The Spectacular Now: The Marvel of Stem Cells

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Diabetes is another area where stem cell therapies could produce tremendous benefit. CIRM is working with ViaCyte Inc., a company in San Diego, Calif., on a human embryonic stem cell-derived therapy enclosed in a device about the size of a credit card. The device would be implanted under the skin and release what are known as progenitor cells that produce insulin in response to blood-glucose levels. “It will react in real time and produce just the right amount of insulin,” Dr. Feigal says. She expects the device to enter clinical trials later this year.

One of the only stem-cell therapies currently in wide use, bone marrow transplants have been conducted for more than three decades, most often in patients with blood cancers such as leukemia. To receive a bone marrow transplant, patients usually need a sibling with a perfect genetic match, but only 25 to 30 percent of people have such a match, says Robert Brodsky, MD, director of the Division of Hematology at the Johns Hopkins University School of Medicine. He and his colleagues have developed a way to perform bone-marrow transplants with “half-match” relatives, such as a parent, a child, and some of their siblings and half-siblings, who share only half of the patient’s genetic material. “That really expands the donor pool,” Dr. Brodsky explains. “Nearly everyone has a match now.”

This broader pool of donors creates options for patients with sickle-cell anemia, a disease in which the body produces misshapen red blood cells, causing excruciating pain. The disease can be treated with bone-marrow transplants, but eligible full-match donors are rare. So Dr. Brodsky and his collaborators developed a protocol to transplant bone marrow from half-match donors in sickle-cell patients. In successful cases, the defective stem cells that produced the sickle-shaped red blood cells are replaced with healthy stem cells, and patients are completely cured.

Results of a preliminary clinical trial showed that among 17 sickle-cell patients, 11 transplants were successful, prompting the Clinical Research Forum to name this one of the Top 10 Clinical Research Achievements of 2012. Dr. Brodsky suspects that a similar treatment could help people with autoimmune diseases by “rebooting” their immune systems, and he is poised to launch a clinical trial that will treat patients with lupus.

While some researchers and clinical trial patients hunger for swift results, still more dream of future developments that sound like science fiction: cures for ALS and a host of rare and devastating childhood diseases; treatments to grow motor neurons in patients with spinal cord injuries; and therapies to stimulate the growth of insulin-producing Beta islet cells in diabetic patients.

Clinical trials are essential to determining whether these treatments will help and are safe. Many of the most promising therapies are only in a Phase 1 or Phase 2 clinical trial and still face many hurdles before FDA approval. However, research is moving forward, says Dr. Scadden. “But, of course, the pace is incredibly slow for people suffering, which is frustrating for those of us wanting to make a difference for those patients. Only through the ongoing commitment of talented young scientists and, frankly, the continued investment on the part of our nation will we be able to get there.”

In the meantime, Dr. Feigal of CIRM encourages interested parties to volunteer for clinical trials. “Participate in the clinical trials so that we can get the answers sooner,” she says. Stem-cell therapies are also a growth area for investment, with pharmaceutical companies just starting to fund research on the use of stem-cell therapies. “Sustainable sources of funding are going to be important to make sure this technology continues to advance and thrive,” Dr. Feigal says. “Opportunities to help the enterprise abound.”

Trials & Tabulations

For investment opportunities with Capricor Therapeutics Inc., visit capricor.com. For investment opportunities with ViaCyte, visit viacyte.com or contact Paul Laikind, PhD, president and chief executive officer, at plaikind@viacyte.com. Dr. Ellen Feigal at the California Institute for Regenerative Medicine (CIRM) can also help with information for investing in regenerative medicine technology as well as philanthropic and investment opportunities across CIRM’s portfolio. efeigal@cirm.ca.gov For a list of stem cell clinical trials that are currently recruiting patients, visit the US National Institutes of Health website, clinicaltrials.gov.

 

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