大医生 | 樊东升：与万名“渐冻人”并肩寻药

Core Insights - The article highlights the ongoing efforts and advancements in the research and treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease or "渐冻症" in Chinese, emphasizing the role of Dr. Fan Dongsheng and his team at Peking University Third Hospital in leading clinical trials and patient care [2][3][4]. Group 1: Research and Clinical Trials - Dr. Fan Dongsheng has been instrumental in conducting clinical trials for new ALS drugs, with recent approvals from the National Medical Products Administration for trials led by his team [5][6]. - There are currently nearly 10 ALS drug clinical trials underway at Peking University Third Hospital, including both international and domestic drug development efforts [8][9]. - The research focuses on various treatment modalities, including traditional Chinese medicine, small molecule drugs, gene therapy, and stem cell therapy [8][9]. Group 2: Patient Engagement and Support - Dr. Fan emphasizes the importance of providing hope and support to ALS patients, many of whom seek participation in clinical trials as a means of gaining access to new treatments [11][12]. - The establishment of a patient database and a new data platform has significantly improved the speed and efficiency of patient recruitment for clinical trials, allowing for real-time updates on patient conditions [19][20]. - Patients are encouraged to participate in trials despite the uncertainty of outcomes, as they receive close medical management during the process [11][12]. Group 3: Challenges and Opportunities - The article discusses the historical challenges in ALS drug development, noting that only a few drugs have been successfully developed over the past decades due to the complexity of the disease [9][10]. - However, recent policy changes and increased attention from the Chinese government towards rare diseases are creating a more favorable environment for ALS drug research and development [9][10]. - The large population of ALS patients in China provides a unique advantage for clinical trials, as it allows for easier recruitment compared to Western countries where patient numbers are lower [10][11].