Deaf gerbils hear again with human stem cells

Scientists have restored hearing to deaf gerbils using human embryonic stem cells in an advance that could eventually help people with an intractable form of deafness caused by nerve damage.

The procedure needs further animal research to assess safety and long-term effectiveness but researchers said on Wednesday the experiment was an important proof of concept, marking a further advance in the growing field of regenerative medicine.

Marcelo Rivolta from Britain's University of Sheffield, who led the research, said the first patients could receive cell therapy for hearing loss in clinical trials in "a few years".

After treating 18 gerbils with complete deafness in one ear, his team reported in the journal Nature that stem cells produced an average 46 percent recovery in hearing function, as measured by electrical signals in the animals' brains.

"If this was a human patient, it would mean going from being so deaf as to be unable to hear a lorry or truck on the street to being able to maintain a conversation," Rivolta told reporters.

"What we have shown here is functional recovery using human stem cells, which is unique."

Gerbils were selected for the test because their hearing range is similar to that of humans, while mice - the usual choice for laboratory tests - hear at higher frequencies.

The animals were deafened using a drug to destroy their auditory nerves before receiving an injection of around 50,000 human embryonic stem cells, which had previously been treated with growth factors to coax them into becoming ear cells.

The response among the gerbils varied, depending on how well the new cells were integrated into the cochlea, the spiral-shaped cavity in the inner ear.

Deafness is caused primarily by loss of sensory hair cells in the ear and auditory nerves. Since these cells are created only in the womb, there is no way to repair them once they have been damaged, resulting in permanent hearing loss.

Original post:
Deaf gerbils hear again with human stem cells

'Berlin Man,' Doctor Convinced HIV Cure Is Real

The first person reportedly cured of HIV said Wednesday he is hopeful that medical advances will allow others suffering from the virus that causes AIDS to be cured, too.

Timothy Ray Brown of San Francisco is known as "The Berlin Patient" because of where he was treated. He and the doctor who treated him, Gero Hutter, made their first joint appearance in the U.S. on Wednesday when Hutter spoke at a symposium on gene therapy at Washington University in St. Louis.

Scientists are studying whether gene therapy can be used to rid the body of HIV. Some doctors remain skeptical that Brown, 46, is cured. His case was first reported in the media in 2008 and described in the New England Journal of Medicine in 2009.

Brown and Hutter, in an interview with The Associated Press during the symposium, said the passage of time is further proof that Brown is cured. Hutter cited the same five-year standard after which some cancer patients are said to be cured.

Brown was diagnosed with HIV in 1995. In 2006, he also developed leukemia while living in Germany. Hutter performed a blood stem cell transplant using a donor with a rare gene mutation that provides natural resistance to HIV. Hutter said that resistance transferred to Brown.

Brown said he feels great, has not needed HIV medication since the 2007 surgery, and is now active in a foundation named for him that seeks a cure for HIV.

Brown grew up in Seattle and moved to Germany in 1993. After the HIV diagnosis, he started on medication to prevent him from developing full-blown AIDS.

He was attending a wedding in New York in 2006 when he became unusually tired. An avid cyclist, within weeks he could barely ride the bike and eventually was diagnosed with leukemia.

Brown underwent chemotherapy but needed a blood stem cell transplant and turned to Hutter, a blood specialist at Heidelberg University.

Hutter suggested they seek a donor with a certain cell feature that gives them natural resistance to HIV infection. Only about 1 percent of the northern European population has this feature. Hutter theorized that a transplant from such a donor could make the recipient resistant to HIV.

Read the original post:
'Berlin Man,' Doctor Convinced HIV Cure Is Real

'Stem cell hope' for deaf people

12 September 2012 Last updated at 13:00 ET By James Gallagher Health and science reporter, BBC News

UK researchers say they have taken a huge step forward in treating deafness after stem cells were used to restore hearing in animals for the first time.

Hearing partially improved when nerves in the ear, which pass sounds into the brain, were rebuilt in gerbils - a UK study in the journal Nature reports.

Getting the same improvement in people would be a shift from being unable to hear traffic to hearing a conversation.

However, treating humans is still a distant prospect.

If you want to listen to the radio or have a chat with a friend your ear has to convert sound waves in the air into electrical signals which the brain will understand.

This happens deep inside the inner ear where vibrations move tiny hairs and this movement creates an electrical signal.

