相关实验视频

Updated: Jun 26, 2025

Establishment and Characterization of Patient-Derived Xenograft Models of Anaplastic Thyroid Carcinoma and Head and Neck Squamous Cell Carcinoma

Establishment and Characterization of Patient-Derived Xenograft Models of Anaplastic Thyroid Carcinoma and Head and Neck Squamous Cell Carcinoma

Published on: June 2, 2023

波纳替尼的历史发展,现状和未来研究的回顾

Hagop M Kantarjian¹, Helen T Chifotides¹, Fadi G Haddad¹

¹Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.

American journal of hematology

|May 10, 2024

概括

波纳替尼 (ponatinib) 是一种强大的第三代氨酸激酶抑制剂 (TKI),有效治疗费城染色体阳性白血病,包括耐药突变. 基于反应的剂量提高了耐受性,并降低了慢性髓性白血病 (CML) 的毒性.

科学领域:

血液学血液学血液学
在瘤学瘤学.
药理学药理学是指药理学的学科.

背景情况:

费城染色体阳性 (Ph+) 白血病,如慢性髓性白血病 (CML) 和急性淋巴细胞白血病 (ALL),是由BCR::ABL1融合基因驱动的.
对第一代和第二代氨酸激酶抑制剂 (TKI) 的耐药性仍然是一个重要的临床挑战,特别是像T315I这样的突变.
第三代TKI为耐药或不耐受Ph+白血病患者提供了新的治疗选择.

研究的目的:

为了审查ponatinib的疗效和安全性,第三代BCR::ABL1 TKI.
突出波纳替尼在治疗T315I突变的Ph+白血病和其他耐药CML和Ph+ALL病例中的作用.
讨论基于响应的剂量和未来的方向对白血病治疗中ponatinib的影响.

主要方法:

对ponatinib的临床试验和研究的文献综述.
在各种BCR::ABL1-阳性白血病环境中分析ponatinib的疗效.
对安全性概况和毒性管理的评估,包括基于反应的剂量策略.

主要成果:

波纳替尼 (Ponatinib) 对Ph+白血病具有很高的疗效,包括T315I突变的CML和Ph+ALL.
在慢性阶段CML (CP-CML) 中基于反应的剂量改善了治疗耐受性和降低了心血管风险.
基于ponatinib的治疗在前线Ph+ALL中显示出优异的结果,将其确立为新的护理标准.

结论:

波纳替尼是T315I突变的CML和Ph+ALL患者以及对先前TKI耐药患者的关键治疗选择.
优化剂量策略,如基于反应的剂量,提高了ponatinib的耐受性和安全性.
第三代TKI和组合疗法的持续发展有望在治疗Ph+白血病方面取得进一步的进展.

更多相关视频

Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3

Scaled-Up Preparation of an Intermediate of Upatinib, ACT051-3

Published on: April 7, 2023

Assessing Cellular Target Engagement by SHP2 PTPN11 Phosphatase Inhibitors

Assessing Cellular Target Engagement by SHP2 PTPN11 Phosphatase Inhibitors

Published on: July 17, 2020

相关概念视频

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...

Targeted Cancer Therapies

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...

Preclinical Development: Overview

Preclinical Development: Overview

Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...

Clinical Trials: Overview

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...

Inhibition of Cdk Activity

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...

Drug Discovery: Overview

Drug Discovery: Overview

Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...