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By Sean Marshall
It was announced this week by erurekalert.com as well as Boston medical and oakland medical that “treatment with the antiplatelet agent prasugrel does not significantly reduce the rate of pain crises or severe lung complications in children with sickle cell disease.” This information came to light by a report by the New England Journal of Medicine. This came as a shock because of the importance of the New England Journal is one of the largest and most geographically diverse international clinical trials on sickle cell disease to date. This was proven by a “trial that was a double blind, randomized, placebo-controlled phase 3 clinical trial held at 51 sites across 13 nations in the Americas, Europe, the Middle East, Asia and Africa. Led by researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and UCSF Benioff Children's Hospital Oakland,” This was all done because the clinic was attempting to actually reach a goal. That goal was “to create a trial the to determine whether prasugrel, a medication used in adult patients to reduce thrombotic cardiovascular events, could also significantly reduce the rate of rate of vaso-occlusive crises (VOCs) -- defined as pain crises or acute chest syndrome -- in children with sickle cell disease.” Prasugrel is a drug that is suppose to “prevent platelets from aggregating by blocking an enzyme called P2Y12.” The good news is that the drug “is approved for use in adult cardiac patients in the U.S. to reduce the risk of clots following angioplasty or insertion of an arterial stent.” This is a mixed bag. On the one hand it is disappointing that a drug that is suppose to be helping is not and there isn't an immediate solution, but it's always a good thing to know that certain medications are still being tested despite use by the public. If there are any comments, questions, or ideas please contact us at [email protected]
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Unfortunately there were no major news updates this week. Check in next week for more coverage.
It was reported by the Nation.com this week that Apollo Hospitals, India's leading super specialty hospitals that “it would collaborate with Nigeria in treating sickle cell anemia and bone marrow transplantation.” It was explained in the article that the “hospital made this known at a two-day continuous medical education (CME) organized by Diamond Helix Medical Assistance for medical doctors in Lagos.”
Other important information displayed was “the hospital will work with the Sickle Cell Foundation Nigeria so that patients would not have to travel to India for bone marrow transplant and other sickle cell disease problems.”What was stressed was that “Nigeria is already doing well in its treatment, adding that more can be done as sickle cell is very much a black people’s problem.” This whole article shows that not only are specialty hospitals taking interest in sickle cell but whole communities, it gives hope for the future and that's something that is always needed. For the original article follow the link below: http://thenationonlineng.net/hospital-partners-sickle-cell-foundation/ If there are any comments questions, concerns or ideas contact us at [email protected] By Sean Marshall
It was announced by Street Insider.com that Mast Therapeutics also known as NYSE:MSTX today released information explaining that their “nonclinical studies investigating vepoloxamer, its lead product candidate, will be featured in three poster sessions at the 57th Annual American Society of Hematology (ASH) Meeting and Exposition.” For those who need an update “The ASH Meeting is being held at the Orange County Convention Center in Orlando, Florida, December 5 through December 8, 2015.” The ASH meetings are all about the sharing of information around hematology, the study of blood. According to ASH's website these meetings are conducted help spread research knowledge as well as “the meetings provides an invaluable education experience and an opportunity to review thousands of scientific abstracts highlighting updates in the hottest topics in hematology.” What this all means is that if NYSE:MSTX is willing to come forward publicly and announce that they have made some sort of breakthrough with hematology treatments it must be a big deal. According to the report “the studies to be presented demonstrate the anti-adhesive effects of vepoloxamer on sickle red blood cells (RBC) as well as its protective effects on the membranes of red blood cells and platelets.”It was later expained by the report that vepoloxamer “was shown to result in statistically significant reductions in the number of adherent sickle RBC to immobilized vascular cell adhesion molecule (VCAM) and reductions in hemolysis in blood from patients with sickle cell disease.” The last part of good news was that “in other studies, vepoloxamer was shown to preserve platelet function under extended storage conditions as well as prevent exposure of a marker of platelet damage (phosphatidyl serine) in blood samples from patients with SCD.” So to summarize Mass Therapeutics newest drug Vepoloxamer is being shown off for what feats it can perform for the sickle cell community. This is great news. It means that people are one step closer to riding the world of sickle cell anemia. Mind you there will always be side effects to every drug and with the amount of publicity generated from these events it means that the drug is most likely not cheap. Either way a cure for sickle cell disease is closer then ever. The original article can be found here: http://www.streetinsider.com/Corporate+News/Mast+Therapeutics+(MSTX)+Announces+Vepoloxamer+Data+Selected+For+Presentation+at+ASH+Meeting/11076025.html ASH's site can be found here: http://www.hematology.org/Annual-Meeting/ If there are any comments questions or even new topic ideas send us an email at [email protected] By Sean Marshall
