Ahcj

uromycitis

How many health studies are wrong. Editor of medpage. Ivan someting

Predatory journals no peer review, take money

Ama journal of ethics

Quartz many scientific truths are in fact false

John Ionnadis reproduceability book. Excerpt?

6 of 53 basic science studies held up

Later stdies maybe 50 percent canmt be reproduced

NEJM mistaken identities plague lab work with human cells

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Retractions are on the rise. Retraction Watch. 650 in 2016 in pubmed.

NEJM has highest retraction rate.

Tumor Biology retracted 100 pgs all at once. Fake peer reviews. s

PubMed commons. Any scientist can comment on any other paper. Important
source.

Pubpeer.com

Get a biostatistician. They will save you from writing about something glowingly.
They love ripping up papers.

Ivan-oransky@erols.com

@ivanorsansky

Studies: phase 0 to phase 4.

Sometimes u have to look at charts to find absolute risk.

Were they clinically significant where they can change practice.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Who funded the study?

How different are the outocmes? How strong are findings/ statistically signficiant?
Clinically significant wehre will change findings.

Meta analyses and systematic reviews. Systematic review. Search of existing lit. on
question using specified protocol that could be replicated.

Meta analysis. Stat. techniqes to synethesie data from several studies into single
estimate or summary effect size.

These can also be wrong if they leave out important studies.

Ahcj website. Core ideas for studiy evidence.

Randomized controlled trials are the point where more serious research.

Abolsute risk vs. relative risk. Relative risk reduction effect dize seems larger

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Absolte risk reduction effect size seems smaller.

Relative risk is change from one to another

Abolsute is % change

What is the denominator? Actual change can be from 2 to 3 percent. Relative risk =
33 percentr reduction. Aboluste risk is only 1 % risk difference

Which # better conveys drugs effect?

Aboslute risk is always best! When you can get it.

More often than not its absolute risk.

Hazard ratios. best indication.

Odds ratios. Abvoid thes

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Essentials is there clinical significant result? Going to make difference to patients
or doctors prace. Major red flags like no control group? Wehre is it on evidence
pyramid? Observational studies are not so good.

Use absolute risk and numbers as much as possible.

Standard deviations. Next to useless for reporting.

RELY ON OUTSIDE EXPERTS.

QUESTIONS TO ASK:

WHAT ARE MOST STRIKING SUPRISING IF ANY FINDINGS HERE? WHY?

WHAT ARE IMPLICATIONS OF THESE FINDINGS?

TO WHAT EXTENT WOULD/COULD FINDINGS AFFECT CLINICAL PRACTICE?

HOW STRONG OR RELIABLE ARE TESE FININDS W/IN CONTEXT OF WHAT WE

ALREADY KNOW?

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

ARE THERE ANY PARTICULAR STRENGHTS OR LIMITATIONS IN THIS STUDY ESP.
WORTH NOTING?

WHAT KEEPS YOU UP AT NIGHT, WORRYING ABOUT UNCERTAINTIES IN THE DATA?

Resources.

Healthjournalism.org/medicalstudies

Kevin Lomangino . Managing Editor HealthNewsreview.org. @klomangino.

@healthnewsrevu. kevinlomangino@gmail.com

PR news releases are a big problem.

Only 23% of press releases included any mention of study limitations. 22% mention
industry funding. Nearly half relied on relative risk descriptions tha are proine to
exaggeration. 23% contained more direct or explicit advice than was found in their
relvant journal articles. News stories more likely to exaggerate if press release does.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Spin in journal abstract leads to spin in press release leads to spin in news.

10 criteria. on plastic card in my wallet

Most stories dont adequately discuss cost. Then : how big are potential enefits.
Scopre of potential harmds. Quality of efience. Alternative options.

Dont use casue and effect language to describe observational studies

Dont exaggerate effect size usually by using relative risk data

Dont use definitieve health claims made from preliminary studies and animal
research.

How did you adjust for confounding factors? I can see you adjusted for that, but how
much can you really fully account for something as complex as that? Was your
method adequate?

Weasel words: could, might, may.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

SENSITIVITY AND SPECIFICITY AND POSITIVE PREDICTIVE VALUE UNDERSTAND
THESE TERMS.

False positives.

Book: News and Numbers

Positive predictive value find out what that is.

GENOME EDITING PANEL

Old tech random insertion and low efficiency

Find and detect chromosome thats broken. Insert into break.

Used to use endonucleases. Zinc finger nucleases or zfns.

Then talens

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Cell repairs itself.

