Seventy-five years ago, when Lou Gehrig was diagnosed with amyotrophic lateral sclerosis, or ALS, at Mayo Clinic-Rochester, regenerative medicine wasn't a term the public had heard of.

But today, most people have at least a inkling of its meaning. Researchers at Mayo are working urgently in concert with patient volunteers to use regenerative medicine to slow the progression of ALS, also known as Lou Gehrig's disease.

"How can we help the body heal itself?" said neurologist Dr. Anthony Windebank, deputy director for discovery in the Center for Regenerative Medicine at Mayo, during a recent presentation at Mayo Clinic's Research Information Centerin the Gonda Building. "We're trying to release that regenerative ability of the body to heal itself."

That's particularly important for diseases like ALS, the diagnosis of which carries a poor prognosis for survival much beyond even a couple of years.

Neurologist Dr. Nathan Staff, who leads the Phase I ALS stem-cell study, said during the presentation that Mayo's effort involves a diverse team, including patients who volunteer for the study, the Mayo neurological research team, the Clinical Research Unit and the laboratory where stem cells are grown from patients' adipose, or fat, tissue.

A tree has a stem, Staff said. And from that stem can grow branches and leaves.

A human stem cell is kind of like that tree stem.

"It has everything it needs to branch out and become everything else," Staff said. Bone-marrow transplants use stem cells that are able to become any type of blood.

Stem cells from fat or bone can become fat, cartilage, connective tissue or muscle.

The idea of Mayo's study is to use stem cells to protect a patient's healthy cells and to protect the patient's remaining neurons. The goal is not to grow new neurons.

The Post-Bulletin reportedabout Mayo's ALS stem-cell safety study in April of this year. At that time, Staff and Windebank reported that, if the study shows it's safe to give stem cells to patients and shows the safety of various amounts of stem cells given to patients, a Phase II studywill begin with the goal of demonstrating that the treatment is both safe and effective.

If that study shows the treatment actually helps patients survive longer, a Phase III trial will begin, evaluating the treatment for side effects.

Hundreds of people signed up to be research participants in the Phase I and Phase II clinical research studies. Mayo in Rochester is one of three sites that will participate in the Phase II portion of the research. Phase II is double-blind and placebo-controlled, meaning neither researchers nor patients will know if a given patient gets stem cells or a placebo.

That's necessary to prove the treatment produces better patient outcomes.

Patients in the Phase I study donate fat tissue and, from that, the Mayo laboratory collects and multiplies stem cells until there are millions available for injection into the patient's spinal column — up to 100 million cells in the final group out of the 25 patients.

"At this point, it's too early to tell whether there's any benefit," Staff said. But, he said, "we're hopeful — and it doesn't appear that it's hurt anyone."