Our second-most-read story of the year was about researchers that leveraged an AI-driven drug discovery process in combination with innovative disease modeling to identify a drug known as vorinostat as a promising treatment for Rett syndrome.
Forging a novel therapeutic path for patients with Rett Syndrome using AI
By Benjamin Boettner (BOSTON) — Rett syndrome is a devastating, rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it, and much fewer boys. It is…
buff.ly
December 29, 2025 at 3:15 PM
Our second-most-read story of the year was about researchers that leveraged an AI-driven drug discovery process in combination with innovative disease modeling to identify a drug known as vorinostat as a promising treatment for Rett syndrome.
Get caught up on new research in Arthritis & Rheumatology →
Colchicine initiation after gout dx assoc. with modestly lower risk of total joint arthroplasty doi.org/10.1002/art....
Vorinostat suppresses IFN-I production in an SLE model
doi.org/10.1002/art....
↓
Colchicine initiation after gout dx assoc. with modestly lower risk of total joint arthroplasty doi.org/10.1002/art....
Vorinostat suppresses IFN-I production in an SLE model
doi.org/10.1002/art....
↓
November 22, 2025 at 1:33 PM
Get caught up on new research in Arthritis & Rheumatology →
Colchicine initiation after gout dx assoc. with modestly lower risk of total joint arthroplasty doi.org/10.1002/art....
Vorinostat suppresses IFN-I production in an SLE model
doi.org/10.1002/art....
↓
Colchicine initiation after gout dx assoc. with modestly lower risk of total joint arthroplasty doi.org/10.1002/art....
Vorinostat suppresses IFN-I production in an SLE model
doi.org/10.1002/art....
↓
Vorinostat suppressed IFN-I production and ameliorated disease severity in an SLE mouse model
Arthritis & Rheumatology
doi.org/10.1002/art....
Arthritis & Rheumatology
doi.org/10.1002/art....
November 18, 2025 at 1:51 PM
Vorinostat suppressed IFN-I production and ameliorated disease severity in an SLE mouse model
Arthritis & Rheumatology
doi.org/10.1002/art....
Arthritis & Rheumatology
doi.org/10.1002/art....
This study used high-resolution crystal structures, computational modeling, and thermal shift assays to investigate the off-target binding of the histone deacetylase inhibitor vorinostat (SAHA) to carbonic anhydrase II (CA II) and a CA IX active-site mimic.
September 23, 2025 at 4:21 AM
This study used high-resolution crystal structures, computational modeling, and thermal shift assays to investigate the off-target binding of the histone deacetylase inhibitor vorinostat (SAHA) to carbonic anhydrase II (CA II) and a CA IX active-site mimic.
Vorinostat, a cancer drug, unexpectedly binds to carbonic anhydrase enzymes, potentially contributing to its side effects. This discovery opens new avenues for developing selective carbon...
🧵 Thread below
Full analysis: https://helixbrief.com/article/adf91303-9da1-46f5-9f56-41455ce4aa4c
🧵 Thread below
Full analysis: https://helixbrief.com/article/adf91303-9da1-46f5-9f56-41455ce4aa4c
September 23, 2025 at 4:21 AM
Vorinostat, a cancer drug, unexpectedly binds to carbonic anhydrase enzymes, potentially contributing to its side effects. This discovery opens new avenues for developing selective carbon...
🧵 Thread below
Full analysis: https://helixbrief.com/article/adf91303-9da1-46f5-9f56-41455ce4aa4c
🧵 Thread below
Full analysis: https://helixbrief.com/article/adf91303-9da1-46f5-9f56-41455ce4aa4c
Crystal structures of a clinically used histone deacetylase inhibitor, vorinostat, bound to the previously unidentified off-target proteins carbonic anhydrases II and IX are reported #HDACInhibitors #SAHA #Vorinostat doi.org/10.1107/S205...
September 11, 2025 at 1:31 PM
Crystal structures of a clinically used histone deacetylase inhibitor, vorinostat, bound to the previously unidentified off-target proteins carbonic anhydrases II and IX are reported #HDACInhibitors #SAHA #Vorinostat doi.org/10.1107/S205...
