2026年9月21日 / 美国东部时间凌晨5:00 / KFF健康新闻
确诊18个月后，化疗未能减缓21岁梅森·亨德森体内罕见脑瘤的扩散，癌细胞已经蔓延至他的脑脊液。于是他离开得克萨斯州东南部的家，前往纽约市参加一项为期三周的临床试验。
但这场试验同样宣告失败，亨德森的治疗前路变得一片迷茫。他所患的癌症极为罕见，世界卫生组织直到2021年才为其命名。因此今年年初，评估了肿瘤深层基因特征的亨德森的医生们，选择了一款由默克和阿斯利康联合研发的药物，商品名为林帕扎（Lynparza）。
这种药物并非亨德森病情的标准治疗方案——实际上根本不存在标准疗法，这在罕见癌症患者中并不罕见。尽管为他治疗的两名专科医生已仔细提交了用药依据，但亨德森的保险公司拒绝承担这笔费用。
“他们没有针对他这种癌症的治疗指南，”亨德森的母亲塔比瑟·洛在今年3月接受KFF健康新闻采访时说道，“他们就是在歧视他，只因为他的癌症太罕见。”
每年约有数万人被诊断出患有与常见肿瘤存在显著差异的罕见癌症，这一群体约占美国所有癌症患者的四分之一。保险公司在决定是否报销此类疾病的治疗费用时，通常会参考美国食品药品监督管理局（FDA）的药品标签和专家指南。
但这类罕见疾病往往缺乏针对性的FDA批准治疗方案，尽管在许多情况下，诊断公司和大学实验室提供的分子检测能够强烈提示哪些药物可能有效。
“保险覆盖范围往往落后于基因检测对患者癌症的揭示结果，也落后于科学研究支持的治疗方案，”威尔康奈尔医学院英格兰德精准医学研究所主任奥利维尔·埃莱门托说道。
亨德森的神经肿瘤学家——贝勒医学院的雅各布·曼德尔和纽约大学朗格尼健康中心的杰西卡·舒尔特——决定尝试将林帕扎（通用名奥拉帕利）与化疗联合使用。舒尔特表示，尽管支持该药物的临床证据并不充分，但存在“生物学上合理的假设”认为它会有效，因为类似亨德森体内的肿瘤细胞存在缺陷，而林帕扎这类药物可以针对该缺陷发挥作用。此前已有多名患者使用该药物后，类似亨德森的脑瘤取得了良好的治疗反应。
“通常情况下，我们会基于涉及数百名患者的大型研究来制定治疗决策，”舒尔特说道。但针对亨德森这种罕见癌症，可能永远不会开展大型临床试验。
舒尔特专门研究青年人群的脑癌，她表示每年只会遇到少数几例亨德森这种类型的病例。
曼德尔于1月16日开具了该药物处方。亨德森的药品福利管理机构Liviniti在1月30日迅速拒绝了报销申请。两周后，该公司发来解释：“林帕扎未被批准用于您提供的诊断病症。”洛表示，自费购买该药物每月约需8700美元。记者多次致电Liviniti寻求置评，但未得到回复。
洛介绍，确诊前，亨德森是一名健康、热爱运动的年轻人，心地善良，信仰耶稣，拥有一群亲密的好友。在得克萨斯州博蒙特以北的伊瓦代尔高中，亨德森曾参加棒球和橄榄球队，并在2022年当选返校节国王。毕业后，他在当地一家造纸厂工作，业余时间喜欢打猎、钓鱼，驾驶全地形车探索丛林。他的梦想是成为一名警察，洛说道。
2024年3月15日，亨德森刚满20岁，他的哥哥冈纳在家中楼梯顶端发现了他，当时他双手抱头。“他正处于癫痫发作后的状态，”洛说道，“他说不出话，一直在哭，想拥抱我，却无法交流。”
在博蒙特的急诊室，核磁共振成像显示他脑部有巨大肿瘤。随后他被转至休斯顿的贝勒圣卢克医疗中心，被诊断为弥漫性半球胶质瘤（H3 G34突变型）。
几天后的手术切除了90%的肿瘤，但舒尔特表示，由于脑瘤嵌入的脑组织极为脆弱，几乎不可能完全切除。
在接受了16个月的放疗和化疗后，2025年9月的扫描显示癌症已扩散至脊髓，即软脑膜癌病，这种病症通常会在数月内导致患者死亡。曼德尔联系了舒尔特，她当时正在主持一项临床试验。该试验包括11天高强度的全脑全脊髓放疗，这让亨德森疲惫不堪。治疗结束时，癌细胞仍未得到控制。
“这家人非常棒，”舒尔特回忆道，“他们信任我们的团队，但也会提出恰当的问题，确保我们真的将梅森当作一个活生生的人来考虑。”
报销遭拒
林帕扎于2014年获FDA批准用于卵巢癌治疗，其作用机制是干扰肿瘤细胞的增殖能力。在药品福利管理机构Liviniti拒绝为亨德森报销后，他的家人转向杰斐逊县寻求帮助。亨德森的继父杰里·洛在该县警长办公室担任直升机飞行员。
该县直接为其雇员的家庭健康保险支付理赔款，拥有最终报销决定权，但同样拒绝了申请。亨德森的家人提出上诉后，县审查委员会授权一名独立医疗审查员审核此案。该非专科医生支持了委员会的决定，并推荐了另一种药物，但亨德森的医生对此表示反对。记者致电委员会寻求置评，未得到回复。
阿斯利康也拒绝了这家人免费获取药物的请求。此时已是3月，距离开具林帕扎处方已过去六周。
脑部原发肿瘤较为罕见——美国每年约有2.5万例新诊断病例，而乳腺癌和肺癌的年新发病例分别为32万和22.9万。舒尔特表示，每年仅有数百人被诊断出亨德森这种类型的脑瘤，且患者多为年轻人。
