[aftermathman]

"Ceregene and Genzyme Announce Partnership for the Development and Commercialization of Cere-120 for Parkinson's Disease".

http://www.earthtimes.org/articles/s...e,126456.shtml

IMHO this shows that gene therapy is now a serious proposition and this secures the financial future of CERE-120 through to launch, (assuming it works !!). With the messing about of stem cell research, gene therapy has to be the best game in town. Ceregene, Genzyme, Neurologix, and soon to be joined by Oxford Biomedica, all have promising gene therapy programs at different stages of the product lifecycle, the more horses we have running, the better the odds one will work.

Full text attached.

GO HARD GENE THERAPY.

Neil.

"Ceregene, Inc. and Genzyme Corporation today announced that they have entered into a partnership for the development and commercialization of CERE-120, Ceregene's proprietary lead program for the treatment of Parkinson's disease.

Under the terms of the agreement, Genzyme will pay Ceregene a $25 million up-front payment in exchange for certain partnership-related expenses. Ceregene will also be entitled to development-related milestone payments of up to $125 million and 50 percent reimbursement of the worldwide Phase 3 development costs. Genzyme will gain marketing rights in all markets outside of the US and Canada, and Genzyme will pay Ceregene tiered royalties based on sales in markets where it has rights. Ceregene will retain exclusive rights to CERE-120 in the US and Canada.

"We are pleased to be partnering CERE-120 with Genzyme-a company with a strong international presence and marketing expertise that will enable us to maximize the worldwide commercialization of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "CERE-120, which is currently undergoing a controlled Phase 2 trial in the United States that is being partially funded by the Michael J. Fox Foundation, has demonstrated strong clinical and preclinical data to date and may have the ability to both improve Parkinson's disease symptoms and slow the disease progression. The resources provided by our new partnership with Genzyme, together with the greater than $27 million initial closing of our Series C financing, which was led by Investor Growth Capital and closed in the first quarter, provides the funding we currently need to advance CERE-120 toward commercialization."

"We are very enthusiastic about the potential that CERE-120 may have to improve the lives of people with Parkinson's disease, which currently affects at least one million Americans," said David Meeker, president, Lysosomal Storage Disorders unit, Genzyme Corporation. "We look forward to helping further the development of this exciting approach, building on our long-term commitment to gene therapy and neurodegenerative diseases. This program - which is focused on preserving neuronal function - will complement our existing Parkinson's clinical trial that is currently underway."

"We look forward to working with Genzyme to accelerate the development of CERE-120 in Europe and other areas outside North America," stated Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer, as well as chairman of the partnership's joint steering committee. "Strong synergies and complementary skills and experience exist between Ceregene and Genzyme. Their deep infrastructure in developing and commercializing innovative therapeutic products and their experience working with international regulatory agencies will be invaluable as we expand the development of CERE-120."

In a complementary program, Genzyme is conducting a separate Phase 1-2 clinical trial of a gene therapy for Parkinson's disease designed to restore the therapeutic effectiveness of levo-Dopa by enhancing the brain's ability to convert it into dopamine. In addition, Genzyme has an extensive gene therapy portfolio that includes two additional ongoing clinical trials."

[KC Tower]

Delivery or Take out?

So with something called "Gene Therapy" what sort of medium will it be delivered in and by what mechanism??

Same for "stem cells" ??

These are just classes of possible therapies and say nothing about the delivery.

Personally I think a solution has to be "take out" or pill equivalent and cannot practically use holes in the head or pumps in the chest.

One large hole in my head already ,,, ken

[KC Tower]

A link to a MSNBC article http://www.msnbc.msn.com/id/19355047/

Also Nathan Klien is quoted as saying about the treatment "I'm elated ... I's Unbelievable" and yet continues to take his regular drugs. Would be nice to know him personally and get a view that is not edited or filtered.

Note that this article shows a hole in head delivery system as well.

,,,Ken

[Thelma]

Treatment.......Treatment..........Treatment...... ...this one is exciting.........thlis one is promising.........this one looks good.........whoops this one failed.......treatments for Parkinsons just on the horizon........sorry no treatments in sight for ALS.........maybe we should look into this or that or what.........................

The worst stinking word in the dictionary...............TREATMENT

Why won't Bush help find the cure ?????????????????????????????????

[jeanb]

A novel and promising approach to the treatment of Parkinson's disease is gene therapy, including viral vector-mediated transfer of candidate genes to enhance dopamine production or the survival of dopaminergic neurons. CERE-120 (AAV-hNGF-hNTN) is a novel gene therapy product that delivers a modified human neurturin (NRTN or NTN) gene (i.e., prepro sequence of human neurturin replaced with the prepro domain of human nerve growth factor b [NGFb]) via an adeno-associated virus type 2 (AAV2)

[aftermathman]

in my laymans terms, CERE uses carriers, called "vectors," to transport the genes into cells. "Similar to the relationship between a delivery truck and its cargo, the vector provides a mode of transport and the therapeutic agent provides the disease remedy. These carriers can be either man-made components or modified viruses. The use of viruses takes advantage of their natural ability to introduce DNA into cells. Gene therapy takes advantage of this property by replacing viral DNA with a specific gene." (Neurologix).

So essentially, take a virus, cut out the bad bit, replace it with the gene therapy bit and inject it into the brain.

Advantages over DBS are less invasive, one injection and its over.

KC, Nathan Klein had his own website which is no longer operational but has been archived and is available on :

http://web.archive.org/web/*/http://www.nathanklein.com

let me know what you think.

Thelma, depends on your definition of cure, stem cells won't cure us as they will continue to die over time in the same way our original cells die. I have little hope for a complete cure, I have real hope of good, effective treatments, such as gene therapy can provide, and when you are dx'ed at 40, as I was, that is critical.

Take care,
Neil.

[KC Tower]

Cure comes with a lot of implications which are probably different for everyone.

Cure for me doesnt mean continuing with the same drugs as well.

Injecting into the brain isnt going to fly for the masses.

Operations around here are expensive and hard to obtain in a timely fashion even without involving brain injections and with most covered by medical.

However all attempts are good and hopefully will lead to something practical for all.

take care ,,, ken

[aftermathman]

gene therapy is not a cure but is largely presented (by Neurologix at least) as a replacement for DBS. This is supported by the decision of Medtronic (DBS "stuff" provider) to invest and partner Neurologix.

Injecting into the brain will fly for the masses as this, and worse, is what underpins DBS. Gene therapy application is a much simpler and less invasive procedure.

I don't know the healthcare cover where you live, but if you can get DBS, then in future, all caveats applying, gene therapy should be simpler and cheaper to obtain. Less hospital time, no need for electronic "stuff", less Neuro time, less chance of complications, etc.

Same logic applies to Spheramine.

Neil.

[jeanb]

Neil,

I agree with you - many people now are having DBS surgery. And it doesn't give the hope of improvement - it's yet another form of medicine that treats the symptoms while PD worsens over time. In addition to the surgery to implant the electrodes in the brain, it also involves major surgery every time the battery needs replacing. (I know someone who has to have the batteries replaced every 6 months! )

I believe that people WILL be interested in gene therapy: CERE-120 and GABA as well as Spheramine (and possibly others in the future) as oposed to DBS surgery. I certainly am.

KC - I've given up on the idea of a 'cure' and now am following hopeful treatments that will stop and may (at least temporarily) reverse the progression of the disease.

[AnnT2]

Parkinson' s has become such a part of who we are. I wonder how we will react to being "Normal" again?

Ann