Advances in Oncology Drug Discovery – Sachs Conference Talk by Roche pRED Head of Oncology DTA, William Pao

9762765983_26a72a44fd_b

A fascinating talk about cancer drug discovery was given by Dr. William Pao, Head of Oncology Discovery and Translational Area (DTA) at Roche Pharmaceutical Research and Early Development (pRED) during the Sachs 16th Annual Biotech in Europe Forum.

He started to explain what is cancer, for us to gain more insights:

  • cancer is a genetic disease: tumors can harbor over 400 somatic mutations
  • cancer is heterogenous: there are more than 200 types of cancers and a single patient tumor displays intra and inter-tumoral heterogeneity
  • cancer can metastasize: once spread, it is virually incurable. Metastatic cancer survival at 5 years is extremely low (between 4 and 28%)

Based on those considerations, treatment is becoming much more complex today with a blend of chemotherapy, targeted medicines and immunotherapies. A right combination could extend survival by several months.

But how to develop drugs with increased efficacy against the smart strategies used by the disease (such as tumor angiogenesis)? According to Dr. Pao, 3 elements are necessary:

  • understanding disease biology as well as druggable targets in the complexity of cancer molecular pathways
  • developing fit-for-purpose molecules allowing to create the right drug with the right format against the right target
  • personalizing healthcare with the administration of the right drug to the right patient at the right time

Beyond a better understanding of the disease, using more than a single strategy to target the cancer:

  • Host directed with cancer immunotherapy. This approach is particularly challenging as some patients do not respond to it (innate or acquired immune escape) and other patients may fully benefit with long term survival
  • Tumor directed with targeted medicines

External innovation, collaborations and partnerships, is fully leveraged in order for Roche to complement existing capabilities in the field (immunotherapy examples: CuraDev, Pieris, BluePrint; targeted medicines: Tensha, C4Therapeutics).

As a conclusion, Roche is well positioned to address the cancer challenges and, since the beginning of innovative cancer treatments, the company has always been perceived as the leader of the therapeutic area.

addressingcancerchallenge

Missing points in his talk were considerations of patient’s quality of life (extending life does not always go with good quality of life because of treatment’s side effects) and drug pricing (adding more and more drugs to the treatment cocktail costs a lot of financial resources, not only paid by the health insurance but also by the patient).

 

Image source

DNA methyltransferase 1 has a role in the establishment and regulation of tissue-specific patterns of methylated cytosine residues. Aberrant methylation patterns are associated with certain human tumors. www.enzymlogic.com. Work done with the molecular visualization VMD program developed at the University of Illinois: www.ks.uiuc.edu/Research/vmd/

The future of medicine? Cells not pills

Siddhartha Mukherjee is a visionary cancer doctor and the writer of an awesome book about cancer published few years ago.

In the TED talk below, he takes us through a fantastic journey about the future of medicine and how it could change soon.

Medicine started with simple principles, such as have disease, take pill and kill something. It worked and is still functioning as of today for specific diseases. However, the future is elsewhere.

The natural world gives us some clues about how one might think about illness. In fact, the natural world is organized hierarchically upwards, not downwards. It begins with a cell that give rise to self-regulating, semi-autonomous units called organs, and these organs coalesce to form humans, and these organisms ultimately live in environments.

Siddhartha Mukherjee takes the example of cancer. To tackle this disease, we started to kill cells with chemotherapeutic agents and targeted therapies, then we realized that it would be a nice idea to harvest the immune system in the war against cancer. This gave birth to immuno-oncology drugs bringing extraordinary outcomes to patients. The next step is to change the environment but it is much more challenging…

Stem cells are also part of the solution according to Siddhartha Mukherjee. However, it raises tons of questions… as what’s at stake is not killing something, but growing something. This means that we need to think upside-down and shift our views as well as our traditional thinking.

