EyePoint Pharmaceuticals Announces $16.5 Million Monetization of ILUVIEN® Royalty with SWK Holdings Corporation
By Dr. Matthew Watson
WATERTOWN, Mass., Dec. 18, 2020 (GLOBE NEWSWIRE) -- EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a pharmaceutical company committed to developing and commercializing innovative therapeutics to help improve the lives of patients with serious eye disorders, today announced a royalty monetization agreement with SWK Holdings Corporation (SWK) for royalties payable to EyePoint under its license agreement with Alimera Sciences, Inc. (Alimera) for ILUVIEN®. EyePoint has received a one-time $16.5 million payment from SWK and, in return, SWK is entitled to receive future royalties payable to EyePoint from the Alimera agreement.
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EyePoint Pharmaceuticals Announces $16.5 Million Monetization of ILUVIEN® Royalty with SWK Holdings Corporation
COVID-19 Clinical Programme Update
By Dr. Matthew Watson
To Read More: COVID-19 Clinical Programme UpdateInivata’s Liquid Biopsy Technology Included in Unique £10m Research Collaboration for Early Detection of Lung Cancer
By Dr. Matthew Watson
Inivata’s Liquid Biopsy Technology Included in Unique £10m Research Collaboration for Early Detection of Lung Cancer
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Inivata’s Liquid Biopsy Technology Included in Unique £10m Research Collaboration for Early Detection of Lung Cancer
ProQR Announces Webcast of Presentation at Upcoming HC Wainwright BIOCONNECT Conference
By Dr. Matthew Watson
LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 18, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that Company management will present during a fireside chat at the upcoming HC Wainwright Virtual BIOCONNECT 2021 Conference. A webcast of the presentation will be accessible from the “Investors & Media” section of ProQR’s website (www.proqr.com) under ‘Events’, beginning January 11, 2021.
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ProQR Announces Webcast of Presentation at Upcoming HC Wainwright BIOCONNECT Conference
MacroGenics Enters Research Collaboration with Janssen to Develop Novel DART® Molecule
By Dr. Matthew Watson
ROCKVILLE, MD, Dec. 18, 2020 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, announced today a research collaboration and global license agreement to develop a preclinical bispecific molecule with Janssen Biotech, Inc. The research collaboration will incorporate MacroGenics' proprietary DART® platform to enable simultaneous targeting of two undisclosed targets in a therapeutic area outside oncology.
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MacroGenics Enters Research Collaboration with Janssen to Develop Novel DART® Molecule
Avenue Therapeutics Announces Publication of Tramadol Non-Medical Use in Four European Countries: A Comparative Analysis
By Dr. Matthew Watson
NEW YORK, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (NASDAQ: ATXI) (Avenue), a company focused on the development of intravenous (IV) tramadol for the U.S. market, today announced that a publication titled “Tramadol Non-Medical Use in Four European Countries: A Comparative Analysis” has been published in Drug and Alcohol Dependence, a peer-reviewed international journal devoted to research, reviews, commentaries, and policy analyses in the area of drug, alcohol and tobacco use and dependence. The publication can be accessed here.
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Avenue Therapeutics Announces Publication of Tramadol Non-Medical Use in Four European Countries: A Comparative Analysis
Avenue Therapeutics Announces Publication of Real-World Data on Nonmedical Use of Tramadol in ASI-MV Network
By Dr. Matthew Watson
NEW YORK, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (NASDAQ: ATXI) (Avenue), a company focused on the development of intravenous (IV) tramadol for the U.S. market, today announced that a publication titled “Real-World Data on Nonmedical Use of Tramadol from Patients Evaluated for Substance Abuse Treatment in the NAVIPPRO Addiction Severity Index?Multimedia Version (ASI-MV®) Network” has been published in Drug Safety, a peer-reviewed international journal covering pharmacoepidemiology and pharmacovigilance. The publication can be accessed here.
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Avenue Therapeutics Announces Publication of Real-World Data on Nonmedical Use of Tramadol in ASI-MV Network
Atea Pharmaceuticals Added to Russell 2000 Index
By Dr. Matthew Watson
BOSTON, Dec. 18, 2020 (GLOBE NEWSWIRE) -- Atea Pharmaceuticals, Inc. (Nasdaq: AVIR) (“Atea”), a clinical-stage biopharmaceutical company, today announced that it has been added to the Russell 2000® Index, effective December 21, 2020, as part of the index's quarterly initial public offering (IPO) additions.
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Atea Pharmaceuticals Added to Russell 2000 Index
Report from the Extraordinary General Meeting of Immunicum AB (publ) on December 18, 2020
By Dr. Matthew Watson
Press Release
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Report from the Extraordinary General Meeting of Immunicum AB (publ) on December 18, 2020
Bavarian Nordic Announces Top-line Results from Phase 2 Clinical Trial of BN-Brachyury in Chordoma
By Dr. Matthew Watson
COPENHAGEN, Denmark, December 18, 2020 – Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) today announced top-line results from a Phase 2 clinical study evaluating the targeted immunotherapy candidate, BN-Brachyury in the treatment of advanced chordoma, a rare cancer occurring in the base of the skull and spine. While the study failed to meet its primary endpoint, it provided evidence of clinical activity.
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Bavarian Nordic Announces Top-line Results from Phase 2 Clinical Trial of BN-Brachyury in Chordoma
Cybin Inc. (NEO: CYBN) Looking to Solve Long-Standing Problem with MDD Treatment
By Dr. Matthew Watson
NEW YORK, Dec. 18, 2020 (GLOBE NEWSWIRE) -- NetworkNewsAudio – Cybin Inc. (NEO: CYBN) announces the availability of a broadcast titled, “Hope on the Horizon: Psychedelics Offer Potential for Transforming Mental Health Treatments.”
