Wednesday, April 09, 2008

Myelofibrosis, CIRM, and patents

An April 7 article by Terri Somers begins:

A one-year scientific collaboration that included the University of California San Diego and the tiny local biotechnology company TargeGen has moved a treatment for a potentially fatal blood disease into human trials at record speed.

The experimental drug is also the first in clinical trials to be funded in part by California's taxpayer-supported stem cell institute [CIRM]. The work on the drug's development is documented in articles published Monday in the scientific journal Cancer Cell.
[The first trial is designed to specifically treat 40 people with a myeloproliferative disease called myelofibrosis, with a market of about 100,000 people in the United States, 15,000 diagnosed per year.]

Somers included details of the work, and several quotes by CIRM president Trounson:

While it can take five to ten years to discover a potential drug and ready it for clinical trials, TargeGen and the scientists collaborated for about a year before the compound started human trials in February.

“The combination of research groups working together with a biotech company is just a superb story and I think it's one of the leading examples supporting the establishment of (the state stem cell institute,)” said Alan Trounson, a stem cell scientist and president of the institute.

“It demonstrates what can be done in this space and the impact of funding stem cell research. It happened in a time frame that I wouldn't have really believed,” Trounson said.

The team was led by the lab of Catriona Jamieson, a physician scientist at UCSD Moores Cancer Center, who used a $642,500 Seed Grant from the stem cell institute to fund part of her work. Also on the team was John Hood, director of research at TargeGen, as well as scientists from Stanford, Harvard and the Mayo Clinic.

The published work related to studies on mice:

In the research documented in the Cancer Cell article, her team put human stem cells engineered to have the mutant gene into mice models to see if the over-expression of the single gene would cause the disease. It did.

To corroborate, she then injected stem cells of people with the disease into the mice, and achieved similar results.

One will not find ANY discussion of patent rights in the Somers article. One does find:

In the past, researchers at different universities weren't quick to share ideas. That is changing because patients who are better educated and networked through the Internet are demanding better care, she [Jamieson] said.

“They remind us that we need to work together like a soccer team, play our positions and make sure we go after things in a way that will be most effective for everyone. In that way, we all win,” she said.

Jamieson's work was also partially funded by the Mizrahi Family Foundation, a philanthropic organization representing one of her patients, Albert Mizrahi, who also allowed his stem cells to be used in the research.

Whether "patient input" is driving collaboration among universities might be questioned.

Of the text, researchers at different universities weren't quick to share ideas one merely has to recall the underlying issue in the Supreme Court case
Merck v. Integra (Scripps v. Burnham???) to see sharing is still an issue.

What about patent rights, Terri?

See also -->

A post at HHMI noted:

Phase I clinical trials of the drug began in March 2008 at the Dana Farber Cancer Institute, the Mayo Clinic, Stanford University Medical Center, MD Anderson Cancer Center, the University of California, San Diego (UCSD) School of Medicine, and the University of Michigan Medical Center. In this drug trial, small doses of the drug will be given to a few patients to see if any toxicities appear.

“The amazing thing about this is that it normally takes eight to twelve years to move from the discovery of a gene to a clinical trial with a drug,” says Gilliland. “This has been remarkably fast by conventional drug development standards. It speaks to the motivation of scientists in academia and industry to move drug development for cancer along at a more rapid pace.”

The three leukemias, polycythemia vera, essential thrombocytosis, and primary myelofibrosis, affect about 80,000-100,000 people in the United States. The diseases are characterized by an overproduction of white blood blood cells, red blood cells and platelets. This surplus of blood cells can contribute to serious complications, such as blood clots and bleeding.

Of TargeGen:

One of the most promising compounds, called TG101348, came from TargeGen, a privately held biopharmaceutical company in San Diego. “This compound has all the properties that we thought would be important in clinical trials,” says Gilliland. The compound specifically inhibits JAK2 while leaving related enzymes untouched. In cell cultures, the compound does not show non-specific toxicity. It also is soluble in tissue, so it can distribute itself throughout the body, and can be taken as a pill rather than as an injection into blood vessels. And finally, the drug has a relatively long half-life, meaning that it will stay in the body long enough to do its work.


On April 17, someone who had done a search for -- Jennell B. Guillory -- attempted to post a comment on this post. Nothing was left though. In passing, Terri Somers never responded to the above query. Obviously, there will be a second part to this post, wherein IPBiz will tell people something Terri Somers declined to tell her readers.


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