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Scientists inject the virus into children’s brains in a desperate attempt to reduce 100% fatal tumors

One day Abbie Mifsud behaved listless for the normally bright and vibrant six-year-old.

A week later she was unable to walk in a straight line – and five months later Abbie had disappeared, killed by the aggressive brain cancer, diffuse intrinsic pontine glioma (DIPG).

When Abbie was diagnosed in 2011, her mother Amanda was shocked and surprised that all doctors could offer her daughter palliative care.

DIPG is 100 percent fatal and, like Abbie, half of the children die within a year of being diagnosed.

After losing Abbie, Amanda started a non-profit called Abbie’s Army, which raises money for research into possible treatments.

Today, families face the same opportunities, but scientists around the world are trying new approaches in desperate attempts to offer patients and their families hope or relief.

Scientists connect everything from drugs to eliminate genetic mutations in the disease to even injecting viruses into the brains of young children, according to studies presented by the American Association for Cancer Research.

Abbie Mifsud (photo) died of DIPG at just six years old, just five months after she was diagnosed with the 100 percent fatal form of pediatric brain cancer

Abbie Mifsud (photo) died of DIPG at just six years old, just five months after she was diagnosed with the 100 percent fatal form of pediatric brain cancer

When it became clear to the Mifsud family that Abbie’s problem was neurological and serious, they were ready for a fight, but of course wanted to know her chances.

“When I asked the consultant the inevitable question, [I thought] because Abbie was so lively that if there was a chance of a percentage that someone would make it, she would be one, “Amanda told Daily Mail Online.

“She is so strong and so smart. I asked: “is it 50/50?”

Amanda laughs sardonically.

“No, no, there is no such thing. It turned out to be a big zero, “she says.

“They call it a fight, but it’s a fight that you can’t win right now.

“I got a panic attack on the bank there. I looked at [the consultant] and said, “What, nobody survives this?”

“And he just looked straight at me and said,” no. ”

DIPG, or diffuse intrinsic pontineglioma, is a rare type of tumor that accounts for between 10 and 20 percent of childhood cancers.

It is fatal within five years for more than 99 percent of children with the devastating disease.

Researchers from the University of Navarro in Pamplona, ​​Spain, believe that their modified virus can change that – but experts say that, like most treatments, the experiment is promising, but still could easily fail, as many previously .

Supporters of parents from all over the world have joined forces to raise funds for groups such as the Institute of Cancer Research (IRC), based in the UK, that pursue virtually every possible therapy for DIPG.

The group is currently focused on genomically sequencing DIPG tumors to develop better targeted therapies, rather than just blindly causing cancer with a garden variety drug that has worked with adult brain tumors.

DIPG is one of the few cancers that has remained virtually untouched by medical progress.

After Abbie's death in 2011, her parents Ray and Amanda (left, right) started Abbie's army and tirelessly raised awareness and money to support DIPG research

After Abbie's death in 2011, her parents Ray and Amanda (left, right) started Abbie's army and tirelessly raised awareness and money to support DIPG research

After Abbie’s death in 2011, her parents Ray and Amanda (left, right) started Abbie’s army and tirelessly raised awareness and money to support DIPG research

While the explosion of immunotherapies and targeted therapies have given years back to even older patients with stage four lung and breast cancer, half of young children with DIPG still die within a year.

DIPG is a type of brain tumor that begins to grow in the brainstem – that plays a vital role in everything from breathing and heartbeat to walking and talking – and spreads aggressively through some kind of fat brain cells.

It usually develops in children between five and ten years, although adults occasionally also get cancer.

The symptoms are often rather vague, which can delay the diagnosis. Abbie, for example, just took it off and wobbly and seemed to lack energy, her mother said.

Initially, the family was told that they should return to the doctor within six weeks if her symptoms worsened. But with DIPG, even an early diagnosis would not allow patients to receive treatment.

The brainstem is so expensive that surgeons cannot operate on tumors there. Until recently, they could not even be safely subjected to a biopsy.

Instead, DIPG is usually treated with radiotherapy.

For 70 percent of patients, this shrinks the tumors and helps relieve symptoms such as vision and hearing problems, difficulty controlling facial muscles, including swallowing, difficulty walking, nausea and vomiting.

But the tumors always overcome the effects of radiation.

Abbie immediately started radiation therapy after her diagnosis. The Mifsud family had to travel 150 miles each day from their home in Ashford, Kent, to the hospital where Abbie received treatment.

Amanda thought that if someone could beat the odds, this would be her “smart, lively” daughter Abbie (photo). But then she was told that there was no chance that her daughter would survive

It was exhausting, tiring and “you know it’s only to get them back,” says Amanda.

“It is soul-destroying that you do it and it is not curative.”

And worst of all, it was clear to Amanda that her daughter knew what was happening to her.

