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The Blog of Drug Discovery News

Did President Reagan suffer from Alzheimer’s disease while in office?

ddn has written countless stories about drug discovery and research efforts in the critical area of Alzheimer’s disease, and one area that researchers, clinics and drug manufacturers seem to be focusing on lately is pinpointing the onset of the debilitating disease. For example, in October, we reported on efforts by the Alzheimer’s Prevention Initiative (API) to test potential Alzheimer’s treatments and identify new biomarkers that could lead to earlier and more accurate diagnoses for Alzheimer’s patients. Researchers at the API told us that although there are many promising treatments being studied in Alzheimer’s symptomatic patients, by the time most people begin to show symptoms of the disease, it has already ravaged the brain, rendering these treatments ineffective.

This cold, hard reality has been making headlines lately with the release of a new book, “My Father at 100: A Memoir,” a close-up account of the life of President Ronald Reagan as seen through the eyes of his son, Ron Reagan. The book, which came out a few weeks shy of what would have been the former president’s 100th birthday on Feb. 6, is “an exploration of his character,” Ron Reagan says, but addresses the ongoing question of whether his father suffered with Alzheimer’s while in office.

President Reagan was diagnosed with Alzheimer’s in August 1994 at the age of 83, and he informed the nation about his diagnosis in a handwritten letter later that year. Although President Reagan’s White House doctors said they saw no evidence of Alzheimer’s while he was president, there was during his time in office widespread speculation that he demonstrated symptoms of mental degeneration. For example, former CBS White House correspondent Lesley Stahl wrote in her own memoir that at her final meeting with President Reagan in 1986, “Reagan didn’t seem to know who I was.” The president regained his alertness at the end of the meeting, Stahl wrote, adding, “I had come that close to reporting that Reagan was senile.”

Ron Reagan writes that that he noticed evidence of dementia as early as President Reagan’s first term. “I felt the first shivers of concern” during the 1984 reelection campaign, he writes, “that something beyond mellowing was affecting my father. My heart sank as he floundered his way through his responses. He looked tired and bewildered.” By 1986, President Reagan “had been alarmed to discover, while flying over the familiar canyons north of Los Angeles, that he could no longer summon their names,” his son writes.

Still, as he hits the press junket, Ron Reagan is careful to say that we cannot know for certain whether President Reagan exhibited signs of Alzheimer’s during his presidency. He also asserts that he believes if Reagan had gotten the diagnosis during his two terms, he would have stepped down.

In this video with TV personality Joy Behar, Ron Reagan clarifies his characterization of his father’s illness in his book.

“One can deduce that the disease must have been present, but I say specifically that I saw no dementia-like signs when he was in office,” he tells Behar. “Let’s recall that this was the oldest president ever elected (President Reagan was in his 70s). By the time he’s reaching his mid-70s, he’s losing his hearing, he’s been shot and nearly killed—which will take a little of the wind out of your sails—and of course I am worried about him all the time, because it’s a very tough job with a lot of stress. Every once in a while I would see—almost like when you are watching television, and it momentarily goes out of focus and snaps back. You think, ‘what did I just see?’ But I didn’t know what it was, I just knew I was concerned about him for all sorts of reasons. In retrospect, it’s possible that some of those early things were signs of Alzheimer’s, but I don’t know, and I can’t really make that claim.”

Some of the controversy, Ron Reagan tells Behar, may stem from “the confusion between Alzheimer’s the disease and dementia, which is a symptom of the disease—which usually arrives in the later stages.”

“Knowing what we know now about Alzheimer’s, that it’s a process that extends for years or even decades before symptoms arise, it’s kind of an academic question as to whether the disease was present when my father had” the debilitating disease, Ron Reagan says in this interview.

I think many of the researchers who read our publication would agree. What do you all think of Ron Reagan’s assertions? How does this “academic question” impact efforts to treat, manage or even reverse damage caused by Alzheimer’s?

January 27, 2011 Posted by | Academia & Non-Profit, Corporate, Labwork & Science | , , , , , , , , , | Leave a comment

Looking for similars parity

So, I’m seeing talk of biosimilars showing up a bit more on the news feeds and in my e-mail inbox. Hard to say yet whether it’s a sign of a rising trend in pharma and biotech or whether it’s just a little jump in the frequency.

