A word about the gene-editing technology, called Crispr-Cas9 – WSJ

DNA_strand

There has been a lot of buzz going around about the Crispr-Cas9 gene-editing technology

But what it is? and what impact could it have on drug development, innovation and new treatments?

What it is?

A fascinating short video created by the McGovern Institute for Brain Research at MIT explains the key characteristics of the technology.

Why is it so exciting?

A Wall Street Journal article reveals what this technology could allow regarding the development of potential new treatments and why it is so exciting.

This new technology will enable the “editing” of defective genes and their replacement by healthy (or normal) genes. Pharmaceutical companies are just starting to explore how to use it as drug discovery would probably be revolutionized. Diseases like cancer, cystic fibrosis,… caused by a mutated gene will eventually find a solution.

Another key advantage is the ease of use. Unless other technologies, Crispr-Cas9 gene-editing technology is much easier to handle than prior methods.

Is it already used?

Yes, it is selected to create genetically engineered research animals for the time being but there is more to come…

Is it dangerous?

It could be, if it is not used correctly and ethically. In April 2015, Chinese scientists used this method to manipulate the DNA of human embryos. This raised fears that it could be used to tune babies up before birth. Manipulating life has to be ethically monitored in order to protect life.

In another article that was then retracted, it seems that Crispr technology caused nearly 2,000 unexpected mutations — 10 times what was previously observed (in two mice that it cured of blindness). It is not the “death” of the technology but it proved that we don’t know yet how it really works.

Studies, published in Nature Medicine, revealed that edited cells might cause cancer.

What are the technical hurdles to overcome before creating a drug treatment based on this technology?

Today we do not know how to deliver a drug that could target the right DNA in the right cells. Inactivated viruses or nanoparticles could lead be used as potential carriers.

What’s next?

Overcoming drug delivery hurdles, perfecting the technology, translating it into drugs able to cure genetic disorders will be a fantastic improvement in health sciences. It will bring hope to many patients suffering from these diseases without any cure today.

As the technology is very promising, scientists and universities are currently fighting on the legal battlefield to know who will be the owner of the patents and who will make money from the technology and its application. More details

Additional Articles

As it moves out of the lab, CRISPR brings tough questions into the clinic – STAT – October 2018

A flawed study shows how little we understand CRISPR’s effects – Wired – April 2018

Biotechnology: At the heart of gene edits in human embryos – Nature – August 2017

Redesigning Life: How Genome Editing Will Transform the World – London School of Economics Book Review – September 2016

Beyond CRISPR: A guide to the many other ways to edit a genome – Nature – August 2016

Do CRISPR enthusiasts have their head in the sand about the safety of gene editing? – STAT – July 2016

Federal panel approves first test of CRISPR editing in humans – Washington Post – June 2016

Riding The Gene Editing Wave: Reflections On CRISPR/Cas9’s Impressive Trajectory – LifeSciVC – May 2016

Scientists solve CRISPR’s “Energizer bunny” problem – STAT – April 2016

CRISP biotech Intellia strikes licensing deal with Regeneron, readies for IPO – BioPharmaDive – April 2016

Bill Gates, together with other well-known investors, recently invested in Editas Medicine as part of CRISP-Cas9 frenzy.

Medical specialists urge more debate on gene-editing technology – Reuters – September 2015

CRISPR player Intellia looks IPO-ready after $70M round – FierceBiotech – September 2015

Editing humanity – The Economist – August 2015

Eight Things You Might Not Have Known Until Editas Filed Its S-1 – Xconomy – January 2016

Historic CRISPR Patent Fight Primed To Become Head-To-Head Battle – Xconomy – January 2016

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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

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Scientists want to make sure medical breakthroughs aren’t just for white men

Precision medicine could reduce the gender gap in clinical trials and genetic databases. This will lead to better care and improved outcomes for women and minorities.

