The first two humans recently received transplanted pig kidneys instead of donated human kidneys. This was big news in the medical world. It is also a foreshadowing of emerging medical genetics investment opportunities. The fact is that we may be at the very beginning of a medical revolution in which xenotransplantation will provide life saving new organs for anyone with the need and without the risk of rejection on donated organs.
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Human Organ Transplants
The first human organ transplants were for a kidney in 1954 and for hearts, livers, and pancreases by the late 1960s. These procedures have saved and prolonged lives. However, in all cases of people with organ failure, there have never been enough human organs to meet the needs of sick patients. In addition, recipients need to have a close enough genetic match with the donor, living or dead, so that the organ is not quickly rejected. Even when there is a close enough match genetically, recipients commonly need to take strong drugs that prevent organ rejection and these drugs have their own long lists of side effects. The long term goal in medical genetics has been to find ways to obtain organs from animals bred to provide the necessary amounts with little or no risk of tissue rejection.
What Is Xenotransplantation?
Xenotransplantation is the term that applies to animal to human organ transplants. However, this is not limited to organs like kidneys. Xenotransplantation applies to any procedure such as infusion, implantation, or transplantation into human recipients of whole organs, live tissue, or individual cells from non-human animal sources. The primary reasons that researchers are working on this are that the needs greatly exceed the supply from human sources and there can be risks to human donors as well.
The use of xenotransplantation eventually will not just be to replace failed kidneys, hearts, and other organs but to help treat conditions like diabetes and various neurodegenerative disorders.
Problems to Be Solved Before Xenotransplantation Becomes Common
There are two big issues to be dealt with before xenotransplantation becomes mainstream. The first is to remove from donor animals or at least modify all genes that could cause rejection in human recipients. The other is to remove or deactivate infectious agents such as hidden retroviruses from donor animals. Currently such transplants, infusions, or implants are only available to those who are critically ill and likely to die soon because there is no other therapy or help available. The goal is to develop therapies that are the first choice for patients in need and not the last line of defense.
What Are the Xenotransplantation Opportunities?
Bloomberg published a useful article about pig kidney advancements and the general state of the related science. They mentioned the two companies that supplied pig kidneys for the first to pig to human xenotransplants, eGenesis and United Therapeutics Corp. In both cases the organs were gene edited kidneys. The process involves CRISPR-Cas9 gene editing technology that removes selected pig genes which in turn are responsible for making antibodies and sugars to which humans react. Additionally, selected human genes are added to the pig kidneys as well.
While eGenesis is a private company, United Therapeutics is a public company organized as a public benefit corporation. News of their pig kidney being used in a human transplant sent their stock price from $240 to $310 a share.
Investing in Xenotransplantation for the Long Term
When the necessary technology is finally sorted out this field could change the face of medicine, cure multiple diseases, and be a huge benefit to the human race. Companies working in this area will likely make huge profits. That day may be a long way off. Spending billions of dollars on research in order to give one pig kidney for the first such transplant is in no way profitable. In order for this to be a profitable business the cost of creating cells, tissue, and organs that will not be rejected will need to be brought down to reasonable levels. And there will need to be thousands if not millions of recipients for these therapies if and when they become extremely affordable. This is a high cost of entry business so we may or may not see competitors for the two current leaders in the field. Late entries would probably come from big pharma such as Johnson & Johnson.
If you are interested in medical genetics investment opportunities you will need to stay alert, put some money in a company like United Therapeutics, and be very, very patient.