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Studies show promising new options to address unmet needs in sickle cell disease and immune thrombocytopenia SAN DIEGO , Dec. 7, 2024 /PRNewswire/ -- Researchers report significant progress toward expanding the therapeutic options available for people with non-cancerous blood conditions in five studies presented during the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The studies offer new hope for addressing unmet needs and improving quality of life among people living with sickle cell disease (SCD) and immune thrombocytopenia (ITP). "It's exciting that we have four terrific studies designed to benefit people with SCD, including treatments that have been available but haven't been used in certain populations, and potential new therapies, as well," said Charles Abrams , MD , Francis C. Wood Professor of Medicine at the University of Pennsylvania School of Medicine in Philadelphia , who moderated the press briefing, Reading Up on the Classics: Treating Not-So-Benign Hematology Conditions . "Additionally, the ITP study shows there's a new option for a group of patients for whom there aren't a lot of great options currently." The first study examines a first-in-class oral medication for the treatment of ITP, an autoimmune disorder that leads to low levels of platelets, fatigue, and a risk of uncontrolled bleeding. The drug brought a durable platelet response in about one-quarter of study participants who had not seen lasting improvements with other available treatments, suggesting it could offer a new therapeutic option for some patients. The other four studies present new hope for individuals with SCD, an inherited disorder that causes red blood cells to become sickle-shaped, which reduces blood flow, leading to decreased oxygen delivery to the tissue. SCD can cause a wide range of symptoms and health impacts, including vaso-occlusive crises (VOCs) – episodes of severe pain caused by oxygen deprivation in tissues – and damage to tissues and organs. There are only a few available treatments for SCD, and many affected individuals lack access to these therapies, cannot tolerate available medications, or do not derive significant benefits from them. There is a tremendous need for new therapeutic options that can improve quality of life and potentially prevent long-term damage. The first SCD study found that pain episodes were reduced by almost half in patients taking the experimental drug etavopivat. The second study reports on tolerability and potential benefits of the SCD drug hydroxyurea in people with hemoglobin SC, a less common variant of the disease; the study represents one of few prospective studies focused on this undertreated patient group. In the third study, a small retrospective analysis, researchers report that fertility preservation procedures are safe for people with SCD under appropriate expert care, underscoring the importance of ensuring access to fertility preservation as more individuals consider therapies that may raise the risk of infertility. The fourth study reports early results from a new, potentially curative gene therapy for SCD, showing both benefit and risk. Rilzabrutinib Found Safe and Effective for Refractory Immune Thrombocytopenia 5 : Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Adults with Previously Treated Immune Thrombocytopenia (ITP): A Phase 3, Placebo-Controlled, Parallel-Group, Multicenter Study (LUNA 3) The experimental drug rilzabrutinib was well tolerated and generated an increase in platelet counts among some adults with immune thrombocytopenia (ITP) who had not experienced lasting improvements with other available ITP treatments, according to the results of a phase III trial. The trial's primary endpoint of durable platelet response was achieved in about one-quarter of patients taking the drug and none of those taking a placebo and continued to improve with longer follow-up. Even among those who did not meet the threshold for the primary endpoint, many patients taking rilzabrutinib experienced secondary benefits including improvements in platelet count, bleeding, and fatigue. "The drug is highly active in a highly refractory group of patients," said the study's lead author, David J. Kuter , MD, DPhil, program director of hematology at Massachusetts General Hospital and professor of medicine at Harvard Medical School in Boston . "In addition to raising platelet counts, bleeding events decreased significantly and quality of life improved. The drug also worked really fast – within 15 days – and those who responded maintained a durable response for a long time." ITP leads to low levels of platelets, a blood cell that is important for clotting. In children, it can resolve on its own, but in adults, it is typically chronic. Several medications are available to help raise platelet counts, but people who cannot tolerate or do not respond to available medications can suffer from uncontrolled bleeding and chronic