By Sean Marshall
Unfortunately for the past few weeks sickle cell news has been light luckily the past few days there has been some recent activity. However there was some major development coming from Gulf News Journal. According to ther medical news site recently “Dilaforette, AGU to collaborate on development of sickle cell disease drug.”
This is big news because Dilaforette is a major Swedish drug company. According to their website they are “developing sevuparin, an innovative, proprietary polysaccharide drug, which has potential to restore blood flow and prevent further microvascular obstruction in sickle cell disease patients.” As well as the AGU or Arabian Gulf University is one of the leading researchers of sickle cell disease. This means the partnership of these two researching giants can only mean great things for the sickle cell community.
According to the article presented on Gulf News Journal “Under the agreement, AGU will give Dilaforette up to $1.2 million in non-dilutive funding and help recruit patients for a phase-two study evaluating the safety of the drug for the treatment of a common painful complication of sickle cell disease known as vaso-occlusive crisis (VOC).”
One of the major issues the aricle touched upon was that “in addition to strengthening AGU's clinical research capabilities, the collaboration deal with Dilaforette also involves the Bahrain-based university receiving royalties on future product revenues.”
What is important isn't the idea of drug companies partnering with universities and other researching institutes in order to fight off diseases isn’t new. What is is the fact that the disease in question is sickle cell. Yes this is ultimately done in the name of profit but at least it's a start.
The original article can be found here:
Dilaforette's site can be found here:
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By Sean Marshall
This week the highlight would be a report from Grand View research outlining. The article produces by News Wire explained how the world Hemoglobin market will only raise in value. If the predictions are correct revenue due to research and cures towards blood born diseases will be expected to reach a U.S. dollar of “8.82 million.”
The article explains that this is all due to a realization that blood born illnesses are increasing in recent years and there are still no real solutions. The article goes so far as to explain that the “rising prevalence of hemoglobin disorders such as sickle cell anemia, thalassemia, Hb C, and Hb E is estimated to be the high impact rendering drivers of the market.” It was also explained that “according to the WHO, hemoglobin disorders are endemic in over 60% of 229 countries affecting over 70% of births. It is also reported that minimum 5% of the world population are carriers of significant variation.”
The article also tackle issues that can cause complication to those with sickle cell disease as well as where sickle cell disease and other blood diseases. These factors included low level hygiene, low awareness levels and the inability to get to proper medical facilities.
Three important facts that the article highlighted about sickle cell that should be passed on were:
“The market for Sickle Cell Disease (SCD) was the largest with revenue valued at over USD 2,290.0 million in 2014. Rising prevalence of hemoglobinopathies and presence of strong product pipeline for sickle cell anemia such as HQK-1001 and Luspatercept are growth promoting factors of this market.”
“Genetic testing for diagnosis of sickle cell disorders is expected to witness lucrative growth over the forecast period. It is estimated to grow at a CAGR of around 7.0%. Increasing awareness levels among people coupled with government programs such as the National Sickle Cell Disease Control Program conducted by the WHO in Africa are anticipated to enhance the usage rates for diagnostic tests pertaining to SCD”
“Hydroxyurea was one of the dominating segments of the SCD therapy market owing to the presence of the FDA-approved drugs such as Droxia and Hydrea by Bristol Myers Squib. SCD. Thalassemia market holds an extensive product portfolio in pipeline and their expected commercialization is anticipated to drive market growth. For instance, The Ellis Lab is developing a gene therapy for SCD in collaboration with Dr.Victor Garcia.”
The original article is fantastic and should be read in detail. What I have presented is only a small portion of what I personally feel is important. I would highly recommend anyone in the sickle cell community or any blood born illness community to read the Grand view researching report or in the very least a news outlet reporting on it.
PR News wire’s article on Grandview's research can be found here: http://www.prnewswire.com/news-releases/hemoglobinopathies-market-worth-88-billion-by-2022-grand-view-research-inc-569930631.html
Unfortunately due to illness the sickle news will be postponed until next week.