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Cybin Inc. (NEO: CYBN) Looking to Solve Long-Standing Problem with MDD Treatment
Medexus Enters into Exclusive License to Register and Commercialize Triamcinolone Hexacetonide (TH) in the United States with Ethypharm
By Dr. Matthew Watson
Drug shortage in the United States driving need for Triamcinolone Hexacetonide Drug shortage in the United States driving need for Triamcinolone Hexacetonide
Roche’s faricimab meets primary endpoint and shows strong durability across two global phase III studies for diabetic macular edema, a leading cause…
By Dr. Matthew Watson
Basel, 21 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from two identically designed global phase III studies, YOSEMITE and RHINE, evaluating its investigational bispecific antibody, faricimab, in people living with diabetic macular edema (DME). Both studies met their primary endpoint and showed that faricimab given every eight weeks and at personalised dosing intervals of up to 16 weeks demonstrated non-inferior visual acuity gains compared to aflibercept given every eight weeks. Faricimab was generally well-tolerated, with no new safety signals identified. The studies each have three treatment arms, with participants randomised to receive either faricimab or aflibercept at fixed eight-week intervals, or faricimab at personalised intervals of up to 16 weeks, following a loading phase. In a secondary endpoint, across both studies, more than half of participants in the faricimab personalised dosing arms achieved an extended time between treatments of 16 weeks at year one. This is the first time any investigational medicine has achieved this level of durability in a phase III study of people with DME. Worldwide, an estimated 21 million people are living with DME, a leading cause of vision loss among working-age adults.1 Whilst anti-vascular endothelial growth factor (VEGF) monotherapy injections have significantly reduced vision loss from DME, the treatment burden associated with frequent eye injections and physician visits can lead to under-treatment and, potentially, less than optimal vision outcomes.2,3 It has been almost a decade since a medicine with a new mechanism of action has been approved to treat DME.4 Faricimab is the first investigational bispecific antibody designed for the eye.5 It targets two distinct pathways – via angiopoietin-2 (Ang-2) and VEGF-A – that drive a number of retinal conditions, including DME.6 “These positive results show that faricimab has the potential to offer lasting vision improvements for people with diabetic macular edema, while also reducing the treatment burden associated with frequent eye injections,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We look forward to discussions with global regulatory authorities, with the aim of bringing this potential new treatment option to people with this condition as soon as possible.” In addition to the YOSEMITE and RHINE studies, the phase III Rhone-X study is investigating the long-term safety and tolerability of faricimab for the treatment of DME.7 Faricimab is also being studied in the phase III TENAYA and LUCERNE studies as a potential treatment for neovascular or “wet” age-related macular degeneration (nAMD), an advanced form of AMD, which can cause rapid, severe and irreversible vision loss.8,9,10,11 Detailed results from the YOSEMITE and RHINE studies will be presented in February at Angiogenesis, Exudation, and Degeneration 2021, a medical symposium presented by Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, and submitted for approval for the treatment of DME around the world. About the YOSEMITE and RHINE studies 5,12,13 YOSEMITE (NCT03622580) and RHINE (NCT03622593) are two identical, randomised, multicentre, double-masked, global phase III studies, evaluating the efficacy and safety of faricimab compared to aflibercept in 1,891 people living with diabetic macular edema (940 in YOSEMITE and 951 in RHINE). The studies each have three treatment arms: faricimab 6.0 mg administered at personalised dosing intervals of up to 16 weeks; faricimab 6.0 mg administered at fixed eight-week intervals; aflibercept 2.0 mg administered at fixed eight-week intervals. In all three arms, sham injections were administered at study visits when treatment injections were not scheduled, to maintain the masking of investigators and participants. The primary endpoint of the studies is the average change in best-corrected visual acuity (BCVA) score (the best distance vision a person can achieve – including with correction such as glasses – when reading letters on an eye chart) from baseline at one year. Secondary endpoints include: safety; the percentage of participants in the personalised dosing arm receiving treatment every four, eight, 12 and 16 weeks, at week 52; the percentage of participants achieving a two-step or greater improvement from baseline in diabetic retinopathy severity at week 52; the percentage of participants achieving a gain of at least 15 letters in BCVA from baseline over time; the percentage of participants avoiding a loss of at least 15 letters in BCVA from baseline over time; and change in central subfield thickness from baseline over time. About diabetic macular edema Affecting around 21 million people globally, diabetic macular edema (DME) is a vision-threatening complication of diabetic retinopathy (DR).1 DR occurs when damage to blood vessels and the formation of new blood vessels causes blood and/or fluid to leak into the retina – a part of the eye that sends information to the brain, enabling sight.14 This leads to swelling, as well as blockage of blood supply to some areas of the retina.15 DME occurs when the damaged blood vessels leak into and cause swelling in the macula – the central area of the retina responsible for the sharp vision needed for reading and driving.14,16 The number of people with DME is expected to grow as the prevalence of diabetes increases.17 The condition is associated with blindness when left untreated and decreased quality of life.14,18 There remains a significant unmet need for more effective, longer-lasting therapies for people with DME.3 About faricimab Faricimab is the first investigational bispecific antibody designed for the eye.5 It targets two distinct pathways – via angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A) – that drive a number of retinal conditions.6 Ang-2 and VEGF-A contribute to vision loss by destabilising blood vessels, causing new leaky blood vessels to form and increasing inflammation.3 By independently blocking both pathways, faricimab is designed to stabilise blood vessels, potentially resulting in better vision outcomes, for longer, for people living with retinal conditions.3 About Roche in Ophthalmology Roche is focused on saving people’s eyesight from the leading causes of vision loss through pioneering therapies. Through our innovation in the scientific discovery of new potential drug targets, personalised healthcare, molecular engineering, biomarkers and continuous drug delivery, we strive to design the right therapies for the right patients. We have the broadest retina pipeline in Ophthalmology, covering early and late stage products, which is led by science and informed by insights from people with eye diseases. Our late stage pipeline includes two potential first-of-a-kind treatments, Port Delivery System with ranibizumab (PDS) and faricimab, which are being evaluated in a number of retinal conditions including neovascular age-related macular degeneration, diabetic macular edema and diabetic retinopathy. PDS is a permanent refillable eye implant that continuously delivers a customised formulation of ranibizumab over a period of months, potentially reducing the treatment burden associated with frequent eye injections.19,20 Faricimab is the first investigational bispecific antibody designed for the eye.5 It targets two distinct pathways – via angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A) – that drive a number of retinal conditions, to stabilise blood vessels, potentially resulting in better vision, for longer.3,6 Our early stage pipeline includes gene therapies and treatments for geographic atrophy and other vision-threatening diseases, including rare and inherited conditions. Applying our extensive experience, we have already brought breakthrough ophthalmic treatments to people living with vision loss through Lucentis®?