Funding windfall rescues abandoned stem-cell trial

NIBSC/SCIENCE PHOTO LIBRARY

Embryonic stem cells may have the ability to repair damaged tissue.

A landmark stem-cell trial is sputtering back to life two-and-a-half years after it was abandoned by the California company that started it. But it now faces a fresh set of challenges, including a field that is packed with competitors.

The trial aims to test whether cells derived from human embryonic stem cells can help nerves to regrow in cases of spinal-cord injury. It was stopped abruptly in 2011 by Geron of Menlo Park, California (see Nature 479, 459; 2011); the firm said at the time that it wanted to focus on several promising cancer treatments instead. Now, a new company Asterias Biotherapeutics, also of Menlo Park plans to resurrect the trial with a US$14.3-million grant that it received on 29May from the California Institute for Regenerative Medicine (CIRM), the states stem-cell-funding agency.

But the field has moved on since Geron treated its first patient in 2010, and the therapy that Asterias inherited is no longer the only possibility for spinal-cord injury. StemCells, a biotechnology company in Newark, California, has treated 12 patients in a safety study of a different type of stem cell, and it plans to start a more advanced trial this year to test effectiveness. And another entrant to the field, Neuralstem of Germantown, Maryland, received regulatory approval in January 2013 to begin human tests of its stem-cell product.

Gerons human trial was the first approved to use cells derived from human embryonic stem cells. But regulators halted it twice, once citing concerns about the purity and predictability of the cells being implanted, and again after the company reported seeing microscopic cysts in the spinal cords of rats that had been treated in preclinical studies. The worry was that the cysts could be teratomas uncontrolled growths that can form from embryonic stem cells, a feared side effect of treatment. Geron later said that the growths were not teratomas, and the US Food and Drug Administration allowed the trial to proceed. But after injecting the cells into five of the ten intended patients, the company said that it had run out of money for the trial.

Geron founder Michael West and former chief executive Thomas Okarma then formed Asterias, which bought Gerons stem-cell therapy last year. The company plans first to treat three patients with spinal-cord damage in the neck, using a low dose of the stem cells; it will then treat different people with higher doses to see if the therapy can restore any sensation or function in the trunk or limbs.

The five patients previously treated by Geron, whom Asterias continues to track, had cord damage at chest level. On 22May, Asterias reported that none of those five had experienced serious side effects from the treatment or developed immune responses to it.

Researchers say that the continuation of the former Geron trial is important because it uses a type of cell different from the fetus-derived ones used by StemCells and Neuralstem. Geron surgically implanted embryonic stem cells that had been coaxed in vitro to grow into immature myelinated glial cells, which insulate nerve fibres when mature. The other companies are using partially differentiated cells derived from fetal brain tissue, which might produce substances that protect surviving tissue and make new connections in the neural circuitry.

Its very good for the field, because we now have multiple cell lines being tested in very similar populations of patients, and this will help us define what is needed to make this approach work, says Martin Marsala, a neuroscientist at the University of California, San Diego, whose work has shown that Neuralstems cells can develop into working neurons and restore movement to rats with cord injuries in the neck.

View post:
Funding windfall rescues abandoned stem-cell trial

Bury cop needs 15,000 for treatment in USA

Bury cop needs 15,000 for treatment in USA

5:19pm Monday 2nd June 2014 in News By David Thomson, Reporter

A BURY police officer who suffered a devastating brain haemorrhage and stroke three years ago is trying to raise 15,000 to pay for groundbreaking stem cell treatment in America.

Gulf War veteran Joseph Duffy, aged 46, hopes that the procedure in Arizona will radically improve his mobility and help tackle a rare vascular disease which may have been responsible for his illness.

His wife Wendy, friends and relatives, are planning a series of fundraising events while the Bury-based police constable has launched his own online YouGiving page to help him reach his target.

The father-of-three joined the Army in 1986 and served in West Germany and Canada as well as in the Gulf War. He joined Greater Manchester Police in 2000.

Mr Duffy suffered a suspected heart attack in March, 2011, and a brain haemorrhage and stroke just two months later. He spent six weeks in hospital.

