Genetic Engineering and Biotechnology News recently published an article describing how engineered cells were implanted into rheumatoid mouse models. The cells used in the study were able to sense and respond to inflammation by distributing therapeutic doses of a drug, reducing inflammation, and preventing bone erosion.
About the Standard Treatment of RA
Although a number of biologic drugs have revolutionized treatment for autoimmune disorders, almost forty percent of RA patients show no response to treatment. RA affects approximately 1.3 million people in the United States.
One of the investigators for the study explained that for current RA drugs to be of benefit, the drugs must be administered at high doses for long periods of time. This, in turn, causes severe side effects.
Another drawback pertains to the biologic drugs’ suppression of the immune system exposing patients to higher risk of infection.
The CRISPR Technology for RA
CRISPR technology was used for reprogramming the stem cell genes. The scientists created cartilage implants (cells on scaffolds) which were placed subcutaneously in mice. This process allowed the cells to stay in the body over longer periods of time and secrete the drug intermittently as a response to inflammation flareups.
The Washington University researchers submitted their report to Science Advances. The original article is available here.
The Washington University study using mice models not only reduced inflammation but also reduced bone damage usually found in RA.
Dr. Yunrak Choi, a co-author, commented that bone erosion is not currently treated by standard biologics. He said that imaging techniques used to examine the study animals found that the new approach was effective against bone erosion. This solves a critical unmet need.
One Size Does Not Fit All
Moving CRISPR-Cas9 gene editing a step further, the researchers point out that it is possible to program the cells to produce many variations of the original drug. Cartilage cells may be engineered to meet the needs of more patients making the treatment specific to the individual and thus afford improved efficacy. The new strategy also offers hope to over 300,000 children in our nation with juvenile arthritis.
