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The cancer girl cured by the HIV virus: Seven-year-old makes extraordinary recovery after U.S. doctors re-wire her immune system to destroy leukaemia cells

• Emily had been fighting leukaemia for two years and relapsed twice by spring this year
• Her parents put her forward for a clinical trial as it was 'the only option left'
• 'We had 48 hours to make a decision or Emily could have started having organ failure', father said
• Therapy, known as CTL019, used disabled form of HIV to reprogramme her immune system to kill cancer cells

By CLAIRE BATES

In April this year, Emily Whitehead's family had almost given up hope.

The brave six-year-old had been fighting leukaemia for two years. But in February she had relapsed for a second time during intensive chemotherapy treatment.

Doctors had exhausted all the traditional treatments as Emily could not remain in remission for long enough to attempt a bone marrow transplant. So Emily's desperate parents, Kari and Tom, started looking at more radical options.



Fighting fit: Emily (seen left during treatment in April) has now been in remission from cancer for seven months


Big hugs: Emily was diagnosed with acute lymphoblastic leukemia (ALL) in May 2010

'We made the decision that we needed to go somewhere else,' said Mrs Whitehead.

'We needed to try something new, different and cutting-edge.'

So they turned to the Cancer Center at The Children’s Hospital of Philadelphia, which is involved in testing a pioneering new therapy.

Doctors suggested they sign Emily up to a clinical trial that would use a disabled form of HIV to carry cancer-fighting genes into her T-cells (disease fighting cells). The hope was that this would reprogramme her immune system to recognise the cancer cells and start killing them.

Several adults had already been enrolled in the study at the Hospital of the University of Pennsylvania and had responded well but as it was so new the treatment wasn't without risks. But time was running out for Emily, who is also known as Emma.

Mr Whitehead said: 'We were told that we were down to 48 hours of making a decision or she could start having organ failure.'

They comforted themselves with the knowledge that even if the treatment didn't work, it would provide doctors with information that could help them save other sick children.

So on April 17, the then six-year-old became the first child to have the therapy known as CTL019.

Emily during her therapy - at first she suffered an adverse reaction, which was treated with an arthritis drug

Now in remission, Emily enjoys walking her dog who comforted her when she was ill

Emily with her proud parents: 'She has never complained' they revealed
All did not go smoothly at first. The family had been warned Emily could experience flu-like symptoms a few days after being infused with her reengineered T-cells.

However, Emily’s symptoms were far more serious than doctors anticipated. She became critically ill and was admitted to intensive care at the children's hospital. On April 24, doctors told her parents she had a one in 1,000 chance of surviving the night, but she pulled through thanks to their expertise.

Trial leader Dr Stephan Grupp and his team realised that the level of a certain protein had become very elevated as a result of the T-cells growing in Emily's body.

This same protein is involved in rheumatoid arthritis, and there is a drug for that disease that turns off production of that particular protein.

The team administered the drug to Emily, with dramatic results: her condition improved faster than anyone could have hoped for. Almost overnight, her breathing improved, her fever dropped and her blood pressure was back to normal.

Mrs Whitehead said Emily inspired them with how she coped.

'She's extremely smart and creative. She's funny - she makes us laugh all the time. She never complains,' she said.


Emily is very creative according to her parents, and loves playing with her dog

Kira and Tom faced the agonising decision of whether or not to put their daughter forward for a clinical trial

Emily during a consultation with Dr Grupp and her parents Tom and Kari

Dr Grupp led the clinical trial, which was life-saving for Emily
Her husband added: 'She told us from the beginning that she would continue to fight and do what we asked as long as we were there with her. We've stuck together as a team. She's definitely our hero.'

Several weeks after her T-cell infusion, they were able to conduct a bone marrow test to find out if the therapy had worked.

'Three weeks after receiving the treatment, she was in remission,' said Dr Grupp.

'Emily completely responded to her T-cell therapy. We checked her bone marrow for the possibility of disease again at three months and six months out from her treatment, and she still has no disease whatsoever. The cancer-fighting T-cells are still there in her body.'

He added that they need to see the remission go on for a couple of years before they can think about whether she is cured or not. It is simply too early to say.

But for Kari and Tom, it’s incredible to think how much has changed in just a few months. After spending years in treatment, Emily went home in June and now enjoys going to school, playing football and walking her dog Lucy. She’s living life like any other little girl.

'T-cell therapy was really the only option left for Emily,' said Mr Whitehead.

