Sohonos (palovarotene)
Research Synopsis
Sohonos (palovarotene)
Palovarotene, a novel retinoic acid receptor gamma agonist for the treatment of emphysema.
London, UK
2 hours ago
1 Received
- Emphysema is a lung condition marked by the destruction of alveoli, with retinoid signaling playing a potential role in their formation.
- Roche Holding AG is developing palovarotene, a selective retinoic acid receptor gamma agonist, which has shown promise in reversing emphysema symptoms in animal studies.
- Early clinical trials demonstrate that palovarotene is well tolerated by patients and may slow disease progression, with a phase II trial ongoing to evaluate its effectiveness further.
$100 - $150
Hourly Rate
Retinoid treatment of Emphysema in Patients on the Alpha-1 International Registry. The REPAIR study: study design, methodology and quality control of study assessments.
London, UK
2 hours ago
1 Received
- Emphysema is a lung disease marked by damaged alveolar walls and larger airspaces, leading to decreased gas exchange, loss of elasticity, and lung hyperinflation.
- The REPAIR study investigates Palovarotene, an experimental drug for treating emphysema linked to alpha-1-antitrypsin deficiency, which can also apply to other smoke-induced emphysema cases.
- The study emphasized rigorous quality control through standardized procedures for accurate lung function measurements, highlighting that CT lung density was the most sensitive indicator of disease progression followed by other lung function tests.
$100 - $150
Hourly Rate
The COPD pipeline VII.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
The COPD Pipeline XIII.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Randomised controlled trial for emphysema with a selective agonist of the γ-type retinoic acid receptor.
London, UK
2 hours ago
1 Received
- Palovarotene is an oral retinoid agonist that showed promising results in animal models for treating emphysema by reducing inflammation and aiding repair.
- A study called REPAIR tested palovarotene's safety and effectiveness over one year on 262 patients with severe α(1)-antitrypsin deficiency and emphysema, comparing it to a placebo.
- Results indicated that, although palovarotene was well tolerated, it did not provide significant benefits in improving lung density or functional outcomes compared to the placebo group.
$100 - $150
Hourly Rate
Effectiveness and mode of action of a combination therapy for heterotopic ossification with a retinoid agonist and an anti-inflammatory agent.
London, UK
2 hours ago
1 Received
- - Heterotopic ossification (HO) is the abnormal formation of bone and cartilage that can occur after severe injuries or surgeries, with a genetic variant seen in Fibrodysplasia Ossificans Progressiva (FOP).
- - Recent studies found that treatments with corticosteroids like prednisone and dexamethasone, along with the retinoic acid receptor γ agonist Palovarotene, significantly inhibit HO in mouse models, each acting on different stages of the condition.
- - The combination of these drugs showed similar effectiveness in reducing bone formation without interfering with each other, while both also decreased inflammatory cell activity at the site, indicating they work through both shared and unique mechanisms.
$100 - $150
Hourly Rate
Palovarotene Inhibits Heterotopic Ossification and Maintains Limb Mobility and Growth in Mice With the Human ACVR1(R206H) Fibrodysplasia Ossificans Progressiva (FOP) Mutation.
London, UK
2 hours ago
1 Received
- * Most FOP patients have a mutation in the ACVR1 gene, which triggers abnormal bone and cartilage growth.
- * Palovarotene, a retinoic acid receptor γ agonist, has been shown to effectively prevent both spontaneous and injury-induced heterotopic ossification in mice with the human-like mutation, while also promoting normal bone growth and overall mobility.
$100 - $150
Hourly Rate
Targeted stimulation of retinoic acid receptor-γ mitigates the formation of heterotopic ossification in an established blast-related traumatic injury model.
London, UK
2 hours ago
1 Received
- * The study tested the retinoic acid receptor γ agonist Palovarotene in a rat model of trauma-induced HO created by blast injuries, revealing that it significantly reduced HO by 50-60% and altered the development of ectopic bone and related tissues.
- * Although Palovarotene effectively inhibited HO, it also showed moderate negative effects on wound healing, indicating the need for careful consideration of dosing and timing in potential treatments.
$100 - $150
Hourly Rate
Cartilage-derived retinoic acid-sensitive protein (CD-RAP): A stage-specific biomarker of heterotopic endochondral ossification (HEO) in fibrodysplasia ossificans progressiva (FOP).
London, UK
2 hours ago
1 Received
- The study investigates the role of cartilage-derived retinoic acid-sensitive protein (CD-RAP) as a potential plasma biomarker for the pre-osseous cartilaginous stage of heterotopic endochondral ossification (HEO) in fibrodysplasia ossificans progressiva (FOP).
- In a transgenic mouse model of FOP, CD-RAP levels peaked during chondrogenesis and were reduced by palovarotene, a drug being evaluated to treat FOP.
- In human patients, CD-RAP showed a significant decrease six months after flare-ups, suggesting its potential use as a specific biomarker for monitoring HEO in FOP cases.
$100 - $150
Hourly Rate
Palovarotene Inhibits Osteochondroma Formation in a Mouse Model of Multiple Hereditary Exostoses.
London, UK
2 hours ago
1 Received
- Multiple hereditary exostoses (MHE), also known as multiple osteochondromas, is a genetic disorder leading to multiple bone tumors due to mutations in the EXT1 or EXT2 gene, affecting cartilage growth.
- Research shows that enhanced bone morphogenetic protein (BMP) signaling, caused by reduced heparan sulfate expression, contributes to the formation of these tumors in MHE.
