A Major Advance Toward An Alzheimer's Drug

Researchers have demonstrated that a protein can help improve brain and skeletal muscle function in aging mice.

The same researchers previously found that the protein can help failing hearts in mice seem healthier as well.

The findings are contained in two separate papers published in the journal Science.

Professors Amy Wagers, PhD, and Lee Rubin, PhD, of Harvard’s Department of Stem Cell and Regenerative Biology (HSCRB), found that injections of a protein known as GDF11, found in humans as well as mice, improved the exercise capability of mice equivalent in age to that of about a 70-year-old human. It also improved the sense of smell in the older mice, who could detect odors as well as younger mice do.

The researchers said they expect to have GDF11 in initial human trials within three to five years.

The findings were published in Science in two separate papers from Rubin and Wagers.

Both studies examined the effect of GDF11 in differing ways. The researchers used a “parabiotic system,” in which two mice are surgically joined and the blood of the younger mouse circulates through the older mouse. They also injected mice with GDF11. The fact that older mice rejuvenated with the blood of younger mice, which has higher levels of GDF11.

Doug Melton, PhD, co-chair of HSCRB and co-director of HSCI, reacted to the two papers by saying that he couldn’t “recall a more exciting finding to come from stem cell science and clever experiments. This should give us all hope for a healthier future. We all wonder why we were stronger and mentally more agile when young, and these two unusually exciting papers actually point to a possible answer: the higher levels of the protein GDF11 we have when young,” he said.

And Rubin said that “it isn’t out of question that GDF11” or a drug developed from it “might be capable of slowing some of the cognitive defects associated with Alzheimer’s disease, a disorder whose main risk factor is aging itself,” Rubin said.

Wagers said that the two research groups are in discussions with a venture capital group to obtain funding to “be able to do the additional preclinical work” necessary before moving GDF11 into human trials.