This week Lagos State governor Akinwunmi Ambode pledged his administration’s support towards completion of a bone marrow transplant center. For those who don't know Lagos is considered the largest city in Nigeria and a major financial center for the country. With an estimated population of 16,348,100. This means that even if a small fraction have sickle cell anemia it is often taken more seriously due to the economic factors in play. It was reported by News 24 Nigeria that the “facility is under construction at the Lagos University Teaching Hospital (LUTH).” The idea behind the center is that it will save lives. In fact the article even pointed out that “envisaged this will be key to save the lives of people suffering sickle cell disorder.” It was also explained that the governor made this proclamation when “the Chairman of Sickle Cell Foundation Nigeria (SCFN), Prof. Olu Akinyanju, and Chief Executive Officer of the Foundation, Anneite Akinsete, visited him at the Lagos House in Ikeja.” Ultimately this means that this was more of a political move to gain good publicity rather then for the betterment of the population. Although that could just be the pessimist speaking. Other information worth mentioning included “the governor added that the state government would provide support in line with the cardinal programs of his administration to touch as many lives as possible.” The original article can be found here: http://www.news24.com.ng/National/News/lagos-offers-support-to-sickle-cell-patients-20151110 If there are any comments questions or concerns or even new topic ideas please email us at [email protected] By Sean Marshall
It was announced this week by news medical net that a key protein in developing stem cells has been isolated. It was explained that these gene is “a key protein controlling stem cell properties that could make them more useful in regenerative medicine,” The protein itself is called zinc finger protein 217 or ZEF17 for short. According to the report ZEF217 regulates “the actions of genes that maintain a balance between stem cell self-renewal and differentiation.” What this means is that ZPF217 can be used to maintain or even create therapeutic stem cells. The main focus is to use this protein to help cure cancer as explained by Martin Walsh PhD. “At the same time, as the human ZNF217 is associated with poor survival in a variety of cancers, understanding how this protein operates in physiological conditions may help to predict cancer risk, achieve earlier diagnosis and provide novel therapeutic approaches." This doesn’t mean however that the implications for sickle cell anemia research isn't there. In fact the sickening of cells could be reversed of even stopped with a better understanding of ZEF217. The idea would be to apply the therapeutic methods on the particular DNA sequence that causes the red blood cells to sickle. Then if applied properly red blood cells could take on the normal non circled trait allowing for a normal life without fear of sickle cell related illness or complications. The original article can be found in the link below: http://www.news-medical.net/news/20151031/Key-protein-controls-stem-cell-properties-that-could-make-them-useful-in-regenerative-medicine.aspx If there are any comments questions or concerns email us at [email protected] By Sean Marshall
This week it was announced that the first national sickle cell anemia survey has closed. The survey was nation wide and had over 600 responses. This included 200 different responses per unique survey. The idea is to not gave them all identical because it can often cause issues with statistical analysis. The survey was designed by the Pricker institute to improve “the quality of health and social care for all, the development and implementation,” the ideas the questions were based off of commissioned work by the National Institute for Health Research Collaboration for leadership in Applied Health Research and Care Northwest London. The short form is NIHR CLAHRC NWL it's a lot to take in and to be honest the length of the name can be overwhelming but the important thing to remember is that they are partnered with Sickle Cell Society and are doing great work. The survey asked patients about their experiences focusing on “aspects of care that are important to them, including access to care services, information and support for their condition, and their views on care across inpatient, outpatient and emergency settings. Survey versions suitable for both pediatric and adult patients, and parents/ carers of young SCD patients, were available.” It was explained that “final data is currently being analyzed by the Picker Institute and the key findings will be shared as soon as they are available.” So we'll be looking forward to the results and keep everyone posted in the mean time. original article can be found here: http://www.blacknet.co.uk/first-national-survey-of-people-living-with-sickle-cell-disease-closes-for-analysis/ Any comments questions of concerns can be sent to [email protected] By Sean Marshall
This week it is officially easier to test for sickle cell anemia then ever before. According to clapway.com as well as medicalpress.com has reported that a new app along with a 3D printer could “hat can accurately diagnose and monitor sickle cell disease in the field or at a remote clinic, using just a few drops of blood.” The team behind the groundbreaking research is led by UConn assistant professor of mechanical engineering Savas Tasoglu his hopes are that anyone who cannot reach a hospital in a developing worlds, often where sickle cell anemia does the most damage, could test their blood and adding only a few drops to a device attached to their phone. The device or platform “contained in a lightweight, compact box that can be attached to a common Android smartphones.” Then the phone would run the app send it to the nearest 3D printer for the most accurate results including a mapping of the bloodstream and where potential treats that could occur. Meanwhile the app should analysis the results of the test and explain the best course of action to be taken by the