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Archive for the ‘Cell Therapy’ Category

On stem cell therapy, benefits

Thursday, March 26th, 2015

Bobby Chia (Foto by Allan Defensor)

THERE is a lot of hype going on in some circles about stem cell therapy: Is it a cure-all? Is it the elusive fountain of youth?

Wikipedia definesstem cell therapy as the use of stem cells to treat or prevent a disease or condition.The process involves the administration of live whole cells or maturation of a specific cell population in a patient for the treatment of the disease as has been done in bone marrow transplants.

Bobby Chia, a Thai national who was in Cebu for a brief visit, said that stem cell therapy has been done in Villa Medica, Germany, since the 1960s. He learned about it 10 years ago when his mother had cancer and he looked around for the best medical care for her and found it in Villa Medica. It made her so much better (she can even play tennis now) that four years ago, Chia bought the clinic being run by Dr. Geoffrey Huertgen, a third generation doctor of that clinic.

The stem cell can be taken from the patient himself, but Chia says this stem source is naturally as old as the patient himself. Villa Medica chooses to use stem cells from fetuses of sheep (he said that stem cells from any mammal would be the same, but sheep stem cells are the ones more readily available). The process for Villa Medica, says Chia, involves the designing of a cocktail of stem cells to address whatever needs correction. If the eyes are not good, we choose the eyes. If the ears are not good, we choose the ears. If the heart is not good, we choose the heart. The procedure has been known to treat diseases and ailments like Parkinsons, diabetes, hypertension, migraine, allergies etc.

It (stem cell therapy) does not make you 18 years old all over again but it energizes you; from not being able to walk to walking again. It gives you a better quality of life. It is not a quick cure, but for a lot of people it is. We offer a choice for people who have no hope, Chia shares. But my main target is people who are not sick, Chia states, referring to athletes, models, professionals who want to have a better quality of life.

Chia says there are four doctors in Villa Medica. Since the cell therapy is patient-specific, the patient needs to stay four days and four nights at the clinic: for a detox program, for physical check-up, for interview, for determining the cocktail of stem cells to be used and how. The result, Chia says,is not immediate. It may take six weeks or even longer for the stem cells to do their work.

Chia says he has had about 150 patients coming from Cebu. Leaf through the pages of the local papers, one of them might just be there!

Published in the Sun.Star Cebu newspaper on March 27, 2015.

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On stem cell therapy, benefits

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Stem cell therapy could reverse Type 2 diabetes, UBC study finds

Tuesday, March 24th, 2015

VANCOUVER -- University of B.C. scientists appear to be one step closer to reversing diabetes using stem cell therapy.

The latest study, published last week in the journal Stem Cell Reports, found that Type 2 diabetes can be eliminated in mice using a combination of conventional diabetes drugs and specially cultured stem cells. Similar methods have already been used to reverse Type 1 diabetes, which usually begins in childhood.

The team simulated Type 2 diabetes in mice by feeding them a high-fat, high-calorie diet for several weeks. In humans, Type 2 usually begins in adulthood and can be a result of obesity, poor diet and lack of exercise.

Like diabetic humans, the diabetic mice treated only with drugs experienced spikes in their blood sugar levels after eating sugary meals.

But the mice that were surgically implanted with pancreatic-like cells grown from human stem cells didnt have those drastic swings and were able to regulate their blood sugar like healthy animals.

Being able to reduce spikes in blood sugar levels is important because evidence suggests its those spikes that do a lot of the damage increasing risks for blindness, heart attack, and kidney failure, said Timothy Kieffer, a professor in UBCs department of cellular and physiological sciences.

So far, the researchers have followed the mice for up to seven months, and theyve remained healthy.

When we removed the transplanted devices and analyzed the cells within, they still appear very healthy so we believe they will function much longer. Ultimately the duration of cell function will need to be assessed in humans, Kieffer said in an email.

Human trials are already underway for stem cell therapy on Type 1 diabetes; the first patient was implanted with cells in October.

The treatment also had a surprising side-effect: weight loss. The mice all returned to the same, healthy weight as the animals in the control group.

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Nevada Pain Now Offering Stem Cell Therapy to Help Patients Delay or Avoid Joint Replacement

Monday, March 23rd, 2015

Las Vegas, Nevada (PRWEB) March 23, 2015

Nevada Pain, the top pain management clinics in Las Vegas and Henderson areas, are now offering stem cell therapy to help patients delay or avoid the need for joint replacement. The treatments are cutting edge, outpatient, and highly effective. Call (702) 323-0553 for more information and scheduling.

