Biologics

Stem Cell Cures Any Closer?

Twelve years after the discovery of the human embryonic stem cell, research is finally picking up steam. Over the past 7 years, the National Institutes of Health have almost tripled its investment in stem cell research to more than $1 billion.

CBS News medical correspondent Dr. Jon LaPook reports the number of embryonic stem cell lines funded by the government has doubled – from 21 to 44 today. More than 200 U.S. companies are researching stem cells. So the report card on stem cell research is promising — but incomplete.

Two years ago, scientists found another way to create stem cells from ordinary skin cells. That breakthrough lets researchers study diseases in a dish. For example, skin cells from a patient with ALS have been turned into the kind of nerve cells attacked by the disease. For the first time, those living nerves can be studied – outside the body – to figure out what goes wrong.

“It’s amazing,” said Larry Goldstein, director of the stem cell program at UC San Diego. “What can you say?”

Goldstein said it’s like investigating a plane crash. “What you’re really looking for is the black box of diseases. What’s the black box of Alzheimer’s disease?”

Once researchers figure out what goes wrong in diseases, they can test drugs in the safety of a petri dish before trying them on patients.

“We’re building foundations right now, so the science is moving along very well,” Goldstein said.

The Problem
The problem? How to harness the tantalizing potential of stem cells.

No one knows that better than Dr. John Kessler. Nine years ago, a phone call changed his life forever. His 15-year-old daughter Allison was paralyzed from the waist down in a skiing accident.

“It was a moment of despair,” Kessler said. “It was a moment of knowing what I faced – what my daughter faced.”

He knew because he’s a neurologist and is now a leader in the field. He’s turned stem cells into nerves and helped heal animals with spinal injury.

“Is this a cure for spinal cord injury? No,” Kessler said. “This is not yet a cure for spinal cord injury. Do we think that its very likely this will help people? We think so.”

Today, Allison is an outgoing 25-year-old. Her father’s greatest wish is that one day his work will help her walk again.

“If there is a cure to find at this day and age with current science – he’s the one who is going to push the envelope,” Allison said.

Allison is in medical school now, and has moved on with her life. Her father however said he’ll “never get over it, never.”

The Solution

The solution can’t come fast enough for patients desperate for help now.

Later this year, Dr. Hans Keirstead of UC Irvine, working with Geron corporation, will begin the first FDA-approved trial of human embryonic stem cells to treat paralyzed patients – within two weeks of injury.

In spinal cord injury, the insulation coating the wires in the cord is destroyed. Electrical messages from the brain can’t get past the point of injury – and function is lost.

“It’s like you have a bunch of wires that are short-circuiting,” Keirstead said. “The tissue that’s left doesn’t work – because they can’t conduct electricity up and down your cord.”

Keirstead and his team figured out a way to turn stem cells into a cell that makes insulation for the spinal cord.

Millions of these cells will be injected into the damaged area. The hope is by restoring the insulation, the spinal cord will work again.

“I expect incremental benefits to the humans that get this,” Keirstead said. “I think they are going to get better, and I’m just dying to find out if I’m right.”

Stem cells have even given scientists a brand new way to think about cancer.

“We think that there are these cells at the root cause of cancer called cancer stem cells,” said Dr. Catriona Jamieson

Stem cells are normally present throughout our bodies and help us replace worn-out cells. But these normal stem cells can produce cancer stem cells that create regular cancer cells and tumors. While chemotherapy kills regular cancer cells as they divide, cancer stem cells survive by lying dormant, then springing to life and causing relapse.

“Getting rid of the most robust cell within the cancer that has the capacity to regenerate the whole cancer is how to really expunge the whole cancer and make sure that people don’t relapse,” Jamieson said.

This new approach may have saved Theresa Blanda’s life. In 2008, tests showed she was close to developing leukemia. Eighteen months ago, she enrolled in a clinical trial using a drug that Jamieson and her team developed. It attacks cancer stem cells in her blood – now she’s back to a normal life.

“I don’t think I imagined myself sitting here, period,” Blanda said.

Even if everything goes right, stem-cell based treatments are still years away. But this may be one of those rare instances in medicine when the hype is actually deserved.

Josh Sandberg

Josh Sandberg is the President and CEO of Ortho Spine Partners and sits on several company and industry related Boards. He also is the Creator and Editor of OrthoSpineNews.

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