A Patient Story: The decision to get a Stem Cell Transplant

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Parachuting from a Crippled Plane

 

By Dave D.

My daughter came to Ohio to provide support to me and my wife during my recent stem cell transplant (SCT). In explaining the process to her I used the analogy of parachuting from a crippled airplane and she found it very helpful. I hope that this analogy might also help others understand my experience. Like with parachuting, there are risks there are steps to take. Each step is a small victory, but the ultimate victory is landing safely.

When I was diagnosed with Primary Myelofibrosis in 2013, it became clear that my high-flying airplane (my body) had a problem. It was still possible that it could fly on for years with relatively few problems, but we needed to keep an eye on it. Medications like Jakafi and Inrebic made the flight a bit more pleasant, but blood counts and bone marrow biopsies indicated that we were losing altitude.

This December I realized through consultation with Dr. Aaron Gerds from the Cleveland Clinic that the plane’s problems were becoming unmanageable. I was now High Risk and I was headed for a fatal crash sooner rather than later. It was difficult to say just how long it would take – but our calculator (MIPPS70) predicted about 5 years – give or take.

At that point I needed to decide whether I would take the risks involved in jumping out of the plane or choose to die in the crash. In consultation with my dear wife and my medical team and with the prayers of my friends and family we considered my options. I decided to make the jump.

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Some people cannot find a good donor match – or they have physical problems that would make SCT pointless. But in my case, everything appeared promising. The team at the Cleveland Clinic put me through tests that showed I was relatively fit. They found multiple 10/10 unrelated donor matches for me. We received necessary insurance approvals. We received logistical support from our family, church family and friends for help throughout the process.

Finally everything was in place. I was giving up on the old airplane that was certainly failing and entrusting myself to the parachute for a safe descent and landing.

  1. I got my final approval to go ahead (negative COVID test. 2/16)
  2. I got the notification that the parachute was in hand (the donor cells had been collected and received by Cleveland Clinic. 2/17)
  3. I prepared myself with the equipment make the jump (my Hickman port was installed through my chest up to my heart. 2/18)
  4. I jumped out the door of the plane (I received chemotherapy to kill my defective bone marrow. from 2/19-2/22)
  5. I put on my parachute and pulled the ripcord (The donor cells were infused into my body. Day Zero – 2/24.

The free fall is not very much fun. The chemo continued to kill off my bone marrow and other fast-growing cells and I didn’t feel well. I had some nausea. I felt very tired. My mouth got sore to the point that I could barely swallow and needed to get most meds through my port. Eventually I needed transfusions of whole blood almost every day and platelets every other day.

And there is always the nagging question of whether or not that parachute would actually open! I was very happy when my fall turned around on day +14. That day my WBC finally went up from 0.050 to 0.090. And it continued to gradually climb so that by day +19 I was able to leave the hospital. At that point I had not needed a transfusion in three days, and they canceled my first outpatient transfusion day.

I am now at day +69 and I have not needed any transfusions for 7 weeks! I feel well. I’m able to exercise. I’m down to one Cleveland Clinic visit each week. Every week they tweak my meds – add one, change the dose of another. We keep watch for any sign of infection or of Graft versus Host Disease. (I’m happy to report there’s nothing much to report so far.)

I am doing well and I am very grateful. I am grateful to God who is the source of my life and my salvation. I am grateful to my beloved friends and family for their prayers and their constant encouragement. I am grateful to the all the people at Cleveland Clinic for their expertise and good care for me. And I am very grateful to the donor who provided me with my parachute – I don’t know him but I do know that he is 25 years old, lives somewhere in the USA and goes out of his way to help strangers!

 

Assessing Best Practices in Managing Pregnancy, Myeloproliferative Neuroplasms

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American Journal of Managed Care


Published April 24, 2021
By Matthew Gavidia

Researchers discuss the unique fetal and maternal challenges for pregnant women with myeloproliferative neoplasms, with insight and recommendations provided on the potential benefit of aspirin therapy, cytoreductive therapy, and systemic anticoagulation.

In women with myeloproliferative neoplasms (MPN), pregnancy events have been reported most frequently in those with essential thrombocythemia (ET), followed by polycythemia vera (PV) and primary myelofibrosis (PMF).

As researchers noted in a critical review published in the American Journal of Hematology, MPNs pose unique fetal and maternal challenges, particularly risk of fetal loss. For ET, live birth rate is estimated at 70% with first trimester loss serving as the major complication in these populations, affecting 30%.

