-
Bevacizumab (Avastin) did not demonstrate significant efficacy in early colorectal cancer - watch an embedded video of the experts discussion. Interesting.
-
Today at ASCO Several Lymphoma Studies Presented in lymphoma. Oddly, they did not mention the romidepsin data in CTCL or MCL though.
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May 2009
May 31, 2009
Lung data at ASCO shows mixed results
Yesterday at ASCO was frantic, there is no other way to describe it as one hurtled from one session to the next. There is little time for reflection in a huge meeting of over 30,000 squeezed into a small space, but one thought crossed my mind while sitting in the lung and colorectal cancer sessions. Namely, the abstracts often contain preliminary data from several months back and the data should be taken with a large pinch of salt. A good example of this was OSI/Genentech's Tarceva, which at first glance suggested a small benefit of only 8 days as maintenance therapy. The data being presented this morning is a little more robust than that, so the media should beware of jumping to hasty conclusions.
Typically, advanced lung cancer patients get 4 cycles of chemotherapy and then their progress is monitored until progression, whereupon another therapy is tried. The big question raised recently, is whether adding a new treatment or combination as maintenance therapy would improve the survival. At this ASCO meeting a number of trials attempted to answer this important question.
Let's look at Tarceva therapy compared to placebo in this setting. The final results of the SATURN trial demonstrate that Tarceva actually significantly improved PFS in chemo naive patients with both squamous and non-squamous cell lung carcinoma by 41% compared to placebo. Patients with an EGFR mutation achieved a 10 fold increase in the time they lived without their disease worsening. Sadly though, the biomarker data suggested that KRAS mutation status was not a predictor of efficacy in NSCLC in patients treated with Tarceva, as had been hoped.
A second trial, called ATLAS, looked at the combination of Tarceva with Avastin following standard chemotherapy as maintenance therapy after first-line treatment with chemotherapy plus Avastin in patients non-squamous histology. Patients treated with this combination saw their cancer growth slow more than a control group treated with Avastin alone.
Over 750 patients were randomized to receive Avastin and placebo, or Avastin and Tarceva. Patients in the Tarceva group survived an average of 4.8 months before the cancer started growing again, compared to 3.7 months for the control group. This eqautes to a 29 percent reduced risk of disease progression for patients who took the Tarceva and Avastin combination compared to Avastin alone.
Vincent Miller, the presenter stated that, "This is the first study to show that adding erlotinib (Tarceva) to maintenance therapy with bevacizumab (Avastin) delays disease progression in patients who have already received bevacizumab as part of their initial chemotherapy."
In a third trial with Alimta (Lilly), 663 non-squamous patients with metastatic lung cancer were randomised to receive Alimta or placebo. Preliminary results showed that patients who took the drug lived 26 percent longer compared to the control group, ie 13.4 months compared with 10.6 months.
The lead author and presenter said that, "Maintenance therapy with Alimta (pemetrexed) offers a new paradigm for patients who have advanced lung cancer, because it has a low toxicity and can be given on an ongoing basis over a prolonged period of time to extend patients' lives."
There was very little hot news to report from yesterday's colorectal cancer sessions, but the data in advanced lung cancer could well change the landscape and significantly improve survival for patients with the disease. That's great news all around. Mind you, I wouldn't like to be one of the companies with ongoing trials in this area though, the standard of care could well have changed with these results and the bar raised a little higher for new entrants. That's good news for patients though!
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May 29, 2009
Follow the aggregated #ASCO Twitter stream
It's that time of year when the annual American Society of Clinical Oncology meeting rolls around, this year from the heat and humidity of Orlando.
Some people are tweeting the different sessions using the #ASCO tag and others #ASCO09. Still others are just typing ASCO or @ASCO, although the poor organisers might be a little overwhelmed by that as all tweets will show up in their @mentions. Rather than do repeated searches on Twitter, you can follow them all via the handy little CoverIt widget below:
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Picture of the Day
I'm off to Orlando to the annual ASCO meeting, so I'll leave you all with a thought for the day before the hurly burly starts. Sometimes, working in the cancer field we need to remember to stop and smell the flowers.