But I will say that in our January issue (heading out in the mail and also available online – key stories here and full issue in PDF form here), we had a sizable commentary about biosimilars. Given that the FDA is supposedly going to finally catch the U.S. up somewhat with its European peers in terms of an approval pathway and regulatory guidelines, I won’t be at all surprised to see a lot more stories on biosimilars like the one below that hit the newswires yesterday:

Merck & Co. and PAREXEL form strategic alliance focused on clinical development of biosimilar candidates

WHITEHOUSE STATION, N.J. & BOSTON—Merck & Co. Inc. and PAREXEL International Corp., a leading global biopharmaceutical services provider, today announced that they have entered into an alliance by which PAREXEL will provide strategic access to global clinical development services for designated biosimilar candidates to Merck BioVentures. Merck BioVentures, a division of Merck, is focused on the delivery of high quality biosimilars to the patients that need them.

“PAREXEL has extensive, industry-leading experience with biosimilar development, and we truly understand the scientific complexities, and regulatory pathways involved,” said Josef von Rickenbach, chairman and chief executive officer of PAREXEL. “We are committed to working with Merck BioVentures to assist in advancing its biosimilar portfolio in this rapidly developing market segment for the benefit of patients worldwide.”

Under the terms of the agreement, PAREXEL will provide Merck BioVentures with strategic access to a broad range of regulatory strategy and clinical development planning capabilities for the development of certain broad classes of biosimilars across various therapeutic areas, including exclusivity for certain candidates. The agreement also provides for the establishment of a dedicated Merck BioVentures unit within the PAREXEL organization. Further details of the agreement were not disclosed.

“Through this agreement, Merck BioVentures has secured broad strategic access to PAREXEL’s proven biosimilar clinical development experience,” said Michael Kamarck, president of Merck BioVentures. “This agreement positions Merck BioVentures for success with an industry leading partner that has the expertise and resources to conduct clinical development of our diverse portfolio of candidates to allow timely delivery of products to the marketplace.”

I think probably the biggest hangup for biosimilars (and thus news about any work on them) really has been the sluggish pace at which FDA seems to have been addressing this topic. I know that biosimilars pose many more challenges than do generic versions of some small-molecule drug or the like, but I can’t help but think that with European regulators already having trod this ground that the FDA has really been dropping the ball (as opposed to simply being wisely cautious).

Of course, that assumes that they agency even makes good on getting workable guidelines out this year. Lot of people and organizations fail at New Year’s resolutions.

January 13, 2011 Posted by | Corporate, Government, Labwork & Science | , , , | 1 Comment

Hoarders: Pharma edition

I can only hope my wife won’t kill me for letting this information out to all of you, but she has some guilty pleasures in terms of television. She doesn’t watch a whole lot of it, but she does have a thing for “General Hospital” when she doesn’t have to be at the office between 3 and 4, and she watches a few reality shows regularly or semi-regularly, such as “Real Housewives of Atlanta.”

But what’s appropriate for my purposes in this post is to mention her love of the show “Hoarders.” OK, love might be too strong a word. It’s more of a love/hate thing, and she watches it almost out of a compulsive fascination, much like the human inability to avoid looking over at a car accident, train wreck, lover’s spat or your spouse’s e-mail inbox…just to see if it’s as bad as you think.

For those of you not familiar, each episode follows the efforts of a clean-up crew and mental health professionals to deal with two different homes in which compulsive hoarding of items, garbage, animals or whatever else has reached crisis proportions. Some episodes require a strong stomach, as you can almost smell the putrid fumes through the television screen.

I wonder how many pharma and biotech companies, particularly the big ones, are about to need some facemasks to deal with some bad smells that could soon be coming out of their servers, computers, databases or what-have-you.

No, I’m not talking about the hardware overheating; I’m talking about data.

As we prepare in the January issue of ddn to delve into a special report series on screening technologies, I find myself wondering if the drug discovery and development world has become a collective mass of hoarders when it comes to data.

I don’t mean that they keep things to themselves to protect their intellectual property. That’s totally understandable. What I mean is that I fear they may have too much data. Don’t misunderstand me, though. I’m not a Luddite. I adore the wonders of next-gen sequencing and computers that can hold terabytes upon terabytes of data. I love that we can sequence genomes and dig deep.

