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The Gentleman’s Hesitation….& The Invention of Stethoscope

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René Laennec (1781 – 1826) was a thorough gentleman. In retrospect, he’d turn out to be a knight in shining white apron.

In 1816, the young French doctor was worried that he could be getting ”inappropriately close’ to a young patient who had been suffering from chest infection. He would recall later, ”…. I was consulted by a young woman laboring under general symptoms of diseased heart, and in whose case percussion and the application of the hand were of little avail on account of the great degree of fatness.The… method of direct auscultation [was] being rendered inadmissible by the age and sex of the patient…”

Laennec resolved the problem of medical diagnostics and social decency in one shot. He ”rolled a quire of paper into a kind of cylinder and applied one end of it to the region of the heart and the other to my ear ”
Within a few months, he had invented that universal symbol of medical science – the STETHOSCOPE




Author Profile:

for sciwri

Anirban Mitra, Ph.D.

Anirban Mitra did his PhD from the Department of Microbiology and Cell Biology, Indian Institute of Science (IISc), Bengaluru and is now a teacher of biology, based in Kolkata. His interests range from biological evolution to history of science and facets of India’s past.

Blog Design and infographics: Abhinav Dey

Featured Image: Ipsa Jain

Creative Commons License
This work by ClubSciWri is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.

MedNess: Healthcare Business News from the Month of May

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Hello everyone and welcome to MedNess: At the frontier of healthcare news. I am back with the news from healthcare business that had the most impact this month Read below to find out more.


To stay on top of scientific advancements, subscribe to Club SciWri (

Neurotrope’s Alzheimer’s candidate fails to yield statistically significant results

Earlier this month Neurotrope reported results from its Phase 2 Alzheimer’s study of Bryostatin. The trial involving 147 patients with moderate to severe Alzheimer’s met its primary endpoint in patients that completed the full course of treatment. Bryostatin improved outcomes in cognition and ability to handle daily activities but failed to achieve statistical significance over placebo (FierceBiotech).

Neurotrope is hopeful of its candidate and plans to take the study forward.

MedNess: This is yet another Alzheimer’s candidate that failed in Phase 2 testing. The successful treatment of dementia is an enigma, and most drug candidates fail larger phase 2 trials. No new drug has been approved for Alzheimer’s for more than a decade. Neurotrope’s shares fell by 63% (Biospace)

Moderna Therapeutics reports interim Phase 1 results from mRNA based H10N8 flu vaccine

The private biotech company; Moderna Therapeutics reported positive interim results from phase 1 study of mRNA-1440 vaccine against avian H10N8 flu. The complete data and results were published in the journal Molecular Therapy.

31 subjects were enrolled in the Phase 1 study, and 23 received 100 µg of the vaccine. All participants achieved HAI titers suggesting seroprotection against seasonal flu. No response was achieved in the placebo arm (8 subjects). (FiercePharma)

MedNess: Since the company is still private, the only investment that can be made in Moderna Therapeutics is through venture capital funds. The company’s valuation is $3B, and primary sources of funding are grants and private investments (Investopedia and The Motley Fool).

Sanofi against affordable pricing for Zika vaccine

Sanofi Pasteur, the leader in vaccine manufacture, was under fire by US army officials and Senator Bernie Sanders after refusing the US Army’s plea for affordable US price for a Zika virus vaccine. Sanofi boasts of about $43 million US research grant money. Sanders and lawmakers have been requesting the US Army to reconsider its term of negotiations with Sanofi that could provide the latter an exclusive license to make and sell Zika vaccine in the US. The vaccine is being developed with the American taxpayer funds, thus prompting both the US Army and Sanders to request an affordable price for the American population.

The spokesperson from the Army states that the decision to provide Sanofi an exclusive licensing is still under consideration and the final decision will be made in the summer (STAT).

Corbus Pharma’s anabasum denied BTD for systemic sclerosis; enrolls the last patient for mid stage-study dermatomyositis

Corbus Pharma’s anabasum (Resunab) was previously provided Orphan Drug designation for Fast Track review by the FDA for systemic sclerosis. However, this month Corbus Pharmaceuticals was trying to gain Breakthrough Therapy status for the drug. The plea was rejected by the FDA. The BTD designation entails recurrent meetings with senior personnel and a rolling review of the New Drug Application. Meanwhile, in another phase 2 clinical trial where anabasum is being tested for dermatomyositis, the last patient was enrolled

MedNess: The failure to gain BTD designation by the FDA worried the investors, slipping shares down by 12%. However, with the news of last patient enrollment, the shares moved up by 3%.

The Belgian-Dutch Biotech Argenx draws $115M post-IPO filing

Argenx filed for IPO last month to draw cash from American investors; in order to push its lead candidate ARGX-113, an antibody directed against autoimmune disorders myasthenia gravis and primary immune thrombocytopenia, to phase 3 trials. It also proposed moving its lead cancer candidate ARGX-110 through mid phase studies. The biotech was able to rope in $115M, 50% above its target goal of $75M.

The biotech started their phase 2 studies with ARGX-113 earlier this year, and the results are expected in the first quarter of next year. A 30% drop in the IgG antibody level would be considered clinically significant (Fierce Biotech, Market Watch).

Incyte reports positive results from selective IDO1 enzyme inhibitor, epacadostat in two separate combined trials

Incyte reported first set of positive results from ongoing combined trials; ECHO-202 in combination with Merck (epacadostat+ Keytruda) and ECHO-204 in combination with BMS (epacadostat + Opdivo). The full sets of results will be announced at American Society of Clinical Oncology (ASCO) meeting next month.

