Geron mit Hammernews!! (Seite 82)

[Sugar2000]

FDA Greenlights Testing of Geron's Stem Cell Treatment

Just four days into the new administration a small biotechnology company is taking a giant step forward in the field of stem cell research.

Geron is announcing that it has received U.S. Food and Drug Administration clearance to do the first-ever test of an embryonic stem cell treatment in people.

The injection will be given to a small number of patients with a specific type of new spinal cord injury to make sure it is safe and to begin to measure how well it works.

In a press release, Geron CEO Dr. Thomas Okarma is quoted as saying, "This marks the beginning of what is potentially a new chapter in medical therapeutics -- one that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells."

Dr. Okarma will appear in a "First on CNBC" live interview on "Squawk on the Street" at approximately 10:40 a.m. ET Friday.

President George W. Bush prohibited federal funding of embryonic stem cell research. President Obama supports it.



Shares of Geron [GERN 5.21 -0.08 (-1.51%) ] closed eight cents lower on Thursday after hitting a new 52-week high of $5.64 intra-day on heavier-than-normal trading volume.
http://www.cnbc.com//id/28804364?__source=yahoo%7Cheadline%7…

[Sugar2000]

Wahnsinn... ein weiterer Meilenstein...

http://www.finanznachrichten.de/nachrichten-2009-01/12914393…23.01.2009 06:02
Geron Receives FDA Clearance to Begin World’s First Human Clinical Trial of Embryonic Stem Cell-Based Therapy
Geron to Study GRNOPC1 in Patients With Acute Spinal Cord Injury

Conference Call and Video Webcast Set for 6:00 a.m. PST/9:00 a.m. EST

Geron Corporation (News) (Nasdaq:GERN) announced today that the U.S. Food and Drug Administration (FDA) has granted clearance of the company’s Investigational New Drug (IND) application for the clinical trial of GRNOPC1 in patients with acute spinal cord injury.

The clearance enables Geron to move forward with the world’s first study of a human embryonic stem cell (hESC)-based therapy in man. Geron plans to initiate a Phase I multi-center trial that is designed to establish the safety of GRNOPC1 in patients with ”complete” American Spinal Injury Association (ASIA) grade A subacute thoracic spinal cord injuries.

”The FDA’s clearance of our GRNOPC1 IND is one of Geron’s most significant accomplishments to date,” said Thomas B. Okarma, Ph.D., M.D., Geron’s president and CEO. ”This marks the beginning of what is potentially a new chapter in medical therapeutics - one that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells. The ultimate goal for the use of GRNOPC1 is to achieve restoration of spinal cord function by the injection of hESC-derived oligodendrocyte progenitor cells directly into the lesion site of the patient’s injured spinal cord.”

GRNOPC1, Geron’s lead hESC-based therapeutic candidate, contains hESC-derived oligodendrocyte progenitor cells that have demonstrated remyelinating and nerve growth stimulating properties leading to restoration of function in animal models of acute spinal cord injury (Journal of Neuroscience, Vol. 25, 2005).

”The neurosurgical community is very excited by this new approach to treating devastating spinal cord injury,” said Richard Fessler, M.D., Ph.D., professor of neurological surgery at the Feinberg School of Medicine at Northwestern University. ”Demyelination is central to the pathology of the injury, and its reversal by means of injecting oligodendrocyte progenitor cells would be revolutionary for the field. If safe and effective, the therapy would provide a viable treatment option for thousands of patients who suffer severe spinal cord injuries each year.”

The GRNOPC1 Clinical Program

Patients eligible for the Phase I trial must have documented evidence of functionally complete spinal cord injury with a neurological level of T3 to T10 spinal segments and agree to have GRNOPC1 injected into the lesion sites between seven and 14 days after injury. Geron has selected up to seven U.S. medical centers as candidates to participate in this study and in planned protocol extensions. The sites will be identified as they come online and are ready to enroll subjects into the study.

