新疗法对于部分没有治疗选择的血癌患者有效 Unprecedented therapy found effective for blood cancer patients with no treatment options

(纽约州纽约市 – 2019年8月21日) – 根据临床试验结果,西奈山的研究人员发现了一种新型治疗方法,可以对特定类型的骨髓癌患者有一定的疗效,这种参加试验的患者已经对几种标准疗法有抗药性。发表于8月份的“新英格兰医学杂志”。

该试验用地塞米松测试了selinexor,这种组合在超过四分之一的患者中显着降低了癌症,其中两名患者得到完全缓解。

蛋白质和信使RNA是癌细胞生长的重要组成部分,selinexor具有前所未有的机制,阻止蛋白质和信使RNA从癌细胞核向细胞质的输出,导致癌细胞死亡。对于患有多发性骨髓瘤(一种称为浆细胞的白细胞癌)的近40%的患者,该疗法多多少少引起一些反应。

“这项研究证明,一种具有新作用机制的新型一流药物可以杀死患者的癌细胞,”该研究的资深作者Sundar Jagannath说,MBBS,多发性骨髓瘤计划主任和医学教授(西奈山的Tisch癌症研究所的血液学和肿瘤内科。 “这证明了这种药物可以用于那些已经用尽所有其他治疗费用的患者,否则他们就会接受临终关怀治疗。”

这项名为STORM Part 2研究的临床试验研究了122名患者在美国和欧洲的试验中对口服药物selinexor和地塞米松的反应。西奈山在国际研究中招募了四分之一的患者。

患者通常在一两个月内看到对药物的反应。虽然没有器官毒性,但副作用包括血液计数低而没有出血,恶心,呕吐,食欲不振或疲劳。

“这项研究对多发性骨髓瘤患者在多种其他疗法上没有取得成功是有意义的,”该研究的第一作者Ajai Chari说,他是西奈山Tisch癌症研究所多发性骨髓瘤计划临床研究主任。 “越来越多的患者对治疗多发性骨髓瘤的标准药物有抵抗力,这些患者的总体生存期很短,有时不到三个月。”

Selinexor于7月初被FDA批准用于对多种疗法有抗药性的患者。该研究由selinexor制造商Karyopharm资助。

Selinexor也正在研究多发性骨髓瘤与其他批准的多发性骨髓瘤药物以及其他恶性肿瘤如淋巴瘤和卵巢癌。

这项研究是西奈山Tisch癌症研究所团队骨髓瘤研究的一个重要里程碑,该研究所最近推出了一个新的多发性骨髓瘤卓越中心。新中心汇集了国际公认的医生,为患有多发性骨髓瘤和相关疾病的患者提供全面,富有同情心的护理,并推动创新研究和个性化治疗,从而治愈。 Jagannath博士将担任该中心的主任,该中心负责治疗该国最多的骨髓瘤患者。

“多发性骨髓瘤的卓越中心是Tisch癌症研究所的一部分,在国家癌症研究所指定的癌症中心最先进的设施中使用最先进的诊断和治疗方法,”医学博士Ramon Parsons说。博士,Tisch癌症研究所所长,肿瘤科学主席,以及西奈山伊坎医学院癌症研究的Ward-Coleman教授。 “协调护理团队包括来自骨髓移植计划,病理学,放射学,免疫学,基因组学,传染病,整形外科,心脏病学和肾脏病学的专家,患者将获得广泛的全面支持服务,包括社会工作者,财务顾问和神职人员。“

NEWS RELEASE 

Unprecedented therapy found effective for blood cancer patients with no treatment options

THE MOUNT SINAI HOSPITAL / MOUNT SINAI SCHOOL OF MEDICINE

(New York, NY – August 21, 2019) — Mount Sinai researchers have found a new type of therapy to be effective for patients with a particular type of bone marrow cancer that is resistant to several standard therapies, according to results of a clinical trial published in The New England Journal of Medicine in August.

This trial tested selinexor with dexamethasone, a combination that significantly knocked down the cancer in more than a quarter of patients, including two patients who went into complete remission.

