Posts Tagged ‘disease’

First UK patient receives stem cell treatment to cure …

Stem Cell Treatment | Posted by admin
Sep 28 2015

Vision loss is as common a problem as dementia among older people. Photograph: John Stillwell/PA

A patient has become the first in the UK to receive an experimental stem cell treatment that has the potential to save the sight of hundreds of thousands of Britons.

By December, doctors will know whether the woman, who has age-related macular degeneration, has regained her sight after a successful operation at Moorfields Eye Hospital in London last month. Over 18 months, 10 patients will undergo the treatment.

The transplant involves eye cells, called retinal pigment epithelium, derived from stem cells and grown in the lab to form a patch that can be placed behind the retina during surgery.

Related: Stem cell therapy success in treatment of sight loss from macular degeneration

The potential is huge. Although the first patients have the wet form of macular degeneration, the doctors believe it might also eventually work for those who have the dry form, who are the vast majority of the UKs 700,000 sufferers.

The surgery is an exciting moment for the 10-year-old London Project to Cure Blindness, a collaboration between the hospital, the UCL Institute of Ophthalmology and the National Institute for Health Research, which was formed to find a cure for wet age-related macular degeneration, the more serious but less common form of the disease.

Prof Pete Coffey of UCL, one of the founders of the London Project, said he would not be working on the new treatment if he did not believe it would work. He hopes it could become a routine procedure for people afflicted by vision loss, which is as common a problem among older people as dementia.

It does involve an operation, but were trying to make it as straightforward as a cataract operation, he said. It will probably take 45 minutes to an hour. We could treat a substantial number of those patients.

First they have to get approval. The trial is not just about safety, but also efficacy. There will be a regulatory review after the first few transplants to ensure all is going well.

The group of patients chosen have the wet form of the disease and experienced sudden loss of vision within about six weeks. The support cells in the eye, which get rid of daily debris and allow the seeing part to function have died.

There is a possibility of restoring their vision, said Coffey. The aim of the transplant is to restore the support cells so the seeing part of the eye is not affected by what would become an increasingly toxic environment, causing deterioration and serious vision loss. The surgery is being performed by retinal surgeon Prof Lyndon Da Cruz from Moorfields, who is also a co-founder of the London Project.

The team chose people with this dramatic vision loss to see whether the experimental stem cell therapy would reverse the loss of vision. But in those with dry macular degeneration, said Coffey, the process is far slower, which would mean doctors could choose the time to intervene if the treatment works.

Helping people to regain their sight has long been one of the most hopeful prospects for stem cell transplantation. Other research groups have been trialling the use of stem cells in people with Stargardts disease, which destroys the vision at a much earlier age.

Stem cells have moved from the drawing board into human trials with incredible speed, scientists say. The first embryonic stem cell was derived in 1989. Using them in eyes was always going to have a big advantage over other prospects, because it is possible to transplant them without an all-out attack by the immune system, as would happen in other parts of the body. Most people who have any sort of transplant have to take drugs that suppress the immune system for the rest of their lives.

Just like conventional medicines, stem cell therapies will very likely have to be developed and marketed by large commercial concerns. The London Project has the US drug company Pfizer on board.

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Induced pluripotent stem cell therapy - Wikipedia, the …

Induced Pluripotent Stem Cells | Posted by admin
Aug 03 2015

In 2006, Shinya Yamanaka of Kyoto University in Japan was the first to disprove the previous notion that reversible cell differentiation of mammals was impossible. He reprogrammed a fully differentiated mouse cell into a pluripotent stem cell by introducing four genes, Oct-4, SOX2, KLF4, and Myc, into the mouse fibroblast through gene-carrying viruses. With this method, he and his coworkers created induced pluripotent stem cells (iPS cells), the key component in this experiment.[1] Scientists have been able to conduct experiments that show the ability of iPS cells to treat and even cure diseases. In this experiment, tests were run on mice with inherited sickle cell anemia.Skin cells were turned into cells containing genes that transformed the cells into iPS cells. These replaced the diseased sickled cells, curing the test mice. The reprogramming of the pluripotent stem cells in mice was successfully duplicated with human pluripotent stem cells within about a year of the experiment on the mice.

