Latest Drugwonks' Blog
Personalized medicine for quicker cures
As we listen to presidential candidates and Congress debate how to control health care and drug costs and raise drug safety standards, most solutions lead to increased government regulation. That is underlined when debate turns to cancer care with high-cost drugs and low returns in quality of life and survival.
But more government regulation on reimbursement rates and overly burdensome drug development requirements will result in less medical innovation and rationing of medical care. What's missing from the debate is the public policy encouragement to produce more "smart" medicine.
One of the hardest parts about being an oncologist is telling patients they have cancer. Even harder, though, is knowing that the drugs prescribed don't work well in most patients and/or will cause severe side effects in others.
That is changing. Scientists working at biotechnology companies are using the knowledge of the human genome to develop drugs targeted to help individual patients. That concept -- known as personalized medicine -- holds the promise of improving care for patients and quickening the pace of drug development so we can "hurt the cancer more than the patient."
The human genome has shown us how our genes affect our health -- including whether we are at risk of disease. Recent advances in DNA research also have taught us how to examine what happens to a cancer tumor's gene or set of genes when they are turned on or off in the presence of a drug.
At my company, Cell Therapeutics Inc., one of our subsidiaries, Systems Medicine, is examining the function of genes in a tumor through a process called genetic profiling. SM examines about 30,000 relevant genes from tumor cells and tests them to determine which gene or genes make the tumor susceptible to the effects of different cancer drugs. Using that technique, we can identify which cancers are sensitive to which drugs. It is a fundamental shift in approaching the dilemma of which is the right drug for the right patient while exposing the least number of patients to a drug's severe side effects.
The power of this technology is enormous. Rather than imposing new regulations on manufacturers or physicians, the most effective way to ensure safety is to use scientific advances to better understand why some patients react badly to some drugs, and encourage development of new diagnostics and therapies that take that into account.
Adoption of personalized medicine will mean that patients will get quicker access to the newest cancer therapies. The current time and cost of drug development is about 16 years and $1.7 billion. Cancer drug development is actually the least-efficient in all of medicine. Genetic profiling could shorten clinical development time and costs because only those patients likely to benefit will be enrolled in clinical trials, making it easier for regulators to make safety and efficacy decisions more quickly.
But, federal regulators need to understand the value personalized medicine can bring and develop policies that encourage development of these new drugs and diagnostics, while doctors need to understand the power of genetic profiling so they can make good prescribing decisions. Some day, instead of physicians diagnosing lung cancer or colon cancer based on where the tissue is from and the way a tissue looks under the microscope, we may be referring to a genetic profile of a tumor in your lung or colon tissue recognizing that the genes that make each of us unique also makes our cancers unique.
The product is Ivory Soap -- and it's only 99 and 44/100th% pure. Should it be taken off the market?
Silly? Not when you consider that the current debate over drug safety is being hijacked by the Panjandrums of the Precautionary Principle. What we need is a debate on smart safety.
Have a look at this new op-ed in The Journal of Life Sciences, "A Balancing Act:."
Here's the link:
www.tjols.com/article-662.html
And here's a taste:
His video interview can be found here:
www.vimeo.com/1045899
(Other "newsmakers" interviews can be found at www.cmpi.org)
Dr. Weber, we are proud to say, serves as Chairman of the Board for the Center for Medicine in the Public Interest (the public policy home of www.drugwonks.com).
According to Bloomberg, two companies that produce different types of stem cells have signed contacts to sell their products to drugmakers, showing the new technology will be used to help discover medicines not just to repair or replace damaged cells.
California Stem Cells Inc., an Irvine, California, biotechnology company that turns embryonic stem cells into neurons, said today it's selling the brain cells to researchers trying to find drugs to treat Lou Gehrig's disease. CellDesign Inc., of
The efforts of these two closely held companies indicate stem cells will aid in the search for old-fashioned drugs long before they're infused into patients. It also suggests that the first businesses to benefit from stem cell technology will be traditional pharmaceutical companies and their suppliers not developers of new kinds of therapies.
``It's similar to what happened in the last century with molecular biology'' and gene therapy, said John Hambor, CellDesign's founding chief executive officer, in a telephone interview yesterday. ``We're now going down a similar path with stem cell biology. While it may lead to cures by itself, it will drive the next generation of drug discovery.''
Here's a link to the complete story:
http://www.bloomberg.com/apps/news?pid=20601124&sid=aYKfThhsUVwI&refer=home
At a recent European Commission-sponsored high level conference, Internal Market Commissioner Charlie McCreevy, commented that the EC wants practical, pragmatic suggestions for fighting the “modern-day highway robbery” known as piracy and counterfeiting. McCreevy stressed that more regulation is not the answer, and that solutions lie with public-private cooperation.
Only governments can put into place enhanced investigatory assets and harsher penalties.
Public/Private cooperation? Certainly. But only governments can catch and keep criminals in jail.
When it comes to discouraging international prescription drug counterfeiting, what we need is a more robust cell culture.