The Rise of the Empowered Health Care Consumer – Deloitte Dbriefs Health Sciences Series

Deloitte-Debrief-EmpoweredConsumer-June2015

An excellent webcast took place yesterday.

The key points are above on the MindMap I did. The presentation is available here: Deloitte_Dbriefs_Empowered_Healthcare_Consumer_Jun2015

The main take-home message: companies really should include the new health care consumer as a stakeholder. The patient has now more power than ever. He/she is more informed, more connected, more commited but also more demanding.

2015 Design of the Year – Human Organ on Microchip – Financial Times

Innovation

Accelerating the path to market for drugs is the dream of many scientists. Reality is closer than you think…

A Financial Times article (subscription required) highlights this breathtaking innovation that could revolutionize the life sciences world.

“These new organs on chips will enable scientists and researchers to mimic responses to drugs and treatments in human tissue without recourse to animal or human testing”.

It will enable researchers and pharmaceutical companies to spend less time and money in animal models as well as in human clinical trials. It will also accelerate the development and allow patients to be provided with the needed drugs sooner than ever before.

Human organ on a microchip has won the 2015 Design Museum’s Design of the Year contest. Thought and developed by a cross-disciplinary team of researchers led by D. Ingber and D. Dongeun Huh at Harvard University’s Wyss Institute, this chip is able to mimick several different types of organs. By using it, it will also be possible to see whether a patient would respond or not to treatments. Personalized medicine will be more precise and more targeted. Additionally, multiple chips can be connected to play the role of the human body.

Have a overview of the technology in their 2-minute video presentation:

 

Go deeper into this amazing technology and its potential application with the TEDx Boston talk by Geraldine Hamilton:

 

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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

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Doctor On Demand Pulls In $50 Million To Continue Expansion Of Its Virtual Doctor Visit Platform

Telemedicine is on the rise. It could be extremely useful for remote areas as well as disabled people unable to visit the GP office. Availability 24/7 is also reassuring for chronic disease patients.
This is clearly one of the main trend in the healthcare industry.

Healthcare – From volume to value – NEJM

stethoscope on open book on a white background

Behavioral economics and physician compensation…

… is the title of an article published last week in the New England Journal of Medicine.

Switching from fee-for-service to value-based payment or pay-for-performance will change the behavior of doctors in prescribing tests, procedures and drugs. As mentioned in the article, “fee-for-service reimbursement tends to promote well-compensated procedures.” Value-based payment will focus on patient outcomes and will drive a change in decision-making. Incentives will be different and drive the behavior of doctors, but incentives are strongly needed to help transition from one system to the other.

Behavioral economics views incentives as fundamental determinants of behavior, but it can help elucidate how the timing, frequency, and amount of payment influences behavior; how to address unintended consequences of incentives; and how to create environments supporting better decision-making.”

Deviations from rational decisions are often triggered by context and benchmarks. Several measures can be implemented to go over these deviations, the article highlights some of them:

– the power of default. By default, when prescribing a drug, the information system shows the generic instead of the brand;

– the loss aversion. Giving payments in advance of mandatory behavior (hand hygiene, electronic prescribing,…) pushes doctors to adhere to guidelines in order not to lose the money won;

– the incentives series. Giving payments in small and fragmented quantities is more efficacious than in larger amounts because immediate feedback is needed to influence behavior;

– the positive self-image. “Pairing performance incentives with appeals to self-image and professional identity provides an additional lever for meeting quality and efficiency goals. Making public some component of physicians’performance, at least within organizations, may enhance the effect of monetary incentives.”

The conclusion speaks for itself: “Not all clinical decisions will or should be amenable to interventions based on behavioral economics insights, but for some transformations in health care delivery and payment, such insights may be a powerful force for change. One challenge is identifying the clinicians, patients, and system inefficiencies for which incentive payments can be most helpful.”

