<![CDATA[Multiple Myeloma - Survival Rate Statistics by Hospital - Myeloma Blog]]>Wed, 11 Dec 2024 16:30:26 -0800Weebly<![CDATA[Open Letter to the HHS & FDA about accelerated orphan drug designations]]>Wed, 02 Aug 2023 19:23:32 GMThttp://myelomasurvival.com/myeloma-blog/open-letter-to-the-hhs-fda-about-accelerated-orphan-drug-designationsPicture

I was wondering if you could help me to resolve an inconsistency in research on orphan drug approvals which have me confused.


Under the Orphan Drug Act of 1983 which was passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myeloma, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States.[1]
They had made outstanding results for orphan drugs with expedited approvals as noted below.  Below it shows an average just about 240 days from an accelerated approval designation to noval drug FDA approval.  This is outstanding!   

Figure 4: Trend in maximum and minimum approval times for novel drug approvals using one or more FDA expedited development and review methods, 2015 to 2017




















​However some myeloma drugs I have followed through the years have not met this outstanding approval timeframe.  And recent history shows a reduction in new orphan drug approvals.  

A list of the orphan myeloma drugs I have followed with at least one expedited approval and the days to drug approval or just the current days from an expedited approval designation (ie. no yet FDA approved) are as follows. 


Ide-cel              Approved after           1215 days
Cilta-cel            Approved after             816 days
CT053.             NOT Approved yet      1367 days
PGEN-3006     NOT Approved yet..       478 days
GPS                 NOT Approved yet      1832 days
Lopofosine       NOT Approved yet      1535 days


The average of those orphan drugs that I follow is 1207 days vs. the chart above at 240 days.  As you can see, this is why I have trouble reconciling this massive difference.  To me 1207 days is a slow boat to approval and NOT a fast track.  If you might help me make this make any sense, I would greatly appreciate your guidance.

Best Regards/editor@myelomasurvival.com
Gary Petersen

]]><![CDATA[Bridging the GAP in Myeloma Survival]]>Sun, 12 Mar 2023 20:35:25 GMThttp://myelomasurvival.com/myeloma-blog/bridging-the-gap-in-myeloma-survival
First of all what is the GAP in myeloma survival?

Currently, The National Cancer Institute provides data on the survival of myeloma patients and they report a life expectancy of just over 5 years, however the myeloma specialists at myeloma centers of excellence have reported a life expectancy of over 10 years.  

This 5 year difference is the GAP between what is and what could be!

A simple explanation  for the difference is only 15 to 20% of patients actually see or have a treatment plan from a myeloma specialists who then it manages by a local hematologist/oncologist.  Patients may want to see a myeloma specialist, but find the wait time is excessive.  I have written an article titled “Myeloma Specialists do not Grow on Trees” which explains this conundrum.

I believe the only way to close the GAP is to provide myeloma specialists expertise, education, and coaching for myeloma patients and local hemotologists and oncologists with an educational platform which helps to provide the information which will help to close this GAP,

I am honored to be able to introduce the CEO of HealthTree Jennifer Ahlstrom who has been building a comprehensive platform and organization to help to close this GAP.

Jenny will be providing her perspective on how the HeathTree platform can help to Close the GAP, and how it may become a template for many other hards to treat diseases.  You can view HealthTree website at the link www.healthtree.org.   

We discussed this with Jenny on March 15th, on CureTalks and you can view the interview on YouTube below.

]]><![CDATA[Very interesting myeloma survival data from The National Cancer Institute]]>Wed, 19 Oct 2022 16:48:51 GMThttp://myelomasurvival.com/myeloma-blog/very-interesting-myeloma-survival-data-from-the-national-cancer-instituteEach year the National Cancer institute has put out a report and statistics on many different cancers.  It usually comes out in April, but has just gone through a massive change in the format.  Just when I got familiar with the old system a new one comes and for me it took a lot of time to get even a little familiar with the new system.    However, with the 2022 edition I found one graph which caught my eye. It was a graph of survival by year of diagnosis.  It was done for 1 year, 3year, 5 year and 10 year survival.  A few of my early observations were: 

1)  Every year for the last 10 years the graph has shown improvement in survival since 2000
2) The improvement is incremental and gradual, and not in a step fashion one might expect as new classes of drugs  are introduced like Revlimid,  Vecade,  Daratumumab, bispecifics, and CAR T.
3)  If we look at the year 2009 there is data for all of the survival years of 1,3,5, and 10.  The first year survival has an improvement from 2001 to 2009 of 71.4%  to 79.7% or 8.3%, 3 year improvement from 49.6% to 62.8% or 13.2%, 5 year improvement from 36.6% to 51.0% or 14.4%, and the 10 year survival from 21.2% to 33.4% or 12.2% improvement. 
4) Could this imply that early treatment could account for a minimum of 8.3/14.4 or 58% of the overall survival improvement?  Dr. Irene Ghobial's and Dr. Sigurdur Kristinsson work is ground breaking in this initiative.
5)  In the 10 year survival you can notice that survival has improved from 2001 at 21.2% to 33.4% in 2009,  and this 58% improvement in survival seems outstanding, however myeloma specialists have been quoting this figure of 10 year survival as the standard for there institution for years.  Does this imply the 85% of patients who are not treated by a specialist must wait until the newest treatments reach the treating community through osmosis?  This would also explain the lack of step improvements as new  drugs are developed. 
6) To aid in getting this to the patients and care givers, Myeloma Healthtree, IMF, LLS, and MMRF do there best to spread the word, but even Heathtree who has 9000 people signed up on their platform and this represents just a small fraction of the more than 150,000 people in the US who are living with myeloma.  For all those who are not being treated by a myeloma specialist your best bet is to join the Heathtree community.  If there was only a way to get past the fear of HPAA restrictions, and each myeloma patient could be informed at diagnosis of the value of early treatment and the best treatments as per Healthtree we could double the patients still living with myeloma to 300,000, or already have found the cure.