patient. The only catch is that the blood “needs to mixed into a solution of gadolinium and sodium salts. This solution is then squirted through a small disposable transparent capillary tube that is clipped into the smartphone attachment between its two magnets. The tube is illuminated by the smartphone attachment’s LED light which is mounted opposite the phone’s camera.” So the only real concern would be the refilling o the solution and the cost of the app and device to attach to a persons phone. Ultimately though the price should hopefully outweigh the cost of life or at the very least outweigh the potential trip to a medical facility which for some is either a non option or a lengthy trip that could cost them many days. On a lighter note tough they app will revolutionize the lives of people in developing nations allowing them to much more easily regulate themselves and their loved ones. The two articles can be found below: http://clapway.com/2015/10/23/smartphone-test-for-sickle-cell-anemia123/ http://medicalxpress.com/news/2015-10-easy-sickle-cell-disease.html If there are any comments questions or concerns email us at [email protected] By Sean Marshall
The Seattle Times covered an article this week published an article on gene editing. It mainly focused on what could be considered ethical. However when the article focused on gene the implications editing can have those who sufferer from sickle cell anemia it became more interesting. The idea behind gene editing is that when a human embryo is forming certain traits in theory, and very soon in practice, a doctor could edit out potential problems. For instance they could edit out the sickle cell trait. As of right now scientists are calling the gene editing technique CRISPR-Cas9 to edit animals mainly but others are making the jump to human beings. The big controversy is that a team in China managed to edit a human embryo potential curing a deadly disease. This then lead to the debate about designer babies. This concept involves changing the entire genetic pattern of ones child to suit the wants of the parent. This is when the article starts to lose any connection with sickle cell and starts becoming something else. Although it doesn't really seem all that morally complicated of an issue to stop sickle cell disease. Most parents aren’t going to go overboard and ultimately this is all theoretical until CRISPR-Cas9 becomes more widespread. If you'd like to learn more about the issue you can find the original article here: http://www.seattletimes.com/seattle-news/health/gene-editing-holds-huge-promise-but-whats-ethical/ Other news this week another biopharmaceutical company engaging in developments for treating rare disease. It was announced that Emmaus Life Scenic inc, a biopharmaceutical company out of Orrance California has decided to develop commercial treatments for “therapies for rare and orphan diseases,” this did include sickle cell disease. There plan was to have “management present an abstract from its completed FDA phase 3 sickle cell disease (SCD) trial at the 9th annual Sickle Cell and Thalassaemia Advance Conference.” The news itself isn't anything spectacular but in recent weeks more and more companies are going public about their developments into possible cures or at the very least life easing drugs that affect sickle cell anemia. In the past month many pharmaceutical and biopharmaceutical companies are not only becoming publicly traded but are becoming much more profitable because they are banking on public interest and support due to claims of a sickle cell free world one day. The original article can be found here:http://news.sys-con.com/node/3489754 If there are any comments questions or concerns email us at [email protected] By Sean Marshall
An article published this week has stated that “Mast Therapeutics shows promise regarding sickle cell treatment as mentioned by opinion leader.” This article featured on Street Wis Journal one of Richmond Vermont’s business trading news organization has reported that Roth Capital health care annalist Michael Higgins has “shed some light regarding the feasibility and potential short and mid-term prospects of Mast Therapeutics.” Mast Therapeutics is a San-Diego based biopharmaceutical company. At least that's what it says on their website. The company itself specializes in treating various disease but as of recently they have been attempting to aid in the treatment and possible curing of sickle cell disease. What is so important about Mass Therapeutics right now is that they are teaming up with it was a the Rutgers Cancer Institute of New Jersey Higgins which has “been treating around 450 patients who have sickle cell anemia.” The reason for the treatment is that the area in recent years has seen an increase in sickle cell anemia. In fact stats given were around 70%. With the combined efforts of a big drug company and a Rutgers Cancer Institute there should be a positive outcome. Especially since Mast Theraputics stocks have risen because of the announcement. The article since it is primary business focused did mention that this has mainly been done in an attempt to bring up stick to 3 dollars a share. Oddly this could have a beneficially outcome. If it does increase to the desired affect it shows that a cure for sickle cell disease is profitable. Perhaps other companies will follow Mast Therapeutics footsteps if all goes as planned. But what is really important is the people who lies should hopefully be improved by the overall research. Especially those in New Jersey. The original article can be found in the link: http://www.streetwisejournal.com/mast-therapeutics-shows-promise-regarding-sickle-cell-treatment-as-mentioned-by-opinion-leader/11426/ If you have any comments questions or concerns email us at [email protected] |
AboutThis section is solely to let our Sickle Soldiers tell their story trials & tribulations alongside things they feel are wrong in the Sickle Cell Community Archives
March 2016
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