- Palovarotene (PVO), a drug candidate previously tested for another bone disorder, significantly reduced osteochondroma formation in mouse models of MHE, suggesting its potential as a therapy for this condition.
$100 - $150
Hourly Rate
Retinoid roles and action in skeletal development and growth provide the rationale for an ongoing heterotopic ossification prevention trial.
London, UK
2 hours ago
1 Received
- Most skeletal elements develop through a process called endochondral ossification, which involves the transformation of cartilage into bone and is crucial for both embryonic development and healing fractures.
- *Heterotopic ossification (HO) can occur abnormally in conditions such as traumatic injuries and genetic disorders like Fibrodysplasia Ossificans Progressiva (FOP), making it a focus for treatment development.
- *Recent studies on synthetic retinoid agonists, particularly a drug called Palovarotene, show promise in preventing or treating HO and are currently undergoing phase 2 clinical trials, with early results appearing positive.
$100 - $150
Hourly Rate
Palovarotene inhibits connective tissue progenitor cell proliferation in a rat model of combat-related heterotopic ossification.
London, UK
2 hours ago
1 Received
- Heterotopic ossification (HO) is a condition that commonly affects wounded service members, especially after severe injuries like blasts and amputations, and there's currently no effective preventative treatment.
- The study examined the drug Palovarotene in a rodent model, finding it reduced inflammation and bone formation related to HO, markedly decreasing specific inflammatory cytokines and progenitor cell activity.
- Additionally, Raman spectroscopy proved effective at detecting early signs of HO formation before visible changes occur, suggesting potential for earlier diagnosis and intervention.
$100 - $150
Hourly Rate
Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity.
London, UK
2 hours ago
1 Received
- * Current treatments, like the drug palovarotene, show promise in reducing the growth of problematic cells involved in this condition but are less effective compared to another treatment that blocks activin A.
- * Even though palovarotene can reduce abnormal bone growth in young mice, it may also cause harmful side effects like joint overgrowth, emphasizing the difficulty of finding effective treatments before the skeletal system fully matures.
$100 - $150
Hourly Rate
Response to comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
London, UK
2 hours ago
1 Received
- The authors address concerns raised by Pacifici and Shore regarding their previous research.
- They studied the drug palovarotene and its severe side effects in a mouse model related to fibrodysplasia ossificans progressiva.
- The response clarifies and defends their findings from the earlier paper published in 2018.
$100 - $150
Hourly Rate
Comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
London, UK
2 hours ago
1 Received
- The authors express their concerns about a study published by Lees-Shepard et al. in 2018.
- They likely find issues related to the study's methodology, conclusions, or implications.
- The communication aims to address these concerns to ensure accuracy and reliability in research.
$100 - $150
Hourly Rate
Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva.
London, UK
2 hours ago
1 Received
- * Recent research has shown that the ACVR1 receptor is overly active when exposed to activin A, contributing to the disease’s symptoms of heterotopic ossification.
- * A new method using phosphorodiamidate morpholino oligomers (PMOs) aims to reduce ACVR1 expression in cells from FOP patients, offering a potential therapy and a way to target other similar genetic disorders.
$100 - $150
Hourly Rate
Current and Emerging Therapeutic Options for the Management of Rare Skeletal Diseases.
London, UK
2 hours ago
1 Received
- Increasing knowledge about rare diseases has led to the development of new treatments targeting specific genetic issues and biological pathways associated with these conditions.
- Several drugs have been approved, such as asfotase alfa and burosumab, while others are in advanced clinical trials, indicating a promising future for therapy in these areas.
- It is recommended that patients receive care at specialized centers to ensure safety and to allow for data collection that can improve treatment strategies, which could also inform family decisions during pregnancy regarding skeletal diseases.
$100 - $150
Hourly Rate
Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva.
London, UK
2 hours ago
1 Received
- - Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder that causes bone to form in muscles and soft tissues, resulting in significant disabilities and often leading to wheelchair dependency by the age of 30.
- - The condition is caused by a mutation in the ACVR1 gene, which disrupts normal signaling in the body and causes unwanted bone growth outside of the skeleton.
- - Although there's no cure for FOP, recent advancements in treatment options, including drugs like palovarotene, REGN 2477, and rapamycin, are currently being studied in clinical trials, offering hope for future management strategies.
$100 - $150
Hourly Rate
The evolving therapeutic landscape of genetic skeletal disorders.
London, UK
2 hours ago
1 Received
- A review of over 140 articles and 80 trials identified more than 20 personalized therapies and novel disease-modifying treatments for over 25 genetic skeletal disorders.
- The discussion emphasizes innovative drug development methods, the challenges of rare disease treatments, and the promise of improved understanding of bone diseases and genetic pathways.
$100 - $150
Hourly Rate
Inflammation in Fibrodysplasia Ossificans Progressiva and Other Forms of Heterotopic Ossification.
London, UK
2 hours ago
1 Received
- The review explores the connection between inflammation and heterotopic ossification (HO), highlighting its relevance in various clinical conditions, including fibrodysplasia ossificans progressiva and traumatic HO.
- Recent studies reveal that both genetic and acquired forms of HO involve similar inflammatory cell types and signaling pathways, such as those associated with macrophages, mast cells, and adaptive immune cells.
- Identifying common inflammatory mediators offers potential therapeutic targets for preventing HO, with future research likely aimed at discovering new targets and testing combination therapies.
$100 - $150
Hourly Rate