Good luck and may God Bless your myeloma journey.
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]]><![CDATA[Are the CDC #COVID19 Vaccine Guidelines Designed To Vaccinate Those Most Likely To Die If Infected? Follow The Facts And Science!]]>Thu, 25 Feb 2021 08:00:00 GMThttp://myelomasurvival.com/myeloma-blog/are-the-cdc-covid19-vaccine-guidelines-designed-to-vaccinate-those-most-likely-to-die-if-infected-follow-the-facts-and-scienceI am writing this for the people like the ones who have reached out to me and others with their story! A brief sample:
- My wife, stage 4 MM and 65 is still refused shot even though her Oncologist ordered it.
- I am 56, in remission, Stem cell transplant recipient, and am still on treatment, and have been writing to my state, Oregon, for the past few months begging that they prioritize people with serious underlying medical conditions regardless of age.
- I am a patient with a rare incurable cancer and cannot receive treatment without being vaccinated and @SecMandyCohen is refusing me. If I die it’s on your hands
- New York Times article “In the Vaccine Scramble, Cancer Patients Are Left Behind”  CLICK HERE
- USA Today article It's a scary time': Cancer patients are frustrated at states making them wait for the COVID-19 vaccine despite federal guidelines CLICK HERE

​A good start is to see who are those that are dying? Two graphs I have found useful are one which shows the death rate by age and the other showing the death rate based on whether you are healthy or have a preexisting condition when you come down with a COVID19 infection.
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If we were to look at just these two charts the obvious choice would be people over 80 with preexisting conditions, followed by over 70 with preexisting conditions,  then 50, then 40, and ending with all who remain without preexisting conditions.  This strategy would reduce deaths by 95 percent by vaccinating just 100 million people with  200 million doses used.   The USA would be 33% complete as of mid February because we have 66 million shots in arms!  

The current death rate is 1.51 deaths per thousand US residents.  So how do the subsets of patients compare to this baseline? Several potential high risk groups have been identified, including health care workers, older Americans, people with preexisting conditions, older citizens with preexisting condition, first responders, teachers, and critical workers like grocery workers.  I will list them by deaths per thousand and how it compares to baseline.

Baseline All USA residents -                   1.51 deaths/1000
Residents over 65 -                                 7.32 deaths/1000 or 4.8 times the baseline 
Residents with preexisting condition -     4.75 deaths/1000 or 3.1 times the baseline 
Over 65 with preexisting conditions -        Likely much greater than 7.32        
All residents under 65 -                              .38 deaths/1000 or 1/3 of the baseline
Teachers(estimate below baseline) -        Between .38 and 1.51 deaths/1000
All active cancer patients -                      12.81 deaths/1000 or 8.5 times the baseline
Health Care Workers (2900 deaths) -        .15 deaths/1000 or 1/10 of the baseline
Transplant and Myeloma patients -         15.10 deaths/1000 or 10 times the baseline
​Assisted Living Residents and staff -       51.60 deaths/1000 or 34 times the baseline
Prison Inmates (possible high risk) -         1.06 deaths/1000 or 7/10 of the baseline

I was surprised by two of these.  Health Care Workers deaths numbered 2900 and the total employment is 19.6 million so the number is accurate, but why? They see the devastation and know the risk so are ultra safe and use PPE properly. Also there are few in the over 65 category. In addition many hospitals have a Covid specific wing which limited the number potentially exposed!

The second surprise was the very high death rate at assisted living facilities.  I would have thought it was closer to the death rate of seniors over 85 or 5 times the baseline or average.  These facilities must be like little Petri dishes for transmission!

We are at the beginning of the vaccination program, but there is  still time to change and improve the system.  If we eliminate  deaths we take the burden off of our critical Heath care workers.  We must focus on assisted living facilities, cancer patients and people over 19 with preexisting conditions, then citizens over 50 without preexisting conditions, the remaining. Once this is done at least 95% of deaths would be eliminated, then all others.  Please follow the facts and science, it will save LIFE!  
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Please let your senators and representatives know if you are having problems getting vaccinated by finding their representatives by CLICKING HERE, and  letting them know your concern and attach this post as support information.

Good luck and may God  bless your journey through this pandemic!