Stem cell therapy for joint arthritis has rapidly become mainstream due to its effectiveness for pain relief. Unlike traditional treatments, the regenerative medicine therapies actually have the potential to change the course of arthritis rather than simply provide a proverbial "band aid" to the condition.

The stem cell treatments are provided by Board Certified, Fellowship Trained pain doctors who have extensive experience with the procedures into all extremity joints including the hip, knee, shoulder and ankle. There are multiple types of stem cell treatments. Some involve amniotic fluid derived treatment, which contains an immense amount of stem cells, growth factors, hyaluronic acid and anti-inflammatory mediators as well.

Bone marrow derived treatment is also available, which also contains an immense amount of stem cells and growth factors. The treatments are provided as an outpatient, and often provide pain relief and functional improvement.

Platelet Rich Plasma Therapy is also offered, which involves a simple blood draw. The platelets are then concentrated and immediately injected into the problem area.

For more information and scheduling with the top regenerative medicine clinic in Las Vegas, call (702) 323-0553.

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Nevada Pain Now Offering Stem Cell Therapy to Help Patients Delay or Avoid Joint Replacement

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Stem Cell Injection Treatment Stem Cell Therapy – Video

Saturday, March 21st, 2015


Stem Cell Injection Treatment Stem Cell Therapy
Salah satu kaedah perawatan menggunakan Sel Induk dari Tulan Belakang.. Untuk maklumat lanjut boleh call/WA/Telegram/SMS ke 013-3530172 atau layarai web http://www.cellmaxx4all.com.

By: KAMARUZAMAN DERAMAN

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Stem cell therapy and me1 – Video

Saturday, March 21st, 2015


Stem cell therapy and me1
Walk without support.

By: drabhijitshendeji

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Stem cell therapy and me1 - Video

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stem cell therapy di indonesia Jakarta tangerang serpong bsd bintaro – Video

Saturday, March 21st, 2015


stem cell therapy di indonesia Jakarta tangerang serpong bsd bintaro
http://youtu.be/3c259RtpzpE.

By: Layar Baru DKI

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Knee arthritis 3 years after stem cell therapy by Harry Adelson, N.D. – Video

Saturday, March 21st, 2015


Knee arthritis 3 years after stem cell therapy by Harry Adelson, N.D.
Donna from Colorado describes her outcome three years after stem cell therapy for her arthritic knee by Harry Adelson, N.D. http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Knee arthritis 3 years after stem cell therapy by Harry Adelson, N.D. - Video

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Amniotic Stem Cell Therapy Discussed by R3 (844) GET-STEM – Video

Thursday, March 19th, 2015


Amniotic Stem Cell Therapy Discussed by R3 (844) GET-STEM
http://r3stemcell.com/stem-cell-treatments/amniotic-derived-stem-cell-injections/ Amniotic derived stem cell therapy has become exceptionally popular due to the benefits that are being seen....

By: R3 Stem Cell

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Stem cell therapy may help treat type 2 diabetes

Thursday, March 19th, 2015

A human embryonic stem cell line derived at Stanford University.(REUTERS/Julie Baker/Stanford University School of Medicine/California Institute for Regenerative Medicine/Handout)

Type 2 diabetes is marked by insulin resistance, or the bodys inability to store sugar and convert it into carbohydrates for energy. Overcoming that resistance is the main hurdle scientists face in creating new treatment for the condition, but researchers in Canada have found a promising means for doing so: combining stem cell therapy and antidiabetic medication.

Type 2 diabetes accounts for nearly 95 percent of the 400 million diabetes cases worldwide. Current treatment involves imprecise insulin injection, and can produce side effects like unwanted weight gain, gastrointestinal issues and low blood glucose levels. Eighty percent of Type 2 diabetes patients are overweight.

In the study, published Thursday in the journal Stem Cell Reports, scientists observed that transplanting human stem cells into mice with Type 2 diabetes symptoms, then administering common antidiabetic drugs, improved the mices glucose metabolism, body weight and insulin sensitivity three hallmark problems associated with the condition.

There have been similar reports looking at treatment of type 1 diabetes by stem cell-based replacement, and there are many people around the world who are interested in that, lead study author Timothy J. Kieffer, a molecular and cellular medicine professor at the University of British Columbia, in Vancouver, told FoxNews.com. Until this point, nobody to our knowledge had tested such a stem cell-based transplant study in a Type 2 diabetes model.

