Author Archives: admin


Stem Cell Clinic > Articles by: admin

BioMarin Licenses Factor VIII Gene Therapy … – Stem Cell Cafe

By Stell Cell Research

SAN RAFAEL, Calif., Feb. 21, 2013 (GLOBE NEWSWIRE) BioMarin Pharmaceutical Inc. (BMRN) announced today that it has licensed a Factor VIII gene therapy program for hemophilia A from University College London (UCL) and St. Jude Childrens Research Hospital. The company expects to select a development candidate this year, initiate and complete IND-enabling toxicology studies next year and initiate proof of concept human studies by the end of 2014. The license and commitment to support the research program was made possible by UCL Business, UCLs wholly-owned technology transfer company, working with Professor Amit Nathwani of the UCL Cancer Institute.

Gene therapy is emerging as a powerful and viable way to treat genetic disorders and is complementary to our current suite of commercial products and research programs, said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. Hemophilia is an attractive target for gene therapy as factor levels in the blood serve as good biomarkers, relatively low factor levels are required for a clinically important benefit in severe patients and the current standard of care of intravenous infusions three times a week is quite onerous. We remain committed to maintaining a rich pipeline with the goal of filing an IND every twelve to eighteen months.

Mr. Cengiz Tarhan, Managing Director of UCL Business said, This is an excellent partnership for UCL Business, which combines the world class translational research strengths of Professor Nathwani and his team with the significant development and commercialization capabilities of BioMarin to progress this ground breaking therapy for hemophilia A.

Professor Stephen Caddick, Vice-Provost (Enterprise) at University College London added, UCL and BioMarin each bring distinct strengths to the partnership. UCL is a world leader in the biomedical sciences, with an unremitting commitment to outstanding research and translation into healthcare benefits for patients. We welcome this partnership which will continue to build on the excellence of our research to fully explore the potential of gene therapy as a life-saving treatment for people with hemophilia.

Andrew Davidoff, M.D., Chair, Surgery, St. Jude Childrens Research Hospital, added, We are pleased that our research with UCL on gene therapy for hemophilia has led to the development of a potential therapeutic tool for treating this devastating disease. This licensing agreement underscores St. Judes commitment to rapidly translating our research into effective clinical interventions.

About Hemophilia A

The current market for hemophilia A products is about $6.0 billion worldwide. There are approximately 90,000 patients in territories where BioMarin has commercial operations and an annual incidence of about 400 new patients in the U.S. The standard of care for the 60 percent of hemophilia A patients who are severe is a prophylactic regimen of IV infusions three times per week. Even with the likely prospect of less frequently dosed products coming to the market, feedback from thought leaders indicates that significant unmet need will remain as factor replacement therapy will inevitably leave patients vulnerable to bleeding events. Many patients on factor replacement therapy still have bleeding events and experience debilitating damage to joints as a result of chronically low factor levels.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The companys product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include BMN-110 (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit http://www.BMRN.com. Information on BioMarins website is not incorporated by reference into this press release.

The BioMarin Pharmaceutical Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=11419

See more here:
BioMarin Licenses Factor VIII Gene Therapy ... - Stem Cell Cafe

Egg cells from Stell Cells: A breakthrough in fertility research | Bionic …

By Stell Cell Research

Its one of the basic truths about biology were all thought in high school: while men can produce a nearly infinite amount of sperm cells throughout their lifetime, women are born with a set number of egg cells and once they run out, well then thats that. Recent research by Jonathan Tilly of the Massachusetts General Hospital in Boston however, has suggested that egg cells can develop from a certain kind of egg-producing stem cells in mice.

Apart from rustling the fundaments of developmental biology, this discovery may also have very useful application in fertility treatments for women. Kutluk Oktay, reproductive biologist at the New York Medical College in Valhalla, compares these findings with discovering a planet in our solar system with a bacterium on it.

Like with many breakthroughs in science, it was not the original goal of the researches to overturn an established dogma. They just found something that didnt seem to stroke with the general consensus and decided to investigate further.