But the truth is that we don’t know what to do with this data in many cases. Companies have it, but they don’t yet know what to look for to make it work for them most effectively. All these massive amounts of data can yield up wondrous gems in the near-term in some cases, but much of it is simply hoarded ones and zeroes on storage devices, waiting for us to catch up with it. Waiting for us to gain enough understanding of genomics, proteomics, metabolomics, biomechanics and so much more so that we know what all the data we have at our fingertips now actually means.

I mostly have faith that all that data will not be a wasted effort and that we will indeed put it to good use. On the other hand, how much of it is totally unnecessary and useless? Worse yet, since we’re already acquiring data far, far, far faster than we can figure out what to do with it, what if that trend continues? What if we gather data at a geometric rate, while our ability to manage it continues at something more like a linear rate?

What are we going to do with all the data? And how much good stuff will we lose in the mess we create?

And will we need a new reality show called “Biotech Hoarders” to run on TLC or the Discovery Channel?

January 4, 2011 Posted by | Corporate, Labwork & Science | , , , , | Leave a comment

Seeking the ‘eye of the tiger’

WASHINGTON—Science funding is getting an infusion of cash after Congress in December passed a $45 billion reauthorization of the America Competes Act—a bill designed to invest in research and science education, and to fund and foster innovations that will help keep the United States competitive in tech-driven industries.

It signals another key milestone for scientific research, and history teaches us that building and maintaining a competitive advantage is vital to our country.

The importance of maintaining a competitive advantage on the global stage can be defined by one striking moment that occurred Oct. 24, 1957: Sputnik I was launched, an event that marked a 20-year race for supremacy in space between the Soviet Union and the United States. For two decades, the two nations traded volleys in the space race, with the United States gaining the upper hand by the late 1960s. By the 1970s, space exploration stood as a symbol of the competitive nature between the world’s two superpowers for technological, military, cultural and intellectual supremacy.

In a less grandiose analogy, the United States must regain the “eye of the tiger.”

In Rocky III, the fictional boxer Rocky Balboa must come back from a rattling defeat to Clubber Lange and the death of his longtime manager, Mickey. He trains with his old nemesis, Apollo Creed, learning along the way that he needs to stay hungry and keep “the eye of the tiger.”

Sadly, the story has changed in recent decades, with the United States slowly losing its competitive edge. It seems as if we no longer place a premium on education, research and innovation. Evidence of this is seen in the World Economic Forum’s recent ranking of the quality of math and science education around the world—the United States stands 48th. We are lagging behind other countries in the issuance of patents and ­even—in an era of high unemployment—American companies consistently suffer from a shortage of individuals with critical skills.

The America Competes Act can serve as an investment in regaining our competitive edge and becoming the world leader in education, research and innovation.

The bill, which has been in legislative limbo throughout the year, sets funding levels for the next three fiscal years for the National Science Foundation, the Department of Energy’s research programs, and the National Institute of Standards and Technology. It also funds education initiatives and a range of other programs.

The legislation provides a total of $23.5 billion for NSF, $16.9 billion for the DOE’s Office of Science, $2.9 billion for NIST, $600 million for education efforts and $1.4 billion for other programs.

The original America Competes Act was approved in 2007. The legislation is based on recommendations outlined in the “Rising Above the Gathering Storm” report released by the National Academy of Sciences in 2005. It urged the United States to work harder to support technological innovation and enhance science, technology and mathematics education.

“In acting to update and extend the America Competes Act, we will spur innovation, invest in cutting-edge research, modernize manufacturing, and increase opportunity,” says House Speaker Nancy Pelosi. “Simply put, we will continue to ‘rise above the gathering storm’—and keep America No. 1.”

Earlier this year, the bill stood in political limbo, with an earlier draft that was nearly double the cost of the version passed through the House and Senate. A compromise was reached during the lame duck session to cut down the term of the bill from five years to three, creating most of the savings from the original $86 billion draft.

“If we are to reverse the trend of the last 20 years, during which our country’s technological edge in the world has diminished, we must make the investments necessary today,” House Science and Technology Committee Chairman Representative Bart Gordon, D-Tenn., said in a statement.