* (ranibizumab injection), the first treatment approved to improve vision in people with certain retinal conditions. About Roche Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI). The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. *Lucentis® (ranibizumab injection) was developed by Genentech, a member of the Roche Group. Genentech retains commercial rights in the United States and Novartis has exclusive commercial rights for the rest of the world. All trademarks used or mentioned in this release are protected by law. References[1] Yau JWY, et al. Global prevalence and major risk factors of diabetic retinopathy. Diabetes Care. 2012;35:556-64.[2] Zhao Y, Singh, RP. The role of anti-vascular endothelial growth factor (anti-VEGF) in the management of proliferative diabetic retinopathy. Drugs in Context. 2018;7:212532.[3] Sahni J, et al. Simultaneous inhibition of angiopoietin-2 and vascular endothelial growth factor-A with faricimab in diabetic macular edema. American Academy of Ophthalmology. 2019;126:1155–70.[4] FDA. Highlights of prescribing information, Lucentis [Internet; cited 2020 November]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2012/125156s0069s0076lbl.pdf.[5] Roche data on file. [6] Khan M, et al. Targeting Angiopoietin in retinal vascular diseases: A literature review and summary of clinical trials involving faricimab. Cells. 2020;9(8):1869. [7] Clinical Trials.gov. A study to evaluate the long-term safety and tolerability of faricimab in participants with diabetic macular edema (Rhone-X) [Internet; cited 2020 November]. Available from: https://clinicaltrials.gov/ct2/show/NCT04432831. [8] Clinical Trials.gov. A study to evaluate the efficacy and safety of faricimab in participants with neovascular age-related macular degeneration (TENAYA) [Internet; cited 2020 November]. Available from: https://clinicaltrials.gov/ct2/show/NCT03823287.[9] Clinical Trials.gov. A study to evaluate the efficacy and safety of faricimab in participants with neovascular age-related macular degeneration (LUCERNE) [Internet; cited 2020 November]. Available from: https://clinicaltrials.gov/ct2/show/NCT03823300. [10] Pennington KL, DeAngelis MM. Epidemiology of age-related macular degeneration (AMD): associations with cardiovascular disease phenotypes and lipid factors. Eye and Vision. 2016;3:34.[11] Little K., et al. Myofibroblasts in macular fibrosis secondary to neovascular age-related macular degeneration-the potential sources and molecular cues for their recruitment and activation. EBioMedicine. 2018;38:283-91.[12] Clinical Trials.gov. A study to evaluate the efficacy and safety of faricimab (RO6867461) in participants with diabetic macular edema (YOSEMITE) [Internet; cited 2020 November]. Available from: https://clinicaltrials.gov/ct2/show/NCT03622580. [13] Clinical Trials.gov. A study to evaluate the efficacy and safety of faricimab (RO6867461) in participants with diabetic macular edema (RHINE) [Internet; cited 2020 November]. Available from: https://clinicaltrials.gov/ct2/show/NCT03622593. [14] National Eye Institute. Facts about diabetic eye disease [Internet; cited 2020 November]. Available from: https://nei.nih.gov/health/diabetic/retinopathy. [15] American Optometric Association. Diabetic retinopathy [Internet; cited 2020 November]. Available from: https://www.aoa.org/healthy-eyes/eye-and-vision-conditions/diabetic-retinopathy. [16] All About Vision. Macula Lutea [Internet; cited 2020 November]. Available from: https://www.allaboutvision.com/resources/macula. [17] Liu E, et al. Diabetic macular oedema: clinical risk factors and emerging genetic influences. Clinical and Experimental Optometry. 2017;100:569-76. [18] Park SJ, et al. Extent of exacerbation of chronic health conditions by visual impairment in terms of health-related quality of life. JAMA Ophthalmol. 2015;133:1267–75.[19] Holz FG, et al. Multi-country real-life experience of anti-vascular endothelial growth factor therapy for wet age-related macular degeneration. The British Journal of Ophthalmology. 2015;99:220-6.[20] Campochiaro, P, et al. Primary Analysis Results of the Phase 3 Archway Trial of the Port Delivery System With Ranibizumab for Patients With Neovascular AMD. American Society of Retina Specialists Annual Meeting; 2020 Jul 24-26. Roche Group Media Relations Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
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Roche’s faricimab meets primary endpoint and shows strong durability across two global phase III studies for diabetic macular edema, a leading cause...
AB Science announces the success of its capital raise for a total amount of 15 million euros
By Dr. Matthew Watson
NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, DIRECTLY OR INDIRECTLY, IN OR INTO THE UNITED STATES, CANADA, AUSTRALIA OR JAPAN – THIS PRESS RELEASE IS NOT AN OFFER TO SUBSCRIBE AND IS FOR INFORMATION ONLY
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AB Science announces the success of its capital raise for a total amount of 15 million euros
Allecra Therapeutics and Shanghai Haini Pharmaceutical Announce Exclusive Licensing Agreement for Cefepime/enmetazobactam for Greater China
By Dr. Matthew Watson
SAINT-LOUIS, France and WEIL AM RHEIN, Germany and SHANGHAI, China, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Shanghai Haini Pharmaceutical Co., Ltd. (Shanghai Haini), the subsidiary of Yangtze River Pharmaceutical Group (YRPG), and Allecra Therapeutics (Allecra) today announced that the companies have signed an exclusive licensing agreement under which Shanghai Haini will manufacture, develop and commercialize cefepime/enmetazobactam within Greater China, including Mainland China with Hong Kong and Macau, and Taiwan.
BetterLife Closes Acquisition of Second Generation Psychedelic Assets of Transcend Biodynamics LLC
By Dr. Matthew Watson
VANCOUVER, Dec. 20, 2020 (GLOBE NEWSWIRE) -- BetterLife Pharma Inc. (“BetterLife” or the “Company”) (CSE: BETR / OTCQB: BETRF / FRA: NPAU) is pleased to announce that further to its press release of December 8, 2020, the Company has completed its acquisition to acquire 100% of the assets in Transcend Biodynamics LLC (“Transcend”) in an all-stock transaction, for a total of 13,333,333 common shares at $0.75 per share, with the transaction valued at $10 million.