Many [of these studies] have been predicted to fail because one of the characteristics of Type 2 diabetes is insulin resistance, and that is in part due to obesity and higher demands of insulin, Kieffer added, and therefore it might be predicted that insulin replacement wouldnt work if were just putting insulin back.

Researchers fed four separate groups of immunosuppressed mice a different diet to try to emulate humans diagnosed with Type 2 diabetes. One group of mice received a 45 percent fat diet; one a 60 percent fat diet; one a high-fat, Western diet; and the last a low-fat diet. No single group of mice developed a phenotype that exactly mimicked a Type 2 diabetes human patient, but all three high-fat groups ended up exhibiting characteristics that mirrored the hallmark features of the condition.

Study authors transplanted human embryonic stem cell (hESC)-derived pancreatic progenitor cells into the mice after they began exhibiting symptoms. These cells are programmed to expand and differentiate when transplanted into the pancreas, and to subsequently secrete insulin.

To transplant the human stem cells, researchers used a macroencapsulation device, a mechanism that is meant to prevent the body from detecting nonnative material as foreign and subsequently rejecting it. Because the mice were immunosuppressed, the device wasnt necessary, but Kieffer said his team used it so their findings would be more relevant for future clinical trials, wherein the patients would not be immunosuppressed. Researchers opted to induce Type 2 diabetes symptoms in immunosuppressed mice instead of using the mice model genetically engineered to assume Type 2 diabetes for that same reason.

The hope in the field is that some sort of device will eliminate the need for immunosuppression when cells are transplanted, Kieffer said.

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CAR T-Cell Therapy – Nebraska Medicine – Video

Wednesday, March 18th, 2015


CAR T-Cell Therapy - Nebraska Medicine
It #39;s the fifth most common type of cancer in U.S. adults. For years, traditional therapies to treat non-Hodgkin #39;s lymphoma (NHL) have included chemotherapy, radiation and a stem cell/bone...

By: Nebraska Medicine

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Stem Cell Therapy Now Being Offered for NonHealing Wounds at Telehealth’s Three Regenerative Medicine Clinics

Wednesday, March 18th, 2015

Orange, California (PRWEB) March 17, 2015

The top stem cell therapy clinics in California, Telehealth, are now offering treatment for nonhealing wounds at three locations. The stem cell therapy for wound healing is being offered by Board Certified doctors at three separate locations in Orange, La Jolla and Upland. Call (888) 828-4575 for more information and scheduling.

Patients with diabetes, neuropathy and autoimmune disorders often find it difficult to heal even minor wounds. This may lead to diabetic ulcers and infections in the soft tissue and/or bone. At times, even the most rigorous conventional wound care fails to heal wounds sufficiently.

At Telehealth, stem cell therapy for nonhealing wounds has been showing exceptional results. Wounds that had basically been unresponsive to traditional methods have displayed quick results with healing when the procedures are performed. The regenerative medicine treatments involve either bone marrow derived stem cells or amniotic derived stem cells. Additional, PRP therapy is included in the treatment at times when necessary.

Along with helping to heal difficult wounds, stem cell therapy is also available for degenerative arthritis, chronic tendonitis, rotator cuff tears, ligament injuries, migraines and much more. Treatments are offered in Orange, Upland and a new La Jolla location by Board Certified doctors with extensive experience.

Most treatments are partially covered by insurance, which helps considerably to keep cost down. Call (888) 828-4575 for more information and scheduling.

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Stem Cell Therapy Now Being Offered for NonHealing Wounds at Telehealth's Three Regenerative Medicine Clinics

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Beware stem cell therapy for lung disease

Tuesday, March 17th, 2015

Clinics in other countries for some time have promised dramatic results in the treatment of lung disease, primarily emphysema, through the use of autologous stem cells obtained from the patient. The stem cells are extracted from adipose (fat) tissue, treated and then injected into the patient. The cells then supposedly go to work regenerating and replacing the damaged lung tissue.

Several of this type of clinic are now popping up in parts of the United States, mostly in California and Florida. Their advertisements are filled with testimonials from patients, extolling the virtues of the treatments. The treatments are quite expensive, and would be an absolute godsend for the 30 million Americans who suffer from some stage of Chronic Obstructive Pulmonary Disease (COPD). If they worked.

Trouble is, none of these clinics or their treatments are approved by the FDA, and the only proof of their effectiveness is anecdotal, coming from selected customers.

