Game changer ! 幹細胞 對 反覆型多發性硬化症 療效

研究顯示：幹細胞治療多發性硬化症試驗效果好 2018-03-20新華網 一項國際合作研究顯示，幹細胞療法治療一種常見類型的多發性硬化症效果好。按照這種療法，醫生借助化學療法"摧毀"患者"已經出現缺陷"的免疫係統，隨後把從患者血液和骨髓中提取的幹細胞重新注入身體，以"重新啟動"免疫係統。在美國芝加哥、英國設菲爾德、瑞典斯德哥爾摩和巴西聖保羅接受治療的100多名多發性硬化症患者中，52人接受幹細胞療法，50人接受傳統藥物療法。他們的病症都屬于這種病中常見的反復復發緩解類型。一年後，接受幹細胞療法的患者中，1人復發；接受傳統藥物療法的患者中，30人復發。平均隨訪3年獲得的結果顯示，前一組患者中對3人的治療失敗，失敗率6%；後一組中對30人的治療失敗，失敗率60%。多發性硬化症損害大腦和脊髓神經，破壞免疫係統，導致疲勞、肌肉無力、行動困難，可能造成不同程度殘障。英國設菲爾德哈勒姆郡醫院血液和骨髓移植專家約翰·斯諾登告訴英國廣播公司，幹細胞療法對治療多發性硬化症有"轉折"意義。美國西北大學教授理查德·伯特説，神經學學術界一直對這種療法持懷疑態度，但研究數據"令人吃驚"，會改變這種態度。新療法費用大約3萬英鎊（約合26.4萬元人民幣），與一年藥物治療費用相當。醫生提醒，幹細胞療法不適用所有多發性硬化症，治療過程由于涉及化療和在醫院隔離數周，讓病人感受"煎熬"。（歐颯）【新華社微特稿】

Stem Cell Transplant for Multiple Sclerosis May Revolutionize Care for One Million Americans  Kate Sheridan Newsweek20 March 2018  People with multiple sclerosis may be able to "reset" their immune system and potentially reverse their symptoms with an infusion of blood-based stem cells. The finding is based on a randomized clinical trial with 110 patients who'd been diagnosed with relapsing-remitting multiple sclerosis. According to the data, stem cell transplant may be an effective treatment for multiple sclerosis (MS). The study built on work by Dr. Richard Burt, a stem cell specialist at Northwestern University's Feinberg School of Medicine, from 2015, the investigation focused on immune cells as a treatment for the illness. The new, highly anticipated data (which have not yet been published or reviewed by experts in the field) were presented on Sunday at the European Society for Bone and Marrow Transplantation in Portugal. The object of the intervention was to stop "the immune attack on the brain," said Burt, who led the new study, too. Stem cells from a person's own blood were collected 10 days after they started a drug regimen and then reinjected. The study compared the stem cell transplant with the best available drug therapy for each patient, as determined by a neurologist. The design kept the neurologists evaluating the data in the dark about which patients received a transplant or an approved standard drug regimen. The main goal of the trial was to see whether the transplant worsened neurological symptoms. According to the abstract of Burt's presentation, about 60 percent of the patients who received drug therapy experienced a decline as measured by neurologists on standardized scales; by comparison, only six percent of patients who received the transplant progressed similarly. Although further details were not provided in the presentation, Burt said that other measurements also favored the stem cell group. "In every outcome parameter we looked at, transplant was superior," he told Newsweek. (Detailed results haven't been made available yet, he said.) Burt did, however, stop short of calling the new treatment a cure. "In terms of whether we're reversing disability or slowing progression," he said, "I'll have to let you wait for that data." Stem cells found in a person's blood may someday treat a form of multiple sclerosis, if results from a clinical trial announced March 18 can be replicated. Courtesy of the National Institutes of Health John Snowden, the director of blood and bone marrow transplantation at the Royal Hallamshire Hospital in Sheffield, who was also involved in the trial, told the BBC that he was "thrilled with the results," calling them "a game changer for patients with drug resistant and disabling multiple sclerosis." About one million Americans are living with MS today, according to the National MS Society. Patients are typically treated with immunosuppresants like interferon. But these treatments often have harsh side effects, including an increased vulnerability to infections. And the drugs can be expensive; patients diagnosed with MS may pay more than $47,000 per year, according to Burt's 2015 paper. As of 2018, the median cost for a year's supply of MS drugs is $80,000, the society noted. Stem cell treatments can have serious side effects, too. In 2016, Burt received a warning letter from the Food and Drug Administration stating that he had not reported deaths of people who had participated in a clinical trial promptly enough. It's not clear from the FDA warning if the patients had MS; however, the locations outside of the U.S. match those of Burt's collaborators. The violations were deemed to be addressed in late 2016. And the treatment isn't perfect; many people treated with stem cell transplantation in the new study continued to show signs of disability.