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Comment Re:AIDS vs. Cancer (Score 2) 130

HIV isn't the only retrovirus or the only virus that targets T-cells. I will admit that the crisis that AIDS has posed has greatly accelerated research into retroviruses, but the development of retroviruses as gene therapy vectors is only tangentially connected to research on AIDS as a disease.

AIDS as a pandemic has from the start been as much or more about public health measures as it has virology, and if we had been far more fortunate, HIV would have remained a fearsome but rare curiosity. Research on it might have remained a backwater of the academic world, and yes, the pace of treatments like this might have been slowed, though I strongly doubt by 50 years. When you ask if success against cancer means AIDS could have been "worth it," I believe that we would have had ample opportunity to obtain this knowledge without the cost of thirty million lives.

Comment Re:Not quite (Score 3, Informative) 50

The researchers involved used a disease called experimental autoimmune encephalomyelitis (EAE). This is a disease with many general similarities with multiple sclerosis (being autoimmune responses against myelin), but there are differences in the course of the disease versus MS. EAE is considered to be closer to a rarer human disease, acute disseminated encephalomyelitis (ADEM) than to MS. Nevertheless, EAE has been used for decades as a model for autoimmune diseases, as it has the major advantage of being able to be reliably induced in animals. The method of immune system modulation used in this study seems general enough to apply to similar autoimmune disorders, but that has not been actually established with studies yet.

Comment Re:Nanoparticles? (Score 2) 50

True, the particles used are an inert support for the proteins, not a treatment in themselves. The idea was to present the proteins as if they were cell-surface proteins which the immune system would recognize and build tolerance for. The original paper's authors performed earlier work with the same proteins attached to white blood cells. This was successful, but in terms of developing a clincal treatment, manufacturing engineered cells presents far more complexity, risk, stability issues, and cost than the manufacture of polymer microparticles coated with the protein. Microparticles, rather than nanoparticles, is the term consistently used throughout the paper, coincidentally. The particles are 500 nanometers in diameter, which is larger than what are usually considered nanoparticles 100 nm).

Comment Re:Generics and Legal Challenges (Score 3, Informative) 100

That's not necessarily true of pharmaceuticals, and in fact, quite a few other blockbuster drugs have come about as re-purposed molecules: Merck originally developed Proscar (finasteride) as a prostate enlargement treatment. Then it submitted finasteride to the FDA again as Propecia for male pattern baldness. Wikipedia tells me, "Merck's patent on finasteride for the treatment of BPH expired on June 19, 2006. Merck was awarded a separate patent for the use of finasteride to treat MPB. This patent is set to expire in November 2013." Bupropion was developed as an antidepressant (Wellbutrin), but there's a separate patent covering Zyban, which is the same molecule when prescribed for smoking cessation. Latisse is the glaucoma drug Lumigan, repurposed when they found it makes your eyelashes grow.
For that matter, Viagra itself is also sold in a different dose as Revatio for pulmonary hypertension- I'm pretty sure that Pfizer's "treatment of erectile dysfunction" patent expiring in 2019 does not apply to that product. I will note that these "use patents" are much more likely to be invalidated in court challenges compared to the "composition of matter" patent (it was considered surprising by many observers when Pfizer won their case with Teva in the US last year), but they are out there.

Comment Re:Insufficient information (Score 3, Informative) 100

Yes, Teva's central argument in the challenge was that in the patent in question, Pfizer never specifically establishes the relationship between sildenafil and the treatment of ED. From the judgement:

As required by s. 2 of the Act, an invention must be novel. In the instant case, the invention is not sildenafil, per se, because this compound was already known. In fact, Pfizer had been investigating sildenafil as a cardiovascular drug when it first suspected that the compound would be useful in treating ED (R.F., at para. 13). The invention is therefore not sildenafil, but the use of sildenafil to treat ED.(italics theirs)

The court ruled that the patent never concretely established this relationship:

Although Patent ’446 includes the statement that “one of the especially preferred compounds induces penile erection in impotent males” (A.R., vol. X, at p. 173), the specification does not indicate that sildenafil is the effective compound, that Claim 7 contains the compound that works, or that the remaining compounds in the patent had been found not to be effective in treating ED. The claims were structured as “cascading claims”, with Claim 1 involving over 260 quintillion compounds, Claims 2 to 5 concerning progressively smaller groups of compounds, and Claims 6 and 7 each relating to an individual compound.

The disclosure in the specification would not have enabled the public “to make the same successful use of the invention as the inventor could at the time of his application”, because even if a skilled reader could have narrowed the effective compound down to the ones in Claim 6 and Claim 7, further testing would have been required to determine which of those two compounds was actually effective in treating ED. As the trial judge stated, at para. 146, “[a] skilled reader would then conduct tests on those two compounds and determine which of those compounds worked.” And as he also stated, at para. 135, “the skilled reader must undertake a minor research project to determine which claim is the true invention”.

Pfizer had the information needed to disclose the useful compound and chose not to release it. Even though Pfizer knew that the effective compound was sildenafil at the time it filed the application, it limited its description to the following statement: In man, certain especially preferred compounds have been tested orally in both single dose and multiple dose volunteer studies. Moreover, patient studies conducted thus far have confirmed that one of the especially preferred compounds induces penile erection in impotent males. [Emphasis added; A.R., vol. X, at p. 173.] It chose a method of drafting that failed to clearly set out what the invention was. Even now, in its factum to this Court, Pfizer offers no explanation as to why — knowing that Claim 7 contained the tested and thus, the useful, compound — it elected to withhold that information.

