Cord Blood Banking & Stem Cells

A new mother talks about her experience with cord blood banking

StemCells, Inc. Receives FDA Approval to Initiate Clinical Trial of HuCNS-SC(R) Cells in a Myelin Disease

StemCells, Inc.  (STEM) today announced that it has received approval from the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of the Company’s proprietary HuCNS-SC product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a fatal brain disorder that mainly affects young children. This Phase I trial is designed to evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for PMD. Currently, there are no approved treatments for this disease.

This is the Company’s second FDA-approved clinical trial to evaluate HuCNS-SC cells as a potential treatment for neurodegenerative diseases. The first such study was the Company’s Phase I clinical trial of HuCNS-SC cells to treat neuronal ceroid lipofuscinoses (NCL), or Batten disease. The Phase I NCL trial will be completed in January 2009.

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ISSCR Releases New Guidelines to Shape Future of Stem Cell Therapy

The ISSCR Releases New Guidelines to Shape Future of Stem Cell Therapy
Regulation needed as new study reveals clinics exaggerate claims and omit risks

Deerfield, IL, December 3, 2008 – Today, the International Society for Stem Cell Research (ISSCR), the world’s leading professional organization of stem cell researchers, released new guidelines for the responsible development of safe and effective stem cell therapies for patients. A Commentary article that summarizes the Guidelines for the Clinical Translation of Stem Cells will be published by Cell Press in the December issue of Cell Stem Cell, the official affiliated journal of the ISSCR.

These guidelines define a roadmap for medical researchers and doctors, outlining what needs to be accomplished to move stem cells from promising research to proven treatments for patients. The new guidelines will accelerate the translation of stem cell research into practice while addressing associated scientific, clinical, regulatory, ethical and social issues. Founded on core principles of scientific rigor and ethical conduct, the recommendations offered in the guidelines include an insistence on expert evaluation and independent oversight, a thorough informed consent process to provide patients with essential information on the unique aspects of stem cell-based treatments, and transparency in reporting of clinical trial results.

“Our guidelines will arm patients and their doctors with the information they need to make decisions about whether to seek stem cell treatments,” said Dr. Olle Lindvall, co-chair of the ISSCR task force that developed the guidelines and professor in clinical neurology at the University of Lund. “Stem cell research holds tremendous promise for the development of novel therapies for many serious diseases. However, as clinicians and scientists, we recognize an urgent need to address the problem of unproven stem cell treatments being marketed directly to patients.”

Too often rogue clinics around the world exploit patients’ hopes by offering unproven stem cell therapies, typically for large sums of money and without credible scientific rationale, oversight or patient protections.

This concern is further emphasized in a Correspondence article from Dr. Timothy Caulfield and colleagues of the University of Alberta, Canada, which also appears in the December issue of Cell Stem Cell. A content analysis of claims made on 19 Web sites offering so-called “stem cell therapies” was performed to assess the portrayal of the services offered by each organization. In addition, the authors assessed whether these claims are substantiated by research reported in the professional medical literature. The authors provide clear evidence that the vast majority of the clinics examined over-promise results and gravely underestimate the potential risks of their offered treatments.

The ISSCR’s new guidelines establish standards that can be used to judge the claims made by stem cell clinics and whether the treatments they offer are being developed responsibly. The ISSCR also offers a handbook for patients and their doctors evaluating a stem cell therapy.

The ISSCR urges governments and regulatory bodies to enact the recommendations outlined in these guidelines. The guidelines call for countries without an official regulatory body to develop a way to monitor new stem cell-based treatments, and the ISSCR has offered to advise agencies that want to build these regulatory capacities.

“Regulators have a responsibility to prevent exploitation of patients in their jurisdictions, and where necessary, to close fraudulent clinics and take disciplinary action against the doctors involved,” said Dr. George Q. Daley, immediate past-president of the ISSCR and associate director of the Stem Cell Program at Children’s Hospital Boston.

To develop these new guidelines, the ISSCR convened an international task force of experts in stem cell science, clinical research and bioethics from 13 countries.  The task force was led by Dr. Lindvall and Dr. Insoo Hyun, ISSCR member and associate professor of bioethics at the Case Western Reserve University School of Medicine.
“Our task force has captured the most current, comprehensive thinking on translational stem cell research. The result – these new guidelines – will be valuable for all members of the stem cell community,” said ­­­Dr. Fiona Watt, president of the ISSCR.

Patients, medical researchers, regulators and those interested should visit the ISSCR’s Web site at www.isscr.org to see the Guidelines, a handbook for patients and more information on stem cell research. In addition, the content of the Guidelines is digested in the Commentary article authored by the task force, which is available online at www.cellstemcell.com/.