Sickle cell anemia is a disease in which the body produces abnormally shaped red blood cells. Red blood cells are flexible and round, moving easily through the blood vessels. Infected cells are shaped like a crescent or sickle (the namesake of the disease). As a result of this disorder the hemoglobin protein in red blood cells is faulty. Normal hemoglobin bonds to oxygen, then releases it into cells that need it. The blood cell retains its original form and is cycled back to the lungs and re-oxygenated.

Sickle cell hemoglobin, however, after giving up oxygen, cling together and make the red blood cell stiff. The sickle shape also makes it difficult for the red blood cell to navigate arteries and causes blockages.[2] This can cause intense pain and organ damage. The sickled red blood cells are fragile and prone to rupture. When the number of red blood cells decreases from rupture (hemolysis), anemia is the result. Sickle cells also die in 1020 days as opposed to the traditional 120-day lifespan of a normal red blood cell.

Sickle cell anemia is inherited as an autosomal (meaning that the gene is not linked to a sex chromosome) recessive condition.[2] This means that the gene can be passed on from a carrier to his or her children. In order for sickle cell anemia to affect a person, the gene must be inherited from both the mother and the father, so that the child has two recessive sickle cell genes (a homozygous inheritance). People who inherit one sickle cell gene from one parent and one normal gene from the other parent, i.e. heterozygous patients, have a condition called sickle cell trait. Their bodies make both sickle hemoglobin and normal hemoglobin. They may pass the trait on to their children.

The effects of sickle cell anemia vary from person to person. People who have the disease suffer from varying degrees of chronic pain and fatigue. With proper care and treatment, the quality of health of most patients will improve. Doctors have learned a great deal about sickle cell anemia since its discovery in 1979. They know its causes, its effects on the body, and possible treatments for complications. Sickle cell anemia has no widely available cure. A bone marrow transplant is the only treatment method currently recognized to be able to cure the disease, though it does not work for every patient. Finding a donor is difficult and the procedure could potentially do more harm than good. Treatments for sickle cell anemia are generally aimed at avoiding crises, relieving symptoms, and preventing complications. Such treatments may include medications, blood transfusions, and supplemental oxygen.

During the first step of the experiment, skin cells (also known as fibroblasts) were collected from infected test mice and put in a culture. The fibroblasts were reprogrammed by infecting them with retroviruses that contained genes common to embryonic stem cells. These genes were the same four used by Yamanaka (Oct-4, SOX2, KLF4, and Myc) in his earlier study. The investigators were trying to produce cells with the potential to differentiate into any type of cell needed (i.e. pluripotent stem cells). As the experiment continued, the fibroblasts multiplied into identical copies of iPS cells. The cells were then treated to form the mutation needed to reverse the anemia in the mice. This was accomplished by restructuring the DNA containing the defective globin gene into DNA with the normal gene through the process of homologous recombination. The iPS cells then differentiated into blood stem cells, or hematopoietic stem cells. The hematopoietic cells were injected back into the infected mice, where they proliferate and differentiate into normal blood cells, curing the mice of the disease.[3][4][verification needed]

To determine whether the mice were cured from the disease, the scientists checked for the usual symptoms of sickle cell disease. They examined the blood for mean corpuscular volume (MCV) and red cell distribution width (RDW) and urine concentration defects. They also checked for sickled red blood cells. They examined the DNA through gel electrophoresis, checking for bands that display an allele that causes sickling. Compared to the untreated mice with the disease, which they used as a control, the treated animals had marked increases in RBC counts, healthy hemoglobin, and packed cell volume levels.[5]

Researchers examined the urine concentration defect, which results from RBC sickling in renal tubules and consequent reduction in renal medullary blood flow, and the general deteriorated systemic condition reflected by lower body weight and increased breathing.[5] They were able to see that these parts of the body of the mice had healed or improved. This indicated that all hematological and systemic parameters of sickle cell anemia improved substantially and were comparable to those in control mice.[5] They cannot say if this will work in humans because a safe way to inject the genes for the induced pluripotent cells is still needed.[citation needed]