“More than half of our economic growth since World War II can be attributed to development and adoption of new technologies. These investments are the path toward sustainable economic recovery and growth and the path toward prosperity for the next 50 years,” Gordon said.

Among the new programs in the act, 98 percent of which will fund new scientific research, is the creation of an inter-agency public access committee in the Office of Science and Technology Policy. The committee will coordinate federal science agency policies related to the stewardship and dissemination of research results, including digital data and peer-reviewed scholarly publications.

Another aspect of the bill welcomed by the research community is the authorization of the funding boost for NSF, NIST, and DOE, which “puts us on a path towards a continued increasing investment in those programs over the next 10 years,” Jennifer Zeitzer, director of legislative relations at the Federation of American Societies of Experimental Biolog.

“It’s a bipartisan bill, which we’re happy about because it sends a signal that investing in science and paying attention to science issues is something that both parties care about,” she said.

Rep. Dan Lipinski, D-Ill., a former professor and one of the few members of Congress trained as an engineer, says he was proud to have helped write and pass the measure, which he says “makes essential, job-creating investments in advanced research and science, technology, engineering and mathematics education.”

“I am grateful for the valuable feedback I received from the Association of American Universities and the Association of Public Land-grant Universities while writing the NSF title of this bill,” he adds in a statement. “Because of their expertise, and because of what I learned from scientists and research administrators across the country, I believe this legislation will have an enduring positive impact on university-based research and STEM education programs.”

The Business Roundtable lauded Congress for its swift action in passing the America Competes Reauthorization Act.

“The bill will attract more young Americans into technical fields, expand the employment horizons and earnings potential of millions of new American workers, and strengthen America’s future. The Act helps increase our nation’s competitiveness by driving recruitment of K-12 math and science teachers, enhancing the skills of existing teachers, and increasing investments in both scientific research and math and science education. The reauthorization of this act will ensure America remains a global leader in technology, innovation and science in the 21st century,” said Larry Burton, Executive Director, Business Roundtable.

Nonetheless, it was a long and winding road to passage for the bill. The scientific community made an urgent call for it to be reauthorized in September—even issuing a stirring update to the original National Academies report—but despite their best efforts the legislation expired in October before the Senate had time to pass it.

The bill was revived in early December with Tennessee Republican Sen. Lamar Alexander among those credited for bringing the bill forward. Alexander is a strong supporter of the Department of Energy’s Office of Science, which is another of the agencies set to benefit from the funding increases mandated by America Competes.

“We’re very pleased. We think it’s a very, very important statement in support of research,” says Robert Berdahl, president of the Association of American Universities in Washington, D.C.

Society of Photographic Instrumentation Engineers (SPIE) leaders are among researchers, engineers, and others in the science and engineering community celebrating the passage today of the America COMPETES Act of 2010.

“We are delighted to see continued strong support for the National Science Foundation and the National Institute of Standards and Technology,” said SPIE CEO Eugene Arthurs.

“We are also happy to see approval for both continued and new spending for Department of Energy (DOE) research, and support for ARPA-E (Advanced Research Projects Agency-Energy),” Arthurs said. “This is a vital step in building a vigorous innovation pathway, linking the excellent R&D produced by DOE and other agencies to successful commercialization and the creation of jobs.”

Deborah L. Wince-Smith, president and CEO of the Council on Competitiveness, says the group applauds the passage of the America Competes Reauthorization Act with bipartisan support.

“By passing this legislation, Congress has taken a critical step in maintaining America’s leadership in innovation and entrepreneurship, which serves as the foundation for economic growth and long-term prosperity,” she says in a statement.

“The America Competes Act reauthorization paves the way for the vital funding of research, STEM education and American innovation, and will help keep America competitive through a time of great economic uncertainty. We encourage the 112th Congress to build upon this reauthorization and show their commitment to raising the standard of living for all Americans.”

December 30, 2010 Posted by | Academia & Non-Profit, Corporate, Government, Labwork & Science | Leave a comment

A “just friends” Viagra?

The holidays are barreling down on me, and things are wrapping up from now until sometime in late December for my ddn activities, so I figured I’d just drop in quickly (and hopefully drop in for a few short posts here and there during the “off time” just in case you’re visiting).

