Last August, the trial was placed on hold after studies showed that some mice had developed cysts at the injury site in previous studies.

The US food and Drug Administration has advised Geron that positive data from an ongoing preclinical study using Geron’s stem cell therapy, may be used to release the current hold on the study.  A lot of work is ahead though for Geron.

If and when the clinical hold is released for Geron, there will be many hurdles to cross.  I expect it may be awhile before the study’s move from the thoracic level to cervical level injuries.

It will be interesting to hear what Geron has to say about these trials at their next quarterly report.

“Aug 27 (Reuters) – Stem cell research company Geron Corp (GERN) said animals injected with its experimental cell therapy for subacute spinal cord injury developed microscopic cysts in the injury site.  However, a manufactured lot of the drug GRNOPC1 that was assessed using new markers and assays showed no cysts in a recently concluded study in rats, the company said in a statement.”

Geron’s IND application, and the trial, was placed on hold last week, as this data was presented to the FDA for further discussion.

I’m not a medical doctor, researcher, or part of the FDA, but I will be surprised if this stem cell trial is allowed to continue.  I was really hoping this trial would show some promising results, but it looks as if it will end before it even gets started.

Next.

High scrutiny will be placed on this historic first-time procedure in the U.S.  Stem cell therapies on humans with sci have been ongoing in other parts of the world such as China, but results have been poorly documented

Here’s a video with Dr. Tom Okarma, and a short blurb of the announcement.

This is a great first step, but as a quadriplegic for 14 years, I know it will be a slow process.  Possibly they’ll have a cure within ten years for me. That’s my timeline now for chronic sci survivors.

President Obama has guaranteed he’ll use the Executive Order overturning President Bush’s stem cell policy, so that should help research in the United States.

Do both administrations support the promise of Embryonic stem cell research?

Republicans and McCain – Palin:  NO

From the 2008 GOP Platform under “Funding Medical Research”, you will find the line, “In that regard, we call for a major expansion of support for the stem-cell research that now shows amazing promise and offers the greatest hope for scores of diseases – with adult stem cells, umbilical cord blood, and cells reprogrammed into pluripotent stem cells – without the destruction of embryonic human life.  We call for a ban on human cloning and for a ban on the creation of or experimentation on human embryos for research purposes.”

The new Republican Administration under McCain not only want’s to stop funding, but completely ban embryonic stem cell research.

Democrats and Obama – Biden:  YES

On page 23 of the 2008 Democratic Platform, you’ll find the line, “We will lift the current Administration’s ban on using federal embryonic stem cells that would have otherwise been discarded and lost forever — for research that could save lives.”

The new Democrat Administration led by Obama want’s an increase in funding for embryonic stem cell research.

In my opinion, we should be pursuing both embryonic and adult stem cell research.  The untapped potential of both research paths may likely help millions of people.

When Obama was asked, “What is your position on government regulation and funding of stem cell research?”  He answered,

“Stem cell research holds the promise of improving our lives in at least three ways—by substituting normal cells for damaged cells to treat diabetes, Parkinson’s disease, spinal cord injury, heart failure and other disorders; by providing scientists with safe and convenient models of disease for drug development; and by helping to understand fundamental aspects of normal development and cell dysfunction.

For these reasons, I strongly support expanding research on stem cells. I believe that the restrictions that President Bush has placed on funding of human embryonic stem cell research have handcuffed our scientists and hindered our ability to compete with other nations. As president, I will lift the current administration’s ban on federal funding of research on embryonic stem cell lines created after August 9, 2001 through executive order, and I will ensure that all research on stem cells is conducted ethically and with rigorous oversight.

I recognize that some people object to government support of research that requires cells to be harvested from human embryos. However, hundreds of thousands of embryos stored in the U.S. in in-vitro fertilization clinics will not be used for reproductive purposes, and will eventually be destroyed. I believe that it is ethical to use these extra embryos for research that could save lives when they are freely donated for that express purpose.

I am also aware that there have been suggestions that human stem cells of various types, derived from sources other than embryos, make the use of embryonic stem cells unnecessary. I don’t agree. While adult stem cells, such as those harvested from blood or bone marrow, are already used for treatment of some diseases, they do not have the versatility of embryonic stem cells and cannot replace them. Recent discoveries indicate that adult skin cells can be reprogrammed to behave like stem cells; these are exciting findings that might in the future lead to an alternate source of highly versatile stem cells. However, embryonic stem cells remain the “gold standard,” and studies of all types of stem cells should continue in parallel for the foreseeable future.