]]><![CDATA[New CureTalks Series -  Small BioTech, The Engine Of Change And Hope For Orphan Diseases eg #Myeloma #AML #TNBC #PediatricCancer]]>Mon, 12 Oct 2020 07:00:00 GMThttp://myelomasurvival.com/myeloma-blog/new-curetalks-series-small-biotech-the-engine-of-change-and-hope-for-orphan-diseasesCLICK ON THE YOU TUBE VIDEO BELOW TO VIEW THE FIRST IN THIS SERIES.
CureTalks has been so kind as to initiate a new series which will highlight the value of the contribution of small biotech to the development of most drugs for orphan diseases.  Myeloma is one of those orphan diseases, and iMids, Proteasome inhibitors, CD38 monoclonal antibodies, and these drugs in combination, are the current standard of care for myeloma treatment.  You can see the small biotech origin of some of these drugs if you CLICK HERE. The average new drug for an orphan disease is just not a big enough money maker to interest any of the large pharmaceutical companies.  As a result the FDA had developed a new program in 1983 called the Orphan Drug ACT to provide incentives, economic help, and FDA assistance to navigate the FDA approval process.  All intended to promote Orphan Drug development. This benchmark program has provided the 7000 orphan diseases with many new drugs, and since the origin of this program these companies have taken the number of orphan drugs from a total of 38 to now well over 500.  A significant improvement,  however this means the vast majority of orphan diseases still have NO APPROVED drugs.
When we say orphan disease we may think this is not a very large number of patients, however 1 in 5 cancers is an orphan cancer, and the have much lower life expectancy  than non orphan diseases.  For myeloma, we have been blessed to have several drugs available, however all major breakthroughs have come from small pharma.  The most likely to be approved are those with dual FDA designations, like orphan drug, another called fast track, breakthrough, etc.  Most all major developments have come from small pharma, and currently we have 8 myeloma drugs with dual FDA designations, but only 2 are backed by Big Pharma, the remaining 6 are Small Pharma.  Big Pharma needs no help getting their products to market, however Small Pharma brings their products to market on a shoe string budget.  They are the ones that put it all on the line, do the heavy lifting, and when they get close to success are often purchased by Big Pharma who then cash in.  I just want the opportunity to highlight these small biotechs which have provided so much to the Orphan Disease community, and hope that we can continue to keep them in the game so they can continue their treatment leadership. 

I am currently in contact with the management of Cellectar and Sellas Life Sciences to provide them with a forum to discuss the technology they bring to market, and the challenges of being small bio tech and bringing life saving drugs to market. We will have our first broadcast in October with Cellectar, which has a targeted universal drug conjugate with a potential to be used for many cancers. Both of these companies have several orphan diseases which have dual FDA designations, and therefore have a high probability of success. Cellectar has 11 dual designations with 4 for pediatric cancers, and Sellas with 3 dual designation with one in Phase 3.  I have myeloma so I have a vested interest to promote the 6 small bio tech stocks with dual designation for myeloma but limited resources  However this forum will be available to all small bio tech with FDA dual designations.   The FDA has an uncanny way of identifying the best drugs for Orphan Diseases, but unfortunately these companies struggle to fund the outstanding life saving treatments.  I hope CureTalks can help to improve the lack of understanding of the importance of small biotech.

Good Luck and God Bless your cancer journey. For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

]]><![CDATA[#COVID19 Vaccine Preface - Why Do People Beat the Average Myeloma Life Expectancy Prognosis?  Or How To Improve Your Multiple Myeloma Survival Rate!]]>Mon, 14 Sep 2020 18:48:26 GMThttp://myelomasurvival.com/myeloma-blog/2020-covid19-preface-why-do-people-beat-the-average-myeloma-life-expectancy-prognosis-or-how-to-improve-your-multiple-myeloma-survival-ratePicture
2021 COVID-19 VACCINE PREFACE 
Myeloma patients, please follow the universal recommendation to get vaccinated for COVID19!  Mayo, International Myeloma Society, IMF, MyelomaCrowd, and MMRF all say the same thing, “Get it as soon as available!”  CLICK HERE or HERE. I just hope our myeloma experts are heard and recommendations followed!

If you are reading this you are one of the lucky ones who has not been infected, or infected and survived, or been vaccinated and IF the vaccine has provided adequate protection.  The Big IF at this point is whether the weakened myeloma patient’s system has been able to provide an adequate defense against infection, or if infected at least keeps us out of the hospital.  No studies have been completed to confirm the efficacy of vaccines in myeloma patients.  The LLS has started a study to do just that, but no results will be published for some time. For a link or to join the study CLICK HERE. We do know from the vaccine clinical trials some vaccines have shown an efficacy of 95% and a 100% chance of not being admitted to a hospital. However, because the MM patients who are not vaccinated and infected with COVID19  have a risk of death 15 times that of the average American, it appears our own immune response is often not able to defend us against severe infection without help from a vaccine.  With the recent loosening of restrictions and the lack of knowledge of the efficacy of the vaccine in myeloma patients, a continued vigilance  is needed. Better safe than sorry! 

You have taken all necessary  precautions outlined in the CDC guidelines and those provided by the myeloma care community,  Some of my favorites include:  Prevention CLICK HERE and Myeloma Treatment CLICK HERE.  I think we are all just exhausted by the pandemic and have been doing our best to comply.  All of which has served us well to date, however I see it in myself, in my fellow advocates, and the population in general, a tendency to let our guard down.  We have all gotten through 12 months of some very hard times, and for us the high risk patients we must  remain more cautious than the average person.  We already were before COVID19, because the risk of infection may be 7 to 10 times higher for myeloma patients than the USA population per MMRF.  So why wouldn’t we be more vigilant during a pandemic?  Try to keep your guard up as the pandemic continues, because COVID19 relentlessly continues and becomes more deadly.  Tragically, in just one year COVID19 deaths have greatly exceeded the total combat deaths(291,000) in the 4 years of US involvement in World War II.