'But we entered her into the trial really hopeful, and from the very beginning we just really had a good feeling about it. So all along we said, "it just has to work, it has to work for Emily" – and it did.'

The scientists said although the results were very promising, much more research needs to be done to see whether the therapy is a viable, safe and long-term solution for controlling certain cancers in children and adults.

But for one family at least, it has provided a new lease of life.

Ken Campbell, Clinical Information Officer at Leukaemia & Lymphoma Research, said the results of the study were encouraging for both children and adults diagnosed with leukaemia.

'Treatments which modify the body’s own immune system to fight leukaemia have shown much promise in recent years,' he said.

'What is significant about [the] therapy is that the severe side-effects associated with this form of treatment seem to be greatly reduced when combined with other drugs.

'This is a small study of just 12 patients. Larger clinical trials are needed to determine how effective this treatment could be and as a result it should be some time before it is available in the UK.'

Researchers from the University of Pennsylvania and Children’s Hospital of Philadelphia presented their latest findings at the American Society of Hematology’s annual meeting in Atlanta.

They found nine out of 12 patients in the trial, which included Emily and one other child, responded to the treatment. Their goal is to treat another 12 patients over the next year.

To read more about Emily's battle visit her awareness page on Facebook

For more information about the trial visit the Children's Hospital of Philadelphia's website



VIDEO: How Far We've Come: Emily's Journey...

How Far We've Come: Emily's Journey from Savannah Smith on Vimeo.
"How Far We've Come: Emily's Journey" photographed and produced by Savannah Smith, The Pennsylvania State University, Visual Communications, 2013. srs0504@gmail.com | Website | LinkedIn

THE PIONEERING THERAPY THAT REWIRES THE IMMUNE SYSTEM TO FIGHT CANCER

Emily Whitehead was diagnosed with the most common childhood cancer, acute lymphoblastic leukaemia (ALL) in May 2010. Unfortunately her case, like 15 per cent of sufferers, was resistant to traditional treatment.

She was put forward for an experimental therapy known as CTL019.

In certain cancers, including the type of ALL that Emily was battling, a subset of cells in the immune system become leukaemia. These are called B cells.

Another set of cells in the immune system, called T-cells, normally recognize and attack invading disease. But in cancers like ALL, the abnormal leukemia cells fly under the radar of the normal T-cells that are meant to kill them.

In the experimental treatment her T-cells were collected from her blood, then reengineered in a lab to recognise and attach to a protein called CD19 that is found only on the surface of B cells. 

To do this they used a gutted HIV virus, called a lentivirus, to carry special receptors into the T-cells.

There is no risk of HIV infection from a lentivirus. 

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When the reengineered cells were put back they dispersed throughout the body to find and kill cancerous B cells.

Emily had an additional drug used to treat rheumatoid arthritis to tackle a side-effect of the therapy and has been in remission for seven months.

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Gene Therapy Cures Adult Leukemia

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CLL Tumors 'Blown Away' in 2 of 3 Patients Given Experimental Treatment

By Daniel J. DeNoon
WebMD Health News

Reviewed by Laura J. Martin, MD

Aug. 10, 2011 -- Two of three patients dying of chronic lymphocyticleukemia (CLL) appear cured and a third is in partial remission after infusions of genetically engineered T cells.

The treatment success came in a pilot study that was only meant to find out whether the treatment was safe, and to determine the right dose to use in later studies. But the therapy worked vastly better than University of Pennsylvania researchers David L. Porter, MD, Carl H. June, MD, and colleagues had dared to hope.

"Our results were absolutely dramatic. It is tremendously exciting," Porter tells WebMD. "These kinds of outcomes don't come around very often. We are really hopeful that we can now translate this into treatment for much larger numbers of patients and apply this technique to other diseases and to many more patients."

Excitement is spreading as oncologists learn about the findings. "I think it is a big deal," says Jacque Galipeau, MD, professor of hematology and medical oncology at Emory University Winship Cancer Center. Galipeau was not involved in the Porter study.

"Here's this guy, the handwriting is on the wall, any hematologist will tell you he is a goner -- this guy was essentially cured," Galipeau tells WebMD. "These genetically engineered cells did what everyone in the field has tried to do for 20 years. The man probably had kilograms of disease in his body, and the cells mopped it up completely."

The treatment uses a form of white blood cells called T cells harvested from each patient. A manmade virus-like vector is used to transfer special molecules to the T cells. One of the molecules, CD19, makes the T cells attack B lymphocytes -- the cells that become cancerous in CLL.

