What is Protein Degradation? 

Protein degradation is a natural process by which cells break down proteins that are damaged or no longer needed so they can be recycled or disposed of.  Scientists at Dana-Farber and elsewhere are harnessing this process to destroy specific proteins involved in cancer, an approach known as targeted protein degradation. In contrast to traditional targeted … Read more

双特异性抗体治疗多发性骨髓瘤:6个必知事项

发布日期:2023年6月12日  作者:Rob Levy  医学审校:Shonali Midha博士(Shonali Midha, MD)  2022年,特立妥单抗(teclistamb)成为美国食品药品监督管理局(FDA)获批的首个治疗多发性骨髓瘤(multiple myeloma)的双特异性抗体药物(bispecific antibody)。专家表示,特立妥单抗是近年来治疗骨髓瘤最重要的进展之一,每年,美国有超过3.5万人确诊这种血癌。  什么是双特异性抗体?它作用的机制是什么?  抗体是呈Y型的蛋白质,在免疫系统中至关重要。数百万的抗体能锁定引发特定疾病的微生物、受感染的细胞或癌细胞。抗体与名为抗原的表面分子结合,抗体或摧毁这些有害微生物、细胞,或诱发免疫系统对其发起攻击。  标准的抗体仅与一个抗原连接,而双特异抗体能够同时与两个抗体连接。例如,特立妥单抗不仅与骨髓瘤细胞表面的BCMA抗原连接,还与免疫T细胞表面的CD3标记物结合。因此,这种连接让骨髓瘤和T细胞更为紧密,从而激活T细胞,促使它们消灭骨髓瘤细胞。  特立妥单抗适用于哪些患者?  至2023年3月,特立妥单抗获批,用于治疗复发性或难治性(无应答)骨髓瘤的成年患者,这些患者需要满足以下条件:此前以接受过至少4次治疗,包括:蛋白酶体抑制剂(防止癌细胞分解不必要的蛋白质)、免疫调节制剂(刺激免疫系统)、在骨髓瘤细胞中与CD38蛋白质连接的抗体药物。您可以和丹娜—法伯医护团队咨询参与相关临床试验的事宜。  特立妥单抗如何给药?  特立妥单抗由皮下注射,并遵循“递增”的方式给药。在住院期间,患者注射前三针,剂量逐级递增。在每一次注射后,我们都会密切观察患者可能出现的不良反应,如:细胞因子(活素)释放综合征(cytokine release syndrome),这是一种快速形成的炎症反应。如果患者在48小时内没有出现不良反应,可注射下一针。没有出现不良反应的患者需住院一周左右,出院后,患者需在诊所内进行药物治疗。  特立妥单抗的疗效如何?  特立妥单抗经美国食品药品监督管理局批准是在临床试验的第2期,有超过60%的患者对治疗有积极响应。近30%的患者完全响应,这也代表着他们的癌症症状全部消失。  有哪些最常见的副作用?  在名为MajesTEC-1的2期临床试验中,最常见的不良反应有发热、细胞因子释放综合征、低血细胞计数、肌肉骨骼疼痛、疲惫、上呼吸道感染、恶心。您可以和医护团队咨询如何应对或防止常见副作用的方法。  还有其他治疗骨髓瘤的双特异抗体药吗?  目前,学界正在对很多双特异抗体药物展开临床试验,以骨髓瘤细胞中BCMA抗原为靶点的特立妥单抗就是其中之一。其他试验以骨髓瘤细胞中的其他抗原为突破口。以特异性抗体和其他疗法结合的试验也在积极开展中,请拭目以待。  医学审校人简介: Midha博士是丹娜—法伯肿瘤内科学医师,专攻多发性骨髓瘤和浆细胞疾病。她在南佛罗里达大学完成内科学住院医师培训,担任首席住院医师,后在墨菲特癌症中心完成血液学和内科学专项医师培训,担任首席专项医师。2021年,Midha博士加入丹娜—法伯癌症研究所杰罗姆·利伯多发性骨髓瘤中心(Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute)。此外,她还在哈佛医学院担任医学讲师一职。Midha博士的临床和科研方向包括:深入研究高风险疾病、新型细胞疗法和免疫疗法在多发性骨髓瘤的有效应用。  转载须知 阁下若有媒体联络需求,敬请致电617-632-4090,请阁下指明与媒体团队洽谈;或请发邮件至media@dfci.harvard.edu。阁下如欲转载本文,请发邮件至kun_ma@dfci.harvard.edu,未经授权禁止转载,感谢合作。

LGBTQ+ Patient Finds Care and Understanding at Dana-Farber 

As a longtime volunteer for the Red Cross, Jeanne Dodge knows the value of gaining as much information as possible about a situation or injured individual before administering aid. Making assumptions without all the facts can result in more harm than good in an emergency.  Accordingly, when Dodge comes to Dana-Farber for her multiple myeloma … Read more

干细胞移植如何治疗癌症?