Targeted edits. But effieincy not great.

Crispr is 3rd major form of gene editing machinery.

Cirspr uses rna to find area.

Editas. Cure type of blindness.

Germline editing. NAS gave yellow light.

Personal Genetics Education Project story? Lesson plans. Johnny Kung. Our team on
website w/ email address.

Tin Wu dept of genetics Harvard. Married to George church.

Protect astroanuts in long-term space travel. Radiation. Have to protect astornauts.

Loss of bone mass.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Loss of muscle mass. Variety of medical issues.

Ethics and policy debates in human gene editing.

Josephine Johnston, Hastings Center.

Rider to bill preventing embryonic stem cell research. or creating them for that
purpose.

Existing oversight:

Recombinant DNA advising committee. RAC existing body that rules on NIH funded
stuff. FDA approval.

Intl conf on ethics of gene editing

Somatic editing research should continue and be evaluated. Germline editing should
not proceed at this stage. Though its potentially valuable.

Safety concerns:

difficulty of predicting harmful effects. Genes interact w/ other genes. Envornmental

interactions. S

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

fear: permanent genetic enhancements to subsets of population. Rich people can
afford

fear of purposefully altering human evolution.

Human Genome Editing: Science Ethics and Governance

Somatic editing to prevent or treat disease should be evaluated w/in existing

frameworks. But germline editing must meet 10- conditions.

Including : only edit genes that cause disease. Continued reassessment of health
and societal risks. Broad input by public.

Societal risk is big theme.

Report 30 years ago was dismissive of concerns.

Gene Editing and Human Flourishing.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

House issued another rider. FDA not allowed to look at anything thats a heritable
change. Some are good but maybe not all. Cant have a bright line.

Bright lines are appealing in rhetorical sense but misleading. No easy rules of
thumb. Nuanced decisions about complex traits.

What is public enagement and how does it work?

Actually look at what concerns are of critics.

Post using Josephine Johnson on bioethics?

Put what were going through now in context of history. How have we weathered
similar advances?

Make sure underserved communiteis are fully part of conversation. Tremendous

distrust of scientisists in some communities.

Working w/ minority coalition for precision medicine.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Guide rna can recognize bases within 20 either way

Crispr heralded bc big step over what as there before. Turns out many different
types of crispr machinery. Is crispr the end, prob. not.

Black pastors interested in sickle cell.

Parenting in the genomic age

The Gene Machine

Eli Y. Adashi

Only 5 yrs ago did it become reasonably practicable to discuss germline editing

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

2012 original crispr paper. Programmable dual-rna-guided dna endoncuelase:
Science journal

2015 Jan 24, 2015 the innovative enomics intitiative forum on bioethics. Ucb and
ucsf

2015 0 nonviable and non transferable embryos worked on. Human gene editing. In
china. Protein cell journal.

NIH statement wouldnt fund any research using gene editing technologies in
human embryos. Per dickey amendment.

April 28 2015: science committee of house. Lamar smith. Doudna was on the panel
as witness. No specific conclusion drawn. But Dr. Dzau announced intiative :
intnational summit on human gene editing. Dec 2015

3 weeks after summit: Dec 2015 congress put moratorium on germline editing in US

IN effect through april 28 2017. Every expectation will be renewed. STORY?

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

We dont know who sponsored this amendment.

No congressional discussion. Unknown motives.

Wisdom of this moratorium?

NAS Human Genome Editing: Science, Ethics and governance. Heritable genome
editing only under certain conditions.

Francis Collin suggested in 2010 that eventually we will have mandatory genome
sequencing.

The Right Not to Know and The Right to Know.

CEO of 23andme Genetic testing is a reponsbility if youre having children.

Julian Savulescu respoinsiblity to have best possible genetic children we can.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Ronald Green. Also feels that we should not harm our children genetically.

Will editing your babys genes be mandatory? The Atlantic

Michael Sandel dangerfs of these techniques. Mistake to think good parenting is

controlling kids genes.

Women who have Downs syndrome babies. Ethical dilemma.

Deaf culture:

Deaf vs. Deaf. Upper refers to socioloingustic communites that use signed language.

Hearing uppercase, refers to community of hearing people.

Gentetics and deafness

Many member of the signing Deaf cultural community worry that the community is
becoming extinct.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Teresa Blankmeyer Burke deaf and bioethics

OMIM lists 591 different gene map loci for deafness.

Genetic screening involves either screening potrential parents regarding their

chances of bering deaf children or screening family members. Some deaf people
WANT to have deaef children.

