In many cancer drugs trials, the "proving" of efficacy is highly statistical in nature, where the size of the trial has to be big enough to show "statistical significance" as the efficacy improvements may be fairly minute as compared to previous standard cancer drugs. A pure Phase 1 usually is fairly small in size, as no attempt is made to make it big enough (with enough patients) to have a chance to show statistical significance with enough certainty, although still big enough to show a fair measure of safety, and dosing limits. A following Phase 2 will enroll enough patients to try to achieve at least a low level of statistical certainty if efficacy is shown. Phase 3 trials will enroll large numbers of patients in multi-centers to attampt to achieve high levels of statistical certainties of the observed efficacy, if successful.
Phase 1/2a probably means a Phase 1 trial "combined" with an initial Phase 2 trial. Phase 1 trial is a purely safety trial where dosages are gradually increased by steps until the top dosage limits are reached, although signs of efficacy may "accidentally" be observed during a Phase 1 trial, there is no attempt to "force" signs of efficacy. A Phase 2 trial will spend its efforts to generate signs of efficacy, and to determine the initial percentage of efficacy. In practice, what is the difference between a Phase 1 trial followed by a Phase 2 trial versus a single combination of Phase 1 and initial Phase 2 trial? If there is no sign of efficacy in a traditional Phase 1 trial that proves to be safe, the drug company will still forge ahead to a Phase 2 trial to look for signs of efficacy. In practice, a combined Phase 1/2a trials that prove safety, but shows no sign of efficacy are often abandoned without continuing into Phase 2 trials.
Theoretically yes, but it may be tricky. The problem of many cancer drugs is that they cause blood thinning (decrease of blood platelets) which limits the dosage and duration of these cancer drugs. Imetelstat also concentrates in bone morrow. One trick is to be able to target the breast cancer cells without effecting the bone morrow normal platelet producing stem cells. This may be done by direct injection, modifying the imetelstat molecule, and the like to more selectively target the breast cancer cells with telomerase inhibition, while more selectively not targeting the normal platelet stem cells in the bone morrow. For example, before imetelstat (GRN163L), Geron has the GRN163 telomerase inhibitor (without the L, which is a lipid molecule attached to make it easier to cross the cell membrane) which may have less blood thinning effects (which is not known as it was not put into human trials after the GRN163L (imetelstat) version was chosen over the GRN163 version.) Direction injection (profusion) is a technique of injecting directly into the effected site or organ, so that higher drug concentrations can be used while minimizing the heightening of side effects in other parts of the body, as after directly injecting into the target site at full concentration, the drug is highly diluted as the drug diffuse to other parts of the body. Direct targeting, where caner drug molecules are attached to guide- molecules which attach only to chosen cancer cells, and not attaching to any normal cells, are still in the research stage in the cancer field.
Why do you say it is not a cancer? Perhaps a search on medical information websites will clarify whether MF is a form of cancer or not.
Before MF, in the ET slide presentation by the investigating doctor of the ET trial, the slide says that the ET stem cells have short telomeres, and use (express) telomerase. Short telomeres and rapid cell division make telomerase inhibition attractive in ET, as cancer stem cells with short telomeres will run down their telomeres after rapid division, if the telomerase is inhibited. It is believed that MF is in the similar class of disease as ET, so that telomerase inhibition may be similarly effective as in ET. Maybe MF stem cells also have short telomeres, and divide rapidly? Perhaps the full MF presentation slides will mention this.
If any big pharma decides to partner with Geron, the big financial institutions will change their assessments in a jiffy, IMO. In the past, big pharma's were afraid of involvements with any embryonic stem cell companies like the old Geron, because big pharm's were afraid of attacks, physical and legal, from the religious rightwing republicans like Merck got attacked with law suites on vioxx soon after it partnered with Geron a number of years ago on the telomerase vaccine (not inhibitor.) Now that Geron has gotten out of the embryonic stem cell business to make it easier for big pharma to get close to Geron without having to look over their shoulder for potential religious right republican attacks, the chances of getting big pharma support has increase, IMO. In the mean time, it is two steps up, and one stem down. Slowly, it is higher highs, and higher lows so far.
You think INCY and other should try to partner with Geron?
One patient Joann has a blog reached by using yahoo search at the top of this screen and clicking "Search Web" after entering search argument "what's the bloody news? joann john"
Yea, 2003 to 2008 are the lost years of Republican Religious Rightwing obstruction years for ESC. Bush's family doctor friend headed up the FDA during the later part of this period, when the FDA placed holds to Geron's first ESC human trial. The week after the Democrats took over the Presidency was when Bush's family friend doctor resigned as head of the FDA, which immediately lifted the hold on Geron's first ESC trial, which had no safety problems, that opened the way for the FDA to approve ACTC's trial to begin about a year later.
