Is the Market for CAR T-Cell Therapy One to Watch?

Cancer continues to be one of the leading causes of death globally, and the path to finding the seemingly unreachable cure is a vast and arduous one. Currently, there are many forms of treatment (e.g., radiation, chemotherapy). There is one, however, which is of particular interest and considered the next pillar of cancer treatment: immunotherapy. This is where the patient’s immune system is harnessed to attack the tumors. 

The Science

One type of immunotherapy gaining significant traction is the use of chimeric antigen receptor (“CAR”) T-cell therapy. CAR T-cell therapy is a process whereby T cells (lymphocytes that are subtypes of white blood cells) are collected from a patient’s blood and are enhanced with synthetic antibodies to be infused back into the immune system. Once reintroduced into the immune system, the CAR T cell is designed to recognize and attack the tumor cells with a specific protein, or antigen. Clinical trials have provided great hope for this therapy, demonstrating a longer-lasting treatment. The first regulatory approval of CAR T-cell therapies was in 2017 when the FDA approved it for treatment of children with acute lymphoblastic leukemia (“ALL”) and adults with lymphomas, both cancers involving liquid tumors. It is still unknown whether the therapy can be effective in attacking solid tumors, such as breast cancer.

The Market

The promise of this research has created excitement within the biotech marketplace. Recent IPOs and financing rounds for the CAR-T industry have reached nearly $2 billion, and market capitalization has been valued at $20 billion, according to a 2018 report on CAR T-cell therapy companies by BioInformant. A separate BCC Research report estimates that the market is likely to experience a 44.1% compound annual growth rate through 2023. There has also been significant M&A activity, highlighted by a $9 billion acquisition of Juno Therapeutics by Celgene in 2018, along with a $11.9 billion acquisition of Kite Pharma by Gilead in 2017; 2019 is off to a fast start with Bristol-Myers Squid announcing plans to buy Celgene at $74 billion. There are now hundreds of companies researching and developing CAR T-cell therapies.

The Challenges

While this therapy seems to have a bright future, there are several key challenges to negotiate:

1. Efforts to create unique antigens to effectively target solid tumors with this therapy have been largely unsuccessful, because many of the tumor antigens are inside the tumor cells and cannot be reached by the CAR T cell.
2. The therapy is designed to be patient-specific, as it is developed directly from the patient’s T cells. So rapid manufacturing, combined with a low fail rate, is both a logistic and expensive barrier.
3. Overcoming payer hurdles will be necessary to ensure patients have access to this treatment.

Despite these obstacles, it does appear that CAR T-cell therapy is promising for both life science companies and patients.