So, I saw this story today on the possible therapeutic effects of Ecstasy (the recreational drug, that is, not the feeling you get thinking a  lot about Halle Berry or George Clooney…depending on gender or sexual orientation), and I find myself both intrigued and flabbergasted.

Intrigued, because the potential value for treating people with post-traumatic stress disorder, autism, schizophrenia would be fantastic if the research bears fruit.

On the other hand, I’m flabbergasted (or perhaps just wary and concerned) about the treatment of “inability to socialize.” I’m not saying this isn’t a serious issue and a potential problem for quality of life, but I worry. Because I remember how Viagra came around (and its successors and peers thereafter) to help men with erectile problems, and then it became something to be used recreationally by many to simply give them more…endurance, shall we say?

I’m not sure I like the idea of taking Ecstasy into the realm of dealing with socialization issues among people who probably just need some good counseling to figure out how to interact with people and make friends. But, as with so many medications, if it ends up going mainstream and prescription-grade, it will likely soon move from being for a select group of people who really, really need it to being purchased en masse by people who simply want it or think they need it when there are better answers.

It’s not a new dynamic, of course, but it is still a worrying thought.

I’m sure there some sort of pill I can head to the pharmacy and get to deal with that feeling.

December 22, 2010 Posted by | Labwork & Science, Market Trends | , , , , | Leave a comment

Scripps researchers make metabolic breakthrough

Normally, this kind of story would be something you’d see in the “Bench Press” portion of our e-newsletter or website (click here) but in this post-Thanksgiving season, our cornucopia of stories was overflowing a bit, and I hate to throw out perfectly tasty and still-fresh leftovers, so enjoy this bit of research news right here at the blog.

Scripps researchers’ synthetic agonist of hormone-mediating proteins could yield treatments for metabolic disorders

By Amy Swinderman

JUPITER, Fla.—Scientists at the Scripps Research Institute have discovered the first synthetic agonist of retinoid-related orphan (ROR) nuclear receptor, protein molecules that mediate hormone activity inside the cell. Publishing their discovery in the November issue of the journal ACS Chemical Biology, the scientists say their finding could pave the way for the development of a therapeutic compound to treat numerous metabolic disorders and even cancer.

The lead author of the study, Dr. Thomas Burris, a professor in the Department of Molecular Therapeutics at Scripps’ Florida campus, and his colleagues have for years focused on nuclear pharmacology and chemical biology, and in particular have been investigating orphan receptors of the nuclear receptor (NR) superfamily. Members of the NR superfamily display a conserved domain structure with highly conserved DNA-binding and ligand-binding domains, and include the receptors for the steroid hormone, thyroid hormone and bile acids and oxysterols. Although many of the 48 NRs found in the human are characterized as ligand-activated transcription factors, a significant number of these proteins still have uncharacterized ligands.

The retinoic acid receptor-related orphan receptors RORα and RORγ are two of these orphan receptors that have been demonstrated to play important roles in regulation of metabolism and immune function. Last year, Burris and his colleagues identified a high-affinity synthetic inverse agonist of this same pair of nuclear receptors.

For this new study, the researchers extended that discovery with the identification of the compound T1317, as a molecular scaffold to synthesize an array of compounds and assess their activity against a number of receptors, including RORα and RORγ. The one compound that stood out was SR1078, which displayed a unique pharmacological profile that indicated it had a high potential for use as a chemical probe for assessing ROR receptor function in general.

The agonist, a compound known as T1317, is a molecular scaffold to synthesize an array of compounds and assess their activity against a number of receptors, including RORα and RORγ. For this study, the Scripps researchers describe the identification of a synthetic RORα and RORγ ligand, SR1078. SR1078 modulates the conformation of

RORγ in a biochemical assay and activates RORα and RORγ driven transcription.

“Pharmacokinetic studies indicate that SR1078 displays reasonable exposure following injection into mice, and consistent with SR1078 functioning as a RORα/RORγ agonist, expression of two ROR target genes, glucose-6-phosphatase and fibroblast growth factor 21, were stimulated in the liver,” Burris says. “Thus, we have identified the first synthetic RORα/RORγ agonist, and this compound can be utilized as a chemical tool to probe the function of these receptors both in vitro and in vivo.”