Now it turns out that Avastin, the drug that Goozner and Mahar love to hate shows considerable promise in extending average progression free survival by up to nine months. Contrary to the twisted narrative put forth by critics, the discovery of the off-label use of Avastin for gliomas came from academia without industry funding (which of course Goozner also criticizes...either way industry is damned, pilloried for inventing useless new uses and attacked for not being innovative)
Here's what happened:
"In the pilot study, the researchers found that dual therapy with Avastin and the chemotherapy drug irinotecan either shrank the tumors or restricted their growth in nearly all cases for up to three months longer than comparative therapies. Three months is a significant advance when dealing with these aggressive tumors, Vredenburgh said; common current treatment normally offers only six to 12 weeks of halted growth before the tumor grows and spreads, ultimately destroying cognitive and physical function and leading to death."
Following this discovery, Genentech, Duke University and NIH worked together to conduct an expanded study.. The FDA initially resisted because of side effects such as bleeding, but since 40 percent of all brain cancer patients experience bleeding for a fatal illness the risk seemed worth it (to everyone except Maggie Mahar www.healthbeatblog.org/2008/02/the-wall-street.html
James Vredenburgh, M.D., a brain cancer specialist at Duke's Preston Robert Tisch Brain Tumor Center and lead researcher on the study noted: "Going forward, we will also explore the efficacy of this treatment in newly diagnosed patients," he said. "Ultimately, our hope is that this will offer a real weapon in what is now a very limited arsenal for treating a very challenging cancer."
www.cancer.duke.edu/btc/modules/news/article.php
That includes patients like Senator Kennedy. Let's hope that his doctors rely upon experts like Vredenburgh in making treatment decisions, not cynical second guessers like Goozner and Mahar who would let hatred of drug companies color life and death decisions.
Ford and From describe the Center this way:
"The American Center for Cures would be a public/private partnership that would function as an independent entity within the National Institutes of Health, targeting research resources from government, academia and the private sector on cure-driven projects. It would pay for high-risk, high-reward research, fund small businesses that have created possible cures but lack the money necessary to test drugs in clinical trials, and work to streamline the clinical trial process."
There is a danger is politicizing science of course, (what else is new?) but if the ACC can roust the NIH out of its current torpor and shift resources towards more partnerships and clinical trial reform, it is worth a discussion.
thomas.loc.gov/cgi-bin/query/F
Biotechnology companies also raised $29.9 billion in investments and loans, the most since 2000. The industry attracted a record $39.4 billion in 2000, the year the first draft sequence of the human genome appeared.
IPOs brought in $2.2 billion, a 21 percent increase over the previous year and the highest total since 2000.
The full Bloomberg story can be found here:
http://www.bloomberg.com/apps/news?pid=newsarchive&sid=a6FjDvLrCCgM
The article mentions that, “Large drugmakers are investing in biotechnology to gain new medicines to bolster revenue as patents on top products expire.”
No doubt. But while acquisitions and licensing is the wave of the present, what will drive the future of pipeline, profit … and public health is the Critical Path. To achieve “A” level health care, industry and academia and government must focus on “B” level issues -- Better science and better tools (biomarkers, bioinformatics, and Bayesian statistical analysis) among others.
So, deal or no deal, the Critical Path must not remain the road less traveled.
Read Boston Globe article
I have noted previously that other friends of mine have received combination of Rituxan and Avastin to treat NHL. (Other drugs for NHL such as Bexxar will hopefully grow in use as treatments are tailored to individual disease progression.) Both of those drugs were not and are not widely available in single payer health systems. Cynics like Merrill Goozner even believe Avastin is worthless. My advice as always is for people to receive medical care consistent with their ideological position and refuse medicines they believe were brought to market or marketed in ways that are corrupting or without the evidence they believe meets their utopian formulary. And don't let your family have access to the same drugs either.
Leave Jon Lester and the rest of us alone.
"A drug company brings a new drug to market based on government-funded research. It charges a huge price for the drug, but since its the insurance companies money, it's everyone's money, which means it's no one's money. So no one complains -- for a while. What does Teva do with the huge cash flow that comes from selling this very expensive drug to a small population of MS sufferers? It funds clinical trials to show it's drug is superior to other in the field, which it shows, sort of. But the trials are never really good enough to prove superiority, just good enough to establish market dominance, which was probably the real goal of the trials. So the government has to sort things out, but it gets back into the game very late and very slowly. The insurance industry, fed up with paying extraordinarily high prices, starts putting the financial onus on patients."
www.gooznews.com/archives/cat_drugs.html
Goozner is referring to the Tier 4 payment category which by itself is crazy. But all of a sudden comparative clinical trials are no good. Maybe they should be bigger and government run. Then there would be no drugs except the one drug selected by government to hit the market. But wouldn't that create a monopoly price? No problem. In the Gooz's world. The price would be slashed by licensing out product to generic companies who would sell the rights to produce the breakthrough at pennies per dose.
Because innovation all flows from government anyways and all the R and D and the marketing and education carried out by private companies is sort of just greedy rent-seeking behavior of the basest sort... I would have no problem have a respectful debate on the best way to advance medical progress but Goozner and others want to shut off debate by attacking motives and funding sources. As I have noted, it cuts both ways but the media and the blogging world are intensely hypocritical on that score.
One other Goozworthy note...He claims I don't support embyronic stem cell research. He infers that because he asserts I am a conservative and CMPI is conservative and therefore assumes I hold down the line social conservative positions. In otherwords, Goozner who never bothered to check or ask me. For the record, I support ESC research though I have tremendous respect for the positions of those that do not and I don't write about it because it is not an area that is CMPI's central focus.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
