A Mother’s Story: Coping with a Sick Child

Young MPN patient “Jedi” with his companion Chewy

In the opening credits of the television show “The Fresh Prince of Bel Air” Will Smith sings, “This is the story/all about how/my life was turned/upside down.” This article is the story all about how OUR lives were turned upside down when our son, who we affectionately refer to as a “Jedi” because of his special blood, was diagnosed with a Myeloproliferative Neoplasm (MPN).

Our story is probably different from other adults/children with an MPN because Jedi wasn’t extremely sick before we discovered he had an MPN. For about two years, Jedi had experienced a variety of unexplained health issues — random fevers, flu, an estimated seven times over the preceding twelve months, and extreme pain in his legs. At the time, I attributed these conditions to allergies, or growing pains, things that boys normally experience at that age. This soon changed, however, when I took him in for his annual physical exam. The doctor suggested a blood test for Jedi after hearing about his recent health issues. I am not one who immediately agrees to testing, but I agreed when the doctor said to me, “if it was my son, I would do the test.”  A week later, the doctor called and told me the blood test was contaminated and to immediately re-test Jedi, which we did. Two weeks later, I received a phone call from him, who told me he believed Jedi had Essential Thrombocythemia (ET), a condition I had never heard used before. His doctor then recommended we see a specialist who specialized in ET. It can be hard to diagnose a child with a MPN, because it is so rare. However, the doctor was a General Practitioner and had seen it in other adults. Thus, Jedi didn’t get extremely sick before diagnosis as so many of the children do.

The following Monday, I called the recommended specialist. The scheduler answered the phone by stating the name of the organization, which was “something something oncology.” Her words, more specifically one word – oncology – startled me. I held it together long enough to explain who I was and to ask for an appointment. Once the call was completed, I hung up the phone, closed my door and cried. Oncology? Why was an oncologist being recommended to see my beautiful eight-year old?

The results of Jedi’s first bone marrow biopsy revealed he had the JAK2 gene mutation. Jedi asked what a gene mutation was. I frantically tried to explain a gene mutation to my child. My first instinct was to tell him he was a mutant. I knew his next question was going to be, “What is my superpower?” I didn’t have an answer for that question, but realized he is like a Jedi, who has midi-cholorians, or special blood. That is what I explained to him.

Pediatric MPN Specialist Dr. Nicole Kucin, MD, MS, New York Presbyterian Hospital/Weill Cornell Medicine. 

After initial difficulty finding a specialist who understood MPNs, we now have a talented team of specialists. He sees a local doctor every month. He also sees Dr. Nicole Kucine, MD, MS, an MPN specialist with Weill Cornell in New York City, click here to learn more. Dr. Kucine is performing a study on children with MPNs through the National Institutes of Health (NIH). If you have a child who has an MPN, I highly recommend contacting her. Last, Dr. Srdan Verstovsek (aka “Dr. V”) who is affiliated with MD Anderson in Houston, Texas, is part of the team. He is an Adult MPN specialist but performs a lot of work related to the JAK2 mutation. He has agreed to consult with Jedi’s Pediatric Hematologist, Dr. Michael Rytting, who is also at MD Anderson. As a result of Dr. V and Dr. Rytting’s recommendation, we have changed Jedi’s treatment plan from Hydroxyurea (HU) to Interferon.

In determining the appropriate treatment plan, some questions we asked were:

What are our options for treatments? What is the difference between each treatment? Are there timeframe limitations for each treatment? (The effectiveness of one of the drugs used to treat ET is limited to 5 years. That was information I did not know but extremely important to know given Jedi’s age – now nine years old!)

Are there any other patients using this same treatment? Have they experienced any side effects not listed on the medicine? Is the basis for our understanding of how this treatment affects the patient based on a different disease? For example, HU is commonly prescribed to patients with sickle cell anemia. Some doctors’ understanding of how HU impacts a patient is based on the their patients who have sickle cell anemia, which is a completely unrelated disease.

Additional questions to consider:

Is there a way to mitigate side effects?

Can we start with a lower dose and see if it works?

How long will it take for the medicine to start working?

What are the risks of not taking any medicine? What are the risks of taking this medicine?

Is there any research being done on these treatments?