Anyone with a chronic, progressive disease, such as COPD, will usually find themselves in a situation of desperation, eager to embrace any promises of a cure. I have been there, and it is a terrible situation.

Sadly, further research shows that institutions that are working on stem cell therapy for lungs unanimously agree that the successful regeneration of human lung tissue is likely decades away. Dr. Hatch, a British researcher, states that he may be able to announce success in about 20 years. Boston University states that stem cell treatment for lungs may be available for our grandchildren or great-grandchildren.

Even the Center for Regenerative Medicine at Wake Forest, which has successfully built working bladders and other of the simpler internal organs, states that we are likely 20 years away from creating a lung.

There have always been those who would separate us from our money with promises of cures of everything from cancer to male pattern baldness. Please beware.

Jim Nelson is a former Glenwood Springs resident who works with regional and national cardiovascular and lung organizations.

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Beware stem cell therapy for lung disease

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The New Botox: Stem Cell Therapy Cream Reviews – Video

Saturday, March 14th, 2015


The New Botox: Stem Cell Therapy Cream Reviews
http://buildingabrandonline.com/buildabrandwithjamalspikes/what-is-jeunesse/ The New Botox: Stem Cell Therapy Cream Reviews Stem cell therapy is the use of stem cells to treat or prevent a...

By: Jamal Spikes

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The New Botox: Stem Cell Therapy Cream Reviews - Video

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Miracle stem cell therapy reverses multiple sclerosis – Video

Saturday, March 14th, 2015


Miracle stem cell therapy reverses multiple sclerosis
Latest research on stem cell therapy in curing MS.

By: Dulci Hill

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Miracle stem cell therapy reverses multiple sclerosis - Video

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Cell Therapy – American Cancer Society

Saturday, March 14th, 2015

Other common name(s): cellular therapy, fresh cell therapy, live cell therapy, glandular therapy, xenotransplant therapy

Scientific/medical name(s): none

In cell therapy, processed tissue from the organs, embryos, or fetuses of animals such as sheep or cows is injected into patients. Cell therapy is promoted as an alternative form of cancer treatment.

Cell therapy as described here is different from the many types of cell therapy that are available as part of proven cancer treatment, such as hematopoietic stem cell transplant and prostate cancer treatment that uses modified human cells. Research continues in this area.

Available scientific evidence does not support claims that cell therapy is effective in treating cancer or any other disease. Serious side effects can result from cell therapy. It may in fact be lethalseveral deaths have been reported. It is important to distinguish between this alternative method involving animal cells and mainstream cancer treatments that use human cells, such as bone marrow transplantation.

In cell therapy, live or freeze-dried cells or pieces of cells from the healthy organs, fetuses, or embryos of animals such as sheep or cows are injected into patients. This is supposed to repair cellular damage and heal sick or failing organs. Cell therapy is promoted as an alternative therapy for cancer, arthritis, heart disease, Down syndrome, and Parkinson disease.

Cell therapy is also marketed to counter the effects of aging, reverse degenerative diseases, improve general health, increase vitality and stamina, and enhance sexual function. Some practitioners have proposed using cell therapy to treat AIDS patients.

The theory behind cell therapy is that the healthy animal cells injected into the body can find their way to weak or damaged organs of the same type and stimulate the body's own healing process. The choice of the type of cells to use depends on which organ is having the problem. For instance, a patient with a diseased liver may receive injections of animal liver cells. Most cell therapists today use cells taken from taken from the tissue of animal embryos.

Supporters assert that after the cells are injected into the body, they are transported directly to where they are most needed. They claim that embryonic and fetal animal tissue contains therapeutic agents that can repair damage and stimulate the immune system, thereby helping cells in the body heal.

The alternative treatment cell therapy is very different from some forms of proven therapy that use live human cells. Bone marrow transplants infuse blood stem cellsfrom the patient or a carefully matched donorafter the patients own bone marrow cells have been destroyed. Studies have shown that bone marrow transplants are effective in helping to treat several types of cancer. In another accepted procedure, damaged knee cartilage can be repaired by taking cartilage cells from the patient's knee, carefully growing them in the laboratory, and then injecting them back into the joint. Approaches involving transplants of other types of human stem cells are being studied as a possible way to replace damaged nerve or heart muscle cells, but these approaches are still experimental.

