Targeted therapies

• Pregnant? Please help
• The newest options
  • Pegylated interferon
  • Targeted therapies
  • JAK2 inhibitors
  • Trial basics
• The science
  • JAK2
  • Scientific papers
  • Testing negative for JAK2
• Links
• For professionals

Targeted therapies are new kinds of drugs on the forefront of medicine. With advances in our understanding of genetic mutations, researchers are developing drugs that pinpoint weaknesses in a diseased cell’s biology.

There are new drugs called “small molecule drugs” or “signal transduction inhibitors” that can attach themselves to the problem proteins in our bodies, those that cause uncontrolled growth in the cells. These drugs stabilize the molecules and prevent them from sending growth signals.

Drug trials are now underway in the European Union and in the United States to find safe and effective small molecule drugs to treat patients who carry the JAK2 mutation. Although new therapies could be many years off, with the discovery of JAK2 we now have the tools we need to develop medications that could one day cure MPDs.

Researchers are currently testing “designer drugs” that target the JAK2 mutation. We hope in the future these drugs will control or cure myeloproliferative disorders. Some JAK2 inhibitors are under trial in the UK and are showing effectiveness in treating myelofibrosis.