QLT announces clinical & regulatory update for oral retinoid program for inherited retinal disease

QLT announced that, following meetings with the U.S. Food and Drug Administration and the European Medicines Agency, the Company believes that it is close to finalizing a pivotal trial protocol for QLT091001 for the treatment of inherited retinal disease such as Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) due to mutations in the LRAT and RPE65 genes, both orphan indications.

The Company expects to provide final guidance on its development plans in these indications before the end of the first quarter of 2014 after final feedback from the European regulatory agency.

Additionally, QLT has initiated recruitment of subjects for a Phase IIa proof-of-concept trial of its drug candidate, QLT091001, in adult subjects with Impaired Dark Adaptation (IDA), a condition that results in decreased ability to recover visual sensitivity in the dark after exposure to bright lights.

The Company also announced the launch of a compassionate use program for QLT091001 in LCA and RP, as well as plans for a patient registry and an update on its retreatment study in these indications.

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Govt accepts Ranjit Roy panel report on approval of new drugs, clinical trials & banning of drugs

The Union Health Ministry has accepted the recommendations of the Prof. Ranjit Roy Chaudhury expert committee.  The committee was constituted by the ministry in February this year to formulate policy and guidelines for approval of new drugs, clinical trials and banning of drugs.

According to senior officials in the ministry, the recommendations of the expert committee were discussed in a meeting with its members recently. During the meeting, clarifications on certain recommendations were obtained from the committee. After the meeting, the ministry in-principle accepted the recommendations of the committee. 

Accepting the recommendations of the expert panel for accreditation of ethics committees, investigators and the clinical trial sites, the ministry decided that in order to strengthen the clinical evaluation of new drugs, the clinical trials should be conducted in accredited sites by accredited investigators with the oversight of accredited ethics committees (Ecs). As this is a long term measure, in the meantime, Quality Council of India (QCI) will be considered for creating a system for accreditation of investigators, ethics committee and clinical trial sites. Although the Drugs & Cosmetics Rules, 1945, already provide for registration of ethics committee, accreditation of such committees will be undertaken following a specific procedure. 

As this requires amendments in the Drugs & Cosmetics Rules, as an immediate measure, CDSCO would initiate steps relating to the process of accreditation by constituting an expert body of 20-25 experts. The names of experts will be finalized by CDSCO in consultation with Dr. Ranjit Roy Chaudhury, Dr. Y. K. Gupta, Prof. & Head, Dept. of Pharmacology, AIIMS, New Delhi and Dr. Arun Aggarwal, Prof. of ENT, Maulana Azad Medical College, New Delhi, senior officials in the ministry said. 

Accepting the recommendations of the committee on the procedure for review of applications of clinical trials and new drugs, officials said that the New Drug Advisory Committees (NDACs) will be renamed as Subject Expert Committees. The members for their meetings will be drawn randomly from a large pool of experts. Applications of clinical trials and new drugs will initially be evaluated by the Subject Expert Committees and their recommendations will be reviewed by the Technical Review Committee (TRC). The TRC will be constituted under DGHS and consisting of experts from each areas i.e. clinical pharmacology, regulatory clinical toxicology/ pathology, medicinal/ pharmaceutical chemistry, pharmacy and immunology including clinicians, basic scientists involved in drug development and subjects specialists (drug indication wise). CDSCO will grant approval of clinical trial and new drugs based on the recommendations of TRC.

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UT Arlington researchers exploring better methods for gene therapies to fight the sight-deteriorating disease Retinitis Pigmentosa.

UT Arlington researchers are exploring a better method for initiating certain gene therapies that could better fight the sight-deteriorating disease retinitis pigmentosa. This research is funded by the National Institutes of Health.

Dr Samarendra Mohanty, Assistant Professor of Physics, at UT Arlington, is focused on using near-infrared ultrafast laser beam to deliver genes that allow expression of light-sensitive proteins, called opsins, into specific cells. That proteins’ expression allows researchers to influence neural activity through optical or light stimulation – a technique known as optogenetics.

In the past, the genes have been delivered to cells by virus. That method can have drawbacks, such as immune responses, in addition to the benefits. In Mohanty’s method, a laser beam creates a transient sub-micrometer size hole, which allows for the gene encoding the proteins to permeate through the cell membrane. It can limit the risk of immune response, as well as delivering larger genes than viral methods, he said.

