在今天下午的全體大會的主題演講環節，第2位登場的專家是來自法國的Agnès Buzyn教授，她的報告是Development of Personalized Medicine in France。以下是Agnès Buzyn教授報告的精彩預告以及本報記者的采訪，先睹為快!
Agnès Buzyn, President, Institut National du Cancer - INCa, France
Advances in the molecular profiling of tumour tissues have opened up an era of personalized, or biologically adapted, cancer treatment where therapies are matched to the molecular profile of the individual tumour.
The challenge is to give the right drug to the right patient by selecting the most-effective therapy and avoiding ineffective, toxic and expensive treatments. In the past decade, several targeted therapies for molecularly-defined subsets of cancers have been successfully introduced into clinical practice. These include imatinib, which has dramatically improved the prognosis of patients with chronic myeloid leukaemia with BCR-ABL translocation. Gene mutations or gene-expression patterns now guide therapy for a variety of solid tumours such as breast and gastric cancers (HER2 overexpression), lung cancer (EGFR mutations) and colorectal cancer (KRAS mutations). Molecular characterization of tumours has, therefore, become a decisive factor in the choice of therapeutic strategies for patients with cancer, and drug approvals for molecularly-stratified tumour subgroups make molecular testing mandatory.
In order to ensure a wide and fair access to tumour molecular profiling, the French National Cancer Institute (INCa) and French Ministry of Health have set up a national network of 28 molecular genetics centres that receive dedicated public funding for this activity. Each molecular genetics centre performs innovative molecular tests for all patients in its region, regardless of the institution where they are treated (university hospitals, cancer centres, public local hospitals or private institutions). Molecular tests are free of charge for patients or health institutions and the centres compensate local pathologists for tumour block shipment, a logistical burden with the increasing number of samples to be tested. The initiative has now been operational for 5 years and has been successful in meeting its initial aims of uniform nationwide test provision and fast implementation of molecular tests for new tumour biomarkers. A specific programme has also been implemented to anticipate the launch of new targeted treatments and reduce time-to-access to new drugs and experimental therapies.
The French screening and targeted cancer treatment initiative is unique and represents an example of a nationwide implementation of personalized medicine into clinical practice. It illustrates that molecular stratification can be successfully integrated into the healthcare system.
■Dialogue
Dr. Agnès Buzyn is a famous haematologist. In an interview with CMT, we asked her opinion on the future of haematology. She said,
"Haematology and oncology follow the same direction towards personalized medicine. However, cell therapy and gene therapy are more likely to reach their goal in the near future due to the accessibility of stem cells. Clinical research should address the question of therapeutic strategies and innovative drugs in severe haematologic malignancies, whose prognosis hasn’t really change for decades. The ultimate purpose would be to avoid allogenic hematopoietic stem cell transplants for patients."
"Personalized Medicine" and "Precision Medicine" are both hot topics in cancer therapy at present,But Dr.Agnès Buzyn believes,"The concept of personalized medicine, the tailoring of medical treatment to the individual characteristics of each patient, is not new and may be considered an extension of traditional approaches to understanding and treating disease. What is new is that advances in a wide range of fields along with increased technical and computational power, are allowing more precise and effective patient monitoring and treatment that better meet the individual needs.
Skillful application of genomics allows researchers to understand at a molecular level underlying causes of functional failure, to develop targeted therapeutic strategies, and to use the results of a genetic test to select the right groups of patients for the drug. This is precision medicine (“treatment strategies that take individual variability into account”) ， which makes use of molecular information to improve the precision with which patients are categorised and treated [Nature Medicine 19, 249 (2013)]. One-third of the new drugs approved since 2011 had some type of genetic or other biomarker data included in the submission to characterize efficacy, safety, or pharmacokinetics. This proportion is increasing.
In October 2013, the US Food & Drug Administration declared that the era of personalized medicine has clearly arrived. Personalised cancer care relies on the principles of thorough molecular characterisation of tumour samples, the extraction of targetable hits and prioritisation of these, followed by target-drug matching and selection of individual drugs or drug combinations per individual patient.
The development of decision algorithms and availability of genomic data will pave the way from ‘precision’ to ‘personalized’ medicine.”
In addition, Dr. Agnès Buzyn added an explanation to personalised medicine. She said, "The definition of personalised medicine adopted by the European Commission makes reference to a medical model using molecular profiling for tailoring the right therapeutic strategy for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely and targeted prevention."(Interviewed by JingHong Xu from CMT)
