CREATIC integrates STEM and social sciences across its research programmes to advance cell and gene therapies and bring meaningful results into clinical use.
The program focuses on rare diseases, spanning basic and translational genomics, clinical care for undiagnosed pediatric patients, and advanced therapy development.
The program combines clinical research in pediatric oncology with laboratory studies of mechanisms
of resistance to immunotherapy.
The program develops innovative model systems for vision and neurodegenerative disease research, and focuses on applied medical microbiology.
Central European Advanced Therapy and Immunotherapy Centre is dedicated to:
- Finding ways, nationally and internationally, to increase the availability of innovative treatment options for patients with rare diseases,
- Researching and developing new treatment options – so-called advanced therapy medicinal products (ATMPs), which are based on the effect of human cells whose therapeutic capabilities have been enhanced in our laboratories,
- Collaboration with physicians, centres and patient organisations that care for patients with rare diseases and those without a diagnosis, both medically and socially, e.g. with the Centre for children without diagnosis at the Paediatric Clinic of University Hospital Brno or patients organisation DEBRA ČR, z.ú.
Advanced therapy medicinal products
Today's science and biotechnology make it possible to enhance the healing properties of various cells of the human body. This is what we study and perform in ACIU-CREATIC laboratories.
The medicinal products developed and manufactured at ACIU-CREATIC belong to the so-called advanced therapy medicinal products (ATMP). We focus on cell or gene therapy products suitable for the treatment of rare diseases.
Gene therapy aims to introduce a normal “healthy” gene into some somatic cells of the patient. This could be a gene that helps immune cells to better recognise a tumour or virus-infected cell. A gene can be introduced also into a malfunctioning mutated cell ensuring that e.g. damaged nerve cells can transmit impulses and thus maintain functional muscles, or ensuring replacement of a missing enzyme needed for the brain to function properly. These introduced genes remain only in the recipient's body and do not have the ability to spread to patient’s offspring. Our medicinal products aim to support cancer treatment.
Cell therapy uses the patient's own cells or those of a healthy donor, which have been promoted in the laboratory to increase their therapeutic abilities. It is currently used, for example, for some complications of Crohn's disease. Our medicinal products aim to promote wound healing and support cancer treatment
Tissue-engineered medicinal products use the patient's own modified cells or those of a healthy donor to replace the damaged or dead cells of the recipient. They are currently used to treat severe corneal injuries or damage to articular cartilage.
Rare diseases
There are currently around 8,000 known rare diseases – medical conditions that occur very rarely in the human population (at most 1 in 2 000) but often threaten the lives of patients or significantly affect their health and daily functioning.
Some of these diseases are already well known to the general public and the professional community – for example, cystic fibrosis, butterfly wing disease, muscular dystrophy and some cancer diseases that occur in childhood. However, many other diseases pass unnoticed and remain known only to the families of patients and the healthcare staff and social workers who care for them.
Rare diseases arise from a variety of causes and have very different manifestations. Patients thus have a variety of difficulties and needs. For most rare diseases, there is no cure yet, but appropriate health care can prolong and improve patients' lives and make care more manageable for their families.
Health care for patients with rare diseases is complicated by:
- > Insufficient scientific knowledge and the slow development of new treatment options,
- > Lack of physicians-specialists,
- > Long path to the final diagnosis and the difficulty of accessing optimal healthcare
Undiagnosed patients
Despite the efforts of treating physicians, some patients can be undiagnosed for a long time. In most cases, these are paediatric patients whose diseases can be genetic.
These patients can receive the best care only in specialised centres. Physicians there can perform the necessary genetic tests and consult with colleagues abroad, which facilitates the identification of the causes of the disease, treatment options both in the Czech Republic and abroad, and collecting and analysing data that can help future patients.