Finance Bob's War against cancer
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My name is Bob, and I'm fighting cancer
Myelofibrosis (MF) is a classified as a blood cancer. The cause is unknown. It is thought to be present in about 20,000 people in the USA; since this number is so low, the World Health Organization has classified it as a rare disease. However, we are starting to learn more about it, and realizing that it might impact many more patients.
MF typically presents with a couple different symptoms, and is very difficult to diagnose correctly. The two symptoms that usually present as definitive are a massively enlarged spleen, and wildly variable blood counts. The patient will suffer from a variety of secondary symptoms (fevers, sweats, chills, debilitating fatigue, cognitive dysfunction) and may well experience significant and severe organic damage (heart attack, stroke, cirrhosis of the liver, internal bleeding); if left untreated, the patient will typically die.
I have many issues. I have severe cirrhosis of the liver; an enlarged spleen; portal vein hypertension; significant connective tissue morphing into bone; and the list goes on. This is just the organic damage caused by the cancer secondarily.
There is no remission phase for this cancer.
There is only one FDA approved medication that was developed specifically to treat this cancer; other drugs might be used to treat some symptoms, but this approach does not treat the cancer itself.
There is only one treatment at present which might lead to a cure: a stem cell transplant from a closely matched sibling. Such transplants involve very lengthy and difficult recovery periods; compared to treatments for other cancers, they have a low success rate; they are suitable only for a small subset of patients; and finally – they are very, very expensive.
Awareness of treatment protocols is lacking. Not only are patients told (as I was) to go home, take it easy, and let death come peacefully, but hematologists don’t realize that this type of cancer requires vastly different treatment protocols from other cancer, including hematological cancers such as leukemia. For example, when taking the one FDA approved drug for myelofibrosis, the patient will, 99% of the time, see a dip and subsequent trough in certain blood counts happen nearly immediately.
In every other blood cancer, this is an indication of bleeding, typically “hidden” internal bleeding and possibly even a stroke, and requires specific treatment to identify and/or just stop the bleeding. However, in our case, this dip and trough are normal side effects of the way the drug works in the body, and treatment is not required. Within a few months, the patient’s counts will climb back to their normal on their own.
Many hematologists are unaware of this.
This lack of awareness is a direct result of current educational practices in medical school and residency. Hematologists and oncologists receive, at best, two days of classroom training on these cancers in all medical schools. This is not nearly enough for doctors to know techniques to best treat patients; none of the standard hematological practices apply to these cancers.
Awareness must also be raised among the "average persons". When I was diagnosed, it was by complete accident; I was rushed to the ER because I thought I was having a heart attack. My case is not an outlier: most patients who I meet are diagnosed secondarily, because some other symptoms suddenly arose. In the case of one young man I know, he was finally diagnosed because of persistent erectile dysfunction!
Of course, raising awareness is not nearly enough. In 2015, I joined a clinical trial for a new, cutting-edge drug called pacritinib. This drug, if approved, would only be the second drug approved to treat MF. When I was part of the trial, the FDA temporarily halted it; that left quite a few patients who were responding to the new drug in a bad position. Suddenly, their treatment was gone. Remember: this is life or death – and suddenly stopping this type of drug was known to cause severe side effects, including death.
Myelofibrosis (MF) is a classified as a blood cancer. The cause is unknown. It is thought to be present in about 20,000 people in the USA; since this number is so low, the World Health Organization has classified it as a rare disease. However, we are starting to learn more about it, and realizing that it might impact many more patients.
MF typically presents with a couple different symptoms, and is very difficult to diagnose correctly. The two symptoms that usually present as definitive are a massively enlarged spleen, and wildly variable blood counts. The patient will suffer from a variety of secondary symptoms (fevers, sweats, chills, debilitating fatigue, cognitive dysfunction) and may well experience significant and severe organic damage (heart attack, stroke, cirrhosis of the liver, internal bleeding); if left untreated, the patient will typically die.
I have many issues. I have severe cirrhosis of the liver; an enlarged spleen; portal vein hypertension; significant connective tissue morphing into bone; and the list goes on. This is just the organic damage caused by the cancer secondarily.
There is no remission phase for this cancer.
There is only one FDA approved medication that was developed specifically to treat this cancer; other drugs might be used to treat some symptoms, but this approach does not treat the cancer itself.
There is only one treatment at present which might lead to a cure: a stem cell transplant from a closely matched sibling. Such transplants involve very lengthy and difficult recovery periods; compared to treatments for other cancers, they have a low success rate; they are suitable only for a small subset of patients; and finally – they are very, very expensive.
Awareness of treatment protocols is lacking. Not only are patients told (as I was) to go home, take it easy, and let death come peacefully, but hematologists don’t realize that this type of cancer requires vastly different treatment protocols from other cancer, including hematological cancers such as leukemia. For example, when taking the one FDA approved drug for myelofibrosis, the patient will, 99% of the time, see a dip and subsequent trough in certain blood counts happen nearly immediately.
In every other blood cancer, this is an indication of bleeding, typically “hidden” internal bleeding and possibly even a stroke, and requires specific treatment to identify and/or just stop the bleeding. However, in our case, this dip and trough are normal side effects of the way the drug works in the body, and treatment is not required. Within a few months, the patient’s counts will climb back to their normal on their own.
Many hematologists are unaware of this.
This lack of awareness is a direct result of current educational practices in medical school and residency. Hematologists and oncologists receive, at best, two days of classroom training on these cancers in all medical schools. This is not nearly enough for doctors to know techniques to best treat patients; none of the standard hematological practices apply to these cancers.
Awareness must also be raised among the "average persons". When I was diagnosed, it was by complete accident; I was rushed to the ER because I thought I was having a heart attack. My case is not an outlier: most patients who I meet are diagnosed secondarily, because some other symptoms suddenly arose. In the case of one young man I know, he was finally diagnosed because of persistent erectile dysfunction!
Of course, raising awareness is not nearly enough. In 2015, I joined a clinical trial for a new, cutting-edge drug called pacritinib. This drug, if approved, would only be the second drug approved to treat MF. When I was part of the trial, the FDA temporarily halted it; that left quite a few patients who were responding to the new drug in a bad position. Suddenly, their treatment was gone. Remember: this is life or death – and suddenly stopping this type of drug was known to cause severe side effects, including death.
Organizer
Sharon Wanamaker
Organizer
Peninsula, OH