Mrs Duffy, aged 42, said: Joseph has severe left side weakness, struggles to walk and has been left with epilepsy. His speech gets a bit slurred when hes tired. He walks with a stick as well as a muscle stimulator, a cuff around his leg which he uses with a remote control to help his movement.

Joseph also suffers from Churgh Strauss syndrome, a rare vascular disease which attacks various parts of his body and which could have been responsible for the brain haemorrhage and stroke.

Mrs Duffy explained: We hope the treatment can be done this year. It involves liposuction and taking stem cells from the fat and putting them back into the body, to areas that have been damaged, to help repair them and to make new pathways for movement and connection.

Read this article:
Bury cop needs 15,000 for treatment in USA

Subtle change in DNA, protein levels determines blond or brunette tresses, study finds

PUBLIC RELEASE DATE:

1-Jun-2014

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center

STANFORD, Calif. A molecule critical to stem cell function plays a major role in determining human hair color, according to a study from the Stanford University School of Medicine.

The study describes for the first time the molecular basis for one of our most noticeable traits. It also outlines how tiny DNA changes can reverberate through our genome in ways that may affect evolution, migration and even human history.

"We've been trying to track down the genetic and molecular basis of naturally occurring traits such as hair and skin pigmentation in fish and humans to get insight into the general principles by which traits evolve," said David Kingsley, PhD, professor of developmental biology. "Now we find that one of the most crucial signaling molecules in mammalian development also affects hair color."

Kingsley, who is also a Howard Hughes Medical Institute investigator, is the senior author of the study, which will be published online June 1 in Nature Genetics. Research specialist Catherine Guenther, PhD, is the lead author.

The researchers found that the blond hair commonly seen in Northern Europeans is caused by a single change in the DNA that regulates the expression of a gene that encodes a protein called KITLG, also known as stem cell factor. This change affects how much KITLG is expressed in the hair follicles without changing how it's expressed in the rest of the body. Introducing the change into normally brown-haired laboratory mice yields an animal with a decidedly lighter coat not quite Norma Jeane to Marilyn Monroe, but significant nonetheless.

The study shows that even small, tissue-specific changes in the expression of genes can have noticeable morphological effects. It also emphasizes how difficult it can be to clearly connect specific DNA changes with particular clinical or phenotypic outcomes. In this case, the change is subtle: A single nucleotide called an adenine is replaced by another called a guanine on human chromosome 12. The change occurs over 350,000 nucleotides away from the KITLG gene and only alters the amount of gene expression about 20 percent a relatively tiny blip on a biological scale more often assessed in terms of gene expression being 100 percent "on" or "off."

"What we're seeing is that this regulatory region exercises exquisite control over where, and how much, KITLG expression occurs," said Kingsley. "In this case, it controls hair color. In another situation perhaps under the influence of a different regulatory region it probably controls stem cell division. Dialing up and down the expression of an essential growth factor in this manner could be a common mechanism that underlies many different traits."

Read the original here:
Subtle change in DNA, protein levels determines blond or brunette tresses, study finds

MetroMDs Advanced Regenerative Therapy Set to Counter Aging; Interesting Price-sharing Model to Make Cosmetic …

Los Angeles, CA (PRWEB) May 30, 2014

MetroMD Institute of Regenerative Medicine, a clinic that specializes in HGH and stem cell therapy, introduces an advanced cosmetic regeneration therapy that makes use of CO2 Fractional Laser System and highly efficient DOT to cure age-related skin problems, sun tanning, improve texture and laxity.

All within minimum discomfort, the Co2 Fractional Laser treatment helps in: 1.Reversing the appearance of aged and sun-damaged skin 2.Improving texture and elasticity of the skin 3.Smoothing Wrinkles 4.Reducing the acne and other scar marks

What does DOT Therapy Do?

Sun can wreak havoc to sensitive skin. Now, for ones who need to spend hours under the sun and suffer from wrinkles, discoloration, sun-spots, or lack of skin elasticity, or all of the above, the MetroMD DOT Therapy brings in a chance to alleviate individuals from all these problems. Persons with scars resulting from acne or other skin conditions and injuries can also benefit from this procedure.

One can restore skins youthful appearance with the DOT Therapy even within a one-hour sitting at the doctors office, says Dr Alex Martin, MD and the cosmetic regeneration specialist at MetroMD. While the aging process cannot be stopped, with proper care you can maintain your rejuvenated skins appearance for many years!

