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(This roundup of news from the ASH 2024 conference first appeared in STAT’s “ASH in 30” newsletter. Sign up here to receive future editions.)
If you’re in San Diego, eat tacos. Amazing. Adam, Jonathan, Meghana and Angus are back for your Sunday ASH recap. And thanks to everyone who packed the house last night for our STAT@ASH event.
Market leaders present a vision on the future of blood cancer treatment
Biopharmaceutical leaders at companies developing blood cancer therapies say we shouldn’t be afraid to use the four-letter word: cure.
That word came up 20 times during a panel discussion with representatives from GlaxoSmithKline, AstraZeneca, Regeneron and Johnson & Johnson at STAT@ASH, STAT’s event at the annual meeting of the American Society of Hematology. Speakers pointed to a growing toolbox of strategies to combat blood cancers – including CAR-T therapy, bispecifics, antibody-drug conjugates and other approaches – allowing drug developers to shift their thinking from treating patients until their disease progresses to treating of cancer. healing.
“Can we give better tolerated medications in earlier lines of therapy?” said Andres Sirulnik, leader of Regeneron’s hematology clinical development program. “Can we possibly heal with these new technologies and medicines that we couldn’t even imagine before?”
He added that there are early signs that the answers to these questions could be “yes,” including the first data Regeneron will present showing 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 generated more than $9 billion in sales last year — significantly reduces the risk that people with smoldering myeloma , a condition that can predict multiple myeloma and subsequent development of the cancer.
Susan Galbraith, AstraZeneca’s Executive Vice President of Cancer Research, pointed to the pharmaceutical giant’s progress in developing cancer drug cocktails that can be used for a set period of time, a feature that could appeal to younger patients who are averse to a lifelong drug regimen. treatment. And GSK highlighted the dramatic rise, fall and potential return of Blenrep, an antibody-drug conjugate the company 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 picture of how this therapy is out of reach for many patients from underserved communities, who are also at greater risk of dying without the treatment.
A scientific team led by the Fred Hutchinson Cancer Center tracked data from five medical centers and found that patients from ZIP codes with lower education levels were 33% less likely to receive a bone marrow transplant compared to people from more educated and wealthier communities. But among patients who were were able to undergo a transplant, the survival rate was not significantly associated with socioeconomic status.
“If patients are able to overcome the barriers to transplantation… perhaps the results could be comparable to those of patients from other socio-economic backgrounds,” Natalie Wuliji, lead author of the study and a hematologist-oncologist at Fred, told IPS. Hutch, to 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 accommodation.
AML is the world’s most common reason for a bone marrow transplant, and the research team now plans to investigate better ways to screen which patients face the greatest socio-economic barriers and test strategies to help them receive treatment to get.
Read more.
Study of September 11 first responders shows higher risk of blood cancer – and potential new treatment target
The attack on the World Trade Center exposed hundreds of thousands of people to toxic dust from pulverized construction and aircraft materials, and the World Trade Center Health Program estimates that more than 30,000 people developed cancer as a result. A study of first responders to the attack is helping discover how this toxic exposure leads to more cancers, and may also have identified a potential target for treating a common precursor to many different types of blood cancer: mutations in blood stem cells known as clonal cancers. hematopoiesis mutations.
To conduct this research, scientists at the Albert Einstein College of Medicine sequenced the blood of approximately a thousand firefighters who responded to the attack, as well as the blood of firefighters who did not respond to the attack and non-firefighters. Divij Verma, the lead author and cancer researcher, found that first responders had a roughly threefold 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 blood cancer.
In another experiment, Verma exposed mice to dust collected from September 11 and found that these animals also developed more clonal hematopoiesis than mice that were not exposed. Verma noticed that these mice experienced “tremendous inflammation,” he said. “And the receptor that senses that inflammation was IL1RAP, which was significantly upregulated on mutant cells.”
When Verma repeated the experiment using mice with an IL1RAP deletion, he found that cells with a particular clonal hematopoiesis mutation grew much more slowly. That could mean that IL1RAP could be an important target in tackling clonal hematopoiesis and could potentially prevent or delay the development of certain blood cancers and other diseases.
Experts call Pfizer ‘tone deaf’ for suddenly stopping the sickle cell drug
Pfizer’s abrupt discontinuation of its sickle cell drug Oxbryta was criticized as “tone deaf” by experts at this weekend’s STAT@ASH panel – drawing attention to systemic shortcomings in the treatment of this historically neglected disease. The drug, which was approved in 2019 and hailed in headlines at the time as a potential “game-changer,” was later found to increase the risk of pain crises and fatal events.
Sickle cell specialists such as Julie Kanter of the University of Alabama at Birmingham also criticized the rollout of Oxbryta, which she said prioritized profit over patient safety and marketed the drug broadly to oncologists rather than sickle cell specialists. The handling of the withdrawal threatens to further undermine confidence in a health care system that often overlooks the needs of the Black community.
“There’s money to be made… but when you enter the sickle cell world, you have to be humble,” says Titilope Fasipe, co-director of the Sickle Cell & Thalassemia Program at Texas Children’s Hospital. “You have to understand what this community has been through and not take this community for granted.”
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