A Cure in Sight?

[billanschell]

Almost everyone on this site is the survivor of a spinal cord injury, in most cases the result of invasive surgery into the spinal cord. Whether your symptoms are pain or loss of function, you can find hope for a cure in efforts by a company called GERON. Below, I'm reprinting text from the company's 2007 Annual Report. I can't say I understand it all, but when I came across this last night, it made me feel a lot more optimistic about the future. I hope it does the same for you...

--Bill

"GRNOPC1 / Glial Cells for Spinal Cord Injury

GRNOPC1 is the lead product candidate within our portfolio of human embryonic stem cell (hESC)-based therapeutic products in development. FDA concurrence of our IND for GRNOPC1 will allow the world's first hESC-based therapeutic product to enter the clinic for human testing. We have published extensively on this approach to repairing spinal neurons damaged by severe spinal cord injury. We have demonstrated that GRNOPC1 cells remyelinate spinal axons in a rodent model of spinal cord injury, leading to durable recovery of function after a single injection of the human cells. We have demonstrated that GRNOPC1 cells secrete neurotropic factors that enhance survival and cause new growth of neurons in the damaged cord. Importantly, we published evidence that GRNOPC1 cells evade direct attack by the human immune system in vitro, reducing the need for immunosuppressive therapy after transplantation. The data in our IND support the safety of the cells, with no teratoma formation, no evidence of significant toxicities and in vivo durability of the myelination. The clinical protocol is finalized, the GMP manufacturing process has been validated and our manufacturing facility has been licensed by the State of California. We are qualifying two completed production lots of GRNOPC1 for potential use in the clinical trial. Needless to say, initiation of this clinical trial is a major milestone for Geron, as well as for the field of regenerative medicine."

[billanschell]

Or Maybe not...

That article was written at the end of 2007. Just last week, the following came out. Disappointing on two fronts: 1) The FDA won't approve (perhaps for good reason); 2) Geron's immediate aim was to treat patients seven to fourteen days post-injury. Most of us are way too late for that, though hopefully the technology could be expanded to treat longer-standing injuries.

UPDATE: Geron Shares Slump On FDA Halt To Stem-Cell Plan
4:13 PM EDT May 14, 2008

DOW JONES NEWSWIRES

Shares of Geron Corp. (GERN) slumped 20% Wednesday after the U.S. Food and Drug Administration placed a hold on the company's embryonic stem-cell application to treat spinal-cord injury.
Such a hold means the agency is delaying a proposed trial or suspending an ongoing investigation.
Geron's stock was recently down 20%, or 98 cents, at $3.91 on volume of 3.9 million compared with average daily volume of 776,500.
"We have not yet received a letter from the FDA explaining the decision to place the submission on hold, so we are unable to comment specifically," said President and Chief Executive Thomas Okarma. "Once we have the letter and have had a discussion with the agency, we will communicate our findings and our thinking to shareholders."
But he expressed disappointment with the move "given the interactions we had with the FDA over four years leading to the filing, and the breadth and depth of the submission, some 21,000 pages."
The biopharmaceuticals firm has been seeking approval to begin testing of embryonic therapies in humans, in Geron's case for treating spinal-cord injuries. It has so far been tested in rats and the company wants to test the therapy in certain patients who have had spinal-cord injuries starting seven to 14 days after the injury.
Steven Bauer, chief of FDA's cell and tissue therapy branch, said last month the agency is considering requiring future therapies derived from human embryonic stem cells to meet tougher effectiveness standards earlier in the clinical development progress than currently required for drugs and medical devices.
Embryonic stem cells have the ability to grow quickly and act like almost any other cell in the body. However, animal tests have shown embryonic stem cells can cause benign tumors called teratomas, and there are concerns the cells have the potential to cause malignant tumors along with other unintended consequences.
-By Kevin Kingsbury, Dow Jones Newswires; 201-938-2136; [e-mail]
(Jennifer Corbett Dooren contributed to this report.)

(END) Dow Jones Newswires
05-14-08 1612ET
Copyright (c) 2008 Dow Jones & Company, Inc.

[msweeney]

I have read about this stem cell therapy. My understanding is that when and if it is ever approved, it could be used on older injuries. The procedure for older injuries would be to go back in surgically and clean out scar tissue and then allow the stem cell therapy to repair the damage in the same way as if it were a new injury. The issue that I had read was the one brought out in the article in Bill's second post. It mentioned the risk of the growth factor that would be introduced into the spinal cord and the side effects that could have (new tumor growth etc...).

My hope is that some day they will get it all worked out and that may be a decade away or two, who knows but hope is hope and we live in a day and age where this stuff may still be in our future!

Hope springs eternal in the human breast;
Man never Is, but always To be blest:
The soul, uneasy and confin'd from home,
Rests and expatiates in a life to come.

-Alexander Pope,

[ScrapHeap]

Thanks a lot for the detailed info and update. I had heard of this only because I was watching Discovery Channel or similar (discovery science i believe) and they touched on this development. I was totally unaware it was so close to the awaiting approval state. I was also completely unaware of the risks of teratomas.

I was like YIPPIE!!! And then I was like BUMMER!!! after reading this post. Much like a roller coaster ride..... the rise up (of instant hope), followed by the rapid descent (of hope). No matter. We have to deal with our various states, post operative, anyway. I try to focus on the bright side. This is a huge step towards a possible cure-possible major improvement potential for us at the very least.

As stated previously, maybe in a few years it can be refined. Being informed is important to me. So again, thanks for that. Even if it wasn't exactly what I had hoped for. I will continue to hold onto hope for a major breakthrough that has less or limited risks/side-effects in the not so distant future.