|Dr. June - Univ Penn|
Early in 2013 I was researching the topic of Immunotherapy for a piece I was putting together for a creative non-fiction writing competition at Eurostemcell.org.
This work was driven by a personal interest in new effective solutions for Cancer. I came across an engineered T-cell program that had some signs of meaningful efficacy results in a few adult patients at the Hospital of the University of Pennsylvania and had been extended to enroll children with relapsed Leukemia at the Children's Hospital Philadelphia (CHOP).
|Dr. Grupp - CHOP|
I wrote the below as the competition entry and later published it on iCell. Although I didn't get the nod by the panel at Eurostemcell it was a cathartic project for me and one which I can look back on as rewarding.
By having a finger on the pulse of some of the next steps in medicine I can relay and communicate. This enables me to further explore my interest in the science and add my energy to the advocacy for such potentially effective treatments. This is why I write & report about the science - specifically cell related science.
The CAR-T and other modified Immune cell technologies are now gaining traction as a result of the early results of the technology pioneered by Dr. June. Many of those lucky enough to gain enrollment in the now many clinical studies underway are alive as a result. That is what matters - traction for cures.
Emily was the first child and helped bring us all closer to the science as a result of her courage and spirit.
Media loves a good story & Emma naturally became the poster child for genetically modified cellular cures.
The science has shown its potential and now has the funding to drive it forward & bring the needed treatments to market.
“In Their Oath We Trust”
I was told of Emily’s case before she arrived at the hospital by some of my coworkers on the floor that said she was 6 years old, had twice failed chemotherapy for Leukemia, and was coming over to see a specialist.
I couldn't imagine being her Mother as I first saw Emily enter our ward, knowing that she didn't stand a decent chance of making it out of here alive. As a nurse I have all too often seen children and their parents swing open the double doors to our corridor, with hope in their step, only to leave one less and heart-broken.
We have a 6 year old little girl of our own at home but still I can’t imagine what it would be like to be here as a victim of Cancer.
As Parents, our first and only thought is to fight for our young. That fight is sometimes futile to all but us and a few professionals willing to try something new. Needless to say we all have the right to fight, in whatever manner available. It is a Parent’s right to decide the level of acceptable risk for our children and to seek out those willing to help restore hope.
This is the story of a struggle between the known and the unknown, of a brave little girl named Emily “Emma,” who had the will to live in the face of death. A desperate battle that too many of us in the medical community have seen countless times but are without the tools to fight.
Emma couldn't have been more normal. She told me often of her simple joys of discovery, of play and her friends. She showed me pictures of when she was carefree and of her family and school. Those images revealed the secret – her dark brown fluid eyes, alive with depth and intelligence. Even to this day I still cherish the photos she left me, as they bring back memories of her inner strength that I myself need sometimes. Her courage and determination was something she was truly blessed with.
Emma was diagnosed with Acute Lymphoblastic Leukemia (ALL) soon after her 5th birthday, the most common form of Cancer in children and tragically seen mostly in the very young, those between 2 and 5 years old.
From one day to another the Leukemia in Emma’s veins multiplied and showed themselves in symptoms that signaled something was terribly wrong… Within a short time Emma was being treated at the University of Pennsylvania’s Hershey Medical Center with the current standard for her Leukemia – Chemotherapy until remission.
Emma was unlucky to be one of the cases where her Cancer was resistant to Chemo. Cancer comes in too many varieties but one which is particularly devastating is unresponsive Blood Cancer in children – Refractory Leukemia – as it almost always kills…
There wasn't much the Hershey Doctors could do once Emma failed Chemo a second time and a Bone Marrow Transplant was ruled out, other than to suggest a risky experimental approach. They referred the case to Dr. Grupp at our Institute, The Children’s Hospital of Philadelphia (CHOP). Dr. Grupp was to be in charge of leading a novel clinical trial using modified T-Cells to treat Emma’s form of Leukemia…
The scientific community at large is tasked with developing treatments and we, the medical practitioners, administer those treatments to the suffering but all too often we don't have a solution. As a nurse I see both sides of the equation and can tell you the Doctors go the distance, taking home a heavy burden, day in and day out. Treating the suffering is truly a calling – but when it comes to getting a possible new approach into the clinic and approved it first must be tested, and tested again, and again and again…..
When Emma walked through those swinging doors with her Parents, her shinny brown hair had fallen out, her skin color had turned grey and her fluid brown eyes had sunken – she was a Cancer victim – outwardly as a result of the toxic chemicals but inwardly as a result of her cancerous cells. Yet they still had hope and put their trust in us….
Emma and her Parents made the only decision they could – they accepted Dr. Grupp’s offer to enter Emma into his experimental trial, to be conducted on our ward. As the first child to receive genetically modified T-Cells, using a deactivated HIV/AIDS virus as modifying vehicle, she was to make medical history. A treatment so revolutionary its design could perhaps be used to treat many other Cancers.
What happened next was straight out of a Sci-Fi film, the battle was on and Dr. Grupp and his colleagues extracted Billions of Emma’s own T-Cells, modified them with the virus and enlisted them into the war. It was a drafted army with a new Cancer killing antibody as a weapon. The cells were dripped onto the battlefield slowly, but constantly, to seek out and kill the Cancerous rogues that had taken over Emma’s system.
Drop by drop the newly equipped warriors multiplied and fanned out in search of their deadly enemy… Emma soon began to react to the battle that raged inside her, burning up at 105F/40.5C and dropping in blood pressure to near death levels. She went into bodily shock, as the armies inside engaged in hand-to-hand combat in a bioreactor like immune system storm…
Dr. Grupp was unable to control the fallout. We watched from the sidelines as the trauma teams circled Emma. She was rushed to intensive care and the diagnostic department went into overdrive to try to find a solution to save her life…
Emma’s family and friends filled the corridors, gathering for the end, as her body distorted, burning up and hardly able to sustain a heartbeat… Death had come knocking and there wasn't anything we could do…. For a week Emma fought for her life in that condition, teetering on the brink in the ICU. The synthetic army was on her side but the battle was causing too much collateral damage and it was clear it was only a matter of time until her will to survive would run dry…
Just when we all thought the worst, a rumor started to circulate amongst us that the Doctors had found a way to potentially stop the effects of the chaos and restore Emma’s immune system balance. We administered an off-the-shelf drug to regulate the immune cell expression level IL-6, which they had detected was one thousand fold above normal…
No one had ever used the drug in such a critical situation but within hours of receiving it Emma stabilized… We had never seen anything like it – from one hour to the next she began her counter-offensive… Within a few days she was awake, a survivor of the ultimate fight.
It seems unreal to take T-Cells out of your body and genetically modify them to specifically target and fight other immune cells. That for much of the medical community was Science Fiction, including me, until now. Now it’s just good science which all in need deserve access to.
It’s become an unspoken thought, that one day soon we too can stand up and say the word – cure – without reservation.
For Emma, I believe, it was simply her destiny to face death, something she was born to do – bravely without fear – for us all to know the power of our own cells.
Cytotoxic T-Cells Killing #Cancer
NIH Sponsored Short