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Will a 'wonder drug' be available in 10 years?

10:50am Monday 29th September 2014 content supplied byNHS Choices

"Wonder drug to fight cancer and Alzheimer's disease within 10 years," is the headline in The Daily Telegraph.

This headline is a textbook example of hope (and hype) triumphing over reality, as the new "wonder drug" is neither available today nor inevitable in the future.

The headline was based on a study that provides new information about the role of the protein N-myristoylation (NMT) in human cells and a mechanism that inhibits it.

The study's authors suggest NMT could be involved in the development and progression of a range of diseases, including cancerdiabetes and Alzheimer's disease.

Inhibiting the actions of NMT could help combat these diseases. But this remains to be seen: if true, this greater understanding may open up new avenues for medical research, which could ultimately lead to new treatments in the future.

While the results are both intriguing and promising, it is very difficult to predict the precise route or timing of future medical developments (drugs, treatments or therapies) based on early laboratory investigations.

Even if treatments based on NMT inhibition were developed and found to be effective, there is no guarantee they would also be safe or free from serious side effects.

All in all, the 10-year timeframe suggested by The Daily Telegraph should be taken with a pinch of salt.

 

Where did the story come from?

The study was carried out by researchers from Imperial College London and was funded by Cancer Research UK, the Biotechnology and Biological Sciences Research Council, the Engineering and Physical Sciences Research Council, the European Union, and the Medical Research Council.

It was published in the peer-reviewed journal Nature Communications.

While The Daily Telegraph's hyped-up headline was a little over the top, the coverage was accurate and balanced.

Optimistic quotes from the study authors, such as, "Eventually we hope this would simply be a pill you could take. It will be perhaps 10 years or so to a drug 'on the market' but there are many hurdles to get over", were counterbalanced with a note of realism from Cancer Research UK's senior science officer: "The next steps will be to develop this idea and make a drug - but there's a way to go before we'll know if it's safe and effective in people".

 

What kind of research was this?

This was a laboratory-based study looking at the structure and function of proteins in human cells.

Proteins are very important in human biology as they are involved in, or carry out, a huge range of biological tasks and processes.

This study looked at a specific chemical modification called N-myristoylation (NMT), which happens to some proteins as they are being made and after they have been made. This is a very common chemical modification of proteins, which in turn affects their function - a form of regulation.

The researchers say NMT has been implicated in the development and progression of a range of human diseases, including cancer, epilepsy, Alzheimer's disease, Noonan syndrome (a genetic condition that can disrupt the normal development of the body), and viral and bacterial infections.

 

What did the research involve?

The study used laboratory-grown human cells to study all the characteristics of the NMT process. This was achieved by identifying all the proteins undergoing the NMT process and finding out what these chemically tagged proteins did inside the cells, what processes they were involved in, other chemicals they interacted with, and whether the protein NMT process could be stopped (inhibited).

The group studied cells in the laboratory during normal cell function and apoptosis - the natural process in which a cell self-destructs in an ordered way, also known as programmed cell death. Apoptosis is often inhibited in cancer cells, causing them to grow indefinitely and not die.

 

What were the basic results?

The researchers' findings include:

  • Identifying more than 100 NMT proteins present in the human cells studied.
  • Identifying more than 95 proteins for the first time.
  • Quantifying the effect of inhibiting the NMT process across more than 70 chemicals (substrates) simultaneously. This showed which chemicals the NMT proteins were interacting with inside the cells.
  • Finding a way to inhibit the NMT process by inhibiting the main enzyme responsible for the chemical modification, called N-myristoyltransferase.

 

How did the researchers interpret the results?

The research team said: "Numerous important pathways involve proteins that are shown here for the first time to be co- or post-translationally N-myristoylated [N-myristoylated during or after their formation]."

Commenting on the wider implications of their research, they said: "These data indicate many potential novel roles for myristoylation that merit future investigation in both basal cell function and apoptosis, with significant implications for basic biology, and for drug development targeting NMT [N-myristoyltransferase]."

 

Conclusion

This laboratory protein study has provided new information about the role of protein N-myristoylation in human cells and a mechanism to inhibit it. The findings suggest proteins undergoing N-myristoylation are involved in many key biological processes and tasks.

Given the researchers' assumption that protein N-myristoylation has been implicated in the development and progression of a range of diseases is true, this greater understanding may open up new avenues for medical research, which could ultimately lead to new treatments in the future.

However, it is very difficult to predict the precise route or timing of future medical developments (drugs, treatments or therapies) based on early findings.

The study's authors struck a balance of optimism and realism when quoted in The Daily Telegraph.

They first said their findings could lead to new treatments in the future and that, "Eventually we hope this would simply be a pill you could take. It will be perhaps 10 years or so to a drug on the market". Balancing this out, they also said: "There are many hurdles to get over".

This study represents one of the first steps on the road to new drug discovery, so the exact path ahead is unclear.

But despite these promising early findings, there are no sure bets in drug development - the history of medicine is full of initially encouraging avenues of research that ended up leading to dead ends. 

Analysis by Bazian. Edited by NHS Choices. Follow Behind the Headlines on Twitter. Join the Healthy Evidence forum.

Summary

"Wonder drug to fight cancer and Alzheimer's disease within 10 years," is the headline in The Daily Telegraph. This headline is a textbook example of hope (and hype) triumphing over reality.

Links to Headlines

Wonder drug to fight cancer and Alzheimer's disease within 10 years. The Daily Telegraph, September 27 2014

Links to Science

Thinon E, Serwa RA, Broncel M, et al. Global profiling of co- and post-translationally N-myristoylated proteomes in human cells. Nature Communications. Published online September 26 2014

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