弥漫性半球胶质瘤的治疗选择极少；脑癌通常也被排除在临床试验之外。对于制药公司而言，脑癌市场规模相对较小。由于大脑对药物的敏感性极高，且药物必须穿过紧密排列的血管壁细胞层（即血脑屏障），针对脑癌的药物测试既存在风险，又难度极大。
不过，随着科学对癌症显著的分子多样性揭示得越来越深入，制药公司正越来越多地瞄准更细分、潜在更精准的药物靶点。
根据2022年发布的指南，FDA已批准九款药物可用于携带特定基因突变的患者，无论癌症最初起源于哪个器官。这些“组织agnostic（组织无关型）”药物仍占少数，但随着基因组测序愈发普及——越来越多的肿瘤科医生会为患者开具该检测——保险公司必须跟上步伐，威尔康奈尔医学院的埃莱门托说道。
美国多个研究团队正在开展名为“篮子试验”的临床实验，根据肿瘤的遗传学特征而非癌症起源器官，为大多处于晚期的癌症患者制定联合用药方案。
美国临床肿瘤学会发起了规模最大的项目之一——靶向药物与检测应用登记库（TAPUR），该项目于2016年启动，目前已招募了3000多名患者。它在美国270多家肿瘤科诊所为晚期癌症患者免费提供超适应症治疗。
该项目创始人、曾任首席研究员的理查德·希尔施基表示，约有一半的参与者从中获益，少数病例中，治疗使患者存活了一年以上，甚至看似治愈。他说，这些研究结果已推动多项治疗指南的更新，而“指南更新通常足以打通保险报销的通道”。
希尔施基表示，研究已发现“相当多”的病例表明林帕扎对多种肿瘤类型有效。但与许多临床试验一样，TAPUR将原发性脑肿瘤患者——比如亨德森——排除在外。
肿瘤科医生对基因发现将在多大程度上改变癌症诊断方式存在分歧。威斯康星医学院癌症中心临床研究副主任拉泽尔·库尔兹罗克表示，目前癌症仍按乳腺、结肠、肺等部位分类，因为病理学家在诊断肿瘤时正是根据细胞来源进行判断的。
但这是“历史遗留的错误”，她说道。“你是基于病理学家对细胞表面的观察做出诊断，而非真正驱动癌症的因素。”
1590年左右，荷兰的一对父子发明了首台用于观察细胞的光学显微镜。人类基因组计划于2003年完成。库尔兹罗克表示，如果如今用于肿瘤分子扫描的基因组新一代测序技术早于光学显微镜出现，“没人会再根据癌症起源器官进行诊断”。
库尔兹罗克主持着I-PREDICT临床试验，每位患者都会根据肿瘤的DNA、RNA和蛋白质模式接受个体化癌症治疗。“在我们的试验中，每位患者得到的都是不同的药物组合，而非固定的A或B方案，”她说道。医生也可以选择使用标准疗法，她补充道，这些患者将作为研究对照组。
其他肿瘤科医生则认为纯基因诊断存在局限性。印第安纳大学肿瘤学教授凯西·米勒表示，某些癌症中心会打着“我们比任何人都更擅长对您的肿瘤进行测序，因此来我院就诊能让您活得更久、恢复更好”的口号做广告。“但目前尚无证据支持这些说法。”
“我不会放弃”
在亨德森的案例中，问题从来都不在于诊断——贝勒医学院的临床医生很快就确定了他的癌症类型。但这种癌症的罕见性和发病部位让治疗变得困难，而经济援助的缺失更是难上加难。
3月8日，塔比瑟·洛在脸书、领英和Instagram上发布了儿子的照片和他的困境描述，并@了阿斯利康、Liviniti以及拒绝报销的县委员会。“罕见癌症患者仅因癌症罕见就被拒绝治疗，”她在其中一篇被转发数百次的帖子中写道。
“我不愿采取这种方式，但为了我的孩子，我无所不能，”她告诉KFF健康新闻，“我哭过，焦虑过，但我不会放弃。”
次日，曾在两周前拒绝她免费用药请求的阿斯利康患者援助项目通过邮件告知她好消息：一瓶60粒装的林帕扎已送往她的药房。公司发言人塔拉·帕塞尔表示，由于患者保密原则，她无法置评具体操作。
洛六周的抗争终于有了结果。如今，“一切都交给上帝了”，她在4月的采访中说道。但到4月中旬，亨德森已经无法行走，随后又出现了语言障碍。“一切都发生得太快了。”
5月4日，在家人搬到客厅的床上，亨德森在服用该药近两个月后去世。数百人参加了他的追悼会，送葬车队绵延了七分钟。
家人以亨德森的名义设立了大学奖学金，颁发给当地高中的毕业生。截至9月，线上筹款活动和鲈鱼钓鱼锦标赛已筹集了近2.4万美元。《鸭子王朝》的威利·罗伯逊、职业匹克球运动员泰森·麦古芬和职业垂钓者汉克·帕克为抽奖活动捐赠了奖品。洛表示，乡村歌手马克·切斯特尼特寄来了两把签名吉他。
“如果能更快用上药会更好，”纽约大学的舒尔特说道，尽管目前很难确定这是否能延长亨德森的生命。
“我会一直心存疑问，”洛在今年夏天的一次电话采访中说道，“在处理文书工作的过程中，癌症不会停下脚步。”
“一想到我本可以花更多时间陪伴梅森，却不得不为医疗保障问题抗争，就格外令人心痛，”她补充道，“我被迫成为了药品福利管理机构、保险公司和研究专家，同时还要尽力做一个母亲。”
