Translational Research: From the Lab to the Clinic

By | April 6, 2016
Marina pipette

Don’t worry! I’m not a real scientist!

I had the coolest field trip today. I visited the Mullally lab, an independent research lab at Brigham and Women’s Hospital, Department of Hematology, with Harvard Medical School. It is a translational hematology research lab. Dr. Ann Mullally and her team of impressive scientists test theories to answer some of the pesky questions people living with  myeloproliferative neoplasms (MPN) ask their hematologists: Why do the mutations occur? Why do the diseases change? How can we fix them?

Shannon Elf, Alex Peed, Marina Peed, Nouran Abdelfattah, Natalie Florescu

Dr. Ann Mullally is a physician researcher, walking the roads of clinical patient care and scientific research. She also takes time to educate the MPN patient community by presenting at patient conferences and symposia. When I sent her an email asking if I could visit her lab during my trip to Boston, she responded right away and welcomed me to meet with Shannon Elf, PhD, and Nouran Abdelfattah in her absence.

Dr. Mullally also sent me the latest research paper published by her team (March 7, 2016) entitled “Mutant Calreticulin Requires Both its Mutant C-terminus and the Thrombopoietin Receptor for Oncogenic Transformation” (Cancer Discovery, American Association for Cancer Research). I read it six times.  I highlighted and underlined key phrases. And I created a list of questions, just in case Shannon or Nouran decided to quiz me.  They didn’t. And I exhaled.

Over the years of living with polycythemia vera and myelofibrosis, I attended 6 conferences for patients living with myeloproliferative neoplasms and numerous support group meetings where the latest science and treatments were shared.  At each event, physicians and researchers answer some of our MPN questions with, “unfortunately, the science isn’t there yet.”

So it is no exaggeration to say that my life and many others depend on scientists who choose to commit their talent and lives to research the intricacies of our incredibly complex cellular biology.  I want to meet them. And thank them. And encourage them. And learn how support them, their work, the field.

At the Mullally Lab, I got to meet actual scientists who wake up every morning thinking about the intricacies of blood behavior.  Not like us patients, who compare CBC values and allele burdens. These women are SCUBA divers who go deep into the bone marrow to study the intricacies of stem cells, gene mutations, signaling, pathways, and receptors. They also study the microenvironment in which the mutations occur including cytokines and inflammation.

There is a lot of talent in this lab!

There is a lot of talent in this lab!

Shannon Elf is a post-doc fellow who is presently studying CALR (calreticulan) mutation. Sarah Rozelle is a post-doc research fellow who studies myelofibrosis. Renata Grozovsky is post-doc researcher and instructor who studies platelets and their complex clotting cascades.   Nouran Abdelfattah is a research associate who is the CRISPR/cas9 queen. (She loves to edit genes). Amy Ko is a research associate and she conducts flow cytometry and western blots and is also a mouse master; she explained how the mice are cared for and the tip of the tail can be snipped to collect the DNA.  Natalie Florescu is a research associate who was working on a western blot while I was visiting. I met more great scientists and regret that I didn’t write all their names.

I shared a bit of my experience with MPN and stem cell transplantation. Because of my “complicated case” experience with thromboses, PV, splenomegaly, MF, marrow failure, transfusion dependence, and allogeneic stem cell transplantation, each researcher’s focus felt deeply personal to me.  I told them that there are thousands of people who are where I was (health wise), and we are grateful and hopeful that they help science catch up with us.

The “basic science” conducted in research labs like this one is essential for humanity. They expand the foundation of knowledge. Facts take time to establish and confirm. In this age of instant gratification, basic science may seem slow and arcane to non-science people.  But it isn’t opinion or hunches or intuition. A lot of time, energy, and money go into expanding our knowledge base. Experiments fail until they succeed.

Mouse spleens

Mouse spleens

Theories are tested repeatedly, findings are reviewed and corroborated before facts are accepted.  The beauty of science is that it belongs to all of us. What is learned here contributes to research and discoveries in other places.  Treatments are developed based on basic science.

The women in this lab come from different places and life experiences. They have varied academic backgrounds (despite the similar sounding topics to this old political science student). What they share is a deep curiosity for understanding blood, a fearlessness to try experiments and learn from failures, and a tenacity to push the edge of knowledge further every year. And student loans. And plans and dreams for their personal lives.

Scientific Research – Did you know?

I asked them what people like me might be surprised to know about medical/scientific research.  They said they experience a lot of failed experiments before they strike upon success that lead to discoveries we read in research and medical journals.  It can be very discouraging to work hard on a theory that seems very sound, only to have it fail. Those who invest in research are eager for discoveries, and those discoveries take time and money.

The mice (I asked):  lab mice are very expensive to procure, maintain, and test. The use of mice is highly regulated and compliance is quite detailed. Lights are on from 7 am – 7 pm where they are kept (no night time experiments); type and frequency of litter, food, and water is specific and monitored; mice must be handled in ways that minimize stress and discomfort; and each experiment requires a string of reviews and approvals.

Research is expensive, but not because the researchers are in the top tax brackets. It is highly regulated work and rent, equipment, tissues, and mice are costly. One joked the rent for the mice is greater than the lab space for the researchers.  Therefore, the quest for grants is continual. This caused flashbacks to my days as a nonprofit executive director: always raising money to keep our programs going.

I know our future is in good hands. And I will limit my science experiments to my kitchen.

Cheers!

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