The ECHO-202 trial is assessing epacadostat (selective IDO1 enzyme inhibitor) in combination with Merck’s Keytruda (anti-PD1 immunotherapy). The efficacy and safety results from the phase I/II trial showed that epacadostat in combination with Keytruda was well-tolerated in the following cohorts: non-small cell lung cancer, renal cell carcinoma, ovarian cancer, triple-negative breast cancer, bladder cancer, and head and neck squamous cell carcinoma.

Alternatively, data from the ECHO-204 trial evaluating the safety and efficacy of epacadostat, in combination with Bristol-Myers Squibb’s PD-1 inhibitor Opdivo showed the combination was well tolerated in melanoma, head and neck squamous cell carcinoma, ovarian cancer, and colorectal cancer (

MedNess: Following the positive results, Incyte’s shares gained 2.3%, rose by 6% by mid-day which rose by 8% towards the end of the day (Thursday, May 18, 2017) with the overall increase of 14.10% by the end of the week (Zacks, Investor Place, The Street, and CNN Money).

About the Author:  

Imit Kaur is a freelance medical writer, editor, and an active science blogger. She pursued her Ph.D. in Pharmaceutics and Pharmaceutical Chemistry from the University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.

Follow Imit on LinkedIn (Imit Kaur) or Twitter (@imit_kaur)



Lantern Pharma: A Game Changing Precision Medicine Initiative

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When the Precision Medicine Initiative was announced by President Obama in 2015, he said: “delivering the right treatments, at the right time, every time, to the right person.” As the next heralded breakthrough in healthcare, simply put it is the ultra-tailored therapy.

Personalization of therapy has always been a practice to a certain extent, however, the combination of technologies to generate patient-derived data, genomic medicine and computer science has added the PRECISION to make it personal.

This time in the Medness Focus edition we will talk about a young pharma company, Lantern Pharma, that could be a game changer in the field of precision medicine. How? Read on to know more!

Lantern Pharma: Personalizing the drug pipeline

Problem to address: Re-purposing of validated, de-risked clinical stage anti-cancer drugs that are discontinued for drug development

Reasons for failure of anti-cancer drugs in pipeline:

  • heterogeneity of cancers,
  • multidrug resistance,
  • inability of one drug to treat some but not all patients


  • Personalized precision drug programs using advanced genomics and artificial intelligence (AI).
  • Repurposing of deserted late-stage clinical drugs that were successful in only a subset of patient population
  • AI- A proprietary machine learning algorithm called Rescue Algorithm for Drug Repurposing (RADR)
  • AI approach Connecting and Analyzing Clinical Trial and Patient Genomics Data to Identify relevant Biomarkers and predict favorable patient responders.

Step 1. Combine Clinical Trial Data with Lab genomic Analysis of Patient Samples

Step 2 a. Data Analysis from Clinical Trails and Patient Genomics 

Step 2 b.  Artificial Intelligence to connect the dots between drug candidates and best responders in a patient population

Step 3. Identify Biomarkers

Step 4. Identify Responders

Drug Pipeline:

  • Tavocept (LP-300) targeting Non-small cell lung cancer
  • Irofulven-1 (LP-100) targeting Hormone-refractory prostate cancer
  • Irofulven-2 (LP-184) targeting ovarian cancer


Opinions of Stakeholders:

“Within the next six years, the precision medicine market is expected to reach nearly $88 billion and has the potential to impact all cancer patients. Drug rescue and repurposing in oncology is a high-growth segment and expected to contribute to as much as 25 to 30 percent of new therapeutic approvals and significantly reduce development costs,” Arun Asaithambi (Co-founder and Director) said. (link)

“Lantern Pharma is leading a wave of innovation in cancer treatment that we believe will bring the best therapies to patients who are most likely to respond,” said Clay Heighten, M.D., founding partner of GPG Ventures. (link)

“We are thrilled to partner with Lantern in order to advance the clinical development of Irofulven, and to advance a new paradigm in cancer drug development by designing and conducting clinical trials around a targeted population of patients identified, via MPI’s diagnostic platform, as the patients most likely to respond to a given drug based on their tumor biology. said Professor Peter Buhl Jensen, M.D., CEO of Oncology Venture and MPI. (link)


“The more clinical and genomic data that is analyzed, the ‘smarter’ the AI becomes, which will lead to additional breakthroughs. We are very excited to assist Lantern in its pursuit to re-invent cancer drug development using advanced AI and genomics. This partnership accelerates the development of therapies at a faster and more efficient rate than ever before,” explains Anand A., Chief Executive Officer of Intuition Systems. (link)

Latest news: “Biotech Startup Lantern Pharma Raises $3.7M to Accelerate Cancer Drug Approval Process”

Who are the other players in the Precision Medicine Start-up market segment and where does Lantern stand?

We compiled a list of similar companies (in USA) who are working in the field of precision medicine and could have matching interests with Lantern (Disclaimer: this is not an exhaustive list and the opinions are based on the information available on the companies’ websites as on 05/05/2017)

Company name Target areas Competition for Lantern?
Syapse Precision medicine data platform for health providers Probably
GenomOncology Translating next generation sequencing data into actionable information Probably
 Centrillion Biosciences Genomics and DNA analyses Partially
4D Healthware Patient engagement software through connected-data approach Partially
Cellular Dynamics International Stem cell technologies for drug development and personalized medicine Partially
NeoDiagnostix Combines advanced diagnostics with therapeutics, enabling clinicians plan treatment Partially
Translational Software Converting genomic data into actionable, relevant intelligence Partially
Advanced Cell Diagnostics Diagnostic tests for personalized medicine Unlikely
Epic Sciences Early cancer detection systems Unlikely
Ignyta New drug discovery efforts in oncology Unlikely
Eve Biomedical Low -cost gene sequencing equipment for medical applications Unlikely
Sctheranostics Drug Discovery efforts on Patient derived cells in cardiology Unlikely
Kuraoncology New drug discovery efforts in oncology Unlikely
Genoptix Specialized oncology diagnostic services Unlikely
Precipio Diagnostics Cancer diagnostics reference laboratory Unlikely
Population Diagnostics Develop personalized DNA-based diagnostics Unlikely
Blueprint Health Startup accelerator, and crowdsources genetic and clinical datasets Unlikely
Rosetta Genomics Discovery, the development and commercialization of diagnostic testing Unlikely
Everist Health A machine learning technique producing mathematical models for cardiac patients Unlikely
Microarrays Array-based technologies for biological research, detection and diagnostics, Unlikely
PHIGENIX Novel molecular therapeutics which target the immune system against cancer. Unlikely
Genomind Pharmacogenetic laboratory testing for psychiatry Unlikely
Permedly Connects doctors & personalized medicine solutions via a software platform Unlikely

