Breakthroughs, breakthroughs... are they finally seeing the beneficial impact to our daily lives?
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Posted: 24 January 2012
PARIS: The first use of embryonic stem cells in humans eased a degenerative form of blindness in two volunteers and showed no signs of any adverse effects, according to a study published by The Lancet on Monday.
Publication in the peer-reviewed journal marks an important step for embryonic stem cells, which were hailed as a miracle cure after they were discovered in 1998 but then ran into technical and political hurdles.
The results of the cautious first-stage test, designed to evaluate whether the treatment is safe, had been previously announced by Massachusetts biotech firm Advanced Cell Technology (ACT) Inc.
The positive outcome in the United States opened the way to the first trials in Europe, which began on Monday.
Embryonic stem cells are extraordinarily versatile cells, found in early-stage embryos, that can differentiate into any tissue of the body.
Scientists have been hoping to turn them into replacement for tissue lost through disease or lost in accidents or war.
The quest to use embryonic stem cells has been arduous.
One problem is biological: that donated stem cells, provoking an immune response, can be rejected by the body or cause cancer. The other is ethical, with moral conservatives contending that an embryo is a human life.
Addressing the biological question, ACT used the stem cells at a so-called "immunoprivileged" site, the eye, where there is not a strong immune response because of a shield known as the blood-ocular barrier.
Around 50,000 embryonic stem cells that had diversified into replacement cells for the pigmented layer of the retina were transplanted into two legally-blind volunteers.
One, a woman in her 70s, had a condition called dry age-related macular degeneration, the leading cause of blindness in the developed world; the other was a woman in her fifties who had Stargardt's macular dystrophy, the commonest form of vision loss among young people.
For the next six weeks, the patients received treatment to prevent their immune systems from attacking the implanted cells, but this was gradually scaled back.
In the first four months, no signs of cancer, rejection or other safety concerns emerged and both patients recovered a little vision, although this was not the point of the test.
At the outset, the older patient was able to read 21 letters on a standard chart of visual acuity. This rose to 33 letters after two weeks before settling at a stable ability to read 28 letters, the study said.
The woman with Stargardt's disease, a former graphic artist, at first could only see hand movements, but this improved after the transplant to being able to see single fingers and to reading five letters of the alphabet.
"However, that doesn't really capture the difference it has made in their life," Bob Lanza, ACT's chief scientific officer, said in an email to AFP.
"The Stargardt's patient reports that she can see more colour and has better contrast and dark adaptation out of the operated eye. In fact, she started using her computer and could even read her watch... (and) says she can even thread a needle now."
Lanza noted that the improvements occurred in patients who were already at a very advanced state of the disease, so the trials were encouraging for patients at an earlier stage of degeneration.
Clinical trials of novel drugs or treatments typically undergo a three-phase process, enrolling a progressively larger number of patients, to make sure they are firstly safe and, secondly, effective.
The Lancet had been scheduled to publish the study on Thursday but released it on Monday as ACT launched its first European trials of the retinal treatment.
Twelve patients with Stargardt's have been cleared by British medical authorities to undergo transplant, with progressively higher doses of cells, at the Moorfields Eye Hospital in London.
- AFP/al
Taken from ChannelNewsAsia.com; source article is below:
Study confirms groundbreaking advance in stem cells
Posted: 17 June 2011
WASHINGTON - The first clinical trials that examine the use of stem cells to treat two forms of blindness are ready to begin now that patients have been enrolled, a US company announced on Thursday.
A total of 24 patients have entered two separate trials at an eye institute in California, said representatives from the Massachusetts-based Advanced Cell Technology (ACT).
ACT was cleared by the US Food and Drug Administration several months ago to begin clinical trials of human embryonic stem cells to treat a form of juvenile blindness known as Stargardt's disease and dry age-related macular degeneration.
Now that patients have been enrolled, the trials will begin "in the very near future," a company spokeswoman said.
The trials aim to check the safety of the treatment before moving on to see whether the therapy can help stop vision loss.
"These trials mark a significant step towards addressing what is one of the largest unmet medical needs of our time, treatments for otherwise untreatable and common forms of legal blindness," said lead investigator Steven Schwartz at University of California Los Angeles Jules Stein Eye Institute.
Dry age-related macular degeneration is the most common form of irreversible vision loss in people over age 55.
There is currently no cure for the disease, which affects around 10-15 million Americans and another 10 million people in Europe, the company said.
Stargardt's disease causes blindness by destroying the pigmented layer of the retina, called the retinal pigment epithelium (RPE). After that follows degradation of photoreceptors, which are cells in the retina that detect light.
Patients often experience blurry vision, difficulty seeing in low light and eventually most lose their ability to see at all. The disease can be inherited by a child when two parents carry the gene mutation that causes it.
The treatment process being tested by ACT worked in animals by creating an abundance of new RPE cells, which are the first cells to die off in Stargardt's and other forms of macular degeneration.
Tests on rats have shown 100 percent improvement in visual performance and "near-normal function" was also achieved in mice, both without negative side effects, ACT has said.
Embryonic stem cell research has been a controversial field ever since the first such stem cells were isolated more than 12 years ago.
