Boston has landed an intriguing biotech startup seeking to tap the New England talent pool. AlloCure, the developer of an adult stem cell therapy for a severe kidney disease, has moved its headquarters from Salt Lake City to Burlington, MA, where it is being led by new CEO Robert Brenner, a veteran executive from AMAG Pharmaceuticals and Amgen.
AlloCure first made news in July 2008, when it nailed down an initial financing of $14.5 million from SV Life Sciences and the venture arm of Danish drug giant Novo Nordisk. This company is still quite lean, with less than 10 employees, but it has global operations with sites in Utah and Hamburg, Germany, where its respective co-founders, Christoph Westenfelder and Axel Zander, are located. Brenner, the new CEO, will be based in Burlington, MA.
The big idea at AlloCure is to come up with an adult stem cell therapy that could be the first effective treatment for acute kidney injury, also sometimes called acute renal failure. This is a condition in which people’s kidneys quit working, and the only real treatments are dialysis or a kidney transplant. People with diabetes or high blood pressure are often at high risk of getting an acute kidney injury in the hospital if they go on a heart bypass machine during surgery, or get certain potent antibiotics, Brenner says. Estimates vary, but as many as 500,000 people in the U.S. suffer from this condition, and various studies put the death rate at 30 to 70 percent within a year’s time of the injury, Brenner says.
“If we could develop a cell therapy for acute kidney injury, it would be a revolution,” Brenner says. “People have been working on this for their entire professional careers.”
Lots of novel ideas have been tried, and all of them have failed. Researchers in the past have experimented with an injectable peptide from Scios, a unit of Johnson & Johnson, as well as Amgen’s blockbuster anemia drug, erythropoietin (Epogen), and a variety of devices.
The founders of AlloCure have a different strategy that hinges on an infusion of adult stem cells that might be able to prevent acute kidney injury from happening, or help reverse the damage in the early stages, Brenner says. AlloCure’s founders have learned how to take a certain kind of adult stem cell found in the bone marrow, called mesenchymal stem cells, and greatly expand their numbers in a consistent manufacturing procedure. Importantly, they’ve shown they can isolate cells without certain markers on the surface, known as antigens, that could provoke the patient’s immune system to recognize the cells as foreign invaders that must be attacked. This means that cells from any individual could be grown in an AlloCure factory, and infused into any patient, without requiring a genetic match, Brenner says.
Because the infusion would be standardized, it’s possible the cells could be kept in stock at hospitals and given “off-the-shelf” when a kidney specialist sees signs of acute kidney injury.
AlloCure hasn’t publicly disclosed any clinical trial results yet on this procedure, but it enticed Brenner to join the company based on results of a study of 16 patients. This study was designed to give patients a single high-dose infusion of these mesenchymal stem cells after they underwent surgery which put them on a heart bypass machine. While patients weren’t randomly assigned to a control group to offer a strong comparative benchmark, researchers did follow other patients who got similar surgeries without the cell therapy infusion, Brenner says. That offered an eye-opening contrast, he says. The results will be presented at an upcoming medical meeting and published in a peer-reviewed journal, Brenner says.
“The results are extremely promising,” he says.
Brenner, 42, doesn’t need to hire a consultant to tell him the difference between good and mediocre data. He’s a medical doctor himself, who did his residency at Brigham & Women’s Hospital in Boston, and a fellowship in nephrology (the study of kidney function) at Stanford University Medical Center. Brenner spent nine years of his career at Thousand Oaks, CA-based Amgen (NASDAQ: AMGN), ending his tenure there in 2008 as executive director of its renal anemia global program. He endured the worst year in the company’s history, 2007, as Amgen was besieged with safety warnings about its anemia drugs for both kidney and cancer patients. After Amgen, he moved up to senior vice president of medical affairs for Lexington, MA-based AMAG Pharmaceuticals, where he was involved in the rollout of that company’s drug for kidney patients, ferumoxytol (Feraheme).
AlloCure, under its new CEO, is now mapping out a course to prove that its therapy really works in a more rigorous mid-stage clinical trial. The company is working on the design of a Phase II trial that will randomly assign patients to get its cell therapy infusion, or something else in a control group. The company is considering different doses and timing for the infusion, to establish whether it can prevent acute kidney injury or help reverse the course of disease once it’s caught in the early stages, Brenner says.
The company has to meet with the FDA to talk about his clinical trial game plan, with hopes of getting the trial going in 2011, Brenner says. AlloCure should have enough money to get the mid-stage trial started, but at some point it will need to consider more financing, from venture capitalists or other sources, he says.
These are, of course, still very early days for AlloCure. Plenty could go wrong in further trials, and the therapy might not work, Brenner says. But if it does, the potential impact for medicine is big. Plenty of companies are testing adult stem cell infusions for various ailments, but no others are pursuing this mode of treatment for acute kidney injury, Brenner says.
“If we can develop a robust treatment, it would be remarkable,” Brenner says. “The benefits to patients and society would be tremendous.”
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