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Stem Cells May Be Next Frontier for Diabetes Drugmaker Novo

  • First clinical trials could start within five years, CEO says
  • Billionaire Sean Parker is among those pursuing type 1 cure

Novo Nordisk A/S, the world’s biggest maker of drugs to treat diabetes, says it’s making progress on the long road to a potential cure.

Novo’s stem cell research may enter clinical trials within five years, Chief Executive Officer Lars Rebien Sorensen said in an interview. The target is a lifelong form of diabetes in which the body’s defense system attacks and destroys cells that make insulin -- the hormone tasked with converting blood sugar into energy. “This will happen,” said Sorensen, who will retire in coming days after 16 years at the helm of the Danish drugmaker.

Novo and others have toiled for years to devise a way to put healthy insulin-producing cells back into patients, a revolutionary process that would effectively cure a disease that afflicts millions worldwide. Recent advances in stem cell science are leading to new ways to tackle the condition, according to the Harvard Stem Cell Institute, which brings together more than 1,000 scientists to accelerate development of treatments.

A solution would be costly, and any success would mark a significant shift for Novo, which has long focused on medicines that patients inject. Novo’s products include the insulin Tresiba and the blockbuster Victoza, part of a class of drugs that stimulate the pancreas to produce more of the hormone.

Shielding Cells

ViaCyte Inc., based in San Diego, said earlier this year that the first clinical trial of a stem-cell therapy in patients with the lifelong form of diabetes known as type 1 had yielded positive preliminary results. The company at the same time acquired the rights to the assets of Johnson & Johnson’s BetaLogics unit. The two are collaborating, and ViaCyte expects to have announcements on its progress early next year, the company wrote in an e-mail.

Research led by Douglas Melton, a Harvard professor, led to the discovery of a method to generate billions of functional, insulin-producing cells, according to Semma Therapeutics, a company he helped start. A partnership between Semma, the Harvard stem cell group and others is planning a clinical trial, and the first transplant is expected to be at least three to four years away. 

Scientists hope one day to use stem cells -- nascent cells that can be made to grow into many different types of tissue -- to treat diseases, yet few therapies have been proved in rigorous trials. A U.S. bill that President Barack Obama signed into law Tuesday may make the path smoother for some and allow regenerative therapies that target serious diseases to get to patients faster.

Cure Mice

“Major wins” in the last 12 months raise hopes for Novo’s stem cell research, Mads Krogsgaard Thomsen, Novo’s chief science officer, said in an interview also at the company’s headquarters in Bagsvaerd, Denmark last month. Novo now can cure diabetic mice with embryonic stem cells that it developed into insulin-producing cells, although “there is still some way to go to take this into humans,” the company wrote in an e-mail.

The challenge when transplanting the cells into patients to replace destroyed ones is that they will be assaulted by the immune system. So the company said it’s working with groups it declined to identify on technology to encapsulate the cells to protect them.

Stem-cell research probably won’t defeat diabetes anytime soon. Trials may take another five to 10 years and the result will probably be too expensive to yield mega-blockbusters, according to the Novo executives. Facing pricing pressure in the U.S., Novo said recently it would end a program to deliver insulin in a tablet, seen as the “holy grail” in diabetes.

That hasn’t stopped people from trying. On the contrary, the more obscure form of diabetes is drawing some big names from the business and academic worlds. Billionaire Sean Parker, the former president of Facebook Inc., donated $10 million last year to create a research center at the University of California at San Francisco that he hopes can find a cure in five years, while drugmaker AstraZeneca Plc is working with the Harvard group to search for potential new medicines that could restore insulin-producing cell activity.

About 5 percent of the estimated 29.1 million Americans with diabetes suffer from type 1, according to the U.S. Centers for Disease Control and Prevention. The disease usually strikes in childhood, adolescence or young adulthood. The remainder have the more common type 2, where the pancreas doesn’t produce enough insulin.

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