However, in about one in 10 people with profound hearing loss, nerve cells which should pick up the signal are damaged. It is like dropping the baton after the first leg of a relay race.

The aim of researchers at the University of Sheffield was to replace those baton-dropping nerve cells, called spiral ganglion neurons, with new ones.

While there is excitement at the prospect of using stem cells to restore nerves in the ear this exact technique will not help the vast, vast majority of people with hearing loss.

Continue reading here:
'Stem cell hope' for deaf people

RNL BIO, a South Korean adult stem cell firm, introduces its autologous stem cell therapeutics in Turkey to treat …

SEOUL, South Korea, Sept. 13, 2012 /PRNewswire/ --RNL Bio (www.rnl.co.kr) announced on Sep 11, 2012 that it signed the agreement with RST Biomedikal Sanayi A.S. (RST), a Turkish company, to license RNL Bio's stem cell technology. Turkey is the 6th country where RNL Bio's stem cell technology has entered. This is one of the major accomplishments that RNL BIO has long focused on establishing the so-called 'Stem Cell Silk Road' with South Korean stem cell technology to give hope to patients with intractable diseases in the world.

RST as a licensee will pay the $5 million fee upfront within 60 days from the agreement and will continue to pay the running royalty of 15% of the revenue, which could be up to $ 200 million. RST will benefit from the geographical advantages of Turkey where Western, Arabic and Oriental cultures are crossed. It plans to establish a GMP facility and invite patients from Europe and Middle East early next year.

Ilknur Erdemin, CEO of RST said, "We expect to improve public health and the quality of life in Turkey through stem cell therapy technology imported from RNL BIO in treating various intractable diseases. We will also grow Turkey to one of the world's most renowned country in regards to medical tourism with RNL's stem cell technology in combination with Turkish World's Heritage." To begin with, RST will focus on the treatment ofdiabeticcomplications, autoimmune diseases, cerebral palsy, and degenerative arthritis with RNL's autologous adipose derived stem cell technology. Stem cell therapy has already been allowed by Turkish health authority since 2011. Turkey actively promotes the industry of regenerative medicine and makes a quick move to expand in related fields.

Dr. Jeong-Chan Ra, president of RNL Stem Cell Technology Institute said, "This licensing deal will be a good opportunity todevelopRNL's stem cell technology to be the world's standards and tofulfillmy goal to make RNL BIO a company that will have treated and helped the most patients suffering from intractable diseases." He had a seminar introducing his stem cell studies to Turkish attendees from related fields and distinguished invitees one day prior to signing licensing agreement.

See the original post here:
RNL BIO, a South Korean adult stem cell firm, introduces its autologous stem cell therapeutics in Turkey to treat ...

Gerbils regain hearing thanks to stem cell therapy

(CBS News) Scientists have restored hearing in gerbils using a stem cell therapy that may hold promise for deaf humans.

Using human embryonic stem cells, researchers at the University of Sheffield were able to implant immature nerve cells into gerbils, which then regenerated and were able to improve hearing ability in the animals. The study was published on Sept. 12 in Nature.

Scientists restore sense of smell to mice who were born with genetic abnormality Marvel team creates deaf superhero called Blue Ear in honor of boy

According to a Nature News article on the study, more than 275 million people have moderate-to-profound hearing loss, many of whom have it caused by a disruption in communication between the inner ear and brain. Senior study author Dr. Marcelo Rivolta, a stem cell researcher at the University of Sheffield told HealthDay that about 80 to 90 percent of deafness is due to problems with cells in the inner ear.

There are two types of inner ear cells. Hair cells translate vibrations into electrical signals that are transmitted via the auditory nerve to the brain. Problems with these cells are typically fixed via cochlear implant, a small device which can bypass the hair cells and directly send signals to intact auditory nerves. Neurons make up the auditory nerve, and when these are damaged, doctors have little to no treatment options available.

It's important to note that the type of deafness that the gerbils had affected only neurons, making it very rare. The Associated Press points out that type of deafness only affects between less than 1 percent to 15 percent of patients. Furthermore, the treatment won't work in all the patients with that disorder.

But, because so many disorders have to do with inner ear cell problems, the research is promising and may have future human applications.

Researchers in the study took embryonic stem cells, which can develop into any other kind of cell in the body, and grew them into a test tube that had molecules that are available when the fetus develops ears, known as fibroblast growth factors (FGFs). Some stem cells developed characteristics similar to hair cells and others turned into cells that looked like neurons.

