GPS for stem cells
SAN ANTONIO—America Stem Cell Inc., a privately held biotechnology company dedicated to the development of technologies to enhance and expand the therapeutic potential of stem cell therapies, last month received approval from the U.S. Food and Drug Administration (FDA) to move forward with Phase I/IIa multicenter, dual-umbilical cord transplantation studies evaluating a novel enzyme treatment that Lynnet Koh-LeMaire, the company's CEO and founder, says would "transform hematopoietic stem cell transplantation."
The enzyme treatment, ASC-101, has the potential to accelerate a patient's immune system and platelet recovery, reducing opportunistic infections and other co-morbidities and improving patient survival.
Koh-LeMaire, who founded America Stem Cell in 2005, is an entrepreneur and self-described "intrapreneur" who has led the successful development of product and technology innovations in several industries, most notably for AT&T Bell Laboratories, where she led the creation of the world's first Caller ID system. Her desire to pursue stem cell technology arose from her experience of losing her parents, grandparents and several friends to cancer.
"I decided to forever remove the pain of cancer," Koh-LeMaire says.
For many patients with leukemia, lymphoma and other cancers, transplants of hematopoietic stem cells derived from bone marrow, peripheral blood after mobilization and umbilical cord are proving to be an effective treatment. Currently, no therapies are available to direct transplanted cells to the site of disease and increase effectiveness. As a result, fatality rates are high, with approximately 50 percent of patients receiving a stem cell transplant dying within the first 100 days.
ASC-101 is an enzyme-based treatment for use in cell transplants. It is comprised of a recombinant enzyme,α-1,3 fucosyltransferase VI (FTVI), plus substrate, GDP-fucose. ASC-101 is designed to add a sugar (fucose) to the surface of cells prior to IV administration.
By increasing the efficiency of stem cell homing and engraftment, ASC-101 offers the potential for improved cancer patient survival and quality of life by accelerating hematopoietic and immune system recovery, and reducing the high rate of opportunistic infections resulting from an extended immune-compromised period after transplant, says Koh-LeMaire.
"We're not only showing best-in-class efficacy and the potential to have a commercial blockbuster, but what sets this product very much apart from everything else is that it is easy to administer," she says. "It's an off- the-shelf product that can be shipped to transplant centers. It's added to cells as a simple, 30-minute ex-vivo incubation at room temperature, and fits seamlessly into standard transplant center protocols, which is important for stem cell clinicians."
Koh-LeMaire stresses that ASC-101 is not a stem cell product: "Think of it like Microsoft software. We are not in the business of producing stem cells, the hardware. We are in the business of providing the operating system to enable stem cells to get to where they need to go—the software," she says.
In July 2012, a Phase I clinical study of ASC-101 was initiated at MD Anderson Cancer Center in patients with hematologic malignancies who are candidates for dual-cord blood transplantation. To date, 12 patients have been transplanted with no adverse effects; nine patients are far enough along to be evaluable for efficacy.
"Post-transplantation, a patient takes about 25 days to produce the neutrophils needed to fight infection. For almost a month, the patient doesn't have an immune system and infection rates are over 95 percent. Today, it takes about 49 days for platelets to recover, which can result in severe bleeding episodes. What our drug is showing so far, which is very exciting, is that the median number of days to neutrophil recovery is 15 days, and platelet recovery happens in about 34 days," says Koh-LeMaire.
Based on these results, the FDA has given approval to America Stem Cell to proceed with a multicenter trial. Additional trials will be conducted at the Texas Transplant Center, the Scripps Clinic in San Diego and Case Western Reserve University in Cleveland, Ohio. The company hopes to recruit approximately 100 patients for Phase III trials and receive FDA approval by the end of 2016.
"With our efficacy data, we are already in discussions with biopharma companies for commercialization," says Koh-LeMaire. "We're looking to partner with someone, not only for transforming outcomes for cancer patients, but to move forward with other cells such as MSCs for disease indications like myocardial infarction and inflammatory diseases, as well as various immune cells and other cell types for a variety of indications."
Koh-LeMaire acknowledges that cord blood transplantation is "the boring part of stem cell research," but laughing that off, she adds that it has been used for the last 40 years and has saved "hundreds of thousands of people around the world."
"There are hundreds of cord blood trials right now," she says. "You hear about embryonic stem cells a lot on the news, but those are controversial. Cord blood cells are not controversial since they are not derived from embryos. So far, we're seeing dramatic improvement in cancer patient survival, and we believe this product will transform stem cell transplantation and the field of cell therapy in general."