The Stream - Living with sickle cell anemia

The Stream - Living with sickle cell anemia

Doctors hope a recent medical breakthrough could lead to a cure for sickle cell anemia, an inherited condition that can cause crippling pain and shortened lifespans for the estimated quarter million babies born with it each year.

Earlier this month, doctors announced the results of an experimental gene therapy that is offering hope to those who suffer from the disease. A French teenager with the genetic condition achieved complete clinical remission after researchers in Paris injected his stem cells with an anti-sickling gene from his bone marrow. So far it seems to be working for the teen. Fifteen months since his last treatment, the patient is no longer dependent on medication, and his blood cells show no sign of the disease. Clinical studies have begun in other parts of the world to address the disease.

The disorder occurs when a child’s mother and father each pass on copies of a defective gene that helps make hemoglobin, a protein that carries oxygen in red blood cells. The defect causes red blood cells to collapse into a crescent shape, clogging blood vessels and preventing oxygen from flowing through the body. This can trigger serious problems like stroke and organ failure.

For most of the 20th century, sickle cell was a relatively invisible disease because it was so often misdiagnosed. Despite advances, race, poverty and stigma remain challenges in treating the blood condition most prevalent in people of African descent. It also affects people of Indian, Middle Eastern, Hispanic and Mediterranean heritage. In the United States, where the majority of people with sickle cell disease identify as black, patients seeking pain relief have been cast as drug seekers, and researchers have suggested racial discrimination affects the care sickle cell patients receive.

[eldavedee]

This was a really good informative stream, it had taken me 35 years before I could openly say I have SC! I hope from more things like this, the stigma will become non existent and knowledge and acceptance will increase.

[califiasrevenge]

love the stream. and 'my friend jen' sounds like a wonderful gift for any child.

[ellejmgmt]

I spoke to my daughter's doctor last week who said this new gene therapy has been done 3 times here in Chicago, IL. But it has only been done on Thalassemia SC patients that are over 18. This therapy has not been done on regular SC patients- as told by this doctor.

[albertagyemang]

Very spirited discussions. I am writing a research paper on it and this really helps. Awesome panel!!!

[ellejmgmt]

I am tryng to get my SC nonprofit off the ground that will focus on helping families directly which no one discusses either. My families has been dispalced (lost housing so many times due to my baby girl having SC SS). There are no programs to help parents/caregivers keep jobs, get assistance when we are in need of services. And I know I am not the only one who has lost a job, marriage and support system to help with managing this disease for their child.