The reprogramming of the induced pluripotent stem cells in mice was successfully duplicated in humans within a year of the successful experiment on the mice. This reprogramming was done in several labs and it was shown that the iPS cells in humans were almost identical to original embryonic stem cells (ES cells) that are responsible for the creation of all structures in a fetus.[1] An important feature of iPS cells is that they can be generated with cells taken from an adult, which would circumvent many of the ethical problems associated with working with ES cells. These iPS cells also have potential in creating and examining new disease models and developing more efficient drug treatments.[6] Another feature of these cells is that they provide researchers with a human cell sample, as opposed to simply using an animal with similar DNA, for drug testing.

One major problem with iPS cells is the way in which the cells are reprogrammed. Using gene-carrying viruses has the potential to cause iPS cells to develop into cancerous cells.[1] Also, an implant made using undifferentiated iPS cells, could cause a teratoma to form. Any implant that is generated from using these iPS cells would only be viable for transplant into the original subject that the cells were taken from. In order for these iPS cells to become viable in therapeutic use, there are still many steps that must be taken.[5][7]

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Charcot-Marie-Tooth Disease - Wu Medical Center - A …

Stem Cell Medical Center | Posted by admin
Jun 04 2015

Stem Cell Therapy for Charcot-Marie-Tooth DiseaseMay 26th, 2015

By Like Wu, Xiaojuan Wang, Sherry Xi, Bo Cheng, Susan Chu

Wu Medical Center

The patient is 34-year-old female. She was presented with involuntary movement of fingers and extremities prior weakness for the past 13 years without any apparent cause. The disease progressed gradually. She had nerve biopsy and gene analysis at a local hospital and was diagnosed with Charcot-Marie-Tooth disease (CMT). She took vitamins without any improvement. Her four limbs were weak. Her balance was bad. She always falls because of bad balance and lower limbs weakness. She had difficulty to hold objects. The distal ends of four limbs were depauperated, numbness and painful.

Physical examination: The general examination was normal. Her speech and spirit was good. The cranial nerves was normal. The distal ends of four limbs were depauperated. The proximal end muscle power of both upper limbs was at level 4, the distal ends muscle power of both upper limbs was at level 3, and the muscle power of all fingers was at level 3-. The proximal end muscle power of both lower limbs was at level 4. The distal ends muscle power of both lower limbs was at level 3. The muscle tension of four limbs was normal. The tendon reflex of four limbs disappeared, the pathology sign was negative. The deep and shallow sensation of her four limbs distal ends were not good.

Diagnosis: Charcot-Marie-Tooth disease (CMT)

Treatment target: Replace the heredodegeneration nerve cells with normal stem cells to repair the nerves, improve the nerve function, and also to improve equilibrium function and motor function.

Treatment procedure and results: We gave the patient 4 times neural stem cells (NSCs) and 4 times mesenchymal stem cells (MSCs) implantation treatment. The stem cells were activated in the patients body to repair the nerve damage. Together with nourishment of the neurons, improvement of circulation and regulating the immune, daily rehabilitation training was incorporated. During the treatment, the patient was happy, had a regular eating and sleeping pattern. With our doctors help, she was able to complete the treatment. After the treatment, the patient had significant improvement, her four limbs had less pain, numbness and weakness. Her exercise tolerance was better. The proximal end muscle power of both upper limbs was at level 5-. The distal ends muscle power of both upper limbs was at level 4, the muscle power of all fingers was at level 4-. The proximal end muscle power of both lower limbs was at level 5-. The distal ends muscle power of both lower limbs was at level 4-. Her balance and coordinate movement were better. Her life had been noticeably improved.

Charcot-Marie-Tooth disease (CMT) is also called Hereditary Motor and Sensory Neuropathy (HMSN), it has visible heredity. The main clinical characteristics: the distal ends of four limbs has progressive weakness, atrophia and sensory disturbance. CMT is one of the most common hereditary peripheral neuropathy (the incidence is around 1/2500). CMT is classified two types according to clinical and electrophysiological characteristics; CMT1 (demyelinating type) and CMT2 (axon type).