Why Do People Beat the Average Myeloma Life Expectancy Prognosis?

I have updated this post several times, and it has been very helpful to many in the myeloma patient community.  I believe one key area which could have the greatest impact on patient survival is the improved dissemination of the myeloma specialist approved best treatments for each individual patient.  The 20% of myeloma patients who see a myeloma specialist or consult with one, are provided with the best current treatment knowledge, but this leaves the remaining 80% underserved.  How then do we make this happen?

One thing has become very clear to me, the pace of change and progress for myeloma has become exponential.   This is a very good thing!  We have had 5 drugs approved for myeloma since 2015 (two are new classes of drugs).  In addition, new targeted treatments like CAR T and ADC(Antibody Drug Conjugates) have exploded with just 3 myeloma CAR T ASH(American Society of Hematology) abstracts listed in 2009, and in 2018 ASH lists 104 myeloma CAR T abstracts, or 35 times as many. 

As a patient advocate I find it nearly impossible to keep up with the avalanche of new data. I can not see how anyone but a skilled myeloma specialist can keep up to date with this rapid pace of change.   But Dr. Lonial, a world class myeloma specialist from Emory University, says it best: CLICK HERE to view his advice.

Most myeloma specialists, and most knowledgeable patients and advocates believe this to be critical.  However, even with the need for a specialist input so obvious, 80% of patients do not see a myeloma specialist or obtain their input for a treatment plan.  I have written about this in a blog post titled, "The Myeloma Conundrum!  Myeloma Specialists Do Not Grow On Trees!"  What this highlights is myeloma specialists are already working at their maximum, so even if the 80% wanted to see a specialist, there is just not enough specialists to handle all of the patients.  The solution identified in this post is to put expert information and guidance into the hands of the local hematologists and oncologists, and patients will receive expert input and achieve much better outcomes.
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Mayo Clinic has developed the mSmart program.  This program provides a template that doctors can follow to provide care outlined designed and developed by some of the very best minds in myeloma research and treatment.  You can go to the mSmart site if you CLICK HERE.   A sample of the information presented on the site is shown to the left.
Currently under development, Dr. Fonseca of Mayo, Phoenix and the Myeloma Crowd have put together what I would call the next step in the process to make expert advice individualized, and in a manner patients can use and understand.  It was designed by myeloma experts, myeloma patients and is called The Health Tree.   You can learn more about the program and find your best individualized treatment if you CLICK HERE.   Until there is a massive increase in the number of myeloma specialists, these two sources are your best bet for the underserved 80%.

In the last two years, the average life expectancy has gone from 4 years to 5.5. years, according to The SEER(Surveillance, Epidemiology, and End Results) data for multiple myeloma published in April of 2018 by the National Cancer Institute.  This is outstanding progress in that life expectancy had been stagnant at 4 years for 5 consecutive years.   Some patients beat the odds and live 10 to 20 years or more. When I was first diagnosed, the data for a person with dialysis-dependent kidney failure was just 3 months, and the average for myeloma patients overall was about 3 years. Now, I am beyond thankful to be a nearly 13 year survivor.   I believe there are three critical components to beating the odds: Part one is early diagnosis and treatment before end organ damage.  Part two is disease dependent, or the hand that you were dealt.  Part three is related to the level of care that is available to you.  For more information on survival rates and life expectancy CLICK HERE. 

Part 1 - Early Diagnosis and Treatment

If you are lucky enough to have a general practitioner who picks up high protein in the blood and finds the disease early while it is smoldering, or stage one, you have won the Myeloma Lottery.  Life expectancy of stage one disease is 3 times greater than if you have been found in stage three.  Treatment guidelines were published in November of 2014 with the express purpose of finding and treating the disease before it has progressed and  causes end organ failure.  A National Institute of health article states the intent of this clearly; "The concept of initiating therapy after end organ damage is analogous to initiating treatment after the development of metastatic cancer in solid tumors. Indeed, screening, early detection and intervention have played a large part in the major curative advances that have been achieved in solid tumors whereas metastatic cancer remains incurable in these same malignancies. It is, therefore, not surprising that MM remains incurable, in spite of all the advances in therapeutic interventions. Could it be because we are waiting too long – until metastatic myeloma occurs – to treat our patients? In such a condition, watchful waiting may actually be more harmful to the patient than early intervention."  To read the whole article CLICK HERE! If you are one of the lucky ones who are found in the early stages of active myeloma or smoldering myeloma, you will have the luxury of time to understand the treatment options, find a myeloma specialist (a must), and plan to confront your disease before permanent end organ damage.  Dr. Rajkumar of Mayo Clinic did a wonderful job of explaining  the new criteria for myeloma diagnosis, and you can read it if you CLICK HERE.  Dr. Irene Ghobrial is doing some great work to follow MGUS and smoldering stages of the disease, to  develop treatments to cure, or at least prevent end organ damage.  CLICK HERE to view a myeloma crowd interview with Dr. Ghobrial on the subject.  Just as a note, the country of Iceland is testing all of its adult population over 40 to screen for MGUS, Smoldering, and active myeloma.  They call it iStopMM, a clinical trial supported by the IMF(International Myeloma Foundation).  This is a future I pray we will all see where we could cost effectively find MM early and treat it before end organ damage.  CLICK HERE or HERE to learn more about iStopMM.