As mentioned before, the presence of egg producing stem cells was only proven in mice at first, and not a lot of people believed that the same kind of cells were also present in humans. Jonathan Tilly however, has shown that some stem cells are present in the ovaries of women, but it still remains to be seen if these stem cells can actually develop in fully functional reproductive cells. Tully remains positive however, that he can somehow control the biological clock.

Full article from Nature Medicine

Like Loading...

.

View post:
Egg cells from Stell Cells: A breakthrough in fertility research | Bionic ...

Jose “Joecon” Concepcion Jr: ‘I’m now a believer in stem-cell therapy. The proof is me!’

By Uncategorized

By Cheche V. Moral Philippine Daily Inquirer

I DID it because it worked on my wifes cousin, and because of what I read about it, says Joe Concepcion of his stem-cell therapy. ALANAH TORRALBA

Last of two parts

Jose S. Concepcion Jr., at 81, still goes to the office daily, and, to his wifes chagrin, is quite restless and bullheaded, sour if he has nothing to keep himself busy.

He does crazy things! Like, if he gets hold of a book, hell bring it and photocopy everything, says Marivic Concepcion, who has been married for 56 years to the RFM Corp. chair, Namfrel (National Citizens Movement for Free Election) founder and former trade secretary. He needs a caregiver but he wont allow it. He could fall!

Her husband, however, insists he feels excellent. I feel so good when I wake up. I dont feel tired, says the activist known to many as Joecon.

Concepcion attributes this renewed strength and vigor to a procedure he had undergone not long ago, and of which he has been a vocal and unpaid advocate: stem-cell therapy.

Astonishing effects

The tycoon received his treatment from a German doctor named Christoph Ganss, who comes to the Philippines every three or four months to speak about his company and the benefits of stem-cell banking, according to the Concepcions. Ganss is the founder of Ticeba (Tissue & Cell Banking), a tissue bank for stem cells and laboratory in Heidelberg.

The couple heard of Dr. Ganss from Ms Concepcions cousin who underwent the treatment in Germany.

Visit link:
Jose “Joecon” Concepcion Jr: ‘I’m now a believer in stem-cell therapy. The proof is me!’

Philippine-based group warns on stem cell medicine

By Stem Cell Treatment

Manila: A Philippine-based group has aired concern over the dangers of stem cell treatment as it warned of the possibility that materials being used for such procedures could have been extracted from hapless donors, particularly human foetuses.

Dr Leo Olarte, spokesman of the Philippine Society for Stem Cell Medicine, said they were alarmed over reports coming locally and from abroad that some stem cell materials were being taken from the unborn.

At the same time he called on the Department of Health to carefully watch stem cell treatment practitioners. The Catholic Church, he said, should unite with health practitioners and put an end to such practices.

Olarte was quoted as saying in reports that allogenic stem cells from aborted foetuses of humans were being exported to the Philippines.

Article continues below

We cannot stand by and merely watch how they exploit people in poverty just to profit while allowing others who are economically well off to benefit from this, he said.

He said there were reports that human stem cells from foetuses and female egg cells were being used in anti-ageing procedures and other medical treatments.

Olarte is also concerned over the sale of supposedly stem cell products that come in the form of injectibles and even soaps.

Dont patronise those products. An example is the stem cell soap. It is unfair that stem cell therapy becomes a quackery, he said.

Earlier, Department of Health Secretary Enrique Ona led a national convention participated in by doctors to discuss the truth behind stem cell therapy.

Visit link:
Philippine-based group warns on stem cell medicine

Cebu Seen As A Major Market For Stem Cell Treatment

By Stem Cell Treatment

CEBU CITY, Cebu - Banking on the popularity of stem cell therapy to enhance a person's general well-being, a Manila-based aesthetic clinic is bent on capturing a huge market in Cebu with its own product line.

Flawless Face and Body Center (Flawless) Chief Executive Officer Rubby Sy said his company will soon launch its own human stem cell therapy following the success of their stem cell product line, from fruit stem cell introduced in the market last December.

''Stem-cell technology is gaining huge popularity in the Philippines and we see Cebu as a big market for this innovation,'' Sy said in a recent press interview here.