KFF健康新闻是一家专注卫生领域深度报道的全国性新闻编辑部，也是独立卫生政策研究、民意调查和新闻机构KFF的核心运营项目之一。
Her son was dying, but his rare cancer made it difficult to get the right drug
September 21, 2026 / 5:00 AM EDT / KFF Health News
Eighteen months after his initial diagnosis, chemotherapy hadn’t slowed 21-year-old Mason Henderson’s rare brain tumor, which had spread to his spinal fluid. So he left his home in southeastern Texas to spend three weeks in a clinical trial in New York City.
But that failed, too, leaving a murky path for Henderson, whose cancer was so rare the World Health Organization had only given it a name in 2021. So early this year, Henderson’s doctors, evaluating his tumor’s deep genetic language, turned to a drug made by Merck and AstraZeneca called Lynparza.
It was not the standard of care for Henderson’s condition — there wasn’t really any standard, which is not unusual for rare cancers. And Henderson’s insurance would not pay for it, despite the careful justification given by the two specialists treating him.
“They have no guidelines for his cancer,” Henderson’s mother, Tabitha Lowe, said in a March interview with KFF Health News. “They’re discriminating against him because his cancer is so rare.”
Every year, tens of thousands of people — representing about a quarter of all U.S. cancers — are diagnosed with tumors that differ enough from frequently identified ones to be called rare. In determining whether to reimburse treatment for such ailments, insurers turn to Food and Drug Administration labels and expert guidelines.
But these rare afflictions often lack targeted, FDA-approved treatment options, even though in many cases, molecular tests offered by diagnostic companies and university labs can provide a strong suggestion of what will work.
“Insurance coverage routinely trails behind what genomic testing reveals about a patient’s cancer and what the science supports,” said Olivier Elemento, director of Weill Cornell Medicine’s Englander Institute for Precision Medicine.
