New Treatment For Sickle Cell Anemia

By Margaret Adams

Sickle Cell Disease (SCD) is a genetic blood disease that primarily affects people of African descent, but also affects people from tropical areas of South America and Asia.

The Center for Disease Control (CDC) estimates that:

• 100,000 people have SCD in the United States,
• 1 of 365 Black/African Americans, and 1 of 16,300 Latinos/Hispanic Americans are born with Sickle Cell Disease and
• 1 of 13 Black/African Americans are born with the Sickle Cell Trait.

Currently, there is no cure for this condition.

Red blood cells carry oxygen to the cells in the body.  Oxygen turns into energy, an essential function to keep the body healthy. 

Normal red blood cells are disk-shaped. When a person has SCD some of their red blood cells mutate and become shaped like sickles.  Sickle shaped cells can block blood vessels and burst, leading to serious infection and damaging the body’s organs.

They can also cause fatigue and swelling of the hands and feet due to blocking blood flow to the chest, bones, and joints, with pain that can vary in duration from hours to days.

Due to the severity of these symptoms some people may require hospitalization. Complications from SCD can be life-threatening.

According to the New England Journal of Medicine the average life expectancy for males is 42 years, and 48 years for females. While this is an improvement in the life expectancy for this group, which was 14.3 years in 1970,  it is still 20-30 years less than the average population.  Incidents of death can occur in children before the age of 3 because their bodies succumb to infection (usually pneumonia) which is why  medical professionals strongly encourage children to get  immunized.

In previous years when people were hospitalized during an SCD crisis, the treatment consisted of managing symptoms, pain management, oxygen support, and fluid hydration.  The need for pain medication has brought to the forefront how people with SCD have often been discriminated against, and stereotyped as exhibiting drug-seeking behaviors.  This bias towards people having a sickle cell crisis has led to people not receiving adequate treatment in a timely manner.

Since this disease is genetically transferred, it is important for people to know their status prior to becoming parents.  People should be aware if they are a carrier (meaning they carry the trait).

Two people who carry the trait increase the likelihood of passing the disease on to their offspring. Fortunately, it is standard practice to screen all newborns for this disease.

Today, however, things are looking up for people with SCD. Dr. Ted W. Love, a Black/African American, was motivated to discover a breakthrough treatment for this condition due to his own personal awareness of discriminatory practices against patients with this disease. In 2014 Dr. Love became aware of an early experimental drug to treat SCD called GBT440.  He is currently the CEO of Global Blood Therapeutics Inc.  The new drug Oxbryta (voxelotor) is used to prevent the hemoglobin in red blood cells from sticking together and forming into the sickle shape that can wreak havoc on the body. This drug and Adakveo were approved by the FDA in 2019.

Obryxta treatment

Dr. Love is an example of why we need more of our students to go into the fields of science so they will have a personal reason to discover ways to alleviate the health issues that plague our community.

For more information and support for people and families dealing with Sickle Cell Disease, please contact the Sickle Cell Disease Association of America.  There is a local chapter in Cleveland, Ohio, (216) 229-8600.

Dr. Ted Love is the president and CEO of Global Blood Therapeutics, which is changing the diagnosis of sickle cell disease from grim to long-term living. He believes research and medicine are the keys to improving the lives of those diagnosed with this disease.

Dr. Love came out of retirement because he was offered the opportunity to become CEO and head of a company that would invest in sickle cell disease. “We’re not going to stop until we’ve done for sickle cell disease what we’ve done for many, many disorders.”