The patient had this disease when she was growing up, and the disease progressed slowly. She gradually had four limbs weakness, the distal ends of four limbs were depauperated, hypesthesia, tendon reflex of four limbs disappeared and balance disturbance. All the accessory examinations supported the diagnosis.

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Scripps Health Receives $7.6M Grant to Regrow Knee Cartilage

Stem Cell Clinic | Posted by admin
Apr 04 2015

Dr. Darryl DLima. Photo courtesy of Scripps Health

Scripps Health announced today it has received a $7.6 million grant to study the repair and regeneration of knee cartilage, and the underlying bone defects and lesions caused by osteoarthritis.

The award from the California Institute for Regenerative Medicine will support ongoing stem cell research by the Shiley Center for Orthopedic Research and Education at Scripps Clinic.

The funding provided by CIRM is essential to the development and support of the research we are doing with regard to tissue regeneration at Scripps, said Dr. Darryl DLima, the Scripps Health director of orthopedic research. With this grant we plan to continue our progress in this field and move toward clinical trials within the next three years.

Scripps researchers are studying a cell therapy that combines stem cells with a natural scaffold made of water-based gels to support the repair of cartilage and bone defects. Such defects, if left untreated, are a major factor in contributing to early osteoarthritis in patients younger than 55.

Caused by the deterioration of cartilage between joints, osteoarthritis affects more than 27 million people in the United States, according to the U.S. Centers for Disease Control and Prevention.

Almost all current strategies to repair knee cartilage involve the removal of healthy cartilage and tissue around the lesion and the creation of artificial defects in the joint to facilitate further treatment or implantation. However, for younger patients with severe arthritis or impending arthritis, there is no treatment that can prevent, cure or even slow the progression of this disease.

Scientists with The Scripps Research Institute and Sanford-Burnham Medical Research Institute are assisting with the project.

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Boosting A Natural Protection Against Alzheimer's Disease

Cell Medicine | Posted by admin
Mar 13 2015

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Newswise Researchers at the University of California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimers disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from adult stem cells.

Our results suggest that certain gene variants allow us to reduce the amount of beta amyloid produced by neurons, said senior author Lawrence Goldstein, PhD, director of UC San Diego Sanford Stem Cell Clinical Center and UC San Diego Stem Cell Program. This is potentially significant for slowing the progression of Alzheimers disease. AD is the most common cause of dementia in the United States, afflicting one in nine people age 65 and older.

The genetic risk factor investigated are variants of the SORL1 gene. The gene codes for a protein that affects the processing and subsequent accumulation of beta amyloid peptides, small bits of sticky protein that build up in the spaces between neurons. These plaques are linked to neuronal death and related dementia.

Previous studies have shown that certain variants of the SORL1 gene confer some protection from AD, while other variants are associated with about a 30 percent higher likelihood of developing the disease. Approximately one-third of the U.S. adult population is believed to carry the non-protective gene variants.

The studys primary finding is that variants in the SORL1 gene may also be associated with how neurons respond to a natural compound in the brain that normally acts to protect nerve cell health. The protective compound, called BDNF, short for brain-derived neurotrophic factor, is currently being investigated as a potential therapy for a number of neurological diseases, including AD, because of its role in promoting neuronal survival.

For the study, UC San Diego researchers took skin cells from 13 people, seven of whom had AD and six of whom were healthy control subjects, and reprogrammed the skin cells into stem cells. These stem cells were coaxed to differentiate into neurons, and the neurons were cultured and then treated with BDNF.

The experiments revealed that neurons that carried disease-protective SORL1 variants responded to the therapy by reducing their baseline rate of beta amyloid peptide production by, on average, 20 percent. In contrast, the neurons carrying the risk variants of the gene, showed no change in baseline beta amyloid production.