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Cell Therapy - American Cancer Society

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Hip and shoulder arthritis six months after bone marrow stem cell therapy by Harry Adelson ND – Video

Friday, March 13th, 2015


Hip and shoulder arthritis six months after bone marrow stem cell therapy by Harry Adelson ND
Mareen describes her outcome six months after her bone marrow stem cell treatment by Harry Adelson ND for arthritis of her hip and shoulder http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Stem Cell Research & Therapy | Full text | Amnion-derived …

Friday, March 13th, 2015

Fauza D: Amniotic fluid and placental stem cells.

Best Pract Res Clin Obstet Gynaecol 2004, 18:877-891. PubMedAbstract | PublisherFullText

Parolini O, Alviano F, Bagnara GP, Bilic G, Bhring HJ, Evangelista M, Hennerbichler S, Liu B, Magatti M, Mao N, Miki T, Marongiu F, Nakajima H, Nikaido T, Portmann-Lanz CB, Sankar V, Soncini M, Stadler G, Surbek D, Takahashi TA, Redl H, Sakuragawa N, Wolbank S, Zeisberger S, Zisch A, Strom SC: Concise review: isolation and characterization of cells from human term placenta: outcome of the first international workshop on placenta derived stem cells.

Stem Cells 2008, 26:300-311. PubMedAbstract | PublisherFullText

Pozzobon M, Ghionzoli M, De Coppi P: ES, iPS, MSC, and AFS cells. Stem cells exploitation for Pediatric Surgery: current research and perspective.

Pediatr Surg Int 2010, 26:3-10. PubMedAbstract | PublisherFullText

Miki T, Marongiu F, Dorko K, Ellis EC, Strom SC: Isolation of amniotic epithelial stem cells.

Curr Protoc Stem Cell Biol 2010, Chapter 1:Unit 1E 3. PubMedAbstract | PublisherFullText

Miki T, Strom SC: Amnion-derived pluripotent/multipotent stem cells.

Stem Cell Rev 2006, 2:133-142. PubMedAbstract | PublisherFullText

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High-Dose Immunosuppressive Therapy and Autologous …

Friday, March 13th, 2015

Importance Most patients with relapsing-remitting (RR) multiple sclerosis (MS) who receive approved disease-modifying therapies experience breakthrough disease and accumulate neurologic disability. High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic cell transplant (HCT) may, in contrast, induce sustained remissions in early MS.

Objective To evaluate the safety, efficacy, and durability of MS disease stabilization through 3 years after HDIT/HCT.

Design, Setting, and Participants Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis (HALT-MS) is an ongoing, multicenter, single-arm, phase 2 clinical trial of HDIT/HCT for patients with RRMS who experienced relapses with loss of neurologic function while receiving disease-modifying therapies during the 18 months before enrolling. Participants are evaluated through 5 years after HCT. This report is a prespecified, 3-year interim analysis of the trial. Thirty-six patients with RRMS from referral centers were screened; 25 were enrolled.

Interventions Autologous peripheral blood stem cell grafts were CD34+ selected; the participants then received high-dose treatment with carmustine, etoposide, cytarabine, and melphalan as well as rabbit antithymocyte globulin before autologous HCT.

Main Outcomes and Measures The primary end point of HALT-MS is event-free survival defined as survival without death or disease activity from any one of the following outcomes: (1) confirmed loss of neurologic function, (2) clinical relapse, or (3) new lesions observed on magnetic resonance imaging. Toxic effects are reported using National Cancer Institute Common Terminology Criteria for Adverse Events.

Results Grafts were collected from 25 patients, and 24 of these individuals received HDIT/HCT. The median follow-up period was 186 weeks (interquartile range, 176-250) weeks). Overall event-free survival was 78.4% (90% CI, 60.1%-89.0%) at 3 years. Progression-free survival and clinical relapse-free survival were 90.9% (90% CI, 73.7%-97.1%) and 86.3% (90% CI, 68.1%-94.5%), respectively, at 3 years. Adverse events were consistent with expected toxic effects associated with HDIT/HCT, and no acute treatment-related neurologic adverse events were observed. Improvements were noted in neurologic disability, quality-of-life, and functional scores.

Conclusions and Relevance At 3 years, HDIT/HCT without maintenance therapy was effective for inducing sustained remission of active RRMS and was associated with improvements in neurologic function. Treatment was associated with few serious early complications or unexpected adverse events.

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Biotech Stock Mailbag: Neuralstem, Genfit, Intercept, Amarin

Friday, March 13th, 2015

BOSTON (TheStreet) --A question about Neuralstem (CUR - Get Report) and its stem-cell therapy for ALS kicks off this week's Biotech Stock Mailbag.