Unfortunately,  Myeloma UK has reported that 1 in 5 myeloma patients die within the first two months of diagnosis, and that it takes nearly a year from the first symptoms to diagnosis for 25% of newly diagnosed patients.  Dr. Morgan of UAMS said it best when he outlined his thoughts on the subject of awareness and delayed diagnosis. He believes the fact that it takes 3 to 6 months and more often 6 months from first symptoms to diagnosis is a bit of a scandal.   To make real inroads in the myeloma we need to get it diagnosed early before we have organ involvement.  We need to make family doctors and family practitioners more aware of the disease.  They should do M spike and light chain tests on patients.  This makes a lot of sense to Dr. Morgan.  It is really tragic when patients develop renal failure when awareness of myeloma by a General Practitioner might have allowed the patient to get a consult or treatment from a myeloma specialist.  A myeloma specialist is critical to a patient's long term care and survival.  It is a disease that does not come on overnight but is years in the making.  Patient organizations can make a difference.     He believes the future of Myeloma will be to get earlier diagnosis, safe treatments, chemo prevention strategy, regular screening for para protein, and early intervention. 

I have kidney damage, a good friend of mine has debilitating bone pain, others collapsed vertebrae, one suffers from a myeloma caused stroke, and many have died from delayed diagnosis.   All of which might have been prevented with a simple test of light chains and M spike costing under $150 without insurance, and no cost if covered by insurance and referred by a General Practitioner.  It is what could be!

Part 2 - Disease Dependent

Some people are just plain lucky and are given a form of myeloma that is not that aggressive.  In other words they have myeloma, but it happens to be smoldering myeloma.   This form of the disease can be present in the patient but not show any outward symptoms.  It can remain in this mode for 5, 10, or even 20 years.

The age of the patient is very important, in that you are 2 times more likely to survive if you were diagnosed at 49 years of age or less.  The average age of  the typical myeloma patient is 70.  You can read more on this subject if you CLICK HERE. 

Some people may have an active disease but do not have any of the negative prognostic indicators.  These indicators include, but are not limited to, deletion of chromosome 17p and  translocation of  4;14 or 14;16 or 14;20.  Your myeloma specialist will run the FISH test or other genetic tests like GEP(gene expression profiling) to determine if you have any of these negative prognostic indicators.  If you are considered high risk(15% of patients) , the life expectancy is less than half of the current average, or just 2 years.  You can read more about high risk multiple myeloma if you CLICK HERE. 

The sensitivity of the disease to treatment is also important.  My myeloma seemed to be very sensitive to the combination of Cytoxan, Thalamid and Dexamethasone, a treatment that put me into remission very quickly.  Some people might have the same experience with Revlimid, Velcade, or Dex, or any combination of these drugs.  If the disease comes back, as it often does, the re-application of the same regimen may continue to work for years.  I know one patient who has taken Thalomid for years as his only treatment and remains in remission. 

And of course if the average is 5.5 years, half of the people will invariably beat the average. 


Part 3  -  Quality of Care


There are some elements that you may or may not have much control over, the first of which is the availability of insurance.  If you do not have insurance or have no access to care, the average life expectancy is less than one year.  However, Medicare has a Compassionate Allowance Program where you can be approved in less than two weeks if you go to your local office and can show that you will not live without care.    To see the program CLICK HERE.  The Affordable Care Act may provide an option for the 15% who are not insured, and Medicare, Medicaid, and drug company assistance programs are also available. In addition, there are  other programs which can provide assistance  listed on the bottom of the home page, to view CLICK HERE.

Multiple Myeloma is a rare blood cancer, so many hematologist/oncologists may not see one patient in a year.  As a result not all oncologists or hematologists are the same. However, some are very skilled and experienced with Multiple Myeloma and have treated many myeloma patients. The data shows these myeloma specialists provide an average life expectancy of 10+ years or more, compared to the average which is at 5.5 years.

For a listing of these exceptional specialists CLICK HERE or for a more extensive list without survival history just CLICK HERE. I chose to get my 2 SCT(stem cell transplants) at University of Arkansas for Medical Sciences, UAMS, which has a myeloma program called MIRT, Myeloma Institute of Research and Therapy.  At the time they had over 10,000 transplants under their belt, and as a result they were expert at the process, and knew what could go wrong and had a plan in place to get you through any potential complications.   I have found from my work on this site that centers like Mayo, Dr. Hari(Medical College of Wisconsin),   UAMS, or Dr. Berenson's (IMBCR) have very different approaches to treatment, but because they are expert in what they do, they have similar results.   You would choose a brain surgeon over any other surgeon if you had a brain tumor, why would you not do the same for myeloma? Find out how to find a myeloma specialist by CLICKING HERE or CLICKING HERE. 

