Prof. Dr. Alain Fischer: The baby we treated with gene therapy is 27 years old today

Hosting a lecture at the university, Prof. Dr. Alain Fischer, Head of the Department of Molecular Biology and Genetics at Acıbadem University, shared his groundbreaking work in the scientific world and the developments shaping the future of medicine with the participants. Known for his pioneering work in pediatric immunology, rare diseases, and gene therapies, Prof. Dr. Alain Fischer is among the architects of a significant transformation in modern medicine, particularly through his research aimed at understanding and treating the genetic foundations of immune system diseases. Prof. Dr. Alain Fischer emphasizes the promising developments offered by gene therapies, as well as the indispensable role of vaccines in public health, with powerful messages… 

During his visit, Prof. Dr. Alain Fischer also toured the laboratories of the Department of Molecular Biology and Genetics at Acıbadem University and received information about the cellular therapy studies conducted there. He stated that cancer treatment applied with CAR-T cells—an innovative approach where the patient's own immune cells are strengthened in the laboratory and reintroduced into the body to destroy cancer cells more effectively—will become much more widespread in the near future. As a scientist who also makes significant contributions to public health in the field of gene and vaccine policies, Prof. Dr. Alain Fischer draws attention to the strategic importance of work in this area…

Stating that he has been tracking genetic diseases for 40 years, Prof. Dr. Alain Fischer says he has largely dedicated his professional life to researching rare diseases linked to the immune system: “We are talking about over 500 different diseases. All of these diseases arise due to genetic disorders and mutations. For over 40 years, I have been trying to understand the genetic causes of these diseases and develop ways to treat them.”

Expressing that the developments in genetics and molecular biology have created a revolution in medicine, Prof. Dr. Alain Fischer says, “Thanks to DNA analysis, we can now detect defects in genes, better understand the mechanisms of diseases, and develop much more effective treatment methods.” 

Treated with Gene Therapy at 6 Months Old, Now a Healthy 27-Year-Old

Speaking about many children he has helped regain their health through gene therapy to date, Prof. Dr. Alain Fischer draws attention to the tangible success of gene therapies and shares the story of a baby born with severe combined immunodeficiency (SCID): 

“Babies with SCID are born with little or no immune system. These children, also known as ‘Bubble Babies,’ usually lose their lives within the first year after birth if not treated. However, with early diagnosis and appropriate treatment, these children can live. In 1999, we applied gene therapy to a baby who was only 6 months old. Today, that patient is 27 years old and living a healthy life. This is one of the most striking indicators of how powerful a treatment method gene therapies are.”

Stating that they focus on treating genetic diseases with gene therapies, Prof. Dr. Alain Fischer says, “Our goal in gene therapies has been to correct the defective gene. About 30 years ago, we developed the approach of treating defective genes by inserting a copy of a healthy, normal gene into human cells. To date, we have cured approximately 60 patients with gene therapy.” Prof. Dr. Alain Fischer emphasizes that this method yields effective results not only in immune system diseases but also in many genetic disorders such as liver, eye, and nervous system diseases.

“Gene Therapy Will Hold a Critical Place in the Future of Medicine”

Stating that gene therapies will not completely replace existing treatment methods but will be one of the most important approaches to complement them, Prof. Dr. Alain Fischer says, “Gene therapies will become one of the most important treatment options, especially for genetic diseases. We foresee that they will be used much more widely in the future.” 

Expressing that scientific developments continue without slowing down, Prof. Dr. Alain Fischer adds that thanks to early diagnosis technologies, diseases could be detected in the womb in the future and treated immediately after birth: “There will be significant developments in the future, especially in rare and genetic diseases. Gene therapies will be a cure for many diseases. Stem cell therapies will also be at the forefront. With the development of gene therapies and stem cell therapies in particular, one day we will find cures for all genetic diseases. Scientific studies are promising. Furthermore, early diagnosis methods are also gradually improving. In the future, we will be able to diagnose many diseases in the womb. Thanks to developments in genetic science, we can now examine the DNA and the entire genetic structure of a baby in the womb. Because we can make a diagnosis as soon as the child is born, we start treatment at a very early stage…” 

Prof. Dr. Alain Fischer points out that in the future of science, artificial intelligence and genetics are “hand in hand.” Touching upon the role of artificial intelligence in scientific research, Prof. Dr. Alain Fischer says, “Artificial intelligence is a very powerful tool that increases the quality of research. When used in conjunction with genetic and biomedical studies, it allows us to reach much faster and more accurate results.” 

“Vaccines Are One of Medicine’s Greatest Achievements”

Known as France’s “vaccine ambassador,” Prof. Dr. Alain Fischer draws attention to the critical importance of vaccines for public health. Prof. Dr. Alain Fischer says, “20 years ago, vaccination rates in France had dropped slightly. Upon this, the Minister of Health at the time wanted to discuss with me what we needed to do to increase the number of vaccinated children. I have been working with vaccines for many years and advising ministries. During the Covid-19 pandemic, we also had important work with ministries for the vaccination of the public. Vaccines are the most effective tool, especially in preventing epidemic diseases. As a pediatric immunology specialist, I always emphasize the importance of vaccines. Vaccines are one of the greatest achievements of medicine.”

Citing data from the World Health Organization, Prof. Dr. Alain Fischer shares the information: “In the last 50 years, approximately 150 million lives have been saved thanks to vaccines against diseases such as measles, tetanus, and Hepatitis B. Covid-19 vaccines alone have saved the lives of approximately 20 million people.” 

“Anti-Vaccination Is Very Dangerous, Do Not Believe Misinformation”

Touching upon anti-vaccination, Prof. Dr. Alain Fischer states that this approach poses a serious public health threat and says, “Claims that vaccines cause autism or heart disease have no scientific basis. These are all fake news. Believing this misinformation causes diseases that were brought under control in the past to re-emerge.”

Worldwide, especially since 2019, there has been a serious rise in child deaths again due to disruptions in access to the measles vaccine and the rise of anti-vaccination. Emphasizing that diseases such as measles are being seen again because vaccines are not being administered, Prof. Dr. Alain Fischer draws attention to the seriousness of the issue, saying, “Since 2019, in some years, more than 100 thousand children have lost their lives due to measles. Yet, these deaths can be largely prevented with vaccines. Not getting vaccinated can lead to very serious and dangerous consequences.”