First human trials of CRISPR outside China have been declared successful

A pair of biotech companies have successfully eliminated symptoms from two patients with different blood diseases

Faisal Khan
Dec 5, 2019 · 3 min read

The growing list of successful treatments by CRISPR technology to treat various diseases has infused hope in revolutionary medical technology to be the next big thing. Preliminary data from the first successful human trial points to the new era human gene editing is upon us. CRISPR Therapeutics and Vertex Pharmaceuticals the results recently.

This is being considered a significant development since this is the first successful human trial outside China. Back in November 2018, He Jiankui, a Chinese biophysicist, rocked the Medical World with his announcing that he had genetically modified the DNA of the twin baby girls before bringing them into the World by using the .

Chinese Scientists were back in the news again with the claim of using the CRISPR tool for trying to treat an HIV & Cancer infected male. And the results were promising. The patient was apparently cured of Cancer but the HIV virus remained. Both these reports were seen with skepticism though, as is the case with most news coming out of the government censored media in the country.

“We are very encouraged by these preliminary data, the first such data to be reported for patients with beta-thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001”

~ Samarth Kulkarni, CEO, CRISPR Therapeutics

The recent study should not only put to rest the doubts about Chinese claims but the innovative new medical tool itself. The two American biotechs began the global trial into their treatment called CTX001 in February of this year. The treatment was developed to treat two types of inherited blood disorders — beta-thalassemia and sickle cell disease.

Both the above-mentioned diseases are caused by a mutation in a single gene which resulted in a severe deficiency of hemoglobin — the oxygen carrier in the blood. The treatment involved gene-editing the patient’s stem cells to turn on a second copy of the hemoglobin gene, which is in most cases not active in adults. After the editing process was completed, the stem cells were returned to patients’ bodies.

The results from the treatment have been nothing less than miraculous. The beta-thalassemia patient, a European native who needed 16 blood transfusions a year but hasn’t needed even one since the treatment and the sickle-cell patient doesn't suffer from the pain attacks associated with the condition anymore.

Both patients did some suffer some side effects from the treatment, but they were primarily related to the removal of the existing bone marrow to be replaced with CRISPR-edited stem cells. A two year follow up is planned to evaluate the safety and efficacy of the treatment. Phase 1 & 2 trails will precede the larger Phase 3 trial needed for final market approval. At best, the final implementation is a few years away.

In the meantime, researchers would be keeping a keen eye on the longevity of the safety profiles for the new treatment. Nevertheless, CRISPR gene-editing techniques are showing they have great potential to treat genetic disorders in humans.

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Faisal Khan

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