In finding a local doctor, some questions we asked are:

Are you willing to work with other specialists in this field of medicine?

How do you propose to communicate with them?

Are you willing to follow the specialist’s instruction when treating my child?

Are you willing to consider diet as part of the treatment?

There a few ways to connect with other with MPN patients. I have gotten great information from Facebook support groups. Attending MPN conferences is another way to become informed and connected. We attended an MPN conference in February. Listening first-hand to specialists providing updates in the field and answering questions was like drinking from a firehose. Thankfully, MPN Advocacy & Education International posted the videos on the website, which allowed me the opportunity to repeatedly watch them to fully absorb the information the specialists provided, view conference videos. Being able to converse with the attendees at the conference was also extremely helpful. They shared their first-hand experiences and provided insight into what my child is going through. It is more difficult for a child to describe how he or she feels because what he or she experiences on an everyday basis is their “normal.” By sharing their experiences with me, the attendees were able to help me find the words to help my child describe how HE is feeling.

Part of this disease is a feeling of loneliness – for Jedi, Jedi’s brother (our other son), and my husband and me. Unlike more common disorders, finding and becoming part of a support group can be difficult for those with an MPN—especially since it is so rare in a child. That is why it is important for us to participate in conferences whenever possible. This Fall MPN Advocacy & Education Int’l is hosting a conference especially for children and young adults with MPNs. This is a fantastic opportunity for both parents and children to meet and get to know one another. We plan to attend this conference. Learn more about the Pediatric MPN event.

Finally, a plea to adults with an MPN. Please consider using the resources the MPN groups has provided, such as the tool that tracks symptoms. I know it can be concerning to share that information with a third party. (Believe me, I am wary of doing that myself.) But, any information YOU provide will help those that come behind you. Working together, we can collectively help each other and future generations better understand how to combat and defeat these diseases.



FDA Approve Investigational New Drug Application for CK0801 for Bone Marrow Failure Syndromes

HOUSTON, June 10, 2018 /PRNewswire/ — Cellenkos™, Inc., a clinical-stage biotechnology company, today announced that the United States Food and Drug Administration has cleared the Investigational New Drug (IND) application which allows for Cellenkos™ to proceed with a phase I clinical trial of CK0801, 3rd party cord blood derived regulatory T cells, in patients with bone marrow failure syndrome including aplastic anemia, myelodysplastic syndrome and myelofibrosis.  Cellenkos™ submitted its IND application on May 11, 2018.  The Phase I clinical trial of CK0801 will commence in the third quarter of 2018 at The University of Texas MD Anderson Cancer Center, Houston, Texas.

The clearance of this IND is a significant milestone for Cellenkos™ and for its’ platform technology, CK0801, as part of the overall clinical development plan of demonstrating adoptive cellular therapy with CK0801 to treat autoimmune diseases and inflammatory disorders”, said Founder and Chief Medical Officer, Simrit Parmar, MD.  “We are excited to launch the development of CK0801 as a novel cellular therapy approach to treat bone marrow dysfunction arising due to irregularities in the patient’s immune system.  CK0801 works to replace and replenish the defective regulatory T cells and blocks the uncontrolled inflammatory signal generated by the cytotoxic T cells against patients’ own tissues. Such an approach may lead to calming of the bone marrow inflammatory microenvironment as a result of the infused CK0801 which may then lead to the reversal of the hematopoietic arrest that will potentially translate into clinical improvement.”

Tara Sadeghi, Senior Clinical Director, added, “The US FDA’s clearance of this IND validates our commitment towards creating an ‘off-the-shelf’ cord blood derived regulatory T cell therapeutic platform for treating autoimmune diseases.  We are confident and eager that this Phase I study will confirm the safety of CK0801.  We remain grateful to all our colleagues and partners in academia, whose constant support and guidance allows us to take this step forward.”

About Bone Marrow Failure Syndromes
Bone marrow failure syndrome refers to the decreased production of one or more major hematopoietic lineages (white cells, red cells and platelets), which leads to diminished or absent hematopoietic precursors in the bone marrow.  Bone marrow failure remains an incurable disease except for those patients who are eligible to receive an allogeneic stem cell transplantation.  For a vast majority of these conditions, the role of immune dysfunction and increased inflammation in the bone marrow is increasingly recognized as important in both the inception as well as maintenance of the defective production of blood cells.