Myeloma specialists have access to drugs that other oncologists do not.  Because they are the thought leaders, they are involved in clinical trials, and can obtain some drugs through other programs that lesser known oncologists do not have access to. Worse yet, oncologists who are not myeloma specialists may not even know that some of these drugs even exist.  For example, some of the well connected specialists have access to drugs or treatments like CAR T, MILs, Venetoclax or Selinexor  which are not approved  treatments. But these experts can get approval for initial therapy through clinical trials or other programs. Or some specialists can use drugs that are only approved for relapse or secondary therapy options (Daratumumab,  Ixazomib, Krypolis and Pomalyst), and obtain approval to use them for newly diagnosed patients.   They also have access to the best clinical trials like KRDD(Krypolis, Revlimid, Darzalex, & dexamethasone)   for first line therapy which provides a response in 100 percent of patients.    When you run out of options with the currently approved drugs, they can provide access to those that have done great in clinical trial, but are not currently available to the general public. Because you need a significant infrastructure to conduct clinical trials at your facility and they cost the facility $15,000 per patient, few local oncologists have access to clinical trials.  Sometimes it is who you know! 

Myeloma patients seldom die from myeloma, they die from the complications from myeloma.    The number one complication is pneumonia, and others include infections, kidney failure, anemia, etc.  This, therefore, brings me to the realization that supportive care for the treatment of the many complications of this disease may just be as important as the cancer treatment itself.  Or a great Defense(supportive care) is as important as the Offense(cancer therapy).   MD Anderson and Mayo Clinic emphasize supportive care in their programs, UAMS actually has a Director of Supportive Care in their myeloma program, Dr. Elias Anaissie, now a Medical Director at Clinical Trial and Consulting Services (CTI).  has an extensive background in supportive care.  Dr. Anaissie has published a well written example of an exceptional supportive care model. You can read this publication if you CLICK HERE. To read my blog post on supportive care CLICK HERE.

I also think the quality of care that you receive can be affected by the knowledge of the patient, and this can be obtained by doing your research on finding the best approaches to care by looking at the work of the best myeloma specialists on-line, and by going to great sites as listed in the Resource Section of www.myelomasurvival.com. To find out how to get educated about multiple myeloma  CLICK HERE.  In addition,  joining a support group of the International Myeloma Foundation or the LLS (Leukemia, Lymphoma, and Myeloma Society) will provide more great information to improve your life expectancy.  I have found that the average life expectancy of most of these support groups far out-performs the average. Knowledge is power!  Additional information on the benefits of support group membership can be found if you CLICK HERE. 

With 30,000 new cases of multiple myeloma in the USA, we can estimate the total number of patients in just the USA at 165,000. If we can move the average life expectancy from 5.5 years to 11 years by having myeloma specialists guide your care, we could save 165,000 times 5.5, or 907,500 years of LIFE.  Many times more if we include the entire world.  You all can help by getting this message out to the myeloma patient community through Facebook and Twitter. You may  know someone who has myeloma or  may have a friend or family member that can be helped by this information.  With your help we can "SAVE LIFE"!

Good luck and God Bless your Myeloma Journey/ editor@myelomasurvival.com

For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1
]]><![CDATA[My Thank You Email To The FDA,  Which Applies To The Entire #Myeloma Care Team!]]>Thu, 20 Aug 2020 14:51:12 GMThttp://myelomasurvival.com/myeloma-blog/my-thank-you-email-to-the-fda-which-applies-to-the-entire-myeloma-care-teamFDA, Thank You Again For giving #Myeloma Patients A new LAST CHANCE AT LIFE!

Thank you for the approval of belantamab mafodotin for late stage myeloma treatment.  The myeloma patient community believes the cooperative efforts instituted in the FDA orphan drug program has given myeloma patients so many added years of life.  When I was diagnosed 13 years ago life expectancy was just 33 month.  It is now TWICE as long!  The following is the reason we have belief we have a future.


Myeloma patients should feel blessed to know they have had 10 new drugs and drug classes approved in the last 16 years all of which all had two FDA designations.   In addition, Myeloma (an orphan disease) has  7 drugs which  all had two FDA designations, but have yet to be approved.  These drugs include the following:

Drug                             Company                 Designations      Description

CLR131                         Cellectar                      OD&FT           Radiotherapeutic phospholipid 

JNJ-4528                       Janssen                       OD&BT          CAR-T with two BCMS-targeting
                                                                                                     Single domain antibodies 
CT053                            CARsgen                  OD, RMAT*       A fully human anti-BCMA CAR--T                                                                                                      

bb2121                          Celgene                       OD&BT          BCMA chimeric antigen receptor
                                                                                                    CAR T
Galinpepimut-S              Sellas                        OD&FT           Immunotherapy vaccine to elicit 
                                                                                                    strong response against WT1
P-BCMA-101                   Poseida                  OD,FT&BT         Autologous chimeric antigen
                                                                                                      receptor(CAR) T-cell therapy
Melflufen                  Oncopeptides                 OD&AA            Activated by aminopeptidases, 
                                                                                                      overexpressed in myeloma
So how have we fared with this list?

AMG420 - has dropped from the list in favor of a kinder gentler similar drug called AMG701, however AMG701 has only one FDA designation.

All of the other drugs on the list are progressing very well, with  bb2121 expected to be approved in the next several months.  

Best Regards/ Gary Petersen editor@myelomasurvival.com]]><![CDATA[Orphan Disease's Biggest Lost Opportunity! The FDA and NCI Need Advocate Input!]]>Mon, 17 Aug 2020 07:00:00 GMThttp://myelomasurvival.com/myeloma-blog/orphan-diseases-biggest-lost-opportunity-the-fda-and-nci-need-advocate-inputIf Orphan cancers were a disease category by itself, it would be one of the top 5 largest killers in the USA just behind Heart Disease, Cancer,  and  #COVID19.  I​ have myeloma and had the opportunity to go to ASH 2019, and it happened to be one of the best opportunities to see the future of blood cancer treatments.  For many of the meetings you will see some of the most significant developments in cancer treatment like CAR T, ADC(Anitbody Drug Conjugates), AWC(Antibody Warehead Conjugates), BiTE’s, etc. and they are held in rooms which can seat hundreds or sometimes  thousands of people.