Company website:

Available jobs

Concluding lines

Lantern’s approach will benefit not only those who are determined to be likely responders but also those patients who are deemed non-responders who will be reprieved from the costs, false hopes, and potential side effects of enduring a futile treatment. In addition, a lot of research dollars spent on the drug discovery efforts from the taxpayers’ money can be recovered by re-discovering their use in smaller subsets of patients.



Featured image source: Adam Simpson

GIF source:

Infographics: Created using



Disclaimer: This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Copyright: The contents of Medness is the copyright of PhD Career Support Group for STEM PhDs (A US Non Profit 501(c)3 is an initiative of the alumni of Indian Institute of Science, Bangalore. The primary aim of this group is to buildup NETWORK among scientists, engineers and entrepreneurs.)

MedNess: At the Frontier of Healthcare Business/ March For Science- Special Report

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Cover Design: Ipsa Jain

Hello everyone and welcome to MedNess: At the frontier of healthcare news. The month of April was pretty crucial for Gilead Sciences and Novartis. Read below to find out more.

Also, this issue of MedNess is special as we are covering a report on March for Science from Club SciWri’s “Reporting from the lab” led by Radhika Raheja, Ph.D. Why are we covering March for Science on MedNess? Science affects us; the scientists, science affects our decisions and perspectives..still need more reasons? Check out our section on March for Science.

To stay on top of major scientific advancements, subscribe to ClubSciWri (

Gilead’s NASH candidate clears early proof concept study

Gilead Sciences presented the first clinical trial data for GS-0976, an acetyl–CoA carboxylase inhibitor in patients with non-alcoholic steatohepatitis (NASH) at the 2017 International Liver Congress. The results are very preliminary but hopeful.

Gilead acquired GS-0976 from Nimbus Therapeutics in a billion-dollar deal last year.

This chronic liver disease affects around 15 million Americans. It is manifested by fat deposition in the liver and can cause scarring and liver fibrosis leading to liver failure (FierceBiotech, SeekingAlpha).

The clinical trial consisted of only 10 patients treated with 20mg dose OD for 12-weeks. GS-0976 blocked the formation of new fat in the liver by 29% and reduced liver fat by 43%. There was also a statistically significant decline in liver stiffness, marker for liver fibrosis, from 3.4 to 3.1kPa.

MedNess: Although on Friday, April 21, 2017, we did not see Gilead stocks soaring after the news, based on 19 analysts polled by TipRanks, majority approve buying Gilead stock while 7 maintain a hold and 0 recommend selling (SmarterAnalyst).

 FDA issues new warnings against the use of opioids in kids and nursing mothers

The Food and Drug Administration (FDA) issued a consumer safety alert on April 20, 2017, ordering major label changes on prescription drugs containing codeine and tramadol. Codeine is found in some prescription pain, and cough medicines and some over-the-counter cough medicines and tramadol is found in some prescription pain medicines. The opioid drugs are metabolized rapidly by children which can lead to breathing problems.

The FDA listed 15 medications and their generics that will be affected by this warning ranging from J&J’s Tylenol with codeine and Ultracet with tramadol, Vertical’s ConZip with tramadol, and Allergan’s migraine medication Fiorinol with codeine (

FDA approves Roche’s Tecentriq as first line treatment for certain patients with advanced bladder cancer treatment

Roche’s Tecentriq gained accelerated approval from the FDA as a first-line treatment in patients with advanced bladder cancer who are ineligible for cisplatin chemotherapy. Tecentriq was earlier approved for treatment in patients with advanced or metastatic bladder cancer whose disease worsened within one year of standard chemotherapy.

MedNess: This immunotherapy was approved earlier for the treatment of non-small cell lung cancer ( The current approval enables label expansion of this immunotherapy. As per Zacks ranking list, Roche stocks are strongly recommended for buying (

Novartis’ CAR-T CTL019 receives FDA “Breakthrough” Tag for the treatment of most common form of lymphoma

Novartis received the US Food and Drug Administration (FDA) Breakthrough Therapy designation for CTL019, an investigational chimeric antigen receptor T-cell (CAR-T) therapy. Last month, CTL109 received the breakthrough designation for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

This is the second indication for which CTL019 has received this designation for relapsed/refractory diffuse large B-cell lymphoma (DLBCL) for the treatment of adults who have failed two or more prior therapies.

The Breakthrough Therapy designation is based on data from the multi-center Phase II JULIET study. The results from JULIET study are expected to be presented soon (

MedNess: Novartis is competing with Kite Pharma over CAR-T therapy. Kite already has breakthrough designations for DLBCL, transformed follicular lymphoma (TFL) and primary mediastinal B-cell lymphoma. After the announcement, Kites shares dropped by 1% and Novartis’s by 0.3% (FierceBiotech)

MedNess # March for Science by Radhika Raheja Ph.D

While we look at FDA approvals and how they affect pharmaceutical companies, it is important to acknowledge the science coming out of academic institutions that steers translational discoveries. Here is a brief report on how science impacts lives and why it is necessary to support scientific research.