About CK0801
CK0801, is a first-in-class, cord blood-derived T-regulatory cellular product, developed using Cellenkos‘s proprietary manufacturing platform, that overcomes immune dysfunction by inhibiting key regulators of inflammation.  CK0801 contains healthy and robust regulatory T cells derived from donor cord blood units and adoptive therapy with CK0801 has the potential to replenish the immune system and impact patient care by providing novel, non-pharmacologic options.  CK0801 will be manufactured at the Cellenkos FDA registered manufacturing facility located in Houston, Texas.

Read more

From Cleveland to Pittsburgh: A Busy Travel Schedule Worth Every Minute


Top left, Dr. James Rossetti, Pittsburgh Support Group Coordinator, Jean Diesch, with Ann Brazeau, Dr. Salman Fazal with Dr. John Mascarenhas and Dr. Raajit Rampal.

On June 7, MPN Advocacy & Education International held its first MPN Patient/Caregiver program in Pittsburgh. Support Group Coordinator, Jean Diesch, invited us many times and we were finally able to make it happen. Two local hematologists, Drs. Fazal and Rossetti joined Drs. Rampal and Mascarenhas for this program.

Dr. Rampal, Memorial Sloan Kettering, presented on new therapies in MPNs, The good news is the development of new JAK inhibitor therapies on the horizon for MPN patients. Click here to view new clinical trials.

Dr. John Mascarenhas, Mount Sinai, discussed new polycythemia vera therapies. Comparing the goals of PV patients to feel better, and the goal of investigators to take what they are doing in the lab and bring it to the patient. Dr. Mascarenhas believes clinical trial participation is the pathway to a cure. This resulted in an excellent discussion with the patients on what they can do to help. Dr. Mascarenhas urged patients to consider donating to a tissue bank. Mount Sinai has a donation program in place, click here to learn more.

Dr. James Rossetti, UPMC Hillman Cancer Center, gave an overview of bone marrow biopsies and aspirations, including how they are done, what is extracted and why, and what they can learn. Many patients have multiple biopsies over the course of their disease.  Biopsies are helpful in creating a baseline for assessing the progression of the disease. (There is a new method being researched to produce non-invasive biopsies, click here to learn more.)

Dr. Salman Fazal, Temple University School of Medicine, reviewed the findings from the MPN Landmark Study. This study highlighted the differences in treatment goals and perception of symptom burden between patients and the hematologists/oncologists who treat MPN patients. While patients listed slowing the progression of the disease as their top priority, physicians listed the reduction in symptom burden, thrombotic events, as their primary goal. For many physicians, this offered unique insights into a patient’s mindset. To learn more about the outcomes of this study click here.

From Cleveland to Pittsburgh: A Busy Travel Schedule Worth Every Minute


MPN Advocacy and Education International hosted its 3rd MPN Patient/Caregiver program at the Cleveland Clinic on May 31. Dr. Aaron Gerds presented information on two MPN topics, Symptom Burden and MPNs 101.

Dr. Gerds helped explain the biology of MPNs and likened the JAK2 mutation to a broken thermostat. In this case, constantly triggering the body to turn up the heat (see slide). His discussion on symptom burden provided a keen insight into why patients endure extreme fatigue, itching and other constitutional issues. Dr. Gerds recommended reviewing the yoga study. The majority of patients who participated found some level of relief and improvements in their quality of life. (read more about the yoga study)


Dr. Betty Hamilton, Cleveland Clinic, presented on bone marrow transplants for MPN patients and how to determine the optimal time to proceed. Are the current treatments reducing the symptom burden? Is the patient healthy enough to recover? It is a matter of finding that point at which it will maximize survival and improve the patient’s quality of life. It isn’t age as much as the overall fitness of the patient. One of the most common issues for patient’s recovering from transplant is graft vs host disease, which affects more than half of transplant patients.

Dr. Kristen Pettit, University of Michigan, gave an overview of clinical trials and the benefits of participation. Patients who participate receive quality care from MPN experts.  The protocols in place are designed to ensure the utmost safety of the patient. Participating in a clinical trial is a big commitment. Patients should be aware of all the necessary requirement.

