Experimental cancer drug that targets common genetic fault can shrink tumours by 64% in six weeks

An experimental cancer drug that targets a common genetic fault has been shown to shrink tumours by up to 67 per cent in just six weeks.

Scientists tested their newly developed anti-cancer drug AMG 510 on four patients, two of whom saw their tumours shrink. It did not work for the others.

In studies on mice, the treatment helped to shrink, and in some cases eradicate, the growing tumours. 

The daily pill works by turning ‘off’ the KRAS gene. Mutated forms of the genes are permanently trapped ‘on’, causing cancerous cells to multiply.

This abnormal activity fuels the development of up to 50 per cent of lung cancers, as well as some pancreatic and bowel cancers.

An experimental cancer drug that targets common genetic fault has been shown to shrink tumours by up to 67 per cent in just six weeks (pictured)

The pharmaceutical company described their findings as a ‘milestone’ for patients with cancer KRAS-mutant cancers. 

The research was led by by pharmaceutical company Amgen Research, based in Thousand Oaks, California.

The findings, published in Nature, are the first to report the effects of inhibiting KRAS with a drug.

The KRAS gene provides instructions for making a protein called K-Ras that tells cells to grow and divide or mature and take on functions.

It belongs to a class of genes known as oncogenes. When mutated, oncogenes have the potential to cause normal cells to become cancerous.  

AMG 510, an oral medication, stops the activity of this oncogene, Dr Jude Canon and his colleagues at Amgen revealed.

In mouse models of KRAS-mutant cancer, the treatment helped to shrink, and in 20 per cent of the cases, eradicated the growing tumour. 

Then, in the first known clinical trial of a KRAS inhibiting drug, AM510 was given to four patients with non-small-cell lung carcinoma (NSCLC) who were not responding to treatment.

In two of the patients, improvements were seen in their tumour size. However, for the other two patients it did not work.

After six weeks of treatment, the patient who received a dose of 180mg saw their tumour shrink by 34 per cent. The second patient, who had 360mg, saw a reduction of 67 per cent.

What’s more, in mice with bowel cancer, the treatment made the tumour more sensitive to other cancer drugs and generated a stronger immune response – an effect known as a pro-inflammatory microenvironment.

Taken together, the results suggest the drug could work well alone or in combination with other anti-cancer strategies.  

KRAS has become an attractive drug target, however pharmaceutical development has been hindered because a binding site isn’t clear. 

The team found a surface groove called His95 in KRAS. AMG 510’s potency is ten-fold higher when it latches on to the groove. 

The drug is a ‘breakthough’, according to Professor Terence Rabbitts, from the MRC Weatherall Institute of Molecular Medicine, University of Oxford, who was not involved in the research.

He said: ‘The protein KRAS is very often altered in cancer in many different ways and these changes are a major factor in cancer development.

‘The KRAS protein had been thought to be undruggable, although it is not the first drug-like compounds to be described that binds to KRAS.

‘It is important to understand that the drug will not be useful in all cancers and in fact only in those cancers where a very special change has occurred in KRAS.’

Professor Eric O’Neill, a professor of cell and molecule biology, University of Oxford, who was not involved in the study, also urged caution.

He said: ‘The milestone is that this appears to have some clinical activity – the initial inhibitor was developed some time ago.

‘Importantly this drug would not work in patients that do not have the specific KRAS mutation (Kras 12C).’ 

Lung cancers, of which around 85 per cent NSCLC, are very difficult to treat and for most people their cancer will return after treatment.

Survival is poor – around 35 per cent of patients will survive their cancer for five years or more after diagnosis despite being diagnosed early. The rates worsen the later the cancer is diagnosed.  


Lung cancer is one of the most common and serious types of cancer. 

Around 47,000 people are diagnosed with the condition every year in the UK.

There are usually no signs or symptoms in the early stages of lung cancer, but many people with the condition eventually develop symptoms including:

– a persistent cough

– coughing up blood

– persistent breathlessness

– unexplained tiredness and weight loss

– an ache or pain when breathing or coughing

You should see a GP if you have these symptoms.

Types of lung cancer 

There are two main forms of primary lung cancer. 

These are classified by the type of cells in which the cancer starts growing. 

They are:

– Non-small-cell lung cancer. The most common form, accounting for more than 87 per cent of cases. 

– It can be one of three types: squamous cell carcinoma, adenocarcinoma or large-cell carcinoma.

– Small-cell lung cancer – a less common form that usually spreads faster than non-small-cell lung cancer.

– The type of lung cancer you have determines which treatments are recommended.

Who’s affected

Lung cancer mainly affects older people. It’s rare in people younger than 40. 

More than four out of 10 people diagnosed with lung cancer in the UK are aged 75 and older.

Although people who have never smoked can develop lung cancer, smoking is the most common cause (accounting for about 72 per cent of cases). 

This is because smoking involves regularly inhaling a number of different toxic substances.

Treating lung cancer

Treatment depends on the type of mutation the cancer has, how far it’s spread and how good your general health is.

If the condition is diagnosed early and the cancerous cells are confined to a small area, surgery to remove the affected area of lung may be recommended.

If surgery is unsuitable due to your general health, radiotherapy to destroy the cancerous cells may be recommended instead.

If the cancer has spread too far for surgery or radiotherapy to be effective, chemotherapy is usually used.

There are also a number of medicines known as targeted therapies. 

They target a specific change in or around the cancer cells that is helping them to grow. 

Targeted therapies cannot cure lung cancer but they can slow its spread.

Source: NHS 


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