How an experimental treatment saved Emily's life

March 26, 2013

Emily Whitehead is kind of a big deal. At age seven, she is the only child to have beaten back leukaemia with the help of a new treatment that turned her own immune cells into targeted cancer killers.

She has been in remission for 11 months and is the first paediatric patient in a growing US trial that is showing signs of success after decades of research and now includes three other children and dozens of adults.


Emily Whitehead: her own white blood cells were
genetically altered toallow them to recognise
and kill cancer cells. Photo: AFP

Her mother said Emily sometimes grapples with her new-found celebrity, which ballooned after the trial's preliminary results were first announced late last year.

 

"When we go to places where there are a lot of people, sometimes they want pictures with her, or sometimes just to touch her, so I think it gets a little overwhelming," Kari Whitehead said.

 

For the most part though, Emily is happy to play with her dog, read, write and explore the outdoors, thanks to an experimental treatment that saved her life after two relapses left doctors admitting they had no other options.

Now, the US researchers behind the method are expanding their quest for a next-generation cancer treatment that may require one dose in a lifetime, and may one day end the use of chemotherapy and bone marrow transplants.

While the word "cure" is something most experts would not whisper until a patient has lived at least five years illness-free, the field of research into targeted immune therapies is generating buzz.

Work at the University of Pennsylvania is supported by Swiss pharmaceutical giant Novartis, which last year announced an exclusive global deal to license chimeric antigen receptor (CAR) technologies for leukaemia and other cancers.



Celebrity status: Emily Whitehead.
Photo: AFP

Novartis is also funding a $US20 million centre for research in Philadelphia as part of the agreement.

The case studies that describe Emily's journey so far, and that of another 10-year-old girl who did not survive after trying the same adoptive T cell therapy, were detailed Monday in the New England Journal of Medicine.

Both girls suffered from acute lymphoblastic leukaemia (ALL), the most common form of childhood cancer. It is often curable, but theirs was a high-risk type that resists conventional treatments.

The method takes a patient's own white blood cells, called T cells, and genetically alters them to allow them to recognise and kill cancer cells, according to Michael Kalos, part of the University of Pennsylvania team of researchers working on the project.

"The concept has been around for at least 50 years, and it has been tried in humans for about 20 years in different clinical trials, with limited success mostly because the T cells that were put into patients had a real hard time surviving in patients," Kalos said.

Greater longevity was achieved when researchers began using a virus in the HIV family to serve as a vehicle for the gene that needs to enter the T cells, said Kalos.

The team, led by Carl June of the Abramson Cancer Centre of the University of Pennsylvania, first published its results in 2011 on three adults who suffered from chronic lymphocytic leukaemia (CLL).

More than two years after treatment, two of the three are still living disease-free, and more than a dozen new patients have begun treatment.

A separate team of researchers at Memorial Sloan-Kettering Cancer Centre in New York also published a study last week in Science Translational Medicine detailing their work on five adult patients with ALL, the type Emily had.

Kalos said University of Pennsylvania researchers are seeing "very strong responses in most of the patients but in a small subset we are not seeing a response and we are trying to understand why that is the case".

"It could be the patient, it could be the product, it could be the tumours or it could be something totally different."

In the meantime, early trials on adult pancreatic cancer patients and people with mesothelioma have already begun. For now, they are only in the United States, but the team hopes to expand globally.

The terrain is brand new. Every patient would need his or her own specialised treatment, and patients need to get antibody treatments to boost their immune systems for years, perhaps indefinitely, to guard against illness.

But, if the recent success continues, a treatment could be on the market within a few years, Kalos said.

"In our case, the data is looking so promising that we are hoping we can devise a phase II study that is so dramatic that we can go to (authorities) and say, 'This is something we'd like you to consider for approval.'"

AFP

http://www.theage.com.au/world/how-an-experimental-treatment-saved-emilys-life-20130326-2grvd.html

Chronic disease fears for Australia

Peter Jean
March 29, 2012

An overwhelming majority of Australians have at least two preventable factors increasing their risk of developing chronic conditions such as kidney disease, heart disease, type 2 diabetes and depression.

More than 87 per cent of Australians have at least two risk factors for chronic disease, according to a report to be released today by the Australian Institute of Health and Welfare.

Only 1.5 per cent of Australians had no risk factors and 11 per cent had a single risk factor.

Institute of Health and Welfare spokeswoman Ann Hunt said that only 40 per cent of Australians did enough physical activity to gain sufficient health benefits and 90 per cent failed to consume the recommended amounts of fruits and vegetables each day.

''This is important because we know that people with low fruit and vegetable intake have higher risks of chronic diseases such as heart disease and Type 2 diabetes,'' Ms Hunt said.

The more risk factors a person had, the greater the likelihood that they would develop some chronic diseases.

Men with five or more risk factors were twice as likely to report experiencing depression than men with two or fewer risk factors.

Women with five or more risk factors were three times more likely to experience a stroke and two and half times more likely to report depression than women with two or fewer risk factors.

''People who consume alcohol at risky levels are more likely to report daily smoking than those who don't, and daily smoking is also more commonly reported by those who have insufficient levels of physical activity,'' Ms Hunt said.

''For people who are obese, high blood pressure is more common as a co-risk factor than for people who are not obese.''

The report said that multiple risk factors could result in either the earlier development of a chronic condition, the increased need for management of a condition or death.

''Individual risk factors can affect a person's health significantly, but at what point they do so is not determined.''

The report found that almost 60 per cent of Australians did not undertake enough exercise to incur health benefits.

''Sufficient activity is defined as at least 150 minutes in 1 week over at least 5 sessions,'' the report said.

''A sedentary lifestyle is increasingly recognised as being detrimental to health, as it can contribute to many chronic diseases as well as an increased risk of mortality. More than 80 per cent of Australians spend more than three hours each day sitting during their leisure time, regardless of whether they undertake sufficient physical activity.

Seventeen per cent of males had five or more risk factors, compared to 11 per cent of females.

http://www.theage.com.au/national/chronic-disease-fears-for-australia-20120328-1vz25.html