First-of-its-kind study into the Ketogenic diet

2 Apr 2024, 10 a.m.

Ace by the sea

A ketogenic diet, which is low in carbohydrates and high in fat, shows promise as an alternative treatment option for infants with drug-resistant epilepsy.

The new study, led by researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH), was the first in the world to compare the impact of a ketogenic diet to further anti-seizure medication for infants aged one to 24 months with drug-resistant epilepsy.

The study found both treatment options to be effective for reducing seizures in infants.

Introducing Ace

Ace, who is now 10 years old was one of the patients who took part in the study.

Ace was just 14 weeks old when she started having partial epilepsy seizures. Despite being put on medication the seizures continued to worsen. At 5 months Ace developed infantile spasms – a severe type of epilepsy that usually occurs under the age of two years which causes stiffening of the body and jerky movements of the head, arms or legs - and was put on high-dose steroids.

It wasn’t until Ace was 12 months when she was diagnosed with a SCN8A gene mutation - a condition that causes developmental delays and learning difficulties, as well as also being a common cause of drug-resistant epilepsy in children.

When steroids failed to control Ace’s seizures, she was started on a new anti-epilepsy drug called vigabatrin.

Mum Rai said: “None of these treatments worked – Ace, who is non-verbal, was still having seizures up to 10 times a day, and the side-effects of the medications were making her tired, floppy and unable to stay awake.”

The family were then referred to GOSH for treatment.

When combining the steroid therapy with the vigabatrin failed to prevent Ace’s daily seizures the epilepsy and dietetic team at GOSH and the UCL GOS ICH agreed that Ace should try a ketogenic diet as a treatment option.

The ketogenic diet is a high-fat and low carbohydrate diet that has just enough protein for the body’s growth. Foods that are high in carbohydrates are either limited or excluded but small amounts of fruits, vegetables and dairy are allowed. The diet is carefully managed by dieticians and patients are continually monitored.

“The ketogenic diet should be considered as another treatment option”

Researchers in the study wanted to investigate whether the diet could reduce seizure frequency, compared with further anti-seizure medication.

The study found both treatment options to be effective for reducing seizures. The researchers also found that there was no significant difference in the quality of life and neurodevelopment experienced by both groups.

Lead author and GOSH dietician, Dr Natasha Schoeler, said: “There is no evidence to show that there is a difference between the effectiveness of a ketogenic diet and further anti-seizure medication in infants with drug-resistant epilepsy.

“To be classed as drug resistant, adequate trials of two anti-seizure medications must have already failed to control seizures. Going on a ketogenic diet gives families a feeling of empowerment over their treatment and gives paediatric neurologists an alternative route of care.

“We can’t overpromise and say that a ketogenic diet is better than another drug. However, it should be considered as another treatment option.”

Headshot of Natasha

Lead author and GOSH dietician, Dr Natasha Schoeler

“The change in her was incredible”

Mum Rai said: “Before the ketogenic diet, Ace was seizing daily but within a few months the change in her was incredible.”

“Quite quickly we also saw her interest in her surroundings improving and she’d start kicking toys in her pram, looking in mirrors, rolling on the floor – all the developmental milestones that weren’t there before. Her daily seizures had reduced to a mild seizure occurring about once a month.”

“When Ace was eight we decided to remove the ketogenic diet and she has been having normal foods since, and has been stable. In fact, for the past few months she hasn’t had any seizures.”

Ace has been attending school full time since the age of 5 and is thriving.

“We’ve been mind-blown by how lucky we’ve been with Ace – I know it may not benefit all patients but the ketogenic diet has really improved our daughter’s quality of life.”

“I hope that other families who want to access the diet for their children with drug-resistant epilepsy are able to.”

Dr Schoeler, said: “The diet is very strict and all recipes are made by dietitians. This means that everything must be weighed out properly. However, there are also prescribable products that some infants can eat too – so it is palatable.

“It depends on the family, but it is generally easier for children to start the diet when they are young.

“They may have to stay on the diet for around two years but then we can have a conversation about coming off it. Previous research in children shows that around 75-80% maintain the response after stopping the ketogenic diet, if they become seizure free whilst on the diet.”

Ace and Rai by the sea

Ace and Rai by the sea

A collaborative effort

Senior author, Professor Helen Cross (Honorary Consultant in Paediatric Neurology at GOSH and Director of UCL Great Ormond Street Institute of Child Health), said: “This study was a real collaborative effort amongst ketogenic diet centres in the UK, demonstrating this therapy should be considered as a second line treatment option in infants with epilepsy alongside other standard anti-seizure medication.”

The research was funded by the National Institute for Health and Care Research (NIHR), Nutricia Advanced Medical Nutrition and Young Epilepsy.

Astronaut Tim Peake ‘launches’ our new NIHR Clinical Research Facility

We were thrilled to have astronaut Tim Peake join us at the official launch of the new National Institute for Health and Care Research (NIHR) GOSH Clinical Research Facility where he met with patients, families, staff and children from the GOSH nursery.

Young people want AI involved in their care, new GOSH study shows

For the first time, a study has asked children and young people across all four UK nations for their views on how they would like AI to be used to enhance their healthcare

World-first genomic testing scheme ensures every child gets the best cancer treatment for them

Children with cancer are being offered treatment plans tailored to their specific cancer thanks to a single genomic test that reads more than three billion letters of DNA to identify cancer-causing mutations.

Important step forward in stem cell therapy for rare bowel disease

A new study led by researchers at UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) and the Centre for Stem Cell Biology, University of Sheffield, has demonstrated the potential of stem cell therapy to treat those with Hirschsprung disease.