Not in her wildest dreams did Anne Faulkner Schoemaker imagine she would one day run the small Malvern company on the frontier of stem-cell science that was the vision of her late husband, Dutch-born entrepreneur Hubert J.P. Schoemaker.
"But here I am," said the interim chief executive officer of Neuronyx Inc., in the Chester County office once occupied by the man who was an inspiration and mentor to many local biotechnology industry scientists and executives.
In 1979, Hubert Schoemaker created Centocor Inc., the region's first profitable biotech company, which was sold to Johnson & Johnson for $4.9 billion in 1999.
Not one to bask in his success, he used some of his wealth to launch Neuronyx in February 2000, and the initial focus was on development of new treatments for neurological disorders based on neuronal stem cells.
But after battling a rare brain cancer for more than a decade, the MIT-educated biochemist died on Jan. 1, 2006, at 55.
After his death, the Neuronyx board asked Anne Faulkner Schoemaker, whose professional background includes commercializing technology from the labs at the University of Pennsylvania and Wistar Institute, to become a director. Members of the Schoemaker family are significant shareholders in the private company, which has raised $41 million from venture capitalists and individuals.
In August, the board decided to transition from being a development-stage start-up to a clinical-stage pharmaceutical company and to search for a chief executive officer with expertise in getting products through clinical trials leading to commercialization, she said.
Hubert Schoemaker had recruited Stephen W. Webster, an investment banker at Paine Webber Inc., to cofound Neuronyx, and since 2003, Webster had been the company's president and chief executive. Webster left Neuronyx in August and has returned to Wall Street as a managing director at First Albany Capital Inc.
So Anne Faulkner Schoemaker, a Juilliard School graduate and pianist with a master's in business administration in finance, joined Neuronyx's executive management committee. In December, the board asked her to serve as interim CEO.
"We have a recruiter who is out looking for the perfect CEO. Until then, for better or worse, here I am," she said. "It's about getting the job done."
Neuronyx and its 23 employees are poised to begin testing its stem-cell therapy in heart-attack patients. The Food and Drug Administration has given the company, which does all its own manufacturing in Malvern, approval to begin a Phase 1 safety trial involving 18 people.
The first four patients are being studied at the Arizona Heart Institute in Phoenix. Scripps Green Hospital in La Jolla, Calif., is also screening patients. The company hopes by the end of the year to have four or five centers recruiting patients.
Neuronyx is the only company in the Philadelphia area believed to be totally focused on adult stem-cell therapy, according to industry experts. The company's technology can produce trillions of cells - six billion patient doses - from a single adult bone-marrow donor that can be used in anyone. A patient's own bone marrow does not have to be harvested.
The cells, in preclinical testing, showed the ability to regenerate damaged tissue.
Neuronyx works only with adult stem cells, not embryonic stem cells that have been the focus of the Bush administration's opposition to federal funding for human embryonic stem-cell research.
Other companies, including Osiris Therapeutics Inc. in Baltimore, are testing adult stem-cell technologies in heart tissue. Neuronyx believes it is the first to inject adult bone-marrow-derived cells directly into heart attack patients.
In Neuronyx's current patient study, the cells will be squirted using a catheter into the area around damaged heart muscle 30 to 60 days after a heart attack. The purpose is to stabilize and restore the damaged tissue and prevent or reduce scar formation and progression to congestive heart failure.
The way it works, said Neuronyx vice president of cellular therapy Joseph Wagner, is the cells secrete a potent "cocktail" of pro-regenerative factors such as cytokines or proteins that go to an area of injury and amplify or boost the host cell's own regenerative capabilities.
"We first made this discovery in spinal-cord injury," Wagner said. "We saw improvement in damage and also in motor function in rats."
Neuronyx hopes to have safety data on the 18 patients, including a 90-day follow-up period, in December and begin a mid-stage Phase 2 study next year. Pharmaceutical companies are required by the FDA to conduct three stages of testing for safety and effectiveness of new drugs in people.
The company's initial focus was on developing treatments for neurological disorders based on neuronal stem cells. Then scientists realized the cells could help regenerate other kinds of tissue and explored additional uses. The data looked most "robust" for treating heart attack, Wagner said.
"We saw these cells were stimulating new blood-vessel formation and they were inhibiting scar tissue" in rats, said Wagner. When the heart becomes scarred, its ability to pump blood is reduced, and that can lead to trouble walking and breathing and ultimately to heart failure.
"We don't think these cells are going to cure everything," Wagner said. "But based on data we have seen, we believe these cells will have the most relevance in acute traumatic or ischemic diseases" where blood flow, and thus oxygen, are restricted to tissue. "That encompasses a lot of diseases" including heart attack, stroke and spinal-cord injury.
Anne Faulkner Schoemaker likes to talk about the company and not herself. "This is just an amazing group of people who are here," she said.
"I don't mean to sound like a Pollyanna, but we are just realizing Hubert's vision. He used to talk about worker bees. We are all the worker bees. I'm just really trying to implement his vision because there is a huge amount of passion. If you talk to any of the people who work here, it will be evident."
Anne Faulkner met Hubert Schoemaker in 1989 when she was at Penn and was trying to license a technology to Centocor, where Schoemaker was CEO. They were together for almost 17 years and married for 10.
Many of those years were spent dealing with Schoemaker's illness. In February 1994, he learned he had medulloblastoma, a fast-growing and usually fatal cancer. He vowed to beat it, and in 1999 as the deal to sell Centocor to Johnson & Johnson was being laid out, Schoemaker was already planning his next move, his wife said.
"I remember the day he said, 'I know what I'm going to do.' And it was all about stem cells," she recalled. "He said, 'This is the next frontier.' And he is right."
The investors that began with Neuronyx are "still very, very supportive of the company," said Gary J. Kurtzman, vice president of life sciences at Safeguard Scientifics Inc., an original investor. The others include Pennsylvania Early Stage Partners, Alliance Technology Ventures L.P., CW Group, and "a couple very high-net-worth individuals."
While a lot of companies are working in stem-cell research, Neuronyx has a "special cell" technology and "manufacturing capability," Kurtzman said. As the company develops more clinical data, it could become "very attractive" to a pharmaceutical or medical-device company as a partner or acquirer.
Use of stem cells in medicine is expected to grow rapidly over the next couple of years, from $35 million this year to $178 million by 2009, said Robin R. Young, a former Wall Street medical analyst who founded Robert Young Consulting Group in Wayne.
"While orthopedics is the hotbed of stem-cell product use today," said Young, who follows 106 companies working on products, "many companies are progressing rapidly with their development of products to treat cardiac, oncologic, urologic and other indications.
"We are finding stem cells have amazing ability to go to the inflammation and treat it," Young said. "Whether adult or embryo, we are beginning to think that what's more valuable than their ability to morph into something else is their ability to create growth factors or stop inflammation."