Latest news with #hematology
Reuters
18-07-2025
- Business
- Reuters
Bristol Myers' drug fails main goal in cancer-related anemia trial
July 18 (Reuters) - Bristol Myers Squibb (BMY.N), opens new tab said on Friday its blockbuster drug Reblozyl in combination with another therapy failed to meet the main goal of a late-stage trial to treat anemia due to a rare bone marrow cancer. The study did not achieve statistically significant results in helping myelofibrosis patients become free from red blood cell transfusions for any 12-week period during the first 24 weeks of treatment. However, more patients receiving Reblozyl saw a reduction in the number of transfusions needed and an increase in hemoglobin levels, a key measure of anemia, the company said. The side effects seen in the trial were similar to those previously reported for Reblozyl. "It is promising to see that Reblozyl led to clinically relevant improvement of anemia for patients with myelofibrosis," said Anne Kerber, head of hematology, oncology, and cell therapy at Bristol Myers. The company remains confident in the treatment's potential to address unmet need, Kerber added. The late-stage trial in myelofibrosis patients tested Reblozyl with a Janus kinase inhibitor drug, a class of medicines that works by slowing down the immune system. Myelofibrosis is a type of cancer, in which the bone marrow becomes scarred and doesn't produce enough healthy blood cells. Reblozyl is already approved for certain types of anemia related to other blood disorders, including myelodysplastic syndromes and beta-thalassemia. Bristol Myers recorded $1.77 billion in global sales of Reblozyl in 2024. Other approved treatments for myelofibrosis-associated anemia include Incyte's (INCY.O), opens new tab Jakafi and British drugmaker GSK's (GSK.L), opens new tab Ojjaara. Reblozyl's failure in the trial might be positive for Disc Medicine (IRON.O), opens new tab, as investors might look favorably at its hepcidin portfolio, given the strength of data and limited treatment options, BMO Capital Markets analysts said.
Medscape
11-07-2025
- Health
- Medscape
Witness to a Revolution: A Blood Cancer Expert's Long View
Hematologist Robin Foà, MD, has done more than simply watch his specialty transform over the past 50 years. He's helped to lead the revolution in blood cancer care, especially for the treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Foà is now a professor emeritus of hematology at Sapienza University of Rome, Rome, Italy, and he says he's 'officially retired' and should be taking it easy. But he's still working. This year alone, Foà has co-authored at least nine published studies, plus a guide for treating adult Ph+ ALL for the journal Blood , a commentary in the Cell Press journal Med , and a review article for The New England Journal of Medicine about 25 years of progress in treating the disease. In an interview, Foà spoke about his professional journey, the past and future of hematology, and his love of chronicling the world through photography. Can you tell us about your early life and how you came to hematology? It's a complicated family story. I was born in England because my father had to leave Italy in late 1938 or 1939 due to the racial laws because he was Jewish. From Torino, Italy, he went first to Paris, France, then to England. When World War II started in 1940, being Italian, he was considered an enemy and interned. In the Lake District he met my mother, who was a teacher from Wallsend in Northumberland. They married in September 1945, after the war finished. Following the war, my family had a choice between reuniting in New York, where my grandparents and my aunt had spent the years of the war, or returning to Italy. They chose Italy. I was borne in Wallsend and grew up in Torino, where I graduated from medical school and initially worked in the pediatric clinic. How did you end up at Hammersmith Hospital in London? That's a strange twist of fate. When I was a medical student in Torino, the professor of pediatrics organized a meeting and invited someone from London — Professor Gordon Hamilton-Fairley, who became Sir Gordon. I was asked to help with translation at the airport. When I took him back, he said if I ever needed help in the future, to get in touch. Years later, when I wanted to work in London, I had two options. Tragically, Professor Hamilton-Fairley was killed by an IRA bomb in London in 1974 — he was taking his dog for a walk when it touched something under a car. The other contact person in London was Daniel Catovsky, MD, at the MRC Leukemia Unit, Hammersmith Hospital, London. What was Hammersmith like in those days? Hammersmith in the mid-1970s was one of the centers of hematology in the world. I spent 3 years there, and it completely changed my life. I went from pediatrics to working on chronic lymphocytic leukemia [CLL], which doesn't exist in children. It's a disease of the elderly. You've witnessed dramatic changes in leukemia treatment. How do today's outcomes compare to what you saw early in your career? It's been a revolution. Take CLL — the most frequent leukemia in the Western world. In the old days, the only option was chemotherapy. Nowadays, chemotherapy is almost not used anymore, at least in developed countries. We have targeted drugs that provide better survival, better disease control, and fewer long-term side effects. Even more striking is acute promyelocytic leukemia. Through research, we understood that it's a matter of blocked differentiation of leukemic cells. Now, acute promyelocytic leukemia is cured in the large majority of cases without chemotherapy, using all-trans retinoic acid and arsenic trioxide. What has your work with Ph+ ALL