Should the deaf community be protected from this potential threat of eradication?
STORY?

What are most cogent grounds for that argument?

-biological diversity is good. Linguistic diversity is good. Modality matters. Group

rights.

Do groups have rights? Does deaf community have right to exist as a community?

Ethical issues and questions:

-harm and bodily integrity.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Trans health

Gender identity unchangeable?

About 1.4 million transgender adults, we believe. And that many teens.

Autistic, monozygogic twins, chromosomal abnormalities. could effect

transgenderism

Insurance and hormone therapy not covered by a lot of insurance

Med schools. Education about trans.

Major employers are advocates. Google.

HRC corporate equality index

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

High rates of murder and violence

Traveling is an issue. TSA.

Hormone replacement therapy is one of 1st steps. Endocrinology.

Gender reassignment surgery not elective surgery.

ACA section 1557? Eliminated exclusions for transgender care.

The Genderbread Person

Dont put ed at end of transgnder.

Dont say born male

assigned male

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Sex reassignment surgery faux pas. Gender confirmation surgery. Language
evolving.

dead name dont ask what previous name was. Name that people used before is
not their name anymore. Dont put that name into your stories. Use name they
refer. Court-ordered name changes can be hard to get.

Images putting clothes on clich.

Name and gender you get from police might not be correct

GLAAD media guide.

Move beyond coming out narratives

Being transgender doesnt mean you need to medically transition

What does medically necessary mean. People crossing state lines to get
procedures.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Section 1557 ACA

Resources: Movement Advancement Project; GLSEN

-----------------------------------------

Immunotherapy in Cancer and Beyond

Clinical advance of the year

Around for over 100 years. Tryied to simulate immune responses.

Cell osf the immune system.

Lymphoid arm and myeloid arm. Myeloid arm trying to under stand and evelop
therapies to target.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Adaptive immune system is the lymphoid arm. Cells are lymphocites t cells or b
cells. Each t cell has specialized receptors. T cell receptors and b cell receptors.
Modified pronouns. 1 x 10 to the 18th specificieties. T cell that recognizes polio is
different than one that recognizes polio, for ex.

Powerful machine for eliminating microbial infections.

Immune system plays important role in cancer development and progression.

Paradoxical.

Immune system has mechanisms against cancer natural killer cells, t cells.
Humoral immunity. Coordinated immune responses.

Coordinated immune response multifaceted, involving different arms of immune

sytem. Has been difficult to achieve.

Immune sytem has mechanisms promoting cac er. Woundm repair inflammation.
Enhance survival pathways. Suppression of anti-tumor cellular immune responses.

Both anti-tumor and pro-tumor is immune system.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Chronicn inflammation is a type 2 response. We want to convert that into acute
inflammation. You get a type 1 response.

We want to develop immune response that can rapidly eradicate tumor.

t-cells infiltrating tumor are specific for antigens expressed.

Inhibition of procancer inflammation. And Boost of anticancer pathways.

Developing cancer vaccines: targeting microbial neoantigens or tumor-sepcific

mutated neoantigens. Nonmutated self antigens

Mutations vary with cancer types. Number of cos. engaged in mutated neo-antigen
vaccine development.

Overexpressed self proteins as source of tumor antigens

Bvaccination to prevent progression of triple negative breast cancer. A new trial.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Monoclonal atntibodies were on of the first immunotherapies

Adoptive t cell therapy with CAR t cells. Yet to make it to clinical trials.

Taking t cells out, growing them, putting back in.

One study 93 % went into remission for a year but a lot of relapses.

Immune checkpoint blockade. Molecule si associated with turning off the t-cells.
Block that and more t-cells. Very toxic bc not specific activates t cells for other
stuff.

Often we dont see resposnes to immune checkpoint blockade?

Goal of cance rimmunot to covnert from chronic eto acute inflammation.

Vaccines can be edeveloped target proteins.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Adoptive t cell making great streides but limited

Antibody therapy

Checkpoint blockade/activation

In the clinical arena. Who benefits and who does not? Excpectaitons of public are
beyond what happens in clinic.

What are costs?

Whats next?

1891. William coley injecting cancer patients with bacteria hopiong immune
response would attack cancer.

1973 0 ID o of dendritic cells. They tell immune system what to attack and what to
ignore.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

2010 first fda approved cancer vaccine. For metastatic prostate cancer.

Approaches: vaccines, immunogregulatory antibodies/adoptive cell

tehrarpy/oncolytic viruses

Sipuleucel-t: first fda approved cacner vaccine.