Rather than restrict the funding of such research, I favor responsible oversight of it, in accord with recent reports from the National Research Council. Recommendations from the NRC reports are already being followed by institutions that conduct human embryonic stem cell research with funds from a variety of sources. An expanded, federally-supported stem cell research program will encourage talented U.S. scientists to engage in this important new field, will allow more effective oversight, and will signal to other countries our commitment to compete in this exciting area of medical research.”

What’s the most important thing in life?  Our car, our house,  the economy, taxes, our money, or our health, and the health of our loved ones?

With a 20,000+ page Investigational New Drug Application submitted, it’s bound to take some time determining how to monitor the studies.

At this point, Geron has not received the written explanation for the clinical hold. Thomas Okarma, Geron’s president and chief executive stated, “Once we have the letter and have had a discussion with the agency, we will communicate our finding and our thinking to shareholders.”

It would be fantastic if the study was steamrolled through.. found successful, and I had gained back my independence. We all know that this is going to be just one more long, patient wait though. Spinal cord injured individuals are accustomed to that.

I do  agree with Don Reed of stemcellbattles though.  If this is some devastating restriction in the 25th hour, supporters of this research must be ready to fight.

I’ve been spinal cord injured for almost fourteen years. It’s refreshing to finally see therapies moving from making rats and mice walk, to potentially help some like myself walk again.

The Student Society for Stem Cell Research comments, ‘The FDA hearings are particularly timely, as Geron is expected to begin human clinical trials using embryonic stem cells for the treatment of spinal cord injury this summer.

“We are talking about weeks, not years, before we see the first clinical trials using embryonic stem cells. This is inspiring to our generation and empowers our hope that the next generation will not have to endure many of today’s dreaded conditions for which stem cell research holds promise,” said Marion J. Riggs, SSSCR Founder.’

The human clinical trials proposed by Geron will be conducted on “acute”, or newly injured individuals.

“* The Reeve-Irvine Research Center (RIRC) has treatments in development that address all 3 phases of SCI; acute, sub-acute and chronic. Since SCI represents a population too small for drug companies to consider profitable, RIRC scientists, like Dr. Hans Keirstead, find characteristics that SCI has in common with other conditions. By grouping these conditions together, 2 important goals can be achieved for people with SCI:
o Create a large enough population to be profitable to big pharma;
o Shorten the trial length of each treatment. Instead of following a person with SCI for 10 – 20 years or more for data, they can use shorter trials in these other conditions.

* Safety studies with IP10 antibodies in ulcerative colitis should report soon. FDA approval for UC will allow this drug to be used off-label in Acute SCI. In animal models, as much as 70% return was noted when IP10 antibodies are given within 6 – 10 hours of SCI.

* In early 2008 Geron is planning to file an IND with the FDA and undertake safety studies using differentiated stem cells that form oligodendrocytes (these remylinate the spinal cord). The Phase I trial will be for people with new SCI, 1-2 weeks post injury.

Multiple studies addressing chronic SCI are underway at the RIRC:

* Animal testing with human embryonic stem cell derived motor neurons is underway. Human clinical trials await FDA approval – those discussions begin January, 2008. The first trials will likely be for infants with SMA-1, and then adults with end-stage ALS. This treatment is also for chronic SCI, without limit to the age of the injury. Decades-old SCIs may benefit from the treatment. The Geron experience taught Dr. Keirstead and team, which is moving the hESC motor neuron therapy forward, a great deal about the FDA approval process and they have been able to streamline their processes to move through the FDA system faster. This will be beneficial to the chronic trials.

* RIRC Director, Oswald Steward, is currently testing FDA approved drugs in chronic cervical SCI models. These drugs were developed for other applications, like heart disease and cancer, but have been found to be neuro-protective in fish models. Dr. Steward is testing 7 drugs now, but has many more ready to go. If any of these show efficacy in rat models, we can very quickly, in a matter of months, go to humans in a Phase II trial because the FDA has already determined them safe.

* Dr. Kim Anderson works with care issues such as sexual dysfunction and the metabolic effects of SCI that make us more prone to diabetes and other conditions. She discussed her current pilot study regarding diabetes in the SCI population.

* We have often talked about how funding is THE obstacle to curative therapies. Dr. Keirstead was careful to point out that funding is not the only issue. FDA approvals also play a pivotal role in how fast or slow a therapy will be tested

* If FDA approvals go as planned, we can reasonably expect chronic SCI clinical trials in humans in 2-3 years with treatments potentially available in as few as 5 years. These predictions are dependant on several factors: funding; FDA approvals and efficacy in humans to follow previous animal models.