Scientists say the cells offer great promise in treating Parkinson's disease, diabetes and a variety of other illnesses. Critics frequently oppose the research on religious grounds because it involves the destruction of human embryos.
- AFP/al
Taken from ChannelNewsAsia.com; source article is below:
First patients enrol in US stem cell trials on blindness
Are we 'seeing' some promise here?
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Posted: 17 June 2011
WASHINGTON - The first clinical trials that examine the use of stem cells to treat two forms of blindness are ready to begin now that patients have been enrolled, a US company announced on Thursday.
A total of 24 patients have entered two separate trials at an eye institute in California, said representatives from the Massachusetts-based Advanced Cell Technology (ACT).
ACT was cleared by the US Food and Drug Administration several months ago to begin clinical trials of human embryonic stem cells to treat a form of juvenile blindness known as Stargardt's disease and dry age-related macular degeneration.
Now that patients have been enrolled, the trials will begin "in the very near future," a company spokeswoman said.
The trials aim to check the safety of the treatment before moving on to see whether the therapy can help stop vision loss.
"These trials mark a significant step towards addressing what is one of the largest unmet medical needs of our time, treatments for otherwise untreatable and common forms of legal blindness," said lead investigator Steven Schwartz at University of California Los Angeles Jules Stein Eye Institute.
Dry age-related macular degeneration is the most common form of irreversible vision loss in people over age 55.
There is currently no cure for the disease, which affects around 10-15 million Americans and another 10 million people in Europe, the company said.
Stargardt's disease causes blindness by destroying the pigmented layer of the retina, called the retinal pigment epithelium (RPE). After that follows degradation of photoreceptors, which are cells in the retina that detect light.
Patients often experience blurry vision, difficulty seeing in low light and eventually most lose their ability to see at all. The disease can be inherited by a child when two parents carry the gene mutation that causes it.
The treatment process being tested by ACT worked in animals by creating an abundance of new RPE cells, which are the first cells to die off in Stargardt's and other forms of macular degeneration.
Tests on rats have shown 100 percent improvement in visual performance and "near-normal function" was also achieved in mice, both without negative side effects, ACT has said.
Embryonic stem cell research has been a controversial field ever since the first such stem cells were isolated more than 12 years ago.
Scientists say the cells offer great promise in treating Parkinson's disease, diabetes and a variety of other illnesses. Critics frequently oppose the research on religious grounds because it involves the destruction of human embryos.
- AFP/al
Taken from ChannelNewsAsia.com; source article is below:
First patients enrol in US stem cell trials on blindness
Posted: 03 January 2011
WASHINGTON: US biotech company Advanced Cell Technology said Monday it was cleared by the government to start its second trial using human embryonic stem cells to treat blindness, this time in older people.
The trial will examine the therapy's ability to safely treat people with a condition known as dry age-related macular degeneration, the most common form of irreversible vision loss in people over age 60.
There is currently no cure for the disease, which affects around 10-15 million Americans and another 10 million people in Europe, the company said.
The Food and Drug Administration (FDA) cleared the Massachusetts-based company in November to begin a similar trial on patients with a progressive form of juvenile vision loss, known as Stargardt's disease.
"ACT is now the first company to receive FDA clearance for two hESC (human embryonic stem cell) trials, and is now a true translational leader in the field of regenerative medicine," said chief executive Gary Rabin.
"It marks a major step forward, not just within the stem cell sector, but, potentially for modern healthcare techniques."
The company hopes to begin the US clinical trials in the coming months, and intends to seek approval for similar trials in Europe. The US and European market for such a treatment amounts to 25 to 30 billion dollars, it said.
ACT's announcement marks the third such trial, after US company Geron broke new ground last year with the first-ever attempt to use embryonic stem cell therapy on a human patient with spinal cord injury.
Embryonic stem cell research has been a controversial field ever since the first such stem cells were isolated more than 12 years ago. Critics oppose the research because it involves the destruction of human embryos.
However scientists say the cells offer great promise in treating Parkinson's disease, diabetes and a variety of other illnesses.
Like the other trials involving human patients, the first step in ACT's Phase I and II trials is to assess whether the therapy is safe before moving on to testing how well it works.
"In a rat model of macular degeneration, we have seen a remarkable improvement in visual performance over untreated animals, without any adverse effects," said Bob Lanza, chief scientist at ACT.
Twelve patients will enrol in the study at various US sites including the University of California, Los Angeles (UCLA) and Stanford University.
The therapy uses retinal pigment epithelial (RPE) cells derived from human embryonic stem cells to replace RPE cells that have broken down in patients with the disease.
Dry age-related macular degeneration, the type that occurs in 90 percent of cases, causes a deterioration in central vision when RPE cells in the patient's macula, in the centre of the retina, lose their ability to function.
Patients often experience blurring in the centre of their field of vision, while peripheral view remains intact.
"As the population ages, the incidence of AMD is expected to double over the next 20 years, further exacerbating this unmet medical need," Lanza said.
-AFP/wk
Taken from ChannelNewsAsia.com; source article is below:US okays stem cell trial on blindness
