A Treatment for Sickle Cell Disease using Hemoglobin Edited Blood Cells (CASE) that Eliminates Debilitating Pain

The treatment aims to eliminate episodes of debilitating pain that are a hallmark of sickle cell disease. It involves editing patients cells outside the body to correct a genetic defect. People with a lot of pain attacks are eligible to receive it. The therapy is designed to be given just once, with benefits potentially lasting years or possibly decades.

“This is a terrible disease,” says Samarth Kulkarni, president and CEO of Crispr Therapeutics. It feels like a lot of work every day. Patients have this constant specter of mortality hanging over them.”

The edited cells produce a form of hemoglobin known as fetal hemoglobin, restoring normal function of red blood cells. The hope is that the therapy, brand name Casgevy, is designed to be a one-time treatment and will alleviate symptoms for a lifetime.

In data presented to the FDA, the treatment solved the severe pain crises for at least eighteen months in 29 of the subjects. The treatment has produced similar results for patients suffering from a related condition known as beta thalassemia.

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They are both very expensive. Vertex said the wholesale price for Casgevy will be $2.2 million. Bluebird set the wholesale price of Lyfgenia at $3.1 million.

Many hospitals cannot offer the complicated procedure that the treatments require. Many patients may find treatment hard to participate in.

Gene-editing, a method that allows scientists to manipulate the basic building blocks of life more easily than ever before, is being studied for treatments for many diseases such as cancer, heart disease, diabetes, AIDS and Alzheimer’s.

The abnormal hemoglobin is a cause of this problem. The problem arises from a mutation in the HBB gene. Everyone has two copies of the gene—one from each parent. Children born with sickle cell disease inherit a copy of the mutated gene from both parents.

Gray was forced to rush to the hospital multiple times for blood and pain drugs. She was unable to finish school or hold a job, so she would often have to look after her children.

Since the treatment, Gray has been able to work full time at Walmart and spend more time with her children who are now teenagers, and she has been much more energetic.

“Since I received the CRISPR treatment, I’ve had a new beginning. Most of all, I no longer have to fear dying and leaving my kids behind without a mother,” Gray says. I have a life that is unlimited now. I’m feeling full of energy. I don’t have any pain. It’s a real transformation.”

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The companies are working with both private and public insurers. Advocates note that the high price could easily be offset by the savings of avoiding a lifetime of sickle cell complications.

Many of the countries where most sickle cells patients live don’t have enough sophisticated medical centers to provide the complicated treatment. It isn’t easy to access in the US due to the treatment not being widely available.

If enough research had been done to detect the effects of the treatment on the genomes, they could potentially cause long-term health problems.

The companies are planning to follow the patients in the study for 15 years to see if the treatment helps them live longer and if there are any long-term side effects.

As well as treating a rare liver condition called amyloidosis and an inherited form of cholesterol called familial hypercholesterolemia, the use of CRISPR based treatments has shown promise.

“This is a major milestone,” says Vence Bonham, acting deputy director of the National Human Genome Research Institute at the National Institutes of Health. “This could truly change the lives of individuals and reduce the burden of pain episodes.”