHA/EPA plus a multidomain behavioral intervention, multidomain behavioral intervention alone—to placebo (Gillette-Guyonnet et al., 2009). Neither intervention, alone or in combination, significantly slowed cognitive decline as measured by a composite score of four tests of recall, orientation, processing and verbal fluency (Andrieu et al., 2017). An amyloid PET substudy compared the effects of the multidomain intervention and DHA/EPA according to brain amyloid status. The multidomain intervention, with or without DHA/EPA, improved composite scores after 36 months compared to placebo in the amyloid-positive, but not -negative, subset; DHA/EPA alone had no effect in either subset (Delrieu et al., 2019; Delrieu et al., 2023). In another PET analysis, cortical amyloid was lower after two years in people who got the multidomain intervention, with or without DHA/EPA, but not in those who received DPA/EPA alone (Hooper et al., 2020). DPS/EPS had no effect on brain structure by MRI, global functional connectivity, or on a composite physical and cognitive endpoint of intrinsic capacity (Sivera et al., 2020; Perus et al, 2022; Giudici et al., 2020). As in the OmegAD study, results suggested that people with low Vitamin B status may benefit less from omega-3 supplementation (Maltais et al., 2022). In this population, low DHA/EPA was potentially associated with psychiatric disorders. Participants with low RBC DHA-EPA at baseline were more likely to be using psychotropic drugs, and those who increased DHA-EPA after treatment were less likely (Gallini et al., 2019).

A subsequent analysis of MAPT data suggested higher odds of cognitive decline for people with low DHA/EPA levels (Bowman et al., 2019). Lower serum DHA was also correlated with more cerebral amyloidosis and atrophy (Yassine et al., 2016Aug 2016 news). In response to those findings, MAPT investigators in April 2018 began LO-MAPT. This 18-month study planned to enroll 400 older adults with low DHA/EPA status and randomize them to 1.53 g/day DHA/EPA or placebo. Participants had to have subjective memory complaints or a family history of AD. The primary outcome is change in a cognitive composite of scores on the Free Cued and Selective Reminding Test, MMSE, and Category Naming Test. The trial offered an 18-month open-label extension. It was completed in 2020, after enrolling 774 participants.

From 2009 to 2011, the company NeuroBioPharm Inc. ran a six-month trial at 14 different sites in Canada to compare soft-capsule formulations of fish oil and krill oil, each containing 100 mg DHA, to placebo in 175 people with mild to moderate Alzheimer's disease. Krill oil contains DHA and EPA in a form claimed to be more bioavailable than fish oil (e.g. Schuchardt et al., 2011). This trial used the Neuropsychological Test Battery as primary outcome; data have not been published.

Between 2013-2015, a study in Taiwan tested six months of daily DHA, EPA, or the combination, against placebo in 163 people with mild cognitive impairment or AD dementia. Doses of 0.7 g/day DHA and 1.6 g/day EPA had no effect on cognitive function or depressive symptoms (Lin et al., 2022).

One DHA study, at Oregon Health and Science University, started in May 2014. According to published baseline data, the trial recruited 102 people age 75 and older who were cognitively impaired but did not have dementia, and suboptimal blood levels of DHA and EPA of less than 110 μg/ml. Participants were randomized to receive a three-year course of 1.65 grams/day of EPA plus DHA or soybean oil placebo (Bowman et al., 2019). Seeking to understand DHA's effect on vascular cognitive aging, this trial used white-matter hyperintensity as primary outcome; secondary and other outcome measures included other brain imaging modalities as well as blood-based indicators of endothelial health and neuropsychological tests. Top-line results were presented at the 2020 CTAD. Supplementation failed to slow accumulation of white-matter hyperintensities in the 45 placebo and 42 treated participants who had a least one follow-up MRI. At the end of the study, 24 of the treatment group had plasma DHA+EPA levels greater than 110 μg/ml. In a prespecified analysis, this group had fewer white-matter hyperintensities and maintained better white-matter integrity than the placebo group. There were no differences in total brain volume, ventricular volume, medial temporal lobe atrophy, excessive cognitive functions, or adverse events.

In June 2016, a pilot study at the University of Southern California began evaluating how much of a DHA supplement enters the central nervous system, and whether CSF levels are influenced by ApoE4 status (for review, see Yassine et al., 2017). The trial enrolled 31 adults older than 55 with a family history of dementia, who took 2 g DHA per day or placebo for 26 weeks. The primary outcome was change in CSF DHA before and after supplementation. According to published results, this dose of DHA resulted in a 200 percent increase in plasma DHA compared to placebo, but only a 28 percent increase in CSF DHA (Arellanes et al., 2020). ApoE4 carriers had slightly lower CSF DHA. For EPA, CSF levels increased 43 percent, but were significantly lower in ApoE4 carriers than in noncarriers. Other endpoints including brain volume and cognitive scores did not differ between supplement and placebo groups.

In September 2018, the same group began a larger follow-up study in 368 healthy adults age 60-80, who have at least one dementia risk factor. Half are to be ApoE4 carriers. Participants take 2g DHA or placebo daily for two years. After six months of treatment, investigators will analyze CSF fatty acids in 168 participants, to determine the effect of their ApoE genotype on supplementation. Changes in MRI measures of structural and functional connectivity and cognition will be assessed in all participants at two years. The study will run through 2025. The trial protocol and baseline data are published (Yassine et al., 2023).

