Celebrating a journey of family, hope, scientists and philanthropy to treat myotubular myopathy in Phase 1 clinical trials, Part 1

Dr. Martin ChildersIf you are a parent you already know the guts, fortitude, desire and village it takes to raise a child from milestone to milestone to adulthood.  Imagine your child is born with a fatal genetic disease affecting the function of all his skeletal muscles.  Meet Alison and Paul Frase, their daughter Isabella, and finally their son Joshua who was born with myotubular myopathy.  If you remember anything about their story, I want you to remember the power of passionate parents working with brilliant, dedicated scientists who tenaciously act and hope for a cure through biomedical research.

The genetic villain in the story is the MTM1 gene, where mutations cause total loss of function in the myotubularin gene.  This gene normally makes an enzyme, myotubularin, which is thought to create and maintain muscle cells.  Kind of important. So when the gene cannot make this enzyme, people (and animals like dogs) experience muscular “floppiness,” breathing issues, feeding issues and scoliosis to name a few.

If you are a scientist, like Dr. Martin Childers, you also know the guts, fortitude, desire and village it takes to conduct research…especially research you hope will treat devastating genetic diseases like the one that affected Joshua.

The Frases, Dr. Childers and other folks have partnered to do something about myotubular myopathy in animals and people.  NWABR is honoring their work at the Speak Up for Research Gala this year.  Want to know more?  Stay tuned for the May newsletter and register for the Gala on May 24th.

The War on Drug Prices

Cost of Drugs_cartoon
Most Americans ask the question, is the price I pay for prescription medications FAIR? In other words, what is the true cost of developing, making and selling a medicine, AND how much profit seems reasonable? This is what we tackled last week during our Community Conversation with experts from the U of Washington’s International Society for Pharmacoeconomics and Outcomes Research (ISPOR).

Tufts estimates that it costs $2.6 billion to develop an FDA approved drug, a number that increases 9% per year.  People in the US pay more for drugs than people in other countries for a few reasons: 1) we use more medications because we have better access to new ones, 2) we have a higher burden of chronic disease like diabetes and obesity, and 3)  in the US, the government protects drug manufacturing monopolies and limits price negotiations while other countries have more price regulation.

Another reason people in the US (and in Canada and Europe) pay more for medications is simply because, on the whole, we can afford to. Picture a world map, and picture all the people who live on Earth.  At 326 million, the US population is 4.4% of the world’s 7.5 billion people, but the US accounts for 50% of all medication sales in the world. Mind blown.

We are looking for VALUE in our medications.  We want medications that are novel and offer leaps of improved quality of life.  We are willing to pay more for medications that achieve this high bar, but there is a point for every person where price will exceed our ability to purchase a medication.

Finally, we learned about the trade-off between innovation and access.  Monopoly protection funds innovation. In turn, industry creates more new drugs in the long run that cost more money in the short run and poorer access. “But if new medicines aren’t invented, then no one can access them in the long term.” (ISPOR)  How can the market incentivize innovation if not through the almighty dollar?

Many in the room were for the first time faced with the reality that the US foots HALF the bill of medications in the world.  I don’t think anyone, besides the facilitators, was prepared for this data.  As the discussion dug deeper, we realized that our drug costs fund innovation of new medications and devices.  Despite the relationship between US drug prices and world drug innovation, many participants still did not think it FAIR that people in the US pay more than those in other countries.

Some held high the banner of altruism and seemed happy having the US play this role.  Others felt perhaps that even within the US that medication prices should be on a sliding scale based on what a household could afford.  Some shared personal stories about themselves or people they know who have had to choose between paying for their medications and paying other bills.

Yet others remained firm that prices could drop while maintaining innovation if pharmaceutical companies could lower their profits to reasonable margins.  Some attendees implied that companies could reduce their expenses by accelerating the clinical research process, a point that was strongly opposed by a former industry employee.  Pharmaceutical companies have no interest in accelerating clinical research in this manner because it would cause less confidence in drug safety.

As we were wrapping up our Conversation, we faced the question of whether or not the way we finance medications is sustainable.  Our vocal attendees had faith in the free market to correct anything that was broken.  Others remarked that the balance of medications on and off patent would also help with price competition once medications go off patent.

Our ISPOR facilitators emphasized that drug pricing is a complex ecosystem.  They recommended that consumers, scientists, policy-makers and health care providers and administrators continue to learn and share their perspectives, in venues such as Community Conversations, as much as possible in order to find a stable and sustainable relationship between medication access and innovation.

To read some excellent resources on the topic, visit the Community Conversation archive and scroll until you find the “war on drug prices” topic.


