Stem Cells And Leukemia Battle For Marrow Microenvironment

From MedicalNewsToday.com

Learning how leukemia takes over privileged “niches” within the bone marrow is helping researchers develop treatment strategies that could protect healthy blood-forming stem cells and improve the outcomes of bone marrow transplantation for leukemia and other types of cancer.

In a paper in the journal Science, available early online Dec. 19, 2008, researchers from the University of Chicago Medical Center show that by blocking one of the chemical signals that leukemic cells release, they could help prevent the cells that mature to become red and white blood cells from being shut down by the cancerous invader.

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Patient-derived induced stem cells retain disease traits

From Genengnews.com

When neurons started dying in Clive Svendsen’s lab dishes, he couldn’t have been more pleased.

The dying cells the same type lost in patients with the devastating neurological disease spinal muscular atrophy confirmed that the University of Wisconsin-Madison stem cell biologist had recreated the hallmarks of a genetic disorder in the lab, using stem cells derived from a patient. By allowing scientists the unparalleled opportunity to watch the course of a disease unfold in a lab dish, the work marks an enormous step forward in being able to study and develop new therapies for genetic diseases.

As reported this week in the journal Nature, Svendsen and colleagues at UW-Madison and the University of Missouri-Columbia created disease-specific stem cells by genetically reprogramming skin cells from a patient with spinal muscular atrophy, or SMA. In this inherited disease, the most common genetic cause of infant mortality, a mutation leads to the death of the nerves that control skeletal muscles, causing muscle weakness, paralysis, and ultimately death, usually by age two.

Genetic reprogramming of skin cells, first reported in late 2007 by UW-Madison stem cell biologists James Thomson and Junying Yu and a Japanese group led by Shinya Yamanaka, turns back the cells’ developmental clock and returns them to an embryonic-like state from which they can become any of the body’s 220 different cell types. The resulting induced pluripotent stem cells, known as iPS cells, harness the blank-slate developmental potential of embryonic stem cells without the embryo and have been heralded as a powerful potential way to study development and disease.

Just one year later, the new work is fulfilling that promise.

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Caption: The nerves that control muscles, known as motor neurons (shown here in red), are lost in the devastating genetic disease called spinal muscular atrophy, causing weakness, paralysis, and early death. A team of UW-Madison stem cell biologists recreated the hallmarks of this disease in the lab using genetically reprogrammed stem cells created from a young SMA patient’s skin. The work gives scientists the opportunity to study the full progression of a disease in the lab and should improve understanding and treatment of genetic disorders. The motor neurons shown here were grown from cells from the patient’s healthy mother.
Photo: provided by Clive Svendsen, cnsvendsen@wisc.edu

FDA Approves Drug that Boosts Stem Cell Yield for Bone Marrow Transplants

From FDA.gov

The U.S. Food and Drug Administration today approved Mozobil (plerixafor), a drug that helps increase the number of blood stem cells for bone marrow transplantation in patients with certain forms of blood cancer.

Mozobil is intended to be used in combination with the growth factor granulocyte-colony stimulating factor (G-CSF), for treatment of adults with multiple myeloma or non-Hodgkin’s lymphomas. Multiple myeloma is cancer of the plasma cell, a cell in the bone marrow that produces antibodies to help fight infection and disease. Non-Hodgkin lymphomas are a diverse group of blood cell cancers derived from lymphocytes, a type of white blood cell.

Prior to receiving high-dose chemotherapy or radiation therapy, patients with these forms of cancer sometimes undergo a procedure known as apheresis in which blood stem cells are collected and stored for reinfusion after therapy. G-CSF is commonly administered to help release and collect stem cells from the bone marrow. Mozobil is an injectable drug that, when used in combination with G-CSF, boosts the number of stem cells released from the bone marrow into the blood stream.

“Collecting the millions of cells needed for a bone marrow transplant can take hours or days,” said Richard Pazdur, M.D., director, Office of Oncology Drug Products, Center for Drug Evaluation and Research, FDA. “Mobozil provides a new therapeutic option for patients with certain types of blood cancers by increasing the number of stem cells collected in a given time period to be reinfused after therapy.”

In two randomized clinical trials – one in patients with non-Hodgkin’s lymphoma, the other with multiple myeloma – Mozobil combined with G-CSF increased the number of stem cells available for collection and transplantation compared with patients receiving G-CSF alone.

The most commonly reported adverse reactions in these trials and other smaller studies were diarrhea, nausea, fatigue, injection site reactions, headaches, joint pain, dizziness and vomiting.

Mozobil is manufactured by Genzyme Corp., Cambridge, Mass.

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.

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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.