Life is for living after all.
May 28, 2009
sanofi-aventis and Exelixis agree licensing deal
This morning I woke up to the news in an email alert that one of my client companies, sanofi-aventis, has been busy on the licensing front again after their recent deal with BiPar. This time they have licensed two compounds from Exelixis, which are PI3-kinase inhibitors, XL-147 and XL-765. You can read more about the deal itself on Mike Huckman's CNBC blog. Regular readers of this blog will recall some previous posts on PI3-kinase inhibition such as here and here.
It's an interesting and relatively 'hot' pathway with the advent of mTOR inhibitors and the role of AKT-PI3-Kinase pathway in potentially reducing drug resistance and limiting the feedback loop seen with mTOR.
The deal with a big Pharma suitor was inevitable after Exelixis signalled their intentions last December during the end of year pipeline update, according to my notes made at the time. The slight development lead in this area turned into a profitable deal for Exelixis.
At the recent AACR meeting in Denver, I noticed there were several others not far behind and some phase I data is expected to be presented next week at ASCO by Semafore on their PI3-kinase inhibitor, SF1126.
According to one of the abstracts:
"SF1126 is composed of the pan PI3K inhibitor LY294002 conjugated to an RGD targeting peptide. It is designed to increased solubility and binding to integrins expressed on tumor vasculature. This targeted prodrug enhances tumor delivery of the active inhibitor, improving antitumor efficacy and tolerability in xenograft models."
Other companies with receptor tyrosine kinase inhibitors (RTKIs) in phase I development include Novartis, who have two PI3-kinase inhibitors BEZ235 and BGT226, Genentech with GDC-0941 and Merck, who have an AKT inhibitor, MK-2206. There are also others in preclinical development. The number of mTOR inhibitors in R&D is now too numerous to mention in addition to the three already available commercially (sirolimus, temsirolimus and everolimus).
In the final analysis though, this looks a very good deal for Exelixis, who get another couple of their portfolio licensed out and relatively low risk for sanofi-aventis but neither PI3-K inhibitor will address the urgent shortfall they face when both Taxotere and Eloxatin go off patent in the very near future. Some late phase II/III compounds are still a glaring gap in the French company's pipeline.
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May 27, 2009
Listening to patients is the most important thing in Pharma Marketing
Yes, that's right; patients. Not doctors, nurses, pharmacists, payers or other HCP's, but patients.
Patients are the people taking the therapies, educating themselves, their families and other sufferers about the disease, and quite frankly, often the marketing people responsible for the commercialisation of drugs. They can make or break your brand as promoters or detractors without you even knowing it, so my advice to Pharma marketers is ignore them at your peril.
This applies to depression, hypertension, cancer, asthma, or just about any disease there is. The silent voice has real power. How many Pharma marketers actually routinely listen or monitor patients though? Some might do it once a year, but really, my opinion of those nebulous and expensive focus groups favoured by market research companies cannot be printed here. The samples are prone to bias and desperation by the recruiters anxious to fill the slots and anyway, how on earth is 20-30 patients representative of a disease universe? The answer is, it isn't nor is it even directional.
One of the coolest things ever invented surely was the internet. I doubt if Tim Berners-Lee ever imagined the sheer power of information that might even be possible with search engines. Patients blog, they chat in forums, they communicate in multiple ways. All of this activity can be a gold mine if you know where to look and how to influence them. Feedback of course, goes both ways - learn to listen, take your lumps on the chin, improve and make things the best you possibly can given the time and resources you have available. Life is not a game of perfect, it's about doing what matters and caring about the patients.
