Effects of ganaxolone: A Synthesis of Findings from 25 Studies

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Original Abstract of the Article

Main Research Findings

Ganaxolone has shown promise as a potential treatment for epilepsy, particularly for drug-resistant epilepsy. 16 found that ganaxolone, when combined with cannabidiol, exhibited a synergistic effect in reducing seizures in adults with refractory focal onset epilepsy. 18 reported that ganaxolone is an alternative treatment option for infantile spasms, a severe form of epilepsy in infants, when other treatments like ACTH or vigabatrin are ineffective. 15 , 21 , 3 , 22 , and 5 highlight ganaxolone's potential as a neurosteroid that modulates GABA-A receptors, a key neurotransmitter system in the brain, to exert anti-seizure effects. Furthermore, 8 and 14 suggest that ganaxolone may play a role in neurosteroid replacement therapy, particularly in promoting brain development, especially in preterm infants. Research suggests that ganaxolone may also have therapeutic potential for other neurological disorders like fragile X syndrome and postpartum depression. 17 and 19 discuss these possibilities.

Benefits and Risks

Benefit Summary

Ganaxolone shows potential as a treatment option for epilepsy, particularly for drug-resistant epilepsy and infantile spasms. 16 , 22 , 10 further highlight its potential in these areas. Moreover, 8 and 14 suggest that ganaxolone could be beneficial in promoting brain development and neuroprotection. Research indicates that ganaxolone may also hold promise for treating other neurological conditions like fragile X syndrome and postpartum depression. 17 and 19 discuss these possibilities.

Risk Summary

While ganaxolone shows potential, it's important to consider its risks. 1 highlights concerns about its safety, especially the need for careful dosage adjustments. 24 suggests that ganaxolone might negatively impact motor function. 3 and 6 mention the possibility of drug interactions. Additionally, 19 and 13 suggest potential mental health impacts. It's crucial to discuss these risks with a healthcare professional before using ganaxolone.

Comparison Across Studies

Commonalities

Many studies agree that ganaxolone works by influencing GABA-A receptors, a critical neurotransmitter system in the brain, to exert its effects. Furthermore, research consistently shows that ganaxolone might promote brain development and neuroprotection. 8 and 14 provide evidence for this.

Differences

There are variations among studies in terms of their focus, methodology, and the types of patients or conditions being studied. For example, 16 focuses on refractory focal onset epilepsy in adults, while 18 investigates infantile spasms. Similarly, 1 explores ganaxolone's potential for treating PTSD, while other studies don't examine this aspect.

Consistency and Contradictions in Results

While research suggests that ganaxolone might be beneficial for epilepsy, 16 , 22 , 10 , a study on PTSD ( 1 ) did not find significant positive results. It's important to note that the effectiveness and side effects of ganaxolone could vary depending on factors such as dosage, administration method, and the specific condition being treated. Therefore, more research is needed to fully understand ganaxolone's effects.

Practical Considerations

While ganaxolone shows potential, it's crucial to consider that it can interact with other medications. 3 and 6 highlight this. Additionally, ganaxolone may have mental health impacts. 19 and 13 discuss this possibility. It's important to consult with a healthcare professional before using ganaxolone and to follow their instructions carefully. 1 emphasizes the need for proper dosage adjustments.

Current Research Limitations

Research on ganaxolone is still in its early stages. 22 states that long-term safety and effectiveness need further investigation. 3 emphasizes the lack of research on drug interactions. Further research is needed to fully understand the effects and potential risks associated with ganaxolone.

Future Research Directions

Long-term clinical trials are crucial to better evaluate ganaxolone's effectiveness and safety. 22 stresses this need. Furthermore, basic research is required to understand the mechanisms behind ganaxolone's actions. 3 underscores the importance of studying drug interactions. Continued research is essential to comprehensively understand the benefits and risks of ganaxolone.

Conclusion

Ganaxolone shows potential as a treatment option for epilepsy and other neurological conditions. 16 , 22 , 10 discuss its potential in these areas. However, 1 and 24 highlight safety concerns and the importance of proper dosage adjustments. It's crucial to consult with a healthcare professional before using ganaxolone and to follow their instructions carefully. 3 and 6 mention potential drug interactions. 19 and 13 indicate that ganaxolone may impact mental health. Continued research is essential to further understand the benefits, risks, and potential applications of ganaxolone.

Literature analysis of 25 papers
Positive Content
25
Neutral Content
0
Negative Content
0
Article Type
1
0
2
8
24
1.

A randomized controlled trial of ganaxolone in posttraumatic stress disorder.

Author: RasmussonAnn M, MarxChristine E, JainSonia, FarfelGail M, TsaiJulia, SunXiaoying, GeraciotiThomas D, HamnerMark B, LohrJames, RosseRichard, SummerallLanier, NaylorJennifer C, CusinCristine, LangAriel J, RamanRema, SteinMurray B

PTSD treatment
Under-dosing

This study investigated the efficacy of ganaxolone, a neuroactive steroid, for the treatment of posttraumatic stress disorder (PTSD). Despite initial promising preclinical findings, the study found no significant difference between ganaxolone and placebo in a double-blind trial. The study suggests that under-dosing may have contributed to the lack of efficacy.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

2.

