By Sean Marshall
Not much new sickle cell news this week but a small yet interesting story did emerge. The good news this week, according to TheTandD.com more than 130 individuals turned out for a three mile awareness walk for sickle cell anemia. The event was held by the Orangburg Area Sickle Cell Anemia Foundation and was called Taking a step Towards Breaking the Sickle Cell Silence.
It was mentioned in the article that “the family of the late Francenia Williams of Orangeburg was honored during the ceremony. The retired teacher had worked with the foundation for more than 30 years.”
During the ceremony, the foundation received $5,000 in donations from both inside and outside the community. The article also explained that four individuals and the local Shriners of Islam Temple No. 3 donated a total of $2,000 while Grand Potentate David Wright from Islam Temple No. 4 of Aiken donated $2,500 and lastly Potentate John Bodrick of Koran Temple No. 88 donated $500.
despite being a relatively small article it shows that people still care about sickle cell disease and are willing to put both their money and time into finding a cure.
If you would like to see the article go to the link here: http://thetandd.com/lifestyles/raising-awareness-for-sickle-cell/article_d1834470-5899-11e4-8353-fb855a53f341.html
By Sean Marshall
Good news from the FDA this week. A press release from U.S. Food and Drug association has outlined what is to come from them to combat sickle cell disease in the future. According to the documents found on their site the FDA plans on dealing with sickle cell disease in all its stages, at least according to one of their spokespeople M.D. Anna T. Farrell.“These potential treatments are in different stages of development, including early and late clinical trials,” explained Farrell
The plan is for the FDA to grant companies the ability to what the press released called “a “fast track” designation.” This is where the drug companies and their sponsor are allowed early and more frequent interactions with the FDA to bring up any and both ethical and health issues concerns as well as any problems with developing, drug testing and the initial trials. It was said by Farrell “the purpose of these consultations is to bring the new product to market faster.”
It was also mentioned that “orphan products” would be designed. These are products that are intended to treat rare diseases affecting 200,00 individuals in the U.S.A per year.
If you would lie to learn more about what the FDA is doing follow the link below:
If you have any comments questions or concerns email us at firstname.lastname@example.org
~And other effective advancements in Transplants for SCD~
Author: Kara Martina B. Sc. Biopsychology
A Semi bone marrow transplant relies on a half-matched sibling to donate unaffected tissue. In the past, BMTs have been heavily and almost solely dependent on close HLA matches. In the States, finding an unrelated match can cost over 50 000 USD and sometimes the chance of finding said donor runs only 30%.
A semi bone marrow transplant sidesteps the restrictive nature of a typical BMT, using partially matched marrow followed by immediate chemotherapy to combat the rejection response. This semi-compatible approach has opened effective treatment for over 95% of children related to a half-match (1). The outcomes have been surprisingly favorable and given the actual limited resources that most people live with, this creative method may be the saving procedure for many with TM, SCD and leukemia (2).
In related findings, researchers have discovered that semi-mature and ‘silenced’ bone marrow cells induce tolerance rather than an undesirable immune response (3). The long foe of BMT, Graft Versus Host Disease (GVHD) can kill patents even after a successful procedure. Using semi-mature cells, with a muted 88 factor allowed rats in the experiment to withstand even fully mismatched grafts.
Another study found that administering donor B-cells immediately following a semi-allegoric transplant prolonged GVHD (4). Usually following a transplant, IV immunoglobulins are readily administered, including T-cells. This study administered only donor B-cells on grafted mice. T-cell depleted mice injected with only donor B-cells never developed GVHD, whereas mice with T-cells died of organ rejection.
Still in the making and forever perfecting, Bone marrow transplants and Semi-bone marrow transplants need further research and a more developed protocol following and preceding procedure. Graft Versus Host disease risks along with the effects of chemotherapy before transplants are both as formidable as the original disease BMTs aim to cure, be it leukemia, thalassemias or Sickle Cell Disease.
When will all the research we fund make a difference? When will the safest and best procedures and protocols develop such that those with blood disorders are not threatened by the very treatments meant to save their lives?
See more on hazardous effects of chemotherapy drugs here.
See more on specific chemo mechanisms here.
What can you do? Be aware, ask your doctor these 20 questions! Don’t just blindly accept or rely on a prescribed chemotherapy.
1. Luznik, L., Engstrom, L. W., Iannone, R. & Fuchs, E. J. Posttransplantation cyclophosphamide facilitates engraftment of major histocompatibility complex-identical allogeneic marrow in mice conditioned with low-dose total body irradiation. Biol. Blood Marrow Transplant. J. Am. Soc. Blood Marrow Transplant. 8, 131–138 (2002). - See more at: http://www.cure2children.org/content/making-cure-possible-even-more-children-using-mother%E2%80%99s-bone-marrow-save-her-child#sthash.ulQXkdVz.dpuf
2. Bolaños-Meade, J. et al. HLA-haploidentical bone marrow transplantation with post-transplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood (2012). doi:10.1182/blood-2012-07-438408 - See more at: http://www.cure2children.org/content/making-cure-possible-even-more-children-using-mother%E2%80%99s-bone-marrow-save-her-child#sthash.ulQXkdVz.dpuf
3. Yang, XJ., Meng, S., Zhu CF., Jiang H. &Wu WX. Semi-mature MyD88-silenced bone marrow dendritic cells prolong the allograft survival in a rat model of intestinal transplantation. Chin Med J (Engl). 2011 Jan;124(2):268-72. 4. Samuel, S., Azar, Y., Corchia, N. & Or R. Improved immune function with donor B-cell infusion after semi-allogeneic bone marrow transplantation in mice. Arch Med Res. 2008 Jan;39(1):61-8.