Reporting from outside the lab – #marchforscience #Boston

This week we are not ‘Reporting from the lab” but from outside the lab where most of the excitement was happening. Thousands of scientists all over the world took to the streets to “March for Science” on April 22, usually observed as Earth Day.

Why do we march for science? We march for science because “ Science is real, denial is deadly”, “ No science, no beer”, “ Progress in science = progress in humanity”, “Climate change is real” and several other reasons. Here in Boston, the research community in the Longwood medical area marched under the motto “ Science is good for your health“. Our rally kick-started with a lineup of extremely eloquent speakers, faculty, students, patients and the Dean of Harvard Medical School (HMS) who spoke about the impact of science on our lives and the ramifications that reckless changes in science policy can have on all of us.

Why do we march for science? Science gives us the opportunity to give back to the community. The Dean for Students at HMS, Fidencio Saldana, emphasized that science affects all of us. “Science is for people who want to invest in the future of our children” he said, as science education creates awareness, teaches our children to think critically, ask questions and creates opportunities for them in the future. Fidencio Saldana ended by saying that there is, “too much at stake for us to remain silent anymore”. On a similar note, Senan Ebrahim, an MD-PhD student at the HMS reminded us, “to raise our voices and speak the truth, because we are blessed with knowledge and it is our responsibility to act on it and share it.” It is our duty to leave behind a legacy of advanced engineering, improved medical care and to safeguard the future for young and bright scientists.

Why do we march for science? Science gives us hope when we are afflicted with disease. This was further exemplified by the stories of patients and doctors on the innovative cures for various diseases including sickle cell anemia, acute lymphoblastic leukemia, neuroblastoma, lymphangioleiomyomatosis that have saved lives, thanks to fundamental scientific research conducted in laboratories within Boston and the nation. In order to continue fostering the promise of scientific discoveries, it is important to ensure our voices are heard. “Scientific research is one of the fundamental pillars of our society, … this is not a fight for our livelihoods, this is a fight for human lives “ said Elorm Avakame, an MD/MPH student at Kennedy School of Public Health.

Why do we march for science? “We march because we are facing a threat to humanity “. The proposed budget cuts within federal agencies like the National Institute of health itself will be dramatic as it has an annual budget of $32billion and propels scientific progress not only in the United states but in the world. “Such budget cuts, if applied, will have tragic consequences that will haunt us for generations, destabilize our economy and pose an existential threat to America’s preeminence as a world leader in biomedicine”, said George Daley, Dean of HMS. He also added that NIH funding supports over 380,000 jobs nationwide and over 31,000 jobs in the state of Massachusetts alone. Research funded by the NIH drives an economic activity of over $65 billion a year. “Scientific progress, scientific discovery is an enduring symbol of what is best and what is most noble about this great nation. Cutting biomedical research funding will eviscerate our ability to relieve suffering here and around the world. It threatens the very core of our mission”.

Boston plays a historic role in creating and nurturing some of the best scientists and physicians trained to alleviate human suffering caused by disease. Nearly half of new cancer drugs in the last 5 years emerged from the hard work and curiosity of scientists in the laboratories at Harvard University funded by grants from the National Institute of Health. In this era of phenomenal advancements in science, it is terrifying to envision the therapeutic landscape for various diseases without support for scientific research.

It was a cold, rainy day in Boston, with temperatures as low as 3oC (37oF) , yet this did not deter the spirit of the students, scientists, physicians, patients and people whose lives have been positively impacted by science to get out and stand up for science. We marched to reaffirm the importance of science and how it benefits our lives, our country, and our planet. We marched because science truly matters!

About the Authors:

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.


Radhika completed her PhD from Cornell University and is currently a Postdoctoral fellow at the Brigham and Women’s Hospital. Her research interests have centered around oncology and neuroimmunology. Among other things, she is striving to effectively communicate scientific discoveries to the community.



FDA Breakthrough A’La CAR-T: Medness Focus on Novartis CTL019

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What’s the big news?

Novartis announced that the US Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational Chimeric Antigen Receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

What’s even bigger?

On April 18 (2017) CTL019 received the FDA granted breakthrough therapy designation for the treatment of adults with relapsed and refractory diffuse large B-cell lymphoma who failed two or more prior therapies. Read more

What is ALL?

Image reference

Acute Lymphoblastic Leukemia (ALL)

Each part of its name tells you something about the cancer itself:

    • Acute: Often fast-growing, requires early detection and treatment. Without treatment, bone marrow cells developmentally impaired, resulting in an unhealthy bone marrow filled with proliferating abnormal lymphocytes.
    • Lymphoblastic: Affects the lymphocytes of a patient’s white blood cells. Alternative term is lymphocytic.
    • Leukemia: Leukemia is a cancer of the blood cells.
  • Most common cancer in children, but it can also occur in adults of all ages (bimodal age distribution, with peaks at 3-7 years and 65 years of age).
  • Clinical presentation is nonspecific:
    1. fever;
    2. infection;
    3. bleeding;
    4. bone pain;
    5. lymphadenopathy;
    6. CNS involvement.
  • Disease classification based on evaluation of cells derived from a bone marrow or tissue biopsy. Clonal cells may be B cells (B-precursor lineage, 75%) or T cells. There are three main different ALL subtypes as follows:
    1. Pre (precursor) B cell ALL – most common in adults
    2. Mature B cell ALL – identified by particular genetic changes
    3. Pre (precursor) T cell ALL – more likely in young adults and more common in men
  • Management involves
    • remission induction with combination chemotherapy.
    • intrathecal chemotherapy is indicated for all patients to prevent CNS relapse.
    • Post-remission, patients undergo 1-3 years of maintenance therapy to eliminate residual disease.
    • Read more


What’s the history ?