The scientists claim that with the minimally invasive near-infrared method, DNA and other impermeable molecules can be effectively delivered only to the places most required. In retinitis pigmentosa, the peripheral retina begins to lose light sensitivity due to loss of photoreceptors. With this method, a laser can deliver the genes only to the required part of the retina, making those neurons respond to light again. This method will be more effective than a virus, where the genes are delivered everywhere, potentially causing complications in areas that are already working fine.

Optogenetic stimulation also holds promise for influencing neurons in the brain. Scientists, including Mohanty’s research group, are researching ways it could be used to understand how the brain works or to intervene in case of neurological disorders or to affect behavior.

Ultimately, Mohanty’s team has a goal of creating all optical, or light-based, control and monitoring of cell activity. So, in addition to the light-assisted delivery of genes, the researchers also will work on refining methods for stimulating the neural activity using near-infrared and visible light. Some of those methods are described in a recently published paper called “Fiber-optic two-photon optogenetic stimulation,” which appeared in the journal Optics Letters.

Mohanty’s lab at UT Arlington also will use a method called phase-sensitive interferometry, to monitor the changes in neurons that result from the activation by light. The interferometry method is called “label-free” because unlike fluorescence, it uses the change in behavior of light rays, rather than staining, to track changes at the sub-nanometer level.

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Technology to detect early diabetic retinopathy

Yissum Research Development Company, the technology transfer arm of the Hebrew University of Jerusalem, has introduced a method for detecting retinal micro-aneurysms that potentially pose a high risk for leakage of blood and blood products, which can lead to visual impairment in diabetic retinopathy (amongst other retinal vascular diseases). This method is likely to enable early diagnosis and treatment of the condition, potentially minimizing damage and saving vision. 

The technology has been patented by Yissum and is currently searching for an appropriate partner for the further development and commercialization of the invention. 

Dr. Yaakov Nahmias and his team from the Center for Bioengineering at the Hebrew University of Jerusalem developed a computational method to identify microvascular regions with high risk of leakage based on fluid dynamics. The method will enable ophthalmologists to identify microaneurysms with a high risk of leakage using adaptive optics. The scientists found that high risk is correlated with increased level of a protein called Von Willebrand factor (vWF), which has been associated with early development of diabetic retinopathy.

Retinal microaneurysms are dilations of small blood vessels in the retina, and an early feature of diabetic retinopathy, one of the leading causes of blindness worldwide. Retinal microaneurysms reduce vision because they eventually cause fluid leakage from blood vessels and retinal edema. Localized leakage can be detected and treated using laser ablation slowing the progression of diabetic blindness.

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Stem cells for treating Diabetic Retinopathy

An article, recently published in the PLOS, refers to pericytes derived from adipose-derived (or in lay terms, fat-derived) stem cells (ASCs), being able to integrate with retinal vasculature, and providing functional vascular protection in animal models.

(c) Robert M Hunt

Pericytes are contractile cells that wrap around endothelial cells of capillaries and venules throughout the body. These cells regulate capillary blood flow, the clearance and phagocytosis of cellular debris, and the permeability of the blood-retinal barrier. A deficiency of pericytes can cause the barrier to break down, leading to leakage of blood and other products.

Diabetic individuals often exhibit loss of pericytes in their retina. This is a characteristic sign early on in the disease. These cells are important for the protection of endothelial cells in the retina. With their loss, microaneuryms form in the capillaries, which is the earliest clinical sign of diabetic retinopathy.

ASCs have been found to differentiate into pericytes, which suggests they may be useful as protective and regenerative cellular therapy for retinal vascular disease.

The PLOS study was undertaken by Thomas A Mendel and his colleagues, a majority of whom are from University of Virginia, Charlottesville. They found that when ASCs were injected intravitreally (inside the eye) into a murine eye subjected to oxygen-induced retinopathy (OIR) (which resembles diabetic retinopathy), the cells were capable of migrating to and integrating with the retinal vasculature. ASCs injected after OIR vessel destabilization and ablation enhanced vessel regrowth (16% reduction in avascular area). ASCs injected intravitreally before OIR vessel destabilization prevented more than 50% of retinal capillary dropout (which is commonly seen in patients with diabetic retinopathy). . Injected ASCs also prevented capillary loss in the diabetic retinopathic mouse model (79% reduction observed 2 months after injection).