Sy said her company intends to offer human stem cell treatment through intravenous and intra-dermal administration, costing some $25,000 and $6,500, respectively.

He disclosed that Flawless has partnered with a medical institute in Russia for human stem cell supply.

''This partner institution works like a blood bank that conducts stringent screening on human stem cells extracted from Russians aged 20 to 25 years old,'' she said.

Sy is confident that stem cell treatment would be widely accepted here given its medical and beauty benefits. Customers, she said, have accepted the product because of its ''immediate effects.''

For his part, Drew Mendoza, the firm's business manager, said that Flawless' stem cell product line is the only stem cell product approved by the Food and Drugs Administration (FDA) and is the first in the Philippine market.

See the rest here:
Cebu Seen As A Major Market For Stem Cell Treatment

Schistosome stem cells could explain how the worms survive for so …

By Stell Cell Research

The parasites that cause schistosomiasis, one of the most common parasitic infections in the world, are notoriously long-lived. Researchers have now found stem cells inside the parasite that can regenerate worn-down organs, which may help explain how they can live for years or even decades inside their host.

Schistosomiasis is acquired when people come into contact with water infested with the larval form of the parasitic worm Schistosoma, known as schistosomes. Schistosomes mature in the body and lay eggs that cause inflammation and chronic illness. Schistosomes typically live for five to six years, but there have been reports of patients who still harbor parasites decades after infection.

According to new research from Howard Hughes Medical Institute (HHMI) investigator Phillip Newmark, collections of stem cells that can help repair the worms bodies as they age could explain how the worms survive for so many years. The new findings were published online on February 20, 2013, in the journal Nature.

The stem cells that Newmarks team found closely resemble stem cells in planaria, free-living relatives of the parasitic worms. Planaria rely on these cells, called neoblasts, to regenerate lost body parts. Whereas most adult stem cells in mammals have a limited set of possible fatesblood stem cells can give rise only to various types of blood cells, for example planarian neoblasts can turn into any cell in the worms body under the right circumstances.

Newmarks lab at the University of Illinois at Urbana-Champaign has spent years focused on planaria, so they knew many details about planarian neoblasts what they look like, what genes they express, and how they proliferate. They also knew that in uninjured planarians, neoblasts maintain tissues that undergo normal wear and tear over the worms lifetime.

We began to wonder whether schistosomes have equivalent cells and whether such cells could be partially responsible for their longevity, says Newmark.

Following this hunch, and using what they knew about planarian neoblasts, post-doctoral fellow Jim Collins, Newmark, and their colleagues hunted for similar cells in Schistosoma mansoni, the most widespread species of human-infecting schistosomes.

Their first step was to look for actively dividing cells in the parasites. To do this, they grew worms in culture and added tags that would label newly replicated DNA as cells prepare to divide; this label could later be visualized by fluorescence. Following this fluorescent tag, they saw a collection of proliferating cells inside the worms body, separate from any organs.

The researchers isolated those cells from the schistosomes and studied them individually. They looked like typical stem cells, filled with a large nucleus and a small amount of cytoplasm that left little room for any cell-type-specific functionality. Newmarks lab observed the cells and found that they often divided to give rise to two different cells: one cell that continued dividing, and another cell that did not.

One feature of stem cells, says Newmark, is that they make more stem cells; furthermore, many stem cells undergo asymmetric division. The schistosomes cells were behaving like stem cells in these respects. The other characteristic of stem cells is that they can differentiate into other cell types.

See original here:
Schistosome stem cells could explain how the worms survive for so ...

First ever double stem cell therapy procedure to be performed by Branchburg veterinarian

By Uncategorized

Now, thanks to participating veterinarians like Dr. Karen Derfuss, DVM, from Branchburg Animal Hospital, who will be conducting a new double stem cell therapy procedure on Tuesday, Feb. 26, and the procedure itself, developed by MediVet America, a new kind of treatment is available for dogs, Auggie and Chloe, and other beloved pets in the Branchburg area.

This new procedure marks a major milestone in regenerative veterinary medicine, said MediVet-America spokesman, Dr. Mike Hutchinson, D.V.M., a leading practitioner in stem cell therapy who has performed more than 400 adipose-derived stem cell treatments on dogs and cats. MediVets superior results made me a believer, and Im excited to share this news with my colleagues.

