When my son Roman Reed was first paralyzed, and lying in the hospital bed, he developed a pressure sore on his right heel: harmless-looking, at first, just a black spot the size of a dime.
But the darkness grew, spreading until it covered his foot, and higher.
The doctor prescribed “moon boots” to take the pressure off the heel. Then he took a marker pen, and made a circle around Roman’s ankle, saying:
“If the blackness spreads above that line, we will have to amputate.”
Imagine being told that one of your legs must be cut off: amputated.
One possible cause of such a nightmare prognosis? Critical Limb Ischemia (CLI), hardening of the arteries.
For many reasons, including the aging of our society, and the increasing numbers of diabetics at risk of limb amputations, CLI is an increasingly common threat.
“…Critical Limb Ischemia…may be present in as many as… two million Americans… it (may) result in amputation due to wounds that refuse to heal….”
Right now, surgery is our main defense. Angioplasty (balloons pushed into the arteries) may clear out the “hardness”. The blockage may be cut with tiny knives. Stents may be used, new tunnels for the blood to flow.
But many such operations are unsuccessful. Amputation may still be required——and even that may not save the patient’s life.
Could there be a stem cell weapon? Spain and California wanted to find out.
John Laird and Jan Nolta of the University of California at Davis cooperated with Immaculada Herrador of the Hospital Universitario Reina Sofia in Spain.
How could they fight this terrifying condition?
With the help of a growth factor (VEGF, Vascular Endothelial Growth Factor) the body might be able to grow new arteries.
VEGF alone would not do the job; it might not go where it was needed.
But what if there was a microscopic “emergency vehicle”, to seek out the problem?
Mesenchymal stem cells (MSCs) can do that. Put into the body, MSCs travel to the trouble: locating the blockages in arteries and veins.
Add growth factor (VEGF) to the mesenchymal stem cells (MSC) and you have MSC/VEGF— the “paramedic van”, self-directing and full of good medicine!
As the scientists put it, in their progress report:
“Mesenchymal stem cells…are remarkably effective delivery vehicles, moving robustly through the tissue, infusing therapeutic molecules into damaged cells.”
Could it work?
“…Injections of MSC…have rapidly restored blood flow to the limbs of rodents who had zero circulation in one leg.
“We propose to use these MSCs as “nature’s own paramedic system”, arming them with VEGF to enhance…blood vessel growth.”
Scientists from California visited the lab in Spain, and vice-versa, making sure the joint effort would be the same, when the human trials began. The only difference would be how the MSC/VEGF was administered: Spain would use an injection into the blood; UC Davis would give the shot into the muscle.
Multiple safety and effectiveness experiments were carried out, each side bearing its own costs. The scientists intended to complete pre-clinical studies, move toward (FDA) regulatory approval, and start the clinical trials.
At the close of the 3rd year of their grant: “We have successfully engineered human MSCs to produce (high levels of) VEGF… and received…approval to proceed to the FDA… for the proposed stem cell gene therapy trial…”
However…while the first grant was awarded, the second was not.
And thereby hangs research: no grants, no science. Someone has to pay for the test tubes.
Why was the grant denied? I am not sure. That kind of information is generally kept private.
But Dr. Nolta shared with me her belief that the grant was turned down because more and different animal tests were required: a seemingly fixable problem.
In the meantime, the University of California system provided funding to develop the required new animal model.
UC Davis is one of California’s premiere sites for stem cell research, and has been awarded designation as an Alpha Clinic to administer promising clinical trials. They may have been denied one grant, but I have no doubt they will try again.
As Dr. Nolta put it: “Thanks in large part to… the California taxpayers who voted for Prop 71, stem cell therapies are changing the way medicine is done…
“In the future, when the mysteries of stem cell therapies are more fully understood, patients may not have to endure such…barbaric surgical techniques as limb amputation.”—Jan Nolta, personal communication.
As for Roman’s leg, it stayed the same for a long time, his foot and ankle black and blue like a bruise—and then, gradually, the darkness faded away…
I wish that success for everyone.
This post originally appeared on HuffPost.
Don C. Reed is Vice President of Public Policy for Americans for Cures, and he is the author of the forthcoming book, CALIFORNIA CURES: How California is Challenging Chronic Disease: How We Are Beginning to Win—and Why We Must Do It Again! You can learn more here.