In 2022, neuroscience research made significant advances by understanding the role of large-scale neuronal connections in disorders. So did cancer research.

In 2022, neuroscience research made significant advances by understanding the role of large-scale neuronal connections in disorders. So did cancer research.

Metabolic health is at an odd juncture. With the advent of glucagon-like peptide (GLP-1) agonists, pharmacologically induced weight loss has matured into a viable therapeutic option at long last. And research into the drug class is continuing apace.

In 2021, Biogen Inc.’s Aduhelm (aducanumab) became the first amyloid-targeting therapy to win U.S. FDA approval in Alzheimer’s disease. After decades and dozens of failed phase III trials, the drug was granted accelerated approval in June 2021. In January 2022, however, the U.S. Center for Medicare & Medicaid Services said it would only cover use of Alzheimer’s MAbs targeting amyloid in NIH trials or trials it approved, thus appearing to call into question the rigor of FDA-approved trials.

2021 was the year Aduhelm (aducanumab, Biogen Inc.) was approved as the first amyloid-β-busting drug for Alzheimer’s disease. And in 2022, there was as much need for an effective AD drug as ever. Aduhelm’s commercial fate was sealed with the decision of the Center for Medicare & Medicaid Services (CMS) that the drug would only be reimbursed in clinical trials approved by the CMS or the NIH.

Biopharma just wouldn’t be biopharma without continuing innovation. Even in a year rife with economic and regulatory turmoil, the industry still achieved major advancements set to change the health care landscape going forward. Standouts for 2022 include cell therapy and gene editing approaches making significant gains, while industry celebrated a new checkpoint inhibitor added to the oncology armamentarium.

Repeat expansions of two or more base pairs cause dozens of neurological disorders – Huntington’s disease, which is caused by an expansion of the triplet CAG in the coding sequence for huntingtin, is perhaps the most famous one. Now, investigators at Stanford University have shown that cancer genomes, too, frequently feature repeat expansions.

CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.

Chimeric antigen receptor (CAR) T cells are astounding. In B-cell cancers, they have been transformative. Yet engineering-wise, CAR T cells are in the equivalent of the Model T era. CAR T-cell engineering has already evolved, with the addition of costimulatory domains, which affect cell expansion and signaling. But once the cells are injected into a patient, there is really no way to affect their behavior.