The authors have concluded that ASC-derived pericytes can integrate with retinal vasculature, adopting both pericyte morphology and marker expression, and provide functional vascular protection in multiple animal models of retinal vasculopathy. The pericyte phenotype demonstrated by ASCs is enhanced with additional (TGF-β1) treatment, as seen with native retinal pericytes. 

The authors feel that ASCs may represent an innovative cellular therapy for protection against and repair of diabetic retinopathy and other retinal vascular diseases.

Advanced Cell Technology initiates higher-dosage patient treatment in European clinical trial for Stargardt's Disease

Advanced Cell Technology, Inc., a pioneer in the field of stem cells for treating conditions such as macular degeneration. has announced treatment of the first patient in the third dosage cohort, and seventh patient overall, in its European Phase I clinical trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). 

The patient was injected with 150,000 hESC-derived RPE cells, as compared with the 100,000-cell dose used in patients of the second cohort. The surgery was performed on Friday, April 19, without any complications, and the patient is recovering uneventfully.

The company declared that they are past the halfway point in all three of their clinical trials on both continents. They have also announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially granted their hESC-derived RPE cells orphan medicinal product designation for the treatment of SMD. This will likely provide a number of benefits to them, as in protection from competition, as well as for the patients through reduced fees.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the study’s primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format.

Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration. Clink here to read more, 

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Phase 2b/3 clinical trial of Emixustat Hydrochloride in subjects with geographic atrophy associated with dry Age-related Macular Degeneration

Acucela Inc., a clinical-stage biotechnology company focused on developing new treatments for sight threatening eye diseases, announced phase 2b/3 clinical trial investigating emixustat hydrochloride in subjects with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) has been initiated. 

AMD is the most common cause of irreversible vision loss in the developed world, the overwhelming majority of which is associated with dry AMD. There are currently no medications approved to treat GA associated with dry AMD. Emixustat hydrochloride is being studied to determine whether it slows the progression of GA in patients with GA associated with dry AMD. 

The SEATTLE study (for Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride) was initiated based on data from the recently completed phase 2a study, the results of which will be announced at the ARVO 2013 Annual Meeting, as well as feedback from the U.S. Food and Drug Administration (FDA).

Emixustat hydrochloride has a unique mechanism of action in visual cycle modulation, offers oral dosing and the ability to specifically target the visual cycle, representing a potentially novel therapeutic approach for the treatment of retinal diseases, such as GA associated with dry AMD.

The SEATTLE study of emixustat hydrochloride is designed as a phase 2b/3 multicenter, randomized, double-masked, dose-ranging study comparing the efficacy and safety of emixustat hydrochloride with placebo for the treatment of geographic atrophy (GA) associated with dry age-related macular degeneration (AMD). Approximately 440 patients with GA associated with dry AMD will be enrolled in the study across 56 sites, primarily in the United States.

There are more than 10 million people in the US and more than 120 million people worldwide who have age-related macular degeneration. AMD is associated with irreversible vision loss, the overwhelming majority of which is due to the dry form of AMD, representing approximately 90% of all cases. Dry AMD occurs when the light-sensitive cells in the back of the eye slowly deteriorate, gradually blurring the central field of vision. As the disease advances, and where patients typically present with GA, the blurred vision slowly progresses to blindness in affected areas of the eye.

To know more about AMD, please click here and here.

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A study for new treatments for wet age-related macular degeneration (AMD)

A University of Wisconsin School of Medicine and Public Health scientist, Dr Nader Sheibani, is spearheading a study of new treatments for wet age-related macular degeneration (AMD). Dr. Sheibani, a professor in the department of ophthalmology and visual sciences, was awarded $6.2 million over five years from the National Eye Institute. In the first year, the collaborators will receive $1.2 million.