David Margolis, MD discusses his team’s work at UNC looking at combination treatment that includes the cancer drug, vorinostat, and immunotherapy as a potential HIV cure. #IDsky #Medsky
Committed to HIV Cure Therapies Through Novel Research
David Margolis, MD discusses his team’s work at UNC looking at combination treatment that includes the cancer drug, vorinostat, and immunotherapy as a potential HIV cure.
www.contagionlive.com
September 4, 2025 at 9:01 PM
Mitchell C. Gulkis et al.: Off-target binding of the histone deacetylase inhibitor vorinostat to carbonic anhydrase II and IX ##HDACInhibitors##SAHA##Vorinostat@uniofleicester@lundsuni...##IUCrhttps://journals.iucr.org/paper?S2053230X25007447
Off-target binding of the histone deacetylase inhibitor vorinostat to carbonic anhydrase II and IX
We determined high-resolution crystal structures of the histone deacetylase inhibitor vorinostat bound to both human carbonic anhydrase II and a carbonic anhydrase IX active-site mimic. Thermal shift assays using differential scanning fluorimetry showed minimal stabilization of either carbonic anhydrase by vorinostat, in contrast to the potent carbonic anhydrase inhibitor acetazolamide.
journals.iucr.org
August 27, 2025 at 12:00 AM
Mitchell C. Gulkis et al.: Off-target binding of the histone deacetylase inhibitor vorinostat to carbonic anhydrase II and IX ##HDACInhibitors##SAHA##Vorinostat@uniofleicester@lundsuni...##IUCrhttps://journals.iucr.org/paper?S2053230X25007447
🔎 Spotted in @commsmed.nature.com!
Researchers from @wyssinstitute.bsky.social, among others, used DSHB's anti-β-tubulin [E7] mAb in a study on the therapeutic use of vorinostat for Rett Syndrome identified by AI.
📄 tinyurl.com/8ncmrf86
🧫 dshb.biology.uiowa.edu/E7_2
Researchers from @wyssinstitute.bsky.social, among others, used DSHB's anti-β-tubulin [E7] mAb in a study on the therapeutic use of vorinostat for Rett Syndrome identified by AI.
📄 tinyurl.com/8ncmrf86
🧫 dshb.biology.uiowa.edu/E7_2
July 9, 2025 at 8:57 PM
🔎 Spotted in @commsmed.nature.com!
Researchers from @wyssinstitute.bsky.social, among others, used DSHB's anti-β-tubulin [E7] mAb in a study on the therapeutic use of vorinostat for Rett Syndrome identified by AI.
📄 tinyurl.com/8ncmrf86
🧫 dshb.biology.uiowa.edu/E7_2
Researchers from @wyssinstitute.bsky.social, among others, used DSHB's anti-β-tubulin [E7] mAb in a study on the therapeutic use of vorinostat for Rett Syndrome identified by AI.
📄 tinyurl.com/8ncmrf86
🧫 dshb.biology.uiowa.edu/E7_2
New paper out, amazing team at Wyss Institute!
www.nature.com/articles/s43...
"AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models"
www.nature.com/articles/s43...
"AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models"
AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models - Communications Medicine
Novak, Lin et al. use AI computational drug prediction and CRISPR-enabled in vivo disease modeling to identify therapeutic strategies for the treatment of Rett syndrome. Findings reveal the therapeuti...
www.nature.com
July 5, 2025 at 2:58 PM
New paper out, amazing team at Wyss Institute!
www.nature.com/articles/s43...
"AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models"
www.nature.com/articles/s43...
"AI-enabled drug prediction and gene network analysis reveal therapeutic use of vorinostat for Rett Syndrome in preclinical models"
An AI-driven approach has identified vorinostat as a potential therapy for Rett syndrome, showing promising effects across multiple organ systems in preclinical models. doi.org/g9r4d6
AI-guided drug discovery forges a novel therapeutic path for patients with Rett syndrome
Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys.
medicalxpress.com
July 2, 2025 at 4:32 PM
An AI-driven approach has identified vorinostat as a potential therapy for Rett syndrome, showing promising effects across multiple organ systems in preclinical models. doi.org/g9r4d6
Wyss researchers identified a drug known as vorinostat as a promising treatment for Rett syndrome, demonstrating disease-modifying abilities across multiple neuronal and non-neuronal tissues in preclinical models. This work was published in @commsmed.nature.com. #RettSyndrome #RareDisease
Forging a novel therapeutic path for patients with Rett Syndrome using AI
By Benjamin Boettner (BOSTON) — Rett syndrome is a devastating, rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it, and much fewer boys. It is cau...
wyss.harvard.edu
July 2, 2025 at 3:15 PM
Wyss researchers identified a drug known as vorinostat as a promising treatment for Rett syndrome, demonstrating disease-modifying abilities across multiple neuronal and non-neuronal tissues in preclinical models. This work was published in @commsmed.nature.com. #RettSyndrome #RareDisease
Here's the trade-off ⚖️: The subclones that respond most strongly to 🦠S. aureus toxins—the aggressive ones—are also the most intrinsically sensitive to 💊anti-cancer drugs when the stimuli are removed.