By Sean Marshall
The news this week was short but included some positive outcomes. It was reported by the Birmingham Business Journal that Alabama University fulfilled their pledge in 2010 to donate $1 million. It may have taken six years but as of last December the goal was met.
What was reported is how the funds have been used. The first detail was that the funds will be providing “are for those patients and will also allow the clinic to hire a full-time social worker to the staff.” It was also mentioned that additional staff such as psychosocial services will be offered for both children and adults suffering from sickle cell disease.
Lastly a startling statistic was provided explaining that, “nearly 3,000 in Alabama struggle to manage sickle cell disease.” It does shed some light on to why the state university was so heavily invested in helping the clinic obtain positive results in the community.
The Original article can be found here: http://www.bizjournals.com/birmingham/news/2016/02/01/uab-receives-1m-in-funding-from-sickle-cell.html
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No sickle cell news updates this week. We apologize for any misconceive and will bring more news as it comes.
If there are any concerns email us at firstname.lastname@example.org
By Sean Marshall
It was a slow news week for sickle cell this week. The only real announcement was that the annual Sickle Cell Telethon in Texas "may have a difficult time this year serving families in the area because a major fundraiser has been postponed."
It was explained in the past that The Central Texas Sickle Cell Anemia Association has risen over $17,000 in funds in 2015 but due to a funding gap they had to postpone to a later date.
If you're interested in the article or how to contact The Central Texas Sickle Cell Anemia Association their details as well as the original article can be found here:
We will continue to follow this story as it unfolds.
If there are any comments, ideas for the future, questions or concerns email us at email@example.com
By Sean Marshall
It was reported this week by Information Nigeria that famous Nollywood actress Mercy Aigbe,37, has just been endorsed as an ambassador of the Dubai Africa Sickle Cell Project. For those not in the loop Nollywood refers to the Nigerian Hollywood film industry and Mercy Aigbe is considered a big deal in her industry.
This was all because the passing of Aigbe's late sister Joy. Her death involved complications due to sickle cell anemia. This was the driving factor that allowed Aigbe to take up the fight against sickle cell.
Although it doesn’t sound like the most prevalent news to most outside of Nigeria the Dubai Africa Sickle Cell Project is hoping for attention as Aigbe is the first ambassador the organization has ever had. If they can receive the spotlight for even a moment to promote sickle cell awareness it will be worth it. Especially if the news even gains some much needed funding as a result.
We have seen this before in the NFL with the McCourty or with supermodel Jourdan Dunn. The concept isn't new but it is always welcome. It reminds many that sickle cell disease is still out there and that it is still dangerous and still has a strong impact on the world.
The Sickle News will continue to monitor news channels to see how Aigbe's presence affects sickle cell awareness in the future.
The original article can be found here:
If there are any comments, questions, concerns or ideas for future articles send them to firstname.lastname@example.org
By Sean Marshall
This week it came to light that by the PBR Regulatory Affairs review board that the “clinic-stage bio-pharmaceutical firm Global Blood Therapeutics (GBT) has secured orphan drug designation from the US Food and Drug Administration (FDA) for GBT440 to treat sickle cell disease.”
That was a lot of information to take in but the basic concept behind it is that a new sickle cell fighting drug is in the clinical stages, making it one step closer to everyday people.
The drug titled GBT440 is meant to be an “oral, once-daily SCD therapy that works by increasing hemoglobin’s affinity for oxygen.” The drug itself would undue one of the biggest risks of sickle cell disease. It could in fact reduce the levels risks of stoke for anyone taking it.
It's also worth noting that Global Blood Therapeutics announced some of their testing plans. “the company is investigating the drug candidate in both healthy subjects and SCD patients in phase ½ clinical trial.” It was also explained that “the efficiency of GBT440 is being evaluated on polymerization of deoxy-hemoglobin, the mechanism of red blood cells sickling.”
All access to the results are to be available late 2016.
The PBR Regulatory Affairs report can be found here: http://regulatoryaffairs.pharmaceutical-business-review.com/news/fda-grants-orphan-drug-designation-to-gbt440-to-treat-sickle-cell-disease-040116-4766214
Other news this week included a Ministry of health announcement from Bahrain's health department that there is no shortage of morphine due to the treatment of sickle cell patients. The country of Bahrain is a small island n the Middle East. Like most of the world they to struggle with sickle cell disease.
