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As CIRM opens world’s largest stem cell bank, scientists ready their research

Sep 1, 2015 A $32 million public-private bank — where California researchers collect stem cells and scientists from around the world can make withdrawals — officially opened Tuesday with the aim of accelerating the use of engineered stem cells to tackle a wide range of diseases. The bank, funded by the California Institute for Regenerative Medicine and located at Novato's Buck Institute for Research on Aging, already is making 300 induced pluripotent stem cell lines available to researchers studying heart, lung, liver and blinding eye diseases as well as epilepsy, autism, cerebral palsy and Alzheimer's Disease. Officials at San Francisco-based CIRM, which is the state's stem cell research funding agency, said the bank is the largest in the world, based on the number of donors, and will have 750 cell lines by February. But it also is a big win for CIRM as critics push the agency to show some bang from the more than $2 billion it has spent since California voters in 2004 approved selling $3 billion in bonds for stem cell research. CIRM in March 2013 approved spending $32 million to create the bank of induced pluripotent stem cells, which are "mature" cells, such as the skin, that are manipulated to return to their embryonic state. At that point, the bank can coax the cells back to a mature state but as heart, liver, skin or any other organ. The process — with major contributions from Nobel Prize winner Shinya Yamanaka of Kyoto University, the Gladstone Institutes and the University of California, San Francisco — means that a researcher isn't required to get true embryonic stem cells from the brain of an autism patient, for example, and can instead get skin cells [...]

Interview with Dr. Charles Cox on New Stem Cell Trial for Cerebral Palsy

Knoepfler Lab Stem Cell Blog, by Paul Knoepfler ~ December 16, 2013 Dr. Charles Cox at the University of Texas (UT) Health Science Center at Houston is starting a new stem cell-based clinical trial for the treatment of young children with cerebral palsy (CP). There is an urgent need for the development of new treatments for CP. You can learn more about the details of this specific trial here on clinicaltrials.gov. The purpose of this specific study is to compare the effectiveness and safety of banked cord blood to bone marrow stem cells. Dr. Cox, who is The Children’s Fund Distinguished Professor and Director, Children’s Program in Regenerative Medicine at UT was kind enough to do an interview with me on the trial. How did you get interested in the possibility of using stem cells to treat CP? Pediatric neurologic injury is a terrible, unsolved problem. We started with preclinical work on investigating using stem cells to treat various injuries of this kind in 2002. This work has included traumatic brain injury in children and adults and now we are starting this trial for CP. Can you please tell us a bit about the design of the trial? This is a Phase IIa trial in which we will compare two therapies (cord blood and bone marrow) to a placebo control. It is a double blinded study. Neither we nor the parents will know who gets what, but later in the study there is an opportunity for parents to have their kids crossover from placebo. We will give a single dose of 2-10 million cells/kg. That dose was chosen based on a variety of pre-clinical data. Then we will follow the kids for a period of [...]

Possible New Cord Cell Treatment for Cerebral Palsy

New umbilical cord blood cell-based treatments may offer hope for people living with cerebral palsy, notes an article in regional newspaper The Australian. Researchers gave 30 children with cerebral palsy a special treatment course, which included donated umbilical cord blood cells, for six months. As noted in the Stem Cells journal, these children saw significant improvements in brain and movement function. The children who made the most progress were those aged three and under. Other patients with cerebral palsy in the study receiving different treatments didn’t exhibit the same improvements. “I think this is the most promising study we've ever seen in the area of stem cells,” says the head of research at the Cerebral Palsy Alliance, an Australian nonprofit cerebral palsy support center. "This is looking like a possible path to a cure." Some background: Umbilical cord blood contains a set of unique immune cells known “regulatory T-cells,” in addition to stem cells. The stem cells can to grow into any of the body’s 200-plus cell types. Because of its unique properties, cord blood is harvested immediately after birth, and then cryogenically frozen for medical use. According to the Australian (which used the Stem Cells article as the basis for its article), further study is needed before doctors can prescribe the treatment. While the research is still in its early stages, the results may come as goods news to the nearly 800,000 adults and children living with cerebral palsy in the United States.

Young Pioneers Test Cord Blood’s Power to Heal

Saturday, April 2, 2011 By: Kate Hagan, The Sydney (Australia) Morning News Australian children with cerebral palsy will be offered a pioneering treatment using their own umbilical cord blood to provide some of the world's first evidence about its effectiveness at repairing damaged brain tissue. Researchers are seeking ethics approval for a trial at Melbourne's Monash Medical Centre that would offer the treatment to 20 children to determine whether it can cure or relieve the symptoms of cerebral palsy. Joanne Kurtzberg, of North Carolina's Duke University, has reinfused the cord blood of about 250 children with cerebral palsy over the past five years, but has published no scientific data on its effectiveness. Brisbane parents Stephen and Gabrielle Archer travelled to the US at a cost of $40,000 to have their son Zac, 5, treated. He was left with cerebral palsy and epilepsy after suffering a stroke aged four months. Since having his cord blood reinfused four months ago the Archers say movement on Zac's right side is improving and he is having about 20 partial (or focal) seizures a day, compared with 100 previously. The blood that remains in a baby's umbilical cord and placenta after birth is rich in stem cells, which have the ability to develop into other body cells. Southern Health's director of obstetrics, Professor Euan Wallace, said animal studies suggested stem cells in cord blood could repair or assist in the repair of brain injuries. One of the theories is that the stem cells go to the damaged area and recruit cells within the brain to repair it, but the exact mechanism is unknown. ''If it doesn't work then Australian families shouldn't be spending $40,000 going to the US. If it [...]