produced? This has been one of my major focuses. Based on the results obtained in chronic myeloid leukemia [CML], at the end of the last century we designed the first protocol to treat older patients with Ph+ ALL using only the TKI imatinib plus steroids in induction — and no chemotherapy. It was revolutionary because this was considered the most lethal hematological malignancy. We found that all patients went into remission. Since then, in all national protocols conducted by the Gruppo Italiano Malattie Ematologiche dell'Adulto[Italian Group for Hematological Diseases of Adults], adult patients — with no upper age limit — received a second- or third-generation TKI, dasatinib or ponatinib, plus steroids. More recently, we added immunotherapy with blinatumomab, a bispecific monoclonal antibody in consolidation. We published the results of the dasatinib-blinatumomab trial in The New England Journal of Medicine in October 2020. It showed virtually all patients achieved remission with 88% disease-free survival at a median follow-up of 18 months. The follow-up, published in the Journal of Clinical Oncology in December 2023, showed that you have survival rates between 75% and 80% at four and a half years, the best data ever reported. Half of the patients never received chemotherapy or transplant. What's the significance of avoiding chemotherapy and transplants? I can give you a real example. Many years ago, a young woman from Eastern Europe came to see me. She must have been in her 20s. She was diagnosed with CML and told she needed imatinib for life. When she asked about the cost — possibly over 50 years of treatment — her family said it was literally impossible to afford. The alternative offered was transplant. The pill would obviously be the best option. So I told her to come back as an undocumented immigrant, present to our emergency room, and we would treat her because in Italy, we're obligated to treat all patients regardless of status. She did that. She came, and we could give her the drugs. This shows the tragedy — offering a transplant with high mortality risk instead of a simple oral medication because of cost. You were the president of the European Hematology Association (EHA). What did you focus on during your tenure? I stayed for 6 years in leadership roles — 2 years as president-elect, 2 years as president, and 2 years as past president. In other years, I was the chairman of the Education Committee and the Outreach Unit. It's been a great part for my life, no doubt about it. EHA has grown to become a very important society. I promoted outreach activities to more or less everywhere in the world. Big societies like EHA and American Society of Hematology have a duty to do this. They can help, and have indeed helped, other people. For all the work done, I was awarded the EHA Education and Mentoring Award in 2018 and the EHA José Carreras Award in 2023. What challenges do you see on the horizon for hematology? The main challenge today is accessibility and sustainability. We have precision medicine that changes prognosis, but costs are high. It's unacceptable that only the very wealthy can afford these treatments. We need to make them feasible for as many people as possible. Also, we need continued funding for research. The world is cutting funds everywhere, and all the advances I've discussed stem from research investment. We have the technologies now — single-cell analysis that's potentially phenomenal but extremely expensive. We need funds to utilize them. What advice would you give young hematologists? Hematology is a beautiful discipline that needs to combine clinical work with laboratory research. You need to work in the lab, understand the technology, and appreciate what can be derived from laboratory techniques. I could tell you stories of patients who write to me 20 years later who should have been dead but are still alive. I'm writing up the story of four older patients with Ph+ ALL who lived many years on a TKI alone, even into their 90s! In the old days, they would have been dead in a couple of months. You're also an accomplished photographer with over 10 published books. How did that passion develop? I always loved traveling, and I started taking shots during my trip to Kenya in 1974 with a colleague who was an excellent photographer. I had a tiny Minox camera — the kind spies were using to take photographs of documents. You can imagine that trying to take a picture of an elephant in the savannah with a Minox was pathetic. When I saw my results compared to his, I got my first proper Nikon camera. I love traveling. Nature has always been a key point for me, but also people. I have so far published 12 photography books. What's next for your career? I'm officially retired, and I should relax. I no longer have any clinical or administrative responsibilities, which is a relief. I do continue to coordinate a large research group in Italy that I've had for 14-15 years, and that'll take me until the end of next year. This certainly keeps me very active. Do you have any final thoughts for our readers? We're living in an extraordinary time in hematology. We've moved from the worst outcomes to treating many leukemias without chemotherapy in a matter of 30 years, which isn't long in medicine. Every year we see advancements and improvements. The relationship with patients is completely different now because you're not telling them they're going to die. You're offering hope and often a cure. The key is combining rigorous science with compassion for patients and never lose sight of the global responsibility we have to make these advances available to all patients, regardless of where they live or their economic circumstances.