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
June 19, 2025 at 11:31 AM
Here's the trade-off ⚖️: The subclones that respond most strongly to 🦠S. aureus toxins—the aggressive ones—are also the most intrinsically sensitive to 💊anti-cancer drugs when the stimuli are removed.
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
Here's the trade-off ⚖️: The subclones that respond most strongly to 🦠S. aureus toxins—the aggressive ones—are also the most intrinsically sensitive to 💊anti-cancer drugs when the stimuli are removed.
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
June 19, 2025 at 11:17 AM
Here's the trade-off ⚖️: The subclones that respond most strongly to 🦠S. aureus toxins—the aggressive ones—are also the most intrinsically sensitive to 💊anti-cancer drugs when the stimuli are removed.
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
This means reducing inflammation could unmask therapeutic vulnerabilities in aggressive clones. 6/
Original: Drs. Centeno, Apkarian et al. (@nufeinbergmed.bsky.social) found that a single injection of an HDAC inhibitor combo (Vorinostat + HPBCD + PEG) relieves neuropathic pain in rodents
—a low-dose, non-opioid approach, long-acting pain therapy
Full: www.jpain.org/article/S152...
—a low-dose, non-opioid approach, long-acting pain therapy
Full: www.jpain.org/article/S152...
www.jpain.org
June 18, 2025 at 12:31 PM
Original: Drs. Centeno, Apkarian et al. (@nufeinbergmed.bsky.social) found that a single injection of an HDAC inhibitor combo (Vorinostat + HPBCD + PEG) relieves neuropathic pain in rodents
—a low-dose, non-opioid approach, long-acting pain therapy
Full: www.jpain.org/article/S152...
—a low-dose, non-opioid approach, long-acting pain therapy
Full: www.jpain.org/article/S152...
- A viral imprint reflected in molecular signatures of EBV, HIV and HSV
Potential therapeutic strategies (at least in silico):
- HDAC inhibitors (e.g. Entinostat, Vorinostat) — particularly interesting due to their epigenetic mechanisms
3/4
Potential therapeutic strategies (at least in silico):
- HDAC inhibitors (e.g. Entinostat, Vorinostat) — particularly interesting due to their epigenetic mechanisms
3/4
June 5, 2025 at 7:25 PM
- A viral imprint reflected in molecular signatures of EBV, HIV and HSV
Potential therapeutic strategies (at least in silico):
- HDAC inhibitors (e.g. Entinostat, Vorinostat) — particularly interesting due to their epigenetic mechanisms
3/4
Potential therapeutic strategies (at least in silico):
- HDAC inhibitors (e.g. Entinostat, Vorinostat) — particularly interesting due to their epigenetic mechanisms
3/4
- Virusimprint durch molekulare Signaturen von EBV, HIV, HSV
Therapieansätze (zumindest in silico)
- HDAC-Hemmer (z.B. Entinostat, Vorinostat; interessant v. a. weil epigenetischer Ansatz)
- Östrogen-Antagonisten (Fulvestrant)
➡️ Evtl.
3/4
Therapieansätze (zumindest in silico)
- HDAC-Hemmer (z.B. Entinostat, Vorinostat; interessant v. a. weil epigenetischer Ansatz)
- Östrogen-Antagonisten (Fulvestrant)
➡️ Evtl.
3/4
June 5, 2025 at 7:23 PM
- Virusimprint durch molekulare Signaturen von EBV, HIV, HSV
Therapieansätze (zumindest in silico)
- HDAC-Hemmer (z.B. Entinostat, Vorinostat; interessant v. a. weil epigenetischer Ansatz)
- Östrogen-Antagonisten (Fulvestrant)
➡️ Evtl.
3/4
Therapieansätze (zumindest in silico)
- HDAC-Hemmer (z.B. Entinostat, Vorinostat; interessant v. a. weil epigenetischer Ansatz)
- Östrogen-Antagonisten (Fulvestrant)
➡️ Evtl.