“Both pregnancy and MPN impart a hypercoagulable milieu, conferring a heightened risk for thrombosis,” said the authors. “In addition, bleeding diathesis may escalate at the time of delivery and in the postpartum phase, especially in the context of treatment with aspirin and/or low molecular weight heparin.”

With hematological and obstetrical challenges becoming increasingly prevalent for patients with MPNs during pregnancy, researchers sought to review these issues, as well as provide their systematic approach to management.

As they highlighted, it is now well established that patients with PV or ET experience a higher rate of both arterial and venous thrombotic events. However, current thrombotic risk stratification models in MPN have limited applicability in determining pregnancy complications, with risk suggested to be relatively low in those without prior thrombotic events and high if otherwise.

“In regards to thrombotic events, risk of venous thromboembolism is increased 4-fold to 6-fold during pregnancy, with the greatest risk in the post-partum phase,” they wrote.

“Both pregnancy and MPN impart a hypercoagulable milieu, conferring a heightened risk for thrombosis,” said the authors. “In addition, bleeding diathesis may escalate at the time of delivery and in the postpartum phase, especially in the context of treatment with aspirin and/or low molecular weight heparin.”

With hematological and obstetrical challenges becoming increasingly prevalent for patients with MPNs during pregnancy, researchers sought to review these issues, as well as provide their systematic approach to management.

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Decitabine With or Without Ruxolitinib Shows Benefit in Advanced Phases of MPN

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Oncology Learning Network

When initiated early in the course of the disease, decitabine with or without ruxolitinib has shown clinical benefit in patients with advanced phases of myeloproliferative neoplasms (MPNs; Acta Haematol. 2021;144[1]:48-57).

“Treatment options are limited for patients with advanced forms of [MPN] including blast-phase disease (MPN-BP). Decitabine has frequently been deployed but its efficacy and safety profile are not well described in this population,” wrote Selena Zhou, MD, Icahn School of Medicine at Mount Sinai, New York, and colleagues.

Using data for 42 patients (16 with MPN-BP, 14 with MPN accelerated-phase [MPN-AP], and 12 with myelofibrosis with high-risk features [MF-HR]) treated with decitabine alone or in combination with ruxolitinib, Dr Zhou et al carried out a retrospective review.

According to the review, the median overall survival (OS) for patients with MPN-BP patients was 2.6 months, and 6.7 months for those given ≥2 cycles of decitabine. The patients with MPN-BP and a poor performance status who required hospitalization at the point of decitabine initiation had a dismal prognosis.

Dr Zhou et al reported that the median OS was not reached after a median follow-up of 12.4 months for patients with MPN-AP and 38.7 months for patients with MF-HR patients; 1 and 2 patients in these cohorts were alive at 60 months, respectively.

Furthermore, the likelihood of spleen length being reduced and transfusion independence within 12 months of decitabine initiation was 28.6 and 23.5%, respectively.

Compared with decitabine monotherapy, decitabine plus ruxolitinib improved OS (21 months and 12.9 months, respectively).

“Decitabine, alone or in combination with ruxolitinib, appears to have clinical benefit for patients with advanced phases of MPN when initiated early in the disease course prior to the development of MPN-BP,” Dr Zhou and co-investigators concluded.—Hina Porcelli

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Australian MPN Specialists

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aussie doc

Dr. Kate Burbury, MBBS(Hons) FRACP FRCPA DPhil
Peter MacCallum Cancer Centre, Victoria

Dr Burbury is a consultant haematologist; stream lead for myeloproliferative disorders (MPD)/chronic myeloid leukaemia (CML) and lead clinician for haemostasis, thrombosis and peri-operative optimisation, including pre-habilitation, for all major cancer surgery patients at the VCCC. Kate is a member of professional societies, scientific committees, council member for Haematology Society for Australia and New Zealand, and actively involved in the development of expert guidelines and governance structures for both the institution and external working parties, including European Leukaemia Network: flow cytometry in myelodysplastic syndromes (MDS).

Dr. Wendy Erber, FRCPA FRCPath FAHMS
The University of Western Australia

Professor Erber graduated in Medicine with 1st class honours from the University of Sydney. She undertook her Haematology training at the Royal North Shore Hospital of Sydney and the University of Oxford as a Rhodes Scholar. In Oxford her research led to Doctorate of Philosophy. She has held Consultant Haematologist posts in Western Australia and in Cambridge, UK. From Cambridge she returned to Australia in 2011 to take up the appointment as Chair and Head of the School of Pathology and Laboratory Medicine at the University of Western Australia. In December 2016 she was appointed Pro Vice-Chancellor and Executive Dean of the Faculty of Health and Medical Sciences. Professor Erber continues to be active in diagnostic and translational research in haematology.

