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What once was lost could be found
Under a three-year deal funded by AstraZeneca, the company's researchers will team up with scientists at the UCL Institute of Ophthalmology to work on new medicines that use the regenerative capacity of stem cells. The institute of higher learning and the pharma hope to come up with a compound in three to five years, followed by clinical development. If successful, the innovative stem cell solution could hit the market in 10 years.
"These tools could be used either to manufacture transplantable material or to directly stimulate new cell growth in the eye to help restore or improve the vision of those with DR," says Dr. Marcus Fruttiger, senior lecturer of the UCL Institute of Ophalmology and leader of the project.
The majority of these patients have type 1 diabetes, and 20 to 30 percent of these individuals will become blind, Fruttiger says. In addition, at least 50 percent of patients with the more common type 2 diabetes will also develop retinopathy over time.
With the rapid spread of type 2 diabetes, which is closely linked to obesity, the need for a retinopathy treatment becomes even more urgent, as more than 438 million people are expected to suffer from diabetes by 2030, Fruttiger says. A study published this year by Oxford University predicted that eight out of 10 men and almost seven in 10 women will be overweight or obese by 2020. The grim forecast is a 98 percent rise in obesity-related diabetes by 2050.
"The prevalence of vision threatening diabetic retinopathy amongst diabetics is 4.4 percent," Fruttiger tells ddn. "This gives you around 10 million diabetics with sight-threatening DR."
The regenerative medicines to be developed in this collaboration are unique in the treatment of this disease.
"That's because diabetes causes damage to the microvasculature, leading to ischemic areas in the retina," Fruttiger says. "This can cause vision degeneration. In a subgroup of patients, an additional complication occurs where blood vessels start to proliferate in an aberrant manner. This complication can be reduced by using laser burns to destroy parts of the retina and there are currently also anti-angiogenesis efforts in progress (i.e. anti-VEGF therapies). However, for the initial microvascular degeneration (the majority of DR patients), there is currently no cure."
The approach of trying to rebuild the damaged microvasculature using stem cell technology is novel, and if it works, it would be the first treatment for non-proliferative DR, Fruttiger says.
If this collaborative effort is successful, its commercial potential would be huge, he adds.
"This obviously depends on how well it will work," Frittiger says. "If the approach is successful, DR may be a model for the repair of many different types of microvascular diseases associated with diabetes. Considering there are 200 to 300 million diabetics worldwide (and many of them in developed countries), there is probably a reasonably big commercial potential."
The partnership is a good fit, he adds.
"At the UCL Institute of Ophthalmology, we have extensive experience and an established track record in basic research relating to eye disease and stem cell biology," Frittiger says. "AstraZeneca, on the other hand, has strong capability in drug screening and long-standing translational experience. This collaboration between academia and industry aims to optimally exploit the strength of both partners."
Alan Lamont, director of Sciences and Technology Alliances at AstraZeneca, says the company believes that regenerative medicine offers new opportunities to develop innovative, more effective and safer therapies, as well.
"We're getting very keen on the whole area of regenerative medicines, which will be part of our research development over the next few years," Lamont says.
Over the next few years, stem cell technology could contribute to a measurable improvement in our ability to discover and develop candidate drugs, and to target those drugs to the right patient population through a better understanding of the disease process.
"We are delighted to be combining our drug hunting expertise with the pioneering research ongoing at UCL," Lamont says.
Prof. Pete Coffey of the UCL Institute of Ophthalmology adds: "This is a great collaborative opportunity, and we're delighted to be working with AstraZeneca to explore the potential of harnessing the regenerative capacity of stem cells to develop a cellular therapy for diabetic retinopathy … AstraZeneca has a proven track record in developing medicines, and their involvement in this type of research is very exciting."
AstraZeneca's U.S. rival, Pfizer, also has a partnership with Coffey and the UCL Institute of Ophthalmology for another eye condition—macular degeneration.
"It's great that Big Pharma is considering regenerative medicines as a serious possibility," Coffey says. "This is British science being developed into a commercial entity with the pharmaceutical industry. It's a good example why the government shouldn't cut funding for biomedical research."
While this is the first time that AstraZeneca has worked on medicines for retinopathy, diabetes has been an area of focus. The company has a new diabetes treatment on the market called Onglyza, which was developed with Bristol-Myers Squibb Co., and the companies are developing a second diabetes drug that could be submitted to regulators for approval later this year.