发布日期:2023年5月18日 作者:Rob Levy 医学审校:Arnold S. Freedman博士(Arnold S. Freedman, MD) 干细胞移植(stem cell transplants)治疗多种与血液相关的癌症。移植的目的是为了治愈疾病,或极大程度地延长患者生命。 干细胞移植可用于治疗以下疾病: 急性成淋巴细胞白血病(Acute lymphoblastic leukemia (ALL) ) 急性髓细胞白血病(Acute myeloid leukemia (AML) ) 慢性淋巴细胞白血病(Chronic lymphocytic leukemia (CLL) ) 慢性髓细胞源性白血病(Chronic myelogenous leukemia (CML) ) 霍奇金淋巴瘤(Hodgkin lymphoma) 非霍奇金淋巴瘤(Non-Hodgkin lymphoma) 多发性骨髓瘤(Multiple myeloma) 骨髓增生异常综合征(Myelodysplastic syndromes) 瓦尔登斯特伦巨球蛋白血症(Waldenström’s macroglobulinemia ) 为什么有些患者需要干细胞移植? 造血干细胞移植用来治疗因骨髓或血细胞病变引起的癌症和一些非恶性血液疾病。骨髓(marrow)是骨骼里呈海绵状的物质,是造血的主要组成部分;红细胞(red blood cells)负责为人体全身的细胞输送氧气,并运走二氧化碳;白细胞(white blood cells)对抗疾病;血小板(platelets)负责凝血。 当癌症在骨髓组织中产生时,后果严重:出现抵御疾病的能力减弱、疲惫和虚弱、体重减轻、容易出血或淤青等问题。 干细胞移植可以清除病变的骨髓和血细胞,形成新的骨髓、血细胞、血小板,让患者的供血有了新的开始。 谁符合干细胞移植条件? 患者需要满足一系列条件才能进行干细胞移植,包括:年龄、以往的治疗、整体健康、癌症的病种和阶段、对干细胞移植治疗响应的可能性,还有最重要的一点:合适捐献者的可用性或使用患者自身干细胞的可能性。 干细胞移植都涉及哪些方面? 自体移植(autologous transplants)是指用患者自身的干细胞进行移植。在移植前,患者要接受大剂量的化疗(chemotherapy)和(或)放疗(radiation therapy)杀死全身的细胞,但这两种治疗的副作用就是损坏了骨髓。 异基因移植(allogeneic transplants)是指从一位合适的捐献人处采集干细胞的移植。这类移植也需要大量的化疗和(或)放疗。移植的干细胞生成白细胞,发起抗癌攻击,也叫“移植对抗白血病”效应(graft-versus-leukemia … Read more

Can Stem Cell Transplants Cure Cancer?

Stem cell transplantation refers to transplants of blood-forming stem cells taken from the bone marrow or collected from the bloodstream. Autologous transplants use the patient’s own stem cells, while allogeneic transplants use stem cells provided by a donor. Stem cell transplants offer some patients with blood-related cancers such as leukemia, lymphoma, and multiple myeloma the … Read more

A Family Navigates Challenges of Rare Blood Cancer Called Waldenström’s Macroglobulinemia

Tom Lomaglio, Jr., learned he had a rare blood cancer called Waldenström’s Macroglobulinemia (WM) in 2000. His father, Tom, Sr., had been diagnosed in 2004, and then his sister, Diane, in 2007. They were all referred to Dana-Farber for care. When Lomaglio visited the first time, he met Steven Treon, MD, PhD, director of the … Read more

Autologous or Allogeneic Stem Cell Transplants: Which Will My Doctor Recommend?

Both allogeneic and autologous stem cell transplants involve treating a patient with chemotherapy followed by an infusion of blood-forming stem cells, with a goal of curing the cancer or extending the time before the disease relapses. In adults, transplants are used to treat a range of blood-related cancers, myelodysplastic syndromes, and non-malignant blood disorders. Autologous … Read more

FDA Approves New Treatment Option For Glioma

In an era when targeted therapies are often effective against multiple types of cancer, the U.S. Food and Drug Administration’s accelerated approval of a two-drug therapy for solid tumors carrying a specific mutation in the BRAF gene is a prime example of this trend. For patients with glioma brain cancer that harbors the mutation, the … Read more

Specially Equipped Natural Killer Cells Show Promise Against Form of AML

CAR T cells’ superpower is to identify cancer-related targets on the surface of tumor cells and order an attack on those cells. But they lack anything resembling X-ray vision to detect nefarious protein targets within tumor cells. That shortfall has limited their effectiveness in diseases like acute myelogenous leukemia (AML), in which tumor cells display few surface … Read more

Hemoglobin and Cancer: What’s the Connection?

Hemoglobin is an essential protein in your red blood cells that carries oxygen to your body’s organs and tissues and transports carbon dioxide from your organs and tissues back to your lungs.  Is hemoglobin affected by cancer?  Anemia is essentially a blood condition marked by a low level of hemoglobin and red blood cells, and in some cases can be … Read more

How Does Cancer Start?

Every cancer starts with a mistake deep inside the nucleus of a single cell, where DNA is stored. Abnormalities in DNA can be inherited from one’s parents or can occur because of exposure to harmful substances in the environment such as chemicals in tobacco, ultraviolet radiation from the sun, and perhaps, chemicals in our diet. … Read more

COVID-19 Booster Shots for Most Cancer Patients: What to Know

If you have been vaccinated against COVID-19, the U.S. Centers for Disease Control and Prevention (CDC) recommends different numbers of vaccines depending on if you are immunocompromised. Immunocompromised people can include people who have: As of December 2022, the COVID-19 vaccination recommendation for immunocompromised people, including active cancer patients, includes: A booster dose is another dose given … Read more

What is a Cytokine Storm?

A cytokine storm is a severe immune system reaction to infection, autoimmune condition, or other disease, including some cancers. It occurs when the body produces extremely high levels of certain cytokines, which are proteins that raise or lower immune activity. The deluge of cytokines into the bloodstream can result in severe inflammation across multiple bodily … Read more