  • CAR T-cell therapy, may appear to be overnight success, has a long experimental history.  Chemist and immunologist, Zelig Eshhar, developed the first CAR-T cells at the Weizmann Institute of Science in Israel in the late 1980s. In 1990, Eshhar took a year-long sabbatical, joined Steven Rosenberg at the National Institutes of Health, and prepared CARs that targeted human melanoma. “We designed CAR T cells to overcome a number of problems in getting T cells to attack cancer,” says Eshhar. The problems being a tumor’s ability to escape immune recognition by preventing the major histocompatibility complex molecules and the immunosuppressive tumor microenvironment.

  • CTL019 first developed by the University of Pennsylvania (Penn) by Carl June‘s group (link to original NEJM paper). Read more.
  • In 2012, Novartis and Penn created a global collaboration to advance research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications. In March 2017, Novartis announced that the FDA accepted the company’s Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.


What is the science behind it?

  • CAR-T therapies exploit the capability of a patient’s immune system to fight their disease, (sometimes referred to as “fifth pillar” of cancer treatment).
  • Therapy involves engineering patients’ own immune cells to recognize and attack their tumors (popularly known as Adoptive Cell Transfer).
  • Renier J. Brentjens, MD (Memorial Sloan Kettering Cancer Center) describes it like “giving patients a living drug.”


What was the outcome of the clinical trials?

  • The Children’s Hospital of Philadelphia (CHOP) study (link) showed disappearance of all signs of cancer (a complete response) in 27 of the 30 patients treated. 19 out of 27 are still in remission
  • The NIH Pediatric Oncology Branch study (link) 14 of 20 patients had a complete response with 10 of them receiving successful stem cell transplant and remain cancer free.
  • The Memorial Sloan Kettering Cancer center (MSKCC) clinical trial study (link) 14 of the 16 participants showed complete response and 7 eligible patients got stem cell transplant staying cancer-free.
  • The NCI-led study (link) showed “Of 15 patients, eight achieved complete remissions (CRs), four achieved partial remissions, one had stable lymphoma, and two were not evaluable for response”. This showed the “effectiveness of treating chemotherapy-refractory B-cell malignancies with anti-CD19 CAR T cells”.
  • Novartis clinical trial (ELIANA) evaluating efficacy and safety of CTL019 (with study enrollment having occurred across 25 centers in the US, EU, Canada, Australia and Japan) found that 82% (41 of 50) of infused patients achieved complete remission.
  • The second global CAR-T trial, JULIET, following the Novartis ELIANA study, led FDA to confer Breakthrough Therapy Designation for Treatment of Adult Patients withrelapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL). The findings from JULIET are expected to be presented at an upcoming medical congress.

What are some of the doubts?

  • CAR-T, which induces an extreme immune response that attacks cancer cells, can create a cytokine storm leading to extreme side effects like high fever.
  • CAR-T might need the best – and presumably the most highly-paid – doctors and healthcare teams to ensure patients can manage the side effects.
  • The laboratory process of extracting immune system T-cells from each individual patient and altering the DNA to create chimeric antigen receptors will create additional costs (totaling upto $500,000-750,000 to treat one patient). Health providers might not be ready to foot the bill.
  • Initial failures from competitor Juno Therapeutics have created doubts on Novartis pulling out of the study. Novartis has previously backed out of large research programs like RNA interference.

What should the patients and their families know?

  • CTL019 is an investigational therapy- safety and efficacy profile not yet established.
  • Access to investigational therapies only available through carefully controlled and monitored clinical trials.
  • No guarantee that CTL019 will ever be commercially available anywhere in the world.

MedNess Quotient

After the announcement, Novartis shares were little changed but the shares of the company making CTL019 raw materials, Oxford BioMedica, rose by more than 4.5 percent. Alternatively, Kite Pharma, Novartis’ rival in CAR-T race, also submitted a rolling application for their chimeric antigen receptor T cell candidate. Rolling applications are allowed for promising new drugs. Kite’s application could be accepted early putting behind Novartis’. Therefore, the winner of the CAR-T race will set the price of the therapy and subsequently the stocks (Reuters and Nasdaq).

References and additional reading:


Featured image source: Twitter

Disclaimer: This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

MedNess: FDA approvals,rejections, M&A and more…

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Illustration: Ipsa Jain

Hello and welcome to MedNess where we bring you the news from healthcare market. We are excited to announce the brand new section known as MedNess Focus, led by Abhinav Dey, Ph.D. that will be published every alternate week. Stay tuned for that next week where he will “Focus” on Novartis’ CAR-T therapy BLA! We are also excited to introduce MedEurope this week, led by Czuee Morey, Ph.D. For other in-depth coverage, please subscribe to @Club SciWri (

FDA grants approval to Gilead Hep C drugs for pediatric patients
The U.S. Food and Drug Administration granted approval to supplemental applications for two Hep C drugs; Sovaldi (sofosbuvir) and Harvoni (ledipasvir and sofosbuvir) for use in pediatric population ages 12 to 17 or weighing at least 35 kg. The drugs are approved to treat hepatitis C in adolescents without cirrhosis or with compensated cirrhosis. Harvoni gained approval for treating pediatric patients with genotype 1, 4, 5 or 6 hepatitis C virus (HCV), while Sovaldi was approved for treating pediatric patients with genotype 2 or 3, in combination with ribavirin. Both of these drugs were previously approved to treat hepatitis C virus (HCV) in adults. The supplemental approval came in as a win for both the medical field and business investment field. These two drugs are the direct-acting antiviral agents approved for the pediatric patients. Direct acting antivirals are efficient in curing HCV in most cases. These agents prevent the virus from multiplying thus reducing the amount of HCV in the body. Alternatively, the supplemental approval of Sovaldi  and Harvoni  also came in as a relief to the slumping Gilead sales (Fierce Pharma)

MedNess: Gilead Sciences tops HCV and HIV treatment market. However; recently, the patent battles and competition from the rivals has given tough time to Gilead’s stocks. While investors have been worried about Gilead’s cranking cash flow, the expanded label approval of Gilead Sciences star HCV drugs; Sovaldi and Harvoni should provide a sigh of relief. There was a slight increase in the stock price after the expanded drug approval use (seeking alpha and The Motley Fool).

