Resumption after an extended hiatus

Things have been really insanely busy on the work front over the last year, necessitating some radical prioritisation and thus I have only posted on Pharma Strategy Blog instead of here and Oncology Market Trends.

It will be changing from this week as things gear up for some interesting new perspectives on the Pharma CI front and I'd like to take the time to thank everyone for their patience during the hiatus.

Normal service will resume shortly.

Trick or treat? When new therapies stretch the price barrier

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that affects around 10,000 people globally. The red blood cells in PNH patients are weak and are destroyed more rapidly than normal. This causes the urine to turn red or dark during an episode (or paroxysm) of red cell destruction (or hemolysis).

Usually the urine is darker first thing in the morning (nocturnal) and clears through the day. Each episode of dark urine usually lasts for a few days and episodes may occur very occasionally or very often. During an attack of dark urine many patients have mild abdominal discomfort. However, some patients with PNH never have attacks of dark urine, so the condition can be erratic.

The standard treatment for PNH was blood transfusions every three months to stave off chronic tiredness, jaundice and difficulty swallowing. Alexion Pharmaceuticals has developed a new treatment for PNH called Soliris (eculizumab), an infusion therapy given every couple of weeks that reduces the need for dependence on blood transfusions. Eculizumab is a humanised monoclonal antibody that inhibits terminal complement activation.

The catch?

The treatment costs approx. $400K per year, something that has raised a lot of concern, as you can see from reports here, here and here.

It's hard to justify such a high price for a therapy when the top end cost for drugs that target around 10,000 patients globally is under $300K a year; even some UK PCT's thought it was not cost effective in the absence of NICE review, as reported recently on BBC News.

Thought for the Day

"The aim of marketing is to know and understand the customer so well the product or service fits him and sells itself."

Peter Drucker


This aphorism is so obvious and true, yet it is surprising how few companies can manage this simple task.

The best leukemia drugs out there not only work, but also fill a deep seated need that hematologists were all craving for - something that truly impacts the quality of their patients lives and makes everyone smile.

Cancer need not be about death and dying, sometimes therapies make a real difference too. The art of marketing in this context is about listening to the customers real needs and lining up what the product can do with those desires. Then you have a winning product and happy customers.

Related articles by Zemanta

• Understanding why customers don't buy is crucial
• Attention, science and money

CML - Gleevec or a take a chance on a transplant?

“The brick walls are not there to keep us out. The brick walls are there to give us a chance to show how badly we want something. Because the brick walls are there to stop the people who don’t want it badly enough. They’re there to stop the other people.”

Randy Pausch

And so it goes for cancer patients, especially those facing a transplant, which can wipe out 20% of patients from infections and treatment related mortality alone. New improved treatments over the years have increased overall survival, but not so much the severity of the regimens.

Which led me to wonder what will happen for patients with chronic myeloid/myelogenous leukemia (CML) patients who have done well on tyrosine kinase inhibitors such as Gleevec, Sprycel or Tasigna. The goal of treatment over the last few years has clearly been to achieve at least a major and possibly even a complete cytogenetic response. Compared to a stem cell transplant though, few have achieved a molecular remission, still less maintained it. These patients are in a twilight zone of chronic stable therapy, living largely a normal life, but unlikely to be cured. Stopping vital therapy may lead to relapse rather than the hoped for remission.

And so that brought me to wonder if some of these patients with a good performance status and under 50 years of age might benefit from a transplant given their cancer burden and hematologic status would be much improved over when they were first diagnosed. The chances of surviving the transplant would probably be much improved. Perhaps it is time to consider this idea, after all, the more patients cured from cancer the better.

A transplant wouldn't be suitable for everyone, but for some CML patients, it could be a life saver.

What would you do if it was you or a family member?

Related articles by Zemanta

• New iPhone apps and Protein Drug Database
• Living with Cancer

In remembrance

To all those who lost their lives on 9/11, there will be no blog posts today.

We will remember them.

Second-line treatment in CML

MD Anderson Cancer Center recently published a new article in Blood looking at second-line treatment with tyrosine kinase therapies with nilotinib or dasatinib after imatinib failure. This is a crucial question - how long should patients receive their new therapy before considering alternative treatments?

The results were clear that after 12 months of second-line therapy, patients achieving a major cytogenetic response (12MMCyR) had a significant survival advantage over patients in minor cytogenetic response or complete hematologic response, with a projected one-year survival of 97% and 84% respectively (P = .02).

