SICKLE CELL DISEASE HAS A TREATMENT NOW!

Sickle Cell Disease is said to be prevalent among about 2 crore Indians, majority of them from Maharashtra. Now, United States of America has approved the first treatment. The US based senior expert Dr. Ravi Godse speaks to http://indiainput.com on the great news, it’s implications on India and the hope that the nation offers. Can India find a cost effective option as regards the treatment? Dr Godse says, it can happen. Read on.

India Input Team

Health check up camps are a frequent feature at government run residential schools in Maharashtra’s tribal district Gadchiroli. The prime objectives include to search and identify students with Sickle Cell anemia condition, so that their health can be monitored regularly.

Sickle Cell Disease (SCD) is a genetic condition, widespread in parts of India where crores of people from tribal and rural population carry Sickle Cell traits. In Maharashtra, several of its eastern districts in Vidarbha have people with Sickle Cell anemia condition that is likely to get worse with age, in many cases. People with sickle cell anemia are said to be at increased risk for stroke and lung, kidney, spleen and liver damage.

On December 8, 2023 the U.S. Food and Drug Administration approved the first CRISPR treatment for sickle cell disease. The treatment, Called as ‘exa-cel’ the treatment is made by the companies Vertex and CRISPR Therapeutics. It features editing a gene involved in red blood cell shape and function.

“Great Weapon, but..”

US based renowned expert Dr. Ravi Godse welcomes the breakthrough but cautions as well.
He says, “This is a technology to treat the disease and is not limited to sickle cell disease alone. It can be used for many genetic diseases like cancer have underlined genetic problems. So, this is a big weapon, we have. We can line up the big diseases and shoot at them using this weapon. But, it is very easy, to shoot ourselves in the foot or in the head with this weapon, too. The side effects are pretty serious. So, we have to watch it very carefully. I thought this will like be a big breaking news all over the world, because sickle cell disease is not all that common in United States. We have about a hundred thousand patients, mostly African Americans. It typically doesn’t affect the white people. The sickle cell capitals of the world, the ninety per cent of the world’s sickle cell population lives in Nigeria, Democratic Republic of Congo and India.”

He further adds, “There are a lot of Sickle Cell traits in some Indian tribes. The Indian Sickle Cell is typically not that severe but it is a significant problem among about two crore people. If a Sickle cell trait person marries another Sickle Cell trait person then the statistical chance that each kid that they have will have a twenty five per cent chance of having the disease, fifty per cent chance of having the trait and twenty five per cent chance of being normal. So seventy five per cent that they will be okay. There is a twenty five per cent chance that they could get in to trouble. When I was eighteen years old, I had recommended that before getting married, we start looking at this Hemoglobin electrophoresis, to look at genetic compatibility. So what exactly is CRISPR? It is clustered, regularly interspaced, short palindromic repeats. what it means is like this the human genome has the DNA , the double standard structure, the genes are on that.. so, there could be one gene that is bad, that is over expressed or under expressed and that is making bad proteins or not making proteins. So, we go by the editing tool and just modify that portion very specifically. So what are the editing tools that we can use? Remember the mRNA vaccine that was much vilified in the social media that we used in the Covid Pandemic? The posterity is going to look at the Covid Pandemic as if it was a boon because although there was a very sad loss of life and all that, but it stimulated the scientific research and it lent it a sense of urgency that it had lacked so far. So, we can even use that mRNA for the genetic edits. The problem is, when you make those edits, you want to make sure you don’t make any offline changes and you have to monitor the patient for a few years. Just to give a funny example, suppose a kid doesn’t like his father because the father keeps telling him to study this and that. And the kid says, “I am going to go back in to the genome of my grand parents and I am going to edit out my dad.” But, the side effect of that, if the dad is edited out, where will the kid come from? So, we have to think long term. What might be the long term effects of that. But, this is a very good treatment. But now like, there is a lot of research.”

“India can make the difference..”

Then he asks, “But, where is the money coming from? How much does it cost? Well, it can cost up to eighteen crore Indian rupees, per treatment. You will say, where are we going to find the money? We are going to find the money in India, not by finding the money, but by not finding the money. I think India is a uniquely capable country. English speaking, democratic and a very smart people. And the people who can think out of the box to reduce the cost of anything. Look at Chandrayan-3. What scientific feat, what triumph that they did at a fraction of the cost! If India takes this technology and downsizes it, keeps it safe, makes it work but they reduce the cost so it can be made available to all. That would be India’s gift to the world. Today’s India is heading in that direction, I am pretty sure.”

During the Covid Pandemic Dr Ravi Godse’s warnings, predictions, fear alleviating analysis and stress relieving lines in lighter vein, were the most sought after as fear mongering ruled the roost.

India Input dot com only hopes, his words prove to be prophetic this time, too.