MetroMDs therapy is FDA approved and helps people feel younger and revitalized again. Patients seeking a cosmetic treatment in MetroMD, however, will have to go through a complete medical examination to ascertain if their body is suitable for the treatment.

Dr. Martin also said that to make the treatment accessible to more men and women, MetroMD have decided to offer the advanced cosmetic skin regeneration therapy at an exciting price. In addition, there are several charitable trusts that MetroMD has collaborated with in making their advanced therapies available to all at incredibly reduced prices!

About MetroMD

MetroMD is a prominent research institute of regenerative medicine based out of Los Angeles. The institute uses the latest medical technologies and has a highly qualified team to treat hundreds of patients who approach them for non-invasive and painless treatment. Involved in the field of cellular therapy for many years, Dr. Martins cosmetic akin and cellular regeneration therapy team renders complete pre and post treatment help.

Read more:
MetroMDs Advanced Regenerative Therapy Set to Counter Aging; Interesting Price-sharing Model to Make Cosmetic ...

Rare stem cell transplant saves life of 59-year-old

A haploidentical or half-matched stem cell transplantation has succeeded in saving the life of a 59-year-old patient with Acute Myeloid Leukemia (AML) that was resistant to all other treatments. The donor was his 25-year-old son.

The procedure was done over a month ago at the Amrita Institute of Medical Sciences here and the patient has now been stepped down from the intensive care unit.

The patient from Thiruvananthapuram had a relapse of myelodysplastic syndrome transformed into AML.

As the search for a complete match of stem cell did not yield any results, the patient was ready for the half-match procedure.

The haploidentical procedure is more complex and expensive. The procedure is not hi-tech but more care-oriented, said Dr. Neeraj Sidharthan, who led a team of experts from haematology, medical oncology, transfusion medicine and other allied departments.

The haploidentical stem cell transplantation is much more expensive and the survival rates are said to be 25-30 per cent compared to the identical stem cell transplantation that has a survival rate of 60-70 per cent.

However, the haploidentical cases were likely to grow as the chances of getting an identical match of stem cell were extremely limited, said Dr. Neeraj.

China, which has a one-child system, had started early in this process, because most of the matches were found among siblings, said Dr. Sidharthan.

Our country has just begun exploring the possibilities, he added.

There are also different methods of going about the haploidentical transplant. We chose a method that was less expensive, he added. The cost of the procedure that included importing medicines was limited to Rs.20 lakh, he said.

The rest is here:
Rare stem cell transplant saves life of 59-year-old

Results in Phase I Trial Targeting Cancer Stem Cells

At the 50th Annual Meeting of the American Society for Clinical Oncology (ASCO), University of Colorado Cancer Center researchers reported results of a Phase I trial of OMP-54F28 (FZD8-Fc), an investigational drug candidate discovered by OncoMed Pharmaceuticals targeting cancer stem cells (CSCs). The drug was generally well tolerated, and several of the 26 patients with advanced solid tumors experienced stable disease for greater than six months. Three trials are now open for OMP-54F28 (FZD8-Fc) in combinations with standard therapy for pancreatic, ovarian and liver cancers, being offered at the CU Cancer Center and elsewhere.

"These are optimistic results for one of the first targeted therapies for cancer stem cells," says Antonio Jimeno, MD, PhD, investigator at the CU Cancer Center, director of the university's Cancer Stem Cell-Directed Clinical Trials Program, and principal investigator of the clinical trial at the CU Cancer Center site. "And it is great to work with such a science-focused sponsor, whose vision aligns with ours: bringing to the clinic cutting-edge drugs and ideas that are focused on targeting CSCs. In the context of the collaboration between the Gates Center for Stem Cell Biology and the CU Cancer Center this was the second clinical trial we offered to our patients with the specific intent to eliminate the CSCs in their tumors."