Yahoo
10-07-2025
- Health
- Yahoo
Diagonal Therapeutics Announces Publication of Data Characterizing the Healthcare System Cost and Impact of Hereditary Hemorrhagic Telangiectasia (HHT) in the American Journal of Hematology
WATERTOWN, Mass., July 10, 2025 (GLOBE NEWSWIRE) -- Diagonal Therapeutics, a biotechnology company focused on correcting dysregulated signaling with clustering antibodies that address the underlying cause of intractable genetic diseases, today announced the publication of study results detailing new health economics and outcomes research (HEOR) that characterizes the significant healthcare utilization and costs associated with hereditary hemorrhagic telangiectasia (HHT) in the American Journal of Hematology. "This study provides the first detailed analysis of the healthcare resource demands and costs associated with HHT, highlighting the substantial economic impact this disease places on patients and the health system," said Hanny Al-Samkari, M.D., the Peggy S. Blitz Endowed Chair in Hematology/Oncology at the Massachusetts General Hospital, Associate Professor of Medicine at Harvard Medical School, and lead author of the study. "The findings underscore the urgent need for treatments that can effectively address the underlying cause of HHT and reduce the complications related to chronic bleeding and anemia, which are major cost drivers requiring hematologic support, hospital admissions, and emergency care." HHT is the second most common inherited bleeding disorder globally, affecting approximately 1 in 4,000 to 5,000 people and an estimated 80,000 in the U.S. The disorder leads to recurrent severe epistaxis (nosebleeds), chronic gastrointestinal bleeding, heavy menstrual bleeding, and arteriovenous malformations (AVMs) in organs such as the lungs, liver, and brain. These AVMs can cause serious or fatal bleeding events and comorbidities, including chronic and recurrent iron deficiency anemia—which occurs in nearly 60% of HHT patients. Chronic anemia is well known to increase risk for hospitalizations, heart failure, and death. Currently, there are no FDA- or EMA-approved therapeutics for HHT worldwide, and treatments are largely limited to off-label use of costly immunomodulatory and antiangiogenic drugs. The study leveraged Komodo Health's Healthcare Map® claims database* to analyze real-world healthcare costs for HHT patients in 2022 and 2023. Key findings from the publication include: Per patient per year (PPPY) costs for people living with HHT are comparable to or surpass those of other rare and resource-intensive diseases. Bleeding and its consequences were identified as the primary drivers of healthcare costs. Mean PPPY costs for all HHT patients were >$19,000 across 2022 and 2023, about 20% higher than those for sickle cell disease. For HHT patients with anemia, the mean PPPY costs were approximately $27,000, comparable to those associated with muscular dystrophy. Patients with HHT receiving hematologic support (iron infusions and/or red blood cell transfusions) had mean PPPY costs of approximately $40,000, comparable to those associated with cystic fibrosis. HHT patients with anemia, while accounting for nearly 60% of the HHT patient population, were responsible for approximately 80% of the direct medical costs. Notably, the prevalence of liver transplantation among HHT patients, arising from complications of liver AVMs, was 40 times greater than in the general U.S. population. 'We are extremely grateful to partner with Dr. Al-Samkari and Cure HHT on this important study, as it puts into stark focus the impact of HHT and its associated complications on individuals and families living with this complex disease,' said Eric Duhaime, Senior Vice President of Corporate Development at Diagonal and co-author of the study. "We are committed to advancing a differentiated treatment that directly addresses the root cause of HHT and are encouraged by the compelling preclinical profile of our first-in-class, bispecific ALK1 agonist antibody. We look forward to advancing development of DIAG723 as a novel, potentially disease-modifying treatment option for people living with HHT." *The Komodo Health's Healthcare Map® claims database includes de-identified data from over 330 million patients across commercial, Medicare, and Medicaid plans in the United States. About Hereditary Hemorrhagic Telangiectasia (HHT) HHT is a rare disease that affects more than 150,000 people in the U.S. and EU, and for which there are currently no approved therapies. In HHT, loss of function point mutations in members of the TGF-ß receptor superfamily complex create abnormal blood vessels that are fragile and susceptible to rupture and bleeding. These bleeding events drastically reduce quality of life, result in emergency visits and hospitalizations, and lead to chronic anemia, necessitating frequent iron infusions or red blood cell transfusions in many patients. Solid organ arteriovenous malformations, if left untreated, are at risk of rupturing, resulting in lung and brain hemorrhage, stroke, heart failure, and death. About Diagonal Therapeutics Diagonal Therapeutics is a biotechnology company advancing novel disease-modifying clustering antibodies that repair dysregulated signaling implicated in a range of illnesses. The Company's DIAGONAL Product Engine combines proprietary computational and experimental techniques to overcome historical challenges associated with antibody drug discovery and efficiently deliver optimized therapeutic assets. Diagonal's pipeline comprises clustering antibodies designed to selectively address the underlying cause of disease across hematology, hepatology, and nephrology, offering the potential to deliver life-changing therapies for patients. DIAG723, Diagonal's first-in-class clustering antibody, recently received orphan drug designation from the U.S. FDA and a positive opinion from the EMA for treating HHT. For more information, please visit Media ContactMarites in to access your portfolio