Take a look at this site, for example. Jerry Mayfield is a quite simply a great guy. A former Louisiana State Trooper, he created a wonderful website in his own time and money after being diagnosed with CML. He regularly added new links to relevant resources and physicians for other fellow sufferers of the disease and a whole cottage industry sprang up around the buzz relating to the Gleevec CML trials way back in 1999-2000. I know, because as one of the marketers, we followed their progress avidly and were humbled, excited and saddened along the way. I am pleased to see that the concept has expanded and now new 'Talks' exist for other new therapies in this area, enabling patients to ask questions, share their experiences and generally provide virtual support for each other.
From a company perspective, the information was incredibly useful as we followed their lives remotely, working on ways to improve things whether it be to push the company to make more drug or speed up the development. The power of a strong patient group can really help your case. They can also tell you about their needs, their goals, their side effects and how well different dosages are working; critical information you could never hope to get in a focus group. It helps you plan ahead, working with key opinion leaders and the medical team to publish papers on practical side effect management, for example. These days, web2.0 and social media monitoring make computerisation and tracking of these sentiments so much easier and quicker compared to 10 years ago and the days of manually reading through hundreds of postings around cyberspace. I ardently wish I had the tools of today available then.
It's been a while since I checked out the patient groups as time and my role has moved on but today I popped back to look at Jerry's site while wondering how how he and several others were doing. After all, before Gleevec, CML patients typically lived 2-4 years and it's now 11 years since the first patient first visit back in 1998. Partly, I was hoping for a minor miracle that perhaps our dream of helping CML patients live to 10 years would be realised. Nervously, I clicked through and found a wonderful posting on the first page of the Gleevec talk posted earlier this month:
"I was dx 1/01
and it took 3-4 years and Gleevec at 800mg to get my bcr 0.000 (up/down
a little, last test 0.004). I learned from this site how to be
proactive in your healthcare. If it wasn't for this site I'd probably
be dead by now. I had to tell my doctor when he needed to increase my
dose. He became confident in me because of the knowledge I expressed
about CML and Gleevev. Because it was very rewarding to be able to
learn more about my condition and then work WITH my doctor in my
treatment, I decided to go back to college to be a nurse. I'm 47 yrs
old and in three weeks will be graduating with my Bachelors degree in
Nursing. This is why I haven't had time to really follow the site and
post. It has been very rewarding (and very difficult) and if I would
never have been dx with CML I probably would not have done this. At
times I almost forget that I have CML. The side effects of Gleevec are
just a part of my life now. I just would like to encourage everyone to
not give up your future goals just because you have CML, live it to the
fullest, and DEFINATELY be proactive in your health and healthcare."
Wow. That's far better than I might even have hoped for after meeting the doctors involved in the phase I clinical trial way back in September 1999. This is true patient power and promotion helping educate her own physician become more confident, willingly sharing knowledge with others and getting on with her life despite a life threatening disease. This is stuff dreams are made of. It gives me goose pimples thinking about turning even one cancer into a chronic disease like diabetes.
Image via Wikipedia
The management was pretty good too - very supportive and encouraging at every step of the way without interfering or micromanaging. You couldn't ask for more. We were also lucky to have some great partners in the agencies and third party companies we worked with. The physicians in the phase I and II trials were some of the best and brightest I've ever worked with, but what made them stand out was they really cared about their patients welfare.
The whole process became like a relay race with the baton being passed around from one team to another as the product moved inexorably towards market approval. For everyone who worked on the STI571/Gleevec development it ceased to become a job per se, instead it became an honour to participate in whatever small way to making a difference to patients lives. The most powerful thing you can listen to is their stories, about their fears, their struggles, their hope and ultimately, overcoming the obstacles. I have yet to hear one that didn't make me cry or be sensitive to the moment.
Real teamwork starts with the right people. Every once in a while we all get a chance to be part of something truly incredible. And it starts with putting the patients first. Do that and the rest will follow.
This post is dedicated to the brave patients who willingly took part in the Gleevec trials, the leukemia physicians who ran them and my team mates who did everything possible to making a real breakthrough in cancer treatment happen. I salute them all.