4. Samuel, S., Azar, Y., Corchia, N. & Or R. Improved immune function with donor B-cell infusion after semi-allogeneic bone marrow transplantation in mice. Arch Med Res. 2008 Jan;39(1):61-8.
By Sean Marshall
Not much in news updates this week however there was one interesting article that surfaced this week that needs to be seen.
An article on science20.com has surfaced shedding some light on cell conversion and how DNA bias may keep some diseases in circulation. It was explained by a new study out of the University of Pennsylvania that investigated the process in the context of evolution of human population and the genetic traits that follow said populations. The study was conducted by researchers Joseph Lachance and Sarah A. Tishkoff and ultimately the study found that “certain types of DNA sequences were found in gene conversion.” This according to the article may be an important factor in why certain hereditary disease persist more heavily in some population around the world then others.
The study outlined some reasons for why certain populations have genetic disorders one such example was the Amish. There research concluded that “have a higher risk of several genetic diseases due in part to a phenomenon called founder effects, whereby certain genes rise to prevalence in populations that originated with a relatively small number of individuals.”
When it came to sickle cell anemia the team had some interesting comments about sickle cell traits, “the classic example is sickle-cell anemia. It's an evolutionary trade-off because people with one copy of a sickle-cell mutation are highly protected from malaria.” If this is true it has some severe implications on how future generation of people with sickle cell can and will be treated.
The article also went on to say one of the reasons why the sickle cell gene is passed from person to person “Previously, researchers have found that during gene conversion DNA is more likely to be retained and copied if the allele that differs contains either a guanine (G) or a cytosine (C) nucleotide. Conversely, the DNA is more likely to be converted, or replaced, if the allele contains an adenine (A) or thymine (T). “
This means if there is a strong gene that is different and can provide some sort of genetic edge it would be copied. So we go back and find out the same gene that causes sickle cell also protect against malaria and is a strong genetic strain. However it was explained that even though the bias is small other factors come into play. “This bias is very small," Lachance said. "It's like a very slightly weighted coin. But over generations and across huge amounts of the genome, flipping the coin over and over again, we thought we would start to see an effect at the population level.
This article is so very important to go over because it is essential outlining why sickle cell exists and why scientists are having such a difficult time in destroying it. I would recommend reading the article in full detail. I've gone over the key points but there is still much more to go over.
The link to the article is here: http://www.science20.com/news_articles/cell_conversion_and_how_dna_bias_may_keep_some_diseases_in_circulation-146184
If you have any questions coments or concerns email us at email@example.com
By Sean Marshall
One story coming out of Philadelphia was that a children s hospital has created a coping kit for the families of young children with sickle cell anemia.
According to a press release from The Children's Hospital of Philadelphia the hospital along with Meghan Marsac, Ph. D., a clinical psychologist working with the hospital, Have created the next coping tool for children and their families who have sickle cell disease. The coping mechanize is called “The Coping Kit.”
It was explained that the kit provides ways of fighting the factors that cause stress, which in her studies showed significant damage not only to the patient but their families as well. To fght off problems like stress the kit includes a stuffed toy named Cellie, coping cards, and a book for caregivers. It was explained that “children use Cellie's zippered mouth to store their coping cards where they can find over 100 tips for dealing with numerous disease-related stresses such as medical procedures, hospital visits and feelings of fear and uncertainty.”
According to a study that took place from 2013-2014 on the effectiveness of the kit it was reported that “Most families learned new information and/or skills from using the The Cellie Kit including new ideas for coping, normal reactions to their disease and its treatment, ways to initiate conversations, how to promote emotional expression, and how to help their children have fun.”
If you would like to read the original follow the link here:http://www.prnewswire.com/news-releases/the-childrens-hospital-of-philadelphia-develops-a-coping-kit-for-pediatric-sickle-cell-disease-patients-and-families-277814141.html#
Other international news comes from the United States were congress has finally put into motion a public service combat sickle cell disease. Representative Davis Danny of Kentucky introduced the “Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act of 2014 and it has three priorities.
The three items were to “collect data on the prevalence and distribution of sickle cell disease, conduct sickle cell disease public health initiatives to improve access to care and health outcomes, and identify and evaluate strategies for prevention and treatment of sickle cell disease complications.”
This is big news the American government is moving forward and fighting sickle cell disease, they are actually bringing this up in congress. Bills are being passed to not just learn and fight the disease but to even improve access to medical equipment to combat sickle cell. This is a big step in the right direction and if the US government is taking action chances are the Canadian government will follow through. And if the worst case scenario occurs and Canada doesn’t follow people with sickle cell can at least travel to a close country for medical support.
Also issues with sickle cell disease have now been moved from “demonstration program from American Jobs Creation Act of 2004 to the Public Health Service Act.” This may all sound foolish but it proves that the disease is being taken seriously. It means Public Health is taking an interest rather then brush sickle cell aside it is being tackled head on.
All of this information has been provided by congresses website but if you would like to learn more you can find out the details here: https://www.congress.gov/bill/113th-congress/house-bill/5124
Like always if you have any questions comments or concerns email us at firstname.lastname@example.org
This section is solely to let our Sickle Soldiers tell their story trials & tribulations alongside things they feel are wrong in the Sickle Cell Community