Animal stem cell bank in Chennai, a first in India

CHENNAI: Every time three-year-old Giggle, a German Shepherd, hides in some corner of the house with her head down, her owner Sridhar Venkatraman, an IT professional, feels the pain. “With each passing month, she is becoming increasingly disabled and racked by pain because of arthritis. Search engines on the internet led me to reports that show how several animals abroad have been treated for the disease with vet stem cell therapy. But veterinarians here don’t offer such treatment,” he says.

Giggle and many dogs like her may some hope now. The Tamil Nadu Veterinary Sciences University (TANUVAS) is planning to establish an animal stem cell bank in Chennai, perhaps the first such in the country. Scientists hope this will eventually lead to better treatment for animals like Giggle who suffer from denegerative diseases.

TANUVAS has submitted its proposal to the department of bio-technology, ministry of science and technology, seeking Rs 15.3 crore to put up the facility. “The file is pending. But we hope we will get to start the project by mid-2009,” says TANUVAS vice-chancellor Dr P Thangaraju. At a time when human stem cell therapy for various ailments is still in the experimental stage, the university plans to offer some certified therapies for animals, besides starting research on new avenues in collaboration with the Japan-based Nichi-In Centre for Regenerative Medicine (NCRM).

The proposed centre would help store adult animal stem cells drawn from bone marrow and fat (adeponectine) tissue for research on corneal, spine and untreatable fractures besides degenerative diseases, Dr Thangaraju says.

Although restrictions on clinical trials on animals are far less than on humans, TANUVAS has decided against using embryonic cells because of the ongoing moral debate about using human embryos. Dr Jestin Williams, principal investigator, TANUVAS, who will be in charge of the stem cell bank, says a small sample (about two tablespoons) of a dog’s own fat drawn from the animal’s belly would be given to NCRM to isolate regenerative cells. The cells will then be returned either in ready-to-inject syringes for therapy or research
, or in vials for storage. “The stem cells will then be injected directly into the animal’s joint or any other area that needs treatment,” he says.

Scientists will also be working on developing cell lines that could replace ailing cells in the animal’s body. “We will work on methods to reprogramme adult stem cells back to embryonic cells. Scientists in Japan have already been able to do this,” says Dr Williams. TANUVAS will also throw options open for animal owners to store animal stem cells for future use. “Though there has not been a great breakthrough in cord blood, many people have invested in storing it for future use. That’s when we realised that there could be an equally good response for companion species such as dogs and horses,” adds Dr Jestin.

Senior veterinarians are enthused by the TANUVAS proposal. “Though we are almost on a par with developed countries when it comes to treatment and management of diseases, we are way behind other countries when it comes to research,” says a senior veterinary surgeon. Experts believe that this will, besides bringing in a new perspective to animal healthcare, provide them vital clues for human trials as well.

“This is the only field where experiments on animals are taking place after they have been conducted on humans. But in research of this sort, we always learn. It will be interesting to see how the university progresses with its research. Perhaps there will be something for us to learn while we work with human cells,” says Dr Rama Shanker Verma, associate professor, stem cell and molecular biology lab, Indian Institute of Technology, Madras.

Complete article here.

Breakthrough Windpipe Transplant Uses Stem Cells

BALTIMORE ’09: Maryland to host World Stem Cell Research Summit

Maryland will host the 2009 World Stem Cell Summit next September in Baltimore, Gov. Martin O’Malley announced Wednesday at the state’s first stem cell research symposium.

The three-day summit, which will be held at the Baltimore Convention Center, is expected to bring in 1,500 stem cell researchers, business leaders and policymakers from across the globe to discuss the future of the field.

“I’m very, very grateful to all of those who made the decision to come to Maryland,” said O’Malley, speaking at the Johns Hopkins Applied Physics Laboratory. “It follows a great deal of recognition that we’ve been receiving over the efforts that we’ve made together.”

Summit attendees are expected to discuss stem cell policy issues, including how to legally transport stem cells across state lines, said Bernard Siegel, executive director of the Florida-based Genetics Policy Institute, which is organizing the event.

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Patient’s stem cells used to treat heart failure

Lisa Rosetta / Salt Lake Tribune
SALT LAKE CITY — University of Utah researchers are going to be the first in the country to inject patients’ own stem cells into their hearts to treat two types of heart failure.

After drawing about three tablespoons of patients’ own bone marrow, researchers will grow cardiac-repair cells — believed to help heart muscles and improve blood flow — in culture for about 12 days.

The cells that survive culture are healthier than the original ones extracted from the patient, said Amit Patel, director of cardiovascular regenerative medicine at the university’s School of Medicine.