 

Additional resources

Should the U.S. Move Away From Fee-for-Service Medicine? – 2015 – Wall Street Journal

The Behavioral Economics Guide – 2014 – A. Samson

 

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Economic crisis, health systems and health in Europe – WHO European Observatory

austerity

How did European Countries cope with the economic crisis and its impact on their national health system?

The last report published by the WHO European Observatory on Health and Health Systems on the impact of the economic crisis on health and health systems allows to take a step back and look at the deterioration of health care in the context of an economic crisis over the last few years in several European countries. It is a desk reference where it is easy to pick the country or the countries that are of interest. Austerity measures have a huge impact on the quality as well as quantity of care in specific countries. Too much cost cutting leads to catastrophic situations, like in Greece.

The structure of the report:

– In the 1st part of the report, 9 countries were chosen for in-depth analysis: Belgium, Estonia, France, Greece, Ireland, Latvia, Lithuania, Netherlands and Portugal. These are probably the most impacted by the economic crisis.

– In the 2nd part of the report, all the countries are covered with regards to their response to the crisis. For each country analyzed in this part, economic trends are evaluated as well as policy responses. Changes are then detailed by category: adjustments to public funding for the health system (budget cut, reforms, subsidies, insurance premiums, cost-containment measures,…), variations to health coverage (population, benefits, user charges,…), revisions to health service planning, purchasing and delivery (price of medical goods, salaries and motivation of health sector workers, payment to providers, overhead costs, provider infrastructure and capital investment, priority setting to change access to treatments, waiting times, prevention,…).

 

Additional resources:

Health and Financial Crisis Monitor – Always up to date

Systematic Review on Health Resilience to Economic Crises – 2015 – PLOSOne

Health, Austerity and Economic Crisis – 2014 – OECD Health Working Paper n. 76

European economic crisis and health inequities: research challenges in an uncertain scenario – 2014 – International Journal for Equity in Health

Effects of the economic crisis on health and healthcare in Greece in the literature from 2009 to 2013: A systematic review – 2014 – Health Policy

Impact of Austerity on European Pharmaceutical Policy and Pricing – 2013 – Deloitte Centre for Health Solutions

Financial crisis, austerity, and health in Europe – 2013 – The Lancet

Global Health and the Global Economic Crisis – 2011 – American Journal of Public Health

 

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Medtronic – The Digital Pancreas – The Future of Diabetes Management? – Fierce Medical Devices

pancreas

A truly interesting article of the transformation of Medtronic Diabetes business

2015 could be a year to remember as the start of a new era in improved diabetes management thank to digital tools.

Diabetes management at Medtronic & deals history

It started several years ago but back in May 2001, a transforming event took place: Medtronic bought Minimed as well as an affiliated company for USD 3.8 billion. Looking only at Minimed, Medtronic paid USD 3.28 billion for USD 400 million in sales (8.2x 2001 sales). Despite its price, quoted as high by some investors and analysts at that time, the deal rationale was pretty compelling as it allowed Medtronic to enter the diabetes management arena. MiniMed offered a beachhead into the field of technological management of diabetes, one of the fastest-growing chronic diseases in the world and one that affects an estimated several millions of people. The devices produced by MiniMed help patients manage their insulin needs and monitor glucose levels.

Since 2001, several deals and partnerships paved the way up to where Medtronic is today. After having a look at my deals database, I can say that some of them clearly stand out:

November 2004: agreement with Novo Nordisk on prefilled insulin cartridges;

– August 2007: co-promotion & co-marketing with Lifescan and Bayer of blood glucose meters (in USA together with Lifescan and outside USA with Bayer extended in 2011). These devices had wireless data transmission to insulin pums;

May 2009: strategic marketing collaboration with Eli Lilly on patient education and disease management;

June 2009: acquisition of PreciSense, a medical device company developing CGM (continuous glucose monitoring) technology. A step forward for “closed loop” systems dedicated to insulin delivery;

January 2013: research collaboration with GI Dynamics on EndoBarrier;

August 2013: acquisition of Cardiocom, a developer and provider of integrated telehealth and patient services for the management of chronic diseases;

June 2014: global strategic alliance with Sanofi, aimed at improving patient experience and outcomes for people with diabetes around the world. The priority is the development of drug-device combinations and delivery of care management services to improve adherence as well as simplify insulin treatment.

