Category: Understanding Science

On the 25th of April, the World Malaria Day 2022 took place. This year’s theme was “Harness innovation to reduce the malaria disease burden and save lives“, since the main goal of the World Health Organization (WHO) was to highlight the necessity of research and development for new therapeutic strategies to eradicate the disease. Today, malaria is entirely preventable and curable disease if the symptoms are recognized in earlier stages, but in some cases, it is unfortunately not possible. Therefore, the estimated number of new cases in 2020 was 241 million, and within that number there were 627 thousand malaria-related deaths in 85 countries. The region at highest risk is the sub-Saharan Africa, where more than two thirds of deaths were reported among the children under the age of 5. Despite the promising and steady advances in controlling the disease between 2000 and 2015, in recent years there was an evident set-back especially in the number of preventable deaths. What are the causes of this stagnation and what can be done to prevent the spread of this highly contagious disease?

“Our primary mission is life critical. Our goal is very clear: to address the gross inequities in child health still existing in the world today. Life or death for a young child too often depends on whether he is born in a country where vaccines are available or not.”

Nelson Mandela, addressing the Vaccine Fund Board 2003 meeting in Johannesburg

The last topic in the series on medical research and clinical trials is related to studies on vaccines and, what better example to explain vaccines research than the recent Covid-19 pandemic. Similar to pharmaceutical products, vaccines trials occur in 3-4 phases (Phase I-III pre-marketing authorization and Phase IV after the vaccine is licensed).

Several studies conducted in the 90’s suggested that prevalence of HIV infection was smaller in patients with sickle cell anemia than in healthy individuals. Although the mechanism behind that is still not fully understood, today we know a lot more than we did back at the end of the century. In order to understand the connection between sickle cell anemia and HIV infection, let us first take a look at both of them separately.

Friedrich’a ataxia (FA) is a progressive neurodegenerative disease that affects 1 in every 50 000 people worldwide. Therefore, it falls under the umbrella of rare diseases. The thing with rare diseases is that it’s hard to get funding for researching their pathophysiology and possible therapies (ergo the name “orphan drugs”). However, with the recent rise of gene therapy, more and more private investors put their money towards finding a cure for 1 in 50 000 people. So don’t be misled by the title of this article – FA is still a rare disease, but its popularity among research groups and institutes has been growing for the past few years. The main reason for such blooming is the emerging field of gene therapy.

What is a clinical trial and how (why) does it work?

A clinical trial is a study conducted on human volunteers to investigate a variety of questions on a treatment/intervention tested:

• is the treatment/intervention safe?
• does the treatment/intervention work?
• does the treatment/intervention work better than what is already available (if there is a similar treatment/intervention)?

“There are no such things as incurable, there are only things for which man has not found a cure.”

Bernard Baruch, 30 April 1954

What is medical research?

Medical (biomedical) research, sometimes referred to as experimental medicine, comprises different types of research, from basic, to clinical, applied research. Different scientific fields are usually included and range from biology, medicine, physics, computational science, mathematics, chemistry, and pharmacology. The overarching intention of such research is to improve human health and well-being.

I believe it is safe to say that those of us who were at some point (or maybe still are) glued to our screens watching Grey’s Anatomy often found yourselves intrigued by some of the innovative treatments used on the patients. One of my personal favorites was a clever use of ultrasound waves to treat a hypothalamic hamartoma in a young boy. After that episode, I rushed to the Internet trying to find anything published about the technique. I was amazed by the idea and was trying to find out more about it. Is it really possible? Can it really be used as a completely non-invasive way of treating brain masses, including tumors? Is it safe? Is it maybe already in use? To my disappointment, I found nothing. I’m not sure whether I did a very bad job at googling those facts back then, or maybe really nothing had been published yet. However, I recently stumbled upon a very interesting article about the use of a head-mounted magnetic device that shrinks tumors. Since it reminded me of the cutting-edge treatment from Grey’s Anatomy, I once again googled it, only this time with greater success. As it turns out, a lot has been done and published upon this subject over the past few years.

In the previous article, we started discovering what is hidden behind the name of CRISPR-Cas9. The main idea of this series of articles is to understand the background of this technology for precise genome editing. It’s truly fascinating how one unusual feature of the bacterial genome has served as an inspiration for further discoveries and developments in biotechnology. Of course, none of this would be possible without the mutual cooperation of scientists all around the world.

CRISPR-Cas9 technology probably needs no special introduction. After all, exactly this system for precise genome editing has started a complete revolution of genetic engineering. Still, the focus of today’s article won’t be the application of CRISPR-Cas9 in biotechnology, no matter how fascinating it gets (but no worries, we will come back to it some other time).

Today, it’s time to take a look at the background of this almost perfect molecular tool, today’s version of which is reduced to only one enzyme and one carefully picked RNA molecule.

Since I started working as a community pharmacist, it has come to my attention that a big part of the general population takes six or more medicines every day, especially the elderly. Although they prepared us for this at university, it still surprised me once I witnessed it in everyday practice. The major problem with polytherapy are drug interactions, which are often neglected, especially in Croatia. The idea of rational pharmacotherapy is just that – to rationalize drug use and consequently assure safer treatments (fewer side effects, minimal risk of sub-dosing or overdosing), less cost to the healthcare system and greater adherence to the therapy; the latter possibly being the most crucial.