For details of clinical trials of DHA in Alzheimer's, see clinicaltrials.gov.

Last Updated: 12 Jan 2024

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Therapeutics

Dimebon

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Overview

Name: Dimebon
Synonyms: Dimebolin, Latrepirdine, Pf-01913539
Chemical Name: 3,6-dimethyl-9-(2-methyl-pyridyl-5)-ethyl-1,2,3,4-tetrahydro-γ-carboline dihydrochloride
Therapy Type: Small Molecule (timeline)
Target Type: Other Neurotransmitters (timeline), Unknown
Condition(s): Alzheimer's Disease, Huntington's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued), Huntington's Disease (Discontinued)
Company: Medivation, Inc.
Approved for: Anti-histamine use in Russia

Background

Dimebon is an antihistamine that has been used in Russia since the 1980s to treat allergic rhinitis.

Dimebon is a pleiotropic drug with activities beyond blocking H1 histamine receptors. A mechanism of action for cognitive benefit has never been conclusively established; however, a broad spectrum of effects on neurologically relevant targets was proposed based on various cell- and animal-based studies. For example, Dimebon has been variously reported to modulate the activity of certain channels and neurotransmitter systems, including L-type and voltage-gated calcium channels, AMPA and NMDA glutamate receptors, α-adrenergic receptors, and serotonergic or dopaminergic receptors.

Rodent models suggested benefits in avoidance tests in rats and hippocampal learning, though animal-behavior studies overall reported mixed results. Dimebon also has been proposed to be neurogenic, to exert protective effects on neuronal mitochondria, to reduce aggregation of misfolded proteins, to upregulate autophagy, and to be neuroprotective via a variety of pathways including blocking Aβ-mediated toxicity (for reviews, see Bezprozvanny, 2010Bharadwadj et al., 2013). 

Findings

Between 2001 and 2010, Dimebon was repurposed for the treatment of Alzheimer's and Huntington's and underwent clinical evaluation for cognitive and psychiatric benefit in those diseases. Phase 1 and 2 trials of a total of 197 Alzheimer's patients in Russia were followed by two Phase 3 trials in some 1,600 patients with mild to moderate AD conducted in the Americas, Europe, Australia, and New Zealand. The Phase 2 trial reported significant improvement over placebo, but neither Phase 3 study detected change in any primary or secondary outcome (Bachurin et al., 2001Doody et al., 2008Mar 2010 news story).

Likewise in Huntington's, a Phase 2 study reported significant improvement on cognitive measures, but a subsequent Phase 3 trial in 403 patients was negative on all outcomes (Horizon Investigators, 2013). A peer-reviewed meta-analysis of all Dimebon trials later confirmed that the drug had no significant benefit on cognition (Cano-Cuenca et al., 2014).  

For detailed news chronicling Dimebon's development history, see Jan 2012 news storyApr 2011 news story; Apr 2010 news storyMar 2010 news storyOct 2009 news storyAug 2008 news storyMay 2007 news story; and all news on Dimebon

See also all commentary on Dimebon and all clinical trials on Dimebon.

Last Updated: 29 Sep 2023

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Therapeutics

Dapsone

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Overview

Name: Dapsone
Synonyms: Avlosulfon, Diaminodiphenylsulfone, DDS
Chemical Name: 4-[(4-aminobenzene)sulfonyl]aniline
Therapy Type: Small Molecule (timeline)
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Inactive)
Company: Immune Network Ltd.
Approved for: Leprosy, malaria, some bacterial skin infections

Background

Dapsone is a small molecule with anti-inflammatory immunosuppressive properties as well as antibacterial and antibiotic properties. It is used clinically to treat leprosy and malaria, and is being investigated for the treatment of a variety of skin infections.  Dapsone acts against bacteria and protozoa  by inhibiting the synthesis of dihydrofolic acid through competition with para-amino-benzoate for the active site of dihydropteroate synthetase. According to Drugbank.ca, the anti-inflammatory action of the drug is unrelated to its antibacterial action and is not fully understood.

Findings

Many studies have linked Alzheimer's disease with inflammation (Halliday et al., 2000) and epidemiological evidence suggests a reduced risk of AD in patients who take anti-inflammatory medications (McGeer et al., 1996), including those with leprosy (McGeer et al., 1992). However, other studies have not observed reduced prevalence of AD in leprosy patients chronically taking anti-inflammatory medication (Goto et al., 1995). 

Last Updated: 09 Jan 2014

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Therapeutics

CX516

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Overview

Name: CX516
Synonyms: Ampalex
Therapy Type: Small Molecule (timeline)
Target Type: Other Neurotransmitters (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Discontinued)
Company: Cortex Pharmaceuticals, Inc.
Approved for: None

Background

CX516 is a so-called ampakine, a molecule that potentiates currents mediated by AMPA-type glutamate receptors. As a class, these drugs enhance AMPA-receptor activity by slowing deactivation and attenuating desensitization of AMPA receptor currents, thus increasing synaptic responses and enhancing long-term potentiation (reviewed in Arai and Kessler, 2007).

Last Updated: 10 Dec 2013

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