Emerging drugs for focal epilepsy.

Author: MulaMarco

Epilepsy treatment

This review discusses current compounds in preclinical and clinical development for the treatment of focal epilepsies, highlighting potential molecular targets. Several compounds, including Everolimus, Ganaxolone, Cannabidavirin, Padsevonil, and Selurampanel, show promise for treating focal epilepsy. Everolimus represents a first-generation disease-modifying agent, while other compounds are in earlier phases of development.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

3.

Novel antidepressant drugs: Beyond monoamine targets.

Author: GondaXenia, DomePeter, NeillJoanna C, TaraziFrank I

Improved efficacy of antidepressants
Reduced side effects

Antidepressants have been largely focused on targeting monoamine neurotransmitters but have limited efficacy and side effects. Novel antidepressants are being developed that target other pathways, including glutamatergic and GABAergic pathways, to improve efficacy, safety, and tolerability. Psychedelic drugs like psilocybin, Ayahuasca, 5-MeO-DMT, and LSD are also being explored as potential treatments for depression.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

4.

Neurosteroid-based intervention using Ganaxolone and Emapunil for improving stress-induced myelination deficits and neurobehavioural disorders.

Author: CrombieGabrielle K, PalliserHannah K, ShawJulia C, HodgsonDeborah M, WalkerDavid W, HirstJonathan J

White matter deficit improvement
Prenatal stress

Prenatal stress in mid-late pregnancy is associated with long-term disturbances in white matter development and behavior in children. The study aimed to examine the effects of prenatal stress on oligodendrocyte lineage components and determine if postnatal therapies directed at ameliorating white matter deficits improve behavioral outcomes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

5.

[New antiepileptic drugs].

Author: VidaurreJorge, HerbstJames

Epilepsy treatment
Side effects

This study explores the effectiveness and safety profile of third-generation antiepileptic drugs for treating epilepsy. It highlights the benefits of newer drugs like brivaracetam, clobazam, lacosamide, and eslicarbazepine in managing different types of seizures and discusses the potential future role of drugs like cannabidiol, fenfluramine, allopregnenolona, and ganaxolone.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : Spanish

6.

GABAkines - Advances in the discovery, development, and commercialization of positive allosteric modulators of GABA<sub>A</sub> receptors.

Author: CerneRok, LippaArnold, PoeMichael M, SmithJodi L, JinXiaoming, PingXingjie, GolaniLalit K, CookJames M, WitkinJeffrey M

Anxiety treatment
Epilepsy treatment
Sleep disorders treatment
Dependence
Motor impairment
Respiratory depression
Sedation
Tolerance

This review examines the therapeutic potential of positive allosteric modulators of GABA<sub>A</sub> receptors (GABAkines) in treating neurological and psychiatric disorders. While traditional GABAkines like diazepam have safety concerns, newer compounds like neuroactive steroids and imidazodiazepines are being investigated for their potential to provide safer and more effective treatment options for conditions such as epilepsy, anxiety, and depression.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Research Support, U.S. Gov't, Non-P.H.S.Research funding provided by US government agencies other than the Public Health Service.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

8.

Neurosteroid replacement therapy using the allopregnanolone-analogue ganaxolone following preterm birth in male guinea pigs.

Author: ShawJulia C, DysonRebecca M, PalliserHannah K, GrayClint, BerryMary J, HirstJonathan J

Neurodevelopmental impairment
Preterm birth

Premature babies, especially boys, have a higher chance of developing ADHD and learning problems. Ganaxolone treatment after preterm birth could potentially reduce these developmental issues.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

9.

Recent advances in the pharmacotherapy of infantile spasms.

Author: RiikonenRaili

Improved cognitive outcome
Infantile spasms treatment
Side effects

This review examines recent developments in the pharmacotherapy of infantile spasms, focusing on first-line treatments such as ACTH, oral corticosteroids, and vigabatrin, as well as other antiepileptic drugs and novel treatments. The article highlights the importance of early intervention and the potential for preventive treatments.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

10.

Neurosteroids and Seizure Activity.

Author: MiziakBarbara, Chrościńska-KrawczykMagdalena, CzuczwarStanisław J

Anticonvulsant activity
Seizure control
Drug resistance

Neurosteroids positively modulating GABA-A receptors have shown promise as anticonvulsant agents in experimental models and early clinical trials. Ganaxolone, a neurosteroid, demonstrated reduced seizure frequency and mild to moderate adverse effects in a phase 2 trial for patients with intractable partial seizures.

Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

11.

Extrasynaptic γ-aminobutyric acid type A receptor-mediated sex differences in the antiseizure activity of neurosteroids in status epilepticus and complex partial seizures.

