Under a three-year deal funded by AstraZeneca, the company'sresearchers will team up with scientists at the UCL Institute of Ophthalmologyto work on new medicines that use the regenerative capacity of stem cells. Theinstitute of higher learning and the pharma hope to come up with a compound inthree to five years, followed by clinical development. If successful, theinnovative stem cell solution could hit the market in 10 years.
"These tools could be used either to manufacturetransplantable material or to directly stimulate new cell growth in the eye tohelp 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 20to 30 percent of these individuals will become blind, Fruttiger says. Inaddition, at least 50 percent of patients with the more common type 2 diabeteswill also develop retinopathy over time.
With the rapid spread of type 2 diabetes, which is closelylinked to obesity, the need for a retinopathy treatment becomes even moreurgent, as more than 438 million people are expected to suffer from diabetes by2030, Fruttiger says. A study published this year by Oxford Universitypredicted that eight out of 10 men and almost seven in 10 women will beoverweight or obese by 2020. The grim forecast is a 98 percent rise in obesity-relateddiabetes by 2050.
"The prevalence of vision threatening diabetic retinopathyamongst diabetics is 4.4 percent," Fruttiger tells ddn. "This gives you around 10 million diabetics withsight-threatening DR."
The regenerative medicines to be developed in thiscollaboration are unique in the treatment of this disease.
"That's because diabetes causes damage to themicrovasculature, leading to ischemic areas in the retina," Fruttiger says."This can cause vision degeneration. In a subgroup of patients, an additionalcomplication occurs where blood vessels start to proliferate in an aberrantmanner. This complication can be reduced by using laser burns to destroy partsof 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 damagedmicrovasculature using stem cell technology is novel, and if it works, it wouldbe the first treatment for non-proliferative DR, Fruttiger says.
If this collaborative effort is successful, its commercialpotential would be huge, he adds.
"This obviously depends on how well it will work," Frittigersays. "If the approach is successful, DR may be a model for the repair of manydifferent types of microvascular diseases associated with diabetes. Consideringthere are 200 to 300 million diabetics worldwide (and many of them in developedcountries), there is probably a reasonably big commercial potential."
The partnership is a good fit, he adds.
"At the UCL Institute of Ophthalmology, we have extensiveexperience and an established track record in basic research relating to eyedisease and stem cell biology," Frittiger says. "AstraZeneca, on the otherhand, has strong capability in drug screening and long-standing translationalexperience. This collaboration between academia and industry aims to optimallyexploit the strength of both partners."
Alan Lamont, director of Sciences and Technology Alliancesat AstraZeneca, says the company believes that regenerative medicine offers newopportunities to develop innovative, more effective and safer therapies, aswell.
"We're getting very keen on the whole area of regenerativemedicines, which will be part of our research development over the next fewyears," Lamont says.
Over the next few years, stem cell technology couldcontribute to a measurable improvement in our ability to discover and developcandidate drugs, and to target those drugs to the right patient populationthrough a better understanding of the disease process.
"We are delighted to be combining our drug hunting expertisewith the pioneering research ongoing at UCL," Lamont says.
Prof. Pete Coffey of the UCL Institute of Ophthalmologyadds: "This is a great collaborative opportunity, and we're delighted to beworking with AstraZeneca to explore the potential of harnessing theregenerative capacity of stem cells to develop a cellular therapy for diabeticretinopathy … 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 partnershipwith Coffey and the UCL Institute of Ophthalmology for another eye condition—maculardegeneration.
"It's great that Big Pharma is considering regenerativemedicines as a serious possibility," Coffey says. "This is British sciencebeing developed into a commercial entity with the pharmaceutical industry. It'sa good example why the government shouldn't cut funding for biomedicalresearch."
While this is the first time that AstraZeneca has worked onmedicines for retinopathy, diabetes has been an area of focus. The company hasa new diabetes treatment on the market called Onglyza, which was developed withBristol-Myers Squibb Co., and the companies are developing a second diabetesdrug that could be submitted to regulators for approval later this year.