In a lab in Philadelphia doctors are re-engineering 9-year-old Rachael Weinstein’s T cells, teaching them to eradicate her leukemia from within.
In a few short weeks Rachael will receive an infusion with her new, stronger cells and then the wait will begin.
She is only the 18th patient in the world to participate in the CART19 trial, but the results have been so promising, her parents Angelique and Craig are hopeful that this will be what will defeat their daughter’s cancer once and for all.
If all goes well Rachael could even be in school on the first day of class this fall.
“Our goal is to have her in school Sept. 3,” Angelique said.
Rachael was first diagnosed with Acute Lymphoblastic Leukemia in February 2012 after she complained of foot pain.
“From there we just started with the standard treatment for leukemia as every child does,” Angelique said. “After a months time they recheck to see where the children are and Rachael was not at the numbers that they like which put us in a high risk category.”
The standard treatment for ALL is two and a half years of intense chemotherapy or a bone marrow transplant.
“We were fortunate that Rachael had a lot of potential matches for her and a lot of families don’t have that option,” Angelique said.
Angelique and Craig opted to go for the transplant as children who receive transplants have a higher rate of achieving long-term remission.
In May 2012 Rachael achieved remission, which is a condition for a transplant, and a month later received the transplant.
“It went well, we had great success, we were passing all the markers, all the doctors were incredibly impressed,” Angelique said. “Then in January she started complaining of foot pain again which had been one of her markers before.”
By March Rachael had officially relapsed.
“We could do another bone marrow transplant with her but we aren’t ready to go down that path with her,” Angelique said.
Angelique and Craig were looking at other options and were considering what studies they could participate in. They heard about the CART19 trial at Children’s Hospital of Philadelphia, which is specifically for children with ALL, through a number of sources before they ever thought they’d need it. Once they knew Rachael had relapsed it became one of their top choices and Rachael met all of the study criteria.
“We are fortunate that we have a plan A and a plan B,” Angelique said.
Another transplant is still an option for Rachael. For many families in the study, she said, it is a last resort because they can’t achieve remission and therefore can’t do another transplant.
Rachael began chemo again in April and once again has achieved remission, but Angelique said they know it is only temporary.
“They can’t find any leukemia cells in her marrow currently but she’s been there before, they’re hiding somewhere in her body,” Angelique said.
Requirements for the study included a relapse of ALL, no cancer in Rachael’s central nervous system, positive B cells — all conditions that Rachael met — and then a preliminary trip to Philadelphia.
In May Rachael and her mom traveled to Children’s Hospital of Philadelphia so her T cells could be collected and the doctors could test her cells to make sure they would grow outside of her body, as they have found that not everyone’s do.
“Rachael’s T cells grew phenomenally,” Angelique said.
When Rachael and her family, including her big sister Aureliana, 11, travel back to Philadelphia later this summer Rachael will receive up to four days of chemo and then the infusion of her new T cells.
“It’s not even a procedure, it’s an infusion, they just put the cells back into her,” Angelique explained.
Angelique said they chose to participate in the study because it focuses on teaching the body to fight the cancer from within, without the side effects and limitations that patients who undergo a transplant face.
“We’ve been living that life already, we’re still living that life for us,” Angelique said. “Our daughter is 9, she needs a life. She needs to go back to school, she needs to have friends again. With CART19 she can do that. She can go back to school in September.”
The last few weeks leading up to the trip are filled with packing and spending time with family as well as looking forward to a future without cancer.
So far there are studies published on the first eight children. For two of them, the trial was unsuccessful — one had lymphoma and one had B negative cells, Angelique explained.
“It means hope,” Angelique said about the study. “It makes sense to us, fighting cancer from within. We’re really excited about this opportunity.”
Angelique said they look forward to participating in a study that can also offer hope to other families.
“Ultimately we just want Rachael to be a 9-year-old again,” Angelique said.
To follow Rachael’s journey visit www.facebook.com/RallyingforRachael.