Friday, January 22, 2010

Promising new treatment for dry AMD in clinical trials

Acucela Inc. has announced Phase 2 clinical trials of it’s ACU-4429 for the treatment of dry age-related macular degeneration (AMD). ACU-4429 works by decreasing the levels of toxic products in the eye thus hopefully stopping the advance of dry AMD.

More than 29 million people worldwide are affected by either “wet” or “dry” AMD. The leading cause of the loss of vision in people over the age of 50 is dry AMD. 90 percent of AMD patients suffer from the dry form of AMD. Currently, there are no FDA approved therapies to treat dry AMD which will make the trials of ACU–4429 ones eagerly watched. Anti-oxidants are the only therapy available to slow or halt the progression of dry AMD

Researchers are pleased with the preclinical and early clinical data for ACU-4429. Thus far, it has demonstrated the ability to decrease toxic by-products which have had a part in the progression of dry AMD.

ACU-4429 is administered as an oral, daily pill unlike other therapies for the eyes which can involve the use of injections into the eyes.

For more information go to www.maculardegenerationassociation.org

Sunday, January 10, 2010

Laureate Pharma and Iconic Therapeutics complete manufacturing hI-con1 recombinant Fc-Factor VII fusion protein

Laureate Pharma, Inc., a full-service biopharmaceutical development and protein production company specializing in the development and GMP manufacture of monoclonal antibodies, fusion proteins and other therapeutic protein products, and Iconic Therapeutics, a company focused on the development of treatments for wet adult macular degeneration (wet AMD) and other ophthalmic diseases, today announced the completion of the manufacture of the first GMP lot of hI-con1(TM) recombinant Fc-Factor VII fusion protein.
"We are very excited that Iconic Therapeutics now has material to test this new therapy for patients with macular degeneration and other diseases," said Robert J. Broeze, Ph. D., President & CEO of Laureate Pharma. "This is a testimonial to our strong partnership with Iconic and our expertise in working with fusion proteins." "Due to the unique properties of Iconic's hI-con1(TM) fusion protein product, this project involved very close collaboration between our process-development team at Laureate and Iconic's product-development team," added Michiel E. Ultee, Ph.D., Vice President of Process Sciences.

"With clinical material now in hand, we have moved a giant step forward in our plans for hI-con1(TM). We are excited about its prospects as a novel and powerful approach for the therapy of wet AMD," said Kirk Dornbush, President of Iconic Therapeutics. "We are very pleased with the strong working relationship that we established with Laureate Pharma."

hI-con1(TM) is a recombinant protein intended to specifically attack and destroy pathological blood vessels (PBVs) with no effect on normal blood vessels. Since wet AMD is characterized by the invasion of PBVs into the eye, a drug that destroys these blood vessels could be very useful in treatment of this disease. In addition, hI-con1(TM) is thought to act differently from other wet AMD treatments, which reduce the effects of pathological blood vessel invasion into the eye but do not appear to kill those blood vessels. In studies with mice and pigs, a single injection of hI-con1(TM) into the eye resulted in dramatic reduction of pathological blood vessels.

For more information go to WWW.MACULARDEGENERATIONASSOCIATION.ORG

Monday, January 4, 2010

New research findings may help stop age-related macular degeneration at the molecular level

Scientists discover the relationship between 2 blood proteins plays a pivotal role in staving off the condition

Researchers at University College London say they have gleaned a key insight into the molecular beginnings of age-related macular degeneration, the No. 1 cause of vision loss in the elderly, by determining how two key proteins interact to naturally prevent the onset of the condition.

In a paper to be published in a forthcoming issue of the Journal of Biological Chemistry, the team reports for the first time how a common blood protein linked to the eye condition reins in another protein that, when produced in vastly increased amounts in the presence of inflammation or infection, can damage the eye.

"By starting to understand these interactions in greater detail, we can begin to devise methods that will ultimately prevent the development of blindness in the elderly," said Zuby Okemefuna, the lead author of the paper to be published Jan. 8.

Age-related macular degeneration, or AMD, is painless but affects the macula, the part of the retina that allows one to see fine detail. One form of the debilitating condition, known as "wet" AMD, occurs when abnormal and fragile blood vessels grow under the macula, leaking blood and fluid and displacing and damaging the macula itself. The second form, "dry" AMD, occurs when light-sensitive cells in the macula slowly break down.

It is believed that both forms start on a common molecular route and then deviate into dry or wet AMD, explained the research leader, Steve Perkins.

"The earliest hallmark of AMD is the appearance of protein, lipid and zinc deposits under the retinal pigment epithelial cells," he said, adding that the yellowish deposits, usually discovered by an ophthalmologist, are commonly known as "drusen."

The researchers studied two proteins involved in drusen formation -- blood protein Factor H and a second blood protein known as C-reactive protein -- and showed that Factor H binds to C-reactive protein when C-reactive protein is present in large amounts, as in the case of infection, to reduce the potentially damaging effects of an overactive immune system.

"In the eye, during the normal processes of aging, cells will die naturally for all sorts of reasons," Okemefuna said. "The blood supply to the eye will bring C-reactive protein with it, and a low level of C-reactive protein activity will enable the normal processes of clearance of dead cells at the retina through mild inflammation. In conditions of high inflammation, the levels of C-reactive protein in the retina will increase dramatically."

Uncontrolled C-reactive protein activity causes damage to the retina, which is followed by more inflammation and then even more damage to the retina, and so forth.

"It's the debris of broken up retinal cells, some of which is caused by this cycle, that is deposited as drusen," Okemefuna said.

The team also found that a genetically different form of Factor H does not bind to the C-reactive protein quite as well as the normal one, making people who carry the modified protein more vulnerable to an immune system attack in the eye and, thus, drusen buildup.

"In normal individuals, further damage to the retina by prolonged exposure to high levels of C-reactive protein is prevented by Factor H. C-reactive protein also prevents Factor H from clumping together and initiating the processes that lead to drusen formation," Perkins said. "Both these 'good' activities of Factor H are much reduced in the genetically different form of Factor H."

While there is no known cure for AMD, existing therapies aim to treat the symptoms and delay progression.

"It is interesting how the interaction of these two blood proteins protects the eye during crisis," Perkins said. "The two proteins also can be involved in a rare and often fatal cause of kidney failure in children. We now are better positioned to begin to work out preventative strategies for these diseases."

For more information go to www.maculardegenerationassociation.org