小児脳腫瘍の治療薬開発には大きな進歩があります。 小児脳腫瘍は、小児においてより一般的な悪性疾患です。 最近の研究では、新しいカクテル薬が一般的な小児脳腫瘍を治療できることが判明しました。
Cancer Cell” magazine recently announced that in the UK, about 400 children develop brain 腫瘍 each year, of which the prevalence of boys is slightly higher than that of girls.
Are we able to take advantage of the results of tumor gene testing and tailor-made treatments, a strategy often referred to as personalized medicine? This treatment strategy can produce very good results for patients with brain tumors.
Neural myeloblastoma (medulloblastoma) is one of the most common 悪性腫瘍 of the cerebellum. This 脳腫瘍 grows rapidly and most often occurs in children around the age of 5. 治療の選択肢 include surgery, radiation, and chemotherapy. Although great progress has been made in treatment methods and techniques, the success rate of treating myeloblastoma still lags far behind other children’s malignancies. In particular, myeloblastoma is a highly aggressive malignancy. Only 40% of patients with 髄芽腫 survive, compared with other tumors of a less severe type-with a survival rate of more than 80%.
Researchers in the United States have discovered a new combination therapy for the treatment of highly aggressive 神経芽細胞腫. In laboratory tests, the drug killed 癌 cells without any toxicity to normal cells, and researchers hope to conduct clinical trials of the drug. Robert Wechsler-Reya, an adjunct professor at the Sanford Burnham Prebys Medical Institute, said: “Our goal is to confirm that the drug has low toxicity properties. Because doctors and patients in this case urgently require new clinical treatment options, we will soon apply the drug from the laboratory to clinical treatment.
他の薬剤と組み合わせることにより、腫瘍を阻害する新しい化合物がinvitroおよびinvivoでスクリーニングされます。
臨床試験 for neuroblastoma are often very challenging because of the limited number of patients. In addition, coupled with the variability of the disease, most treatments are only effective for one subtype of patient. Understanding which patients will respond to this treatment is one of the main goals of the trial.
「腫瘍遺伝子に基づいたオーダーメイドの治療法（一般に個別治療と呼ばれる戦略）を開発できれば、特定の腫瘍の患者に大きな福音をもたらす可能性があります。」
神経芽細胞腫には40つの異なるタイプがあり、腫瘍の80番目のグループの患者の予後は最悪です。患者のXNUMX％だけが長期生存します。 対照的に、他の神経芽細胞腫の長期生存は比較的楽観的であり、患者の約XNUMX％が長期生存することができます。
神経芽細胞腫の患者の第XNUMXのグループのほとんどは、制御不能な細胞分裂と腫瘍の形成の原因であるMYC癌遺伝子の高発現を示しています。
There was a study on mice with a third type of neural tube 細胞腫瘍 that showed histone deacetylase inhibitors (HDACIs) and phosphatidylinositol 3-kinase inhibitors (PI3KIs) might stop mice and people from making neurotubular glioblastomas without doing too much damage to normal cells.
We found several histone deacetylase inhibitors that can kill MYC oncogene-activated neural tube cell tumors without harming normal cell agents (HDACIs),” said Pei Yanxin, an assistant professor at the National Children’s 医療センター ワシントンDCで
The most effective of these compounds is panobinostat, which has entered clinical trials in other がんの種類, but has not yet been tested on neuroblastoma.” Dr. Kun-Wei, a postdoctoral researcher at Stanford University, added: “Several other studies have revealed that the mechanism of action of panobinostat is to promote the activation of the FOXO1 gene that can interfere with the oncogenes of MYC.
Phosphatidylinositol 3-kinase inhibitors (PI3KIs) are also thought to have the effect of activating the FOXO1 gene. We hypothesized that panobinostat and phosphatidylinositol 3-kinase inhibitors (PI3KIs) could work together to block がん細胞 生存。
“It is true that the combined treatment of these two drugs can significantly increase the survival of patients with tumors carrying the MYC gene compared to using a single drug alone.”