  • Could your medicine be a cell and not a pill?
  • How would we grow these cells?
  • What we would we do to stop the malignant growth of these cells? Could we implant suicide genes into these cells to stop them from growing?
  • Could your medicine be an organ that’s created outside the body and then implanted into the body? Could that stop some of the degeneration? What if the organ needed to have memory?
  • Could your medicine be an environment? In every culture, shamans have been using environments as medicines. Could we imagine that for our future?

It is very often said that the reason we haven’t had the transformative impact on the treatment of illness is because we don’t have powerful-enough drugs, and that’s partly true. But perhaps the real reason is that we don’t have powerful-enough ways of thinking about medicines.

Using “Tumour-Traps” to Monitor The Spread of Breast Cancer

Excellent & insightful article on how it is now possible to track cancer progression.

Snackable Science

Breast cancer is the most common form of cancer in the UK, with approximately 50,000 British women diagnosed with the disease every year. Despite this high occurrence, breast cancer patients have relatively good prospects and almost 80% of sufferers are still alive 10 years after their initial diagnosis. One factor that influences a patient’s chances of survival is the presence or absence of secondary tumours, which occur when cancer cells spread from the breast and establish themselves in other parts of the body.

In research published this month in the journal Nature Communications, a team of scientists led by the University of Michigan’s Dr Lonnie Shea describe an exciting new material, which can be implanted under the skin to capture invasive cancer cells as they move throughout the body. “Tumour traps” like this could be used in the future as part of an early warning system to alert doctors to the…

View original post 497 more words

Scientists are developing an x-ray pill you can swallow for colonoscopy

Less invasive technology is clearly the future. It will allow more cases to be diagnosed and treated early leading to better care and cure.

Can Finance Cure Cancer? Andrew W. Lo Talk at the University of Geneva – June 29th 2015

I had the chance to attend this refreshing talk about what finance could do to help drug development. My summary of the talk is shown below.

CanFinanceCureCancerAWLGenevaJune2015

 

Memorial Sloan Kettering Cancer Center Interactive Drug Calculator – What will it bring?

DrugPrices

A lot of buzz has been around these days about an initiative launched by a well-know cancer center. We take a step back and think about the rationale and the benefits.

The Memorial Sloan Kettering Cancer Center launched an interactive cancer drug calculator comparing the cost of several cancer drugs with a “fair” price based on factors such as the benefits (extended life expectancy for example) and the issues (like the side effects, development costs,…). The calculated price and the official list price do not match in many cases.

The concept

The rationale

Several doctors, especially oncologists, complained and are still angry about the escalating costs of cancer drugs in the US. It is not rare to see price tags around USD 100’000 per patient per year of treatment. It is clearly unsustainable for the whole system and could be a huge issue for patients as they are required to pay a portion of the cost. See more in one of my past blog posts.

The project leader, Dr. Peter Bach, said: “Prices for many new cancer drugs don’t reflect their value to doctors and patients. Right now, manufacturers have total price control, and total control of prices has led to irrational pricing behaviors.” This situation is not easy to manage.

With the Abacus Tool, we can see the official list price and the “abacus” price, that will be determined by different factors chosen by the user.

The benefits

This tool reflects the implementation of value-based pricing, discovered by M. E. Porter. More on this concept by following this link.

As said earlier by Daniel Goldstein, “currently cancer drug prices are not linked to the benefit they provide. They’re priced on what the market can bear, which is an unsustainable system.”

Patricia Danzon agrees and states that “assigning a monetary value to an additional year of life and discounting a drug’s toxicity should be key components of any pricing system.”

Each user will find a different price depending on the value of each factor he/she decides to select. It is a real personalizable tool for each individual confronted to cancer and its financial burden.

Conclusion

Paying for a specific value is true in any industry apart from the pharmaceutical area. Value-based pricing should be put in place to bring benefits to patients as well as to guarantee the reimbursement of life-saving treatments to each person without destabilizing the health system budget. The ASCO initiative is also working on a conceptual framework to assess the value of cancer drugs (see below).

The value-based pricing for drugs will work its way and it is definitely worth it for all the stakeholders!

