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Translational medicine opens a gate for drug R&D

time:10/01/2017 page views: resource:BioBAY
After more than 10 discussion sessions on drug discovery, preclinical study and early clinical study, the event finally came to the end, the closing ceremony. 
Dr. Jiang Ningjun, CEO of CSTONE Pharmaceuticals and one of the chairpersons for the event, presided over the closing ceremony. Three guests gave keynote speeches and a number of experts participated in specialized discussion.  

The first speaker is Dr. Elias Zerhouni, president of Global Research & Development of Sanofi. He gave a speech on “New Trends for Future Research and Development”.

▲ Dr. Elias Zerhouni 

According to Dr. Zerhouni, a series of new DNA and antibody technologies have led to dramatic changes in pharmaceutical industry. More new drugs have emerged. The drug researchers and developers are paying more and more attention to pathogenesis. 

Dr. Zerhouni then expounded on translational medicine, bio-medicine and precision medicine. 

Translational medicine is an recently emerging field which focuses on using what is learned in pre-clinical studies to do smarter things in the clinic (from bench to bedside). Dr. Zerhouni demonstrated the process of the development of a new drug, adding that the first-in-human stage is the hardest part, because it is a turning point for transformation from preclinical study to human body study. 

Nowadays, the human beings’ understanding of molecules has been increasingly enhanced. Dr. Zerhouni gave an introduction to the basic gene functions and related molecular signaling. He said an “ideal molecule” should be applicable to more than one target spots so that several diseases can be cured by the same drug.

Dr. Zerhouni mentioned Th2 cells as an example. Th2 cytokine inhibitor can be used to treat various allergic diseases. Dupixent, an antagonist of IL-4/13 receptor alpha jointly developed by Sanofi and Regeneron, is just a drug of this kind. It targets IL-4R α and inhibits Il-4 and IL-13 signaling. 

▲Dr. Elias Zerhouni 

Another example is acid sphingomyelinase deficiency, also known as Niemann-Pick disease, which is caused by lack of acid sphingomyelinase. Genzyme, a subsidiary of Sanofi that specializes in products for rare diseases, developed the olipudase alfa, a recombinant human acid sphingomyelinase, for the treatment to such disease. Dr. Zerhouni also presented the pharmacological model for the disease. 

It is the same to the treatment to diabetes, obesity and NASH, according to Dr. Zerhouni, who said that Sanofi is developing dual and triple antagonists (GLP/GIP/Glucagon) for that. 

Dr. Zerhouni said Sanofi is on the way for a critical strategic transformation, from micromolecule to macromolecule, from single target spot to multiple ones. Based on that, Sanofi’s scientists have developed a host of proprietary technologies. 

In addition, Dr. Zerhouni also introduced a paper that was released on Science on Sept 21, 2017. It is about the trispecific broadly neutralizing HIV antibodies that can be used at three different spots to neutralize AIDS virus. It helps prevent HIV and reduce drug resistance risks that are frequently caused by existing drugs of similar kind. The new drug is expected to be put into Phase I clinical trial next year. 

Concerning the latest digital medicine, Dr. Zerhouni said big data is opening a door for drug research and development. The digital solutions will accelerate the research and development of drugs and improve the prediction accuracy.

Finally, Dr. Zerhouni concluded that translational medicine creates new opportunities. 

Dr. Wang Chen, director of China-Japan Friendship Hospital and academician at the Chinese Academy of Engineering, then gave a speech on “How to Set up a New Drug Research, Development and Evaluation System in Hospital: International and Chinese Modes”. 

▲Dr. Wang Chen

Wang said currently there are a multitude of opportunities and challenges in innovative drug development, as many demands for clinical treatment have not been satisfied. Great achievements have been seen in new drug development, along with many new therapies, such as cell therapy.

Recently, the state government has released a list of specialized significant new drugs. Priority has been given to drugs on the list in the process of review. In recent years, CFDA has introduced a series of new regulations and policies, which meet international norms and the actual conditions in China. This helps facilitate new drug development and enhance the effectiveness of related accountability systems. 

According to Wang, the clinical research mechanisms in most of Chinese hospitals were established based on advanced international practices and executed through multiple modes.

The Chinese hospitals have carried a lot of clinical trials. For example, Peking Union Medical College Hospital, West China Hospital and PLA General Hospital respectively launch hundreds of clinical trials each year. However, Wang said, it is still less than that in European and American hospitals, which have thousands of high-quality underway clinical trials each year. 

▲ Dr. Wang Chen

Wang also gave an introduction to the framework of the clinical drug research platform established in his hospital. The platform incorporates the Phase I pharmacological study based on the early Phase II and III studies.  

Wang pointed out a series of existing problems in current clinical trials, including the lack of accountability system for doctor in charge of treatment, the low efficiency caused by three-level ward round and the problems in GCP execution. 

Wang then explained the problems in traditional Chinese medicine development based on real cases. 

Concerning study methods, Wang introduced the randomized controlled trial (RCT), cohort study, real-world study and application of artificial intelligence technology.

Finally, Wang called on paying attention to digestive tract diseases, which have caused heavy burden in China. 

The last speaker is Prof. Dong Chen, director of Tsinghua University School of Medicine. He gave a speech on “Promoting Medicine Education and Innovation”.

▲ Prof. Dong Chen

Prof. Dong said the Chinese government has launched the “Thousand Talents” program to support industrial leading talents, especially introducing overseas high-level talents and research projects. This provides support for the research and development of new drugs. 

▲ Prof. Dong Chen

Prof. Dong then gave an introduction to Tsinghua University’s major moves to promote medicine education and innovation as well as explained in details the organization of the hospital. He mentioned that the hospital has set up an immunotherapy innovation center. 

He expounded on the Tsinghua University’s 3+2+3 education mode, which consists of three years’ learning at Tsinghua University, two years’ learning at overseas research institutes and three years’ clinical practices. 

Prof. Dong then gave an introduction to Tsinghua University’s study on translational medicine and clinical study. 

The closing ceremony included a summit on the development and future of translational medicine.

Jiang Ningjun presided over the summit. The attendees included: Dr. He Ruyi, chief scientist at CFDA Center for Drug Evaluation, Elias ZERHOUNI, Dr. Wang Chen and Prof. Dong Chen (Left to right). 

The experts exchanged their opinions on the prospect and advantages of translational medicine in China, related main technologies as well as the cooperation between government, academic circle, clinicians and industrial circle. They also interacted with the audiences and answered their inquiries.