Speak Up For Research Education Fund

How did science hook you?

Strawberry DNA extraction

Strawberry DNA extraction

Did you have a teacher whose lab was your favorite place to hang out in high school? Were you a biology graduate student who fought for better treatment of animals and found a calling in animal care or health ethics? Did you travel and see suffering that would be diminished with the right vaccination?

I got hooked as a member of the St John Ambulance Brigade in New Zealand where as a teenager I was able to volunteer in ambulances, hospitals and rest homes and saw evolving treatments driven by research.

I’m both excited about science and concerned about its future. I see a growing distrust in biomedical research, waning science literacy and an almost perverse celebration of anti-science sentiments; this all of course at a time when new biomedical research breakthroughs are occurring on a daily basis. Here at NWABR, we see the possibilities of science and are excited by the opportunities for young people to get hooked into fascinating and important science fields—but we also see a gap in the science education for the general public that results in twisted logic, misinformation, hijacked conversations and bad policy decisions.

NWABR bridges that gap, combats that misinformation, and leads spirited and informative conversations about complex issues related to biomedical research.  And we need your help.

Today, I’m asking readers you to join our newest fundraising initiative: the Speak Up For Research Education Fund.  Over the last two years more that 1,400 people have joined NWABR at a series of events:

  • Perhaps they volunteered for our popular Bio Expo that engaged close to 700 high school students;
  • Or they attended a Community Conversation on the ethics of end of life care, or vaccinations, or direct to consumer genetic testing.
  • Perhaps they are a professional dedicated to ethical protections for humans and animals in research and you attended one of our research conferences;
  • Or they attended our Security Conference and joined colleagues from across the country who are committed to keeping scientists, their facilities and their work safe.

This campaign to create a Speak Up For Research Education Fund is about protecting the belief and trust in biomedical research and ensuring that this work can continue robustly into the future.

Join the Speak Up For Research Education Fund and make a donation today by visiting:  https://donatenow.networkforgood.org/nwabr?code=Speak%20Up%20For%20Research

Alternatively you can send a contribution to the Northwest Association for Biomedical Research, 2633 Eastlake Ave E., Suite 302, Seattle WA 98102.

All supporters will be thanked by name in our public materials, unless of course they request to make an anonymous contribution.  All contributions will also be acknowledged with a tax deduction receipt.

This is a vital time for science – with the support of the Speak Up For Research Education Fund we can continue and expand the work of engaging students, families and communities with science.  With the support of this fund then one student at a time, one family at a time, one community at a time we will build support for, and trust in, biomedical research.

Thank you for Speaking Up For Research.

Kind Regards


Ken Gordon – Executive Director

Northwest Association for Biomedical Research

(P) 206-957-3337 (C) 206-595-2450



Our Bioteh Future

Good morning all

In all of the election coverage over the last few days you may have missed this guest editorial in the Seattle Times from H. Stewart Parker.  Ms. Parker in this Op. Ed. charts the development of a number of key biotech firms and products here in the Northwest and talks about the diaspora of staff from Immunex the company that invented Enbrel, a breakthrough drug for the treatment of rheumatoid arthritis.


H. Stewart Parker, MBA

Biomedical research and biotechnology combine to provide 34,200 direct jobs in WA state and a total of 92,400 total jobs once you count all of the ancillary support people (like me).  In total this sector makes up $11.4 Billion of the state’s GDP and provides $7.1 Billion in personal income.

Unfortunately even though this sector makes up the 5th largest sector in the state, with life science organizations in 74 cities – it often feels like the sector flies under the radar screen with people seeing the major economic drivers in WA and the NW as being IT and aeronautics.  Ms. Parker’s Op. Ed. reminds us all of the contribution of the sector – both in terms of economics and also in terms of the life saving changes being brought to us on a daily basis.

Talking about life saving changes – I had the pleasure of attending a fund raising luncheon hosted by the Benaroya Research Institute this last Friday.  They have a very simple mission – they want to cure 80 plus autoimmune diseases, like Type 1 diabetes, Crohn’s Disease and MS.  In one word BRI’s work is – inspiring.  Their new brand of “BRIng it on” is both fun and a challenge to us all to do better.

Have a great day.