Additional resources

‘Financial toxicity’ looms as cancer combinations proliferate – Nature Biotechnology (subscription required) – 2015

How Much Should Cancer Drugs Cost? – 2015 – WSJ

Another initiative by ASCO: American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options – 2015 – Journal of Clinical Oncology

Image Source

Addressing the Financial Burden of Cancer Treatment – From Copay to Can’t Pay – JAMA Oncology

anguish

Cancer care is not only about individual health but also financial health

A new article in JAMA Oncology is raising awareness of the disastrous consequences cancer treatment could have on the financial health of patients.

There is substantial evidence that high financial burden could lead to decreased clinical benefits due to poor treatment adherence and deteriorating quality of life. This is a challenge for oncologists as they are looking to provide the best care for each patient. But what happen if she or he cannot afford it?

The authors suggest several measures to improve patients’financial health:

Restructure cost sharing and insurance design. Due to the current US insurance system designed with deductibles, copays and tiering, cancer patients could face extreme financial burden and, consequently poor health outcomes. Deteriorating health is tied to unaffordable treatments involving lack of adherence.

Eliminate low-value prescribing practices. Common high-cost practices that do not improve clinical outcomes should be excluded in order to preserve patients’financial health.

Create tools to evaluate patient risk regarding financial distress. The routine assessment of financial health should be done early and, if necessary, patient could be addressed to dedicated programs to facilitate care access like patient assistance programs offered by charities.

Improve cost transparency. Providing this information as well as out-of-the-pocket cost information are valuable and allow patients to choose healthcare providers to minimize the impact on their personal budget.

Provide financial counseling as part of cancer care. Patients need to understand what impact(s) their cancer diagnosis could have on their private life (employment, future income, family financial security,…). It is crucial in order to avoid financial pressure and improve planning for the patient’s family and relatives.

Express Scripts is also concerned about the price of cancer drugs as shown in a recent article. As a payer, it would like to really focus on value-based reimbursement and implement indication-based formularies. They will allow to better decide on which treatment/drug works best for which patient. Today, several tools are available to improve treatment decision like tumor testing, genetic analysis, predictive analytics,… Integrated care along with better claims management will complement the measures discussed above in order to provide real solutions and benefits for the patients.

Additional resources

1,495 Americans Describe the Financial Reality of Being Really Sick – NYT – 2018

Out-of-Pocket Costs, Financial Distress, and Underinsurance in Cancer Care – JAMA Network – 2017

Cancer patients skipping medicines or delaying treatment due to high drug prices – STAT News – 2017

Financial toxicity: 1 in 3 cancer patients have to turn to friends or family to pay for care – STAT News – 2016

Drug Abacus – Interactive Exploration of Drug Pricing – 2015 – Memorial Sloan Kettering

How Much Should Cancer Drugs Cost? – 2015 – WSJ

New Cancer Drugs are Expensive, but Price Controls are Misguided – 2015 – Forbes

Cost of Cancer Drugs Should Be Part of Treatment Decisions – 2015 – ASCO

Pricing in the Market for Anticancer Drugs – 2015 – Journal of Economic Perspectives

The High Cost of Cancer Drugs and What We Can Do About It – 2012 – Mayo Clinic Proceedings

Image Source

Hacking the Human OS – IEEE

health_keyboard

A mind-blowing report from IEEE about the human body’s operating system

Can we harvest digital health data from trackers and sensors to improve our health and well-being? I summarized below what you will find on the IEEE dedicated web page. I was amazed by the variety and depth of all the innovations presented in these pages. Some of them have certainly the power to be game changers in the health care world. I’m looking forward to seeing all the advances coming to life!

Reading the Code

Several technologies are close to the market and will help us monitor our health. A very good example is the biostamps developed by John Rogers from the University of Illinois. These tiny, stretchable and skin-like sensors are able to send information and data to our smartphone alerting us whether something is happening in our body. More and more complex data could be sensed such as blood oxygen, blood glucose and even muscle weakness or sleep patterns.

