Personalized Medicine: Wave of the future
By Thomas A. Sternberg/William Blair Investment Management
This is personalized medicine, which involves getting the right drug to the right patient at the right time, and there are three primary areas of focus: gene therapy, cell therapy, and gene editing.
Gene therapy modifies or replaces existing genes, paving the way to cure hereditary diseases. One gene-therapy drug, which treats a hereditary retinal condition that can lead to blindness, is available today. We expect to see at least two more gene-therapy drug approvals in the United States and Europe in 2019.
Cell therapy involves the transplantation of human cells back into the body to replace or repair damaged cells or tissue.
One of the most common forms of cell therapy used today is CAR T-cell therapy, in which cells are extracted from a patient’s body, modified in a laboratory to attack cancer cells in the blood, then reinserted into the patient’s body.
CAR T-cell has distinct advantages over other treatments, including that it’s given once and stays in the body, reducing hospitalization time and automatically treating recurrences of the cancer. There are two approved CAR T-cell drugs available today, each treating a different type of blood cancer.
Lastly, gene editing gives scientists the ability to change an individual’s DNA. Genetic material is added, removed, or altered at particular locations in the genome.
Gene editing took off in 2012 with the discovery of CRISPR-Cas9 technology, which is faster, cheaper, more accurate, and more efficient than other methods.
Although it’s not quite as advanced as other gene therapy and cell therapy approaches, gene editing has a variety of applications. For example, with gene editing we could alter humans’ DNA to make them resistant to certain diseases (like malaria) or even prevent them from acquiring certain diseases (like cystic fibrosis).
Personalized medicine should lead to better patient outcomes and economic outcomes, because we won’t be giving drugs to patients who might not actually benefit from them. And there are several investment implications.
First and foremost, the pharmaceutical companies that produce and sell these therapies will be watched closely, and there will be challenges along the way. There are complexities in the production, supply chain, and delivery of these drugs to patients that must be resolved, for instance. And new payment models will have to be developed, as these are very expensive but one-time therapies.
We also think there will be opportunities for investors beyond the pharmaceutical companies. For instance, there will also be a greater need for equipment and supplies in the production of these therapies and the supply chain in terms of delivering them, and there will be companion diagnostics.
It will be a fascinating area to watch unfold over the next several years, and we expect there to be opportunities for healthcare investors globally.
The views expressed herein do not constitute research, investment advice or trade recommendations, do not necessarily represent the views of William Blair nor TEXPERS, and are subject to revision over time.
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