* Research for Cure accepts donations for RIRC and Dr. Keirstead.

* Hopefully, everyone reading this will be inspired to raise funds and awareness for SCI research. Tania Cusack and Dr. Maura Hofstadter are the contacts at RIRC that can help with fundraising and research questions.”

“I am pleased to report that the Seventh Annual Research for Cure Dinner was a huge success, due to 1. Dr. Keirstead’d presentation plus his easy manner of cruising through the attendees, allowing everyone an opportunity to speak to him about their own concerns. 2. The efforts of a growing number of volunteers.
Below is a brief synopsis of Dr. Keirstead’s presentation. It lacks the details and the excitement that Dr. Keirstead has for this work, which is quite contagious.

Keirstead worked with Dr. Thomas Lane at UCI, whose interest is multiple sclerosis, to develop a means of reducing the inflammatory response immediately after trauma or injury to the nervous system. Together they developed a treatment for multiple sclerosis, which Dr. Keirstead’s group then adapted for use in acute SCI. A phase 1 clinical trial began in 2006 on individuals with ulcerative colitis (same inflammatory mechanism that causes degeneration), and is funded by Medarex Corporation.

Using hESCs, Dr. Keirstead’ team has had great success turning them into oligodendrocytes, with a purity level above 95%. Injection of the cells into sub-acute paralyzed rats (within 2 weeks of injury) resulted in improvement of function. Four other laboratories replicated this approach. Clinical trial dates have been pushed forward several times, as this will be the first hESC therapy clinical trial and safety is the primary consideration. Being the first trial, new procedures and standards have been created and revised and revised again. Clinical trials are now scheduled to begin in 2008, and will be funded by Geron Corporation.

Dr. Keirstead also has a team working on scar tissue removal. Scar tissue is the reason that the oligodendrocyte treatment outlined above will only work on acute injuries – there is no scar tissue in acute injuries. If the scar tissue is removed, the sub-acute injuries become like acute injuries and treatments successful on acute injuries may be successful.

Chronic studies are being pursued with a passion equal to my own for treatments. Motor neurons, in conjunction with cAMP and a “designer” virus, is hoped to restore function in chronic SCI (months to decades after injury). This is another wonderful example of collaboration equaling faster answers to recovery treatments. Dr. Keirstead and collaborators from California Stem Cell Inc. have put their knowledge together and now can produce clinical grade human motor neurons in large volumes at 95% purity. Dr. Keirstead has provided these cells to Dr. Doug Kerr at Johns Hopkins, and both have both begun animal testing. Because of Dr. Keirstead’s experience with the FDA during his work with Geron Corporation, the path now exists to move through the process much quicker. The first human trials are planned for babies with spinal muscular atrophy. These poor babies generally only live about 12 months, and die as a result of motor neuron death within the spinal cord. There are currently no treatments for the disease. The trials would be relatively quick, as the babies’ lifespan is so short. As with other clinical trials, once a treatment has been proven safe for humans, it can be used for other conditions.

We all left with hope, based on science and, hopefully, a desire to help politically for increased funding for hESC research, or a desire to volunteer time in efforts to raised money or, if able, donate money towards this exciting research OR all three!”

Thanks Karen!

Once again, President Bush has vetoed a bill that would allow NIH funding for stem cell research. His narrowminded view will only accomplish stalling medical research. His legacy will show a President more interested in allowing Americans to die, than live or be cured.

The Coalition for the Advancement of Medical Research (CAMR) writes, “With this veto, President Bush ignored the overwhelming majority in the medical, scientific, and spiritual communities who believe stem cell research holds the key to unlocking the future of better treatments and cures.

“With this veto, President Bush turned the page to start yet another chapter in a Presidential legacy of errors in judgment and bad decisions that needlessly cost American lives in the process.

“More than 100 million Americans suffer from debilitating diseases and disorders for which embryonic stem cell research holds great promise. Despite our anger and disgust at this second, most misguided veto, we will continue to do whatever we can to advance embryonic stem cell research in America . We will always be grateful for the strong, bipartisan support on this issue in both the U.S. Senate and House of Representatives, and among the American people.”

It’s a shame that stem cell research in the United States has been slowed down by the narrow viewpoint of one individual. If one of his daughters, or close family members sustained a spinal cord injury, I wonder if he’d change his tune.