Those properties, Burris says, give SR1078 potential as a possible therapeutic compound for many different metabolic disorders. Nuclear receptors have become drug development targets for diseases like type 2 diabetes, atherosclerosis and metabolic syndrome, and have also been implicated in the progress of a number of cancers.

However, Burris cautions that this was a proof-of-principle study, and additional experiments are needed to examine the pharmacological profile of this compound in vivo.

“We’re certainly interested in merging the academic side of this with the drug development side,” says Burris, who spent 10 years working for pharmaceutical companies Johnson & Johnson and Eli Lilly & Co. “We’re still looking around for folks who will take it further, because of course on the academic side, we can only take it so far.”

The first author of the study, “Identification of SR1078, a Synthetic Agonist for the Orphan Nuclear Receptors RORα and RORγ,” was Yongjun Wang. Others authors include Naresh Kumar, Philippe Nuhant, Michael D. Cameron, Monica A. Istrate, William R. Roush and Patrick R. Griffin. The study was supported by the National Institutes of Health.

December 8, 2010 Posted by | Academia & Non-Profit, Labwork & Science | Leave a comment

Research efforts can help fragile young lives

Whether we are researchers or journalists, as we explore the landscape of the drug development world we are reminded just how fragile human life can be.

This week, I came across a story that a team of pediatric cancer researchers have identified variations in a gene as important contributors to neuroblastoma, the most common solid cancer of early childhood.

The study team, led by researchers at The Children’s Hospital of Philadelphia, found that common variants in the LMO1 gene increase the risk of developing an aggressive form of neuroblastoma, and also mark the gene for continuing to drive the cancer’s progression once it forms.

The team’s work appears online in Nature. According to their report, a cancer of the sympathetic nervous system that usually occurs as a solid tumor in the abdomen, neuroblastoma accounts for 10 percent of childhood cancer deaths.

According to its website, The Cancer Center at The Children’s Hospital of Philadelphia cares for more children with cancer than any other pediatric hospital in the United States. It is ranked second in children’s cancer care in the U.S. by U.S.News & World Report.

It is so sobering to think that the youngest and most vulnerable—our children—sometimes are engaged in a fight for their lives as they battle myriad issues that come with adult-sized problems.

The researchers found a significant association between neuroblastoma and the LMO1 gene, located on chromosome 11, detecting the strongest signal among patients with the most aggressive form of the disease. This portion of the study identified SNPs, changes in a single letter within the DNA sequence, which predispose a child to developing neuroblastoma.

The research team utilized genetic tools to decrease LMO1’s activity, and showed that this inhibited the growth of neuroblastoma cells in culture. Increasing LMO1 gene expression had the opposite effect, causing tumor cells to proliferate.

Because other genes in the LMO family are known to be active in acute leukemias, other researchers have been investigating potential anti-leukemia drugs to target portions of the LMO pathway.

This is just one example of all of the great work going on in labs in hospitals, academic institutions and other facilities in the United States and around the world.

The research offers great hope for children battling cancer. It also is further proof that the work being done in research labs around the world are yielding results that can take steps to eradicate the diseases that take a great toll on us emotionally and physically, regardless of sex, age or race.

Further, the study by researchers at The Children’s Hospital of Philadelphia shows how we can expand our knowledge base for translating genetic discovery to clinical issues through integrative genomics, combining SNP discovery arrays with gene expression arrays and other functional approaches.

With the great results coming from this work, perhaps the suffering of some of the small and meek can be eased.

December 7, 2010 Posted by | Academia & Non-Profit, Announcements and Events, Labwork & Science, Uncategorized | Leave a comment

Oversaturation vs. tunnel vision

One of the things that drives me nuts when I go to the grocery story is heading down the aisles and seeing a slew of new products that seem to be designed solely to part me with my money and make less space for established items that I already like more than they do to enhance my life. Truly, I have to wonder if adding marshmallow bits to a cereal or making a chocolate version of a healthy oat-based staple of the breakfast table is really necessary. And cheeseburger flavored Doritos? Don’t get me started.

I admit that I sometimes feel the same way when I see multiple iterations of drugs, whether over-the-counter or prescription, or when I see large-scale research efforts that cover the same ground but with different budgets and different researchers.

