Revolutionary stride in Medicine: Doctor and team invented an effective and potentially cheaper treatment for autoimmune diseases

Dr. Huji Xu. Credit: Qilai Shen/ Nature

As the year comes to a close, it’s the perfect time to spotlight groundbreaking scientific achievements. Recently, Nature released an article on 10 scientists that they felt had made immense achievements in the scientific community, called "Nature's 10". While reading that, I came across Dr. Huji Xu's profile, which caught my eye, as he had made a groundbreaking achievement in medicine - that is he had invented a world-first treatment for autoimmune diseases. Let's dive deep and learn of his achievement and the benefits it renders to humanity, in detail.

What is autoimmune disease?

Autoimmune disease is a condition that results in the loss of the immune system's tolerance to self-antigens, which are cellular markers that immune cells rely on to recognise cells from the host body. Due to this condition, organ or system damage occurs. 

T-cells and B-cells are immune cells that are involved in autoimmune responses, which usually occur when the immune system identifies foreign pathogens invading the body system. However, autoimmune diseases are often engendered by the malfunctioning of T-cells and B-cells.

B cells are responsible for the production of antibodies that attack foreign invaders. When B cells malfunction, they produce autoantibodies which basically kill the body's own cells, thereby rendering organ and systemic damage. 

What did Dr. Xu and his team achieve?

Dr. Xu's treatment involves delivering engineered immune cells to autoimmune diseased patients. The cells are also known as chimeric antigen receptor (CAR) T-cells, which have been instructed to eliminate malfunctioning B cells. By eliminating these B-cells, the disease can be alleviated.   

Dr. Xu and his team follow a set of steps in engineering the therapeutic T-cells. The T-cells are obtained from an independent donor, which is unusual as previous autoimmune disease treatments commonly utilize cells from the patients. Thereafter, the CRISPR-Cas9 gene-editing tool is used to inactivate 5 genes from the T-cells, to prevent the patient's body from rejecting the engineered T-cells. 

Wish to learn more about CRISPR and CRISPR gene editing? Check out part 1 of my CRISPR series to learn more about CRISPR. Part 2 will discuss CRISPR gene editing in detail and how it benefits humanity.

How is Dr Xu's treatment pathbreaking?

CAR T-cell therapy is frequently depended upon to treat blood cancers involving malignant B-cells. However, they have not been widely used to treat autoimmune diseases. Although Germany had previously used this therapy to treat several autoimmune conditions, Dr. Xu's method differs from the treatment used in Germany because it uses independent donor cells rather than from patients. This donor strategy could allow for the mass production of CAR T-cell treatments, reducing cost and thus making it available to the masses. 

As a novel treatment that had never been tested on autoimmune patients, the path forward was fraught with significant risks. When Dr.Xu's team approached several doctors to try out this treatment for autoimmune disease, they backed out. Nevertheless, Dr. Xu remained steadfast and took the risk to trial this novel treatment for autoimmune disease. 

For the first three patients that were selected for this therapy, the results were beyond astounding. The first patient, who had an agonising disorder characterised by immense muscle weakness, reported to the nurses that she had regained adequate strength to lift her arms, two weeks after receiving treatment. The next 2 patients, with a different condition, said that their symptoms started vanishing within a matter of days. all three patients were in remission, more than 6 months after receiving the treatment. 

Currently, Dr. Xu's team has delivered this treatment to another 24 patients. It is without a doubt that this groundbreaking invention of his has made a remarkable impact in medicine, through developing an effective treatment for a debilitating disease that could be made more available to the masses.

What other areas of medicine do you think could benefit from CAR T-cell therapy? And what other medical topics would you like to hear for me? Write your opinions in the comments below. Thanks a lot for reading!

References

1. Hampe, C. S. (2012). B cell in autoimmune diseases. Scientifica. https://pmc.ncbi.nlm.nih.gov/articles/PMC3692299/
2. Arango, M.-T. (2013, July 18). Infection and autoimmune diseases. Autoimmunity: From Bench to Bedside [Internet]. https://www.ncbi.nlm.nih.gov/books/NBK459437/

3. Mallapaty, S. (2024, December 9). The daring doctor behind a world-first treatment for autoimmune disease. Nature News. https://www.nature.com/articles/d41586-024-03895-0