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New data on Abiraterone in prostate cancer
I was away for the Memorial Day weekend last Friday and hence missed the big news in oncology that Johnson and Johnson were purchasing Cougar Biotechnology for $1 billion. Note the whole company, not just a licensing deal for the promising prostate cancer compound, abiraterone.
Wow.
The data hasn't even been announced at ASCO yet, that's this weekend coming, so one can only speculate that suitors got a sneak peek of the data under an NDA. A billion dollars certainly gets attention and raises the ante for future small oncology biotech deals.
Just now, Cancer Research UK tweeted about the new data:
Twitter is rapidly becoming my real time alert system for accurate competitive intelligence. You can read more about CRUK's perspective HERE, but essentially the data tells us the following:
"Researchers at the Institute of Cancer Research and The Royal Marsden Hospital tested the drug on 54 men with aggressive, advanced prostate cancer. In most cases, the cancer had spread around the body, causing many of them discomfort and pain. The men were given abiraterone daily and, twelve weeks later, were given a PSA test and a CT scan to see if the drug had worked. The scientists found that abiraterone worked for around two-thirds of the men on the trial, lowering their PSA levels, causing the tumours to shrink and relieving pain. It wasn’t a permanent cure, as the effects only lasted an average of 8 months. But, interestingly, for men who carried a faulty version of a gene called ERG, the effects lasted much longer – up to 18 months.
This may not sound like long, but it’s certainly a significant improvement on what might be expected for men with such advanced cancer."
Regular readers of Pharma Strategy Blog will know that this is one compound I'm quite excited about. The abiraterone data has been consistent and appears to have fewer toxic side effects compared to standard of care chemotherapy, Taxotere plus prednisone, which can induce severe myelosuppression in many patients.
What was particularly interesting about the new data is that patients with the mutated ERG gene appeared to do better on abiraterone than those without. That's a potentially interesting biomarker to look out for because linking a specific biomarker with therapy for patients most likely to benefit from the treatment is increasingly the way to go in oncology. Now, I'm not sure how many prostate cancer patients might have the mutated ERG gene, so will have to research it unless anyone out there knows?
{UPDATE: Thanks to Twitter and Martin Fenner, it appears that the TMPRSS2:ERG fusion is present in approximately 50% of localized prostate cancers according to this paper}.
Hopefully, there will be more information available at ASCO next week, so watch this space.
Attard, G., Reid, A., A'Hern, R., Parker, C., Oommen, N., Folkerd, E., Messiou, C., Molife, L., Maier, G., Thompson, E., Olmos, D., Sinha, R., Lee, G., Dowsett, M., Kaye, S., Dearnaley, D., Kheoh, T., Molina, A., & de Bono, J. (2009). Selective Inhibition of CYP17 With Abiraterone Acetate Is Highly Active in the Treatment of Castration-Resistant Prostate Cancer Journal of Clinical Oncology DOI: 10.1200/JCO.2008.20.0642
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May 26, 2009
A life in the day of a Pharma marketing strategy consultant...
Recently, a client asked what a typical day as a management consultant is like compared to being in big Pharma. That's a good question, because although much of the work is similar, the work flow is very different indeed.
The three things I absolutely do NOT miss are the perpetual cycle of endless meetings to address someone else's crisis, the politics and insane travel schedule that takes up every other weekend. For some reason, politics is sadly rather prevalent in Pharma. That said though, the huge advantages of running your own company is that a) you are the boss b) you don't have to stress about a twice daily commute that eats up time and c) with all your associates working remotely, every one gets on with their work and doesn't have to worry about keeping up appearances like Mrs Bucket or getting sucked into meetings and politics.
The things I do miss, however, are the water cooler chats about industry news and happenings, how they affect your franchise and what you might need to do about them. Most of us are social creatures so human interaction is important.
Another cool thing about consulting is that you get to balance getting out to seeing clients and potential clients with days in the home office (sometimes the garden table in nice weather, that can't be beat, although the birds chirping is a dead giveaway on phone calls) either beavering away on research and reports or doing business development. Until becoming a consultant, I hated the telephone and avoided it like the plague as colleagues who encountered a full VM box will attest. As a consultant it is a central part of the day, so I have grown to love my Bluetooth gadget that works so seamlessly with the iPhone. It really has revolutionised my life, especially with all the useful apps that allow you to share data across computers to the cloud.