BDNF is found in everyones brain, said first author Jessica Young, PhD, a postdoctoral fellow in the Goldstein laboratory. What we found is that if you add more BDNF to neurons that carry a genetic risk factor for the disease, the neurons dont respond. Those with the protective genetic profile do.

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New ALS gene and signaling pathways identified

Stem Cell Medical Center | Posted by admin
Mar 09 2015

IMAGE:Induced pluripotent stem cell-derived motor neurons from an ALS patient (left) compared with normal cells (right). The cells are being used to study the role of the genes TBK1 and… view more

NEW YORK, NY (February 19, 2015)–Using advanced DNA sequencing methods, researchers have identified a new gene that is associated with sporadic amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. ALS is a devastating neurodegenerative disorder that results in the loss of all voluntary movement and is fatal in the majority of cases. The next-generation genetic sequencing of the exomes (protein-coding portions) of 2,874 ALS patients and 6,405 controls represents the largest number of ALS patients to have been sequenced in a single study to date.

Though much is known about the genetic underpinnings of familial ALS, only a handful of genes have been definitively linked to sporadic ALS, which accounts for about 90 percent of all ALS cases. The newly associated gene, called TBK1, plays a key role at the intersection of two essential cellular pathways: inflammation (a reaction to injury or infection) and autophagy (a cellular process involved in the removal of damaged cellular components). The study, conducted by an international ALS consortium that includes scientists and clinicians from Columbia University Medical Center (CUMC), Biogen Idec, and HudsonAlpha Institute for Biotechnology, was published today in the online edition of Science.

“The identification of TBK1 is exciting for understanding ALS pathogenesis, especially since the inflammatory and autophagy pathways have been previously implicated in the disease,” said Lucie Bruijn, PhD, Chief Scientist for The ALS Association. “The fact that TBK1 accounts for one percent of ALS adds significantly to our growing understanding of the genetic underpinnings of the disease. This study, which combines the efforts of over two dozen laboratories in six countries, also highlights the global and collaborative nature of ALS research today.

“This study shows us that large-scale genetic studies not only can work very well in ALS, but that they can help pinpoint key biological pathways relevant to ALS that then become the focus of targeted drug development efforts,” said study co-leader David B. Goldstein, PhD, professor of genetics and development and director of the new Institute for Genomic Medicine at CUMC. “ALS is an incredibly diverse disease, caused by dozens of different genetic mutations, which we’re only beginning to discover. The more of these mutations we identify, the better we can decipher–and influence–the pathways that lead to disease.” The other co-leaders of the study are Richard M. Myers, PhD, president and scientific director of HudsonAlpha, and Tim Harris, PhD, DSc, Senior Vice President, Technology and Translational Sciences, Biogen Idec.

“These findings demonstrate the power of exome sequencing in the search for rare variants that predispose individuals to disease and in identifying potential points of intervention. We are following up by looking at the function of this pathway so that one day this research may benefit the patients living with ALS,” said Dr. Harris. “The speed with which we were able to identify this pathway and begin our next phase of research shows the potential of novel, focused collaborations with the best academic scientists to advance our understanding of the molecular pathology of disease. This synergy is vital for both industry and the academic community, especially in the context of precision medicine and whole-genome sequencing.”

“Industry and academia often do things together, but this is a perfect example of a large, complex project that required many parts, with equal contributions from Biogen Idec. Dr. Tim Harris, our collaborator there, and his team, as well as David Goldstein and his team, now at Columbia University, as well as our teams here at HudsonAlpha, said Dr. Myers. “I love this research model because it doesn’t happen very frequently, and it really shows how industry, nonprofits, and academic laboratories can all work together for the betterment of humankind. The combination of those groups with a large number of the clinical collaborators who have been seeing patients with this disease for many years and providing clinical information, recruiting patients, as well as collecting DNA samples for us to do this study, were all critical to get this done.”

Searching through the enormous database generated in the ALS study, Dr. Goldstein and his colleagues found several genes that appear to contribute to ALS, most notably TBK1 (TANK-Binding Kinase 1), which had not been detected in previous, smaller-scale studies. TBK1 mutations appeared in about 1 percent of the ALS patients–a large proportion in the context of a complex disease with multiple genetic components, according to Dr. Goldstein. The study also found that a gene called OPTN, previously thought to play a minor role in ALS, may actually be a major player in the disease.

