The importance of clinical trials in genetic diseases
Why clinical trials are important in genetic diseases
Clinical trials for genetic diseases are conducted with people who have agreed to participate. And, in reality, it would not be possible that there was a scientific progress without those essays .
It should also be noted that clinical trials for these diseases follow a very clear chronology, divided into several phases. Initially, the trial focuses on a small group and is subsequently expanded to define its effectiveness. This is another key element of the advances in genetic research .
Here we'll tell you about their main advantages, which will help you understand why they're traditionally used. Take note, this will interest you:
1. Improved diagnosis
The adequate diagnoses Early detection of genetic diseases is essential. Well, this can be skillfully achieved through each test. This way, we can detect with greater certainty whether there is a pathological diagnosis and improve screening processes.
One of the recurring problems is the lack of accurate diagnosis. And if this is important for all diseases, it's even more so when it comes to making an early diagnosis, because it helps improve treatments.
2. Experimentation with treatments
Clinical trials have an experimental aspect, but in many cases what is done is to test a New treatment . In this case, although the risks are greater, if proven effective, they can be very useful for generalizing new treatments. This is the first step toward implementing therapeutic innovations.
Obviously, to experiment with a treatment, you need to have some elements that presuppose the chances of a cure. But, in any case, at this stage, the element of randomness is much greater.
3. Measuring the effectiveness of treatments
A clinical trial is useful not only to find out if it works, but also to Measure its effectiveness . Thus, depending on the patient, the reaction may be more or less effective. Ultimately, a cure percentage must be calculated for treatments because not all organisms respond the same way. Therefore, it's advisable to take this into account because it will be easier.
Commensurability of treatment effectiveness is essential for better performance. And it's no small feat, since, without it, decision-making is much more complex for both patients and physicians.
4. More accurate identification of biomarkers
In genetic diseases, the accurate detection of biomarkers It's key. Well, when it comes to clinical trials, it's easier to work with these molecules to see the body's actual reaction to the disease, but also to the treatment. A better understanding of the disease is essential if you want to treat it correctly.
Biomarkers are especially useful, and even more so when it comes to genetic issues.
5. Establishment of new diagnostic and treatment protocols
Finally, the establishment of new protocols for diagnosis and treatment of diseases is very necessary, and this can only be achieved through prior clinical trials. The idea is that, when there is a sample sufficiently broad and with conclusive results, the protocols can be adapted to each group of patients.
Furthermore, it is desirable for diagnostic and treatment protocols to be clear and transparent. Therefore, it is important to have all the information available to the various stakeholders, which will benefit everyone.
Main clinical trials being conducted today for genetic diseases
The clinical trial techniques There are several options for detecting and treating genetic diseases. However, we can highlight the following that have been established recently:
Clinical trials for genetic diseases using CRISPR techniques
The CRISPR technique Gene editing is, without a doubt, the greatest contribution to treating genetic diseases in recent years. Although it is a very recent technique (2012), it allows defective sequences to be eliminated from the DNA chain and edited with other, healthy fragments. These are some of the genetic diseases that have been treated thanks to this new possibility:
- Sickle cell anemia
- Thalassemia
- Hemophilia
- Angioedema
- Glaucoma
- Muscular dystrophy
- HIV
In the first two cases, these therapies have already been approved by the UK health services. This is already a significant boost in this regard. In any case, it should be noted that, in the medium term, we can expect CRISPR treatments and clinical trials to increase.
Trials for cystic fibrosis
Numerous trials have also been implemented for the treatment of cystic fibrosis . In this case, they are being used CFTR modulators to reduce mucus secretion, which is the main cause of the problems. To date, these trials appear to have had moderate success. This will allow us to work toward improving the quality of life for patients.
Trials for different types of cancer
The Trials for different types of cancer , even if their genetic origin is tangential, could also be applied using various techniques. For example, the CRISPR technique can be used for brain tumors. Another option is the use of inhibitors for ovarian, breast, or thyroid cancer. Therefore, there are different techniques that can be useful, depending on the case.
Huntington's disease trials
In recent years, new trials have been applied to the treatment of Huntington's disease . Specifically, antisense oligonucleotides (ASOs), which are particularly useful for reducing mutations, are used, along with other medications. The ultimate goal is to increase therapeutic possibilities.
In summary…
Clinical trials for genetic diseases are diverse, necessary, and will contribute to a better quality of life for people. Would you like to undergo a private genetic test to determine or rule out any risk? Genotics We have a comparison tool for different tests by type, cost, methodology, and delivery time. Get to know us better!