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BetterLife Closes Acquisition of Second Generation Psychedelic Assets of Transcend Biodynamics LLC
Comparison of concentrated fresh mononuclear cells and …
By daniellenierenberg
Autologous bone marrow mononuclear cell (BMMNC) transplantation has been widely studied in recent years. The fresh cell cocktail in BMMNCs, without going through the in vitro culture process, helps to establish a stable microenvironment for osteogenesis, and each cell type may play a unique role in bone regeneration. Our study compared the efficacy of concentrated fresh BMMNCs and cultured bone marrow-derived mesenchymal stem cells (BMSCs) in Beagle dogs for the first time. Fifteen-millimeter segmental bone defects were created in the animals tibia bones. In BMMNCs group, the defects were repaired with concentrated fresh BMMNCs combined with -TCP (n = 5); in cultured BMSC group, with in vitro cultured and osteo-induced BMSCs combined with -TCP (n = 5); in scaffold-only group, with a -TCP graft alone (n = 5); and in blank group, nothing was grafted (n = 3). The healing process was monitored by X-rays and single photon emission computed tomography. The animals were sacrificed 12months after surgery and their tibias were harvested and analyzed by microcomputed tomography and hard tissue histology. Moreover, the microstructure, chemical components, and microbiomechanical properties of the regenerated bone tissue were explored by multiphoton microscopy, Raman spectroscopy and nanoindentation. The results showed that BMMNCs group promoted much more bone regeneration than cultured BMSC group. The grafts in BMMNCs group were better mineralized, and they had collagen arrangement and microbiomechanical properties similar to the contralateral native tibia bone. These results indicate that concentrated fresh bone marrow mononuclear cells may be superior to in vitro expanded stem cells in segmental bone defect repair. 2020 The Authors. STEM CELLS TRANSLATIONAL MEDICINE published by Wiley Periodicals LLC on behalf of AlphaMed Press.
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How Researchers Are Making Do in the Time of COVID-19 – The Wire Science
By daniellenierenberg
Image: UN/Unsplash.
One of the astonishing aspects of the human response to the COVID-19 pandemic has been how quickly scientists pivoted to studying every facet of the virus in order to mitigate the loss of life and plan for a return to normalcy. At the same time, a lot of non-coronavirus research ground to a near halt.
With research labs and offices shuttered for all but essential workers, many scientists were stuck at home, their fieldwork and meetings canceled and planned experiments kicked down the road as they struggled to figure out how to keep their research programs going. Many took the opportunity to catch up on writing grants and papers; some in between caring for kids came up with strategic workarounds to keep the scientific juices flowing.
To gauge how researchers in different fields are managing,Knowable Magazine spoke with an array of scientists and technical staff among them a specialist keeping alive genetically important strains of fruit flies, the maintenance chief of an astronomical observatory working to keep telescopes safe and on standby during the lockdown, and a paediatrician struggling to manage clinical trials for a rare genetic disease. Here are a few slices of scientific life during the pandemic.
Agnieszka Czechowicz, Stanford University School of Medicine
Czechowicz is a paediatrician in Stanfords division of stem cell transplantation and regenerative medicine, where she manages a research group that develops new therapies and conducts clinical trials on rare genetic diseases.
Agnieszka Czechowiczs father suffers from severe Parkinsons disease. The coronavirus pandemic forced him to remain indoors and away from people, robbing him of the physical conditioning and social interactions he needs to cope with his disease. A recent fall left him in the hospital, bringing the additional worry that he might contract COVID-19 there and isolating him further.
For Czechowicz, his situation brought into sharp relief the challenges the coronavirus has forced upon those carrying out clinical trials, including those she is running, which involve patients traveling to hospitals around the country. Would I have him travel to any clinical site right now for a new Parkinsons treatment? she says. Absolutely not.
The pandemic forced Czechowicz to halt clinical trials she oversees for a rare genetic disease of children called Fanconi anAemia, a condition that impairs the bodys ability to repair damaged DNA and often leads to bone marrow failure and cancer. The treatment Czechowicz and colleagues are testing involves extracting blood-forming stem cells from the patients bone marrow, inserting a healthy copy of a missing or malfunctioning gene and then reinfusing those cells back into the patient.
Every aspect of what I do is massively impacted by the pandemic, Czechowicz says. One of her early-stage clinical trials involves testing the safety of the therapy. But during the initial shutdown which started in mid-March and lasted for two months her patients could not readily travel to Stanford for the necessary follow-up visits, and remote monitoring was difficult.
Theres special blood testing and bone marrow testing that we need to do. In particular, its critical to get the samples to make sure the patients, for example, arent developing leukAemia, she says. Theres no way to know that without really checking the bone marrow. She had to clear large hurdles to get her patients evaluated.
Another early-stage trial, designed to determine the effectiveness of the therapy, also had to stop enrolling new patients. Because speed is important when it comes to treating Fanconi anaemia the children are likely losing stem cells all the time any delay in treatment can be a source of great anxiety for their parents. Czechowicz had to explain to them why the trials were temporarily halted. It was really challenging to have these discussions with the families, she says.
With the easing of travel and workplace restrictions, the families began traveling to Stanford in June but with infections back on the rise, many families are becoming hesitant again, says Czechowicz. Fortunately, her trials are small, so she can guide each family through the process of safely resuming the trials and continuing with follow-up. Her own team also has to follow strict safety protocols. For example, even though her lab has 10 members, only two can be in the lab at any one time, and only one parent is allowed into the clinic with the child.
Not all clinical trials can pay such close attention to individual patients. Large trials with hundreds of patients can involve multiple sites and require much more monitoring, so resuming those remains difficult. Also, restrictions on working full bore are slowing the pipeline for new therapies. The impact of that, were not going to see for many years to come, Czechowicz says.
Abolhassan Jawahery, University of Maryland, College Park
Jawahery is a particle physicist and a member of LHCb, one of the main experiments at the Large Hadron Collider (LHC) at CERN, the particle physics laboratory near Geneva.
In December 2018, well before the coronavirus pandemic began, the LHC shut down for upgrades. Housed in a 27-kilometre-long tunnel about 100 meters underground, the LHC accelerates two beams of protons, one clockwise and one counterclockwise, and makes them collide head-on at four locations. There, four gigantic subterranean detectors ATLAS, CMS, LHCb and ALICE sift through the debris of particles created by the collisions, looking for evidence of new physics. (For example, ATLAS and CMS found the Higgs boson, the fundamental particle of the Higgs field, which gives all elementary particles their mass.)
For its next set of experiments, which aim to probe the properties of subatomic particles with greater precision, the LHC needed to increase the intensity of its proton beams. Consequently, the four detectors needed to be upgraded too, to handle the resultant higher temperatures and increased radiation at the sites of the particle collisions. The work was on track for a restart around May 2021 until the pandemic swept all the scientists careful plans away.
The LHC and its four detectors are each run by a separate collaboration. CERN, which manages the LHC, is hopeful it can restart the collider by February 2022. They think that they can get the accelerator going if there are no more major catastrophic events, says physicist Abolhassan Jawahery. But the impact on the four detectors is less clear.