Another fascinating example that could change the life of Type 1 diabetes patients is the artificial pancreas. It links “data from an implanted blood-sugar sensor to a computer, which then controls how a pump worn on the hip dribbles insulin under the skin through a pipette. In its fully realized form, the machine would take the patient out of the decision-making loop”. Advanced versions of the system are currently in clinical trials. Continuous monitoring is a huge advance in the field of disease management. It could strongly lighten the daily burden of each patient.

Another field where wearable are very popular: athletes. They are always eager to test the last innovation in the wearables arena. Physiological measurements can be extremely useful to optimize training and rest periods, improve performance and avoid injuries. Sleeves, wristbands, sensors equipped with highest technology can really make a difference in the way we monitor and track performance.

A device rapidly diagnosing any medical condition or disorder… Sounds like science fiction, right? Like in Star Trek… Some of you may recall the tricorder. And guess what? It’s about to become reality thank to a competition launched by Qualcomm. 300 teams registered, 10 finalists which are about to deliver their prototypes very soon. Once the winner has been chosen, real life clinical trials will start and we will know if it’s really working as expected. It’s a huge step forward as it will allow the diagnosis (and maybe the start of a treatment) for a lot of people, not only in US or Europe but also in emerging countries where the lack of medical infrastructure is killing human beings…

 

Analyzing the Code

Technology companies showed their interest in healthcare only recently… It’s welcomed because without technology you cannot do anything with data sets. However, some people are afraid of their data becoming public and being hacked. I think that between these two extreme opinions, we can take the good from both sides and see what this can bring us. “Apple, Google, Microsoft, and Samsung, have all launched e-health initiatives, mostly based around smartphones and wearables. Indeed, the fast-growing health care business would seem a natural next step for the tech giants”. A lot of deals have been signed between pharmaceutical companies and technology firms: Google and Novartis; IBM, Apple, Medtronic & JNJ… These are deals to follow in order to analyze the outcomes. Great initiatives could really emerge and I think we are at the beginning of a new era!

Long term analysis could help us understand in a more detailed way how we get sick, how the disease develop and how we could have anticipated it by looking at biomarkers trends.

The new era of precision medicine is making a big difference for patients. An open-source platform has transformed the way patients are being treated. Surgery is not always the best option in oncology for example and sometimes drug treatment is much more effective. A thorough and careful analysis of all the parameters will help doctors taking the right decision for the right patient at the right time.

Real-time epidemics modelling could have saved lives. Building treatment centers at the right locations, anticipating the spread of the disease (in this case, Ebola) and how to limit the contagion were several of the criteria used to run the model. We will never know what would have happen without such a model but globally we can say that modelling is critical in disease management. Additionally, it is not the use of a single model that will be helpful but the customized and accurate modelling for each and every epidemic, according to its characteristics.

 

Changing the Code

Performing surgical interventions at very small scales is becoming a reality. “Thanks to developments in microfabrication and other areas, researchers are pushing the limits on the size and capabilities of objects small enough to move through the human body”. “With the right design, researchers say, a microrobot—or a swarm of them—could deliver a highly targeted dose of drugs or radioactive seeds, clear a blood clot, perform a tissue biopsy, or even build a scaffold on which new cells could grow”. For the time being, tests have only been run in animals.

A new emerging medical field: electrical therapy. Vagus nerve stimulation has the potential to treat several conditions from migraine to asthma, even immune diseases. Progress is very slow and several failures have made history… but new startups are created and renew the interest in this type of technology.

W like Watson, the digital MD. Watson is based on machine learning: “bringing together computer scientists and clinicians to assemble a reference database, enter case studies, and ask thousands of questions. When the program makes mistakes, it self-adjusts. Researchers also evaluate the answers and manually tweak Watson’s underlying algorithms to generate better output. Here there’s a gulf between medicine as something that can be extrapolated in a straightforward manner from textbooks, journal articles, and clinical guidelines, and the much more complicated challenge of also codifying how a good doctor thinks.” Progress is under way.

