New cellular therapy uses stem cells harvested from an umbilical cord after birth to help boost a patient’s immune system and speed their recovery from cord blood transplants.
Crystal Day received a cord blood transplant in 2014, after she went in for a six-month check-up that revealed leukemic blasts throughout her blood. After three years of being symptom-free, Day’s leukemia had returned.
“I went into shock,” Day says. “They told me that I couldn't do just chemotherapy again. If I wanted to actually treat the leukemia, it would have to be with a transplant.”
Day is of Latino and Caucasian descent. She couldn’t find a bone marrow transplant donor match, and time was running out. Dr. Colleen Delaney, from the Fred Hutchinson Cancer Research Center, approached Crystal about participating in a randomized trial for a cord blood transplant. She received the cord blood transplant at the Seattle Cancer Care Alliance, and almost two years later, she is leukemia-free.
“I’m a lot healthier now with this baby's donated cord blood, because he must have been really healthy,” Day says. “My blood counts are higher than they've ever been in five years.”
Delaney has been championing the virtues of cord blood stem cells for the past 15 years. She says the adaptive, universal-donor nature of the cells, and low side effects from the transplant, have given many patients like Day a second chance at life.
“If you're of mixed-race or minority background, your chances of finding a donor are much … it's much more difficult,” says Delaney. “Cord blood — we can find patients for 99 percent of those people, because you don't have to do that matching so stringently. So for moms who are currently pregnant, and their child is a mixed-race infant, collecting that cord blood unit is much more important for patients like Crystal.”
Delaney says that cord blood transplant patients currently have the same outcome and success rate as a bone marrow transplant recipient. The question then arises: Why bother with bone marrow transplants at all?
“So, why isn't everyone doing it? Everyone isn't doing it because of this issue of the small number of cells in a cord blood unit,” says Delaney. “That means that they are at risk for early complications, and that’s where our product is meant to improve things.”
Cord blood transplants traditionally involve a lengthy engrafting period (when the patient’s body accepts the donor’s blood and begins making its own blood), and recovery period, sometimes up to three months. During this time, the immune system is weak and vulnerable to infection. This is where expanded cord blood cell therapy can help. Delaney says the therapy can speed up recovery time from a cord blood transplant by generating new cells at a faster, earlier rate post-transplant. This translates into lowered risk of complications and infections and shorter hospital stays.
Delaney says that their primary focus is for patients getting a cord blood transplant to receive this product, which will give them an immune boost. Day was one of the first patients in a clinical trial to receive the new expanded cord blood cellular treatment.
“I think patients in the hospital are usually there for a long time — waiting for your blood to engraft — but mine did it in a few weeks, and so I was able to leave the hospital faster,” says Day. “They [the doctors] checked and saw that I was already making blood.”
Delaney says Day represents the overall positive results she’s seen. Data from the initial small clinical trial conducted in 2014, which involved 15 cord blood transplant patients, showed a survival rate of 86 percent for patients who received the expanded cord blood therapy, versus a survival rate of 67 percent of patients who did not.
“One thing I will say is that here at the Hutch we have excellent overall survival,” says Delaney. “So 67 percent in and of itself is an amazing survival rate for cord blood recipients.”
Delaney is in the process of commercializing the expanded cord blood therapy through co-founding NOHLA Therapeutics. While the cord blood transplant clinical trials have been regulated by the FDA, Delaney says commercialization is the only way she can get FDA approval for public distribution.
“If there was no NOHLA, or some entity, I was never going to make it to the FDA on my own as an academic center. That’s not how it works,” says Delaney.
According to Delaney, commercialization will provide worldwide access to a therapy that not only benefits cord blood transplant patients, but potentially many more cancer patients as well.
“If we gave this product to everyone who needs a cord blood transplant, it’s in the thousands,” says Delaney. “But then, if you say, ‘I'm going to give it to anyone who's at risk for an infection from chemotherapy,’ then we're talking tens of thousands who could potentially benefit from this product.”
NOHLA Therapeutics opened its doors in December, 2015. The company is a spinoff of the Fred Hutchinson Cancer Research Center, where Delaney heads the cord blood program. Dr. Delaney will continue her role at Fred Hutch, while also serving as NOHLA’s Chief Medical Officer. The expanded cord blood therapy’s clinical trials are scheduled to begin their final phase in about a year. This trial phase will take about two years to complete, and after that, Delaney says they are optimistic about gaining FDA approval.
“Cell therapy is something I think people are starting to hear more and more about: engineered t-cells, expanded cord blood cells,” says Delaney. “These are all cellular therapies, and it is my belief that cellular therapies are the wave to treatment in the future.”