Industry leaders lay out vision for the future of blood cancer treatment

Biopharma leaders at companies developing blood cancer therapies say we shouldn't be afraid to use the four-letter word: cure.

That word came up two dozen times during a panel discussion with representatives from GlaxoSmithKline, AstraZeneca, Regeneron, and Johnson & Johnson at STAT@ASH, STAT's event at the American Society of Hematology annual meeting. Speakers pointed to an expanding tool kit of strategies to combat blood cancers — including CAR-T therapy, bispecifics, antibody-drug conjugates, and other approaches — that is allowing drug developers to shift their thinking from treating patients until their disease progresses to treating to cure.

"Can we give better tolerated drugs in earlier lines of therapy?" said Andres Sirulnik, leader of Regeneron's hematology clinical development program. "Can we potentially cure with these new technologies and drugs that before we couldn't even imagine?"

He added there are early signs the answers to these questions could be "yes," including initial data Regeneron will present suggesting that Orspono, a bispecific antibody, has potential to treat newly diagnosed patients with follicular lymphoma.

Mark Wildgust, J&J's global vice president of oncology medical affairs, noted that the company will present data at ASH showing that Darzalex — an antibody drug that brought in more than $9 billion in sales last year — significantly reduces the risk that people with smoldering myeloma, a condition that can presage multiple myeloma, go on to develop the cancer.

Susan Galbraith, AstraZeneca's executive vice president of cancer research, pointed to the pharma giant's progress in developing cancer drug cocktails that can be used for defined durations, a feature that could appeal to younger patients averse to being on a treatment regimen for life. And GSK highlighted the dramatic rise, fall, and potential return of Blenrep, an antibody-drug conjugate the company has developed for multiple myeloma. (Keep an eye out for a story on our site tomorrow about Blenrep's survival benefits, as well as a look at J&J's smoldering myeloma readout.)

Bone marrow transplants can be transformative for AML patients — if they can get them

For patients with acute myeloid leukemia, or AML, a bone marrow transplant can be lifesaving. But research presented at ASH provides a detailed view of how this therapy is out of reach for many patients from disadvantaged communities, who are also more likely to die without the treatment.

A scientific team led by Fred Hutchinson Cancer Center tracked data across five medical centers and found that patients from ZIP codes with lower levels of education had 33% lower odds of receiving a bone marrow transplant compared to people from more-educated and wealthier communities. But among patients who were able to receive a transplant, survival rate was not significantly linked to socioeconomic status.

"If patients are able to overcome the barriers to transplant … perhaps the outcomes could be comparable to patients from other socioeconomic backgrounds," Natalie Wuliji, the study's lead author and a hematologist-oncologist at Fred Hutch, told STAT's Jonathan Wosen.

Socioeconomic status can create access barriers for patients in a number of ways. Receiving and recovering from a transplant can take months, and during that time patients must stay near a treatment center and have a caregiver. That means time away from work and, often, paying for a hotel or other accommodations.

AML is the world's most common reason for a bone marrow transplant, and the research team now plans to explore better ways to screen which patients face the greatest socioeconomic barriers and test strategies to help them get treatment.

Study of 9/11 first responders finds a higher risk of blood cancer — and a potential new treatment target

The attack on the World Trade Center exposed hundreds of thousands of people to toxic dust from pulverized building and airplane material, and the World Trade Center Health Program estimates over 30,000 individuals have developed cancer as a result. A study of first responders to the attack is helping to uncover how this toxic exposure is leading to more cancers, and it may have also identified a potential target to treat a common precursor to many different blood cancers — mutations in blood stem cells known as clonal hematopoiesis mutations.

To do this study, scientists at Albert Einstein College of Medicine sequenced blood from about 1,000 firefighters who responded to the attack, as well as blood from firefighters who didn't respond to the attack and non-firefighters. Divij Verma, the lead author and a cancer researcher, found that first responders had a roughly three-fold increased risk of clonal hematopoiesis mutations compared to the other groups. These 9/11 responders who had clonal hematopoiesis were also three times more likely to develop any blood cancer.

In another experiment, Verma exposed mice to dust collected from 9/11 and found these animals also developed more clonal hematopoiesis than mice who weren't exposed. Verma noticed that these mice were experiencing "enormous inflammation," he said. "And the receptor that senses that inflammation was IL1RAP, which was significantly upregulated on mutant cells."

When Verma repeated the experiment with mice with an IL1RAP deletion, he found that cells with a certain clonal hematopoiesis mutation grew far more slowly. That might mean that IL1RAP could be an important target in tackling clonal hematopoiesis and potentially preventing or delaying the development of certain blood cancers and other diseases.

Experts call Pfizer 'tone deaf' for sudden withdrawal of sickle cell drug

Pfizer's abrupt withdrawal of its sickle cell drug Oxbryta was criticized as "tone deaf" by experts at this weekend's STAT@ASH panel — highlighting systemic flaws in treating this historically neglected disease. Approved in 2019 and hailed in headlines then as a potential "game-changer," the drug was later found to increase risks of pain crises and fatal events.

Sickle cell specialists like Julie Kanter of the University of Alabama at Birmingham also criticized Oxbryta's rollout, which she said prioritized profits over patient safety and broadly marketed the drug to oncologists instead of sickle cell specialists. The handling of the withdrawal risks further eroding trust in a health care system that often overlooks the needs of the Black community.

"Money needs to be made … but if you enter the sickle cell space, you need to be humble," said Titilope Fasipe, co-director of the Sickle Cell & Thalassemia program at Texas Children's Hospital. "You need to understand what this community has been through, and do not take this community for granted."