Then I realize that it’s just that growing “grumpy old guy” persona that started to kick in just before I officially entered middle age. Yes, sometimes pharma companies do simply re-tool a product or create a knock-off version of something for the sole purpose of capturing market share and providing something that seems new but really isn’t.

But so many other times, products that seem similar (particularly when it’s a new chemical compound from the same company for the same target or a similar-acting one from a rival company) need to be pursued for the very reason that subtle differences can have big impacts in terms of efficacy, adverse reactions and off-target effects. I was reminded of that in writing a story yesterday for the web site on Amarin’s promising new omega-3-based triglyceride-lowering drug, which on the surface seems a lot like GSK’s Lovaza.

On the research side, I also got the reminder of how looks can be deceiving when I wrote two stories for the upcoming December issue of ddn that cover the 1000 Genomes Project and the PGP-1K, respectively. When you have two high-profile projects doing complete human genome sequencing with the magic number 1,000 involved, you start to ask questions like: “How is your project different from that one?”

But the fact is that they do have different focuses and different approaches, and because of that, they aren’t duplicated efforts that are redundantly spending money, but rather two projects doing work that will likely provide support to each other’s efforts in the long run, as well as each provide distinct and unique contributions to genomics.

Because, in the end, if there’s anything we’ve learned from decades of pharma and biotech breakthroughs, failures and other newsworthy outcomes, the human body and human diseases are an arena where we aren’t ever likely to have all the answers, and might be lucky to get even a handful of them, no matter how many similar or even duplicative efforts we have.

Too much money poured into certain areas, even important ones like cancer, can mean oversaturation and dilution of research dollars that might be needed on other disease areas. But too little diversity in the research and development sphere, and we can end up with tunnel vision…and look where that got us when we took our eyes off new antibiotic research and ended up with a whole lot of resistant pathogens and not enough ways to fight them.

November 30, 2010 Posted by | Corporate, Government, Labwork & Science | , , , , , | Leave a comment

Putting a face on diabetes isn’t too hard

In my role with ddn, whether in the print publication or the online venues, I get to write about myriad diseases that affect the human population on this planet.

From Huntington’s and Alzheimer’s diseases to diabetes and cancer, companies that we cover each month are working to find new ways of battling these insidious diseases.

In talking with many of the people on the front lines of the research effort to develop these treatments, I get to put a name and face to efforts to improve our chances of surviving and thriving when these illnesses strike.

Over the years, the pages of ddn have featured stories about diseases that strike particularly close to home for those of us writing the articles. For me, it’s diabetes.

November is Diabetes Awareness Month, and it serves as a reminder for us all of a disease that is a growing problem.  According to some health studies, by 2050, one in three adults could be diabetic.

Today, nearly 24 million Americans have diabetes – including an estimated 6 million Americans who have it and don’t know it. It is estimated that another 57 million adults in the U.S. have pre-diabetes, placing them at increased risk for developing type 2 diabetes. Type 2 is the most common form of diabetes, accounting for 95 percent of all cases.

I don’t have to look too far to put a face on diabetes. My wife was diagnosed with type 2 diabetes just over a year ago. Her father was diagnosed five years ago, but he lived with the disease for many years and didn’t know it, choosing to ignore the warning signs.

As a result of years of living with uncontrolled diabetes, my father-in-law today is 64 years old, legally blind and must undergo kidney dialysis three times a week. For him, it’s a matter of life or death.

After being diagnosed, my wife felt as if she were just handed a death sentence. I knew from the stories I’d written that there are two ways to live – you can control the disease or it can control you.

We’ve learned that type 2 diabetics can take a number of oral medications to help their body metabolize sugars, increase insulin production or block the absorption of carbohydrates.

My wife also has a key advantage over her father in her fight against diabetes – early detection. While a diagnosis is a life-changing experience, knowing the what you’re up against is instrumental in effective treatment.

It also is key in thwarting the ravages of diabetes, which include neuropathy, which causes tingling, numbness and destruction of nerves. Because of neuropathy, diabetics must check their feet regularly to ensure they have no wounds. If undetected, a wound can lead to infection that could lead to amputation. Diabetes may also lead to kidney disease, heart disease and blindness.