Medtronic has built its Diabetes franchise over the last several years and is still fully committed to be the leader in this field. This pledge could lead to the achievement of the digital pancreas, a fully autonomous device (closed loop system) monitoring blood glucose continuously and adjusting insulin doses as perfectly as the biological pancreas without human interaction. Some researchers already have prototypes, studies are ongoing, universities and hospitals are teaming up. The field is really at its boiling point!

Deals and partnerships in 2015

In this FierceMedicalDevices article, we can see Medtronic advancing its franchise by investing with determination and dedication in new technologies.

Many partnerships have been signed in 2015: Diabeter (a diabetes clinic and research center dedicated to providing comprehensive and individualized care for children and young adults with diabetes), DreaMed (artificial pancreas technology for integration into future Medtronic insulin pumps). Beyond healthcare companies, Medtronic is also expanding its network into consumer electronics with Samsung (integration of mobile and wearable devices to improve disease management with an Android mobile app) and diabetes data with a startup called Glooko. It also partnered with IBM Watson Health for next-generation disease management solutions.

All these deals are clearly accelerating innovation at Medtronic but in a patient-centric fashion as in every deal we can see the benefits for them and the management of their disease. What could be the next step? A deal with Apple in order to nearly fully cover all the mobile OS in the world.

For my conclusion I will quote Medtronic CFO, Gary Ellis: “We’re focused on transforming our diabetes group from a market-leading pump and sensor company into a holistic diabetes management company focused on making a real difference in outcomes and costs,” summed up Medtronic CFO Gary Ellis on the most recent quarterly conference call.

 

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The Vaccine for Type-1 Diabetes Is Moving Forward

Fascinating use of the BCG vaccine – it could bring hope for Type 1 diabetes patients in reversing their disease. Looking forward to seeing the results in 5 years! A story to follow!

TIME

A promising vaccine that has the potential to reverse the symptoms of type I diabetes—an autoimmune disease often diagnosed in childhood—is heading on to a phase II trial, which will test the vaccine on humans with the chronic disease.

The vaccine, called bacillus Calmette-Guérin (BCG) has succeed in reversing type 1 diabetes in a trial among mice and in a phase I trial in 103 humans. The new trial, which the researchers announced on Sunday at the Scientific Sessions of the American Diabetes Association, will last for five years and will test the effect of the vaccine on people with type 1 diabetes among adults between ages 18 to 60. The vaccine may be able to improve the disease in people who have small but detectable levels of insulin coming from their pancreas. Lead researcher Dr. Denise Faustman, director of immunobiology at Massachusetts General Hospital (MGH), estimates that about one…

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Hacking the Human OS – IEEE

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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.

 

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8 emerging technologies that could revolutionize the life sciences – Medium

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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.

 

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Immunology or Metabolism – What can we say about Type 1 Diabetes?

Diabetes

Not always easy to categorize this disorder. What is being said today? An overview of opinions around the litterature

I conducted a small litterature search with the keywords “Type 1 Diabetes”, “Immunology”, “Metabolism”, “Metabolic”. PubMed returned 534 results. Google Search returned over 5 million…

I will start with some definition as well as resources and then try to answer the question.

 

Definition

Diabetes is not a single disease.

