Evidence map›Paper›PMID 38496939›Full record

ArticleACS omega2024

Identification of New Modulators and Inhibitors of Palmitoyl-Protein Thioesterase 1 for CLN1 Batten Disease and Cancer.

Ana C Puhl, Renuka Raman, Tammy M Havener, Eni Minerali, Anthony J Hickey, Sean Ekins

Open access · goldAbstract read
In one paragraph

Article in ACS omega, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.4field-weighted citation impact, top 20% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 6 citations in OpenAlex.

  1. ProteinJournal of pharmaceutical analysis · 2026
    Review
  2. Article
  3. Article
  4. Applications of artificial intelligence in drug discovery for neurological diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 2 institutions in 1 country.

Ana C PuhlCollaborations Pharmaceuticals, Inc., 840 Main Campus Drive, Lab 3510, Raleigh, North Carolina 27606, United States.
Renuka RamanCollaborations Pharmaceuticals, Inc., 840 Main Campus Drive, Lab 3510, Raleigh, North Carolina 27606, United States.
Tammy M HavenerUNC Catalyst for Rare Diseases, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, United States.ORCID https://orcid.org/0000-0002-4613-0498
Eni MineraliCollaborations Pharmaceuticals, Inc., 840 Main Campus Drive, Lab 3510, Raleigh, North Carolina 27606, United States.
Anthony J HickeyUNC Catalyst for Rare Diseases, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, United States.
Sean EkinsCollaborations Pharmaceuticals, Inc., 840 Main Campus Drive, Lab 3510, Raleigh, North Carolina 27606, United States.ORCID https://orcid.org/0000-0002-5691-5790
Collaborations Pharmaceuticals (United States) · USUniversity of North Carolina at Chapel Hill · US

Funding

Centralized assay datasets for modelling support of small drug discovery organizationsR44GM122196 · NIGMS · COLLABORATIONS PHARMACEUTICALS, INC. · PI EKINS, SEAN · 2018 to 2022
$3.3M
Manufacture of an intracerebroventricular Enzyme Replacement Therapy for CLN1 Batten DiseaseR44NS107079 · NINDS · COLLABORATIONS PHARMACEUTICALS, INC. · PI EKINS, SEAN · 2022 to 2023
$3.0M
Enzyme Replacement Therapy For Infantile Onset Neuronal Ceroid LipofuscinosesR43NS107079 · NINDS · COLLABORATIONS PHARMACEUTICALS, INC. · PI COOPER, JONATHAN D, EKINS, SEAN · 2018 to 2019
$412k
MegaPredict for predicting natural product uses and their drug interactionsR43AT010585 · NCCIH · COLLABORATIONS PHARMACEUTICALS, INC. · PI EKINS, SEAN · 2019 to 2020
$225k
NCCIH NIH HHS R43 AT010585NIGMS NIH HHS R44 GM122196NINDS NIH HHS R43 NS107079NINDS NIH HHS R44 NS107079
6 · The paper itself

Abstract

Palmitoyl-protein thioesterase 1 (PPT1) is an understudied enzyme that is gaining attention due to its role in the depalmitoylation of several proteins involved in neurodegenerative diseases and cancer. PPT1 is overexpressed in several cancers, specifically cholangiocarcinoma and esophageal cancers. Inhibitors of PPT1 lead to cell death and have been shown to enhance the killing of tumor cells alongside known chemotherapeutics. PPT1 is hence a viable target for anticancer drug development. Furthermore, mutations in PPT1 cause a lysosomal storage disorder called infantile neuronal ceroid lipofuscinosis (CLN1 disease). Molecules that can inhibit, stabilize, or modulate the activity of this target are needed to address these diseases. We used PPT1 enzymatic assays to identify molecules that were subsequently tested by using differential scanning fluorimetry and microscale thermophoresis. Selected compounds were also tested in neuroblastoma cell lines. The resulting PPT1 screening data was used for building machine learning models to help select additional compounds for testing. We discovered two of the most potent PPT1 inhibitors reported to date, orlistat (IC

Identifiers

PMID38496939
PMCPMC10938339
OpenAlexW4392238435

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.