September 19, 2016 at 1:23 pm #47319
Historic Spinal Cord Stem Cell Treatment Is a Game Changer for Biotech
Dear TransTech Reader,
The Keck Medical Center of USC recently released news about a patient who participated in a clinical trial of stem cells for treatment of complete cervical spinal cord injury. Included was video of Kris Boesen, a former quadriplegic, lifting a barbell above his head.
Since I first wrote about this breakthrough, people have e-mailed me saying that stem cells couldnt have caused Boesens improvement. They theorized that Boesens spinal cord hadnt been completely severed and thought he must have naturally recovered.
This FDA-approved clinical trial required that only patients with catastrophic spinal cord injury could participate. It could have been possible, though, that a single patient was misdiagnosed by the experienced and highly qualified clinical and surgical team.
All 8 patients in the trial show great improvement
Data has now been released about all eight clinical participants with catastrophic spinal cord injury. Boesen wasnt an anomaly. His results werent even the most impressive. All eight attained benefits that are both spectacular and historic.
The first three patients, in fact, only received the same small number of stem cells used in earlier mouse studies. Yet, they have shown some improvement.
The other five quadriplegics received half the therapeutic dose. They have gained enough upper body movement to care for themselves in just three months (the trials FDA guidelines allow twelve months).
This kind of recovery just doesnt happen. My cousins husband, for example, suffered this sort of spinal cord damage decades ago due to an off-road vehicle accident. Today, he can barely move his arms while floating in a swimming pool.
We dont yet know if larger doses given sooner to patients will lead to full sensation and movement in the lower body. I hope so, of course, but this therapy is already a breakthrough. If it can turn quadriplegics into paraplegics, it borders on miraculous.
Quadriplegics need full, constant care for their entire lives. This impacts their personal lives in ways that are hard to contemplate. It also imposes millions of dollars in care and medical costs per patient. Paraplegics, though, can take care of themselves and work productive jobs.
Participants in this trial who received only half the therapeutic dose can now text and feed themselves. For more data, you can find the link to a detailed PowerPoint presentation using information included in the press release.
So what does it mean?
Clearly, this will be transformational for people suffering spinal cord injuries. About 15,000 spinal cord injuries occur annually in the US alone. Those who have less catastrophic injuries may respond to stem cell treatment even better.
Though markets dont seem to have processed that fact, this breakthrough will change medicine as we know it. The larger implication is that the field of embryonic or pluripotent stem medicine will finally overcome the skepticism and controversy that have plagued it for over twenty years.
The stem cell controversy
From the start, embryonic stem cells (eSCs) have been at the center of a scientific and ethical debate. For that reason, many people vested hope in adult stem cells. We now know, though, that this hope was largely misplaced.
It had been thought that adult stem cells can do everything that eSCs do but without the moral complexities. Biologically, this isnt true. Embryonic cells have complete regenerative ability. They can also engraft, which means they can integrate themselves into other tissues. The cells used in the spinal cord trial were able to do that.
Adult stem cells release compounds that aid healing, but they dont engraft. They also age and over time must be replaced with new adult stem cell lines. This does not just raise issues of cost and safety. Most people dont know that embryonic stem cells become adult stem cells about eight weeks after conception. This is when the embryo quite suddenly becomes a fetus. It seems that some stem cell companies that tout their use of adult stem cells actually use cell lines derived from aborted fetuses.
eSCs are derived from unused fertilized ovum created to treat infertility using in vitro fertilization (IVF). These cells are routinely discarded following IVF, which is a common procedure. According to sources cited in Forbes, there are about 350,000 IVF babies born each year. There are likely more than 900,000 people who were IVF babies now alive in the US.
Embryonic or pluripotent stem cells are biologically immortal. This means they can replicate under the right conditions essentially forever. So the handful of eSC lines approved for government funding under President George Bush and Barack Obama will never have to be replenished. In theory, they could form the basis of treatments for an infinite number of patients forever.
Induced pluripotent stem cells are superior to eSCs
In practice, eSCs are obsolete. Their use will end simply because there is now a superior alternative. Today, scientists can create cells that are basically identical to embryonic stem cells using a process of genetic engineering called induced pluripotency. My skin cells have been transformed into induced pluripotent stem cells (iPSCs). These cells are virtually identical to the embryonic cells I am derived from.
Some of those cells are being used for research. Many millions are being stored cryogenically in a lab in Northern California. More can be replicated at will. Each could, in the right conditions, become a copy of me. This means that I could be a real life Jango Fett. (So far, no one seems interested, but I remain hopeful.)
So, why are we still using eSC lines?
The reason is that government doesnt allow iPSc therapies and restrictions on federal funding make it extremely unlikely that eSCs will be replaced by iPS cells in the near future. There are, though, thousands of scientists working with pluripotent stem cells today. Now, they have a model in this successful spinal cord therapy. At present, there are ongoing stem cell trials for Age-related Macular Degeneration (dry AMD). This is the most common cause of blindness in older people.
In animal studies, pluripotent stem cells are being used to repair hearts. These cells will engraft heart muscles that have been impaired by aging or damaged due to heart attack. This is good news.
Other scientists are making rapid progress in orthopedics because of pluripotent stem cells. Unlike adult stem cells, these cells have the genetic intelligence to become whatever connective tissue is needed to repair aged joints and related arthritis.
In fact, every organ in the body, including the skin, is now a target for regeneration by pluripotent stem cells. In time, regenerative medicine will significantly extend health spans and cut the cost of age-related diseases. For that reason alone, we should be excited about the historic results from this first phase of the still ongoing spinal cord stem cell therapy.
Editor, Transformational Technology Alert
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