The procedure uses adult animal stem cell technology and the bodys own regenerative healing power. No embryonic stem cells are used. MediVets kit and equipment enable veterinarians to remove a sample of fat from the pet, separate stem cells from the fat, then activate and inject the cells into the affected area. The entire procedure is all done in-clinic and on the same day.

Within about one month, most animals can be removed from pain and anti-inflammatory drugs. Results show animals that had been unable to climb stairs or jump, now are walking, running and playing.

MediVet is a global leader in veterinary science, with over 1,000 clinics practicing this incredible technology in the US, Mexico, Canada and 28 other countries

About the two dogs:

Auggie is a nine year old loving beagle and a Fathers Day Gift for dad. He is suffering from hip dysplasia, which is an abnormal formation of the hip socket that, in its more severe form, can eventually cause crippling lameness and painful arthritis of the joints. Derfuss is the only doctor in the area who has been certified by MediVet America to perform this double procedure. The stem cell procedure is an affordable and non-invasive alternative to FHO (femoral head osteopathy) surgery, which was originally planned for Auggie.

Chloe is a beautiful 11 year old golden retriever who suffers from bilateral arthritic hips and long term inflammation issues. It was decided to use this new technology to treat Chloes ailments as an alternative to expensive and invasive hip replacement surgery.

Go here to see the original:
First ever double stem cell therapy procedure to be performed by Branchburg veterinarian

Stem cell heart repairs: 21st century medicine in action

By Stem Cell Treatment

Gerard Cuomo loves to dance.

Until recently, however, the 70-year-old couldnt even do a two-step.

After having three heart attacks in the early 1990s, Cuomos heart was severely damaged. The scar tissue that had formed around his heart left him easily fatigued.

I felt like an old man, said Cuomo of Aventura. I could barely climb the stairs. I could walk for about a quarter of a mile. Shopping at the mall I wish I did not have to sit down all the time.

In May 2010, he participated in a University of Miami Miller School of Medicines clinical trial in which doctors injected stem cells directly into his heart muscle. The stem cells, because they are not fully formed, have the potential to grow into different kinds of cells, internalizing information from their environment to determine their future growth.

The study found that the injections built up the healthy heart tissue and reduced scar tissue by 33 percent a dramatic improvement, said Dr. Joshua Hare, director of UMs Interdisciplinary Stem Cell Institute, which conducted the study with Johns Hopkins University. The new tissue remodeled the heart to look more like a healthy, football-shaped heart.

This is a real example of 21st century medicine, said Hare, the Louis Lemberg Professor of Medicine in the Cardiovascular Division. Without doing any specific manipulation, we didnt coax them, they knew where to go. They work in ways that make a lot of sense.

The results of the study, funded by the National Institutes of Health, were published in November by the Journal of the American Medical Association. Half of the 30 men enrolled in the study received injections of their own stem cells, while the other half got stem cells from a third-party donor.

Using a donor is a huge convenience factor, Hare said. We can store large quantities of the stem cells in a cell bank and use them whenever the need arises.

Cuomos stem cells were extracted from his bone marrow. He had to wait about six weeks after extraction to have them re-injected into his heart. During that time, doctors cultivated and tested the cells.

See the original post:
Stem cell heart repairs: 21st century medicine in action

Florida Hospital Pepin Heart Institute, USF partner … – Stem Cell Cafe

By Stell Cell Research

Feb 23

Cardiovascular disease specialists at Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute affiliated with the University of South Florida announced they have enrolled their first patients into a clinical trial testing a novel gene therapy for the treatment of heart failure after ischemic injury. The therapy may promote the regeneration of heart tissue by encouraging the body to deploy more stem cells to the injury site.

Dr. Charles Lambert, Medical Director of Florida Hospital Pepin Heart Institute and Dr. Leslie Miller, Director of the USF Heart Institute, are leading the way for the randomized, placebo-controlled trial which spans 10 sites across the United States. The study, called the STOP-HF, will enroll 90 patients nationwide.