For the LHCb upgrade, Jawaherys team at the University of Maryland had been working on building about 4,000 extremely sensitive electronic circuit boards. These boards have to be burned in before they can be sent to CERN. We put them in an oven, literally cooking the boards and then running extensive tests in order to get them ready so that we can put them in the accelerator and run them for 10 to 20 years, says Jawahery. And none of that could be done during the pandemic shutdown.
The team resumed its work in June, but with restrictions put in place by the state of Maryland. Jawahery runs two labs, and for months was allowed only two people at a time in one lab and three in the other, making progress extremely slow. Still, his team is fortunate that it does not depend on supplies from countries hit hard by the coronavirus. Other labs werent so lucky. Scientists in Milan, for example, built some electronics and detector components for the LHCb, and a lab at Syracuse University in New York built sensors that relied on shipments from Milan. When Milan was completely closed down at the height of the pandemic, Syracuse, too, stopped working on Milan-dependent components.
For Jawahery the lockdown had a silver lining. The LHCs most recent run had produced about 25 gigabytes of data per second but his team had found little time to analyse any of it before the pandemic. We were complaining that we were spending all our time building the new instrument and the data keeps on coming, he says. When he and his team were locked out of their labs, they turned to their data backlog. We could do actual physics, he says. We are already getting ready to publish some papers.
Gordon Gray, Princeton University
Gray is a professionalDrosophila specialist in the department of molecular biology.
Gordon Gray has been called the chef de cuisine of Princetons fly kitchen, where he has been feeding flies for 46 years. He concocts meals for millions of fruit flies, at least 150 litres each week. When the pandemic hit in March and universities around the world shut down, Princeton deemed Grays work an essential service: The Drosophilafruit flies could not be allowed to die off.
Princetons flies include mutant and transgenic strains everything from ones that allow researchers to study the genes that influence normal development of a fly embryos organs, to those that have cancer-causing mutations. If the flies starved, researchers would need months or years to recreate these strains, says Princeton molecular biologist Elizabeth Gavis. And often, as techniques in molecular biology improve, the biologists reexamine flies they had studied earlier to get a more fine-grained understanding, making it worthwhile to preserve the strains.
Normally, if a lab had to shut down, researchers would send their flies to stock centres, such as one at Bowling Green State University in Ohio, that preserve the flies as part of their genetic library. But the stock centres couldnt handle Princetons flies, so Gray found himself on his own. Its basically catch as catch can with regards to the various labs here, just to keep things operational, he says.
For months, university pandemic restrictions have allowed only one person to be in Grays kitchen at a time. This has caused problems. Before the pandemic began, Gray, who is in his late 60s, had started training someone as a backup. But because of the one-person restriction, Gray and his trainee havent been able to work together. Gray envisions doing so soon, while wearing masks, keeping nearly 12 feet apart and communicating using hand signals.
To whip up a batch of fly food, or media, Gray uses a 50-litre steel cauldron, to which is attached a mixer that looks like an outboard motor. Gray fills the cauldron with water and adds agar, sugars, yeasts, salts and cornmeal, then brings it to a boil, all the while stirring watchfully. You dont want it to boil over, because when it does you wind up with a gigantic pancake on the floor, which you have to scoop up immediately because it gels, he says. Once the suspension cools to the right temperature, Gray adds an acid to inhibit mould, then dispenses precise amounts of the media into bottles and vials.
Even before the pandemic, Grays kitchen was isolated, to keep errant fruit flies from contaminating the pristine media. But at least he could work regular hours, because he knew the rhythms of the 10 or so fly labs he cooked for. That has changed. Labs, restricted to two occupants at a time, are now working seven days a week on rotating shifts. Gray comes in to work at all hours, because he cannot predict when each batch of fly food will run out and hell need to cook more.
He tries to work mostly at night to avoid coming into contact with others. But he still worries for his health, given his asthma and age-related risk. The relentless pandemic is taking a toll. Its exhausting, he says. It doesnt help not knowing when we will return to a sense of normalcy.
Celeste Kidd, University of California, Berkeley
Kidd is a child developmental psychologist who uses behavioural tests and computational methods to understand how children acquire knowledge.
When UC Berkeley locked down in March, Celeste Kidd found herself closeted at home, dealing simultaneously with virtual meetings and her three-year-old son. During the early days of the pandemic, Kidd kept a supply of treats handy, and when her toddler came up to her during a meeting shed sneak him some under the desk. But she hadnt accounted for how long the pandemic would last. It turns out thats not a good strategy, long term, she says. I was very literally rewarding him for bad behaviour.
Kidds son soon learned that acting up during her meetings meant more candy. I knew that would happen. I did it anyway because I didnt have the bandwidth to come up with a better solution, she says. But Kidd knew from her own research that children are also extremely flexible and can unlearn behaviours. Eventually, she had a chat with her son. First, she admitted to him that she had made a mistake by giving him candy when he disrupted her meetings, and that was bad of her. Then she brought in new rules: no candy for misbehaving and misbehaviour could even mean no treats for the rest of day. We had some meltdown moments, says Kidd. But he gets it now and he doesnt do those things.
Her son may be the only child Kidd gets to interact with during the pandemic. Thats a huge loss for her research, because the bulk of her work focuses on young children. In normal times, families would bring their children to her lab, where her research team would track their gaze as they watched videos. In one study, for example, infants about seven to nine months old would look away (demonstrating lack of interest) when the events in the video were either too complex or too simple, suggesting that infants use their cognitive resources for stimuli that have just the right amount of information.
Such work, of course, requires the presence of parent, child and researchers, all in the same room. None of that is going to happen anytime soon, she says. Those families are not going to feel comfortable coming in for a while.
Kidd is also concerned about the impact of the pandemic on younger scientists. One of her undergraduate students had spent six months working on a study aimed at exploring the complexity of kids play patterns using physical objects and their relation to working memory and other cognitive resources. The university had approved the protocol, but shelter-in-place orders went into effect the week the first child was to come for the experiment. I feel so bad for her as a young scientist, to have done all this hard work and then right when you get to the fun part, which is collecting the data and finding out if her ideas have lasting merit, she doesnt get to do that part, Kidd says.
The situation might be even worse for grad students and postdocs. All of them are experiencing a big blow to morale in general, because there is so much uncertainty about what the future holds, she says. University budget cuts mean fewer slots for graduate students and fewer jobs for postdocs. Its very hard to stay motivated and get things done when youre not sure if there will be a payoff in the future, says Kidd. Thats literally a study that we ran in the lab so were all acutely aware of it.