Although the primary endpoint of the trial is safety, the protocol includes secondary endpoints to assess efficacy, such as improved neuromuscular control or sensation in the trunk or lower extremities. Once safety in this patient population has been established and the FDA reviews clinical data in conjunction with additional data from ongoing animal studies, Geron plans to seek FDA approval to extend the study to increase the dose of GRNOPC1, enroll subjects with complete cervical injuries and expand the trial to include patients with severe incomplete (ASIA grade B or C) injuries to enable access to the therapy for as broad a population of severe spinal cord-injured patients as is medically appropriate.

Preclinical Evidence of Safety, Tolerability and Efficacy

Geron submitted evidence of the safety, tolerability and efficacy of GRNOPC1 to the FDA in a 21,000-page IND application that described 24 separate animal studies requiring the production of more than five billion GRNOPC1 cells. Included in the safety package were studies that showed no evidence of teratoma formation 12 months after injection of clinical grade GRNOPC1 into the injured spinal cord of rats and mice. Other studies documented the absence of significant migration of the injected cells outside the spinal cord, allodynia induction (increased neuropathic pain due to the injected cells), systemic toxicity or increased mortality in animals receiving GRNOPC1.

In vitro studies have shown that GRNOPC1 is minimally recognized by the human immune system. GRNOPC1 is not recognized in vitro by allogeneic sera, NK cells or T cells (Journal of Neuroimmunology, Vol. 192, 2007). These immune-privileged characteristics of the hESC-derived cells allow a clinical trial design that incorporates a limited course of low-dose immunosuppression and provide the rationale for an off-the-shelf, allogeneic cell therapy.

Also included in the IND application were published studies supporting the utility of GRNOPC1 for the treatment of spinal cord injury. Those studies showed that administration of GRNOPC1 significantly improved locomotor activity and kinematic scores of animals with spinal cord injuries when injected seven days after the injury (Journal of Neuroscience, Vol. 25, 2005). Histological examination of the injured spinal cords treated with GRNOPC1 showed improved axon survival and extensive remyelination surrounding the rat axons. These effects of GRNOPC1 were present nine months after a single injection of cells. In these nine-month studies, the cells were shown to migrate and fill the lesion cavity, with bundles of myelinated axons crossing the injury site.

Production and Qualification of GRNOPC1

GRNOPC1 is produced using current Good Manufacturing Practices (cGMP) in Geron’s manufacturing facilities. Geron’s GRNOPC1 production process and clean-room suites have been inspected and licensed by the state of California. The cells are derived from the H1 human embryonic stem cell line, which was created before August 9, 2001. Studies using this line qualify for U.S. federal research funding, although no federal funding was received for the development of the product or to support the clinical trial.

Geron’s H1 hESC master cell bank is fully qualified for human use and was shown to be karyotypically normal and free of measurable contaminants of human or animal origin. Production of GRNOPC1 from undifferentiated hESCs in the master cell bank uses qualified reagents and a standardized protocol developed at Geron over the past three years. Each manufacturing run of GRNOPC1 is subjected to standardized quality control testing to ensure viability, sterility and appropriate cellular composition before release for clinical use. GRNOPC1 product that has passed all such specifications and has been released is available for the approved clinical trial. The current production scale can supply product needs through pivotal clinical trials. The existing master cell bank could potentially supply sufficient starting material for GRNOPC1 to commercially supply the U.S. acute spinal cord injury market for more than 20 years.

Intellectual Property

The production and commercialization of GRNOPC1 is protected by a portfolio of patent rights owned by or exclusively licensed to Geron. Patent rights owned by Geron protect key technologies developed at Geron for the scalable manufacturing of hESCs, as well as the production of neural cells by differentiation of hESCs. The fundamental patents covering hESCs are exclusively licensed to Geron from the Wisconsin Alumni Research Foundation (WARF) for the production of neural cells, cardiomyocytes and pancreatic islets for therapeutic applications. The validity of these patents was recently confirmed by the U.S. Patent and Trademark Office in a re-examination proceeding. Geron funded the original research at the University of Wisconsin-Madison that led to the first isolation of hESCs. The production of oligodendrocytes from hESCs is covered by patent rights exclusively licensed to Geron from the University of California. These patent rights cover technology developed in a research collaboration between Geron and University of California scientists.