Henderson’s neuro-oncologists, Jacob Mandel of the Baylor College of Medicine and Jessica Schulte of NYU Langone Health, decided to try Lynparza, also known by the generic name olaparib, in combination with chemotherapy. There wasn’t a wealth of evidence behind the drug but there was a “biologically reasonable” assumption it would help, Schulte said, because cells in tumors like Henderson’s have a flaw that drugs like Lynparza can target. Providers in several previous cases had seen brain cancers like Henderson’s respond well to the drug.
Tabitha Lowe and her son Mason Henderson, who was diagnosed with a rare brain cancer. Tabitha Lowe
“In general, we try to base our treatment decisions on large patient studies” involving hundreds of patients, Schulte said. But large clinical trials will probably never be conducted for a cancer as rare as Henderson’s.
Schulte, who specializes in brain cancers in young adults, sees only a few of Henderson’s type each year, she said.
Mandel prescribed the drug on Jan. 16. Liviniti, Henderson’s pharmacy benefit manager, responded with a quick refusal on Jan. 30. Two weeks later, the company sent an explanation: “Lynparza is not approved for the diagnosis provided.” Out-of-pocket, the drug would cost about $8,700 per month, Lowe said. Liviniti did not respond to phone calls seeking comment.
Before his diagnosis, Henderson was a healthy, athletic young man with a big heart, faith in Jesus, and a tight group of friends, his mother said. At Evadale High School, north of Beaumont, Texas, Henderson played baseball and football and was homecoming king in 2022. After graduating, he worked at the local paper mill, spending his free time hunting, fishing, and exploring the woods on an all-terrain vehicle. He wanted to be a police officer, Lowe said.
Henderson was 20 on March 15, 2024, when his brother Gunner found him at the top of the stairs in the family home with his head in his hands. “He was in the post-seizure state,” Lowe said. “He couldn’t talk. Was crying. Trying to hug me. Could not communicate.”
At an emergency room in Beaumont, an MRI revealed a large tumor. He was transferred to Baylor St. Luke’s Medical Center in Houston and diagnosed with a form of brain cancer called diffuse hemispheric glioma (H3 G34-mutant).
Surgery a few days later cut out 90% of the tumor, but brain cancers are almost impossible to remove entirely, because of the delicacy of the tissue they’re embedded in, Schulte said.
After 16 months of radiation and chemotherapy, a September 2025 scan showed the cancer had spread to his spinal cord, a condition called leptomeningeal disease that usually proves fatal within a few months. Mandel contacted Schulte about a clinical trial she was leading. It consisted of 11 days of brutal craniospinal irradiation, which left Henderson exhausted. When it was over, the cancer was still there.
“The family was wonderful,” Schulte recalled. “They were trusting in their team, but they asked appropriate questions to make sure that we were thinking about Mason as a person.”
Coverage refused
Lynparza, approved by the FDA in 2014 for ovarian cancer, works by interfering with tumor cells’ ability to multiply. After Liviniti, the pharmacy benefit manager, refused coverage for Henderson, his family turned to Jefferson County. Henderson’s stepfather, Jerry Lowe, flies helicopters for the county sheriff’s office.
The county, which had the final say on reimbursement because it pays claims directly for its employees’ family health coverage, also refused. When Henderson’s family appealed, the county review board authorized an independent medical reviewer to look at the case. The nonspecialist supported the board’s finding and recommended another drug, but Henderson’s doctors disagreed. The board didn’t respond to a request for comment.
AstraZeneca had also turned down the family’s request for a donation of the drug. By then it was March, six weeks after Lynparza was prescribed.
Cancers that start in the brain are unusual — only about 25,000 cases are diagnosed in the U.S. each year, compared with 320,000 breast cancers and 229,000 lung cancers. Only a few hundred people each year, mostly young adults, are diagnosed with Henderson’s type, according to Schulte.
Treatment options for diffuse hemispheric glioma are few; brain cancers in general are often excluded from clinical trials. They represent a relatively small market for a pharmaceutical company. Testing drugs against them is risky, because of the brain’s sensitivity, and difficult because the drug must pass through the tightly packed cell walls lining the blood vessels, known as the blood-brain barrier.
Still, drugmakers are increasingly homing in on narrower and potentially more accurate drug targets as science reveals more of cancer’s remarkable molecular diversity.
Under guidance issued in 2022, the FDA has approved nine drugs to be used for patients whose tumors have specific mutations, regardless of the organ where the cancer first appeared. These “tissue agnostic” drugs are still a tiny minority, but as genome sequencing becomes more common — growing numbers of oncologists order it for patients — insurers will have to keep up, Weill Cornell’s Elemento said.
Several U.S. research groups are hosting clinical experiments known as “basket trials,” in which mostly late-stage cancer patients are put on drug combinations based on tumor genetics, rather than the organ of origin.
