Health & Wellbeing

America’s first CRISPR trial is still nearly 100% effective 3 years on

America’s first CRISPR trial i...
The new data reports nearly 100 percent success in 75 patients treated with efficacy persisting up to three years beyond the initial treatment
The new data reports nearly 100 percent success in 75 patients treated with efficacy persisting up to three years beyond the initial treatment
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The new data reports nearly 100 percent success in 75 patients treated with efficacy persisting up to three years beyond the initial treatment
The new data reports nearly 100 percent success in 75 patients treated with efficacy persisting up to three years beyond the initial treatment

Incredible new data presented recently at the European Hematology Association Congress has revealed an experimental CRISPR gene editing therapy is both safe and effective up to three years after treatment. The follow-up results come from one of the longest-running human trials using CRISPR technology to treat a pair of rare genetic blood diseases.

The first human trial in the United States to test CRISPR gene editing technology started back in 2019. The trial focused on two rare blood diseases: beta-thalassemia and sickle cell disease.

The treatment involves first gathering stem cells from a patient’s blood. Using CRISPR technology a single genetic change is made, designed to raise levels of fetal hemoglobin in red blood cells. The stem cells are then re-administered into the patients.

Initial results were extraordinarily promising. The first two patients treated were essentially cured within months, but questions over long-term efficacy remained.

A follow-up announcement last year continued the impressive results with 22 patients treated and all demonstrating 100 percent success. Importantly, seven of those patients were 12 months past the initial treatment with no waning of efficacy.

Now, a new data release is offering results from 75 patients treated with the groundbreaking CRISPR therapy, now dubbed exa-cel (exagamglogene autotemcel). Of those 75 patients treated, 44 were suffering transfusion-dependent beta thalassemia (TDT) and 31 had severe sickle cell disease (SCD).

All but two of the 44 patients with TDT were essentially cured of their disease, needing no more blood transfusions. The two TDT patients still requiring blood transfusions had 75 percent and 89 percent reductions in transfusion volumes respectively. All 31 SCD patients were also free of disease symptoms at long-term follow-up.

The data, yet to be published in a peer-reviewed journal, also reports no waning of treatment efficacy with the longest follow-up period stretching to 37 months beyond the initial therapy.

“These robust data from 75 patients, of which 33 have one year or more of follow-up after exa-cel infusion, further demonstrate the potential of this investigational therapy as a one-time functional cure for patients with transfusion-dependent beta thalassemia or severe sickle cell disease,” said Chief Medical Officer Carmen Bozic, from Vertex, the biotech company developing the treatment.

These Phase 3 trials are fully enrolled, and are the most advanced CRISPR trials in the United States. The researchers are looking to evaluate each patient with a follow-up period of 24 months.

The U.S. Food and Drug Administration (FDA) has given exa-cel a Fast Track designation. Vertex is hoping to submit exa-cel to the FDA for market approval by the end of the year. If it is authorized in early 2023, it will be the first CRISPR-based gene editing treatment to be approved for public use in the United States.

Source: CRISPR Therapeutics, Children’s Hospital of Philadelphia

Great news with hope for all. Any words on severe aplastic anemia?
Rocky Stefano
And this treatment is going to cost???????
Curing an inherited disease. Damn. Diseases that couldn't even be explained a few generations ago are on the way to the dustbin.
This research - along with the mRNA mouse studies (and with previous CRISPR & mRNA proofs) is an advancement in medical application skills. I have been in medicine for more than 40 years and have seen breakthroughs develop into standard of care. I have seen cutting edge expensive techniques become less expensive, easily accomplished therapies. While using CRISPR to transform hematopoietic stem and progenitor cells to produce fetal hemoglobin, effectively mitigating the disease, expect to see genetic induced aplastic anemia tackled in the next decade or so. Aplastic anemia has different causes and therefore different therapies, there is ALWAYS ongoing research on current therapies for our afflictions!
Rocky - if you have to ask???? then you likely can't afford it this year. Do you mean how many millions of dollars did it take to get to this stage or just how much was required for the medical hours and procedures on each patient? And is that really relevant for anything that is not standard of care?
Thanks Rich!
Rusty Harris
Hope it works! One thing I'd be concerned about is genetically altered DNA, through 2nd, 3rd generations.
There wouldn't be any way to tell if it has any effects multigenerations down the line.
@Rusty Harris
Wouldn’t a DNA test on the patient’s descendants would reveal all?
Rusty Harris & Robt - The research is dealing with altering the operating DNA expressions in the organism - remove the wanted cells, utilize CRISPR to re-engage fetal hemoglobin transcription (alter the genotype yes, but not alter the gene line), and reinfuse the cell line to the organism's (human's) bloodstream to impact their disease state, essentially curing them for the moment.

Ongoing monitoring will yield percentage cure of these SCD &TDT patients. Nothing has been done to alter their offspring, or alter their "Germ-line" if you will. This will be shown by any fertility testing, not DNA testing of the recipients. What and how their gametes develop depends on many more factors but they likely will pass on the conditions that led to their own diseases unless their germ-line is intentionally altered by as yet undeveloped techniques.