Conference Call and Video Webcast

Thomas B. Okarma, Ph.D., M.D., will host a conference call and video Webcast presentation for investors and the media at 6:00 a.m. PST/9:00 a.m. EST today. Participants can access the conference call via telephone by dialing 866-783-2145 (U.S.) or 857-350-1604 (international). The passcode is 89631672. The video Webcast presentation is available at http://phx.corporate-ir.net/phoenix.zhtml?p=irol-eventDetail… All participants are encouraged to view Dr. Okarma’s presentation on the Internet. The video Webcast will also be accessible through a link that is posted on the home page of Geron’s Web site at http://www.geron.com. Participants are encouraged to log on at least 15 minutes prior to the beginning of the presentation in order to download any necessary software. The video Webcast will be available for replay through February 23, 2009.

Background information about human embryonic stem cells, GRNOPC1 and the spinal cord injury clinical trial can be found at http://eon.businesswire.com/news/eon/20090122006356/en.

Geron is developing first-in-class biopharmaceuticals for the treatment of cancer and chronic degenerative diseases, including spinal cord injury, heart failure and diabetes. The company is advancing an anti-cancer drug and a cancer vaccine that target the enzyme telomerase through multiple clinical trials. Geron is also the world leader in the development of human embryonic stem (hESC) cell-based therapeutics. The company has received FDA clearance to begin the world’s first human clinical trial of a hESC-based therapy: GRNOPC1 for acute spinal cord injury. For more information, visit www.geron.com.

This news release may contain forward-looking statements made pursuant to the ”safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that statements in this press release regarding potential applications of Geron’s human embryonic stem cell technology constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Geron’s periodic reports, including the quarterly report on Form 10-Q for the quarter ended September 30, 2008.



Contacts:

Russo Partners, LLC
David Schull, 858-717-2310 (Media)
[e-mail]
or
Geron
Anna Krassowska, 650-473-7765
Investor and Media Relations
[e-mail]

[-Salem-]

Geron hat sich extremst gut entwickelt nach dem Low bei 1,9$. Und ich muss ehrlich sagen, bis auf die guten Studiendaten zu GRN163L, gibt es eigentlich nüschts wesentlich Neues. Die Umsätze wieder weit über Average und nicht nur heute rel. stabil. Aber GERN braucht unbedingt Erfolge und Partnerschften, denn Stammzellen+Oligonucleotide sind elendig teure Forschungsgebiete. Und 174 Mios Cash sind zwar solide, werden aber sicher nicht über die volle Distanz reichen.


Übrigens ist Geron zu Gast bei dem Girindus Oligonucleotid Symposium ende März.

[LuckyCat]

Video-Interview mit CEO Tom Okarma zu Embryonalen Stammzellen:

http://news.bbc.co.uk/2/hi/health/7828800.stm


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[LuckyCat]

Nicht lange her, dass Geron Neuigkeiten zum MM-Versuch von GRN163L sowie die Probleme mit der Toxizität (Thombozytenschwund) publizierte: Hier ein paar Fachkommentare:


My take on today's news ...

First a summary of the main result. In one of six ongoing studies, the monotherapy MM study, two patients exhibited bulk tumor telomerase inhibition of 78% and 48%, and 33% and 63% inhibition in the "myeloma stem cell-containing fraction of their bone marrow".

Both of these patients were treated at 4.8 mg/kg, probably with a weekly dosing.

We don't know how many other patients exhibited less TI or no TI and were not reported in Geron's PR. It may be that only a small subset of the MM patients got pre and post treatment bone marrow biopsies, or it may be that all of them did. But it is likely that these two patients had the best TI results from among those tested. We do know that data from 13 of the MM patients was presented at ASH.

According to the ASH MM abstract, "The MTD for continuous weekly dosing of GRN163L in this heavily pretreated, relapsed and refractory MM population appears to be ≥ 4.8 and < 7.2 mg/kg."