So what does a typical week look like in Pharma?
Arrive, grab coffee while the network takes an eternity to boot, do VM and attempt to answer some emails, rush off to meetings while wondering while such meetings exist. Debate between grabbing a late hasty lunch at desk and attempt a few more emails and VMs or sit down for half an hour with colleagues. Get back to office, but get pulled into another meeting. Then your assistant reminds you about a meeting with a vendor you forgot about. It is 3.30pm and you wonder where the day has gone. Finally get some 'me' time to do some real work. Colleagues pop in and out with questions or wanting a chat. Finally leave the office later than you hoped and dice with the tail end of the rush hour traffic. Arrive home stressed and tired, needing to do some more work after dinner and pack for a 6.30am flight to some conference the next morning.
Sound familiar?
Last week was a typical one for me. A couple of days working on client projects, all in different diesases and cancers with different outputs, two days of business meetings, a day of business development and administration, including working on the concept for a new brochure on forecasting. The only travel was to client or supplier meetings, no daily commute and no wasted time. This week is similar, more work to finish, some client/prospect meetings, some short teleconferences and travel to the ASCO conference in Orlando. The end result is less stress, more fun, but also more uncertainty. At least working for a Pharma company guarantees a regular paycheck every month, consulting does not.
Time is more focused as a consultant, less waste and less meetings. Oh, but it's not all hunky dory: I'm still bad at expenses though, please excuse me while I dash and catch up on three months worth before the next major conference hits. Unfortunately, some things never change.
If you enjoy reading these blog posts, you can subscribe and read them offline at your convenience. I do this for a lot of my favourite blogs myself, reading them in bunches on my iPhone. It's much more efficient while you're whiling away time on a bus, subway, train or plane, though wouldn't recommend it while cycling home though!
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May 25, 2009
Thinking and focusing on the BIG picture
Alan Webber in his book Rules of Thumb came up with a startling rule that made me sit up and pay attention. In it he essentially said keep two lists:
- One that holds what gets you up in the morning
- One for what keeps you up at night.
"Managers and leaders have got to know
themselves before they know their businesses. They've got to have
passion for their work and concern for their world. Otherwise they're
just punching the time clock and risking everyone's future."
That made me think and focus.
I love what I do, getting up in the morning is rarely a problem and enjoy working with a lot of fun people in the Pharma and Biotech industry on cool and interesting projects.
Quieting my brain and getting to sleep is another matter entirely. With the economy in the tank, I worry about my mother as she slips into the twilight of her life with the accompanying physical deterioration, my little brother who is finding business very hard right now and friends who are less fortunate and have been laid off. Some 50,000 jobs have been lost in the Pharma industry alone over the last 18 months, leaving fewer, more stressed people doing yet more work.
Image by Getty Images via Daylife
I try to stay positive, but it isn't always possible.
And then you hear the tragic news that Phil Mickelson's young wife, Amy, has been diagnosed with breast cancer. That puts things into perspective and my thoughts are with her and husband who has just left the PGA tour.
What about you?
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May 21, 2009
Useful iPhone medical apps
This post was stimulated by Martin Fenner, a clinical fellow in oncology in Hannover, Germany. We 'met' in The LifeScientists room on Friendfeed and on Nature Networks. He won a competition for a free iPhone app, specifically the Clinical Trials app but unfortunately couldn't use it in Germany as it is US-only. His loss turned out to be my big gain because I use the NIH website a lot in my daily job but would never have paid $25 for it. Last night I decided to test drive it and also look more closely at a couple of other medical apps I use. In this blog post, I'll review the clinical trial app and review the others in subsequent posts.
Clinical Trials on the iPhone Home Page:
All new apps start off on my first Home page and get moved pretty quickly if not used regularly. I have a feeling that this app is definitely one for keeps on the coveted Home page.