OMP-54F28 (FZD8-Fc) is an antagonist of the Wnt pathway, a key CSC signaling pathway that regulates the fate of these cells. The Wnt pathway is known to be inappropriately activated in many major tumor types, including colon, breast, liver, lung and pancreatic cancers, and is critical for the function of CSCs. Because of this extensive validation, in the Jimeno lab and elsewhere, the Wnt pathway has been a major focus of anti-cancer drug discovery efforts. OMP-54F28 (FZD8-Fc) and a sister compound also developed by OncoMed, vantictumab (OMP-18R5), are two of the first therapeutic agents targeting this key pathway to enter clinical testing. In multiple preclinical models, OMP-54F28 (FZD8-Fc) has shown its effectiveness in reducing CSC populations, leading to associated anti-tumor activity, either as a single agent or when combined with chemotherapy.

"The ongoing line of work with this drug is an excellent example of the bench getting even closer to the bedside -- our lab work with the drug in patient-derived xenograft models of disease makes possible the clinical trials taking place at the University of Colorado Hospital next door," Jimeno says.

The Phase I clinical trial of OMP-54F28 (FZD8-Fc) is an open-label dose escalation study in patients with advanced solid tumors for which there was no remaining standard curative therapy. Patients are assessed for safety, immunogenicity, pharmacokinetics, biomarkers, and initial signals of efficacy. The trial is conducted at Pinnacle Oncology Hematology in Scottsdale, Arizona, the University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, and the CU Cancer Center under the direction of Principal Investigators Dr. Michael S. Gordon, Dr. David Smith and Dr. Antonio Jimeno, respectively.

The most common adverse events, mild to moderate and manageable, included dysgeusia (altered taste), fatigue, muscle spasms, decreased appetite, alopecia and nausea. One related Grade 3 or greater adverse event of Grade 3 increased blood phosphorus was reported. One moderate sacral insufficiency fracture occurred in one patient at the highest tested dose of 20 mg/kg every three weeks after 6 cycles.

"The drug is now being developed in combination with standard of care in three Phase 1b clinical trials, with the CU Cancer Center being one of the active sites," Jimeno says. "In pancreatic, ovarian and liver cancers, we hope that by adding anti-cancer stem cell drugs to standard of care, we can control proliferating cells within the tumor that could otherwise help the tumor regenerate in the face of existing chemotherapies."

Story Source:

The above story is based on materials provided by University of Colorado Cancer Center. Note: Materials may be edited for content and length.

Originally posted here:
Results in Phase I Trial Targeting Cancer Stem Cells

Results in Phase I trial of OMP-54F28, a Wnt inhibitor targeting cancer stem cells

PUBLIC RELEASE DATE:

31-May-2014

Contact: Erika Matich erika.matich@ucdenver.edu 303-524-2780 University of Colorado Denver

At the 50th Annual Meeting of the American Society for Clinical Oncology (ASCO), University of Colorado Cancer Center researchers reported results of a Phase I trial of OMP-54F28 (FZD8-Fc), an investigational drug candidate discovered by OncoMed Pharmaceuticals targeting cancer stem cells (CSCs). The drug was generally well tolerated, and several of the 26 patients with advanced solid tumors experienced stable disease for greater than six months. Three trials are now open for OMP-54F28 (FZD8-Fc) in combinations with standard therapy for pancreatic, ovarian and liver cancers, being offered at the CU Cancer Center and elsewhere.

"These are optimistic results for one of the first targeted therapies for cancer stem cells," says Antonio Jimeno, MD, PhD, investigator at the CU Cancer Center, director of the university's Cancer Stem Cell-Directed Clinical Trials Program, and principal investigator of the clinical trial at the CU Cancer Center site. "And it is great to work with such a science-focused sponsor, whose vision aligns with ours: bringing to the clinic cutting-edge drugs and ideas that are focused on targeting CSCs. In the context of the collaboration between the Gates Center for Stem Cell Biology and the CU Cancer Center this was the second clinical trial we offered to our patients with the specific intent to eliminate the CSCs in their tumors."

OMP-54F28 (FZD8-Fc) is an antagonist of the Wnt pathway, a key CSC signaling pathway that regulates the fate of these cells. The Wnt pathway is known to be inappropriately activated in many major tumor types, including colon, breast, liver, lung and pancreatic cancers, and is critical for the function of CSCs. Because of this extensive validation, in the Jimeno lab and elsewhere, the Wnt pathway has been a major focus of anti-cancer drug discovery efforts. OMP-54F28 (FZD8-Fc) and a sister compound also developed by OncoMed, vantictumab (OMP-18R5), are two of the first therapeutic agents targeting this key pathway to enter clinical testing. In multiple preclinical models, OMP-54F28 (FZD8-Fc) has shown its effectiveness in reducing CSC populations, leading to associated anti-tumor activity, either as a single agent or when combined with chemotherapy.