All this has been done before. These genetically engineered cells are called chimeric antigen receptor (CAR) T cells. They kill cancer in the test tube. But in humans, they die away before they do much damage to tumors.

What's new about the current treatment is the addition of a special signaling molecule called 4-1BB. This signal does several things: it gives CAR T cells more potent anti-tumor activity, and it somehow allows the cells to persist and multiply in patients' bodies. Moreover, the signal does not call down the deadly all-out immune attack -- the feared "cytokine storm" -- that can do more harm than good.

This may be why relatively small infusions of the CAR T cells had such a profound effect. Each of the cells killed thousands of cancer cells and destroyed more than 2 pounds of tumor in each patient.

"Within three weeks, the tumors had been blown away, in a way that was much more violent than we ever expected," June says in a news release. 'It worked much better than we thought it would."

CLL Patient Describes CAR T-Cell Treatment

The treatment was not a walk in the park for patients. One of the three patients became so ill from the treatment that steroids were needed to relieve his symptoms. The steroid rescue may be why this patient had only a partial remission.

"Those engineered T cells don't hug the cells to death. They release an array of substances, nasty things that have evolved to clear virus- infected cells from your body," Galipeau says. "But now they are using this to melt down a couple of pounds worth of tumor burden, you will get some side effects."

One of the patients, whose case is reported in the New England Journal of Medicine, described his experience in a University of Pennsylvania news release. The patient chose not to identify himself by name, although he discloses that he has a scientific background. He was diagnosed with CLL at age 50; 13 years later his treatment was failing. Facing a bone-marrow transplant, he jumped at the chance to enter Porter's clinical trial of CAR T cells.

"It took less than two minutes to infuse the cells and I felt fine afterward. However, that fine feeling changed dramatically less than two weeks later when I woke up one morning with chills and a fever," he says. "I was sure the war was on. I was sure the CLL cells were dying."

A week later the patient was still in the hospital when Porter brought him the news that the CLL cells had disappeared from his blood.

"It was working and I was winning," the patient says. "It was another week later that I got the news that my bone marrow was completely free of detectable disease. It has been almost a year since I entered the clinical trial. I'm healthy and still in remission."

Is he cured? Doctors hate to declare a cure until patients have been cancer-free for at least five years. But there are signs the CAR-T cells persist in patients' immune memory, ready to mop up any CLL cells that reappear.

And there's a big downside. The CAR T cells that fight CLL also kill off normal B lymphocytes. These are the cells that the body needs to make infection-fighting antibodies.

As long as the CAR T cells persist -- which may be for the rest of patients' lives -- patients will require regular infusions of immune globulin.

Hope for Cancer Patients, but Treatment Years Away

CLL is the second most common form of adult leukemia. In the U.S. there are about 15,000 new cases and about 4,400 deaths each year.

Cure is possible, but it requires a risky bone marrow transplant. About 20% of patients don't survive this treatment -- and even when they do, there's only a 50-50 chance of a cure.

CAR T cells appear to be a much better option. But the amazing success now reported came very early in the development of this new treatment. Only a few of the thousands of CLL patients facing death will be able to enter the still-small clinical trials testing CAR T cells.

"The distressing thing is the need will far, far, far outweigh any slots in clinical trials," Galipeau says.

But Porter says his team is energized by the early success and is pushing forward as quickly as possible. Even so, a lot of work remains to be done.

"We've treated only a very small numbers of patients," Porter says. "So part of the goal is to see these results in more people, see that the results are sustained, and that it is safe over time. We need to find the appropriate dose and to make incremental modifications. And now we have shown activity, we can try and apply it earlier in the course of the disease. We have reason to think treating patients sooner may be even safer and more effective.

Although the CAR T cells in the study were designed to fight CLL, there's good reason to hope they can be effective in other forms of cancer. The catch is that it can work only on tumor cells that carry markers flagging them for destruction. Normal cells that carry the same markers will also be destroyed.

Many cancers are known to carry such markers, and there's hope of finding more.

"We have a clinical trial at the University of Pennsylvania with an anti-mesothelin molecule [which marks mesothelioma, ovarian, and pancreatic tumors]," Porter says. "There are other trials around the country trying to target renal cell carcinoma [kidney cancer] and myeloma [skin cancer]. We are hoping to identify other tumor targets, particularly in other leukemias, to adapt this technology."

Porter, June, and colleagues report their findings in the Aug. 10 early online versions of two major journals: The New England Journal of Medicine and inScience Translational Medicine.

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