Maxime Boccas, ESO Paranal Observatory
Boccas is the head of maintenance, support and engineering at the European Southern Observatorys Paranal Observatory in Chile.
When the massive domes of the Very Large Telescope, a constellation of four 8-meter-class telescopes atop Mount Paranal in Chiles Atacama Desert, open to the night sky each evening and the telescopes get ready for observations, its like a dragon waking up.
When the pandemic hit in March, the dragon on Mount Paranal closed its eyes to the cosmos and slept the first shutdown in its 20-year history, which included a major earthquake in 2010 that paralyzed much of the rest of Chile. For those who had to leave Paranal, it was like being sent away from home. We spend 40% of our life here, says Maxime Boccas, who oversaw the process of ensuring an orderly shutdown of the sites scientific and technical facilities. We work and sleep here, and we stay here eight days in a row. Some of Boccass colleagues have been doing that for 20 to 25 years. Leaving Paranal was like leaving their second home. That was a weird feeling.
The skeleton staff just 20 of the normal 150 or so people remained on site kept the observatory safe, ensuring that essential systems continued working: computers that astronomers were accessing remotely, the fire detection system and the earthquake protection system essential for protecting the 8-meter-wide primary mirrors from Chiles frequent quakes. The mirrors will likely never be made again, says Boccas. All the factories that cast and polished them are dismantled. If we lost a mirror, it would take between 5 and 10 years to build up the factory again and fabricate it. So each mirror has an airbag a tube that inflates around it when the system detects tremors and other protections.
During the shutdown, astronomers kept their fingers crossed. They were anxious that no big thing, like a supernova in our galaxy, would explode, Boccas says. The heavens have been quiet, but the six-month shutdown harmed research that involves continuously monitoring the same patch of the sky for transient phenomena such as gamma ray bursts. It creates a hole in their science program, says Boccas.
The observatory began a slow return to normalcy on September 9. Boccas is overseeing the reawakening of each telescope, one at a time. The staff still less than full strength is now working in shifts that have doubled from 8 to 15 days to limit the amount of travel to and from the site. The four large telescopes are now up and running again, and Boccas hopes they will be back to working together as one by the end of January.
Boccas, his crew and a few lucky astronomers are glad to be back at Paranal. It really feels like a family and I think everyone has noticed that, he says. Even in the kitchen, they have to cook for 30 people instead of 150, so the quality of the food is different, its slightly better.
But even as people return to the observatory, Boccas worries about long-term effects of the shutdown. Given the reduced staff, he has had to cut down on the frequency of preventive maintenance tasks, such as changing belts and lubricating motors, potentially shortening the lifetime of some components. We will not know until six months, a year or three years from now, he says.
This article is part ofReset: The Science of Crisis & Recovery, an ongoing series exploring how the world is navigating the coronavirus pandemic, its consequences and the way forward. Reset is supported by a grant from the Alfred P. Sloan Foundation.
Anil Ananthaswamy is a science journalist who enjoys writing about cosmology, consciousness and climate change. Hes a 2019-20 MIT Knight Science Journalism fellow. His latest book is Through Two Doors at Once. http://www.anilananthaswamy.com.
This article originally appeared in Knowable Magazine, an independent journalistic endeavour from Annual Reviews.
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How Researchers Are Making Do in the Time of COVID-19 - The Wire Science
Stem Cell or Bone Marrow Transplant Side Effects
By daniellenierenberg
Problems soon after transplant
Many of the problems that can happen shortly after the transplant come from having the bone marrow wiped out by medicines or radiation just before the transplant. Others may be side effects of the conditioning treatments themselves.
Your transplant team can help you cope with side effects. Some can be prevented, and most can be treated to help you feel better.This is not a complete list and you should tell your doctor or transplant team about any problems you have or changes you notice. Some of these problems can be life-threatening, so its important to be able to reach your doctor or transplant team at night, on weekends, and during holidays. Ask for their after hours contact numbers to makesure you will be able to do this.
Mucositis (inflammation or sores in the mouth) is a short-term side effect that can happen with chemo and radiation. It usually gets better within a few weeks after treatment, but it can make it very painful to eat and drink.
Good nutrition is important for people with cancer. If mouth pain or sores make it hard to eat or swallow, your transplant team can help you develop a plan to manage your symptoms.
Because chemotherapy drugs can cause severe nausea and vomiting, doctors often give anti-nausea medicines at the same time as chemo to try to prevent it. As much as possible, the goal is to prevent nausea and vomiting, because its easier to prevent it than it is to stop it once it starts. Preventive treatment should start before chemo is given and should continue for as long as the chemo is likely to cause vomiting, which can be up to 7 to 10 days after the last dose.
No one drug can prevent or control chemo-related nausea and vomiting 100% of the time. In many cases, two or more medicines are used. Youll need to tell your transplant team how well the medicines are controlling your nausea and vomiting. If they arent working, they will need to be changed.
For at least the first 6 weeks after transplant, until the new stem cells start making white blood cells (engraftment), you can easily get serious infections. Bacterial infections are most common during this time, but viral infections that were controlled by your immune system can become active again. Fungal infections can also be an issue. And even infections that cause only mild symptoms in people with normal immune systems can be quite dangerous for you. This is because right after the transplant you don't have many white blood cells that are working well, and they are the primary immune cells that fight off infections.
You may be given antibiotics to try to prevent infections until your blood counts reach a certain level. For instance, pneumocystis pneumonia (often called PCP) is a common infection thats easy to catch. Even though the germ doesnt harm people with normal immune systems, for others it can cause fever, cough, and serious breathing problems. Antibiotics are often used to keep transplant patients from getting this.
Your doctor may check you before the transplant for signs of certain infections that may become active after transplant, and give you special medicines to keep those germs under control. For example, the virus called CMV (cytomegalovirus) is a common infection that many adults have or had in the past. Adults with healthy immune systems may not have any symptoms because their immune system can keep the virus under control. But, CMV can be a cause of serious pneumonia in people who have had transplants, because the transplant lowers the amount of white blood cells they have. Pneumonia from CMVmainly happens to people who were already infected with CMV, or whose donor had the virus. If neither you nor your donor had CMV, the transplant team might follow special precautions to prevent this infection while you are in the hospital.
After engraftment, the risk of infection is lower, but it still can happen. It can take 6 months to a year after transplant for the immune system to work as well as it should. It can take even longer for patients with graft-versus-host disease (GVHD, see below). It's important to talk to your cancer care team about your risk for infection during this time.