Dr. Cecily Forsyth, MBBS FRACP FRCPA
Jarrett Street Specialist Centre, NSW

Dr Forsyth has worked as a clinical haematologist on the Central Coast of NSW for 20 years after training in haematology at Royal Prince Alfred Hospital. She is passionate about improving rural and regional patients’ access to disease information, education and clinical trials, and providing educational opportunities for haematology trainees and haematology nurses. Her main clinical and research interest is myeloproliferative neoplasms and she has collaborated on Australian and International studies in these disorders. She is a member of the ALLG CML and MPN Disease Group Committee and has established the Myeloproliferative Neoplasms Registry (MPN01) on behalf of the ALLG.

Dr. Steven Lane, MBBS, PhD FRACP FRCPA
Royal Brisbane and Women’s Hospital, QLD

Associate Professor Lane is a clinical haematologist interested in all aspects of benign and malignant haematology, with a particular focus on myeloid disorders such as acute myeloid leukaemia, myelodysplasia and myeloproliferative neoplasms. He achieved his medical degree from the University of Queensland and subsequently completed his clinical training at the Royal Brisbane and Women’s Hospital and Princess Alexandra Hospital. As a research head at QIMR Berghofer Medical Research Institute, his lab concentrates on basic science research to discover new treatments for myeloid disorders such as myeloproliferative neoplasm and acute myeloid leukaemia.

Dr. Andrew Lim, MBBS, FRACP, FRCPA
Austin Hospital, Victoria

Dr Andrew Lim is a clinical haematologist at Eastern Haematology Oncology Group and a staff specialist in the Department of Clinical Haematology at Austin Health. Andrew obtained his degree from the University of Melbourne and has trained in haematology at Austin Health, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital. He holds dual Fellowships from the Royal Australasian College of Physicians and the Royal College of Pathologists of Australasia. Andrew has held appointments at Western Health and Dorevitch Pathology.

Dr Lim has experience in all aspects of haematology including laboratory diagnosis of various haematologic conditions; management of haematologic cancers, blood clotting and disorders of iron; and dealing with abnormal blood test results. He also has expertise in bone marrow transplantation.

Dr. David M. Ross, MBBS PhD FRACP FRCPA
SA Pathology, South Australia

Dr Ross is a consultant haematologist at SA Pathology with clinical appointments at the Royal Adelaide Hospital and Flinders Medical Centre in Adelaide, Australia. His PhD was on the topic of minimal residual disease and treatment-free remission in chronic myeloid leukaemia (CML). He has been an investigator in numerous clinical trials, including the landmark COMFORT-1 study of ruxolitinib in myelofibrosis. He supervises the diagnostic haematology service at SA Pathology and is an examiner for the Royal College of Pathologists of Australasia. His clinical and research interests include CML and Ph-negative myeloproliferative neoplasms. He is a Senior Research Fellow in the South Australian Health & Medical Research Institute.

Nobody Wants Cancer. But a ‘Big C’ Label Has Surprising Upsides.

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Source: New York Times

Classifying a rare blood disorder as a cancer opened new doors for disease investigation, treatment and hope for a cure.

By Ruth Fein Revell

I have a rare blood disorder. When it was diagnosed 25 years ago, it was called an “orphan disease,” meaning the small number of people affected didn’t justify research investments by major pharmaceutical companies.

Enter the World Health Organization and its 2008 decision to classify the condition as a blood “cancer.” This opened new doors for disease investigation and understanding. It prompted a growing number of super specialist practitioners, and most important, hope for some 300,000 people living in the United States with what are now called myeloproliferative neoplasms, or MPNs.

Nobody likes to hear a cancer diagnosis, particularly for a disease they thought was benign. In fact, when I first saw my blood disorder referred to as a cancer, I wrote to the MPN Research Foundation, a nonprofit research and advocacy group, and suggested they remove the “Big C” word from their website. How, I thought, could they be so misleading?

That’s when I discovered I was behind the times. MPNs were indeed reclassified as a malignant condition of the bone marrow, affecting sometimes one, two or all three types of blood cells: white cells, red cells and platelets.

The average age people are diagnosed with an MPN is 60-something. My diagnosis came at age 38. For the next 15 years, I was treated for essential thrombocythemia, a type of MPN that caused my bone marrow to produce significantly higher than normal numbers of platelets. The proliferation of platelets put me at risk for dangerous clots and ultimately led to complete blockages of two important veins of the portal system, which carries blood to the liver.

The Big C label began to look like good news.