To start off, you get an easier to use menu driven database, which is intuitive and obvious:
My only beef with the search page is that while you can sort by location etc, you can't sort by therapeutic intervention, as per the advanced search on the web page where there is a list you can click and choose from. This is a major omission because different drugs might be entered by codename, generic or brand name. The app forces you to type in the drug in the generic search box and take a chance. It is more designed for patients this way who might be looking for trials in a particular cancer type, so it is probably a niche grumble, although my guess is that many Pharma people would also search the same way.
Yesterday, I was interested in Bcl-2 type drugs, and specifically, Mcl-1 targeted therapies. Thus I typed in Bcl-2 to see what it would produce:
Ok, it returns a general list of trials involving Bcl-2 therapies that you can scroll through and review. The titles are important - they tell you a lot about the trial. The little note underneath each entry is also useful, explaining the intervention, condition tested and the status of that trial.
Scrolling through, I found this interesting page:
Now, I happen to know that Obatoclax is both a Bcl-2 and Mcl-1 inhibitor and was specifically interested in whether there were any trials in malignant melanoma after reading yesterday that Mcl-1 may be an important target in that disease. Interestingly, I found some trials in hematologic malignancies but not melanoma. That was useful to know.
Suppose one is interested in a particular trial, what then? Well, clicking on one of the above selections gives more details about the trial itself like this:
What's useful is that you can email the trial page to anyone, including yourself for later browsing. Also, if you were doing competitive intelligence on mantle cell lymphoma, this sort of granular information is vital if trials are just opened or recruiting. Knowing when recruiting or the study has finished is also helpful in anticipation of the results and the impact on other therapies and competitors in that market space.
Overall Analysis:
I use the full NIH website ClinicalTrials.gov all the time, but often wish for easier access on the road or while visiting clients. This handy little app will help considerably, especially as it is quick and easy to use. The only downside will be searching a big tumour type such as breast or lung cancer and getting hundreds of entries but as an adjunct for finding specific competitor information, I think it will be useful and definitely a keeper in my app library. Would I pay the hefty $25 price tag? I don't know, it's too early to tell, but I sure am very pleased with my freebie copy courtesy of Martin :-)
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May 20, 2009
What are you reading this week?
I'm a voracious reader of books, magazines and RSS feeds, learning is just great fun. Aside from Sporting News and USA Sports Weekly (for my fantasy baseball and football teams, you understand), here's an eclectic selection of things I'm currently reading:
- Three rules for these times (Harvard Business)
- Aberrant miR-182 expression promotes melanoma metastasis by repressing FOXO3 and microphthalmia-associated transcription factor (PNAS)
- Start-up Strategy: To Change the Game, Change the Economics of How It’s Played (Tim Ferriss 4HWW)
- Twelve Key Steps to Address International Pharmacopeia Harmonization (Laboratory Equipment)
- The comment Dungeon - warning, amusing! (Pharnyngula)
- What should come first, marketing or products (Eric Garland's Competitive Futures)
I'll write a bit more later about the last one in Pharma and drug development but what are you all reading and why?
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May 19, 2009
Alzheimers Disease: amyloid-beta and sticky plaques
I thought it would be fun to do a series on hot new trends in science and medicine based on different mechanisms of action, partly because this is what science is all about and partly because hearing about creative new scientific strategies gives me a sense of awe and wonder.
One idea that hit my mailbox today was a fascinating article on new targets for Alzheimers disease (AD) in SciBX, a Nature publication. For a limited time the leading articles are downloadable free of charge. You can find the link to the Alzheimers article here, which said:
"A study by a team at Genentech Inc proposes a new mechanism for Alzheimers disease pathogenesis by outlining a process that could be an early step in the neurogenerative disorder. The work also identifies a trio of new AD targets, which the company is pursuing with preclinical programs."