3/4
www.nature.com/articles/s41...
"Furthermore, HDAC inhibitors, vorinostat ( #SAHA) and panobinostat ( #LBH589) regulated CRTH2 expression and PGD2 production, and CRTH2 inhibitor AZD1981 and high-concentration PGD2 enhanced their anti-tumor effects in DLBCL."
"Furthermore, HDAC inhibitors, vorinostat ( #SAHA) and panobinostat ( #LBH589) regulated CRTH2 expression and PGD2 production, and CRTH2 inhibitor AZD1981 and high-concentration PGD2 enhanced their anti-tumor effects in DLBCL."
PGD2 displays distinct effects in diffuse large B-cell lymphoma depending on different concentrations - Cell Death Discovery
Cell Death Discovery - PGD2 displays distinct effects in diffuse large B-cell lymphoma depending on different concentrations
www.nature.com
April 22, 2025 at 11:55 PM
www.nature.com/articles/s41...
"Furthermore, HDAC inhibitors, vorinostat ( #SAHA) and panobinostat ( #LBH589) regulated CRTH2 expression and PGD2 production, and CRTH2 inhibitor AZD1981 and high-concentration PGD2 enhanced their anti-tumor effects in DLBCL."
"Furthermore, HDAC inhibitors, vorinostat ( #SAHA) and panobinostat ( #LBH589) regulated CRTH2 expression and PGD2 production, and CRTH2 inhibitor AZD1981 and high-concentration PGD2 enhanced their anti-tumor effects in DLBCL."
KCB's resource, RRID:CVCL_0004, was just reported to be used in "Vorinostat impairs the cancer-driving potential of leukemia-secreted extracellular vesicles". RRIDs like this improve reproducibility in scientific research. #reproducibility #ReproducibleResearch
doi.org
April 14, 2025 at 7:01 AM
KCB's resource, RRID:CVCL_0004, was just reported to be used in "Vorinostat impairs the cancer-driving potential of leukemia-secreted extracellular vesicles". RRIDs like this improve reproducibility in scientific research. #reproducibility #ReproducibleResearch
The #MAVORIC trial led by Dr. Francine Foss demonstrated greater efficacy with mogamulizumab vs. vorinostat in relapsed/refractory patients with #CTCL, including those with more advanced disease.
pubmed.ncbi.nlm.nih.gov/39894454/
@yale-hematology.bsky.social @yaleschoolofmed.bsky.social
pubmed.ncbi.nlm.nih.gov/39894454/
@yale-hematology.bsky.social @yaleschoolofmed.bsky.social
Insights into treatment of patients with mycosis fungoides or Sézary syndrome using mogamulizumab - PubMed
MAVORIC demonstrated greater efficacy with mogamulizumab <i>vs.</i> vorinostat in relapsed/refractory patients with CTCL, including those with more advanced disease. Concomitant steroid use improved O...
pubmed.ncbi.nlm.nih.gov
February 21, 2025 at 7:41 PM
The #MAVORIC trial led by Dr. Francine Foss demonstrated greater efficacy with mogamulizumab vs. vorinostat in relapsed/refractory patients with #CTCL, including those with more advanced disease.
pubmed.ncbi.nlm.nih.gov/39894454/
@yale-hematology.bsky.social @yaleschoolofmed.bsky.social
pubmed.ncbi.nlm.nih.gov/39894454/
@yale-hematology.bsky.social @yaleschoolofmed.bsky.social
タイトル: マンダリンフィッシュ（Siniperca chuatsi）におけるフォトトランスダクションの重要性を示すトランスクリプトーム解析
実験対象: マンダリンフィッシュの幼魚
要点1: マンダリンフィッシュの幼魚がアルテミアを摂食するメカニズムは不明だったが、比較トランスクリプトーム解析により、フォトン伝導シグナル経路の遺伝子発現が重要であることが判明した。
要点2: Vorinostat（SAHA）処理により、マンダリンフィッシュ幼魚がアルテミアを摂取することが促進され、フォトトランスダクション経路の遺伝子発現が増加した。
実験対象: マンダリンフィッシュの幼魚
要点1: マンダリンフィッシュの幼魚がアルテミアを摂食するメカニズムは不明だったが、比較トランスクリプトーム解析により、フォトン伝導シグナル経路の遺伝子発現が重要であることが判明した。
要点2: Vorinostat（SAHA）処理により、マンダリンフィッシュ幼魚がアルテミアを摂取することが促進され、フォトトランスダクション経路の遺伝子発現が増加した。
Transcriptome analysis reveals the importance of phototransduction during the first-feeding in mandarin fish (Siniperca chuatsi).