FDA rejects Merck’s plea to drop cardiovascular risks from sitagliptin label
The FDA sent a complete response letter to Merck rejecting the drug maker’s appeal to include outcomes from its TECOS trial. This heart study showed that Merck’s drugs used for the treatment of diabetes; Januvia and its related combos Janumet and Janumet XR had “no signal” of heart failure (FiercePharma).

MedNess: Merck has been facing competition with Eli Lilly’s and Boehringer Ingelheim’s SGL2 medicine Jardiance and Novo Nordisk’s GLP-1 Victoza which have shown to reduce the combined risks of heart attack, stroke, and death from the cardiovascular disease. Amongst these rivals, Merck wanted to follow suit. The stock prices of Merck fell after the letter on Friday (Investor’

FDA authorizes 23andMe Personal Genome Service Genetic Health Risk tests
The FDA approved the marketing of genetic health risk analysis. The results of these tests will provide information directly to customers without requiring physician’s prescription.
The genetic health risk tests can detect a genetic predisposition to 10 diseases including Parkinson’s disease, late-onset Alzheimer’s disease, and Gaucher disease. But there are certainly gray areas with the testing system. The tests are designed to provide information on genetic predisposition that can help make lifestyle changes, but the results are not expected to be entirely valid or “fully penetrant.” This implies, even if a person is shown to be at higher risk genetically, he or she might never develop a disease in their lifetime and vice versa. In addition, the reports provided by these test results will be independent of the family history. This is particularly concerning as for some diseases; family predisposition might increase the risk in general. Therefore, customers are advised beforehand to not to rely on the test results completely and not to get emotionally upset with the unfavorable test results (STAT).

NIH’s Zika vaccine enters Phase 2 trial amongst budget cut frenzy
The Phase 2 trial testing a Zika vaccine, developed by scientists at the National Institute for Allergy and Infectious Diseases (NIAID), began at Baylor College of Medicine in Houston. The phase 2 study results are expected by the end of this year.
The clinical trial involves a DNA-based vaccine, lacking a live Zika virus, but from which proteins are placed into small pieces of DNA. NIAID is a part of the NIH and is obviously affected by President Trump’s recent budget proposal cut. However, given the importance of this study, the institute has dedicated $100 million funding for the Phase 2 work. If the Phase 2 study is successful, NIAID will partner with commercial sources to bear the costs of larger Phase 3 trials (STAT)

Novartis CAR-T therapy BLA granted FDA priority review
Novartis announced that the US Food and Drug Administration has accepted the company’s first Biologics License Application (BLA) filing and granted the priority review for CTL019 (tisagenlecleucel-T) in relapsed and in relapsed and refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). CTL019 is an investigational chimeric antigen receptor T-cell (CAR-T) therapy.  The priority review designation is expected to shorten the anticipated review time to six months.

In the Phase II ELIANA study, 82% (41 of 50) of patients infused with CAR-T cells achieved either complete remission or complete remission with incomplete blood count recovery after three months. The study enrolled patients globally across the US, EU, Canada, Australia, and Japan.

CAR-T therapy, one of the most controversial treatments involving gene therapy, utilizes reengineered patient’s T cells. These T cells (immune system’s killer cells) are filtered from patients blood and altered in the lab and injected back intravenously making it a “living drug.”(

MedNess: After the announcement, Novartis shares were little changed but the shares of the company making CTL019 raw materials, Oxford BioMedica, rose by more than 4.5 percent. Alternatively, Kite Pharma, Novartis’ rival in CAR-T race, also submitted a rolling application for their chimeric antigen receptor T cell candidate. Rolling applications are allowed for promising new drugs. Kite’s application could be accepted early putting behind Novartis’. Therefore, the winner of the CAR-T race will set the price of the therapy and subsequently the stocks (Reuters and Nasdaq).

MedEurope by Czuee Morey, Ph.D.

Astellas Pharma expands its portfolio to women’s health by acquiring Belgian biotech Ogeda
Japanese Astellas Pharma announced its plans to acquire privately owned, clinical-stage drug discovery company Ogeda. The acquisition comes three months after Ogeda achieved positive results from a Phase IIa study of fezolinetant (ESN364), its lead drug candidate. Fezolinetant is a potential non-hormonal treatment for menopause-related vasomotor symptoms (MR-VMS) such as hot flushes and night sweats and is an antagonist of the GPCR known as tachykinin receptor neurokinin3 (NK3). The phase II data showed statistically significant reduction in both frequency and severity of menopausal hot flushes versus placebo in 80 women.
Astellas’ offer consists of €500 million ($530 million) upfront followed by an additional €300 million ($318 million) if Ogeda meets its development and regulatory milestones. Astellas will also benefit from a few pre-clinical assets under investigation for autoimmune diseases and ulcerative colitis. The companies expect the deal to close in the second quarter, at which point Ogeda will serve as an Astellas subsidiary. This acquisition will help to expand Astellas’ pipeline that is primarily focused on oncology.