Then the neuron-like cells, which were called otic neural progenitors (ONPs), were transplanted into the ears of gerbils that had been given ouabain, a chemical that damages the neurons in the auditory nerves but not the hair cells.

Ten weeks later, the cells had grown and some connected to the brain stem. The gerbils on average had a 46 percent overall improvement in hearing, with many of the animals registering brain activity at much fainter sounds after the transplant.

Go here to see the original:
Gerbils regain hearing thanks to stem cell therapy

Doctor claims cure for HIV, cites stem cell treatment

ST. LOUIS More than five years after a radical treatment, a San Francisco man and his German doctor are convinced that he remains the first person cured of infection with HIV, the virus that causes AIDS.

Timothy Ray Brown, who is known as "The Berlin Patient" because of where he was treated, and Dr. Gero Hutter made their first joint appearance in the U.S. on Wednesday when Hutter spoke at a symposium on gene therapy at Washington University in St. Louis. Scientists are studying whether gene therapy can be used to rid the body of HIV.

Brown, 46, was diagnosed with HIV in 1995. In 2006, he developed leukemia while living in Germany. Hutter performed a blood stem cell transplant using a donor with a rare gene mutation that provides natural resistance to HIV. Hutter said that resistance transferred to Brown.

Brown said he feels great, has not needed HIV medication since the 2007 surgery, and is now active in a foundation that seeks a cure for HIV.

Hutter said enough time has passed to say without hesitation that Brown is cured, citing the same five-year standard after which some cancer patients are said to be cured.

Brown, who now lives in San Francisco, grew up in Seattle and moved to Germany in 1993. After the HIV diagnosis he started on medication to prevent him from developing full-

blown AIDS.

He was attending a wedding in New York in 2006 when he became unusually tired. An avid cyclist, within weeks he could barely ride the bike and eventually was diagnosed with leukemia.

Brown underwent chemotherapy but needed a blood stem cell transplant and turned to Hutter, a blood specialist at Heidelberg University.

Hutter suggested they seek a donor with a certain cell feature that gives them natural resistance to HIV infection. Only about 1 percent of the northern European population has this feature. Hutter theorized that a transplant from such a donor could make the recipient resistant to HIV.

View post:
Doctor claims cure for HIV, cites stem cell treatment

Stem cells restore hearing to deaf gerbils

Eighteen gerbils were given a drug to make them deaf in one ear, before being given an injection of 50,000 progenitor cells into the cochlea, which translates sounds into nerve impulses which can be sent to the brain.

On average about a third of the cells grafted themselves to the ear and replace the damaged nerve cells. Brain scans showed that the gerbils typically recovered 45 per cent of their hearing after 10 weeks.

In humans this would translate to someone who could formerly not hear a lorry passing by their window gaining the ability to follow a conversation in a crowded room, researchers said.

They added that the results were variable, with some gerbils recovering up to 90 per cent of their hearing and others seeing very little improvement, depending on how many of the cells took hold.

More research is needed to establish that the benefits of the treatment are lasting and that it is safe for use on humans, but the study represents a "huge step forward" in deafness research, the team said.

Dr Marcelo Rivolta, who led the study, said: "Stem cells have been used in animal models of deafness before, mostly the mouse, with different results, but none have shown functional recovery. What we have shown here is functional recovery using human stem cells, which is unique.

"It is difficult to say when we might be able to treat patients. We are hoping in a few years, but first we need to understand more about the biology of the system and whether it is sustainable in time and safe."

View original post here:
Stem cells restore hearing to deaf gerbils

Vitro Biopharma Signs Agreement With StemGenesis for Distribution of Its Stem Cell Products in China

GOLDEN, Colo., Sept. 12, 2012 (GLOBE NEWSWIRE) -- Vitro Diagnostics, Inc. (VODG), dba Vitro Biopharma, announced an agreement with StemGenesis, Inc. (http://www.stemgenesisinc.com) for the exclusive distribution of its products into the following Chinese Provinces: Shanghai, Beijing, Tianjin, Jiangsu, Shandong, Zhejiang, Anhui, Fujian, Hebei, Liaoning and Heilongjiang. The agreement provides for distribution of the entire Vitro Biopharma product line under specific terms and conditions. The agreement covers Vitro Biopharma's adult mesenchymal stem cell (MSC) lines & derivatives, the MSC-Gro(TM) brand of MSC cell culture media and the LUMENESC(TM) assay kits for the determination of MSC potency, quality and response to toxic agents. StemGenesis, Inc. was incorporated in Nevada in 2011 and now has US and Chinese operations through facilities located in Sacramento, CA and Qingdao, Shandong Province, China. StemGenesis is a medical device company with focus on blood transfusion, blood banking and stem cell products. The goal of the company's presence in China is to work with the government at different levels to enhance quality of cell therapy as well as stem cell therapy by introducing the state-of-the-art technologies and products from developed countries.

