Young girl’s ‘cure’ signals new path against cancer

Source: TheNews.com.pk

PENSYLVANIA: 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.

Her mother said Emily sometimes grapples with her newfound 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 told AFP.

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.

Novartis is also funding a $20 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 recognize 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 told AFP.

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 specialized 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.’’

 

School Celebrates Prom in Honor of Teen With Cancer

Source: ABC

Disco balls hung from the ceiling, balloons and other decorations covered the room and teens in suits and dresses milled about. There was a blue cake with the night’s theme, “Katie in the sky with diamonds” iced in white.

At the center of it all was crown-wearing Katelyn Norman, 14, who was presented with a “Prom Queen” sash from her white suit-wearing date.

It would be just like every other prom except that this one took place in a hospital room and the prom queen wore an oxygen mask as she lay in a bed.

For one magical night, Katelyn’s hospital room was transformed into the bucket list prom she had wished for, even if it wasn’t the prom that was going on in another part of town.

Katelyn is dying from osteosarcoma, an aggressive bone cancer. After fighting the cancer for two years, she was told last week that it has spread and there’s not much more doctors can do. She was sent home to spend her last days.

She made a bucket list that included a prom, a last slow dance, learning to drive a car, seeing Italy and a day with each sibling.

A fundraising page to help pay for Katelyn’s wish list has produced an outpouring of affection and generosity towards the teen. The page has raised more than $62,000 since Tuesday morning.

Katelyn’s prom was planned for Tuesday night in LaFollette, Tenn., but during the day Katelyn was having difficulty breathing and had to be taken to the hospital. Doctors told her she couldn’t go to her special prom, but she didn’t want that to stop others for going.

“Katelyn wanted the prom to go on. That’s her. That’s Katie bug,” Sharon Shepherd told ABCNews.com today.

Shepherd works at Campbell County High School, where Katelyn is a freshman. She has known Katelyn since she was 5 and has grown even closer to the teen since her diagnosis in eighth-grade.

Shepherd calls Katelyn “my little short and sassy Katie bug” and says she considers Katelyn “one of my own.”

Thousands of people lined highway 63 with candles to show support for Katelyn in an event called “Light the Night for Kate.” Another crowd gathered outside her hospital, standing in a heart formation.

“They wheeled her over to the window and propped her up to where she could wave outside to the crowd,” Shepherd said.

Meanwhile, the planned prom went on and some of the guests closest to Katelyn were told they could go to the hospital afterward to celebrate with her there.

Shepherd was told that Katelyn was “being her spunky self” at her hospital prom, talking and laughing with her guests.

Her best friend Brandon Huckaby planned “Light the Night for Kate” and helped run Tuesday’s events. At one point, Campbell County Mayor William Baird surprised the community with a special proclamation, declaring Tuesday Katelyn Norman Day in Campbell County.

Later on, Huckaby headed to the hospital to see his friend.

“Kate reached for me and pulled me against her. She made me promise to start the nonprofit that I talked to her about earlier this month,” Huckaby told ABCNews.com in an email today.

“She then told her mom to tell me about Nashville,” he wrote. “Apparently, Kate and her family were invited to the capitol to advocate for cancer research. Kate had told her mom before that if she doesn’t make it, then she wants me to go in her place and speak to the politicians on her behalf. As you can imagine, it was emotional.”

Dying Teen’s Bucket List Create Outpouring of Affection and Donations
An outpouring of donations have been made to Katelyn’s fundraising page in the past day. Donations will be withdrawn from the site on April 2. The check will be sent to Katelyn and her family, along with copies of all of the messages of support.

The website, GoFundMe.com, takes a percentage from each donation, but the rest of the money will be sent to the family.

“It was just wonderful how the community rallied,” Shepherd said. “It’s almost breathtaking. It’s overwhelming the impact she’s had.”

Shepherd said she does not yet know what the family’s plans are for the money or what their situation is with expenses or insurance. She said the family’s main focus right now is on Katelyn and her health.