Author: ReddyDoodipala Samba, CarverChase Matthew, ClossenBryan, WuXin

This study investigates the potential mechanisms behind sex differences in the antiseizure activity of neurosteroids. The researchers examined differences in the expression and function of extrasynaptic δ-subunit GABA-A receptors, a possible explanation for the observed sex-specific differences in seizure susceptibility and neurosteroid effectiveness.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.

Language : English

12.

Shank3 deletion in PV neurons is associated with abnormal behaviors and neuronal functions that are rescued by increasing GABAergic signaling.

Author: PaganoJessica, LandiSilvia, StefanoniAlessia, NardiGabriele, AlbanesiMarica, BauerHelen F, PracucciEnrico, SchönMichael, RattoGian Michele, BoeckersTobias M, SalaCarlo, VerpelliChiara

Phelan-McDermid syndrome treatment

Phelan-McDermid syndrome (PMS) is a neurodevelopmental disorder caused by haploinsufficiency of the SHANK3 gene. The study hypothesized that SHANK3 haploinsufficiency might lead to alterations in the inhibitory system, which could be linked to the excitatory/inhibitory imbalance observed in models of autism spectrum disorder (ASD).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

15.

Reversal of behavioral effects of pentylenetetrazol by the neuroactive steroid ganaxolone.

Author: BeekmanM, UngardJ T, GasiorM, CarterR B, DijkstraD, GoldbergS R, WitkinJ M

Anticonvulsant effect
Anxiety reduction

Ganaxolone, a neuroactive steroid, effectively reversed the behavioral changes induced by pentylenetetrazol (PTZ), a convulsant, and may have potential therapeutic applications in epilepsy and other conditions.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Research Support, U.S. Gov't, P.H.S.Research funding provided by the Public Health Service (PHS).

Language : English

16.

Isobolographic analysis of adjunct antiseizure activity of the FDA-approved cannabidiol with neurosteroids and benzodiazepines in adult refractory focal onset epilepsy.

Author: GolubVictoria, RamakrishnanSreevidhya, ReddyDoodipala Samba

Epilepsy treatment
Seizure prevention

This study evaluated the adjunct antiseizure activity of a clinical CBD product in an adult 6-Hz model of focal refractory epilepsy. CBD was found to be effective both as a monotherapy and as an adjunct therapy in combination with GABAergic ASMs.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, U.S. Gov't, Non-P.H.S.Research funding provided by US government agencies other than the Public Health Service.

Language : English

17.

A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome.

Author: LigsayAndrew, Van DijckAnke, NguyenDanh V, LozanoReymundo, ChenYanjun, BickelErika S, HesslDavid, SchneiderAndrea, AngkustsiriKathleen, TassoneFlora, CeulemansBerten, KooyR Frank, HagermanRandi J

FXS symptom improvement

Ganaxolone, a GABA<sub>A</sub> receptor modulator, is being investigated as a potential treatment for fragile X syndrome (FXS). This review explores the rationale for using ganaxolone in FXS, highlighting the role of GABA system deficits in the syndrome's pathophysiology.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

19.

[GABAergic approach of postpartum depression: A translational review of literature].

Author: VerbeJ, DubertretC, El-HageW, Bonnet-BrilhaultF, DuriezP

PPD treatment

Postpartum depression (PPD) affects 10-15% of mothers after giving birth, impacting their bond with their baby and the health of both mother and child. The causes of PPD may be different from other types of depression. Current treatments focus on managing symptoms, but research is exploring how hormones and the brain's GABA system may be involved in PPD.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : French

21.

Experimental GABA A Receptor Agonists and Allosteric Modulators for the Treatment of Focal Epilepsy.

Author: JankovićSlobodan M, DješevićMiralem, JankovićSnežana V

Focal epilepsy treatment
Desensitization
Physical dependence
Tolerance

GABA A receptors are diverse and play a key role in epilepsy. New anticonvulsants targeting specific GABA A receptor subtypes are being developed to address drug-resistant focal epilepsy. While promising, these drugs are still in early clinical trials and will take time to reach the market.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

23.

Expanding the Treatment Landscape for Lennox-Gastaut Syndrome: Current and Future Strategies.

Author: StrzelczykAdam, Schubert-BastSusanne

LGS treatment

This review provides a comprehensive overview of current and future options for treating Lennox-Gastaut syndrome (LGS), a severe childhood-onset epileptic encephalopathy. The review covers pharmacologic and non-pharmacologic treatments, highlighting the need for tailored treatment approaches for this complex syndrome.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

24.

Anticonvulsant doses of ganaxolone do not compromise motor performance in immature rats.

Author: MaresPavel, StehlíkováMariana

Anticonvulsant effect
Motor function compromise

Ganaxolone, a neuroactive steroid, showed anticonvulsant effects in rats, specifically suppressing epileptic afterdischarges (ADs) in 25-day-old rats and delaying their onset in 12-day-old rats.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

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