Gene therapy based on the Crispr tool promises a revolution in medicine: eliminating diseases at the root, genes. But how many diseases are really genetically determined? What does the pharmaceutical industry do today? In the future we still need drugs? A brief overview of what is already possible, of what is still science fiction and why drugs are still very, very active.
When we talk about genetically conditioned diseases, we are not only talking about rare cases such as cystic fibrosis or Huntington disease. Most of the diseases that afflict us in the course of life (cancer, diabetes, heart disease, depression) have at least a partial genetic basis. It is estimated that from 70 to 80 percent of all chronic diseases are somehow connected to genetics.
This does not mean that our genes are the only guilty. The environment, lifestyle and case also play an important role in most diseases. But the fact remains: without genes the diseases would not exist, or would be very different.
While rare diseases are usually caused by a mutation in a single gene (there are about 8,000 diseases of this type), the most common diseases are much more complex. Several genes are involved, in addition to the environment, diet, stress and habits. We call them polygraenic diseases and represent the greatest health challenge of our times.
Pharmacy: huge, diversified and (for now) indispensable
Most of the drugs we know and use today do not eliminate the cause of the disease, but they relieve the symptoms or slow its progression. For example, type 2 diabetes drugs help to regulate blood sugar, but do not correct the genetic defect that could contribute to the disease.
Currently the pharmaceutical industry covers most of the needs of patients: it is estimated that from 70 to 90 percent of people suffering from diseases with a genetic component already receives some form of treatment. But for monogenic rare diseases the coverage is much lower: for most of these 8,000 diseases there is no cure or only an experimental therapy is available.
This is not because science does not know. Above all because developing a drug for rare disease is risky from a financial point of view, extremely expensive and uninteresting from the market point of view. The development of a genetic drug can cost hundreds of millions of euros, but it is sufficient to treat only a few hundred people in the world.
CRISPR: the technology that cuts DNA and opens a new chapter
CRISPR is a tool that allows a precise repair of DNA, as a kind of molecular scissors that cut the wrong piece of genetic code away and replace it with the correct one. In theory, it can eliminate the cause of the disease, not only its consequences. In practice, however, we are still at the beginning of the trip. Although it is thanks to the artificial intelligence that things seem easier today than a few years ago.
We have seen the greatest progress so far in the treatment of rare genetic diseases And some forms of cancer. Therapies such as zolgensma for spinal muscle atrophy already exist, but they cost more than two million euros per patient. In addition, only a few are approved in Europe, mainly for diseases that caused by a single gene.
For the most common diseases, the situation is much more complex. These diseases are not the result of a single mistake, but Networks of genetic and environmental factors. And CRISPR – for now (we do not know what the future reserves) – is not precise and safe enough to treat them effectively and reliablely.
There are also ethical and legal limitations. In most countries the genetic modification of embryos (the so -called germ therapy) is prohibited, which would allow to eliminate hereditary diseases before birth. And even if this were allowed, the question of those who decide what is genetically “normal” would still remain unresolved.
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Because CRISPR will not replace (still) pharmaceutical products
Although it seems that Crispr can replace drugs, in practice it is not that simple. Technology has enormous potential, but it works mainly in very specific cases. Most of the diseases we take care of today require a different approach: long -term, gradual and often combined.
Even the pharmaceutical industry will not go back. Large companies such as Pfizer, Novartis or Gilead are already investing in CRISPR Search. Not because they are threatened by technology, but because they want to be part of the next phase of medical development. In the future we will probably witness a hybrid model: some patients will receive gene therapy, while most will continue to take drugs.
Even for the diseases that Crispr can theoretically treat, drugs will often be necessary to control symptoms, prevent complications or improve the quality of life.
What about Slovenia?
Slovenia has a well -developed genomic diagnostics, which means that we are able to detect genetic alterations associated with diseases. We also have several research groups involved in the development of gene therapies. But access to Crispr treatment is (and will remain) limited, mainly due to the price, regulation and dependence on foreign countries.
Our pharmaceutical companies, such as Krka and Lek, are focusing on generic medicines, which remain essential for the health system. Gene therapy is not a therapy intended to become part of the daily health practice shortly, neither in Slovenia nor in the rest of the world. Although things are changing very quickly thanks to the development of artificial intelligence, implementation, especially the large -scale one, will require at least a decade. Despite some discoveries accelerated in the following years.
Conclusion: CRISPR is not the end of the pharmaceutical industry, but his new partner
Gene therapy is the future, but it is a future that comes slowly and selectively. CRISPR will change the way some diseases are treated, in particular rare and genetically simple ones. But most patients will need drugs that relieve symptoms, improve the quality of life and allow the daily management of the disease for a long time.
Instead of replacing pharmaceutical products, Crispr will integrate them. And if in the next decades the technology will become safer, more accessible and cheaper, it could play a fundamental role. Until then, the drugs will remain the fundamental pillar of health care, also for genetically determined diseases.