fatigue. Rilzabrutinib is a first-in-class oral Bruton tyrosine kinase inhibitor designed to boost platelet counts by simultaneously reducing the production of platelet-targeting autoantibodies, reducing the destruction of platelets by macrophages, and inhibiting B cell activation and inflammatory pathways. While other drugs increase the rate of platelet production, the mechanism of action for rilzabrutinib is unique in its multifaceted approach to ITP. For this phase III trial, researchers enrolled 202 adults with persistent/chronic ITP that was not sufficiently managed with standard-of-care therapies. Participants had seen no lasting benefit from a median of four prior therapies before enrolling in the trial. Two-thirds of the participants were randomly assigned to take rilzabrutinib and one-third took a placebo for a 24-week double-blind period. After the first 12 weeks, 64% of patients on rilzabrutinib and 32% on placebo achieved a platelet response (platelet count ≥50×10 9 /L or ≥30–Justice Department Drops Cases Against Trump: A Legal Milestone
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EAST RUTHERFORD, N.J. (AP) — The New York Giants organization got exactly what it deserved in getting blown out by Baker Mayfield and the Tampa Bay Buccaneers. The Giants were embarrassed in Sunday's 30-7 loss , taunted by Mayfield after a touchdown run just before halftime. And then they saw their fans walk out on them again when the Bucs extended their lead to 30-0 and sent New York (2-9) to its sixth straight loss. The losing streak is the longest for the Giants since 2019, when they dropped a franchise-record nine straight games to finish 4-12. That led to the firing of coach Pat Shurmur after two seasons. Third-year coach Brian Daboll is clearly in trouble, with the Giants guaranteed a second straight losing season. They were 6-11 in a 2023 season that featured a lot of injuries. Daboll, who denies he has lost the team, isn't the only one whose job is in jeopardy. General manager Joe Schoen is on the hot seat and so is this entire franchise, which is celebrating its 100th year. It's one thing to lose. It's quite another to give up, and that's what the organization did when it decided to bench Daniel Jones a week ago and then release him on Friday after the 27-year-old asked co-owner John Mara to let him walk away. While he wasn't playing well, Jones was the Giants' best quarterback. He gave them more a of chance to win than either Tommy DeVito or Drew Lock. Removing him from the picture was all but certain to make the Giants worse, even if it was a good business decision. If Jones was hurt and unable the pass his physical before the 2025 season, the team would have been on the hook for a $23 million cap hit. The problem is the players care about now. By getting rid of Jones and elevating DeVito to the starting role, the front office was telling the team it didn't care about winning with seven games left in the season. So the players gave a lackluster effort. Defensive tackle Dexter Lawrence called the team soft. Rookie receiver Malik Nabers said he was sick of losing. Left tackle Jermaine Eluemunor said he saw a lack of effort by some players. What they all were saying was they were angry at being betrayed. Money is never more important than winning, and the Giants made that mistake. At this point in the season? Nothing. The offense once again. The Giants have scored a league-low 163 points, including only 60 in six games at MetLife Stadium, where they are winless this season. They have scored in double figures at home twice. Daboll's team has been held scoreless in the first half in three of 11 games and it has been held without a first-half touchdown seven times. Daboll said he will continue to call the offensive plays. S Tyler Nubin. The rookie has had a team-high 12 tackles in each of the last two games. His 81 tackles for the season are just two behind team leader Bobby Okereke. RB Tyrone Tracy. The rookie leads Giants running backs with 587 yards on 116 carries — a 5.1-yard average for the fifth-round pick. But holding onto the ball has been a big issue. Tracy's fumble in overtime cost New York a chance to win in Germany against Carolina. He also lost the ball in the third quarter at the Bucs 5-yard line with New York down 23-0. It earned him a seat on the bench. LT Jermaine Eluemunor (quad) and OLB Azeez Ojulari (toe) left Sunday's game in the first quarter. Chris Hubbard filled in at tackle and the Giants luckily got back DL Kayvon Thibodeaux this past week after he missed five games with a broken wrist. DeVito was banged up but Daboll expects him to start against the Cowboys. 10 — The Giants have gone 10 consecutive games without an interception, tying the NFL record held by the 1976-77 San Francisco 49ers and the 2017 Oakland — now Las Vegas — Raiders. The Giants and Raiders now share the single-season mark. A national showcase on Thanksgiving Day for the NFC-worst Giants at Dallas. AP NFL: https://apnews.com/hub/NFL