Proteins and messenger RNAs play an important part of cancer cell growth, and selinexor has an unprecedented mechanism that blocks the export of protein and messenger RNAs from the nucleus of the cancer cell to the cytoplasm, causing the cancer cell to die. This therapy caused at least a minimal response in almost 40 percent of patients who had multiple myeloma, a cancer of a type of white blood cell called a plasma cell.

“This study proved that a novel, first-in-class drug with a new mechanism of action can kill a patient’s cancer cells,” said the study’s senior author, Sundar Jagannath, MBBS, Director of the Multiple Myeloma Program and Professor of Medicine (Hematology and Medical Oncology) at The Tisch Cancer Institute at Mount Sinai. “This proved that the drug worked in patients who had exhausted every other treatment and who would have been placed on hospice care otherwise.”

The clinical trial, called the STORM Part 2 Study, studied the response of 122 patients taking selinexor and dexamethasone, both oral drugs, in trials across the United States and Europe. Mount Sinai enrolled a quarter of the patients in the international study.

Patients generally saw a response to the drugs within one or two months. While there was no organ toxicity, side effects included low blood count without bleeding, nausea, vomiting, lack of appetite or fatigue.

“This study is meaningful for patients with multiple myeloma who haven’t had success on multiple other therapies,” said the study’s first author Ajai Chari, MD, Director of Clinical Research in the Multiple Myeloma Program at The Tisch Cancer Institute at Mount Sinai. “An increasing number of patients have resistance to the standard drugs used in the treatment of multiple myeloma, and the overall survival in these patients is short, sometimes less than three months.”

Selinexor was approved by the FDA for patients resistant to multiple therapies in early July. This study was funded by Karyopharm, the manufacturer of selinexor.

Selinexor is also being investigated in multiple myeloma in combination with other approved multiple myeloma drugs as well as in other malignancies such lymphoma and ovarian cancer.

This study is a major milestone in myeloma research for the team at The Tisch Cancer Institute at Mount Sinai, which recently launched a new Center of Excellence for Multiple Myeloma. The new Center brings together internationally recognized physicians to provide comprehensive, compassionate care for patients with multiple myeloma and related diseases and to advance innovative research and personalized treatments that lead to cures. Dr. Jagannath will serve as Director of the Center which treats the largest number of myeloma patients in the country.

“The Center of Excellence for Multiple Myeloma is part of The Tisch Cancer Institute and uses the most advanced diagnostic and treatment approaches within state-of-the-art facilities of a National Cancer Institute-designated cancer center,” said Ramon Parsons, MD, PhD, Director of The Tisch Cancer Institute, Chair of Oncological Sciences, and Ward-Coleman Professor in Cancer Research of the Icahn School of Medicine at Mount Sinai. “Coordinated care teams include experts from the Bone Marrow Transplant Program, pathology, radiology, immunology, genomics, infectious diseases, orthopedic surgery, cardiology, and nephrology, and patients will have broad access to comprehensive supportive services, including from social workers, financial counselors, and clergy.”

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About Mount Sinai Health System

The Mount Sinai Health System is New York City’s largest integrated delivery system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai’s vision is to produce the safest care, the highest quality, the highest satisfaction, the best access and the best value of any health system in the nation. The Health System includes approximately 7,480 primary and specialty care physicians; 11 joint-venture ambulatory surgery centers; more than 410 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report’s “Best Medical Schools”, aligned with a U.S. News & World Report’s “Honor Roll” Hospital, No. 12 in the nation for National Institutes of Health funding, and among the top 10 most innovative research institutions as ranked by the journal Nature in its Nature Innovation Index. This reflects a special level of excellence in education, clinical practice, and research. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report’s “Honor Roll” of top U.S. hospitals; it is one of the nation’s top 20 hospitals in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Geriatrics, Gynecology, Nephrology, Neurology/Neurosurgery, and Orthopedics in the 2019-2020 “Best Hospitals” issue. Mount Sinai’s Kravis Children’s Hospital also is ranked nationally in five out of ten pediatric specialties by U.S. News & World Report. The New York Eye and Ear Infirmary of Mount Sinai is ranked 12th nationally for Ophthalmology and the South Nassau Communities Hospital is ranked 35th nationally for Urology. Mount Sinai Beth Israel, Mount Sinai St. Luke’s, Mount Sinai West, and South Nassau Communities Hospital are ranked regionally.

For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

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