Steve writes, "If 47% of the people responded well and their progression of the disease slowed considerably, then I see this as a huge success for a disease with no cure. I don't have ALS or know anyone that does, but if I had it, I would immediately want the treatment knowing that there is a 47% chance that I will respond positively to it and it would DRASTICALLY slow the progression of the disease. Wouldn't you agree, or am I missing the point somewhere?"

Eight of the 15 (53%) ALS patients enrolled in the study saw their ALSFRS scores fall from an average of 40 to 14 over nine months. This is a rapid decline in muscle function and suggests NSI-566 accelerates the progression of ALS.

If you believe 47% of patients in the Neuralstem study benefit from NSI-566, you can't ignore the 53% of patients who fare far worse and may actually be harmed by the stem cell therapy.

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Biotech Stock Mailbag: Neuralstem, Genfit, Intercept, Amarin

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UCLA Research Shows Promising Method For Correcting Genetic Code To Treat Sickle Cell Disease

Thursday, March 12th, 2015

Posted: Thursday, March 12, 2015 7:08 PM

UCLA stem-cell researchers have shown that a novel stem-cell gene therapy method could one day provide a one-time, lasting treatment for the most common inherited blood disorder in the U.S. sickle cell disease. Publishedin the journal Blood, the study outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, the gene correction method leads to the production of normal red blood cells. The study was directed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member, Dr. Donald Kohn.

People with sickle cell disease are born with a mutation in their beta-globin gene, which is responsible for delivering oxygen to the body through blood circulation. The mutation causes blood stem cellswhich are made in the bone marrowto produce distorted and rigid red blood cells that resemble a crescent or sickle shape. Consequently, the abnormally shaped red blood cells do not move smoothly through blood vessels, resulting in insufficient oxygen supply to vital organs. Anyone can be born with sickle cell disease, but it occurs more frequently in African Americans and Hispanic Americans.

The stem-cell gene therapy method described in the study seeks to directly correct the mutation in the beta-globin gene so bone marrow stem cells then produce normal, circular-shaped blood cells that do not sickle. The fascinating gene correction technique used specially engineered enzymes, called zinc-finger nucleases, tocut out the mutated genetic code and replace it with a corrected version that repairs the beta-globin mutation.

For the study, bone marrow stem cells donated by people with the sickle cell gene mutation were treated in the laboratory with the zinc-finger nucleases enzyme cutting method.Kohn and his team then demonstrated in mouse models that thecorrected bone-marrow stem cells have the capability to replicate successfully. The research showed that the method holds the potential to permanently treat the disease if a higher level of correction is achieved.

This is a very exciting result,said Dr. Kohn, professor of pediatrics atUCLAs David Geffen School of Medicine, professor of microbiology, immunology and molecular genetics in Life Sciences at UCLA, member of the UCLA Childrens Discovery and Innovation Institute at Mattel Childrens Hospital and senior author on the study. It suggests the future direction for treating genetic diseases will be by correcting the specific mutation in a patients genetic code. Since sickle cell disease was the first human genetic disease where we understood the fundamental gene defect,and since everyone with sickle cell has the exact same mutation in the beta-globin gene, it is a great target for this gene correction method.

To make the cut in the genetic code, Dr. Kohn and his team used zinc-finger nucleases engineered by Sangamo BioSciences, Inc., in Richmond. The enzymes can be designed to recognize a specific and targeted point in the genetic code. For the study, scientists at Sangamo BioSciences engineered the enzymes to create a cut at the site of the mutated genetic code that causes sickle cell disease. This break triggered a natural process of repair in the cell and at the same time, a molecule containing the correct genetic code was inserted to replace the mutated code.

The next steps in this research will involve improving the efficiency of the mutation correction process and performing pre-clinical studies to demonstrate that the method is effective and safe enough to move to clinical trials.

Symptoms of sickle cell disease usually begin in early childhood and include a low number of red blood cells (anemia), repeated infections and periodic episodes of pain. People with sickle cell disease typically have a shortened lifespan of just 36-40 years of age. The disease impacts more than 250,000 new patients worldwide each year. The only cure currently available for sickle cell disease is a transplant of bone marrow stem cells from a matched sibling, but matches are rare or can result in rejection of the transplanted cells.

This is a promising first step in showing that gene correction has the potential to help patients with sickle cell disease, said Megan Hoban, a senior graduate student in microbiology, immunology and molecular genetics and first author on the study. The study data provide the foundational evidence that the method is viable.

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