Science Is What Works

Such as this:

A four-year-old girl has become the first patient in Spain to recover from brain cancer after being treated with stem cells from her own umbilical cord blood. The announcement of the girl's recovery came March 7 from the company Crio-Cord, a stem cell bank in Spain.

Alba was born healthy in 2007, but at age two she was diagnosed with a rare form of brain cancer. Her treatment consisted of extracting the majority of the tumor from her brain. She was then given chemotherapy to reduce and eventually eliminate the remainder of the tumor. Alba's blood system was destroyed during the final round of chemo, thus requiring a transplant of cord blood stem cells.

The procedure was carried out in 2009 by Dr. Luis Madero of the Department of Oncology and Hematology at the Nino Jesus Hospital in Madrid. Today, four year-old Alba is a healthy girl.

Periodic Reviews

Sixty days after the transplant, Alba was given new stem cells taken from her peripheral blood in order to accelerate the production of platelets. Fourteen months after the transplant, her blood system was completely restored, and she has since enjoyed a normal life.

Dr. Madero called her case unique in Spain. “The use of stem cells to regenerate the blood system is an extended treatment for this form of cancer,” he said. What makes her case unique, he added, “is that for the first time in our country, the stem cells came from a patient’s own umbilical cord, preserved from birth. In recent years, transplants of cord blood stem cells have become increasingly common. In the case of siblings, these stem cells are the best therapeutic option that exists,” he said.

“Our best investment”

Alba’s father, Santiago, who is a computer engineer, and her mother, Teresa, a literature professor, agreed that keeping the blood from Alba’s umbilical cord was the “best investment” they ever made. Santiago said he had previously seen a report “on the treatment for Parkinson’s using stem cells … and was sympathetic to the idea of using stem cells to treat degenerative diseases.” “Keeping the umbilical cord is a wager for the future, a life insurance policy that you don’t know if you will need but that could save a life,” Teresa added.

The head of Crio-Cord, Guillermo Munoz, also said he was pleased at the results of the therapy. He noted that the organization was “proud to have participated in Alba’s healing process.” Cases like these confirm “that umbilical cord blood is an excellent source of stem cells. Being the youngest cells of their kind in the human body, they have great potential to cure,” Munoz explained.

And this:

The fiery subject of medicine based on human stem cells usually evokes roiling controversy, often generating more questions than answers. So, stem cell therapies have yet to catch on among investors -- and on Wall Street. No wonder a tiny company like NeoStem (NBS), which focuses on stem cell research and its medical applications, continues to be underappreciated, if not ignored.

And yet it has attracted several big-name investors that are intrigued by some unexpected events, including the Catholic Church's surprising embrace of NeoStem's research and NeoStem's foothold in China's vast health care market. The Vatican has decided to invest $1 million in NeoStem, a biopharmaceutical company developing proprietary cellular therapies. NeoStem is aiming to become a single source for the collection, storage and manufacture of adult stem cells -- rather than embryonic stem cells from aborted fetuses -- for cell-based medicine and regenerative science.

What's the Vatican's interest in NeoStem? In particular, the Church is very interested in the company's work on adult stem cells, and what they imply on theological and philosophical grounds.

"A Fundamental Shift"

"It's the Vatican's first-ever contractual collaboration with an outside commercial venture to advance adult stem-cell research," says Rev. Tomasz Trafny, of the Vatican's Pontifical Council for Culture. In a public statement in New York last May when the Vatican quietly made public its involvement with NeoStem, Rev. Trafny said the Vatican's charitable foundation, STOQ International, will make a commitment of $1 million to start its collaboration with NeoStem. It will spearhead, he said, an educational campaign to generate awareness of the "cultural relevance of such a fundamental shift in medical treatment options, particularly with regard to the impact on theological and ethical issues."

Explaining further, Rev. Trafny said the initiative will partner NeoStem's and the Vatican's charitable organizations to expand research and raise awareness toward adult stem cell therapies, and to explore their clinical application in the field of regenerative medicine, as well as the cultural impact of such research. Considering the potential implications of scientific investigation, medical applicability and the cultural impact of research on adult stem cells, "we view the collaboration with NeoStem as a critical effort," says Rev. Trafny. "We are particularly excited about NeoStem's VSEL [very small embryonic-like] stem cells technology," he adds.