So (assuming we can trust the assay) we can say that TI has been seen in at least two 163L-treated patients at or below the MTD.

That is PD. That is molecular activity. That is first base. That is excellent news.

Now ... is it sufficient TI to give rise to clinical efficacy? Any TI has the potential to slow down the cancer growth. From other studies, one needs a rather high amount of TI to actually cause the cancer to regress rather than simply grow more slowly. (Something like 75% for many weeks, if memory serves.) Clearly clinical efficacy has not been close to demonstrated at this point. But let me wax optimistic for a moment.

While there are many unanswered questions, I think we are beginning to get some evidence that there may be a *potential* therapeutic window. To remove the word "potential" it is necessary to show that TI can be converted into some sort of clinical efficacy in humans, such as an increase in the TTP. The therapeutic window may be small, due to the serious AEs seen at and below 7.2 mg/kg and the fact that the MTD is less than 50% above the dose where TI was seen. But there is a very real potential that that the therapeutic window can be widened by playing the dosing games that Geron has been working on for some time.

My initial reaction is that the probability that GRN163L may someday result in commercial sales has been increased from ~10-15% to ~20-25% with this news.

Other views would be most appreciated.

micro


**************************************************************

My quick take on this is that Telomerase Inhibition is not directly causing the thromocytopenia, however since thromocytopenia results from prolonged weekly dosing of GRN163l at levels approaching 7.2mg/kg that is a side effect of GRN163L.
Dr OKarma describes a dosing schedule that includes a skipped week every third week that at least at 4.8mg/kg demonstrated that the progression to thromocytopenia is controlled. He showed graphs of thromocytopenia development in two cohorts, with the alternate dosing showing the platelet count staying above the 50. Thromocytopenia begins at 10. He stated that the alternating dosing schedule should allow dosing beyond the 7.2mg/kg but did no show trial results that demonstrated it.
Although I would like to think that the heavy pretreatment of the patients predisposed the patients to thromocytopenia when dosed with 163 the graph of platelets showed values at initiation that were in the normal range.
However the patients that demonstrated the worse thromocytopenia apparently had previously received bone marrow transplants. This may indicate that the rebuilt blood producing hemeopoetic system is fundamentally weak and more suseptible to the impact of 163.
I find it interesting the LPC is one of the patients that demonstrated Telomerase inhibition of 78% in the bulk cells, but at this time it is unclear whether there has been a beneficial effect. However he was being dosed only at 4.8mg/kg. If we can really get closer to 7.2 mg/kg with alternate dosing, and still get a long enough period of inhibition then the amount of Telomerase inhibition may become very significant.

RX7171




Grüße
LuckCat

[[KERN]Codex]

[meislo]

Aufwärtstrend weiter in takt

[meislo]

Chronologie der Stammzellforschung

1981 In Embryonen von Mäusen werden Stammzellen entdeckt. Sie haben das Potential, sich in jedes Gewebe eines Mauskörpers zu verwandeln.



1996 Das Schaf Dolly kommt zur Welt. Es ist der erste Klon eines Säugetiers, geschaffen aus einer Euterzelle eines erwachsenen Schafs. Erst im Frühjahr 1997 wird das Experiment bekannt.

1998 Im November gelingt es James Thomson erstmals, menschliche embryonale Stammzellen (hES-Zellen) zu züchten. Dafür hat er etwa zehn Tage alte Embryonen zerstört. Die ethische Kontroverse um die Stammzellforschung beginnt, denn die Zellen könnten auch einmal dazu dienen, krankes Gewebe zu ersetzen und defekte Organe zu reparieren.

2002 Ein Team um Rudolf Jaenisch zeigt, dass das "therapeutische Klonen" bei Mäusen funktioniert: Die Forscher klonten dazu eine Maus mit Gen-Defekt, stellten aus dem Klon-Embryo Stammzellen her, reparierten deren Gen-Defekt mittels Gentherapie und pflanzten die gesunden Zellen kranken Mäusen ein.