Ken Gordon

Executive Director


NWABR Urges Congress to Preserve Biomedical Research Funding

Sequestration could cost WA at least $70 million in grant funding

Now that the election is finally over, it’s easy to be distracted from the ongoing work of the current congress, and the looming threat that budget sequestration poses to the biomedical research industry. The Budget Control Act of 2011 requires that across-the-board cuts to be applied to a large portion of the federal budget on January 2, 2013, unless Congress reverses it. For domestic programs, around $39 billion in cuts would be applied to “discretionary” programs, which include the National Institutes of Health (NIH) and the National Science Foundation (NSF).  According to a United for Medical Research report, if NIH funding is cut by 7.8% as part of budget sequestration, the state of Washington will be hit especially hard, with a loss of 1,184 jobs and $72.2 million in grants supported by this funding. And that is just the beginning.

This week, members of Research!America, a nonprofit advocacy alliance, are headed to Capitol Hill to make the case that sequestration is harmful not just for biomedical research, but also for our economy. As part of their Save Research campaign, NWABR was proud to sign a letter urging congressional leaders to reject any deficit reduction proposals that would cut research funding or hinder incentives that support biomedical innovation.

The full letter is posted below. Please take a moment to reach out to your congressional delegation and ask them to preserve funding that will help combat disease and spur private sector innovation in the Northwest and beyond.

Dear Mr. Reid, Mr. McConnell, Mr. Boehner, and Ms. Pelosi:

As advocates for biomedical and health research, we are writing to urge you to refrain from deploying deficit reduction strategies, like sequestration, that would slow medical progress.

Our nation leads the world in biomedical and health research, a function of public sector support and private sector ingenuity. The National Institutes of Health (NIH) is unrivaled in spurring the basic discovery that lays the path for private sector innovation. Peer-reviewed NIH funding reaches all 50 states and congressional districts, spurring discovery at universities, hospitals, small businesses, and independent research institutes. In fact, NIH-supported research has:

  • Supported nearly 500,000 jobs in 2011 in every state
  • Generated $62 billion in economic activity in 2011
  • Helped increase life expectancy from 47 years in 1900 to 78 years in 2009

But this is not just about NIH.  The National Science Foundation (NSF), Food and Drug Administration (FDA), Centers for Disease Control and Prevention (CDC) and the Agency for Healthcare Research and Quality (AHRQ) all provide a positive return on investment to our nation, protecting American lives and promoting American prosperity.

  • NSF’s grant portfolio is designed to identify and pursue the best scientific opportunities across the spectrum  of scientific disciplines, including biomedical research.
  • FDA is a key conduit between medical discovery and medical progress, laying the path for safe and effective  medical products to reach the marketplace.
  • CDC conducts and supports the public health research needed to contain disease outbreaks, promote wellness, and in other ways provide basic supports for a safe and healthy society; and
  • AHRQ combats entrenched and insidious problems in our nation’s health care system – like preventable medical errors and needless administrative red tape — that take lives and inflate the cost of taxpayer funded health programs and private insurance alike.

Disinvesting from biomedical and health research – and the infrastructure and expertise needed to conduct it – would contravene the goal of deficit reduction. This research is one of the fundamental underpinnings of our economy, a reality well understood by other nations, which are ramping up their investment and building out their research infrastructure.  Research is a catalyst that creates businesses large and small, and generates jobs in research, manufacturing, distribution, exports, health care and a host of other sectors. Those businesses and jobs supply federal revenue needed to reduce the deficit.

In addition, research can help stem runaway federal healthcare spending, which is driving our deficit. While new treatments may require additional cost at the outset, research has shown the offsetting effects of reduced hospitalizations, fewer visits to providers, reduced home care, a reduction in the Social Security disability roles, and improved productivity.  As you well know, the cost of treating diseases like Alzheimer’s, Parkinson’s and other diseases are exploding. There is no high-impact alternative to research as a means of addressing this crisis.

When it comes to the fiscal health of our nation, biomedical and health research are part of the solution, not part of the problem.  Whether it is appropriations policy or entitlement and tax reform, we urge you to discard any proposal that cuts funding or mutes incentives for public- and private-sector supported medical innovation.   Compassion and pragmatism intersect in the decision to do so.

Thank you for your consideration.

NWABR Members Speak Up For Research

These two exceptional editorials from leaders in the Oregon Health and Science University community stand out for their thoughtful approach to advancing public understanding of biomedical research.

Please read, share, and comment:

Youth Ethics Summit 2011 :: Stem Cells :: Science and Ethics

Group photo :: Youth Ethics Summit 2011 :: Stem Cells :: Science and Ethics

On April 9, 2011 the Youth Ethics Summit brought together students from across the Puget Sound region to learn about topics related to ethics, medicine, and biomedical research that are of special relevance to young people.

Presented by NWABR and UW’s Institute for Stem Cell and Regenerative Medicine (ISCRM), this year’s summit focused on stem cells and featured tours, panels, and breakout discussions. The Summit provided an opportunity for students from different schools to meet and to participate in discussions and presentations about ethics in science issues.

