From Molecule to Medicine: How A Cure Is Born

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24 Aug 2025

8 Min Read

Dr Lee Fong Kai (Academic Contributor)

IN THIS ARTICLE
Dr Lee Fong Kai

Contributed by Dr Lee Fong Kai, whose research focuses on medical and health sciences, pharmacy, and medicinal chemistry. He can be reached at fongkai.lee@taylors.edu.my.

Every time you swallow a pill, whether it’s for a stubborn headache, seasonal allergies or a serious illness, you’re holding the result of years of science, patience and determination. Behind that small tablet lies a journey that often begins more than a decade earlier, filled with late-night lab work, unexpected setbacks and breakthrough moments that change lives.

Finding a Needle in a Molecular Haystack

Before a new medicine ever reaches the pharmacy shelf, scientists may have examined more than 10,000 different molecules in the hope of finding just one that works. The search begins with identifying ‘hits’ — molecules that could influence a biological target linked to a disease.

Penicillin Mould

In the past, these discoveries sometimes happened entirely by chance. Penicillin, for example, was famously uncovered when a forgotten petri dish revealed its bacteria-killing powers. Today, the hunt is far more deliberate and precise.

Researchers now use High-throughput screening (HTS) to test thousands of chemical compounds in rapid succession. Powerful computer programmes, such as molecular docking and virtual screening, then sift through the results to highlight the most promising candidates. From there, medicinal chemists step in, applying structure–activity relationships (SAR) to fine-tune the molecule — adjusting its shape and properties to increase effectiveness, reduce unwanted side effects, and help it remain active in the body for longer.

High-throughput screening (HTS) (noun)

 

HST is like having a super-speedy detective team working around the clock. Instead of testing one chemical at a time, scientists use highly automated machines to test thousands of different molecules in a matter of days. Each molecule is checked to see if it interacts with a specific ‘target’ — often a protein linked to a disease. It’s a bit like trying thousands of different keys in a lock to find out which ones even have a chance of opening it.

Structured-activity Relations (SAR) (noun)

 

Once a few ‘keys’ seem to fit, SAR take over. This is where medicinal chemists become the locksmiths, carefully reshaping and adjusting the key to make it work better. They might make the molecule stick more tightly to the target, reduce unwanted side effects, or help it stay active in the body for longer. Through SAR, a promising but imperfect molecule can be transformed into a powerful and precise medicine.

Preclinical Trials: Testing in the Lab and on Animals

Once a promising molecule is chosen, it must first prove itself in preclinical testing before it ever reaches a human volunteer. This stage acts as the medicine’s first real audition, where scientists study its safety, how it is absorbed, and what happens to it inside the body.

 

Testing begins in vitro — experiments carried out in the lab, often using cells in dishes. This is where researchers can see how the molecule interacts with its target and check for early signs of toxicity without involving a living creature.

 

If results look promising, the work moves in vivo — studies in living animals, commonly mice, rats, or rabbits. These studies reveal how the drug behaves in a complex, living system, showing how it is processed, how long it stays active, and whether it causes any harmful effects.

Scientist's Hands Grabbing White Mouse

Animal testing remains a sensitive and often debated step, but it plays a crucial role in protecting human volunteers. By identifying potential risks early, researchers can refine the drug or halt its development before it reaches people.

Only after passing rigorous safety and quality checks can scientists apply for approval to begin human trials through an Investigational New Drug (IND) application. Many breakthrough medicines, including imatinib (Gleevec) for certain cancers and trastuzumab (Herceptin) for breast cancer, began their journey to saving lives by passing through these essential preclinical stages.

From First Human Trials to the Long Road Ahead

Once a drug clears preclinical testing, it is finally ready to be tried in humans — but the journey is still far from over. Clinical trials unfold in carefully planned stages, each designed to answer different questions about the medicine’s safety and effectiveness.

 

Phase I trials are the cautious first step, involving a small group of healthy volunteers (usually 20 to 100 people). Here, researchers focus on safety: finding the right dose, monitoring how the drug moves through the body, and watching closely for any side effects.

 

If the results are encouraging, the medicine moves into Phase II, where it is given to a few hundred patients who actually have the disease. This stage asks: does it work, and is it still safe? Promising treatments then graduate to Phase III, large-scale trials involving thousands of patients across multiple locations to confirm how well the drug works and uncover any rare or unexpected side effects.

 

Even after approval, the process continues with Phase IV, or post-market surveillance, where the medicine is monitored in the real world to ensure it remains safe and effective over the long term.

A scientist using a pipette with a microtiter plate and a petri dish

This careful, step-by-step process is one reason why developing a new drug can take 10 to 15 years. Regulatory agencies such as the FDA in the United States, the EMA in Europe, and Malaysia’s NPRA require clear proof that a drug is safe, effective, and manufactured to the highest quality standards. Yet, despite years of work, only about one in five drugs that enter human testing ever makes it to market. Many fail because they are not effective enough, have unexpected safety issues, or turn out to be commercially unviable.

There is also the pressure of time. Drug patents typically last 20 years from the date they are filed, which often happens during the earliest stages of discovery. This means companies are racing to recover their investment before the patent expires and generic versions can be made.

 

In special situations such as a public health emergency — regulatory bodies may allow fast-track approvals, breakthrough designations, or emergency use authorisations to speed up the process, as seen with COVID-19 vaccines. But in most cases, the priority remains the same: ensuring that every medicine released to the public is as safe and effective as science can make it.

Is the Future Faster

The pace of drug discovery is accelerating, thanks to new technologies that are reshaping how medicines are developed. Artificial intelligence (AI) and machine learning can forecast how a molecule will behave, flag potential safety risks before any laboratory tests begin, and even design entirely new compounds. By doing so, they can shorten the early stages of drug discovery from years to months, bringing life-changing treatments closer to reality.

 

From there, tools like DeepMind’s AlphaFold help researchers predict protein structures with remarkable accuracy, guiding more effective drug design. CRISPR gene editing is opening the door to curing genetic diseases such as sickle cell anaemia and inherited blindness. Personalised medicine is tailoring treatments to an individual’s DNA — for example, using genetic testing to guide breast cancer therapy in patients with BRCA mutations.

Stay curious, be imaginative, and work hard—these are the keys to making real impact in science. If you're passionate about discovering life-saving medicines, your creativity and dedication could lead to the next big breakthrough. Your ideas could shape the future of medicine!

— Dr Lee Fong Kai

Conclusion

From the first spark of an idea in a laboratory to the moment a medicine is handed over at a pharmacy counter, drug development is a journey defined by patience, precision and purpose. It is a process that blends science with compassion, driven by the hope of improving or even saving lives.

 

Pharmacy is not just about dispensing tablets; it is about being part of the story that begins with a single molecule and ends with a cure in someone’s hands. Every breakthrough carries with it years of dedication, setbacks turned into lessons, and moments where science pushes beyond what once seemed possible.

 

So here’s the question: if you had the chance to help create the next life-saving treatment, would you take it — knowing it might change someone’s life forever, perhaps even your own?

The world of pharmacy is where science meets purpose—and it could be your future. If you're curious about how medicines are made and imagine yourself helping develop the next breakthrough, now is the time to take the first step. Start with the Foundation in Science programme, and progress into the Bachelor of Pharmaceutical Science and be part of the journey that turns molecules into medicine.

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