Related Experiment Video

Updated: Dec 27, 2025

A Protocol for Rapid Post-mortem Cell Culture of Diffuse Intrinsic Pontine Glioma DIPG
08:46

A Protocol for Rapid Post-mortem Cell Culture of Diffuse Intrinsic Pontine Glioma DIPG

Published on: March 7, 2017

17.4K

ALK2: A Therapeutic Target for Fibrodysplasia Ossificans Progressiva and Diffuse Intrinsic Pontine Glioma

Katsuhiko Sekimata1, Tomohiro Sato2, Naoki Sakai3

  • 1Drug Discovery Chemistry Platform Unit, RIKEN Center for Sustainable Resource Science.

Chemical & Pharmaceutical Bulletin
|March 3, 2020
PubMed

Insights

Fibrodysplasia ossificans progressiva (FOP) and diffuse intrinsic pontine glioma (DIPG) are rare childhood diseases lacking effective treatments. Targeting abnormal Activin receptor-like kinase-2 (ALK2) signaling offers a promising therapeutic strategy for both conditions.

Area of Science:

  • Biochemistry
  • Genetics
  • Pediatric Oncology

Background:

  • Fibrodysplasia ossificans progressiva (FOP) and diffuse intrinsic pontine glioma (DIPG) are devastating childhood diseases with no current cures.
  • Both conditions are characterized by severely reduced life expectancy.
  • Activin receptor-like kinase-2 (ALK2), encoded by the ACVR1 gene, is crucial for normal development but implicated in these diseases.

Purpose of the Study:

  • To describe FOP and DIPG.
  • To elucidate the role of ALK2 signaling in FOP and DIPG pathogenesis.
  • To highlight ALK2 inhibitors as potential therapeutic targets.

Main Methods:

  • Review of current literature on FOP, DIPG, and ALK2 signaling.
  • Analysis of the molecular mechanisms linking ALK2 mutations to disease.
  • Identification of ALK2 inhibitors in development.

Main Results:

  • Constitutively active ALK2 mutants cause FOP.
  • Abnormal BMP signaling mediated by ALK2 is involved in DIPG tumorigenesis.
  • ALK2 has emerged as a key therapeutic target for both FOP and DIPG.

Conclusions:

  • Targeting ALK2 signaling presents a novel therapeutic avenue for FOP and DIPG.
  • Development of ALK2 inhibitors offers hope for patients with these currently incurable diseases.
  • Further research into ALK2 inhibitors is warranted for FOP and DIPG treatment.

Keywords:
activin receptor-like kinase-2 (ALK2)diffuse intrinsic pontine glioma (DIPG)drug discoveryfibrodysplasia ossificans progressive (FOP)rare disease

More Related Videos

Study of the Functions and Activities of Neuronal K-Cl Co-Transporter KCC2 Using Western Blotting10:08

Study of the Functions and Activities of Neuronal K-Cl Co-Transporter KCC2 Using Western Blotting

Published on: December 9, 2022

2.5K
Assessing Cellular Target Engagement by SHP2 PTPN11 Phosphatase Inhibitors08:45

Assessing Cellular Target Engagement by SHP2 PTPN11 Phosphatase Inhibitors

Published on: July 17, 2020

6.5K

Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
8.6K
Inhibition of Cdk Activity02:34

Inhibition of Cdk Activity

The orderly progression of the cell cycle depends on the activation of Cdk protein by binding to its cyclin partner. However, the cell cycle must be restricted when undergoing abnormal changes. Most cancers correlate to the deregulated cell cycle, and since Cdks are a central component of the cell cycle, Cdk inhibitors are extensively studied to develop anticancer agents. For instance, cyclin D associates with several Cdks, such as Cdk 4/6, to form an active complex. The cyclin D-Cdk4/6 complex...
5.5K
Electron Transport Chain: Complex I and II01:46

Electron Transport Chain: Complex I and II

The mitochondrial electron transport chain (ETC) is the main energy generation system in the eukaryotic cells. However, mitochondria also produce cytotoxic reactive oxygen species (ROS) due to the large electron flow during oxidative phosphorylation. While Complex I is one of the primary sources of superoxide radicals, ROS production by Complex II is uncommon and may only be observed in cancer cells with mutated complexes.
ROS generation is regulated and maintained at moderate levels necessary...
18.2K
Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
873
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.6K
Drugs that Stabilize Microtubules01:15

Drugs that Stabilize Microtubules

Microtubules are dynamic structures that undergo cycles of catastrophe and rescue. The microtubules play a central role in cell division by forming the spindle apparatus for segregating the chromosomes. This makes them ideal targets for regulating dividing cells in tumors and malignant cancer cells. Microtubule stabilizing drugs help stabilize the microtubule formation and promote its polymerization. Paclitaxel was the first microtubule stabilizing agent used as anticancer drug in chemotherapy...
2.5K