Because of the increased risk, you will be watched closely for signs of infection, such as fever, cough, shortness of breath, or diarrhea. Your doctor may check your blood often, and extra precautions will be needed to keep you from being exposed to germs. While in the hospital, everyone who enters your room must wash their hands well. They may also wear gowns, shoe coverings, gloves, and masks.
Since flowers and plants can carry bacteria and fungi, theyre not allowed in your room. For the same reason, you may be told not to eat certain fresh fruits and vegetables. All your food must be well cooked and handled very carefully by you and family members. You might need to avoid certain foods for a while.
You may also be told to avoid contact with soil, feces (stool, both human and animal), aquariums, reptiles, and exotic pets. Your team may tell you to avoid being near disturbed soil, bird droppings, or mold. You will need to wash your hands after touching pets. Your family may need to move the cats litter box away from places you eat or spend your time. Also, you should not clean pet cages or litter boxes during this time. Instead, give this task to a family member or friend.
Your transplant team will tell you and your family in detail about the precautions you need to follow. There are many viruses, bacteria, and fungi that can cause infection after your transplant. You may be at risk for some more than others.
Despite all these precautions, patients often develop fevers, one of the first signs of infection. In fact, sometimes fever is the only sign of infection, so it's very important to contact your cancer care team if you have one or if you have any other signs of infection. You'll probably be asked to take your temperature by mouth every day or twice a day for a while. And your cancer care team will let you know when you should call in your temperature to them. If you get a fever, tests will be done to look for possible causes of the infection (chest x-rays, urine tests, and blood cultures) and antibiotics will be started.
After transplant, youre at risk for bleeding because the conditioning treatment destroys your bodys ability to make platelets. Platelets are the blood cells that help blood to clot. While you wait for your transplanted stem cells to start working, your transplant team may have you follow special precautions to avoid injury and bleeding.
Platelet counts are low for at least several weeks after transplant. In the meantime, you might notice easy bruising and bleeding, such as nosebleeds and bleeding gums. If your platelet count drops below a certain level, a platelet transfusion may be needed. Youll need to follow precautions until your platelet counts stay at safe levels.
It also takes time for your bone marrow to start making red blood cells, and you might need red blood cell transfusions from time to time as you recover.
For more information on the transfusion process, see Blood Transfusion and Donation.
Pneumonitis is a type of inflammation (swelling) in lung tissue thats most common in the first 100 days after transplant. But some lung problems can happen much later even 2 or more years after transplant.
Pneumonia caused by infection happens more often, but pneumonitis may be caused by radiation, graft-versus-host disease, or chemo rather than germs. Its caused by damage to the areas between the cells of the lungs (called interstitial spaces).
Pneumonitis can be severe, especially if total body irradiation was given with chemo as part of the pre-transplant (conditioning) treatment. Chest x-rays will be taken in the hospital to watch for pneumonitis as well as pneumonia. Some doctors will do breathing tests every few months if you have graft-versus-host disease (see next section).
You should report any shortness of breath or changes in your breathing to your doctor or transplant team right away. There are many other types of lung and breathing problems that also need to be handled quickly.
Graft-versus-host disease (GVHD) can happen in allogeneic transplants when the immune cells from the donor see your body as foreign. (Remember: The recipients immune system has mostly been destroyed by conditioning treatment and cannot fight back, so the new stem cells make up most of the immune system after transplant.) The donor immune cells may attack certain organs, most often the skin, gastrointestinal (GI) tract, and liver. This can change the way the organs work and increase the chances of infection.
GVHD reactions are very common and can range from barely noticeable to life-threatening. Doctors think of GVHD as acute or chronic. Acute GVHD starts soon after transplant and lasts a short time. Chronic GVHD starts later and lasts a long time. A person could have one, both, or neither type of GVHD.
Acute GVHD can happen 10 to 90 days after a transplant, though the average time is around 25 days.
About one-third to one-half of allogeneic transplant recipients will develop acute GVHD. Its less common in younger patients and in those with closer HLA matches between donor and the patient.
The first signs are usually a rash, burning, and redness of the skin on the palms and soles. This can spread over the entire body. Other symptoms can include:
Doctors try to prevent acute GVHD by giving drugs that suppress the immune system, such as steroids (glucocorticoids), methotrexate, cyclosporine, tacrolimus, or certain monoclonal antibodies. These drugs are given before acute GVHD starts and can help prevent serious GVHD. Still, mild GVHD will almost always happen in allogeneic transplant patients. Other drugs are being tested in different combinations for GVHD prevention.
The risk of acute GVHD can also be lowered by removing immune cells called T-cells from the donor stem cells before the transplant. But this can also increase the risk of viral infection, leukemia relapse, and graft failure (which is discussed later). Researchers are looking at new ways to remove only certain cells, called alloactivated T-cells, from donor grafts. This would reduce the severity of GVHD and still let the donor T-cells destroy any cancer cells left.
If acute GVHD does occur, it is most often mild, mainly affecting the skin. But sometimes it can be more serious, or even life-threatening.
Mild cases can often be treated with a steroid drug applied to the skin (topically) as an ointment, cream, or lotion, or with other skin treatments. More serious cases of GVHD might need to be treated with a steroid drug taken as a pill or injected into a vein. If steroids arent effective, other drugs that affect the immune system can be used.
Chronic GVHD
Chronic GVHD can start anywhere from about 90 to 600 days after the stem cell transplant. A rash on the palms of the hands or the soles of the feet is often the earliest sign. The rash can spread and is usually itchy and dry. In severe cases, the skin may blister and peel, like a bad sunburn. A fever may also develop. Other symptoms of chronic GVHD can include:
Chronic GVHD is treated with medicines that suppress the immune system, much like those used for acute GVHD. These drugs can increase your risk of infection for as long as you are treated for GVHD. Most patients with chronic GVHD can stop the immunosuppressive drugs after their symptoms improve.
Hepatic veno-occlusive disease (VOD) is a serious problem in which tiny veins and other blood vessels inside the liver become blocked. Its not common, and it only happens in people with allogeneic transplants, and mainly in those who got the drugs busulfan or melphalan as part of conditioning, or treatment that was given before the transplant.
VOD usually happens within about 3 weeks after transplant. Its more common in older people who had liver problems before the transplant, and in those with acute GVHD. It starts with yellowing skin and eyes, dark urine, tenderness below the right ribs (this is where the liver is), and quick weight gain (mostly from fluid that bloats the belly). It is life-threatening, so early diagnosis of VOD is very important. Researchers continue to find ways to try to measure a person's chances of getting VOD so that treatment can start as soon as possible.