“Remarkably, the TBK1 protein and optineurin, which is encoded by the OPTN gene, interact physically and functionally. Both proteins are required for the normal function of inflammatory and autophagy pathways, and now we have shown that mutations in either gene are associated with ALS,” said Dr. Goldstein. “Thus there seems to be no question that aberrations in the pathways that require TBK1 and OPTN are important in some ALS patients.”

The researchers are currently using patient-derived induced pluripotent embryonic stem cells (iPS cells) and mouse models with mutations in TBK1 or OPTN to study ALS disease mechanisms and to screen for drug candidates. Several compounds that affect TBK1 signaling have already been developed for use in cancer, where the gene is thought to play a role in tumor-cell survival.

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MS stem cell treatment hailed 'miraculous' as patients make dramatic recovery

Stem Cell Treatment | Posted by admin
Mar 09 2015

Pioneering treatment has allowed wheelchair-bound patients to run again Patient given high dose of chemotherapy to wipe out faulty immune system Therapy then uses person’s own stem cells to fight the devastating disease It may be the first ever treatment tosuccessfullyreverse symptoms of MS

By Fiona Macrae for the Daily Mail

Published: 13:27 EST, 1 March 2015 | Updated: 02:54 EST, 2 March 2015

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Britons left wheelchair-bound by multiple sclerosis can walk, run and even dance again after being given a pioneering stem cell treatment.

Doctors have described the recoveries as miraculous, while patients say they have been given their lives back.

The treatment uses a patients own stem cells the bodys master cells to fight the disease.

Recovery: MS sufferer Holly Drewerybecame wheelchair-bound after the birth of daughter Isla, but thanks tothe stem cell transplant shecan dance, run and chase after Isla in the park

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Ciplas Stempeutics gets US patent for stemcell product

Stem Cell Treatment | Posted by admin
Mar 06 2015

Drug used to treat limb disorder

Mumbai, March 4:

Stempeutics Research has received a process patent from the United States Patent and Trademarks Office for its novel stem cell-based drug Stempeucel.

The company is part of the Manipal Education and Medical Group and drugmaker Cipla entered into a joint alliance with it in 2009.

The drug will initially be used for the treatment of Critical Limb Ischemia (CLI), the companies said in a joint note, adding that the treatment option directly addresses the root cause of the disease, unlike others that treat the symptoms and not the disease.

CLI is a progressive form of peripheral arterial disease that blocks arteries in the lower extremities of the body, resulting in reduced blood flow.

It is a debilitating disease which affects patients with severe pain in the feet or toes, besides the development of sores and wounds, the note said.

If left untreated, patients may have to undergo amputation of the affected limb, it added.

Stempeutics has also submitted its applications to the regulatory Drug Controller General of India (DCGI) to obtain marketing authorisation for Stempeucel.

The availability of the product in the local market is subject to DCGI approvals.

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Cell Cure Neurosciences Ltd. Provides Update on its Product Development and Partnering Activities

Stem Cell Treatment | Posted by admin
Feb 18 2015

ALAMEDA, Calif. & JERUSALEM–(BUSINESS WIRE)–BioTime, Inc. (NYSE MKT: BTX) and its subsidiary Cell Cure Neurosciences Ltd. (Cell Cure) today provided an update on Cell Cures product development and partnering activities.

On February 16, 2015, Cell Cure opened the clinical trial of OpRegen titled Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients with Advanced Dry-Form Age-Related Macular Degeneration with Geographic Atrophy at Hadassah University Medical Center in Jerusalem, Israel. Patient enrollment is expected to begin shortly. OpRegen consists of animal product-free retinal pigment epithelial (RPE) cells with high purity and potency.