 

Image Source

8 emerging technologies that could revolutionize the life sciences – Medium

DNA_1

Google Ventures managing partner’s fascinating view on the next wave of innovations in life sciences

Bill Marris is a key influencer to follow in the life sciences industry, but only on Google+… On Twitter, it’s better to follow Google Ventures. Anyway… There is no need to present him.

He is a visionary for new technologies, new business models and he’s very smart when it comes to spot the next breakthrough innovation in life sciences. In this context, he wrote a captivating article on the 8 emerging technologies that could revolutionize the life sciences.

I propose a summary below but I agree it is a pure pleasure to read the entire article, so do it!

Many of the technologies that will transform healthcare already exist but have not yet been expoited at their full potential. Here is the list:

1. Artificial intelligence in healthcare – Machine learning and computer power will help drive better diagnosis and improved data analysis in order to support medical decision-making.

2. Understanding the brain – Decoding the brain is the key to find a cure for brain diseases as well as spinal cord injuries. With recent discoveries, we are at the start of a new era! Check here too!

3. Reinventing antibiotics – Widespread use as well as misuse are at the origins of the emergence of resistant bacteria. We have to really focus on this emerging threat by limiting the use of antibiotics and promoting research of new molecules. Have a look here for more!

4. Battling cancer – Amazing progress has been done over the last years and it is not finished yet! There is more to come. Additionally a real innovation will also be to make these treatments affordable for every patient.

5. Genetic repair – CRISPR, the gene-editing technique, allows the replacement and/or repair of any genetic mutation. Wow!

6. Understanding the microbiome – The 2.5kg of bacteria living on us are a treasure trove! The microbiome is an organ in itself, responsible for our health and wellbeing as well as our diseases and bad moods…

7. Organ generation – 3D cell culture and 3D bioprinting could be the solution to our ageing bodies… Spare parts…!

8. Stem cells – How to maintain the regenerative power of stem cells into adulthood?

All these fields are promising and could give hope to thousands of people in the world. More technology into healthcare for increased value is unavoidable. Let’s hope health and quality of life won’t become unaffordable.

 

Image Source

Blood: Underappreciated Resource in the Health/Disease? – The Next Element

Bloodanalysis

Very well written and insightful post about the use of blood in medicine. Worth a look!

Image Source

The Next Element

Alternative title: Pitching a VC on Disruption of Blood Testing

You may have seen some recent editorials about the necessary frequency of blood tests for healthy individuals, many of them prompted by a series of tweets from Mark Cuban:

MarkCubanTwitter BloodTests

Although there are certainly potential dangers in expecting any and all test results to be immediately informative and/or actionable, there are benefits in disrupting the status quo of how and when we collect information. A potential parallel is the “wearables” market, which has seeped from the “enthusiasts” (so-called quantified self-ers) to being used in trials of new drugs as well as disease research.

Blood Testing: Cost

One significant area of potential benefit to increased attention is cost. Generally when your physician prescribes a drug, you are asked what pharmacy to send the prescription. When told to get blood tests or imaging services, patients (in my experience) are sent to the affiliated…

View original post 1,446 more words

Global Oncology Trend Report 2015 – IMS Institute for Healthcare Informatics

NonSmallCellCarcinomaOfTheLung

A must-have in oncology market analysis

A new report has been published by IMS Institute for Healthcare Informatics about trends in oncology.

It is really informative (stats, charts, visuals…) and insightful. An outstanding overview of what are the main trends (biomarkers, complexity…), the challenges (financial burden, expenditures…) and the opportunities (improved survival, new approaches…) in oncology today.

Key points

biomarkers are more widely used in treatment selection, especially for tumor types with low survival rates and few treatment options;

– new complexity for doctors, payers and patients;

– rising expenditures dedicated to cancer care linked to earlier diagnosis, longer treatment duration as well as increased effectiveness of drug therapies;

USD 100 billion of cancer expenditures registered for 2014 (CAGR 2009-2014: 6.5%);

– increasing financial burden faced by patients (patient access varies widely by country);

– growth in the use of social media by patients in order to find support to cope with their disease;

rise in 5-year survival rates for major cancers;

– promising new approaches in immuno-oncology.