Students Say

Students who experienced the Summit said:

  • I learned just how much control we have/might have soon. Knowing where to draw the line isn’t easy, and it’s something we all need to discuss and understand in order to make wise choices as individuals and as a society.
  • We were able to express our own ideas and see what other people thought about them … the discussions we had in our breakout groups were very thought-provoking … listening to different view points on things helped me learn a lot more about them.
  • The tours gave me insight on what real life stem cell research would be like and how it would be to work in a lab in the future.
  • It was absolutely amazing going into three different labs focusing on the application of stem cells, the stem cells themselves, and the use of robots in research. The groups were small, we had the opportunity to look at both embryonic and induced pluripotent stem cells through microscopes, and the researchers were all extremely informative.
  • In the laboratories we toured, I saw myself in the scientist gown, handling the different machines.
  • It was a wonderful learning experience that I would recommend anyone who is interested in bioethics … I loved the chance to meet similar-minded teens in the Seattle-area and talk about this fascinating topic.

Stem Cells 101

We began with a brief presentation of “Stem Cells 101” by Professor Tony Blau, MD, Director of the Institute for Stem Cell and Regenerative Medicine.

“If you took a drop of blood from my finger, put it on a glass slide, smeared it and stained it and looked at it under the microscope, you’d see different types of cells, including what?” Blau asked. Hands shot up, and Blau took three fast answers, one each from three students: white blood cells, red blood cells, platelets. “And they would look obviously different from each other,” Blau continued, describing what each looks like under a microscope, “but they all come from the same mother cell, a stem cell.”

Dr. Tony Blau

Dr. Tony Blau, Professor of Medicine, Hematology, Adjunct Professor of Genome Sciences, and Co-Director, Institute for Stem Cell & Regenerative Medicine, University of Washington School of Medicine

In each of us, we might have a trillion cells in our blood, but we have about 10,000 blood-generating stem cells. “Where are these stem cells?” Blau asked and another student answered: in the bone marrow.

The professor next defined leukemia (cancer of the blood or bone marrow) and one life-saving treatment for it, dependent on stem cells and developed “next door” at the Fred Hutchinson Cancer Research Center. Stem cell transplantation with bone-marrow-derived stem cells was led by Dr. E. Donnall Thomas, whose work was recognized in 1990 with a Nobel Prize.

Dr. Tony Blau

Dr. Blau explained the basics of hematopoietic stem cell transplantation, of regenerative medicine as studied at the ISCRM, and he introduced what we would see for ourselves, next — in tours of several research labs on campus at UW South Lake Union.

Tour One: Tony Blau Lab – cancer biology and stem cells

There are about 500 researchers at UW South Lake Union. Neighbors include the Seattle Cancer Care Alliance, Seattle Childrens’ Research Institute, Seattle Biomedical Research Institute, Novo Nordisk, PATH, Fred Hutchinson Cancer Research Center, et al.

Outside the Blau Lab at ISCRM
Upstairs at his lab’s front door, Dr. Blau pointed out a few notable neighbors in biomedical research in Seattle’s South Lake Union neighborhood.
Tony Blau, Chris Miller, and Kyle Rattray of the Blau Lab

Researchers Tony Blau, Chris Miller, and Kyle Rattray of the Blau Lab

Researchers Kyle Rattray and Kathy Davidson at the Blau Lab

Researchers Kyle Rattray and Kathy Davidson at the Blau Lab

Tony Blau Lab - cancer biology and stem cells

Tony Blau Lab - cancer biology and stem cells

Tour Two: Mike Laflamme Lab – cardiovascular research

Professor Laflamme’s lab researches cardiac applications for human embryonic stem cells, including repair and regeneration of ventricular, atrial, and other cells from embryonic stem cells.

Professor Mike Laflamme

Professor Mike Laflamme, Pathology, Molecular and Cellular Biology, University of Washington

Researcher Jay Gantz, UW Bioengineering

Researcher Jay Gantz, UW Bioengineering

Researcher Jay Gantz, UW Bioengineering

Researcher Jay Gantz, UW Bioengineering

Tour Three: Tim Martins, Co-Director of the Quellos High Throughput Screening Core – screening molecules for drug development

Entering the Quellos High Throughput Screening Core

Dr. Tim Martins, Co-Director of the Quellos High Throughput Screening Core

Dr. Tim Martins, Co-Director welcomes us to the Quellos High Throughput Screening Core, full of the robotics and automation which have vastly improved biomedical research with improved speeds for identifying therapeutic drug candidates.

