CRISPR Tests Being Done for The First Time in U.S. Cancer Patients

The CRISPR technology started off on the wrong foot, publicity-wise, in 2018. In November 25 of that year, He Jiankui announced that he created the first CRISPR-edited babies (i.e., twin girls) and a media firestorm soon followed.  But in 2019, scientists want to introduce the cutting-edge technology to the public on a more favorable footing. 

For the first time, the cutting-edge CRISPR technology will be used in the treatment of cancer patients in the United States. The patients are participants in a study being conducted by the University of Pennsylvania in Philadelphia; they are on treatment for sarcoma and myeloma using the technology. 

The reintroduction of the CRISPR technique is notable because it comes just a few months after He Jiankui mishandled it. While it’s unclear whether he actually did what he claimed, his use of the technique for genetic editing on infants earned him the ire of his fellow scientists and of his government.

But looking beyond this fiasco, the technology actually has numerous practical – and ethical – applications, especially in comparison with traditional scientific techniques. Known also as CRISPR-Cas9, it is currently studied for its possible applications in the treatment of certain neurological disorders like Huntington’s disease.

Scientists are also looking into its applications in sickle cell anemia and inherited blindness. In an Editas Medicine-sponsored blindness study, scientists will attempt gene editing in the body. 

2019 seems to be a favorable year for the CRISPR technique particularly in terms of a more favorable public opinion. He’s CRISPR fiasco will likely be placed on the back burner, especially in light of the recognition of He’s numerous mistakes in handling the technology. While He worked with Rice and Stanford scientists, he didn’t work within the required institutional setting and he didn’t release his findings in an academic journal.

There are high hopes for the success of the cancer study conducted by the University of Pennsylvania and led by Dr. Edward Stadtmauer. Basically, the scientists will harvest immune system cells from each patient, attempt to genetically change them, and then reintroduce the altered cells into the patient’s body. The altered cells will target and kills the cancerous cells, thus, curing the patients of their cancer.

The study has 18 participants.

While there are no results yet, the study has apparently been cleared according to the standards of scientific ethics. The world is watching and it’s a promise that the scientists involved study are well aware of, especially after the bad rap CRISPR technology experienced in 2018. 

What is CRISPR? Considered as the most revolutionary technology in medicine and in science, CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. Essentially, it’s a technology that allows scientists gene editing in living cells.

Francisco JM Mojica, a Spanish microbiologist at the University of Alicante, pioneered the CRISPR technique. He first observed the repeats in two archaeal organisms in 1993.

In 2013, Dr. Feng Zhang of the Massachusetts Institute of Technology (MIT) made the first demonstration that CRISPR can be used in genome editing in human cells. The technology has grown by leaps and bounds with scientists finding newer, better applications for it. 

As previously mentioned, CRISPER has numerous possible applications being studied. These include curing genetic diseases, treating HIV, and treating cancers, even in removing the threat of malaria. Scientists are also looking into DNA editing in live human embryos and in bioengineering in agriculture. 

Many scientists, in fact, have been successful in finding viable applications.  In 2018, for example, Kite Pharmaceuticals and Juno Therapies created human T-cells altered using the CRISPR technique. These T-cells were then used in inducing remission in terminal leukemia.

Dr. Feng, who’s connected with Editas Medicine, is leading clinical trials in the use of CRISPR technology in the treatment of rare diseases. These include Duchenne muscular dystrophy, Usher syndrome 2a, and Leber congenital amaurosis. 

Researchers at Temple University also removed HIV-1 from T cells in cultures using the technique. Scientists at the University of Wisconsin are developing a CRISPR antibiotic that will selectively target super-resistant bacteria.

In China, scientists have corrected deadly mutations in human embryos. In the United Kingdom, researchers have eliminated malaria in new strains of mosquito with the CRISPR technique. 

Due to its wide range of applications in the medical and scientific fields, CRISPR technology has attracted investors and companies aiming to benefit from it. The legal battles have been fierce, such as in the case of Dr. Feng and Jennifer Doudna of UC Berkeley who fought in court over patent rights and royalties; the former won.  

The three largest publicly-traded CRISPR-centric companies include Crispr Therapeutics, Editas Medicine, and Intellia Therapeutics have benefited from the technology, obviously. There three companies tripled their stock prices and secured billion-dollar capitalization, and these don’t have any marketable treatments yet! The investments are obviously made in anticipation of future financial gains. 

The value of CRISPR, both in its scientific applications and monetary gains, continues to rise. In the process, we will be seeing more of it in the mainstream media.