Click here for a list of questions to consider before participating in a trial

Click here for a list of current mpn clinical trials


Dr. Naveen Pemmaraju, MD Anderson, discussed what is on the horizon for MPN patients. He encouraged physicians to pay attention to what patient’s are dealing with beyond the visible symptoms. “No one understands the war going on inside your body.” MD Anderson is currently working on several clinical trials for MF patients:

Evaluation of Ruxolitinib in Combination With PU-H71 for Treatment of Myelofibrosis.

Testing of SL-401 in Advanced, High Risk MPN Patients


From left, Dr. Aaron Gerds, Dr. Naveen Pemmaraju, Dr. Kristen Pettit and Dr. Betty Hamilton

Structure of protein pair provides blueprint for future drugs

Walter and Eliza Hall Institute researchers have visualised for the first time how the protein SOCS1 ‘switches off’ cell signalling to dampen immune responses and block cancer growth.

The atomic-level structure of SOCS1 binding to its partner protein JAK could guide the development of drugs that alter disease-causing cell signalling pathways, and may have applications for treating some blood cancers, including leukaemias.

The research, led by Dr Nick Liau, Dr Nadia Kershaw, Associate Professor Jeff Babon and Professor Nick Nicola, was published in the journal Nature Communications.


– The SOCS1 protein binds to JAK proteins to ‘switch off’ cell signalling, which dampens processes including immune responses and cancer growth.

– Our researchers have used structural biology to visualise how SOCS1 binds to JAK proteins in never-before seen detail.

– The detailed structure may guide the development of new drugs that modify JAK activity, amplifying or dampening cell responses, with potential applications in cancer therapies.

Learn more about clinical trials


Read more

MPN Explanations in Multiple Languages

MPN Advocacy & Education International’s website can be translated into multiple languages.  From the home page, select the language from the drop down menu in the upper-right hand corner.

Below are brief explanations of MPNs by MPN Specialists in various languages.


Ver la explicación del Dr. mesa de MPN en Español

Para ver el sitio web en Español Haga clic en la opción de idioma en la esquina superior derecha del sitio web


الدكتور الجنادي ، رئيس قسم أمراض الدم ، جامعة ولاية ميشيغان ، يقدم شرحًا موجزًا ​​عن الأورام النقوية (MPNs) باللغة العربية.

Video Presentations: San Antonio Patient/Caregiver Program 2/24/18

MPN Advocacy and Education International hosted its very first patient and caregiver program in San Antonio, Texas on February 24. Dr. Ruben Mesa, our scientific advisor and frequent speaker, initiated and assisted with this program’s agenda. Drs. Mesa, Michaelis, Verstovsek, Scherber, Mascarehnas, Gotlib, Palmer and Lyons presented to a packed audience. We will return to San Antonio in 2020.

Click the video screen below to view each presentation

Ann Brazeau, CEO, MPN Advocacy & Education International

Dr. Jason Gotlib, MD, MS, Stanford University Medical Center

Dr. Laura Michaelis, MD, Medical College of Wisconsin/Froedert Hospital


Dr. John Mascarenhas, MD, Icahn School of Medicine

Dr. Srdan Verstovsek, MD, PhD, MD Anderson Cancer Center


Dr. Jeanne Palmer, MD, Mayo Clinic


Panel Q & A


Dr. Robyn Scherber, MD, Mayo Clinic, Arizona


Dr. Ruben Mesa, MD, UT Health San Antonio Cancer Center

A Veteran’s Story: The Frustrations of Filing a Claim with the VA

By Wayne E.

MPN Patient and Vietnam Veteran Wayne E.

I served in the USAF Security Service, 6924th Security Squadron, stationed in Da Nang, Vietnam for one year (1970-1971) and was exposed to the deadly Agent Orange/Dioxin. In 2007, after a simple pre-op blood test, I was diagnosed with essential thrombocythemia (ET). Upon further study I was told I had an incurable, but manageable, blood cancer, coupled with a gene mutation (JAK2). The word cancer scared me. I had never heard of ET and I was at a loss for what to do. I didn’t know where to go next. After much reading about these potentially deadly diseases, I found out I was one of many Vietnam Veterans who had an MPN.