Comment Re:Generics and Legal Challenges (Score 4, Informative) 100

Just to follow on, the Canadian Supreme Court decision is here and the invalidated patent is here. What caused the patent to be invalidated was that the patent is basically written to cover a wide array of similar molecules, all derivatives of a central molecular skeleton. Often, minor alterations to a molecule can be made that do not change the behavior of the molecule as a drug. What it appears Pfizer was looking to do here was prevent competitors from developing ED drugs that were simple derivatives of sildenafil (Viagra) (like adding a methyl group or a fluorine atom somewhere it would have no significant effect). That's considered acceptable strategy, and as a result, other ED drugs like vardenafil (Levitra) and tadalafil (Cialis) have differences in their core structures that keep them from infringing.

However, the Canadian court found that Pfizer had failed to essentially zero in on sildenafil with their claims. When it came down to actually stating that this molecule is the one that lab studies have found treats ED, Pfizer only ever mentions the core skeleton (known as "formula I") and never uniquely identifies sildenafil. It mentions sildenafil (not even by name, only by its R groups) in one claim, but never connects it and only it to ED. The court judgement notes that "formula I" represents 260 quintillion possible compounds, and therefore rejected the patent for vagueness.

Comment Generics and Legal Challenges (Score 5, Informative) 100

This particular patent fight is also occurring in other places. In the US, there was a decision last year between Pfizer and Teva that was ruled the opposite way, and if no other challenge is successful, I believe Viagra will continue to exist under patent for Pfizer until 2019. At issue is that Viagra really has two patents- one for sildenafil and its formulation into a drug and one for the use of sildenafil to treat erectile dysfunction. Sildenafil was originally developed for blood pressure and cardiovascular disease, so the application for the ED indication patent trailed the formulation patent by several years. The formulation patent expired this year, but the indication patent lasts until 2019. In the US, a regulatory framework known as a Paragraph IV challenge exists for generic drug manufacturers to either argue that their proposed generic does not violate the existing patents, or that the patents themselves are invalid. This potentially allows them to open up the generic market years before the patent was originally set to expire, so this sort of legal action is not uncommon, particularly for blockbuster drugs.

Comment Re:Once again (Score 3, Informative) 43

These happen to only be "adult stem cells" in the sense that they are not totipotent embryonic stem cells (cells from the very earliest stages after fertilization that can differentiate into any cell type), but I'd like to point out that doesn't mean they come from an adult brain. They're only "adult" by the meaning of having matured to the multipotent stage (neuronal stem cells can differentiate into neurons or glia, but not muscle cells or liver cells, for instance). The biotech company that provided these cells, StemCells, Inc. cultures them from donated fetal brain tissue.

The first production step comprises a proprietary method for purification of HuCNS-SC cells from donated fetal brain tissue procured from an FDA-registered, not-for-profit agency, in compliance with Good Tissue Practice (GTP) and all other applicable state and federal regulations. As part of the purification process, cells from the tissue are “tagged” with a monoclonal antibody that recognizes human neural stem cells. High-speed Fluorescence Activated Cell Sorting (FACS) is then used to isolate the cells tagged by the monoclonal antibody. The FACS-isolated HuCNS-SC cells are then placed in cell culture.

Comment Re:Flowers For Algernon (Score 3, Informative) 43

The stem cells aren't turning into neurons, actually, despite coming from "neuronal stem cells." The intent of the treatment is for the stem cells to differentiate into oligodendrocytes, which are a type of glial cell (which in turn are several types of cells that provide support functions to neurons). Oligodendrocytes are interesting cells because they wrap around neurons like insulation around a wire (which is exactly their purpose). These cells play an important role in nerve conduction and in overall brain function, but they're just tubes filled with fat.

Comment First Edition! (Score 4, Funny) 371

Anyone here teaching a course might be interested in the comprehensive new textbook I'm writing. It has an attractive hard cover, a quality binding, and a single page inside which lists the URLs for Google and Wikipedia. My planned retail price is $499, but I'm willing to offer a volume discount.

Comment Re:We already know soda drinkers are fat (Score 4, Informative) 388

Well, they shouldn't, if they're drinking out of 2L bottles, most of which are polyethylene terephthalate and generally do not contain BPA, which is why the focus here is on the epoxy liners of many aluminum cans. They did try to control for caloric intake in the study:

Controlling for race/ethnicity, age, caregiver education, poverty to income ratio, sex, serum cotinine level, caloric intake, television watching, and urinary creatinine level, children in the lowest urinary BPA quartile had a lower estimated prevalence of obesity (10.3% [95% CI, 7.5%-13.1%]) than those in quartiles 2 (20.1% [95% CI, 14.5%-25.6%]), 3 (19.0% [95% CI, 13.7%-24.2%]), and 4 (22.3% [95% CI, 16.6%-27.9%]).

However, they also admit in the conclusions, "Explanations of the association cannot rule out the possibility that obese children ingest food with higher BPA content or have greater adipose stores of BPA."

Comment Re:Pancreas (Score 1) 544

Often, there aren't any early symptoms of pancreatic cancer, or whatever symptoms appear are nonspecific, which is a major reason why it is such a deadly cancer. It tends to be an aggressive cancer otherwise, so early detection is not always a lifesaver, but it certainly is a problem that pancreatic cancer is rarely diagnosed until it has become advanced. Frequently, the first signs of trouble are liver symptoms, i.e., after the cancer has already metastasized there.

Comment Re:Anyone here have PKD? (Score 1) 544

I don't have it, but PKD definitely runs in my family, so I feel for you knowing what some of my family has gone through. One of my kidneys is already in my sister, so I'm afraid I've literally already given until it hurt. I can say that a successful transplant really can work miracles for the disease, so best of luck to you. Of course, if we're able to just print organs out, it'd be nice to print myself a replacement kidney. I mean, I have an open expansion slot and everything....

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