【Funct Integr Genomics】<AbstractText>The mandarin fish (Siniperca chuatsi), as a typical freshwater carnivorous fish, has high ...
pubmed.ncbi.nlm.nih.gov
October 25, 2024 at 7:49 PM
タイトル: マンダリンフィッシュ（Siniperca chuatsi）におけるフォトトランスダクションの重要性を示すトランスクリプトーム解析
実験対象: マンダリンフィッシュの幼魚
要点1: マンダリンフィッシュの幼魚がアルテミアを摂食するメカニズムは不明だったが、比較トランスクリプトーム解析により、フォトン伝導シグナル経路の遺伝子発現が重要であることが判明した。
要点2: Vorinostat（SAHA）処理により、マンダリンフィッシュ幼魚がアルテミアを摂取することが促進され、フォトトランスダクション経路の遺伝子発現が増加した。
実験対象: マンダリンフィッシュの幼魚
要点1: マンダリンフィッシュの幼魚がアルテミアを摂食するメカニズムは不明だったが、比較トランスクリプトーム解析により、フォトン伝導シグナル経路の遺伝子発現が重要であることが判明した。
要点2: Vorinostat（SAHA）処理により、マンダリンフィッシュ幼魚がアルテミアを摂取することが促進され、フォトトランスダクション経路の遺伝子発現が増加した。
'Algranati, Oren et al. therefore set out to test whether combining an HDACi known as Vorinostat with a drug that blocks MYC activity (Sulfopin) can act as an effective treatment for this cancer (Diffuse midline gliomas).'
elifesciences.org/digests/9625...
elifesciences.org/digests/9625...
Stronger together
Combining two drugs that target separate biological pathways involved in the emergence of a deadly type of brain cancer helps to reduce tumor growth in cancer models.
elifesciences.org
August 8, 2024 at 10:05 AM
'Algranati, Oren et al. therefore set out to test whether combining an HDACi known as Vorinostat with a drug that blocks MYC activity (Sulfopin) can act as an effective treatment for this cancer (Diffuse midline gliomas).'
elifesciences.org/digests/9625...
elifesciences.org/digests/9625...
Die DNA wird wieder enger aufgewickelt und das Gen an dieser Stelle ist stillgelegt.
Vorinostat hemmt die Desacetylierung und führt damit zu einer erhöhten Aktivität vieler Gene. Letztlich kann das zum Absterben der Tumorzellen führen.
Vorinostat hemmt die Desacetylierung und führt damit zu einer erhöhten Aktivität vieler Gene. Letztlich kann das zum Absterben der Tumorzellen führen.
December 10, 2023 at 6:50 PM
Die DNA wird wieder enger aufgewickelt und das Gen an dieser Stelle ist stillgelegt.
Vorinostat hemmt die Desacetylierung und führt damit zu einer erhöhten Aktivität vieler Gene. Letztlich kann das zum Absterben der Tumorzellen führen.
Vorinostat hemmt die Desacetylierung und führt damit zu einer erhöhten Aktivität vieler Gene. Letztlich kann das zum Absterben der Tumorzellen führen.
PharmBlog #Adventskalender Türchen 10: Vorinostat
Vorinostat ist ein Histon-Desacetylase-Hemmstoff, der zur Behandlung von Krebserkrankungen verwendet wird. Es beeinflusst die epigenetische Regulation der Genaktivität.
Vorinostat ist ein Histon-Desacetylase-Hemmstoff, der zur Behandlung von Krebserkrankungen verwendet wird. Es beeinflusst die epigenetische Regulation der Genaktivität.
December 10, 2023 at 6:49 PM
PharmBlog #Adventskalender Türchen 10: Vorinostat
Vorinostat ist ein Histon-Desacetylase-Hemmstoff, der zur Behandlung von Krebserkrankungen verwendet wird. Es beeinflusst die epigenetische Regulation der Genaktivität.
Vorinostat ist ein Histon-Desacetylase-Hemmstoff, der zur Behandlung von Krebserkrankungen verwendet wird. Es beeinflusst die epigenetische Regulation der Genaktivität.