DRUG RESEARCH
There is no cure for SPATA5 in the entire world. The disease is rare and therefore does not attract the attention of large pharmaceutical companies. However, science and technology have already advanced to such a level that there is hope of finding and developing a cure for SPATA5.
Experts at the Czech Center for
Phenogenomics of the Institute of Molecular Genetics of the Academy of Sciences
of the Czech Republic are working on research and development of a
drug for SPATA5.
The
research is led by PD. Dr. rer. nat. habil. Radislav Sedláček.
You
can read an interview with him here (in czech).
1. Pilot phase
During the pilot phase, detailed research into the gene and design of a treatment procedure will take place.
We estimate the duration of this phase to be 12-18 months.
The estimated cost of this phase is 120.000 - 160.000 EUR.
2. Preclinical study
The result of the preclinical study will be approval for human testing of the drug from the Czech Institute for Drug Control
and the European Medicines Agency (EMA).
This phase will last 2-3 years.
The estimated cost is 800.000 – 1.000.000 EUR.
3. Clinical study
As part of the clinical trial, the drug will be administered to the first patients.
This phase will last at least 4-5 years.
It is difficult to estimate the costs of this phase, but they may be around 400.000 – 800.000 EUR.
There is no budget in the Czech Republic for research and development of rare diseases, everything must be paid for from our personal resources, by us - the parents of children with SPATA5. The research is an expensive matter exceeding our personal financial resources, which is why we are looking for support from the public.