Once MPNs were classified as blood cancers — including essential thrombocythemia, polycythemia vera and myelofibrosis — interest grew from research laboratories, major medical centers, and small and mega pharmaceutical companies, which now saw these rare and poorly understood conditions as an opportunity. Perhaps pieces to the MPN puzzle could shed light on more common blood cancers, like leukemia and lymphoma. And perhaps treatments for those widely studied cancers could be used to treat MPNs.

“The cancer designation did open up significant new funding opportunities, for example from the National Cancer Institute,” said Barbara Van Husen, board chair of the MPN Research Foundation. “It has definitely accelerated research.” There are more than 200 clinical trials underway for various MPNs, as well as ongoing research into stem cell transplantation, currently the only potential cure for these rare cancers.

In my case, for reasons researchers are working to understand, my bone marrow flipped a switch. I was no longer making excessive platelets. Instead I was producing too many red cells and was given a revised diagnosis of polycythemia vera, a distinctly different MPN. I went for regular phlebotomies, the modern version of bloodletting in which pints of my blood were drained into a collection bag to reduce blood volume. Think blood transfusion, reversed.

Doctors increased the dose of the 30-year-old chemotherapy drug I had been taking since my blood clots first appeared. The drug is still considered standard of care “if well tolerated.” It effectively adjusted my red counts. Unlike newer, more targeted drugs, however, it does not discriminate, potentially killing off not just red cells but white cells and platelets as well. Read more

Ruth Fein Revell, a health and environment writer, serves on a patient advisory board of the MPN Research Foundation.

BET Inhibitor CPI-0610 Shows Potential as Treatment in MF

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Lucia Masarova, MD, an assistant professor in the Department of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses the efficacy of CPI-0610 in the phase 2 MANIFEST (NCT02158858) trial in patients with myelofibrosis.

Masarova says that CPI-0610, a bromodomain, and extra-terminal (BET) inhibitor holds promise in patients with myelofibrosis since it could alter the transcription factors and potentially overcome the patient’s resistance to ruxolitinib (Jakafi). The MANIFEST trial investigating this drug had 3 arms; the second arm combined ruxolitinib and CPI-0610 in patients who responded suboptimally to ruxolitinib monotherapy and the third looked at the combination in the front-line setting.

For Arm 2, there were 70 patients enrolled and median follow-up of 28 weeks. There were 64 patients enrolled and median follow-up of 24 weeks in Arm 3. The second arm showed that patients could achieve further improvement in their spleen or their symptoms when receiving CPI-0610 in addition to ruxolitinib. The upfront combination in previously untreated patients demonstrated superior responses in terms of spleen size and symptoms. Spleen volume reduction was seen in about 60% of these patients, which is more than what is observed in the single-agent ruxolitinib trials, according to Masarova.

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CTI BioPharma: Potential Light At End Of The Rainbow

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Source: Seeking Alpha

Summary

  • Today, we take our first in depth look at CTI BioPharma, that looks like it might finally garner its first FDA approval.
  • The company is aiming its primary drug candidate as a lucrative niche of the myelofibrosis market.
  • An analysis on CTI BioPharma is presented in the paragraphs below.

Today, we take our first in-depth look at a small developmental company called CTI BioPharma (CTIC). We have received occasional inquiries on this name over the years, but I believe this is our first analysis on it. Given the company might finally be close to garnering its first FDA approval, it seems an appropriate time to dig under the covers. An analysis follows below.

Company Overview

CTI BioPharma is a small ‘Tier 4‘ biotech concern out of Seattle. The company is focused on developing novel targeted therapies for blood-related cancers. The company was once known as Cell Therapeutics but changed its name in 2014 over 15 years after going public. The stock trades just above $3.00 a share currently and sports an approximate market capitalization of $240 million.

The main asset of the company is a compound called pacritinib. The company provides this description of this drug candidate on its website.

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2 and IRAK1 developed for the treatment of patients with myeloproliferative diseases including myelofibrosis. Unlike other JAK2 inhibitors pacritinib does not inhibit JAK1. Inhibition of JAK1 has been associated with immune dysfunction, lymphomas and may also worsen thrombocytopenia and anemia.”

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COVID-19 and blood cancer: Evolving science on a dangerous comorbidity

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More than a year into the COVID-19 pandemic, new information emerges daily about the virus and its effects on various vulnerable populations.

The CDC’s list of underlying medical conditions that elevate risk for severe illness due to COVID-19 is described as a “living document” that will likely remain in flux as knowledge about the virus evolves.

Cancer has been identified as one disease likely to increase risk for poor COVID-19 outcomes.

Because bone marrow plays an essential role in immune function, hematologic malignancies are a particular area of concern. Individuals with blood cancers may have compromised immune systems due to the cancer, its treatment or both.

