Drug Targeting Common Causative Genes in Different Cancers: Drug for Practical Application

Recently NHK News on-line reported the following:

異なるがんで共通する原因遺伝子標的に 治療薬実用化

2026年6月22日17:35

医療・健康

異なる種類のがんで共通する特定の遺伝子変異を標的とすることでさまざまな希少がんや小児がんなどにも効果が期待できる薬を開発し、国の承認を得たと国立がん研究センターなどのグループが発表しました。患者数が少ないがんの患者の新たな治療につながるとしています。

がんの治療薬の開発はがんの種類ごとに行われることが多く、患者数が少ない希少がんや小児がんは薬の開発のための治験に参加する患者を集めるのが難しいことなどが課題となっています。

国立がん研究センターなどのグループは、希少がんや小児がんの中には、一般的ながんと共通する遺伝子変異が原因とみられる患者がいることに着目し、このうち肺がんなどの治療に使われる「ALK（アルク）」という遺伝子の変異を標的とする薬が、ほかのがんにも効果があるか治験を行いました。

その結果、骨や筋肉にできる希少がんや小児がんなど、さまざまながんに効果があることが確認され、5月、小児がんを含めた固形がんに対する治療薬として国の承認を得たということです。

国立がん研究センターの間野博行 理事長は「臓器ごとではなく、がんの原因となる共通の遺伝子に着目して新薬を開発する手法は今後さらに増えていくはずだ。患者数が少ないがんでもより早く薬を開発できるような体制を確立したい」と話していました。

Translation

Drug Targeting Common Causative Genes in Different Cancers: Drug for Practical Application

A group including the National Cancer Center announced that they we’re developing drugs that were expected to be effective against various rare cancers and pediatric cancers by targeting specific gene mutations common to different types of cancer. They had received national approval for it. This could lead to new treatments for patients with rare cancers.

The development of cancer drugs was often conducted separately for each type of cancer, and for rare cancers and childhood cancers which affected a small number of patients, recruiting patients for clinical trials had been a challenge.

The group including the National Cancer Center focused on the fact that some patients with rare cancers and childhood cancers appeared to be caused by gene mutations common to common cancers. They conducted clinical trials to determine if a drug targeting the "ALK" gene mutation which was used to treat lung cancer could also be effective against other cancers.

As a result, it was confirmed that the drug was effective against various cancers, including rare cancers of bone and muscle, and childhood cancers. In May, it had received national approval as a therapeutic drug for solid tumors, including pediatric cancer.

Dr. Hiroyuki Mano (間野博行), president of the National Cancer Center, said, "We expect to see an increase in the development of new drugs that focus on common genes that cause cancer, instead of on each individual organ. We want to establish a system that will allow us to develop drugs more quickly, even for cancers with a small number of patients."

So, Japan has announced that they are developing drugs that are expected to be effective against various rare cancers and pediatric cancers by targeting specific gene mutations common to different types of cancer. Apparently, this new approach will develop drugs more quickly, even for cancers with a small number of patients.