The American Society of Clinical Oncology has recruited more than 3,000 patients into one of the biggest efforts, the Targeting Agent and Profiling Utilization Registry, or TAPUR, which began in 2016. It provides off-label treatments at no cost to advanced-staged cancer patients at more than 270 U.S. oncology practices.
About half the participants have benefited, and in rare cases the treatment kept patients alive for a year or more or seemingly cured them, said Richard Schilsky, the program’s founder and its principal investigator until recently. The results have led to changes in several treatment guidelines, he said, and a change in guidelines “usually is sufficient to create a pathway to reimbursement by insurance.”
Research has uncovered “quite a few” cases in which Lynparza was effective against a variety of tumor types, Schilsky said. But like many clinical trials, TAPUR excludes patients with primary brain tumors — like Henderson’s.
Oncologists disagree on how broadly genetics discoveries will transform cancer diagnosis. Cancers are currently identified as breast, colon, lung, etc., because those are the cells that pathologists see when diagnosing a tumor, said Razelle Kurzrock, the associate director of clinical research at the Medical College of Wisconsin Cancer Center.
But that’s a “mistake of history,” she said. “You’re making the diagnosis based on the pathologist’s view of the surface of the cell rather than what’s actually driving the cancer.”
A Dutch father and son invented the first light microscope to peer at cells around 1590. The Human Genome Project finished in 2003. If genome-enabled next-generation sequencing, now used for molecular tumor scans, had come before the light microscope, “no one would look at organ of origin,” she said.
Kurzrock leads I-PREDICT, a clinical trial in which every patient gets individualized cancer therapy based on DNA, RNA, and protein patterns in their tumor. Instead of getting drug combination A or B, “in our trial everyone gets a different set of drugs,” she said. Physicians can instead use standard therapies, she said, and their patients are the study controls.
Other oncologists see limitations to purely genetic diagnosis. Certain cancer centers advertise by saying, “‘We’ll sequence your tumor better than anyone else, and therefore you’ll live longer and do better if you come here,’” said Kathy Miller, a professor of oncology at Indiana University. “But the evidence doesn’t support those claims right now.”
“I wouldn’t give up”
In Henderson’s case, the problem was never diagnosis; Baylor clinicians identified his cancer type quickly. But its rarity and location made the tumor hard to fight, and the lack of financial help made it even harder.
On March 8, Tabitha Lowe went on Facebook, LinkedIn, and Instagram with photos of her son and descriptions of his plight. She tagged AstraZeneca, Liviniti, and the county board that had denied his reimbursement. “Rare cancer patients are denied treatment simply because their cancers are rare,” she wrote in one of the posts, which were shared hundreds of times.
Tabitha Lowe took to Facebook to try to get her son Mason Henderson access to the brain cancer treatment his doctors sought for him. Tabitha Lowe
“I hated to take this route, but when it comes to my kids there’s nothing I won’t do,” she told KFF Health News. “I’ve cried, I’ve stressed out, but I wouldn’t give up.”
The next day, AstraZeneca’s patient assistance program, which had turned down her request for the drug two weeks earlier, emailed her with good news: A bottle of 60 Lynparza pills had been shipped to her pharmacy. Company spokesperson Tara Parsell said patient confidentiality prevented her from commenting on its actions.
Lowe’s six-week battle had paid off. Now, “it’s in God’s hands,” she said in an April interview. By mid-April, however, Henderson could no longer walk. Then came issues with his speech. “It all happened so fast.”
On May 4, in the family’s living room, where his bed had been moved, Henderson died, after taking the drug for nearly two months. Hundreds attended his memorial service; their cars made a procession seven minutes long.
The family has created a college scholarship in Henderson’s name for graduates of the local high school. An online campaign and bass fishing tournament had raised nearly $24,000 by September. Willie Robertson of Duck Dynasty, professional pickleballer Tyson McGuffin, and pro fisherman Hank Parker donated items for a raffle. Country singer Mark Chestnutt sent two signed guitars, Lowe said.
“Faster treatment would have been better,” although it’s hard to know whether it would have extended Henderson’s life, NYU’s Schulte said.
“I will always wonder,” Lowe said in a phone interview this summer. “Cancer don’t pause while the paperwork’s in progress.”
“There’s something especially painful thinking about how much time I spent fighting healthcare instead of being with Mason,” she added. “I was forced to become a PBM, insurer, research expert, all while trying to be his mother.”
KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — the independent source for health policy research, polling, and journalism.