“Hematologic malignancies are diseases of the blood, the bone marrow and lymphoid organs, and that’s where most immune functions take place,” Nathan A. Berger, MD,Hanna-Payne professor of experimental medicine, professor of medicine, biochemistry and oncology, and director of the Center for Science, Health and Society at Case Western Reserve University School of Medicine, said in an interview with HemOnc Today. “We think the mechanism of that increased susceptibility is because of immune suppression or immune deficiency associated with the disease.”

However, the paucity of mechanistic data on this association reflects an overall lack of understanding about the interplay between hematologic malignancies and COVID-19. As the pandemic continues to unfold, researchers strive to make real-time treatment decisions based on limited information.

“It’s tough, because we don’t have much evidence out there,” Andrew Ip, MD, hematologist/oncologist at Hackensack Meridian Health John Theurer Cancer Center, told HemOnc Today. “We think convalescent plasma helps, but it’s still not strongly evidence-based. We’re waiting on larger research trials to publish their data. It’s very hard to make judgments when there’s not much good data.”

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MPN Patients Ask the Experts

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Three Key Relationships Bring New Drugs to the MPN Community

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 Byline:  Ann Brazeau, CEO & Founder

MPN Advocacy and Education International prides itself on bringing the experts to the MPN Community to ensure they are receiving accurate and updated information about clinical trials, treatment options, quality of life issues, and everything that could affect them medically.  It also brings biopharmaceutical companies together with the researchers/clinicians at all of their programs in an effort to inform both physician and patient who is doing what in the laboratories around the world that could bring a new drug to commercialization for their use.

These three relationships are critical to new drug development.  Our Industry Partners’ research and development teams work tirelessly in their labs to find answers to perplexing questions myeloproliferative neoplasms present.  The MPN specialists who give so much of their time to speak at our programs, spend hours in their labs and also see patients throughout each day to understand the complexities of MPNs and how to best treat each patient, and many participate in heading trials at their academic and medical institutions.  Patients participate and commit to lengthy clinical trials to help discover what works and what doesn’t.  Without them, drugs would not be developed or approved. Drugs have to be tested for safety and efficacy in patients.  There is no way around this fact.  Hats off to those who volunteer to participate in clinical trials in hopes that the drug may one day benefit others who have the same condition as theirs.  Participation in a clinical trial is a major contribution to our society.

Learn more about MPN clinical trials

This intricate connection is the key to so many discoveries that have saved lives, cured the common headache, stopped seizures, and cured some cancers.  While preclinical research answers basic questions about a drug’s safety, it is not a substitute for studies of ways the drug will interact with the human body.  People living with a chronic disease, like an MPN, benefit enormously from the pipeline of medications brought to market by hard-working clinical researchers who design and carry out clinical trials that show promise after testing in animals.

Education is critical to the success of clinical trials and participation.  If physicians that treat you are not aware of the numerous trials currently underway, is it your responsibility to share what you know? We would say yes.  Our programs for physicians that treat MPN patients always includes updates about current trials to be sure they are made aware of every single trial in the MPN space.  This information coupled with site locations and trial criteria are essential to connecting the patient to a specific trial.

In this era of individualized treatment protocols and combination therapies, which will eventually be widespread in not only MPNs but other disease areas, it would be a shame to miss opportunities where potentially good therapies might fall off the grid for lack of trial participants and trial sites due to already strained programs.  With the robust activity in MPNs currently, the challenges accompany the gains.  Thus, the relationships among the three groups is even more important.

MPN Advocacy and Education International understands the challenges experienced by all three entities.  Each plays an integral role in bringing new drugs to the shelves and ultimately to patients.  Researchers who participate in trials and see patients can be overwhelmed.  Patients may have financial issues that prohibit a commitment to a trial even if they want to participate.  There are social and cultural factors that have historically left many trials with one demographic of participants, namely white, insured, and often retired.

The good news is organizations and our partners want to understand the challenges and want to help in all ways.  In the seventeen years I’ve been working in MPNs, I am seeing the fruits of our advocating and educational efforts not only for patients, but in our relationships with biopharmaceutical companies and the hundreds of physicians who are not necessarily MPN specialists that see MPN patients.  Our engagement and desire to grow a deeper understanding of the many challenges patients face has created a real interest in finding ways to close the gaps in areas of access, participation, education, and support.

Without collaboration and communication, the momentum needed for new drug development would quickly lose steam.  MPN Advocacy and Education International remains committed to providing a platform for the dialogue necessary to keep the wheels in motion.

View our MPN Clinical Trials 101 Webinar