Heart failure (HF) can occur when the muscles of the heart become weakened and cannot pump blood sufficiently throughout the body. The injury is most often caused by inadequate blood flow to the heart resulting from chronic or acute cardiovascular disease, including heart attacks. Considerable scientific evidence has emerged over the past decade demonstrating the high therapeutic potential of regenerative medicine for a host of diseases. Heart failure is a leading cause of death, disability and hospitalization.

Dr. Charles Lambert is performing the gene therapy by direct injection into the heart using an investigational system in the catheterization laboratories at Florida Hospital Pepin Heart Institute.

Pepin Heart and Dr. Kiran C. Patel Research Institute and USF are exploring and conducting leading-edge research to develop break-through treatments long before they are even available in other facilities, Dr. Lambert said. Stem cells have the unique ability to develop into many different cell types, and in many tissues serve as an internal repair system, dividing essentially without limit to replenish other cells. This trial is unique in that it uses gene therapy to turn on a process leading to cell regeneration rather than simply administering stem cells directly.

The Pepin Heart Institute has a history of cardiovascular stem cell research as part of the NIH sponsored Cardiac Cell Therapy Research Network (CCTRN) as well as other active cell therapy trials. Locally, the STOP-HF trial is the first of several regenerative medicine clinical trials teaming the USF Heart Institute with Florida Hospital Pepin Heart Institute, which is adjacent to the USF Health campus.

Follow this link: Florida Hospital Pepin Heart Institute, USF partner for novel gene therapy trial

More here:
Florida Hospital Pepin Heart Institute, USF partner ... - Stem Cell Cafe

Cell therapy a little more concrete thanks to VIB research

By Uncategorized

Public release date: 21-Feb-2013 [ | E-mail | Share ]

Contact: Sooike Stoops sooike.stoops@vib.be 32-924-46611 VIB (the Flanders Institute for Biotechnology)

Cell therapy is a promising alternative to tissue and organ transplantation for diseases that are caused by death or poor functioning of cells. Considering the ethical discussions surrounding human embryonic stem cells, a lot is expected of the so-called 'induced pluripotent stem cells' (iPS cells). However, before this technique can be applied effectively, a lot of research is required into the safety and efficacy of such iPS cells. VIB scientists associated to the UGent have developed a mouse model that can advance this research to the next step.

Lieven Haenebalcke (VIB/UGent): "iPS cells have enormous therapeutic potential, but require more thorough testing before they can be used for such purposes. Using our new mouse model, we can study which mechanisms determine the identity of a cell. This knowledge is essential before we can use cell therapy for regenerative medicine."

Jody Haigh (VIB/UGent): "If we want to give cell therapy a future, then we must continue this type of research and invest in the further development of such technologies. This will result in an improved insight into cellular identity and in the long term safer options of applying iPS cells or cells derived from iPS cells in clinical studies."

Cell therapy replacing cells to provide a cure

Cell therapy is the replacement of lost or poorly functioning cells in patients. For example, such cell therapies could be used to repair the heart muscle after a heart attack, joints affected by arthritis, the pancreas in diabetes or the spine in certain forms of paralysis. This requires cells that are able to multiply in the laboratory and that can be converted to healthy cells of the desired cell type. Human embryonic stem cells meet these criteria, but they are ethically controversial.

iPS cells a promising alternative to embryonic stem cells

Shinya Yamanaka recently developed a fairly simple method to reprogram differentiated cells such as skin cells back to stem cells, so-called "induced pluripotent stem cells" (iPS cells). This earned him the Nobel Prize for Medicine in 2012 (shared with John Gurdon). These iPS cells can be generated using only 4 "reprogramming factors".

As is the case with embryonic stem cells, these iPS cells can be used to produce other cell types, such as heart muscle cells or nerve cells. They can also be cultured indefinitely and there are no ethical objections as they are not obtained from human embryos left over after IVF, but from adult individuals. Furthermore, iPS cells are obtained from the patient and this reduces the risk of rejection during therapeutic applications.

Go here to see the original:
Cell therapy a little more concrete thanks to VIB research

→←