When Novartis dissolved its gene and cell therapy unit a few years ago, a bunch of projects the Swiss pharma giant had incubated were tossed aside. One of them was the work of surgeon Suzanne Ildstad, who has spent decades trying to develop a new way to improve the health outcomes of patients who need organ transplants.
That work has just been salvaged. If it plays out as Ildstad hopes, her company might not only help transform treatment for kidney failure patients—but possibly, for people suffering from other illnesses, such as autoimmune disorders.
Talaris Therapeutics, a Louisville, KY, startup founded by Ildstad (pictured) in 2002 and long known as Regenerex, has secured a $100 million Series A investment led by Blackstone Life Sciences, the biopharma investing arm of private equity firm Blackstone Group. The cash breathes new life into an experimental cell therapy known as FCR001, a one-time treatment meant to make it possible for patients who need a kidney transplant to get one from any donor—without the need for a life-long regimen of immunosuppressive drugs afterwards to prevent rejection of the outsider’s organ. There may be a significant bonus effect—the treatment might also block the continuing threat from underlying autoimmune diseases that, in some patients, caused their own kidneys to fail in the first place.
Ildstad has already tested the technique in humans and seen some promising results. But the work had stalled amidst the cell therapy unit’s revamp at Novartis, which licensed the technology from Regenerex in 2013. With the new cash, Talaris can now fund a Phase 3 study that should start by the end of the year, says CEO Scott Requadt, a former Clarus Ventures managing director and, most recently, a Blackstone venture partner.
There are more than 113,000 people currently on the waiting list for organ transplants in the US alone, and 84 percent of them (94,973) need a donor kidney because one of their kidneys has failed, according to the Organ Procurement & Transplantation Network. The nonprofit group Donate Life America estimates that patients have to wait an average of three to five years for a kidney from a deceased donor.
The wait is shorter—a year or less—if a workable living donor is found, and volunteers. But securing the right organ is still very difficult. Patients have to be “matched” with a donor, a complex process that involves a variety of factors. One of them is finding compatibility between both parties’ immune systems, a test known as “HLA (human leukocytic antigen) typing.” The more mismatched patients are—and a perfect match is rare—the more likely the body will see the donated organ as a threat, and try to destroy it—a condition called graft versus host disease. This threat of rejection is why many transplant patients have to take immunosuppressive drugs for life.
Those drugs come with a cost, something Ildstad—a longtime transplant surgeon, director of the Institute of Cellular Therapeutics, and a professor of surgery at the University of Louisville—has seen first-hand. Suppressing a patient’s immune system with drugs can lead to heart problems or malignancies, and boost the risk of dangerous infections. Heart disease was the leading cause of death among US kidney transplant recipients between 1996 and 2014, according to a paper published last year in the American Journal of Nephrology. “Transplants are wonderful and life enhancing, but the immunosuppressive drugs really take their toll,” Ildstad says in an interview.
Scientists have spent decades trying to find ways to get our bodies to tolerate implanted donor organs—to accept that two kinds of genetically different tissues, from donor and recipient, are inhabiting the same body, a condition called chimerism.
Agnieszka Czechowicz, an assistant professor of pediatrics at Stanford University’s Division of Stem Cell Transplantation and Regenerative Medicine, who isn’t associated with Talaris, notes that the field has made strides. “Various [academic] groups,” she says, have shown that tolerance to transplanted solid organs is possible through cell therapy procedures and other methods. This 2017 paper in the Journal of Organ Transplantation, for instance, highlights some of those efforts at Stanford, Northwestern University, Massachusetts General Hospital, and elsewhere.
Such a therapy, if shown to be safe and effective, “would be a game changing thing for patients,” she says. But it’s been tough to scale that work up and test it in studies large enough to prove their worth.
“Now these efforts are trying to get more rigorous,” Czechowicz says.
Ildstad’s FCR001 is among those efforts, and it’s been in development since she founded her company in 2002.
In her treatment approach, patients not only receive a donor’s kidney, but they also receive an infusion of the same donor’s stem cells and other cells, which in Talaris studies have