An excellent starting point is the Diabetes Journal Article: Diagnosis and Classification of Diabetes Mellitus. It says that “Diabetes mellitus is a group of metabolic diseases characterized by hyperglycemia resulting from defects in insulin secretion, insulin action, or both. The chronic hyperglycemia of diabetes is associated with long-term damage, dysfunction, and failure of various organs, especially the eyes, kidneys, nerves, heart, and blood vessels. Several pathogenic processes are involved in the development of diabetes. These range from autoimmune destruction of the β-cells of the pancreas with consequent insulin deficiency to abnormalities that result in resistance to insulin action.” We have a good point here saying its origins are coming from an immune system dysfunction.

The American Diabetes Association provides in-depth overview of the symptoms, diagnosis, Type 1 and Type 2 as well as gestational. The website is full of stats and resources.

The CDC also contributes to knowledge and awareness with this comprehensive infographics. The IDF – International Diabetes Federation – is another source of information together with the WHO dedicated page on diabetes and the European Foundation for the Study of Diabètes.

CDC_diabetes-infographic

 

Some possible answers – Immunology vs. Metabolism

Coming back to Type 1 diabetes and my quick litterature search. From the 534 articles, I selected 5 among them seeming more relevant to start answering the question.

One of the first article I read is clearly stating that “Type 1 diabetes mellitus (T1DM) is a T cell-mediated autoimmune disease characterized by the destruction of pancreatic β cells”. But authors of another article insist on the metabolic abnormalities in Type 1 diabetes: “Clinical onset of type 1 diabetes (T1D) is thought to result from a combination of overt beta cell loss and beta cell dysfunction. However, our understanding of how beta cell metabolic abnormalities arise during the pathogenesis of disease remains incomplete. Despite extensive research on the autoimmune nature of T1D, questions remain regarding the time frame and nature of beta cell destruction and dysfunction.” “Determining the time frame of beta cell destruction and identifying metabolic mechanisms that drive beta cell dysfunction has high potential for successful interventions to maintain insulin secretion for individuals with established T1D as well as those with prediabetes.”

A study has been done to see whether any genetic link could justify the apparition and severity of T1D in children by looking at autoimmune diseases in the extended family. The short conclusion is that approximately 30% of children with newly diagnosed T1D have a 1st and or 2nd-degree relative affected by an autoimmune disease like autoimmune thyroiditis, celiac disease, Addison’s disease, pernicious anemia, rheumatoid arthritis and multiple sclerosis. Moreover, between 9 and 19% of the children with T1D have another autoimmune disease.

An interesting point is made about the influence of the microbiome both on metabolism and immune system. For the time being only mice have been used but the results have good chances to be extrapolable to humans. “Emerging evidence suggests that both host metabolism and immune function is crucially regulated by the intestinal microbiome. Recently, we showed that in the non-obese diabetic (NOD) mouse model of Type 1 Diabetes (T1D), the gut commensal microbial community strongly impacts the pronounced sex bias in T1D risk by controlling serum testosterone and metabolic phenotypes”.

Additionally, LADA (Latent Autoimmune Diabetes of the Adult), Type 1 diabetes diagnosed during adulthood seems to have a mix of genetic characteristics from childhood-onset Type 1 diabetes and Type 2 diabetes. “Metabolic changes at diagnosis reflect a broad clinical phenotype ranging from diabetic ketoacidosis to mild non-insulin-requiring diabetes”, as said in the article. In this case, we probably have a mix of immunological and metabolic changes leading to the disease.

I’m not sure whether one day we will be able to really discover the autoimmune or metabolic origins of T1D. It is probably a mix of both as complex disruptions and dysfunctions in the human body are leading to T1D onset.

 

Selected articles used

Latent autoimmune diabetes of the adult: current knowledge and uncertainty – 2015

Alteration of regulatory T cells in type 1 diabetes mellitus: a comprehensive review – 2015

Metabolic abnormalities in the pathogenesis of type 1 diabetes – 2014

Microbiome manipulation modifies sex-specific risk for autoimmunity – 2014

Extended family history of autoimmune diseases and phenotype and genotype of children with newly diagnosed type 1 diabetes – 2013

 

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