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Seattle Children’s immunotherapy trials offer new hope

There is a tremendous effort to find a cure for pediatric cancer that doesn't include the harsh side effects of traditional therapies.

Scientific studies with names like 'Brain Child' and 'Strive' are the tip of the cancer-fighting spear at Seattle Children’s.

Families travel great distances seeking help for their children who have relapsed from chemo, radiation and bone marrow transplants.

One success story is Bernie Mier, who came to Seattle Children’s for help, with his sister and parents.

“Everything started two and a half years ago when Bernie was diagnosed with acute lymphoblastic leukemia. He was eleven months old,” said Paola Renpening, Bernie’s mother.

Nearly three-years-old, little Bernie has been through much more than a lifetime of medical care in the last 2 and a half years.

“We started the treatment just under treatment, and he actually started doing very good, right. He got into remission after 30 days which was standard protocol, but then. Five months later he relapsed,” explains Paola.

That initial treatment took place in Mexico City where Paola and husband Leon live; both had careers, a home, family, and friends, but all that is on hold.

“Whenever you have your son with cancer, you need to spend 110 percent of your time and energy on his treatment, but also you leave other things aside and things that really matter like a sister, like work, like the relation between us,” says Leon Mier, Bernie’s father.

After that relapse, Bernie’s next step was a bone marrow transplant. Unfortunately, Mexico does not participate in a donor registry. But, with the help of their oncologist, they located a match in Texas where the transplant took place.

Unfortunately, after a couple of months, Bernie relapsed again.

Their story is not uncommon; some children don’t survive.

A lucky few find their way to immunotherapy trials.

The family was referred to Seattle Children’s where they were able to modify Bernie’s T-cells in the lab, and after being reintroduced the new CAR T-cells attacked leukemia in his blood.

“He has a type of leukemia that we think has a higher risk of outsmarting the T-cells. And so, for the best chance for him to stay in remission long term we believe is to proceed with a stem cell transplant again now that he's back in remission,” explained Dr. Rebecca Gardner, an attending physician at the Cancer and Blood Disorders Center, at Seattle Children’s.

Researchers at Seattle Children’s discovered children who get re-engineered T-cells and get a bone marrow transplant experience great remission success.

“What's really remarkable about CAR T cells is that it gets you into remission and it happens fast. So, you know one to three weeks after you've received your car T cells you're in remission, and then your body has a chance to heal itself,” says Dr. Gardner.

“He got the cells on September 14, which are basically his own cells just fortified and they started to do this magic inside his body,” says Paola.

Bernie’s mother and father are all smiles because of how Bernie has recovered.

And it’s because of years of work on trials that have built upon each other that doctors have the tools and knowledge to boost survival rates.

“In terms of our studies now we have PLAT 02, 03, 04, 05 and 06 for leukemia. And then we have two trials open for brain tumors, and another two trials have been for solid tumors. So, we're really trying to be able to capture all of our pediatric malignancies,” says Dr. Gardner.

Dr. Gardner admits the progress at Seattle Children’s has been astonishing.

They refer to it as bench to bedside, back to bench, back to bedside.

She’s talking about real-time adaptation as doctors work closely with researchers to study therapy results and strategize ways to outsmart cancer.

“T-cells are most happy when they can attack something so the CAR T-cells, in this case recognize a protein called CD 19, when you get rid of all the leukemia in your body, and you get rid of your normal B cells which also express CD 19 there's not much for the T-cells to go after and this sometimes causes this T-cells to go away too quickly. And so, our vaccine strategy, we actually take T-cells from the patient that we collected when we made their CAR T-cells and instead of modifying them with a CAR (chimeric antigen receptor), we modify them to express this protein CD 19 and then we can give these cells back to the patient. It's a nice little meal for the CAR T-cells that are there, where they can say, Oh here's something for me to go after, I can expand now, I'm happy again, and I'll stick around. The initial results that we've seen from that are really encouraging, and we have patients who we would have predicted their CAR T-cells would have gone away after two months. And now our initial patient is about ten months out and still has T-cells,” explains Dr. Gardner.

That example is one of many where pediatric oncologists adapt and build upon already established therapies.

“I think we are doing remarkable, in taking our early findings from our clinical trials and quickly turning them around to make new clinical trials. We really think that’s the way to push this field forward. So, ten years from now we can say your child has A.L.L. (acute lymphoblastic leukemia), we're going to give them some CAR T-cells and then they go back to school because that's what we all want to get to, to say here's this little bump in the road and I think we are well on our way now to getting there,” says Dr. Gardner.

Which will hopefully allow mother; Paola, father Leon, little sister Cata and almost 3-year-old Bernie to go back home and get back to a life where the cancer was just a bump in the road.

“Other than faith that is the first thing that you need to have is resilience. And once when you have faith, and you have resilience and some love for your family. I mean, you can tackle a lot of things,” says Leon.

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