Chapter 6
It’s in Your Genes? — 3
Our immune white blood cells designated as T, which derive their name from our thymus gland’s role in training them, are fundamental for fighting infections, differentiating between our own proteins and foreign proteins, and protecting us from diseases, such as cancer. T cell engineering, with or without genome editing, is emerging as a treatment for “liquid” tumors, blood diseases like leukemia, and is in early testing for hard-to-treat solid tumors, like pancreatic cancer. While it was previously considered far too expensive and impractical, the “off the shelf” potential has altered that perception.
The reversal of scarring (fibrosis) that occurs in many organs, previously considered as irrevocable, is being actively pursued in clinical trials with engineered T cells, known as chimeric antigen receptor (CAR-T) treatment, and new drugs, one of which was discovered using generative AI. CAR-T directed against fibrosis was used in mice to restore heart function. It was also used to achieve long-term remission of asthma and in the experimental model of multiple sclerosis to deplete a specific type of T cell population that attacks the body’s cells. This groundbreaking work is not just in animal models: a single shot of engineered T cells has achieved remissions, without the need for immunosuppression therapy, in patients with lupus and other autoimmune conditions.
In the short term, we’re going to get much smarter about individualized, optimal diets, with a $189 million investment by the National Institutes of Health (NIH), building on prior knowledge that each of us responds to food quite differently. Here the use of AI to help develop algorithms for what I call Diet 2.0 is likely to represent a challenge to the old food pyramids and one-size-fits-all food recommendations—as if all human beings are the same.
With the astounding effectiveness of human papilloma virus vaccines to prevent cervical cancer, and COVID vaccines for preventing hospitalizations and deaths during the pandemic, there is keen interest to build on these successes. When it comes to empowering the immune response against cancer, there have been multiple reports that a vaccine directed to a person’s cancer cell proteins, known as neoantigens, can increase successful treatment beyond the existing drugs known as checkpoint inhibitors. Ultimately, there will be cancer vaccines produced to prevent specific cancers in people at high risk or show the earliest molecular signs of onset (such as via a liquid biopsy), but these will require a jump from current efforts that are directed at augmenting the immune response in people who have already developed cancer.