SAN ANTONIO—America Stem Cell Inc., a privately heldbiotechnology company dedicated to the development of technologies to enhanceand expand the therapeutic potential of stem cell therapies, last monthreceived approval from the U.S. Food and Drug Administration (FDA) to moveforward with Phase I/IIa multicenter, dual-umbilical cord transplantationstudies evaluating a novel enzyme treatment that Lynnet Koh-LeMaire, thecompany's CEO and founder, says would "transform hematopoietic stem celltransplantation."
The enzyme treatment, ASC-101, has the potential toaccelerate a patient's immune system and platelet recovery, reducingopportunistic infections and other co-morbidities and improving patientsurvival.
Koh-LeMaire, who founded America Stem Cell in 2005, is anentrepreneur and self-described "intrapreneur" who has led the successfuldevelopment of product and technology innovations in several industries, mostnotably for AT&T Bell Laboratories, where she led the creation of theworld's first Caller ID system. Her desire to pursue stem cell technology arosefrom her experience of losing her parents, grandparents and several friends tocancer.
"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, peripheralblood after mobilization and umbilical cord are proving to be an effectivetreatment. Currently, no therapies are available to direct transplanted cellsto the site of disease and increase effectiveness. As a result, fatality ratesare high, with approximately 50 percent of patients receiving a stem celltransplant dying within the first 100 days.
ASC-101 is an enzyme-based treatment for use in celltransplants. 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 IVadministration.
By increasing the efficiency of stem cell homing andengraftment, ASC-101 offers the potential for improved cancer patient survival andquality of life by accelerating hematopoietic and immune system recovery, andreducing the high rate of opportunistic infections resulting from an extendedimmune-compromised period after transplant, says Koh-LeMaire.
"We're not only showing best-in-class efficacy and thepotential to have a commercial blockbuster, but what sets this product verymuch 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'sadded to cells as a simple, 30-minute ex-vivo incubation at room temperature, and fits seamlessly into standardtransplant center protocols, which is important for stem cell clinicians."
Koh-LeMaire stresses that ASC-101 is not a stem cellproduct: "Think of it like Microsoft software. We are not in the business ofproducing stem cells, the hardware. We are in the business of providing theoperating system to enable stem cells to get to where they need to go—thesoftware," she says.
In July 2012, a Phase I clinical study of ASC-101 wasinitiated at MD Anderson Cancer Center in patients with hematologicmalignancies who are candidates for dual-cord blood transplantation. To date,12 patients have been transplanted with no adverse effects; nine patients are farenough along to be evaluable for efficacy.
"Post-transplantation, a patient takes about 25 days toproduce the neutrophils needed to fight infection. For almost a month, thepatient 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 insevere bleeding episodes. What our drug is showing so far, which is veryexciting, 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 toAmerica Stem Cell to proceed with a multicenter trial. Additional trials willbe conducted at the Texas Transplant Center, the Scripps Clinic in San Diegoand Case Western Reserve University in Cleveland, Ohio. The company hopes torecruit approximately 100 patients for Phase III trials and receive FDAapproval by the end of 2016.
"With our efficacy data, we are already in discussions withbiopharma companies for commercialization," says Koh-LeMaire. "We're looking topartner with someone, not only for transforming outcomes for cancer patients,but to move forward with other cells such as MSCs for disease indications likemyocardial infarction and inflammatory diseases, as well as various immunecells 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 thatit has been used for the last 40 years and has saved "hundreds of thousands ofpeople around the world."
"There are hundreds of cord blood trials right now," shesays. "You hear about embryonic stem cells a lot on the news, but those arecontroversial. Cord blood cells are not controversial since they are notderived from embryos. So far, we're seeing dramatic improvement in cancerpatient survival, and we believe this product will transform stem celltransplantation and the field of cell therapy in general."