We’ve learned from the endocrinologist that the goal of diabetes treatment is the prevention of long-term complications. We’ve also had to talk to dietitians and diabetes educators, learning that it truly takes a village to keep a diabetic well.

I’ve learned that even a simple trip to the optometrist can heighten anxiety in my wife. She has her father as a constant reminder of what can happen with this disease.

Type 2 diabetics can take a number of oral medications to help their body metabolize sugars, increase insulin production or block the absorption of carbohydrates, and that’s where we are at. Thanks to the work of countless researchers over the decades, these treatments are available to help diabetics keep their blood sugar levels in check.

Researchers today are at work to discover new drug candidates that have greater efficacy against these diseases. Stepping outside of my role as a journalist and speaking as a husband and father, I have to say just how grateful I am for the work of these researchers. Whether you work at the biggest pharma or the smallest of labs, your work is having an impact on the lives of people the world over.

Some of you may even be motivated in the lab by your own personal experience. We all can share that common bond in the human condition. The faces of these diseases are all around us, and the battle is no less important today. That key discovery that could deliver the knock-out punch to a disease could be occurring in a lab right now.

And to all of the researchers who toil each day to find the Holy Grail, I thank you.

November 17, 2010 Posted by | Academia & Non-Profit, Corporate, Government, Labwork & Science | , , , | Leave a comment

For stem cell case plaintiff, faith and science go hand-in-hand

Yesterday, we shared a Q&A with the lead plaintiffs in the controversial federal lawsuit challenging federal funding for embryonic stem cell (eSC) research, Dr. James L. Sherley, a biological engineer at Boston Biomedical Research Institute, and Dr. Theresa Deisher, research and development director at AVM Biotechnology LLC in Seattle.

Both researchers agreed to field questions about their beliefs regarding eSC research. Deisher, who has been especially vocal about her Catholic faith and how it informs her research, also agreed to take a few questions about the connection she sees between her beliefs and science.

According to Deisher’s bio, she has 17 years of experience in scientific and corporate leadership positions involving research, discovery, production and commercialization of human therapeutics. She obtained her Ph.D. in molecular and cellular physiology from Stanford University. Prior to founding AVM Biotechnology in 2007, Deisher held positions at Repligen Corp. in Cambridge, Mass., ZymoGenetics Inc., Immunex and Amgen in Seattle and CellCyte Genetics Corp. in Bellevue, Wash. She has had 23 patents issued and has published numerous scientific manuscripts.

ddn: Do you find any conflict between your faith and the scientific research you engage in?

Deisher: I do not find any conflict between my faith, which is Catholic, and my research. My faith enhances my work. My Christian faith calls me to focus on drugs and treatments that are affordable so that the greatest number of people will benefit. My faith calls me to use reason and the order of natural law to determine, for instance, the stem cell most optimal for clinical use. My faith calls me to focus only on those treatments that will be effective. My faith also calls me to respect the intrinsic dignity of human life in my work.

ddn: How does your faith impact your research approach?

Deisher: My faith is completely complementary to my research, which focuses currently on stem cells for regenerative medicine and alternative vaccines.

Adult “self” stem cells, meaning a patient’s own stem cells, are affordable, compared to all other stem cell therapies. For the most part, therapies using adult stem cells will cost about $25,000 compared to Geron’s projected $500,000 for embryonic stem cell-based therapies. Adult “self” stem cells are found naturally in every organ, in each of us, and they are “preprogrammed’” to perform the functional regeneration that patients require. They also lack the issues of immune rejection or tumor formation that plague pluripotent stem cells such as embryonic stem cells. Adult “self” stem cells are far advanced in clinical trials, and in comparison to “patented” stem cell lines, they show more effectiveness in patients. Whether one believes in God or Darwin, one can arrive at an optimal stem cell for patients using objective measures, common sense and business criteria to generate the greatest good for the most people.

I would apply these same criteria to any type of treatment that I would work on, including biologics and small molecules: Will the therapy be affordable, or will only the very few benefit? Will the therapy be effective or merely enhance my stock price or financing temporarily? Will the therapy be undermined by adverse side effects? These criteria are sound business objectives and compatible with my faith.

November 11, 2010 Posted by | Academia & Non-Profit, Corporate, Government, Labwork & Science, Uncategorized | , , , , , , , , , , , , | Leave a comment