The report

Global Oncology Trend Report 2015: IIHI_Oncology_Trend_Report_2015

 

Image Source (Non Small Cell Carcinoma of the Lung)

Doctors Are Now Inviting Patients To Help Design Their Own Medical Treatment – Huffington Post

Doctors are now proposing more than one option to cancer patients

CollaborationIt is a great step forward in collaborative thinking for the treatment of patients. Including patients opinion in the decision for their own treatment and quality of life will empower them and allow them to feel they have a voice which is heard.

In many hospitals around the world, oncologists and surgeons simply tell cancer patients what treatments they should have. But in the US, at UC San Francisco, under a formal process called “shared decision making,” doctors and patients are working together to make choices about care.

Each patient is unique and there is no single answer to treat them all with the same combination of drugs and therapies.

Huffington Post article    Collaborative thinking in healthcare

Image Source

How can we address the high price of cancer drugs? – Mayo Clinic

The high price for cancer drugs is not only a burden for patients but also for the whole society

Affordability is one of the key word coming back every time we speak about cancer drugs. This time, oncologists are joining the cancer price revolt. The Mayo Clinic published a 4-minute video really worth watching and an article detailing all the points where oncologists can act upon.

Cancer care is not only the issue for the patient but for all the stakeholders (doctors, healthcare providers, insurances, governments, relatives,…). We all need to team up to find solutions. Mayo Clinic is giving us a starting point.

The Mayo Clinic Article    The Mayo Clinic Proceedings Article

A Faster Way to Try Many Drugs on Many Cancers – NYT

A new way to give hope to patients

doctor_physician_surgeryCancers often tend to be fueled by changes in genes, or mutations, that make cells grow and spread to other parts of the body. There are now an increasing number of drugs that block mutations in cancer genes and can halt a tumor’s growth.

While such an approach has worked in a few isolated cases, those cases cannot reveal whether other patients with the same mutation would have a similar experience.

Now, medical facilities are starting coordinated efforts to find answers. And this spring, a federally funded national program will start to screen tumors in thousands of patients to see which might be attacked by any of at least a dozen new drugs. Those whose tumors have mutations that can be attacked will be given the drugs. The studies of this new method, called basket studies because they lump together different kinds of cancer, are revolutionary, much smaller than the usual studies, and without control groups of patients who for comparison’s sake receive standard treatment.

The NYT article

AbbVie boosts cancer drug pipeline with $21 billion Pharmacyclics deal – Reuters

AbbVie is going for the big home run with the Pharmacyclics deal

jumpAbbVie is to buy Pharmacyclics for about $21 billion, giving it access to what is expected to be one of the world’s top-selling cancer drugs and expanding its reach in the profitable oncology field.

The deal — the latest example of a big drugmaker swooping on a biotech firm to refill its medicine pipeline — confounds expectations that Pharmacyclics would sell out to Johnson & Johnson.

More on the deal

The Value Of Cancer Care In The US Compared To Europe – Health Affairs

More value in Europe…

pillsDespite sharp increases in spending on cancer treatment since 1970 in the United States compared to Western Europe, US cancer mortality rates have decreased only modestly. This has raised questions about the additional value of US cancer care derived from this additional spending. Researchers Samir Soneji and Jae Won Yang calculated the number of US cancer deaths averted, compared to the situation in Western Europe, between 1982 and 2010 for twelve cancer types. They also assessed the value of US cancer care, compared to that in Western Europe, by estimating the ratio of additional spending on cancer to the number of quality-adjusted life-years saved. Read the abstract

Cancer Immunotherapy Companies: Looking Forward and Looking Back – Xconomy

Cancer immunotherapy successes and failures with very good examples: Dendreon and Juno Therapeutics

packs_of_pillsWhat scientific, legal, and business challenges does the immunotherapy companies face? Stewart Lyman looks at some of them: intellectual property concerns, technology issues, huge manufacturing costs, pressures on pricing and reimbursement, obsolescence and limited patient populations. Find out more