VSEL stem cells have physical characteristics typically found in embryonic stem cells. In 2007, NeoStem acquired a technology focused on VSELs that has an ability to detect cells found in different types of tissues that could interact with a specific organ to help in repairing damaged or diseased tissues.

A Three-Day Conference in Rome

The Vatican isn't the only big backer. On Jan. 13, the U.S. Defense Department awarded NeoStem $1.7 million to help fund the company's research in using its VSEL technology for the treatment of osteoporosis and improve bone health. One of the highlights of the Vatican-NeoStem partnership will be a three-day international conference, to be held at the Vatican in November, on adult stem cell research, including NeoStem's VSEL stem cell technology.

"This technology opens the door to the possibility of achieving the positive benefits associated with embryonic stem cells without the ethical or moral dilemmas and other negative effects associated with embryonic stem cells," says Dr. Robin Smith, NeoStem's chairman and CEO. For more than 40 years, she says, physicians have been using adult stem cells to treat various blood cancers. But only recently, she notes, has the promise of using adult stem cells to treat other diseases begun to be realized.

The Vatican-NeoStem collaboration hasn't yet generated much enthusiasm from Wall Street or the medical community. But it did nudge some large U.S. institutional investors to take a look at NeoStem. Among those that have bought its shares are BlackRock Institutional Trust, which has acquired a 1.22% stake; D.E. Shaw, which purchased 0.91%; State Street Global Advisors, which now owns 0.60%; Northern Trust Investment, with 0.33%; and Vanguard Group with 0.24%.

Rising 12-Month Targets

"We think an investment in NeoStem is a good way to participate in the growing potential of stem cells, where a significant breakthrough could happen over the next five years," says Shiv Kapoor, biotech analyst at investment firm Morgan Joseph. And it's also a great way to benefit, through NeoStem, from the large and growing Chinese health care market, he adds. In 2009, NeoStem acquired 51% of Suzhou Erye Pharmaceuticals in China, which produces a series of antibiotics, including penicillin and cephlasporins. Suzhou in 2009 generated revenues of $11 million and $6 million in earnings. "We expect the Chinese pharmaceutical business to grow at least 20% over the next decade," says Kapoor.

NeoStem is licensed to use stem cell-based orthopedic technology in Asia and has now set up a program in China to offer orthopedic treatments using adult stem cells through a network of hospitals. "NeoStem is developing one of the best cell therapy platforms in the industry," which includes stem cell storage, banking, and autologous stem cell-based treatments, says Kapoor. He rates NeoStem's stock, now trading at $1.49 a share, a buy with a 12-month target of $3.50.

Yale Jen, biotech analyst at investment firm Maxim Group, also rates NeoStem a buy, but with a higher 12-month price target of $5 a share. The stock remains undervalued, says Jen, based on the increasing revenues generated by its stem cell operations in China and the advancement of its VSEL technology.

An Enhanced Portfolio of Services

NeoStem has now become a global one-stop-shop on cell therapy, says CEO Smith, as a result of its January acquisition of Progenitor Cell Therapy, a privately owned cell therapy company that owns cell manufacturing and storage facilities. As a contract manufacturer, Progenitor was a big factor in the success of Dendreon (DNDN) in its manufacture of Provenge, the first autologous cellular immunotherapy to get U.S. Food and Drug Administration approval for the treatment of metastatic prostate cancer. Provenge has since become a blockbuster drug.

The purchase of Progenitor enhances NeoStem's portfolio of services and allows the in-house development of all stages of regenerative therapeutics, notes Reni Benjamin, biotech analyst at investment firm Rodman & Renshaw. He says that on a sum-of-the-parts analysis, NeoStem, which he rates as outperform, is worth $3 a share.

The company's regenerative medicine franchise, he adds, provides it with "a blockbuster potential." He figures that although NeoStem will remain in the red through 2011, revenues have ballooned. They jumped from $11.6 million in 2009 to an estimated $69.9 million in 2010. For 2011, Benjamin forecasts revenues will leap even more, to $93.4 million.

There's little doubt that NeoStem will start attracting more institutional and individual investors seeking to participate in the still-evolving field of stem-cell therapies. As NeoStem solidifies its leading role in that still underappreciated sector, it seems likely to emerge as a suitable way to invest in the nascent field of stem cell therapy.