However, the one thing all of the drugs under development have in common is they all must  obtain FDA approval. The FDA had two meetings scheduled. It was for this reason I wanted to make sure I attended the meeting titled “FDA Dialogue with Patient Representatives and Advocates”.  Myeloma clinicians and advocates had just gone through a very difficult approval process for Selinexor, and I wanted to find out what I could learn at this meeting.  I almost missed the meeting because most are in large halls, and this meeting was in a meeting room, so I kept walking past it thinking this little room could not be the venue for a meeting as important as it seemed to me.   As you can see in the following image the advocates meeting was in OfficeW206, and the other FDA meeting and most meetings in a hall the size of the grand Hall D.

At this meeting the FDA was well represented with 38 in attendance, and they seemed to outnumber the advocates by a factor of two to one.  I was frankly shocked at the poor turnout of advocates, in that these FDA officials held the final judgment on approval or rejection. 
Picture
Myeloma had two representatives so we were fairly well represented, and we were also quite willing to participate in the discussion.   We had the opportunity to learn about the programs for orphan diseases which are designed to Increase the number of orphan diseases which have FDA approved drugs.  Of the 7000 orphan diseases this FDA program had provided just over 500 new drugs. and  this is just since the first Orphan Drug Act was passed in 1983.  Prior to this FDA program few, if any, new drugs were ever approved for orphan diseases with less than 50 FDA approved orphan disease drugs available.  The average orphan drug has annual sales of $350 million dollars each year, so most large pharmaceutical companies find this level of added sales is not worth their effort and the $2 billion dollars to bring them to market.  The FDA orphan drug program has developed a designation which drug companions can apply, and this provides tax benefits and help in getting through the FDA approval process.  It is because of these incentives that many smaller companies have become the engine of orphan drug development.  The FDA has added other designations like Fast Track, Priority Review, and Breakthrough designations which help to expedite drugs through development.  In addition, the National Cancer Institute, American Cancer Society, MMRF, LLS, Academic Institutions, and many others provide research grants to help fund Orphan Drug development.  All of these organizations and the researchers and disease specialists are our allies in this life and death struggle.  We can only thank these groups for their efforts to help to promote orphan drug development, without which there would be NO PROGRESS!

We were able to provide inputs to the group, praised them for final approval of Selinexor, asked for more representation specific to the disease to be at the the Oncology Drug Advisory Committee meetings to discuss the FDA concerns, and to recognize this is a last chance at life for late stage patients.  For example if there were just one more myeloma specialist (or a total of two) of the 13 members on the ODAC(oncology drugs advisory committee), Selinexor would have been approved months earlier.  

Small Biotech companies are at a significant disadvantage in the universe of drug companies.  They are small and find it hard to fund themselves as they work through a 8 to 10 year FDA approval process.  They seldom profit during these initial development years and find it impossible to borrow money, so they must depend on  the capital markets for funding.  As I had written before, the SEC has few tools to protect these baby biotechs from Capital Vultures(CLICK HERE for the article) who manipulate the stock, an illegal tactic  driving the stock price down.  This makes it even more difficult to fund their trials and these baby biotechs are forced into bankruptcy.  I would love to see the SEC with the enforcement power to put these animals in jail, as well as the government provide a guaranteed loan program as another funding avenue, and  prevent the use of viscous short sale attacks designed to profit from the drastic decline in a vulnerable biotech stock price.  Because of these disadvantages we need all the allies we can get and must have more protections for small biotech firms.

One way as advocates and disease representatives to champion your orphan disease would be to attend any of these FDA/Advocate programs.  Or send this article to your Senate and House representatives (CLICK HERE for contact information for your senators and representatives), @secazar, and @SteveFDA .

Good Luck and may God Bless your family's cancer journey.  You can see more information on myeloma at my site www.myelomasurvival.com.
]]><![CDATA[#Myeloma Patients Health - It's A Question of Balance]]>Thu, 21 May 2020 21:32:10 GMThttp://myelomasurvival.com/myeloma-blog/myeloma-patients-health-its-a-question-of-balancePictureThe COVID19 DREAM TEAM
Like everyone who is in the high risk populations for death from the CORONA virus, myeloma patients have heightened resistance to going to the hospital for fear of getting exposed to the virus.  Unfortunately, this is heightened by the findings, 40% of all infections are caused by asymptomatic carriers, and the average death rate for COVID19 positive Americans is 5.9% but over 30% for myeloma patients. 

I have tried to practice what I preach and follow the guidelines which #birx and #fauci have recommended. Until recently you could not get a virus test unless you were symptomatic and had a doctors order, and it was not covered by Medicare.  This has now been changed and is covered by Medicare, and I have since gotten both the virus test, and the antibody test(courtesy of myeloma crowd).   I do not have the virus or the antibodies to the virus, so I still remain vigilant.  It would be nice if I had the virus and recovered but with a death rate of 30%, the best approach is to stay virus free until a vaccine or cure is found and available to all.