Over the last 30 years, scientists have tried to treat DIPG with chemo, but to no avail. None of the different chemotherapy therapies they tried even offered benefits over radiation.

For about six weeks, radiation seemed to relieve some of Abbie’s symptoms.

But when its effects began to decrease, it began to decrease immediately and dramatically.

The Mifsud family spent about a week in the hospital.

As the tumor continued to infiltrate Abbie’s brain, she lost the ability to swallow, so Amanda and her husband, Ray, had to suck fluid from her throat to prevent them from “drowning” in secretions, Amanda says.

‘Bless her, she has actually become a head on a pillow.

“You have to ask yourself:” how bad must it get before you want them to go? ”

After a week in the hospital, the Mifsuds took Abbie home and called her family to say goodbye.

Cancer has been a scourge for Amanda’s family, where her father (left) died of lung cancer after her daughter died of DIPG. She calls the two “her angels”

Abbie had a home there last night, nestled among her parents. By the early morning, on September 13, 2011, she was gone.

“It’s unbearable – there’s no other word for it,” says Amanda.

But although some families have tried to get as far away from the disease as possible, Amanda and Ray leaned to the DIPG community and started Abbie’s army.

“What compelled us to do it was that it didn’t make any sense to us that our daughter was here who would die in a very short time and would not be given any medicine to treat the disease,” says Amanda.

There are more than 40 clinical trials recruiting DIPG patients, according to the US-based DIPG Registry. In the UK, Amanda says that only a few such studies are actively seeking patients.

Most research on DIPG is still in the pre-clinical phase, when scientists develop therapeutics by studying cells or animal models.

Research often shows a remarkable promise at these stages, but fails to treat children with DIPG.

For example, Spanish scientists are injecting tumor-killing viruses into the brains of children with the incurable pediatric cancer, DIPG, according to research by the American Association for Cancer Research.

The team uses a modified adenovirus to kill tumor cells. The virus also increases an immune response and stimulates attacks against the cancer.

The location of DIPG in the brain stem makes it difficult for immune therapies – usually administered via an IV or pill – to reach the fast-growing tumors.

To circumvent that problem, the scientist devised a long, thin tube that is drilled into a small hole in the skull and subtly navigated to the tumor to bring the drug directly to its target.

The effects of therapy in mice are remarkable, even curing the tumors in some iterations.

So far, the group has only recruited eight of the 12 patients they are looking for for a phase 1 study.

At this stage they are not looking for how effective the treatment is – how toxic it is.

Abbie received radiation therapy for six weeks, making it easier for her to walk, talk and swallow, but the effects are temporary. She died five months after the diagnosis

Abbie received radiation therapy for six weeks, making it easier for her to walk, talk and swallow, but the effects are temporary. She died five months after the diagnosis

Abbie received radiation therapy for six weeks, making it easier for her to walk, talk and swallow, but the effects are temporary. She died five months after the diagnosis

They found no toxicity, two patients recruited when their tumors were already large and invasive died and one stepped out of the study.

The five remaining patients have so far seen a reduction in the size of their tumors – but none of them have been in the study for more than 28 weeks (seven months).

Dr. Martinez Velez says that one patient is still alive even 12 months after the virus is injected and there are no signs of tumor presence.

It is a new approach to the DIPG problem, but the treatment is not yet tested for efficacy, only for safety.

Dr. Chris Jones of ICR did not work directly on the study, but contributed to the genomic sequencing that formed the trial.

Dr. Jones calls the research “a potentially interesting path,” but notes that what happens in mice does not necessarily happen in humans.

Amanda has spoken with the researchers, but does not yet want to draw conclusions.

“It’s just too early, it’s another way that needs a lot of research,” she says.

She notes that, like many other treatments that have failed in children with DIPG, this method is based on a method used in adults.

“We have this entire field of research that is completely dependent on studies in adults,” but often does not translate.

“It is another area that is worth investigating, but we still cannot say whether there is an extension of the lifespan or an improvement in life expectancy.”

She is more encouraged to create a treatment in light of the discovery of a mutation in the tumors associated with the ALK2 proteins.

Treatments under development regarding the mutation are “biology based,” she says, rather than shooting in the dark.

A group called Medicines 4 Kids (M4K) Pharma has made rapid progress toward a therapeutic agent that blocks the tumor-inducing activity of ALK2, but it may take a year or more before they can begin clinical trials.

This is the frustration for the families who are the driving force – and funding – behind the DIPG study: everything takes time and so much research is still in its infancy.

“It’s a huge challenge, because on the one hand you want to offer treatments that are data-driven,” says Amanda.

“But for a parent who is actually trying to save a child, you would do anything.

“If someone had said to me,” Cut yourself in half and it will work, “I may have done it.”

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