In 2011, I filed my first claim with the VA. Until this filing, I was unable to get any substantial information from my primary care physician (PHP) or my hematologist/oncologist, as to what may have caused or contributed to my ET. They knew virtually nothing about Agent Orange. I contacted the National Institutes of Health, The Centers for Disease Control, and as many online medical sites as possible, all ending with a bigger question mark. Nothing could be explained to satisfy my inquiry.

It was by chance that I connected with a most remarkable group, MPN Advocacy and Education International. I could never thank them enough for the compassion and the understanding they extended to me.

After my initial rejection from the VA, I filed three more times and each time I was denied because MPNs are not on the “presumptive” list of Agent Orange-related illnesses. The same message I kept getting was I needed “clinical rationale” to support my claims. My doctors have not been able to provide me with this needed information. I don’t know what to do today. I understand there are many Vietnam vets that have won their appeals and now get benefits, but there are many others who were not approved and just gave up. I don’t plan to give up.

To my fellow Vietnam Veterans who may be dealing with one of these MPNs, don’t give up. If you have been denied, file an appeal. There is hope, comfort, and assistance available. With the help of MPN Advocacy and Education International.

 Learn more about filing a claim with the VA

 Learn more about Veterans and MPNs

A Physician’s Perspective on the use of Social Media

By Dr. Naveen Pemmaraju, MD, MD Anderson Cancer Center

“The forum of social media is for information that is meant to be a general outline, not personalized therapy for an individual.”

For physicians who are balancing a heavy workload, how do you view the role of social media?

I think at least a working knowledge of social media usage is essential in today’s modern era of information. I know many hematologists and other physicians who get a good chunk of information for the day from social media such as Twitter, including myself. In five minutes, one can quickly glean important items for the day in the hematology/oncology-specific areas of interest. These posts can be “liked,” saving it for later reference when more time permits, serving as a personal bookmarking tool. Dr. Mike Thompson, my own Twitter mentor, published two really nice primers on this exact topic, and they provide a nice guide to getting yourself involved:

Twitter 101 and beyond: introduction to social media platforms available to practicing hematologist/oncologists.

Social Media and the Practicing Hematologist: Twitter 101 for the Busy Healthcare Provider.


What are your thoughts on the use of social media for patients? Pros and Cons? 

We live in an era of readily accessible information. Overall, this is a wonderful thing for our patients and providers alike. With this, it means we have information readily available, 24-7 at our fingertips. This includes online formats in the newer platforms such as social media.

For patients the pros include the ability to see up-to-the-minute information and announcements from key opinion leaders and professionals in the MPN field via twitter using #MPNSM (this is a grassroots twitter community started by me  along with other academic investigator); the access to online support groups such as on facebook for interaction, support, group discussions, information, comfort, and caregiver help; the ability to contribute one’s own valuable experiences to the public using blogs, websites, private facebook groups. (go to MPN Advocacy’s twitter page)

On the con side, one must understand that what you post comes great responsibility. Myself and others have written in the medical literature about the potential pitfalls and unknowns in this space. As with any form of media, we all need to be very careful and mindful of what we post (all is archived!), and that we treat others with the utmost respect. We must always remember to obey HIPAA and other privacy rules /laws. One must be vigilant about misinformation and be ready to identify and report spam, and any incorrect postings.

There are many different ways a person can participate on social media, from using it to gather news, information and opinions, to re-tweeting posts (twitter) or sharing (fb) items that resonate with an individual. Ultimately, one must be comfortable enough to post original content. Take your time, follow others on topics you care about, and assess and re-assess what sources are providing you with relevant, helpful information.

How do you and should you advise others on what they post on social media?

One rule I try to follow and advise others to follow is to remember to keep things accurate and keep things general. What I mean by this is always strive to give authentic, truthful accounts – whether it is about your impressions from a meeting, detailed scientific analysis, or experiences. Remember, all items on social media are archived-they are forever. Refrain from giving specific medical advice for specific situations. Instead, direct patients to make a formal appointment.

Dr. Pemmaraju has been a pioneer in the early stages of developing social media and its interface with hematology/oncology. We are pleased to have him offer his insights on the use of social media for both patients and medical professionals.
Dr. Pemmaraju will be joining us at the MPN Patient/Caregiver Program in Cleveland, learn more.