MedNess: The global MR-VMS market was valued at US$3.77 bn in 2014 and is projected to grow at a CAGR of 3.7% from 2015 to 2023 to reach US$5.28 bn by 2023. Ogeda is also running Phase II trials with the lead candidate to treat polycystic ovary syndrome (PCOS) and uterine fibroids. Other companies developing neurokinin antagonists are UK based NeRRe Therapeutics for a range of conditions from sex hormone imbalances to opiate use and US-based Millendo Therapeutics for polycystic ovarian syndrome, both in Phase II.
Astellas stock, which trades on the Tokyo Stock Exchange, was up about 2% to ¥1,492 ($13.39) on Monday morning after the announcement.
(PRNewsWire,, Seeking Alpha)

About the author:

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.


Multiple Sclerosis, Single Lead- Medness Focus on Ocrevus

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What’s the news?

The multiple sclerosis community has been eagerly awaiting the approval of the drug Ocrevus (ocrelizumab), which will be used to treat patients who have relapsing MS (RMS) and primary progressive MS (PPMS). The FDA’s decision of final approval came on March 28, coinciding with Multiple Sclerosis Awareness Week.

Ocrelizumab is the first and only investigational drug

  • to show greater efficacy in both RMS and PPMS in clinical studies.
  • to consistently and significantly reduce disease activity and disability progression compared with a standard-of-care high-dose interferon (Rebif®).
  • to significantly reduce the progression of physical disability in primary progressive MS in a large Phase III study (ORATORIO).
  • that has the potential to address an important unmet need in MS.

What is Multiple Sclerosis?

MS is a chronic, typically progressive disease involving damage to the sheaths of nerve cells in the brain and spinal cord.

Patients with MS may show paresthesias (tingling sensation), blurred vision, optic neuritis (painful unilateral vision loss), clumsiness, muscle weakness, cognitive decline, and urinary dysfunction. Unfortunately, the neuron in the picture is also feeling some of these signs. The Lhermitte sign is caused when neck flexion creates electric shock-like sensations down the back and limbs.

Types of MS

1.    Clinically Isolated Syndrome (CIS) First episode of neurologic symptoms caused by inflammation and demyelination in the central nervous system.

2.    Relapsing-remitting MS (RRMS) is characterized by clearly defined attacks of new or increasing neurologic symptoms.

3.    Primary Progressive MS (PPMS) worsening neurologic function (accumulation of disability) from the onset of symptoms, without early relapses or remissions.

4.    Secondary Progressive MS (SPMS) Most people who are diagnosed with RRMS will eventually transition to a secondary progressive course in which there is a progressive worsening of neurologic function (accumulation of disability) over time.

The History

In the past 20 years, we’ve seen a lot of improvement in the battle against MS to benefit the 2.3 million people worldwide who have this ailment. Notwithstanding these developments, people with RMS continue to need medications that offer the possibility for greater efficacy than standard-of-care interferons, with a favorable safety profile.

For people with PPMS there were no approved treatments before Ocrevus. Previous Phase III trials with investigational medicines have been unsuccessful in demonstrating a significant effect on disability progression in PPMS.

What is the science behind it?


  • targets myelin-attacking B-cells (unlike similar medications attacking T-cells)
  • is an anti-CD20 humanized monoclonal antibody

Image source

How was the drug developed?

Genentech’s Medical Director, Peter Chin, said “The journey of ocrelizumab in MS started about 15 years ago, when Genentech began collaborating with academic researchers at major universities to investigate the importance of B cells in MS and their potential as a therapeutic target. The first small proof-of-concept studies showed that CD20+ B cells appeared to play a more important role in MS than anybody previously thought.”

Read the full interview with Genentech’s Peter Chin here

What was the outcome of the trials?

More than 1,600 MS patients enrolled in clinical trials for Ocrevus and 94 percent of participants had fewer brain lesions during the 96 weeks of treatment.

A summary of the data from the OPERA I, OPERA II and ORATORIO studies that support this approval can be found here.

What are some of the doubts?

There was little increased risk of infection (link). 

The clinical trials for Ocrevus also found that while patients taking the drug did have a slightly increased risk of common colds and flu, they had no significant increased risk of other infections when compared to patients taking the alternative medication, Rebif.

Some concerns like “Targeting B cells in MS patients appears to be Ocrevus’ strength, but depleting B cells also raises safety concerns” were addressed to Dr Chin. He responded saying, “Ocrelizumab selectively binds to CD20, a cell surface antigen expressed by a subset of B cells. CD20 is not expressed on stem cells or antibody-producing plasma cells, and therefore pre-existing humoral immunity and the ability to reconstitute B cells may be preserved. Since the CD20 protein is not found on many other cells of the immune system, they can continue to fight infection and other illnesses.

What should the patients and their families know?


  • will be administered by intravenous infusion every six months.
  • will be used for treating primary progressive MS.
  • will also be used for treating relapsing MS patients.
  • may have potential serious side effects which may include infusion reactions, infections and malignancies where only routine screening is required based on age and medical history

Medness Quotient from Imit Kaur

“Analysts forecast annual sales exceeding $3 billion by 2021 as reported by Reuters. After the approval news, Biogen stock fell by 2%, and Roche stock rose by a fraction. Novartis’s drug for MS treatment, BAF312, for secondary progressive MS is expected to receive regulatory approval in the first half of 2017. Until then, Roche can bask in glory  (Reuters, Investor’s Business Daily, FiercePharma).”

Featured image source: Pixabay



Disclaimer: This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

MedNess- March Mania

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Hello and welcome to yet another exciting week of MedNess. In this March mania, we bring the news from medicine and healthcare with the greatest impact.