"The ongoing line of work with this drug is an excellent example of the bench getting even closer to the bedside our lab work with the drug in patient-derived xenograft models of disease makes possible the clinical trials taking place at the University of Colorado Hospital next door," Jimeno says.

The Phase I clinical trial of OMP-54F28 (FZD8-Fc) is an open-label dose escalation study in patients with advanced solid tumors for which there was no remaining standard curative therapy. Patients are assessed for safety, immunogenicity, pharmacokinetics, biomarkers, and initial signals of efficacy. The trial is conducted at Pinnacle Oncology Hematology in Scottsdale, Arizona, the University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, and the CU Cancer Center under the direction of Principal Investigators Dr. Michael S. Gordon, Dr. David Smith and Dr. Antonio Jimeno, respectively.

The most common adverse events, mild to moderate and manageable, included dysgeusia (altered taste), fatigue, muscle spasms, decreased appetite, alopecia and nausea. One related Grade 3 or greater adverse event of Grade 3 increased blood phosphorus was reported. One moderate sacral insufficiency fracture occurred in one patient at the highest tested dose of 20 mg/kg every three weeks after 6 cycles.

"The drug is now being developed in combination with standard of care in three Phase 1b clinical trials, with the CU Cancer Center being one of the active sites," Jimeno says. "In pancreatic, ovarian and liver cancers, we hope that by adding anti-cancer stem cell drugs to standard of care, we can control proliferating cells within the tumor that could otherwise help the tumor regenerate in the face of existing chemotherapies."

Continued here:
Results in Phase I trial of OMP-54F28, a Wnt inhibitor targeting cancer stem cells

Global Stem Cells Group to Host the First International Symposium on Stem Cells and Regenerative Medicine in Buenos …

Miami (PRWEB) May 30, 2014

GlobalStemCellsGroup.com will host the First International Symposium on Stem Cell Research in Buenos Aires, Argentina Oct. 2, 3 and 4. The symposium will provide an opportunity to showcase advancements in stem cell research and therapies on a global level and establish a dialogue among the worlds leading stem cell experts. Pioneers and luminaries in stem cell medicine will be featured speakers as well as accomplished guests prepared to share their knowledge and experience in their individual medical specialties.

Regenerative medicine as a field is still in its infancy, and Global Stem Cells Group President and CEO Benito Novas believes it is time to clear up old misconceptions and change outdated attitudes by educating people on the wide range of illnesses and injuries stem cell therapies are already treating and curing. The first step, Novas says, is establishing a dialogue between researchers and practitioners in order to move stem cell therapies from the lab to the physicians office.

Our objective is to open a dialogue among the worlds medical and scientific communities in order to advance stem cell technologies and translate them into point-of-care medical practices, Novas says. Our mission is to bring the benefits of stem cell therapies to the physicians office for the benefit and convenience of the patient, safely and in full compliance with the highest standard of care the world has to offer.

An interdisciplinary team of leading international stem cell experts will provide a full day of high-level scientific lectures aimed at medical professionals.

Among the growing list of speakers are some of the worlds most prominent authorities on stem cell medicine including:

The objective of Global Stem Cell Groups international symposium is to educate the public and the medical community, and at the same time establish a dialog between physicians, scientists, biotech companies and regulatory agencies in order to advance stem cell technologies so they can be used to benefit people who need them.

Global Stem Cells Group is also joining forces with some of the most prestigious regenerative medicine conferences in South America including:

Stem cell therapies are revolutionizing the anti-aging aesthetics industry while offering new hope for sufferers of serious chronic debilitating diseases

For more information on the Global Stem Cell Group First International Symposium on Stem Cells and Regenerative Medicine and the events lineup of speakers, visit the Global Stem Cells Symposium website, email bnovas(at)regenestem(dot)com, or call 305-224-1858.

See more here:
Global Stem Cells Group to Host the First International Symposium on Stem Cells and Regenerative Medicine in Buenos ...