On October 31, 2014, the United States Food and Drug Administration (FDA) cleared Cell Cure’s Investigational New Drug (IND) application to initiate the clinical trial of OpRegen in patients with the severe form of age-related macular degeneration (AMD) with geographic atrophy (GA). While treatment options exist for the treatment of the wet form of AMD, it amounts to only about 10% of the disease prevalence. There is currently no FDA-approved therapy for the dry form occurring in approximately 90% of those afflicted with AMD. Cell Cure intends to transplant OpRegen as a single dose into the subretinal space of patients eyes in order to test the safety and efficacy of the product in this leading cause of blindness.

The Phase I/IIa clinical trial, will evaluate three different dose regimens of OpRegen. Following transplantation, the patients will be followed for 12 months at specified intervals, to evaluate the safety and tolerability of the product. Following the initial 12 month period, patients will continue to be monitored at longer intervals for an additional period of time. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen to engraft, survive, and moderate disease progression in the patients. In addition to thorough characterization of visual function, a battery of ophthalmic imaging modalities will be used to quantify structural changes and rate of GA expansion.

Cell Cure also announced today that the option granted to Teva Pharmaceutical Industries Ltd. (Teva) under a Research and Exclusive Option Agreement of October 7, 2010 to license-in rights to its OpRegen product has expired without having been exercised by Teva. Cell Cure will therefore be continuing the clinical development of OpRegen on its own and pursuing discussions with other potential strategic partners, including those that have already indicated interest in participating in development and commercialization of the product.

Cell Cure also announced that US patent No. 8,956,866 relating to a proprietary method of manufacturing RPE cells (the active ingredient of OpRegen) is expected to issue on February 17, 2015. This patent combined with other patents and patent applications in the BioTime family of companies provides significant patent protection for this novel therapeutic modality for AMD.

The large markets currently associated with therapies for the wet form of AMD combined with the elegance of RPE replacement therapy for the larger unmet needs associated with the dry form, highlights why Cell Cure has prioritized the development of this product, said Dr. Charles Irving, CEO of Cell Cure. We look forward to initiation of the trials and providing updates in the coming months.

About Age-Related Macular Degeneration

Age-related macular degeneration (AMD) is one of the major diseases of aging and is the leading eye disease responsible for visual impairment of older persons in the US, Europe and Australia. AMD affects the macula, which is the part of the retina responsible for sharp, central vision that is important for facial recognition, reading and driving. There are two forms of AMD. The dry form (dry-AMD) advances slowly and painlessly but may progress to geographic atrophy (GA) in which RPE cells and photoreceptors degenerate and are lost. Once the atrophy involves the fovea (the center of the macula), patients lose their central vision and may develop legal blindness. There are about 1.6 million new cases of dry-AMD in the US annually, and as yet there is no effective treatment for this condition. About 10% of patients with dry-AMD develop wet (or neovascular) AMD, the second main form of this disease, which usually manifests acutely and can lead to severe visual loss in a matter of weeks. Wet-AMD can be treated with currently-marketed VEGF inhibitors. However, such products typically require frequent repeated injections in the eye, and patients often continue to suffer from continued progression of the underlying dry-AMD disease process. Current estimated annual sales of VEGF inhibitors for the treatment of the wet form of AMD are estimated to be in excess of $5 billion worldwide. The root cause of the larger problem of dry-AMD is believed to be the dysfunction of RPE cells. One of the most exciting therapeutic approaches to dry-AMD is the transplantation of healthy, young RPE cells to support and replace the patients old degenerating RPE cells, which may prevent progression of the atrophy as well as the development of wet-AMD. Pluripotent stem cells, such as hESCs, can provide an unlimited source for the derivation of such healthy RPE cells for transplantation.

About OpRegen

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Treatment for Chronic Kidney Disease using Stem Cell Therapy in India - Video

Cell Therapy | Posted by admin
Feb 07 2015



Treatment for Chronic Kidney Disease using Stem Cell Therapy in India
Learn how Chronic Kidney Disorder can be treated using stem cell therapy: http://reelabs.com/stem-cell-therapy/treatable-diseases/chronic-kidney-disorder.html Chronic renal failure, also known…

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Treatment for Chronic Kidney Disease using Stem Cell Therapy in India – Video

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