2004 Der südkoreanische Tierarzt Hwang Woo-Suk wird weltberühmt, weil es ihm angeblich gelungen ist, einen menschlichen Embryo zu klonen und daraus Stammzellen zu gewinnen. Zum Jahreswechsel 2005/2006 wird Hwang jedoch der Fälschung überführt.

2006 Im Frühjahr veröffentlicht der japanische Arzt Shinya Yamanaka ein Rezept, mit dem sich Zellen ausgewachsener Mäuse biochemisch in den Embryonalzustand zurückversetzen lassen. Erstmals lassen sich hES-ähnliche Zellen herstellen, ohne dass dafür ein Embryo zerstört werden muss.

2007 Zehn Monate nach Yamanakas erstaunlichem Bericht kann er sein Rezept verbessern. Viren schleusen dabei vier Gene in die Zellen, die normalerweise nur in embryoähnlichen Zellen aktiv sind. Durch den technisch einfachen Eingriff wird die biologische Uhr der erwachsenen Zellen auf Null zurückgestellt. Die verwendeten Viren und zwei der Gene können jedoch die Entstehung von Krebs begünstigen.

Im November demonstrieren zwei Arbeitsgruppen unabhängig voneinander, dass das Rezept auch mit menschlichen Zellen funktioniert. Wieder ist Yamanaka beteiligt. Die entstehenden iPS-Zellen haben die Fähigkeit, sich in alle Gewebe des menschlichen Körpers zu verwandeln - das können sonst nur embryonale Stammzellen.

2008 Gleich nach dem Jahreswechsel verkündet die kalifornische Firma Stemagen, sie habe einen Menschenklon für einige Tage im Labor wachsen lassen. Stammzellen wurden nicht isoliert.

Im April berichten britische Forscher, dass sie einem Herzpatienten Zellen entnommen und mit dem Yamanaka-Rezept in iPS-Zellen verwandelt haben. Sie tragen dieselben genetischen Informationen wie der Patient und sollen die Krankheit im Kleinmaßstab nachbilden, um sie besser erforschen zu können. Dies könne der Entwicklung neuer Medikament dienen, so die Hoffnung. Bis Ende des Jahren liegen weltweit etwa 20 iPS-Zelllinien für verschiedene Krankheiten vor.

Im Verlauf des Jahres gibt es Berichte über vereinfachte Rezepte zur iPS-Herstellung. Manche verzichten auf die Krebs-Gene, andere auf die Viren.

2009 Die Bemühungen, die Rückverwandlung erwachsener Zellen in einen embryonalen Zustand einfacher und sicherer zu machen, werden weitergehen. Eine der wichtigsten Fragen ist zudem, ob die iPS-Zellen die embryonalen wirklich ersetzen können. Das Biotech-Unternehmen Geron hofft auf eine Genehmigung für Versuche mit umgewandelten embryonalen Stammzellen. Es geht darum, Verletzungen im Rückenmark zu heilen. Die über 20 000 Seiten mit Vorstudien und Anträgen liegen seit fast einem Jahr bei der zuständigen Behörde.hach

[-Salem-]

Antwort auf Beitrag Nr.: 34.591.020 von -Salem- am 26.07.08 03:29:06

Seht, ick hatte recht... Wir Berliner sind eh die schlausten. Nicht so blöd wie der Rest der deutschen Nation.(auch eine im Suff getroffene Aussage stimmt)

Siehe heutige Veröffentlichung. GRN163L (L wie Legastheniker) dürfte Geron´s Top Story werden. Die Stammzellen sind ne Sache für die nächsten 20-45.500 Jahre. GRN163L kommt früher und ischt heißer.

GRN163L kommt übrigens von einem deutschen Unternehmen (WKN 588040).

http://biz.yahoo.com/bw/081208/20081208005642.html?.v=1

[Sugar2000]

Antwort auf Beitrag Nr.: 35.951.156 von meislo am 14.11.08 20:53:57

auf der anderen seite würde GERN wieder in den Fokus der Anleger geraten...