| | | |  | Greetings! This is Adam Feuerstein, Angus Chen, and Damian Garde, and we are incredibly excited to be in New Orleans for the annual meeting of the American Society of Hematology. It’s humid down here! Hang with us for the next four days, as we bring you all the news, analysis, and quirky color from this year’s conference. And don't forget to sign up for our live ASH recap, scheduled for Wednesday. | | |  The view from Jackson Square. (Adam Feuerstein/STAT) Durability weighs on gamma-delta T cell therapy Off-the-shelf cell therapies of all types do a pretty good job of inducing remissions in patients with blood cancers. Maintaining those remissions for a long period of time, however, has been a challenge. Adicet Bio, developer of a unique therapy derived from so-called gamma-delta T cells, came into this year’s meeting with some high expectations. Data presented last June showed an impressive 80% of patients with B-cell lymphoma achieved complete remissions following a single infusion of its therapy, called ADI-001. With longer follow-up, however, patients are relapsing. In a study update, Adicet said 33% of patients have maintained complete remissions for at least six months — a closely watched marker of durability. Based on another analysis, the durable remission rate fell to 20%. The company and its study investigator called the updated results encouraging and sufficient to justify moving ADI-001 into a pivotal clinical trial. But the data are also likely to fuel more concerns that off-the-shelf cell therapies like ADI-001 are still struggling to match the long-term efficacy of currently approved, CAR-T treatments that are custom-made for each patient. | New drugs, new genetic targets for adult leukemia An emerging class of genetically targeted drugs is inducing remissions in about one-third of patients with advanced leukemia, according to updates today from separate clinical trials. Two biotechs — Syndax Pharmaceuticals and Kura Oncology — are developing their own versions of so-called menin inhibitors that have the potential to become effective treatments for certain types of genetically defined acute myeloid leukemia, a rapidly progressing bone marrow cancer that affects adults. Read more. | In pursuit of cure: A 5-step formula for multiple myeloma Today, though people with multiple myeloma are living longer than ever before, they live with the reality that more treatment lies ahead. As a pioneer in multiple myeloma research, Janssen Oncology is advancing innovative thinking and a scientific strategy focused on delivering potentially curative therapies to patients one day. Learn more about this unique approach. | Treating cancer with less pain and no ‘gruel’ While most conversations at ASH focus on the shiny new tools that might change the standard of care, a few researchers came to the conference with data challenging why that was the standard in the first place. Take, for instance, acute myeloid leukemia. For patients with relapsed or refractory disease, it’s common practice to hold off on a stem cell transplant until their cancer is in complete remission, which means administering intensive rounds of chemotherapy. But that appears to be an unnecessary step. In the first randomized trial of its kind, researchers in Germany enrolled nearly 300 patients with advanced AML. Half went through the standard of care, and half skipped the intensive chemo and went straight to transplant. In the end, both groups had similar rates of remission and survival, but the ones who didn’t go through heavy chemotherapy spent significantly less time in the hospital and had far fewer severe side effects. Another study, focused on mantle cell lymphoma, found that Imbruvica, a targeted cancer therapy from Johnson & Johnson, was non-inferior to stem-cell transplant for patients with advanced disease, suggesting many could avoid the costly, unpleasant process that is now the standard of care. A third concluded that using a high dose of chemotherapy might make post-treatment steroids unnecessary for certain children with leukemia. Even hospital food came under scrutiny. Patients undergoing stem-cell transplant are severely immunocompromised, and, in the name of avoiding infections, roughly 90% of clinics put them on what’s called a neutropenic diet, forbidding raw produce, cold cuts, yogurt, and honey, and demanding that all foods be cooked to nearly 200 degrees Fahrenheit. But it turns out those restrictions might be wholly unnecessary. In a study conducted, perhaps unsurprisingly, in Italy, researchers recruited about 250 transplant patients and randomized them either to a neutropenic diet or one in which they could eat pretty much anything other than uncooked meat. They found no difference in rates of infection, mortality, nausea, hospitalization, or graft-versus-host disease. Also unsurprisingly, the patients with an unrestricted diet reported significantly higher quality of life. “I love this because it really upends the dogma,” said Mikkael Sekeres, a hematologist at the University of Miami who was not involved in the study. “For decades we have been essentially feeding patients gruel in the hospital. We should eliminate these silly neutropenic diets, let patients eat what they want, and give them a better quality of life.” | Accounting for equity in paying for gene therapy The debate over whether one-time gene therapies, priced in the millions of dollars, are cost-effective usually focuses on durability, standards of care, and something called quality-adjusted life years. Researchers from Harvard and Yale suggest adding in a new vector: equity.
Looking at gene therapies for sickle cell disease, which primarily affects Black people, a group led by Yale hematologist George Goshua crafted a complicated formula, whose mathematical details we will spare you, to account for the fact that such treatments would benefit a historically marginalized population. The basic idea is to account for health inequities in sickle cell disease by tinkering with the cost-effectiveness math such that society is willing to spend more money to benefit a population that has received less care.
Assuming a one-time gene therapy for sickle cell disease costs about $2.1 million, that would land outside the cost-effective parameters of a traditional analysis, Goshua said. But if you include an equity weight, such a treatment is cost-effective at between $1.4 million and $3 million. And “if we actually care about equity, we can see that this could be a truly equitable treatment for our patients,” he said. | Olé, olé, olé The ASH meeting attracts an international crowd, so when it happens to fall during the World Cup, attention spans tend to wander … toward soccer.
Yesterday, crowds of ASH attendees gathered at “World Cup viewing areas” equipped with jumbo TVs and audio systems to watch the dramatic quarter-final clashes between Brazil and Croatia and Argentina and the Netherlands. After Croatia’s dramatic win in penalty kicks, a group of supporters decked out in the team’s red-and-white jerseys belted into song.
Overheard on a convention-center escalator this morning: “I canceled all my afternoon meetings. There’s no way I’m missing the England-France match.”
Same. | | | |
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