Two weeks later, the newly grown stem cells will be injected directly into the left ventricle of the patients’ hearts during a minimally invasive surgery developed by Patel, the national principal investigator of the Aastrom Biosciences-sponsored Phase II clinical trial.

Most patients will leave the hospital two to three days after surgery — and within six months, will start to feel the results, he said.

In stem cell clinical trials conducted outside of the country, patients experienced between 20 percent and 100 percent improvement.

“This is truly unique in the type of patients and the type of delivery,” Patel said.

The one-year Cardiac Repair Cell Treatment of Patients with Dilated Cardiomyopathy study — the first trial of its kind in the country — will provide “patients who have limited to no other options with a viable treatment,” Patel said.

“By using a patient’s own cells, we eliminate the concern of rejection and the need for potentially harmful immunosuppressive drugs.”

The treatment is targeted at patients with two types of heart disease. Ischemic heart disease occurs when hearts don’t get enough blood and oxygen because of heart muscle damage caused by coronary artery disease.

Non-ischemic heart disease describes muscle damage caused by other means, including viruses or drug use, Patel said.

A subset of these patients has dilated cardiomyopathy, a condition that leaves the heart weakened, enlarged and unable to pump blood efficiently.

“For most of these patients, the only option has been a heart transplant,” said David Bull, professor and division chief of cardiothoracic surgery in the University of Utah’s medical school.

Patel said about 50 out-of-state patients — many of whom are following him from Pittsburgh, where he moved from three months ago — are “lined up” to participate.

3 UW spinoffs form major stem cell company

By RYAN J. FOLEY | Associated Press Writer

MADISON, Wis. – Three companies founded by star University of Wisconsin-Madison stem cell researcher James Thomson are merging into a single entity that aspires to be a world leader in the field.

Under a deal announced Monday, Cellular Dynamics International is joining forces with Stem Cell Products Inc. and iPS Cells Inc. Backed by $18 million in private venture capital, the new company is keeping CDI’s name and its headquarters in Madison.

“The new company has an ambitious goal: it intends to be the world leader in the industrialization of basic stem cell technology,” said Bob Palay, its chairman and chief executive.

Thomson said the company would focus first on supplying human heart cells made from stem cells to pharmaceutical companies for drug testing. He has long predicted stem cells would be most useful first in speeding drugs to market and pinpointing potential side effects.

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Stem Cells Spawn First Drug-Free Windpipe Transplant

By Michelle Fay Cortez
Nov. 19 (Bloomberg) — Doctors operating on a 30-year-old Colombian woman restored her ability to breathe freely with the world’s first transplanted windpipe specially treated to prevent organ rejection.

The airway connecting Claudia Castillo Sanchez’s left lung to her windpipe collapsed after a persistent tuberculosis infection, leaving her short of breath and unable to perform routine daily activities. Efforts to prop it open failed, leaving Spanish doctors two options: remove the lung or replace the airway using an experimental technique tried only in animals.

The donor windpipe, or trachea, was washed 25 times to strip away all vestiges of live tissue, which could trigger rejection, then coated with cartilage cells grown from Castillo’s own adult stem cells to trick her body into accepting the transplant, the doctors said in The Lancet. She was released from the hospital 10 days after the June operation and isn’t taking drugs to suppress her immune system, a standard approach to prevent the body from rejecting transplanted organs.

“We think that this first experience represents a milestone in medicine and hope that it will unlock the door for a safe and recipient-tailored transplantation of the airway in adults and children,” said the physicians, led by Paolo Macchiarini, head of the thoracic surgery department of the Hospital Clinic in Barcelona.

Difficult To Treat
Damage to the large airways — the trachea that runs down the throat and the bronchial tubes that connect it to the lungs – – is difficult to treat. Previous grafts of more than six centimeters (2.4 inches) have failed, so the successful transplant of the seven-centimeter windpipe for Castillo’s left main bronchus suggests tissue-coated replacements may become an alternative for treating diseases of the upper airways.

Castillo hasn’t produced any antibodies against the donor windpipe and her lung function is in the normal range for a woman her age, doctors said. She is able to walk 500 meters (1,600 feet) without stopping, climb stairs and take care of her children, aged 4 and 15.

Macchiarini performed the operation in Barcelona and was the lead investigator on the grant used to conduct the preliminary work. The cleansing of the trachea, from a 51-year-old woman who died of a brain hemorrhage, was done using a technique created at the University of Padua in Italy.

Stem Cells
Castillo’s stem cells were extracted from her bone marrow then grown into cartilage cells at the University of Bristol in the U.K. The donated trachea was coated on the outside with the cartilage cells and lined on the inside with Castillo’s healthy epithelial cells to create a hybrid organ using an incubator developed at Politecnico di Milano in Italy.