One of the other items on Katelyn’s bucket list was to see the band Of Mice & Men in concert and get an autographed T-shirt from them. The band’s fans flooded their Facebook page and Twitter accounts with links to Katelyn’s story, urging them to reach out to her.

“Dear Katelyn Norman, our set was for you tonight,” the band’s lead vocalist Austin Carlile tweeted Tuesday night. “My mind couldn’t stop thinking about you the whole time. You inspire me, and I love you <3"

"NYC I'm sorry for the show. My head was somewhere else the entire time and the last thing I could think about was music. I'm so heartbroken," Carlile tweeted later in the night. "KATELYN is an inspiration to me. I didn't even want to be on stage tonight. I wanted to be with her."

Donations to Katelyn's bucket list fund can be made here.

DNA test reveals 80 markers for inherited cancer risk

Source: BBC

This chip was used to identify genetic markers

More than 80 genetic markers that can increase the risk of developing breast, prostate or ovarian cancer have been found in the largest study of its kind.

The DNA of 200,000 people – half of them with cancer and half without – was compared, revealing an individual’s inherited risk of the diseases.

British scientists, who led the research, believe it could lead to a DNA screening test within five years.

They also hope it will boost knowledge of how the cancers develop.

The research was led by scientists at the University of Cambridge and the Institute of Cancer Research (ICR) in London and funded by Cancer Research UK (CRUK) and the Wellcome Trust.

The main findings are published in five articles in the journal Nature Genetics.

Study author Prof Doug Easton said: “We’re on the verge of being able to use our knowledge of these genetic variations to develop test that could complement breast cancer screening and take us a step closer to having an effective prostate cancer screening programme.”

Inherited cancer risk

The scientists looked for common genetic variations – known as single nucleotide polymorphisms (Snps) – linked to the three cancers.

Each alteration raised the risk of cancer by a small amount. However, a small minority of men with lots of the markers could see their risk of prostate cancer increase more than fourfold and for women the breast cancer risk increase threefold.

By contrast, the test can also identify those with a smaller than average risk of developing the cancers.

A woman’s lifetime risk of breast cancer is one in eight, but among the 1% with lots of these newly identified genetic variations the risk rises to one in two.

The test could also help the one in 300 woman who carry a faulty gene known as BRCA1 or BRCA2. Two-thirds of them will develop breast cancer before the age of 80 and 45% who carry BRCA1 will get ovarian cancer.

At present the options to reduce their risks are limited – a double mastectomy or having their ovaries removed.

By combining the gene test for BRCA1 and BRCA2 with this extra genetic information, women who have a high number of the newly identified markers could find they have a nearly 100% risk of getting breast cancer.

In contrast, those with the protective versions of the genetic changes could see their risk drop to as low as 20%.

Dr Antonis Antoniou, CRUK senior fellow at the University of Cambridge, said: “Our research puts us on the verge of being able to give women a much more accurate picture of how likely they are to develop breast or ovarian cancer and would help to guide them about the most appropriate type and time of prevention or monitoring options for them.”

Prostate

For men, the lifetime risk of developing prostate cancer is one in eight. But for 1% who carry a significant number of genetic alterations, the risk rises to one in two.

Unlike for breast cancer, there is no screening programme for the disease.

The prostate-specific antigen or PSA test, looks for protein markers in the blood and high levels may be an indicator or prostate cancer. But it is unreliable.

Furthermore, about two-thirds of men who get prostate cancer have a slow-growing “indolent” form of the disease that will not kill them.

Treatment options include prostate removal, radiotherapy and hormone treatment, But for every life saved through treatment for prostate cancer, it is thought that between 12 to 48 men are treated unnecessarily. Many patients opt for “watchful waiting”, monitoring the cancer.

Sixteen of the 23 newly identified genetic markers are associated with aggressive forms of the disease so may help clinicians and patients decide on the best form of treatment.

Prof Ros Eeles, from the ICR, said: “These results are the single biggest leap forward in finding the genetic causes of prostate cancer.

“If further studies show such men benefit from regular screening, we could have a big impact on the number of people dying from the disease, which is still far too high.”