Zawya
01-07-2025
- Health
- Zawya
Dubai hosts the 6th edition of the Middle East, Africa and Russia Hematology Summit 'MASH'
Saudi Arabia - The Hematology Medical Conference, sponsored by AbbVie, the global research and development company in the field of biopharmaceuticals, kicked off for the sixth consecutive year at the Intercontinental Festival City, Dubai, UAE. This summit represents a major regional collaboration for medical affairs in the field of hematology, with a robust history of collaboration in five previous successful versions. The summit featured a group of elite national and international experts and more than 25 speakers from across the Middle East, Africa, and Russia, as well as international speakers, including a hematologist from Canada and an AI expert from healthcare practitioners from the United States. This year's Hematology Summit, in its sixth version, focused more on clinical experience, devoting a full day of its program to workshops for hematologists and clinical pharmacists to share their experiences in managing challenging cases. Additionally, specialized workshops on AI role in healthcare and precision medicine, particularly in hematological diseases, were led by an international expert in artificial intelligence. Dr. Ayman Al-Hijazi, Consultant Hematologist, King Abdulaziz Medical City, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia, commended the holding of the 6th Hematology Summit and the attendance of that elite group of consultants and hematology experts. The conference showcased the latest diagnostic methods and various advanced treatments in hematology oncology, including AML, CLL, lymphoma, and others. Dr. Ayman also praised the valuable lectures included in the conference, which showcased advancements in the treatment of recurrent or refractory multiple myeloma, as well as revolutionary advances in cancer research and the transformative use of artificial intelligence in diagnosis and personalized treatment strategies. Dr. Abdulwahab Al-Babtain, Consultant Hematologist and Cellular Therapist at King Faisal Specialist Hospital and Research Center in Riyadh, welcomed everyone who participated in the success of this summit. He added that the summit addressed new developments in the treatment of benign and malignant blood diseases and tumors. The conference also featured extensive scientific and medical content, including scientific papers presented and workshops, including workshops on lymphoma and leukemia, management of complications resulting from blood cancers, treatment sequencing for chronic lymphocytic leukemia, and the importance of detecting genetic mutations in acute myeloid leukemia. Dr. Abdulwahab explained that there has been significant development in medications for treating blood diseases, which will significantly contribute to controlling the disease and alleviating patients' suffering. This is based on the latest clinical studies and how to choose the appropriate treatment for each individual case, in accordance with the latest scientific recommendations in this field. Dr. Ashraf Daoud, General Manager of AbbVie Saudi Arabia, emphasized that such scientific summits align with AbbVie's mission and its focus on medical education, training, scientific research, and providing distinguished medical care, in line with the Kingdom's Vision 2030. This was evident in this version of the conference, which showcased the role of AI in accurate disease diagnosis, discussed new treatments for unmet clinical needs, and highlighted the importance of early detection and optimal management of the disease, with the goal of improving patients' lives.
New York Times
28-06-2025
- Health
- New York Times
Maybe It's Not Just Aging. Maybe It's Anemia.
Gary Sergott felt weary all the time. 'I'd get tired, short of breath, a sort of malaise,' he said. He was cold even on warm days and looked pale with dark circles under his eyes. His malady was not mysterious. As a retired nurse-anesthetist, Mr. Sergott knew he had anemia, a deficiency of red blood cells. In his case, it was the consequence of a hereditary condition that caused almost daily nosebleeds and depleted his hemoglobin, the protein in red blood cells that delivers oxygen throughout the body. But in consulting doctors about his fatigue, he found that many didn't know how to help. They advised Mr. Sergott, who lives in Westminster, Md., to take iron tablets, usually the first-line treatment for anemia. But like many older people, he found a daily regimen of four to six tablets hard to tolerate. Some patients taking iron complain of severe constipation or stomach cramps. Mr. Sergott felt 'nauseated all the time.' And iron tablets don't always work. After almost 15 years, he found a solution. Dr. Michael Auerbach, a hematologist and an oncologist who is the co-director of the Center for Cancer and Blood Disorders in Baltimore, suggested that Mr. Sergott receive iron intravenously instead of orally. Now Mr. Sergott, 78, gets an hourlong infusion when his hemoglobin levels and other markers show that he needs one, usually three times a year. 'It's like filling the gas tank,' he said. His symptoms recede, and 'I feel great.' Want all of The Times? Subscribe.