The early cytogenetic response was strongly predictive of achievement of 12MMCyR, with less than 10% of patients showing no cytogenetic response at 3 to 6 months eventually attaining the target of 12MMCyR.

The researchers concluded that patients receiving second-line therapy who did not experience a cytogenetic response at 3 to 6 months should be therefore be considered for alternative therapies, ie patients on nilotinib could switch to dasatinib and vice versa.

Forgotten side of the Iraq war: shortage of meds for cancer patients

This interesting (and sad) letter in the New England Journal of Medicine highlights the shortage of chemotherapy treatments for children suffering from leukemia:

"There was a significant inverse relationship between the amount of prescribed chemotherapy that was administered and the risk of relapse."

The study reinforced the notion that the most important factor in improving survival, even in children with leukemia, is adequate treatment.

Cancer: tracking progress for the treatment of CML

This fascinating short video interviews some of the top leukemia doctors around the world and looks at how tracking faulty chromosomes is now routine for people with chronic myeloid leukemia. Experts such as Dr Brian Druker and Prof. John Goldman explain what sophisticated laboratory tests can tell about treatment success:

Video

Precise location of an oncogene may determine the onset of childhood leukemia

The white blood cells in our body combat foreign intruders, such as viruses and bacteria. In leukemia, the formation of white blood cells goes haywire and the cells that should develop into white blood cells multiply out of control without fully maturing. This process disrupts the production of normal blood cells, making patients more susceptible to infections. T-ALL, a particular form of leukemia, is the most prevalent cancer in children under 14 years of age and occurs predominantly between the ages of two and three.

With intensive treatment involving chemotherapy, over half of children are cured. But scientists hope to be able to develop targeted therapies that are less toxic than chemotherapy, based on knowledge of the biological processes behind T-ALL.

Oncogenes are often at the root of cancer. Scientists around the world are theefore concentrating on identifying oncogenes and their related proteins. Recent research by the Flanders institute of Technology in Belgium (VIB-K.U.Leuven) indicates that the location in the cell where these proteins are found plays an important role in the entire carcinogenic mechanism.

In collaboration with the Nederlands Kanker Instituut, Amsterdam and Harvard Medical School, Boston, the VIB researchers have demonstrated that NUP214-ABL1, a fusion of two proteins, is carcinogenic only when it is in a protein complex near the nucleus of the cell. Located at another place in the cell, NUP214-ABL1 does not lead to cancer. This finding sheds new light on the study of carcinogenic processes.

Many forms of cancer are caused by genetic defects in which a certain kinase becomes too active and this is the case with NUP214-ABL1. The most obvious solution is to make the carcinogenic kinase inactive, and so kinase inhibitors are usually used to combat these kinds of cancers. However, the carcinogenic kinase often becomes resistant to these inhibitors, which is certainly true for T-ALL. new approaches are therefore being actively sought.

The recent research results now offer a possibility. It has been shown in cells that NUP214-ABL1 is no longer carcinogenic when it cannot bind with the protein complex in the vicinity of the cell nucleus. On the basis of these results, the researchers want to further investigate the therapeutic possibilities of compounds that render binding between the complex and NUP214-ABL1 impossible. This study also indicates that the location of proteins can play an important role in other forms of cancer/leukemia as well.

Stand Up to Cancer!

Stand Up To Cancer, a new initiative to raise philanthropic dollars for accelerating ground-breaking research, launched recently through a large collaboration uniting the major television networks, entertainment industry executives, celebrities and prominent leaders in cancer research and patient advocacy.

ABC, CBS and NBC will donate one hour of simultaneous commercial-free prime time for a nationally televised fund raising event to air on September 5, 2008 (8 pm EDT and PDT), aimed at rallying the public around the goal of ending cancer's reign as a leading cause of death.

In addition to the nationally televised network fundraising event, other key elements of the initiative include:

Standup2cancer.org

With both interactive applications and rich content, the SU2C website will foster an online community for everyone affected by cancer, utilizing the same approach as the televised special: it will move, educate and even entertain users. Features include: The Constellation: For a dollar donation or more, users can launch a star in honor of anyone who has received a cancer diagnosis.

The Stand: An interactive Facebook application to illustrate that the ‘cancer community' encompasses everyone and that we are all connected by this disease. SUTV: Features video segments rich in scientific and research information, as well as ones that confront the personal and human side of cancer's impact. SU2C Magazine: Offers seven sections of diverse content written by leading voices in every field.

Public Service Announcement (PSA) Campaign

A series of TV, radio and print PSAs featuring celebrities and members of the general public to mobilize support for the campaign will air and appear in publications.