Tom Price justifies NIH’s “indirect” budget cuts

Secretary of Health and Human Services (HHS) defended Trump’s administration proposed National Institute of Health’s budget cuts. The Trump administration proposed $5.8 billion, about 18% cut for the fiscal year 2018. In addition to that, an addendum proposed an additional $1.2 billion cut for the current fiscal year. When questioned by both democrats and republicans about the nature of budget cuts, Price explained to reduce the “overhead” costs to streamline the system. According to the Secretary of HHS, the “indirect” cost takes up about 30% of the grant money, which could otherwise be available for research.
This came in as a second blow to the medical research community. Earlier this year, Trump administration pushed deregulation of FDA to accelerate the drug approval process. The research community did not welcome this proposal. Also, both pharmaceutical companies and insurance companies did not approve of the proposition (STAT and Science).


March for Science, a scientist’s view:  For our readers, “overhead” or “indirect” costs constitute of anything required for carrying out research safely, smoothly and efficiently! Some of the “indirect” costs include lab equipment, electricity, custodial services and other utilities. The list is not inclusive but clearly, emphasizes the importance of overhead charges. The budget cuts will not only affect the advancement of research but will also impact jobs and outreach of science. For non-science professionals, say, politicians, the overhead cost will include, electricity, custodial services, security, dinner, travel, etc. Again, the list is not inclusive! Of course, these expenses are required for the smooth functioning of the government. Before I wrap up the section, just a thought: yes, we need the stronger military to defend the country, but we need to ask this question, who are we protecting; the people and the Mother Nature. Therefore, we need an effective EPA and NIH. We need the healthy and clean environment and disease-free children and adults.

Amgen’s LDL-lowering drug Repatha: effective drug with good data for a bad price?
Amgen presented Phase III FOURIER results on Repatha (Evolocumab) at the 2017 American College of Cardiology conference. Repatha is an LDL-lowering PCSK9 inhibitor. Repatha targets PCSK9 proteins in the blood stream thus preventing it from binding to and breaking down LDL cholesterol receptors in the liver. The trial results were impressive. This wonder drug is believed to break down the most stubborn cholesterol. The FDA approved drug marketing in 2015 after the drug’s addition to statins reduced the LDL levels by about 63%. At the ACC, Amgen reported the outcome of long-term FOURIER trial and the results showed that Repatha reduced the risk of heart attack and stroke by 15% or more. Repatha met both its primary and secondary composite endpoint in the secondary prevention trial demonstrating superiority to statin therapy. However, analysts are not too impressed with the data and question the high price of the drug. Earlier, analysts with BioPharmInsight suggested that the high price of Repatha could be justified if the cardiovascular event risk reduction is at least 35%. Repatha has been price tagged for $14,000 annually. The trial findings and analyst’s reports will also affect insurance coverage of the drug (MedPage Today).

MedNess: PCSK9 is the hottest target in the field of cardiovascular research. While Amgen’s drug can crush the most stubborn cholesterol molecules, the investors were not impressed, or at least the stock market trend did not concede with it. After the results had been announced, Amgen’s stock price went down by 10%. However, a lot of analysts are still keeping their faith in Amgen’s stocks and considering this temporary dip as an opportunity for investment. On the contrary, the competition from other biosimilars is getting fiercer, and the dip in stock price might not be temporary after all. Other contenders in this area with Amgen, are the drugs from Sanofi and Regeneron. Both the companies are locked in a patent battle with Amgen. Another drug in the race is The Medicine’s Company’s inclisiran. Unlike Amgen’s, Sanofi’s and Regeneron’s drug, inclisiran interrupts PCSK9 synthesis. The analysts look at this drug as efficacious as Repatha but with fewer annual doses. If this assumption is correct, Amgen will have a hard time convincing insurance companies for their drug price. The best bet might be therefore to either wait or invest wisely (The Motley Fool and Seeking alpha).

FDA approves Roche MS drug Ocrevus after 3-month delay

The FDA approved Roche MS drug Ocrevus after initial delay caused by regulator’s concerns over manufacturing issues.
Ocrelizumab, becomes the first U.S.-approved medicine for the primary progressive multiple sclerosis. It has also been approved for relapsing-remitting multiple sclerosis (RRMS). Biogen’s MS drug has been used to treat RRMS. Biogen will receive up to 24% royalty on U.S. sales of Ocrevus. According to the pharmaceutical giant; Ocrevus is expected to be available for use to people within two weeks.


MedNess: Analysts forecast annual sales exceeding $3 billion by 2021 as reported by Reuters. After the approval news, Biogen stock fell by 2%, and Roche stock rose by a fraction. Novartis’s drug for MS treatment, BAF312, for secondary progressive MS is expected to receive regulatory approval in the first half of 2017. Until then, Roche can bask in glory  (Reuters, Investor’s Business Daily, FiercePharma).

FDA approves Tesaro Inc’s Niraparib for the treatment of Ovarian Cancer

Tesaro’s Niraprib (Zejula) gained an early approval by FDA for the treatment of recurrent ovarian cancer. Zejula is a PARP inhibitor causing DNA damage. It is a first drug in the class that can be used to treat all women with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer without requiring BRCA mutation or biomarker evaluation. This is unlike the rival drug Lynparza by AstraZeneca. In addition, Zejula acquired orphan drug designation for its use in the treatment of recurrent epithelial ovarian cancer.

MedNess: According to EvaluatePharma, Zejula is one of the top drug launches of 2017 with 2022 sales expectations of $1.9 billion. Tesaro Inc’s shares were up 7.78 percent after the drug gained FDA approval (FiercePharma and BusinessInsider).

Illustration: Ipsa Jain

About the Author

Imit Kaur is a freelance medical writer, editor and an active science blogger. She pursued her PhD in Pharmaceutics and Pharmaceutical Chemistry from University of Utah. She is experienced in the field of oncology, hematology, pharmacology, nanotechnology and drug development.

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