Harvard professor Doug Melton is having quite a week. His work on potential cell therapies for diabetes just fueled the launch of a well-funded biotech startup, and now he's signed up with AstraZeneca ($AZN) to help the drugmaker spotlight new treatments for the disease.
Under a 5-year deal, Melton's team at the Harvard Stem Cell Institute will use its technology for engineering insulin-producing beta cells to better understand how both types of diabetes develop. The plan is not to use the cells as treatments unto themselves--that's the remit of the newly un-stealth Semma Therapeutics--but instead to probe how the decline of beta cell function leads to diabetes and tap AstraZeneca's vast library of compounds to see if a small-molecule drug can disrupt the process.
As HSCI explains, patients with Type 1 diabetes endure the destruction of beta cells at the hands of their immune systems, while Type 2 is driven by faulty or outmatched beta cells. Scientists have spent years investigating the details of each process, but limited access to testable human beta cells has made that a difficult proposition, according to AstraZeneca. Now, using Melton's proprietary method for turning stem cells into insulin-generating beta cells, the company believes it may have found the key to a new approach to diabetes R&D.
To get started, AstraZeneca's cardio division will simultaneously fund work in Melton's lab and set up an in-house team at its Mölndal, Sweden, outpost. The two groups will work together on fleshing out the biology behind the degradation of beta cells and use high-throughput screening to find drug candidates that could restore them.
"Professor Melton's group has made an extraordinary breakthrough in the differentiation of human stem cells into human beta cells and our scientists are extremely excited to be working alongside his team," Marcus Schindler, AstraZeneca's head of cardiometabolic R&D, said in a statement. "Harnessing this new technology has the potential to transform the research and development of new treatments for patients with diabetes."
Meanwhile, Semma, the other offshoot of Melton's discovery, is getting started with $44 million in venture and partnership cash with hopes of developing a stem cell therapy for Type 1 diabetes. Using the process Melton's team described in a Cell paper, the biotech plans to engineer human beta cells in the lab and transplant them into patients, thereby reversing the root cause of the disease. And Semma plans to design a device that will allow for the safe introduction of those cells into the body without the need for immunosuppression, hoping to circumvent a common pratfall in cell therapy.
- read the announcement