Improved survival in glioblasgtoma

One patient 11 yar cancer free of glioblasgoma, getting monthly vaccinations

Concerted effort to know who will benefit vs who wont. Hot vs. cold tumors.

Mutations/neoantigens/ presence of t cells/expression of immune markers are

factors.

Roughly 1/3 pateitne enefit. Costs 85k to 100k for a course of treatment for single
checkpoint inhibitors.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

$3billion in global sales predicted for 2917.

16-30 percent have severe side effects. Most managed successfully.

Identifying those who can benefit vs those who cant is important bc of side effects
and expense.

Thats a major emphasis in the field.

Exploration of combination of treatment approaches is a theme.

Immunotehrapy applications in other diseases MS, parkinsons alzheimers etc.

Duane A. Mitchell, florida. Good presentation.

Up to Date. Cochran Reviews. Health News REiew has guide on immune therapy.

Finding who thse drugs are best used in is critical.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Whose this for? Is it being overpromoted? Is it a marketing tool? Adverse events
what % have those. How serious?

GET ABSOLUTE RATHER THAN RELATIVE RESPONSE RATES. DOUBLING RATE FROM 1
TO 2 PERCENT IS NOT SO GREAT.

SMALL phase 2 trial is much less relevant than a large phase 3 trial.

Diffiuclt to know what to highlight. Important to temper the overall storyline to

reflect how far along the therapy is.

Were in a different era in which fda approved drugs have been shown to improve
large # of patients.

Presentations at conference are preliminary not peer reviewed ytet.

Diabetes Type 1 increasing.

Outomces not great in type 1. Dont meet glycemic goals.

DCCT trial.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Diabetes technology. Human factors. Regimens can involve various combos of
devices and tools. Multiple daily injections paired w/ blood glucose meter.

Insulin pumps 40-60 of adults

Continuous glucose monitoring systems.

Requires 6-7 days a week of wearing. Pediatric noncompliance.

Stress. Psychocoail factors impact.

Transdisciplinary care versus usual care for type 1 diabetes in adolescence.

Telemedicine. Effects of delivering transdisciplinary care via telemedicine.

Artificial pancreas.

Trying to get patines to wear sensors

New technology, have to ask what will adherence be?

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Science of metabolism

Control of body weight.

Quantitiative bioenergetics.

Obesity epidemic took off in 1980. Type 2. Obesity a major cause of type 2.

Eat less exercise more. 5 critical hormones that tell brain to increase or decrease
amount of food you eat and amount of calroies you born or not. When u restrict your
hormones, satiety hormones are decreased. Made in the intestines.

Leptin. Peptide yy, insulin, amylin, cholescstokinin

Ghrelin. Grumbling hunger hormone. Gets turned up when u lose weight.

Fidgeting is inerhited. You can lose weight.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Can burn up to 50 calories per day expelling gas.

To lose 5 to 10 percent of bodyweight cut 120 calories per day.

Carolimetry. Room that meausres oxygen in and out. Can tell what calories burning
and if fat or carbohydrates. When u reduce you burn less calories become more
efficient. But hunger hormones increase so very hard.

Abdominal body fat is harmful. Fat on hips and thighs good for health.

Fat cell in stomach vs. fat cell in hips and thigh regions.

Epigenetics. Modification of gene sequence w/changing it

Gene sequence is highlighted in tummy fat and redacted in other fat

Steven.r.smith@msomething hospital.

diabetes technology

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

kudya.yogish@

nobel prize for insulin awarded 1923

last 12 years can show info about glucose in real time. Medtronic ipro 2. Continuous
glucose monitors.

Dexcom g4 plat pro

Cgm plus glucose pump

Medtronic 670G- artificial pancreas

Medtgronic 670G. clinical practice sue started march 2017. Was approved last
September. Insurance coverage evolving.

4 nih funded studies.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Bionic pancreas. Boston

Stem cell based insulin producing

----------

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.

Tara Haelle. Translating Medical Research Into Journalism

2 types of clinical studies

Clinical trials. Used to measure an intervention. Interventional studies

Drug devices, prods procedures, screenings, behavior. Can provide evidence of

causation

2) epidemiological/observational studies. Coffee and chocolate and wine oh my

Phase 0: exploratory. Behave in humans as it does in animals?

Phase 1: test intervention in small group for safety, dosage range, side effects,

Phase 2: larger group for safety and effectiveiness

Phase 3: larger groups. Side effects, compare t other agents.

Phase 4: post licensure.