According to Genentech's website:
"Anti-Abeta is a humanized monoclonal antibody, which binds to amyloid beta (Abeta). Abeta is the main constituent of amyloid plaque in the brains of patients with Alzheimer's disease and is proposed to be causative in the development of the disease."
It has long been observed that Alzheimer's patients develop a build up of amyloid plaques and neurofibrillary tangles made of misfolded proteins in their brain. The plaques are abnormal accumulations of sticky amyloid-beta
protein between the neurons. This situation arises when the
amyloid precursor protein is incorrectly cut into pieces. The tangles exist as a filamentous collection of twisted and hyperphosphorylated tau found in the cell body of a neuron.
The NIH has a beautiful illustration of the difference between a healthy and diseased neuron as well as other images on its website that are free to download:
A number of drugs have tried to slow the progression of the disease, with limited success. Once the plaques and tangles have formed, it is difficult to effect any change in the disease.
Earlier this year Yale researchers reported that cellular proteins called prions activate the process by which amyloid-beta peptides impair brain function in people with the disease. Cellular prion proteins are usually harmless and exist in all cells, but they can change shape and cause disease. Essentially, in Alzheimer's, they appear to start the cascade that makes the neurons sick.
These concepts raise several questions - can the amyloid plaques be stopped or slowed down from developing or can they be eliminated or reversed in some way.
The study by the Genentech team, which was reported in Nature, suggests a new mechanism for the pathogenesis of Alzheimer's disease by outlining a process that could be an early step in the neurodegenerative disorder.
The work also identifies a trio of new AD targets, which the company is pursuing with preclinical programs. The study suggests that the previously ignored amino-terminal portion of amyloid precursor protein (APP), called N-APP, might be the main culprit behind AD. APP is a transmembrane protein that gives rise to N-APP and the much more commonly known amyloid (A), a fragment that forms the amyloid fibres and plaques that are hallmarks of AD, although the precise role of these in the disease pathology is unclear. A schematic is shown below, from the Nature article:
Of course, none of this new research is going to help those of us who are struggling with parents with AD, but perhaps by the time we are seniors there will be some improvements in available therapies and earlier detection of this devastating disease. There is some justification for that raised hope because a newly discovered mutation, the A673V mutation, is associated with familial AD. The
inheritance pattern is recessive, meaning a patient needs two mutant
alleles in order to acquire the disease risk. This is unusual because most AD is sporadic and not inherited.
Regular readers of this blog will be familiar with wild type and mutant mutations seen in cancer conferring different prognosis. It seems that they also appear in AD. Interestingly, in combination with wild type allele, A673V doesn’t cause AD. The presence of mutant protein prevents the wild type protein from forming amyloid fibrils, even under very favorable in vitro conditions.
As we learn more about the biology of Alzheimers Disease, hopefully new and better treatments will be developed. Perhaps we might even understand one day what actually causes or triggers the disease to begin it long slow process in the first place. Now that would be something.
Osherovich, L. (2009). Genentech's new parADigm Science-Business eXchange, 2 (8), 1-5 DOI: 10.1038/scibx.2009.300
Di Fede, G., Catania, M., Morbin, M., Rossi, G., Suardi, S., Mazzoleni, G., Merlin, M., Giovagnoli, A., Prioni, S., Erbetta, A., Falcone, C., Gobbi, M., Colombo, L., Bastone, A., Beeg, M., Manzoni, C., Francescucci, B., Spagnoli, A., Cantu, L., Del Favero, E., Levy, E., Salmona, M., & Tagliavini, F. (2009). A Recessive Mutation in the APP Gene with Dominant-Negative Effect on Amyloidogenesis Science, 323 (5920), 1473-1477 DOI: 10.1126/science.1168979
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What we do
Icarus Consultants is a management consulting company focusing on marketing strategy and new product development to the Pharma and Biotech industry. We focus on specialist areas such as oncology, hematology, immunology, respiratory and HIV.
Our particular areas of interest include social media monitoring, key opinion leader research, competitive intelligence and landscape opportunity assessments. We use primary qualitative market research and buzz metrics to support our strategic analyses.
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