The NFLPA names a Community MVP each week to recognize the work done by players off the field, but they broke with tradition for this week’s award.

It’s being shared by Patriots defensive backs Jason and Devin McCourty for the work they’ve done together to raise money for sickle cell research and awareness. The twin brothers held a fundraiser on October 22 that brought in more than $200,000 to benefit Boston Medical Center and the non-profit Next Step, which supports young people with life-threatening illnesses.

“Sickle cell disease is a sickness that has caused a lot of pain in our family, specifically for our Aunt Winifred. So to be able to bring awareness for others and raise money to help those impacted by this terrible disease has been humbling,” Jason McCourty said in a statement.

 

The phrase “know your status” is synonymous with one potentially life-threatening disease, but singer Jordin Sparks wants you to be educated about another that also disproportionately affects African-Americans —and she’s working with “Generation S” to lead the conversation.

“Generation S is a group of those who have sickle cell disease sharing their stories and bringing more awareness to what the disease is,” Sparks said at the 46th Annual National Sickle Cell Disease Association of America in Baltimore.

“Obviously you can’t tell by looking at someone if they have it. With this it gives them an opportunity to show their face and say hey look, “I have this, this is my experience and everybody’s experience is different.”

 

In a bid to improve quality of care for persons with sickle cell disorder, Nigeria Sickle Cell Foundation Nigeria, SCFN, and RHIEOS Ventures yesterday signed a Memorandum of Understanding, MoU, to establish the first multi-centre Sickle Cell Disorder, SCD, registry in Nigeria.

The registry will be used to support and improve quality of treatment and care for persons with SCD as well as for policy formulation around sickle cell decision-making and research. The registry will be used to facilitate the implementation of National Guidelines for the management of SCD.

SCD is a major genetic condition in Nigeria. The country has by far the largest burden of the disorder anywhere in the world. Over 40 million Nigerians are carriers of the sickle cell gene and an estimated 150,000 babies are born every year with sickle cell anaemia (Hb SS).

Warrior University is an online, curriculum-based educational platform, powered by the patients and caregivers of the Sickle Cell Consortium. All current classes, presentations and workshops are classified into five warrior university pillars:

• Disease Education (ex. The Expert Patient Series)
• Community-Based Organization/Certified Community Advocate Training & Best Practices
• Medical/Scientific Research & Updates
• Legislation & Policy
• Warrior/Caregiver Life Skills

Warrior University will be hosting a series of classes for ALL partners, patients, caregivers, and everyone interested in the research of sickle cell disease to gain more information about how we may be able to develop cures for those individuals who suffer significantly from this illness.

The upcoming classes will be held every Thursday at 7 p.m. EST.

Women’s soccer sophomore midfielder Alexis Williams was playing with her club high school team at a tournament in Colorado when she found she could not keep up her endurance on the field.

“It was the weirdest thing because I’ve never had problems running or anything, but I would have to stop every second,” she said. “I couldn’t run.”

Williams knew she was physically fit, but questions from her coaches asking if she was out of shape frustrated her – especially because being quick on the field was a hallmark of her play.

She forgot about the incident when she returned home to New Jersey, where she was able to return back to her normal ability on the field.

But the moment in Colorado would return to Williams’ mind when she went in for a sickle cell trait test before her freshman year of college – an NCAA requirement for athletes before their first season – and it came back positive.

 

SCD is a group of genetic disorders that cause red blood cells to become misshapen and break down. From an early age, patients with sickle cell disease have a high risk of vascular disease and even stroke. Current models to test drugs used to treat SCD are not reliable and cannot accurately predict how a person’s body will respond to the medication. It is also difficult to predict which patients with the disease have the highest risk of suffering a stroke versus those who have the lowest.

The team is designing an organ-on-a-chip, consisting of a device that will mimic an artery of a pediatric sickle cell disease patient, and the associated biological readouts will provide information on the progression of vaso-occlusion, including during drug treatment.

 

A case series of individuals with sickle cell disease who lived into their ninth decade revealed potential genetic and environmental factors associated with their longevity. The case report — published in Blood — included data from four women with sickle cell disease who have lived longer than age 80 years. Three women are American and received treatment at Thomas Jefferson University’s Sickle Cell Center. The fourth woman resided in Brazil. One patient remained alive and in her 80s at the time of reporting. The remaining three patients are deceased and had lived into their 80s. Although individuals with sickle cell disease are living longer than ever, most patients do not live beyond the age of 60 years, according to Samir K. Ballas, MD, FACP, emeritus professor of medicine and pediatrics at Thomas Jefferson University.

Nearly 20 years ago, scientists stunned the world when they announced they had decoded the genes that make up a human being. They hoped to use that genetic blueprint to advance something called gene therapy which locates and fixes the genes responsible for different diseases.

Now, a clinical trial at the National Institutes of Health is doing exactly that in an attempt to cure sickle cell anemia, a devastating genetic disease that kills hundreds of thousands of people around the world every year.

For the past 15 months we've been following the scientists, and patients, who are ushering in a genetic revolution.

In the laboratory, Dr. Tisdale and his collaborators created a gene with the correct spelling. Then, to get that gene into the patient, they used something with a frightening reputation: HIV, the virus that causes AIDS. It turns out HIV is especially good at transferring DNA into cells.

Here's how it works. The corrected gene, seen here in yellow, is inserted into the HIV virus. Then, bone marrow stem cells are taken from of a patient with sickle cell anemia. In the laboratory those cells are combined with the virus carrying that new DNA.

 

There's new hope for patients who have blood diseases. Johns Hopkins Kimmel Cancer Center researchers say a new protocol offers the possibility of a cure. One patient who tried the treatment says it didn't just change her life, it saved it.

For most of her life, Simone Day had a very different experience at the hospital than the one she's having now. For Day, sickle cell meant acute pain crises. She'd be in the hospital for weeks. "I was essentially OK with dying at that point," Day said. It's different now, thanks to a bone marrow transplant in 2017.

The new protocol doesn't only mean using what they call "half matched" donors, like Day's mother, but also lowering the dosages of chemotherapy to make it safer on the patient.

 

Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma giant is investing millions in developing potential treatments and gene therapies for rare disorders ranging from sickle cell disease and hemophilia to ALS and Duchenne muscular dystrophy.

Michael Wajnrajch, MD, is senior medical director for Pfizer’s Rare Disease Group and pediatric endocrinologist on the faculty of New York University. Wajnrajch outlined his company’s vision for gene therapies at the 2nd International Congress on Advanced Treatments in Rare Diseases, held March 4-5 in Vienna, Austria.

“Sometimes we’re doing the right thing for the right reasons, and it’s not about the money,” he told some 100 delegates. “Sickle cell disease predominantly affects patients with very limited ability to pay. We’re not going to make much money on this, but we know it’s an unmet medical need in many parts of the world.”