Tim Martins, Co-Director of the Quellos High Throughput Screening Core

Dr. Martins was asked about making mistakes in experiments. He replied "I make mistakes, but I'm not afraid to make mistakes," while explaining failure rate in research and the importance of confidence.

Dr. Tim Martins with ready answers on our tour

Dr. Tim Martins with ready answers on our tour

It isn't *only* high-tech at the Quellos High Throughput Screening Core

Robots! at the Quellos High Throughput Screening Core

Robots! at the Quellos High Throughput Screening Core

Dr. Tim Martins at the Quellos High Throughput Screening Core

Hands-on with Planaria and Play-dough

After our tours and lunch, we enjoyed hands-on activities with planaria and Play-dough — to model human embryonic development.

Dr. Reitha Weeks, PhD, introduces planaria

Dr. Reitha Weeks, PhD, Program Manager for Science Outreach at NWABR introduces planaria

Planaria are “the regeneration experts” explains Reitha Weeks of NWABR — if you separate one worm into 279 pieces, they grow into 279 worms!  Planaria also serve as model organisms for understanding human stem cells.

Plenty of PlanariaPlenty of Planaria

NWABR offers resources for teaching about biomedical research and ethics, including our popular Stem Cell unit with “Plenty of Planaria” to model stem cell function, development, and the complexity of tissue regeneration.

The curriculum is geared towards high school students and available for download free of charge.

Plenty of Planaria

Microscope, camera, and monitor loaned to us by Leica Microsystems, Inc. Thank you!

Plenty of PlanariaPlenty of Planaria

Next up, modeling early embryo development — with play-dough!

Play-dough Egg and Sperm

Play-dough Egg and Sperm

Jeanne Chowning, MS, Director of Education at NWABR

Jeanne Chowning, MS, Director of Education at NWABR leads the activity

modeling embryonic development with play-dough

modeling embryonic development with play-dough

Students in Dawn Tessandore's AP Biology class

modeling embryonic development with play-doughmodeling embryonic development with play-dough

Breakout Groups: Ethical Issues in Stem Cells

After the above activities, we broke out into groups to discuss ethical issues more closely. A few of the groups were photographed, as below. Group leaders and subjects included:

  • TONY BLAU, MD — Stem Cell Treatments: Considering the risks and benefits of testing stem cell treatments in humans.
  • DAVID EMERY, PhD — Embryonic Stem Cells: How far should we go in seeing if they can grow into embryos?
  • ERICA JONLIN, PhD — Savior Siblings: “My Sister’s Keeper” – what if you were a genetic “designer baby” created to save your sick sister?
  • KATHY DAVIDSON, PhD — Embryos and Research – Creation and Donation: Should researchers be allowed to encourage couples to donate embryos?
  • KYLE RATTRAY, MD/PhD Program — Social Justice: Disease Research and Stem Cells: What diseases should be prioritized in stem cell research?
  • CHRIS MILLER, PhD — Knowing Your Future: What Can Your DNA Tell You? How much do we want to know about the relative risks of what potentially lies ahead for us?

TONY BLAU, MD -- Stem Cell Treatments: Considering the risks and benefits of testing stem cell treatments in humans

TONY BLAU, MD -- Stem Cell Treatments: Considering the risks and benefits of testing stem cell treatments in humans

KATHY DAVIDSON, PhD -- Embryos and Research - Creation and Donation: Should researchers be allowed to encourage couples to donate embryos?

KATHY DAVIDSON, PhD -- Embryos and Research - Creation and Donation: Should researchers be allowed to encourage couples to donate embryos?

KYLE RATTRAY, MD/PhD Program -- Social Justice: Disease Research and Stem Cells: What diseases should be prioritized in stem cell research?

KYLE RATTRAY, MD/PhD Program -- Social Justice: Disease Research and Stem Cells: What diseases should be prioritized in stem cell research?

Youth Ethics Summit 2011 was blogged by Brian Glanz for NWABR

Youth Ethics Summit 2011 was blogged by Brian Glanz for NWABR

Please reuse and remix! We share with a Creative Commons Attribution License.

Photography by Mohini Patel Glanz.

Youth Ethics Summit 2011 was presented by:

University of Washington School of Medicine


Northwest Association for Biomedical Research -- logo

This program was supported by a Collaborations to Understand Research and Ethics (CURE), 1R25RR0251131, a Science Education Partnership Award from the National Center for Research Resources. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center for Research Resources or the National Institutes of Health.

Collaborations to Understand Research and Ethics, a Science Education Partnership Award from the National Center for Research Resources at the National Institutes of Health