Dr. Jim Musick, Vitro Biopharma's President & CEO, said, "We are very pleased to announce our distribution agreement with StemGenesis, Inc. There are substantial markets for our stem cell-based products within China and we now have an appropriate partner for us to pursue these markets. I met with StemGenesis at the annual ISCT meeting held in Seattle, WA in June of this year and we have mutual interest in serving the needs of the stem cell industry through the development, manufacture and distribution of high-quality medical devices serving specific needs within stem cell research and clinical development. This vision is now shared through our distribution agreement and there are also opportunities for us to expand this collaboration. "

The CEO of StemGenesis, said, "I recently visited the operations of Vitro Biopharma in Golden, Colorado and we are very excited to work with Vitro Biopharma. The current market demand in China for the advanced stem cell products and technologies is developing too fast to be met. As an experienced cell therapy medical device company having offices in both US and China, we provide full technical service and product support. We carefully select the best products from US and Europe to market in China. We believe Vitro Biopharma's products, especially its stem cell culture media and assays, will offer high quality products with unique features for our Chinese customers. We are looking forward to collaborating with Vitro Biopharma."

About Vitro Biopharma

Vitro Diagnostics, Inc. dba Vitro Biopharma (VODG) (http://www.vitrobiopharma.com), owns US patents for production of FSH, immortalization of pituitary cells, and a cell line that produces beta islets for use in treatment of diabetes. In 2011, Vitro out-licensed its intellectual property related to treatment of infertility to Dr. James Posillico, a renowned expert in Assisted Reproductive Technologies. Vitro also owns a pending US patent for generation of pluripotent stem cells and an additional pending patent for methods of mesenchymal stem cell (MSC) generation and related materials. Vitro's mission is "Harnessing the Power of Cells(TM)" for the advancement of regenerative medicine to its full potential. Vitro operates within a modern biotechnology manufacturing, R&D and corporate facility in Golden, Colorado. Vitro manufactures and sells "Tools for Stem Cell and Drug Development(TM)", including human mesenchymal stem cells and derivatives, the MSC-Gro(TM) Brand of optimized media for stem cell self-renewal and lineage-specific differentiation. Vitro recently formed a strategic alliance with HemoGenix(R), Inc. (http://www.hemogenix.com/) to jointly manufacture and distribute the LUMENESC(TM) quantitative assay for determination of MSC quality, potency and response to toxic agents.

About StemGenesis, Inc.

StemGenesis (http://www.stemgenesisinc.com) is an US-registered cell therapy technology company. Based in California, the company's business focuses on the market in great China including mainland China, Taiwan and Hong Kong. StemGenesis is involved in two cell therapy-related businesses: medical devices and cell therapy.

In the medical device business, we offer the following services to our customers:

In our cell therapy business, we provide the following services:

Safe Harbor Statement

See the original post:
Vitro Biopharma Signs Agreement With StemGenesis for Distribution of Its Stem Cell Products in China

Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Two slightly different viral vectors were tested in the study, each modified to deliver healthy ADA genes into the bone marrow cells of the patients so the needed enzyme could be produced and make up for the cells that don't have the gene. Four of the 10 patients in the study remained on their enzyme replacement therapy during the gene therapy study. There were no side effects, but their immune systems were not sufficiently restored, Kohn said.

In the next six patients, the enzyme therapy was stopped and a small dose of chemotherapy was given before starting the gene therapy to deplete the ADA-deficient stem cells in their bone marrow. Of those patients, half had their immune systems restored. The human findings confirmed another study, also published recently in Blood by Kohn and UCLA colleague Dr. Denise Carbonaro-Sarracino, which tested the techniques in parallel, using a mouse model of ADA-deficient SCID.

The rest is here:
Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Originally posted here:
UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'