The project is a collaboration among Dr. Sheibani’s lab and others in the department of ophthalmology and visual sciences; researchers in the UW department of pediatrics; scientists at Northwestern University Center for Developmental Therapeutics and Feinberg School of Medicine; and the University of Nebraska Center for Drug Delivery and Nanomedicine. Each specialist and team brings different skills and techniques to the question of treatment of exudative AMD.

Wet or exudative AMD is the leading cause of blindness among aging Americans. The global rate of AMD is expected to double in the next decade as the population ages.  A cause of vision loss in this type of AMD is associated with angiogenesis, the growth of new blood vessels which are leaky with severe consequences to vision.

Ophthalmologists have developed treatments known as anti-vascular endothelial growth factor (VEGF) to slow or stop new vessels from forming. The anti-VEGF treatments require frequent injections into the eye. They are an important tool for ophthalmologists to help save vision and their use has demonstrated that inhibiting VEGF slows damage from AMD. However, VEGF is essential for normal ocular integrity and function. Therefore, there is a great need for new treatments which preserve vision without interfering with normal functions.

Dr. Sheibani and his collaborators hope to develop treatments using small peptides that mimic those of the body’s own inhibitors of angiogenesis. These treatments will counter VEGF activity and inhibit vascular growth in eyes with exudative AMD while providing an environment resistant to new vessel growth. 

This study will develop peptide mimetics – shorter versions of the proteins the body makes – that will stop vessel growth. Researchers at Northwestern University are developing the peptides and those at the University of Nebraska are developing the drug delivery nanotechnology. Dr. Sheibani and his staff will test whether the treatments work in preclinical models of the disease for its translation to humans.

The hope is that this class of drugs, because they mimic the body’s own defenses, will be more effective and have fewer side effects than the treatments already available. Although human clinical trials are years away, this research is truly translational, with the potential to greatly impact quality of life by preventing a common cause of vision loss.

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Mum with ROP manages with a guide dog

Wendy Wright thought she was exceedingly clumsy in her early adulthood. The 49-year-old has managed her condition, known as Retinopathy of Prematurity (ROP), for decades, but now has a new-found freedom thanks to guide dog, Freya.

The mum of five has no peripheral vision, a result of her premature birth.

Ms Wright has had Freya (3) for six months and said it was “amazing” how much of a difference the dog had made to her life.

Freya and Ms Wright graduated as a guide dog team on Tuesday.

from www.inmycommunity.com.au

Ms Wright, manager of the |Needle and Syringe Exchange Program, commutes to work in the Perth CBD by public transport with Freya every morning.

“I can walk down the street, turn my head and look into a shop window without walking into the back of someone, if there’s a person there Freya will guide me around them,” she said.

“I had so much trouble using a white cane, I’d walk into people with their iPod headphones in and they’d get annoyed.

“She’s also taken it upon herself to be my companion, which I didn’t expect, I had some very bad news recently and she didn’t leave my side – that bond after six months just amazes me.”

In 2013 the Association for the Blind, which runs Guide Dogs WA, celebrates 100 years of service to the WA community.

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Being the person, not the 'blind' person...

"...The accompanist’s rapidly moving hands on the keyboard spark curiosity about the source of the prominent piano line. But curiosity may linger for those that notice he is not using sheet music. The pianist is Shane Dittmar, a freshman music education major, accompanying the Department of Performing Arts’ production “It Gets Better” last October. For more than four hours, Dittmar played an entirely memorized piano part in the back-to-back productions." from The Pendulum, Elon University's Student News Organization And for 18 years, Dittmar has been living with a disability. Dittmar is blind. Diagnosed with a genetic disease called Leber Congenital Amaurosis (LCA), Dittmar and his twin brother Derek have been blind since birth. Just like Dittmar had to memorize music measures for “It Gets Better,” he has had to memorize innumerable daily methods in order to adjust to life being blind.

Shane has also been trying out musical theatre.

from The North Raleigh News With a flash of casting instinct from the mind of a high school theater director, Shane Dittmar has claimed his home on stage - the musical theater stage, that is. That's not necessarily an unusual thing for a high school sophomore who is a perfect-pitch tenor with a charismatic personality - punctuated by an enviable wit and outstanding student achievement honors. Difference here: Shane is blind.

Some of Shane's videos...enjoy his voice, his music and his wit!