June 9, 2014

New Hope: Stem Cell Trial for ALS Patients

By centerforregmedmc

From the pages of Mayo Clinic Magazine.

Ted Haakonson

Ted Haakonson

Regenerative medicine at Mayo Clinic harnesses its collective knowledge, resources and skills to teach the body to heal from within. This unprecedented science offers patients definitive solutions for devastating diseases and conditions.


Patient Number Five

“We thought, ‘jeez, this cure has to start somewhere, someone has to be patient zero,’” says Ted Haakonson. He is patient number five in the Center for Regenerative Medicine’s clinical trial testing stem cells to treat amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease).

He was shocked when his local doctor told him he had ALS two years ago. The only symptom he had noticed was a weak thumb. But by the time the 49-year-old received the stem cell treatment at Mayo Clinic a year later, both his hands were weak, his legs twitched uncontrollably and he fell asleep several times a day.

Within a couple of weeks, his legs became steadier and his energy levels rose.

“I’m of the belief that it has helped quite a bit,” Ted says.

“The rate of progression seems to have slowed, and I’m still doing whatever I want to do. Some things like baking and woodworking take a lot longer, but I hate to think of where I’d be right now if I hadn’t been accepted into the study or gotten the stem cells. It was like winning the lottery.”


Engineering a Cure

In ALS, nerve cells in the brain and spinal cord progressively degenerate and eventually die, leaving the brain unable to control muscle movement. There is no cure. But Mayo Clinic’s Anthony J. Windebank, M.D., deputy director-Discovery of the Center for Regenerative Medicine, and Nathan P. Staff, M.D., Ph.D., are providing new hope.

The team in the Human Cell Therapy Lab takes cells from fat tissue and re-engineers them into stem cells. They then enhance the ability of these cells — technically known as mesenchymal stem cells — to promote nerve health.

As part of the country’s first phase one stem cell trial for ALS patients, the team placed these cells directly into Ted’s spinal fluid, believing the stem cells would secrete growth factors and protect nerve cells from further degeneration.

The investigators quickly caution that the primary goal of phase one studies is to measure safety, which makes the investigators grateful to participants like Ted who selflessly enter the unknown. The investigators also caution that not all trial participants describe the same improvements as Ted, though they are confident the technology is on the right track to benefit patients with this uniformly fatal disease.

Learn more about how researchers are studying the use of intraspinal deliver of mesenchymal stem cells to the cerebral spinal fluid of patients with ALS using a dose-escalation study below:

Tags: ALS, clinical trial, Lou Gehrig, regenerative medicine, Research, stem cells

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