NeoStem completing enrollment in heart trial

NeoStem (NASDAQ:NBS) is on track to complete enrollment later this year in a Phase 2 clinical trial of a bone marrow-derived cell therapy to treat patients for acute myocardial infarction, with a data readout six-to-eight months later.

“AMR-001 represents the first compound in a class of cell therapies to have a highly defined cell population and an identified biologically effective therapeutic dose, both of which tie back to the biological mechanism of action that the outcomes of the current study are intended to demonstrate,” chairman and CEO Dr. Robin Smith says in an interview with

Dr. Smith explains that NeoStem’s AMR-001 drug candidate brings a natural repair system, consisting of enriched CD34+ cells, to the heart in order to preserve heart function following a stent placement in patients with a type of heart attack known as ST segment elevation myocardial infarction (STEMI).

She says the technology involves collecting stem cells from the patient’s bone marrow and enriching them to increase their potency. The cells are being tested to save damaged tissue that would otherwise die within days or weeks, through the release of chemicals and proteins that grow blood vessels through a process called angiogenesis.

“STEMI patients are at a high risk of a progressive deterioration in heart muscle function that leads to arrhythmia, recurrent myocardial infarction, congestive heart failure, and premature death,” she points out.

The Phase 2 PreSERVE study, which is enrolling 160 patients at 60 hospitals in the U.S., is being conducted by NeoStem’s Amorcyte subsidiary. Progenitor Cell Therapy (PCT), another NeoStem company, is supporting manufacturing, product supply, and logistics for the trial.

In an earlier Phase 1 study, the company demonstrated that patients dosed at or above a threshold of 10 million AMR-001 cells showed significant improvement in heart perfusion. “Not one patient who received 10 million cells or more had a worsening of left ventricular ejection fraction after six months, while in the five million cell group or in the control group, 30% to 40% got worse over time,” Dr. Smith contends.

The Amorcyte unit, which is focused on the AMR-001 stem cell product for cardiovascular disease, is also preparing for a Phase 1b/2a study in patients with congestive heart failure as well as ongoing preclinical development for traumatic brain injury.

Since 2006, NeoStem has positioned itself to be an integrated leader in cell therapy, with holdings acquired largely through M&A activity. In addition to PCT and Amorcyte, NeoStem’s assets include its Athelos unit, which focuses on autoimmune disorders, and its VSEL, or very small embryonic-like, stem cell technology platform, which is targeting tissue regeneration.

Earlier this month, the company raised $40.25-million in a stock offering to be used for R&D of its cell therapeutic product candidates, including AMR-001, expansion of its business units, strategic transactions, and other general corporate purposes.

Cell therapy is a process that introduces new cells into a tissue to prevent or treat disease or to regenerate damaged or aged tissue. “We firmly believe that this is the future of medicine,” Dr. Smith contends, citing its potential to improve clinical outcomes and reduce overall health care costs.

“Very early on, we realized there was a lot of confusion out there between embryonic and adult stem cells, so we began a program to educate people of all ages, religions, and cultures on the potential of adult stem cells to treat chronic diseases as part of this next frontier in medicine,” Dr. Smith recalls.

“Our focus is on adult stem cells because they are more advanced [and] safer, and they don’t have the same ethical and moral issues associated with embryonic stem cells.”

In 2010, NeoStem and its Stem for Life Foundation began a five-year collaboration with the Vatican’s Pontifical Council for Culture and its foundation, STOQ International, to advance education on the benefits of adult stem cells and reduce misconceptions about cellular research.

In a statement last November, Msgr. Tomasz Trafny said, “[The Vatican] acknowledges that insights from natural sciences play a crucial role in our society, having consequences for anthropology, philosophy and even theology. It is our mission and our duty to explore these dynamics, to offer the best tools for pastoral care, and even to encourage understanding of changing culture.”

Earlier this year, the group held its second international conference at the Vatican to explore the interconnections between scientific breakthroughs, faith, culture, and ethics. The group also has authored a new book, The Healing Cell: How The Greatest Revolution in Medical History is Changing Your Life, that included an introduction by Pope Benedict XVI.

Pointing to the trend of using the body’s own natural repair mechanism to treat disease, Dr. Smith points out that there are more than 27,000 clinical trials now underway in cell therapy, of which about 4,500 are using adult stem cells.

NeoStem’s PCT contract development and manufacturing arm currently serves more than 35 clients, making cell-based therapies for clinical trials. More than 6,000 patients have received products manufactured at PCT plants in Allendale, NJ and Mountain View, CA.

Dr. Smith says both plants are in the process of adding capacity, and PCT is also pursuing expansion overseas.

She figures that if the company can ink a commercial manufacturing contract for an approved cell-based therapy, it could generate annual sales between $38-million and $250-million, depending on the complexity of the product.

In its Athelos unit, which is 20% owned by Becton Dickinson, NeoStem is developing a novel approach to restore a patient’s immune balance by enhancing the number and function of T-regulatory cells, or Treg cells.

Dr. Smith explains that immune-mediated diseases (such as graft-versus-host-disease, autoimmune disorders such as Type 1 diabetes and multiple sclerosis, and allergic conditions) are a result of an imbalance between T-effector cells and T-regulatory cells.

In the Treg program, the company is collaborating with the University of California at San Francisco (UCSF) and the laboratories of Jeffrey Bluestone and Qizhi Tang to develop a therapy for the treatment of Type 1 diabetes.

The collaboration aims to advance the Treg program into a Phase 2 trial in 2014 to evaluate the efficacy of autologous Treg cells in Type 1 diabetes. According to Dr. Smith, this will effectively advance the pipeline more quickly than if the company had developed a program for this clinical indication independently.  “It’s our IP and their IND.”

NeoStem is developing a therapy with Treg cells in collaboration with three research experts in field, Dr. William Busse of the University of Wisconsin, Dr. Mario Castro of Washington University in St. Louis, and Dr. Prescott Woodruff of UCSF, aimed at treating severe asthmatics who are resistant to steroids.

NeoStem’s tissue regeneration program falls under its VSEL technology platform. According to Dr. Smith, preliminary preclinical data generated by third party collaborators have indicated that highly enriched human VSELs were able to integrate, differentiate and potentially regenerate into all basic cell types: mesoderm, ectoderm, and endoderm.

The program is expected to soon move into early clinical studies to assess the therapeutic potential of VSELs in wound care, bone regeneration, and/or macular restoration.

“If substantiated by further research, this could imply significant potential for restorative healing,” she contends.

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