Grafts fail when the body does not accept the new stem cells (the graft). The stem cells that were given do not go into the bone marrow and multiply like they should. Graft failure is more common when the patient and donor are not well matched and when patients get stem cells that have had the T-cells removed. It can also happen in patients who get a low number of stem cells, such as a single umbilical cord unit. Still, its not very common.
Graft failure can lead to serious bleeding and/or infection. Graft failure is suspected in patients whose counts do not start going up within 3 to 4 weeks of a bone marrow or peripheral blood transplant, or within 7 weeks of a cord blood transplant.
Although it can be very upsetting to have this happen, these people can get treated with a second dose of stem cells, if they are available. Grafts rarely fail, but if they do it can result in death.
The type of problems that can happen after a transplant depend on many factors, such as the type of transplant done, the pre-transplant chemo or radiation treatment used, the patients overall health, the patients age when the transplant was done, the length and degree of immune system suppression, and whether chronic graft-versus-host-disease (GVHD) is present and how bad it is. The problems can be caused by the conditioning treatment (the pre-transplant chemotherapy and radiation therapy), especially total body irradiation, or by other drugs used during transplant (such as the drugs that may be needed to suppress the immune system after transplant). Possible long-term risks of transplant include:
The medicines used in transplants can harm the bodys organs, such as the heart, lungs, kidneys, liver, bones/joints, and nervous system. You may need careful follow-up with close monitoring and treatment of the long-term organ problems that the transplant can cause. Some of these, like infertility, should be discussed before the transplant, so you can prepare for them.
Its important to find and quickly treat any long-term problems. Tell your doctor right away if you notice any changes or problems. Physical exams by your doctor, blood work, imaging tests, lung/breathing studies, and other tests will help look for and keep tabs on organ problems.
As transplant methods have improved, more people are living longer and doctors are learning more about the long-term results of stem cell transplant. Researchers continue to look for better ways to care for these survivors to give them the best possible quality of life.
The goal of a stem cell transplant in cancer is to prolong life and, in many cases, even cure the cancer. But in some cases, the cancer comes back (sometimes called relapse or recurrence depending on when it might occur after a transplant). Relapse or recurrence can happen a few months to a few years after transplant. It happens much more rarely 5 or more years after transplant.
If cancer comes back, treatment options are often quite limited. A lot depends on your overall health at that point, and whether the type of cancer you have responds well to drug treatment. Treatment for those who are otherwise healthy and strong may include chemotherapy or targeted therapy. Some patients who have had allogeneic transplants may be helped by getting white blood cells from the same donor (this is called donor lymphocyte infusion) to boost the graft-versus-cancer effect. Sometimes a second transplant is possible. But most of these treatments pose serious risks even to healthier patients, so those who are frail, older, or have chronic health problems are often unable to have them.
Other options may include palliative (comfort) care, or a clinical trial of an investigational treatment. Its important to know what the expected outcome of any further treatment might be, so talk with your doctor about the purpose of the treatment. Be sure you understand the benefits and risks before you decide.
Along with the possibility of the original cancer coming back (relapse) after it was treated with a stem cell transplant, there is also a chance of having a second cancer after transplant. Studies have shown that people who have had allogeneic transplants have a higher risk of second cancer than people who got a different type of stem cell transplant.
A cancer called post-transplant lymphoproliferative disease (PTLD), if it occurs, usually develops within the first year after the transplant. Other conditions and cancers that can happen are solid tumor cancers in different organs, leukemia, and myelodysplastic syndromes. These other conditions, if they occur, tend to develop a few years or longer after the transplant.
Risk factors for developing a second cancer are being studied and may include:
Successfully treating a first cancer gives a second cancer time (and the chance) to develop. No matter what type of cancer is treated, and even without the high doses used for transplant, treatments like radiation and chemo can lead to a second cancer in the future.
Post-transplant lymphoproliferative disorder (PTLD) is an out-of-control growth of lymph cells, actually a type of lymphoma, that can develop after an allogeneic stem cell transplant. Its linked to T-cells (a type of white blood cell that is part of the immune system) and the presence of Epstein-Barr virus (EBV). T-cells normally help rid the body of cells that contain viruses. When the T-cells arent working well, EBV-infected B-lymphocytes (a type of white blood cell) can grow and multiply. Most people are infected with EBV at some time during their lives, but the infection is controlled by a healthy immune system. The pre-transplant treatment given weakens the immune system, allowing the EBV infection to get out of control, which can lead to a PTLD.
Still, PTLD after allogeneic stem cell transplant is fairly rare. It most often develops within 1 to 6 months after allogeneic stem cell transplant, when the immune system is still very weak.
PTLD is life-threatening. It may show up as lymph node swelling, fever, and chills. Theres no one standard treatment, but its often treated by cutting back on immunosuppressant drugs to let the patients immune system fight back. Other treatments include white blood cell (lymphocyte) transfusions to boost the immune response, using drugs like rituximab to kill the B cells, and giving anti-viral drugs to treat the EBV.
Even though PTLD doesnt often happen after transplant, its more likely to occur with less well-matched donors and when strong suppression of the immune system is needed. Studies are being done to identify risk factors for PTLD and look for ways to prevent it in transplant patients who are at risk.
Most people who have stem cell transplants become infertile (unable to have children). This is not caused by the cells that are transplanted, but rather by the high doses of chemo and/or radiation therapy used. These treatments affect both normal and abnormal cells, and often damage reproductive organs.
If having children is important to you, or if you think it might be important in the future, talk to your doctor about ways to protect your fertility before treatment. Your doctor may be able to tell you if a particular treatment will be likely to cause infertility.
After chemo or radiation, some women may find their menstrual periods become irregular or stop completely. This doesnt always mean they cannot get pregnant, so birth control should be used before and after a transplant. The drugs used in transplants can harm a growing fetus.
The drugs used during transplant can also damage sperm, so men should use birth control to avoid starting a pregnancy during and for some time after the transplant process. Transplants may cause temporary or permanent infertility for men as well. Fertility returns in some men, but the timing is unpredictable. Men might consider storing their sperm before having a transplant.
For more information on having children after being treated for canceror sexual problems related to cancer treatment, see Fertility and Sexual Side Effects.
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Stem Cell or Bone Marrow Transplant Side Effects