When Gary took over there were about 300 to 600 million shares outstanding. Now 2 billion and some? At least three times dilution on the long suffering shareholders. This guy is none technical, so that he appears to contribute little on the technical side, which one of the biggest issue is to find out how to increase the success rate of treatments, so that it is not only three patients out of about two dozen getting remarkable success, but many more is needed. On the stock price side, he dilutes without end.
Serious investors like Buffett don't touch high risk techie stocks like ACTC it appears. It is unlikely most average investors deal with risky early stage development high tech micro stocks either.
It appears to me there are two major types of people involved with this type of stocks: people who like to deal with early stage hi-tech biotech stocks, and shorts who like to take advantage of early stage micro cap stocks. Because this stock involves an unusual area of ESC's, there is a third type involved with this stock: religious-right- wing-Republicans. As ESC entails a lot of religious and conservative politics, it appears that this message board is saddled with hordes of religious and political bashers, who may in addition be shorts.
There are a few investors of the forth type, who are either patients, or who are potential patients. There are very few of this type of investors. It can be safely say that few super rich people invest in this stock to prepare for the day when they get old, and when they may have a need for this kind of regenerative medicine. Recently, someone posted that a billionaire donated 25 million dollars to a research university, which is developing similar eye regenerative treatment. Maybe a multimillion dollar donation to a university is tax deductible, while a loss on a biotech is less deductible (limited by 3K per year, but more can be used to offset stock gains.) It appeared that a certain billionaire did not invested in a small cancer drug development company, which is a few that was working on his type of cancer. He appeared to have the means to easily taken over the company, and using his vest business building skills to create a cure. But, that is not the way the super rich people thinks. Maybe biotech is too hi-tech for them.
Thus, we are left with the two types of investors: biotech investors, and the shorts who prey on early stage biotech stocks, plus in this case, the rightwing-religions-republican-libertarian bashers
Big oil companies, and other big companies have the money to hire dream teams of lawyers to fight other big companies in contract disputes.
Little people cannot afford to hire lawyers to fight against big companies in contract disputes. It is a one sided competition between the big companies of the super rich people versus the common individual people without having the government to protect the common people from being bullied and pillaged by the super rich, and their big companies with their huge and powerful dream teams of lawyers, and lobbyists.
The market playing field is completely uneven and tilted to benefit the super rich and their big companies with their armies of lawyers and lobbyists, unless a liberal government steps in to even the playing field. You should not fail to see how only a liberal government is strong enough to level the market playing field against the heavy weight of the super rich and their big companies.
True, not only shorts "hope" that patients do badly, but shorts, in addition, have actually "caused" patients to suffer from drug development delays caused by shorting, which lowered the price of biotech stocks so that biotech companies get less funding from the issuing of their stocks, and by shorts bashing in financial journals, as well as stock message boards to scare away investors. There used to be this Rob Cambell fellow who played shorting games by penny downward steps using his commission-free company account, and bashed on this board literally 24/7 with none stop bashing on this board. Anybody know what happened to him?
In retrospect which was obvious to many at the time, agree that Geron should had gone offshore, as it did not have the funds to outlast the FDA drawn out trials of baby step small dosages (under the political pressure from the right wing.) Going offshore is better than having to abandon the Phase 1 trial, due to the lack of funds to complete the drawn out trial.
Another far out issue is whether high incubation temperature is needed for these post-embryonic/fetal stage cells to grow rapidly and robustly. In higher animals, eggs and fetuses are incubated at high temperatures for rapid growth. Hens and birds spend a lot of energy and effort to sit on their eggs to keep the growing embryo/fetus hot. Mammals "incubate" their fetuses in the warm womb. Lower animals like fishes and insects just lay their eggs into the environment without incubating at high temperatures, which higher animals require like chicken egg incubators selling on Ebay are set at 99.5 degrees F. In ACTC's eye trial, the first two patients were injected during the summer of July 12, 2010, and the first two patients injected during the summer had measurable improvements, that were published in a scientific journal publication. The next four patients were inject during the winter and spring, and nothing were published about them.
Evolution wise, it is beneficial to not having to spend the time and energy to incubate, as insects proliferate by the zillions not having to incubate their eggs at high temperatures. Higher animals have some as yet unclear (to the human intellect) need for the high temperature incubation, which is time and energy consuming, slowing down the reproductive rates.