Modern Express News (Correspondent Wu Minyue, Reporter Li Nan) Recently, a primary immunodeficiency disease science popularization campaign with the theme of "perceiving diseases with art" was held at China Pharmaceutical University. The Modern Express reporter learned that this is also the first time that the popularization of science about primary immunodeficiency diseases has entered domestic medical universities. The popular science lectures full of dry goods and the interactive experience devices full of meanings have made the students of the school gain a lot, but also let them have a more three-dimensional understanding of primary immunodeficiency diseases, and strengthen their belief in the research and development of new drugs for rare diseases through efforts.
It is reported that this event was jointly sponsored by the Cord Center for Rare Diseases and the PID Cheer Baby Care Center. Primary immunodeficiency is a clinical syndrome caused by mutations in a single immune gene.
Wang Xiaochuan, Director of the Department of Clinical Immunology, Children's Hospital of Fudan University
"The main problem with primary immunodeficiency is that the immune function is compromised. Wang Xiaochuan, director of the Clinical Immunology Research Office of the Children's Hospital of Fudan University, introduced that immunity has three major functions, one is the defense function. If there is a problem with the immune defense, it is more susceptible to infection than normal people, not only in children, but also in adults. The second is immune self-stability and tolerance function. For example, it is prone to autoimmune diseases or recurrent severe allergies. The third is the monitoring function. Tumors are detected in early childhood and are checked for immunodeficiency disorders.
Tian Man, Vice President of Nanjing Children's Hospital
Tian Man, vice president and second-level chief physician of the Children's Hospital of Nanjing Medical University, said that with the enhancement of people's awareness of diseases and the improvement of medical testing capabilities, more and more primary immunodeficiency diseases have been detected, and this data has also risen from more than 100 to more than 500. "Some patients with primary immunodeficiency disorders can live as normal people with medical intervention and regular treatment," she said. Intelligence, development, etc. are not affected. ”
In recent years, the hospital has also continued to popularize the knowledge of primary immunodeficiency diseases. Tian Man said that the popularization of knowledge can not only allow more clinicians and families to detect children's abnormalities as soon as possible and avoid delaying the best treatment period, but also allow more people to understand the disease, look at patients with normal eyes, and let children with such diseases adapt to normal life. More importantly, patients with rare diseases should receive more care and call on more caring people to provide them with care and funding.
Interactive scene
"Some patients with primary immunodeficiency disease are severe at birth, but some patients do not appear until adulthood," Wang said. As clinicians, we also hope that experts and scholars in the field of pharmacy will work together to think, research, and design new drugs for primary immunodeficiency diseases. ”
The cycle of new drug research and development is very long, and the research and development of a new drug often takes more than ten years, with an investment of more than one billion, and the final success rate is less than 10%. Such a long chain of R&D work requires close cooperation between hospitals, research institutes and schools. At present, Shanghai Gaobo Cancer Hospital focuses on hematological diseases, tumors and rare diseases, and has established a relationship with the affiliated hospital of China Pharmaceutical University in the hope of increasing investment in the research and development of new drugs.
Feng Chen, vice president of Global Medical Group and executive director of Shanghai Global Cancer Hospital affiliated to China Pharmaceutical University, said that the number of patients with rare diseases is small, the cost of research and development is higher, and some drugs are "sky-high prices". "We hope to increase the accessibility and affordability of new drugs through new drug research and development, so that more patients can afford them. Participating in today's event is also to hope that students will pay more attention to this field and devote themselves to it. ”
(Photo provided by the correspondent)