L-Glutamine: A new FDA approved medication for sickle cell disease

2017 was a banner year for sickle cell disease drug therapy!
 For almost 20 years, the only FDA approved medication for sickle cell anemia was Hydroxyurea. Hydroxyurea was approved for adults in 1998.  There is much evidence for its effectiveness and safety in infants as young as 9 months, and it has been safely used in children as well as adults since 1998. In 2017, the FDA officially approved Hydroxyurea therapy for children with sickle cell anemia--types HBSS and HBS beta zero thalassemia--starting at age 2 years.
In 2017 the FDA also approved L-glutamine (Endari), therapy for sickle cell disease.
What is L-glutamine?
L-glutamine was approved by the FDA for sickle cell disease in July 2017. As of March 2018, it is on the market as brand name Endari. It is approved for children over age 5 years and adults with sickle cell disease.
L-glutamine is a natural amino acid that our bodies make or that we receive when we eat meat or plant proteins such as red cabbage, beans and raw spinach. This product has been studied in critically ill patients, patients with allergies and patients with gut problems in the past. You may have even seen L glutamine on the shelves with supplements used by body builders.
The Emmaus Company has studied this treatment since the late 90s in sickle cell patients. The L-glutamine is manufactured from the sugar cane plant, so is actually vegan. It is purified to pharmaceutical grade as brand name Endari. This makes it different from the L-glutamine capsules that are on the shelves of the supplement stores as these cannot be  guaranteed for purity.
How do you take L-glutamine?
Endari comes in a powder form. It has to be mixed well in 8oz of a cold drink . It can also be mixed into yogurt and applesauce. Hot drinks cannot be used as they make L-glutamine inactive.
It is taken twice a day and the number of packets depends on your weight:
A child less than 66 pounds needs 1 packet twice a day.
A person 66 to 123 pounds needs 2 packets twice a day.
A person over 143 pounds needs 3 packets twice a day.
How does it affect sickle cell patients?
A study carried out in 31 centers in the United States led to FDA approval. Patients on the study could continue their Hydroxyurea. Patients had fewer pain crises in a year (an average of 3 a year for those on the study, and 4 a year for those not on the study).  They had fewer acute chest syndrome complications. The study also showed that patients had fewer hospital visits than those not taking Endari, and that their hospital stays lasted a shorter time. Patients also experienced longer times between their pain crises.
What else should I know about L-glutamine?
The most common side effects reported with its use were nausea, constipation and cough. Don’t use Endari if you have trouble with liver or kidney function. It can be used with Hydroxyurea if you are already taking this or plan to start this.
How does it work?
The way L-glutamine works is not yet fully understood. Red blood cells usually live for 120 days but sickle red blood cells are prone to dying quickly, causing anemia. This red blood cell death is called hemolysis. One of the main reasons for hemolysis is damage to the red blood cell membrane. This damage is caused by natural byproducts in our body called reactive oxygen species. Our red cells are protected against this damage by the NAD (nicotinamide adenine dinucleotide) system. L-glutamine helps us build up our NAD and allow better red cell protection. L-glutamine may also make red blood cells less sticky, which could reduce the number of painful crises.  
How can I learn more about L-glutamine?
Speak with your Hematologist about L-glutamine (Endari). Your sickle cell care team will help you decide if you need one or both of the two preventive medications for sickle cell care (Hydroxyurea and Endari). Your Hematologist also knows if your liver and kidney function are healthy enough for you to tolerate Endari. Your insurance will review your records and approve the Endari on a case by case basis with the help of your Hematologist.
Promising medications in the pipeline
This recent drug approval after a gap of almost 20 years is very promising for our sickle cell community. We have many medications that are in development by several companies. The only way these can be approved for use is with the help of sickle cell patients who participate in clinical trials.
We look forward to the day when we have many different medications options out there to help all our child and adult warriors with sickle cell disease live life to the fullest!

Angeli Rampersad
Pediatric Hematologist