Believe me,2017 was the year of innovation in the medical field.From the use of microbiome to diagnose and treat disease to the discovery of the first drug to treat sickle cell disease complications, the year was filled with the new innovative concept.Among all innovation, the approval of endari made a big headline in various newspapers and News channel.
On june 20,2017 famous rapper Prodigy died with a complication of Sickle cell disease.Since then, the rare disease Sickel cell anemia gained its popularity in news headline. On july 7, 2017 FDA approved endari( L-glutathione oral powder) for the treatment of sickle cell disease.
Today I will be discussing sickle cell disease and its primitive treatment method.Based on research paper, I will be comparing the effectiveness of endari with another available treatment method of sickle cell disease
Understanding sickle cell disease(SCD)
It is a group of a rare form of genetic disorder of blood. Sickle cell anemia is the commonest variety of sickle cell disease.It is the abnormality of oxygen carrying protein haemoglobin-S.As the name suggests, RBC becomes sickle shaped.Blood becomes sticky and rigid.
source
According to CDC
i)SCD affects approximately 100,000 Americans.
ii)SCD occurs among about 1 out of every 365 Black or African-American births.
iii)SCD occurs among about 1 out of every 16,300 Hispanic-American births.
iv)About 1 in 13 Black or African-American babies is born with sickle cell trait (SCT).
114,800 deaths were reported in 2015.
The death count is very high.This is where the importance of Endari comes.If it can reduce death count by 5% that will make a huge difference considering the affordable price.
Am I suffering from SCD ?
Well, A blood test can be done to check for abnormal hemoglobin.But nobody will go for direct investigation without having few symptoms.Symptoms are milder to very severe which might take away your life.Since affected red blood cells are not capable of carrying enough oxygen patient will initially present with fatigueness, breathlessness, pale hand and sole. When the rigid and sticky RBC blocks blood vessel the symptoms of pain begins.Along with this, there is swelling of limbs , difficulty in vision and frequent infection.
Different sign and symptoms begin after 5 to 6 months of age.
What can I do now ?
Visit a doctor. Doctors are always looking for symptomatic relief.Reducing pain and infection can give symptomatic relief.This is what doctors are doing for a long period of time.Believe me, treating any genetic disorder is the greatest challenge a doctor can have.A doctor can always be sure that if the patient is discharged, he will certainly meet that patient very soon.Genetic disorder is like that-You can't fully satisfy your patient.
Life expectancy of people with SCD is about 40-60 years.This is quite high.But life threatening symptoms will not allow you to live free life.You will be spending most of your life in hospitals-still you don't know what exactly can happen next day!
Hydroxyurea is the first drug approved in the treatment of SCD.In the past,It was single hope for the patient with SCD.Now the time has changed. This graph sums up all the advances in the treatment of SCD.
Timely blood transfusion and bone marrow transplant are the only effective treatment.
On july 7, FDA approved new treatment method i.e Endari. And now the treatment method been changed.
What is Endari?
Endari ( L- Glutamine) is a newer drug for the treatment of SCD developed by Emmaus Life Sciences.It is the first treatment for the pediatrics group and first treatment for adult after almost 20 years.
How endari works?
There is an alteration in the globin protein ( which carries oxygen).Due to this alteration, RBC are susceptible to oxidative stress.
Increased oxidative stress is associated with the decreased flexibility of RBC, which later acquires sickle shaped.Less flexible than normal cell means higher viscosity. Due to this there is obstruction of the blood vessel.
Endari increases the amount of glutamine in the blood.These glutamine are taken up by sickle cell RBC .Glutamine helps in the formation of anti-oxidant.This generated anti-oxidant helps to neutralize the oxidative stress allowing them gaining the normal flexibility.
In SCD, all complications are due to increased rigidity of RBC and occlusion of Blood vessel.Since Endari makes RBC flexible, complications are dramatically reduced.
Endari vs Normal Treatment
Clinical trials conducted using endari for 48 weeks showed reduced in frequency and length of hospital visit. One of the major complication, Acute chest pain has reduced by 67%.
Endari Vs Hydroxyurea Vs Bone marrow transplant
With no doubt, A Bone marrow transplant is the best treatment.The cost of Bone marrow transplant is high as $800,000 in the U.S.A.So, most of the people suffering from SCD can't afford it.The cost of treatment of Endari comes to be around $600 for a single packet.The requirement of packets depends upon the severity of complication.Use of Hydroxyurea is associated with the risk of developing skin cancer and leukemia.
At last,
Better late than never, endari is a good choice of drug for the patient who can't afford treatment like bone marrow transplantation.With few side effects, a patient responds well to the treatment.The cost of treatment is quite high.
sources:-
1)https://www.cnbc.com/2017/08/07/new-sickle-cell-anemia-drug-endari-by-emmanus-is-fda-approved.html
2)https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4765496/table/T1/
3)https://en.wikipedia.org/wiki/Sickle-cell_disease#Bone_marrow_transplant
4)https://www.medigo.com/blog/medigo-guides/bone-marrow-transplant-cost-guide/
5)http://www.rxdrugs.xyz/2017/07/Endari-cost-side-effects-dosage-uses-for-sickle-cell-disease.html
6)http://www.emmausmedical.com/
7)https://www.ncbi.nlm.nih.gov/pubmed/20301551
Thanks for reading .
Any feedback and comments are highly welcome !