I have been putting off surgery for a hip replacement for years, but it is now bone on bone and just too painful.  It was originally scheduled for April 22nd but was canceled by Mayo because of the pandemic.  Talking with my doctors they believe now is the time for treatment because we are over the curve and going down, but with the opening of the country we will likely see another wave this fall and winter.

I have had insurance issues trying to get pain medications. Tylenol is no help, and I can not take NSAID's because  I have myeloma caused kidney damage. I could take a topical NSAID cream which does work and is easy on the kidneys, but BCBS of Florida has turned it down 3 times.  Their response is I have to take oral NSAID's first, but I have told them oral NSAID's would damage my kidneys and put me back on dialysis.  So we all have this Question Of Balance.  We all have to weigh the risks vs. the benefits of our health care decisions. Is the risk of getting COVID19 significant enough to cancel the surgery? 

I chose to be pain free. I will be going to Mayo in Jacksonville, and the Mayo system has a number of safety measures and procedures in place.  So I come to the conclusion, I have minimized the risks, will use a N95 mask at all times, keep my distance if possible, and sanitize hands and things I touch frequently.  I asked a number of questions and found out the OR staff have all been tested for the virus, all patients are tested before surgery, all nursing staff are masked and surveyed for fever or symptoms, as are food service personnel, and there are two separated areas, COVID and non COVID, where staff never crosses over.

I think I am making the correct choice, but we all must answer our own "Question of Balance"!

Good Luck and May God Bless OUR myeloma journey. For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

A Question Of Balance by the Moody Blues
WHY? Just because I like it!

]]><![CDATA[Enough Bad News, Some Great News For #Myeloma Patients!  The Myeloma Miracle Continues!]]>Thu, 07 May 2020 15:44:03 GMThttp://myelomasurvival.com/myeloma-blog/enough-bad-news-some-great-news-for-myeloma-patients-the-myeloma-miracle-continuesPicture
With the challenges of COVID19, it becomes hard to remember the wonderful improvements seen in myeloma over the years, and the critical part the leadership of myeloma scientists, specialists, advocates, drug companies provide and the remarkable work of the FDA and its Orphan Drug Designation, without which orphan drug progress would have languished, as it had for decades.
In September of last year I wrote the blog post(CLICK HERE) outlining how all myeloma drugs in the last 16 years had two FDA designations for each approved drug. One being an  Orphan Drug Designation,  and the other being Fast Track, Priority Review, Breakthrough, etc.  Remember there are more than 6000 orphan diseases and majority of these diseases have not one approved orphan drug.  Myeloma patients should feel blessed to know they have had 10 new drugs approved in the last 16 years.  In addition, Myeloma (an orphan disease) has  7 drugs which  all had two FDA designations, but have yet to be approved.  These drugs include the following:

Drug                             Company                 Designations      Description

AMG420                        Amgen                        OD&FT          (BCMA) Bispecific T-Cell Engager
                                                                                                (BiTE®) Antibody Construct
CLR131                         Cellectar                      OD&FT           Radiotherapeutic phospholipid drug
                                                                                                 conjugate (PDC™)
GSK2857916               GlaxoSmithKline           OD&BT          (BCMA) antibody-drug   
                                                                                                 conjugate (ADC)   
bb2121                          Celgene                       OD&BT          BCMA chimeric antigen receptor
                                                                                                 CAR T
Galinpepimut-S              Sellas                          OD&FT          Immunotherapy vaccine to elicit a
                                                                                                 strong response against WT1
P-BCMA-101                 Poseida                     OD,FT&BT      Autologous chimeric antigen receptor
                                                                                                 (CAR) T-cell therapy
Melflufen                        Oncopeptides              OD&AA         Drug activated by aminopeptidases,
                                                                                      overexpressed in myeloma
So how have we fared with this list?

AMG420 - has dropped from the list in favor of a kinder gentler similar drug called AMG701, however AMG701 has only one FDA designation.

All of the other drugs on the list are progressing very well, with GSK2857916 and bb2121 expected to be approved in the next several months. 

For the first time in 16 years,  two new drugs have been approved without dual FDA designations. However, one  is another  CD38 antibody similar to Daratumumab called Sarclisa® (isatuximab-irfc) by Sanofi for patients with relapsed refractory multiple myeloma.  Another approval is subcutaneous Daratumumab called Darzalex Faspro.

In addition we must add two more to  the list of candidates with two  or more FDA disignations. 

Drug                             Company                 Designations      Description

JNJ-4528                         Janssen                    OD&BT              CAR-T with two BCMS-targeting
                                                                                                     Single domain antibodies
CT053                             CARsgen               OD, RMAT*          A fully human anti-BCMA CAR--T                                                                                                         Therapy
* The RMAT designation is given to regenerative therapies intended to treat, modify, reverse, or cure a serious condition.  

Other drugs which already have an orphan drug designation and have the potential to obtain another FDA designation include: Amgen's AMG701(replaces AMG420), Molecular Partners MP0250, DARA BioSciences KRN5500, and SUTRO's STRO-001. 
Historically each approval of a new class of drugs (eg. Imids and Proteasome Inhibitors) have added a year or more to myeloma  life expectancy, so approval of these drugs could double the current myeloma life expectancy.  Now that is GREAT NEWS!

Good Luck and May God Bless OUR myeloma journey. For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1                                                                                     
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