Unfortunately there have been rumors of a shortage of morphine. This in turn lead many with sickle cell disease to go through the pain their affliction causes without any form of relief their government could provide.
Luckily their ministry has stressed that this was just a rumor and that “it is closely monitoring all medication needs of the sickle cell patients in accordance with the new treatment protocol taken in light of the recommendations of a group of experts from the US-based Johns Hopkins Hospital.”
he press release can be found here: http://www.bna.bh/portal/en/news/704563
Like always if anyone wants to comment ask questions or bring up new ideas The Sickle Cell News can be reached at email@example.com
By Sean Marshall
Welcome back to the Sickle Cell news. After a short break due to the holiday season we are proud to be updating and is better then ever. According to the Mercury News the Stanford School of Medicine is utilizing pipettes DNA for gene editing of stem cells. This is all in an attempt to eradicate harmful diseases like sickle cell anemia.
As of right now it has been reported that “tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering.” This means that the breakthrough that is gene editing is getting closer and closer.
This is all capable because of new technology called “CRISPR-Cas9, acts as a microscopic scalpel, performing genomic surgery with a precision, efficiency and affordability once thought unimaginable.” The technology know as CRISPR-Cas9 actually “acts as a microscopic scalpel, performing genomic surgery with a precision, efficiency and affordability once thought unimaginable.”
This technology has been mentioned before on sickle news but this is one of the first instances that the technology has been used to actually cure or in the very least make progress towards curing. That isn't to say that CRISPR-Cas9 has not been used to it's full potential but most news coverage is on the device itself and what it could do rather then what it actually is doing.
It was also explained that “the research being done at the Stanford School of Medicine, led by Dr. Matthew Porteus, is part of an accelerating research movement made possible using the new technique to try to cure genetic diseases such as sickle cell anemia and muscular dystrophy.”
It's also worth mentioning that this wouldn't have been possible without companies raising large sums of money to keep the labs afloat. This was so prevalent that the article actually mentioned that the university takes outside donations in order to keep the very expensive equipment running.
This could mean however that if sickle cell anemia is cured it could be month or even years before it makes it's way to the general public and that said cure would be pricey and most likely involve a length stay in a hospital. This is of course speculation and only time will tell what truly happens all we can do is hope a cure does come fast.
The original article can be found here: http://www.mercurynews.com/science/ci_29333667/stanford-bringing-gene-editing-patients-deadly-diseases
Like always if you have any comments, questions, concerns or idea topics email us at firstname.lastname@example.org
By Sean Marshall
It was announced this week in an article published on Medical Press' website that “an established drug for recurrent multiple myeloma might effectively be re purposed to improve the survival and day-to-day lives of patients with devastating sickle cell disease.”This was all according to “revealing new research by a Feinstein Institute for Medical Research scientist.”
For those who don't know myeloma is also known as plasma cell myeloma,myelomatosis, or Kahler's disease is a cancer of plasma cells, a type of white blood cell normally responsible for producing antibodies
According to the article the study was by Lionel Blanc, PhD, assistant investigator at the Feinstein Institute. AS well as the fact that the report will be publicly available on the 17th of December. Meaning we can all see the details first hand. The data can be found in the journal Blood.
Other important information the article gave was that “Dr. Blanc's research, was the first to identify how the drug pomalidomide increases production of fetal hemoglobin, known to interfere with the so-called "sickling" of red blood cells implicated in sickle cell disease.”
Other important details included Illuminating “pomalidomide's mechanism of action offers proof of concept that the US Food and Drug Administration (FDA)-approved medication could potentially be used to treat SCD, representing better outcomes compared to current drug treatment for the 100,000 Americans with SCD, an inherited disorder causing poor oxygen delivery, organ damage and even death.”
This is all good news and we hope the Lionel Blanc study continues. The original article can be found here: http://medicalxpress.com/news/2015-12-multiple-myeloma-drug-revolutionize-treatment.html
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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