Masaryk University Establishes Centre For Research and Treatment of Rare Diseases

CREATIC is devoted to research, development and production of medical treatments for diseases which currently have none. Credit: MUNI.

Brno, Sep 22 (BD) – Over half a million people in the Czech Republic suffer from one of the roughly 7,000 diseases categorised as rare, and many other patients are still waiting for a diagnosis. The number of such diseases is growing continuously. In response to this issue, the Central European Advanced Therapy and Immunology Centre (CREATIC) has been launched at the Masaryk University Faculty of Medicine, devoted to research, development and production of medicinal treatments for diseases which currently have no medical solution.

“In my original profession as a doctor, I encountered many times a situation where it was no longer possible to help the patient, and believe me, these are powerful moments for the attending physician as well,” said the university rector Martin Bareš. “But now, thanks to the Faculty of Medicine and its CREATIC project, we have kindled an imaginary light of hope for at least some of them.”

The new centre will have its headquarters in Pavilion C03 of the Faculty of Medicine, within the Bohunice University Campus, which is due to begin reconstruction in February 2024. 

“Universities should also be the solvers of societal problems, and in the case of rare diseases, I believe that research, development and production of medicines on academic grounds will in the future be a necessary supplement to the activities of the pharmaceutical industry, for which it is neither capacity nor economically possible to develop all medicines for the rapidly growing the number of very rare diseases,” said Martin Repko, dean of the MUNI Faculty of Medicine.

The head of the CREATIC project is Professor Regina Demlová, head of the faculty’s Institute of Pharmacology. In recent years, a team of her laboratory colleagues has devoted a lot of effort to the development of somatic cell drugs for a pair of diagnoses for which commonly available chemical drugs do not work: epidermolysis bullosa, known as butterfly wing disease, and a rare cancer that mainly affects children. Pharmacologists have already developed two medicinal products for these patients from the most modern therapies. 

The first of them is an anti-tumour vaccine, used for paediatric oncology patients. “This vaccine works on the principle of activating the patient’s own immune system. It recognizes tumour cells in the body and destroys them in a targeted manner,” explained Demlová. So far, more than 40 patients have been treated with this type of therapy in a clinical trial. A number of them are in long-term remission of the disease. 

The second medicinal product assists the healing of wounds in patients with butterfly wing disease. “Both types of preparations make it possible to treat rare diseases in a very precise and targeted manner, and they can be produced on a small scale for a limited number of patients,” said Demlová.

The limited possibilities of treatment for patients with rare diseases and the increase in the costs of already existing therapies are a priority issue for the European Commission, who are following the work of the team from the Institute of Pharmacology with interest. The institute has received a prestigious European grant for the next six years from the Teaming Horizon Europe research and innovation program, to the amount of EUR 15 million, as well as over CZK 500 million from the national Jan Amos Komenský Operational Program (OP JAK). 

Among other things, the purpose of the CREATIC project is to connect academic and clinical workplaces across the region. Scientists from Brno will receive the support of experienced colleagues from the German Fraunhofer Institute IZI and the University of Leipzig, who already have many years of experience in the development and production of gene and cell therapies. Partners from the University of Copenhagen will offer their expertise in matters of legislation or work with data. 

“The experience with the development and non-commercial production of gene therapies at Fraunhofer IZI is completely unique at the national level, and thanks to the partnership and sharing of know-how, we will be able to offer treatment with these medicinal products, developed at the university, to Czech patients as well,” said Demlová.

In this way, a scientific centre of excellence will be created in Brno, where medicines will be developed not only for patients with butterfly wing disease, but also for other diseases caused by a specific gene mutation. One of CREATIC’s priorities is also the opening of a social discussion on the topic of the availability of care within the public healthcare system and the issue of “fair medicine”, i.e. ensuring that such drugs are produced without profit maximisation at prices transparent for payers. This debate should lead to treatments becoming available to patients for whom there is currently no solution, or who have legislative and financial difficulties accessing the treatments they need. 

“In connection with ever-improving diagnostics and treatment, the number of patients has increased by a third over the past three years, and since the cost of therapy for rare diseases is in the order of tens of millions of crowns, securing resources for the development of this care is a societal challenge. The development and production of such medicinal products on a non-commercial basis is one of the ways to ensure the availability of care and the stability of public budgets,” concluded Demlová.

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