“Just four days after transplantation the graft was almost indistinguishable from adjacent normal bronchi,” Macchiarini said in a statement. “After one month, a biopsy elicited local bleeding, indicating that the blood vessels had already grown back successfully.”

The initial results of the surgery are impressive, wrote Toshihiko Sato and Tatsuo Nakamura, from Kyoto University’s Institute for Frontier Medical Sciences, in a comment that accompanied the study. More information and follow-up is needed to fully evaluate the results, they said.

The bio-engineered approach of using donor scaffolds coated with tissue grown from the patient’s own stem cells may one day be used for other types of transplants, the investigators said.

“Surgeons can now start to see and understand the very real potential for adult stem cells and tissue engineering to radically improve their ability to treat patients with serious diseases,” said Martin Birchall, who oversaw the growing of the stem cells at the University of Bristol. “We believe this success has proved that we are on the verge of a new age in surgical care.”

To contact the reporter on this story: Michelle Fay Cortez in London at mcortez@bloomberg.net

Stem cell companies awaiting sea change

By David Morrill
Staff writer, Insidebayarea.com

A cure for diabetes? New organs? Spinal injury repair?

For more than a decade Bay Area scientists and companies have thrown out grandiose possibilities that might be possible via stem cell research.

But for the better part of that time, stem cell companies hopes have been hog-tied by a Bush administration that limits funding available to research. President Bush has been opposed to the science because he feels the destruction of human embryos is immoral.

President-elect Barack Obama has made it clear that one of his first executive orders will be to reverse this policy. Companies such as Alameda-based Biotime, Menlo Park-based Geron and South San Francisco-based VistaGen finally see this as a sign that their chance to operate freely might soon be near.

“It is a fact that President Bush’s policies have had a dampening effect on research over the past years,” said Michael West, chief executive of BioTime. “Many companies, both smaller biotech and larger Pharma companies, have been reluctant to invest in the technology due to the uncertain federal policy.”

Embryonic stem cells are early-stage cells capable of being grown into hundreds of cell types that are used in the human body. Stem cells created prior to 2001 are not restricted by research funding, but only about 21 of those lines are available, most created in ways that preclude use in humans.

“I think it’s going to be a significant positive because it will open up
additional grant funding for research,” said Ralph Snodgrass, CEO of South San Francisco-based VistaGen. “The last years have been difficult and frustrating on a number of different levels.”

The biggest change would likely be that the National Institutes of Health will be able to open up additional grant funding for research.

When scientists isolated the first stem cells from a human embryo in 1998, it was heralded as a breakthrough. A decade later, no company has sought approval for a therapy using embryonic stem cells, and the $150 million “stem cell market” consists entirely of equipment used to study the technology, according to research firm TriMark Publications. Most investors have instead focused on the $300 billion U.S. drug market that has been seen as less of a risk.

It also has allowed other countries that do embrace embryonic cell research, such as China, to catch up, said Michael Werner, president of Washington, D.C.-based the Werner Group, which consults life science companies.

“Not only will this have a direct implication on the federal funding, but hopefully now any kind of stigma or cloud over the embryonic stem cell industry will be lifted,” Werner said. “This science is so valuable, and now we will really be able to pursue all these avenues of research.”

West says he knows “concrete examples” of companies and academia not doing anything in stem cell research because of fear over uncertain federal policy.

As early as last week there’s been a shift in attitudes.

Pfizer Inc., the world’s largest drugmaker, is scheduled to open a new research center this month to research stem cells to treat nervous system disorders. The second largest drug maker, GlaxoSmithKline, has put money into stem cell development as well.

At the BioTime headquarters, there is state-of-the-art equipment ready to be utilized to study the technology once funding to do so is received.

Some analysts say the new challenge for these companies will now be to get private funding from venture capital firms.

Shareholders seem to have their eyes set on stem cell companies as well.

On Monday, shares of BioTime climbed 16 percent to $2.01. Since May 19, when shares were 46 cents, it has climbed 357 percent during one of the most challenging times on Wall Street.

West says both he and his colleagues hopes Obama will set ambitious goals for science, similar to President Kennedy’s target to land a man on the moon.

“A clear signal from President-elect Obama that he will make it a priority to put in place federal policies accelerating new medical technologies to alleviate human suffering and cut the medical costs associated with the baby-boom generation age wave, could have a dramatic effect on the industry,” he said.

The Associated Press contributed to this article. Staff writer David Morrill covers biotechnology. He can be reached at dmorrill@bayareanewsgroup.com.