BIG #MYELOMA NEWS WEEK!  #ASH19 Showed Excellent Progress Towards The CURE, & The House Passed A Drug Cost Control Bill!

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Myeloma Had An ASH Kicking In Orlando This Year!

I had an opportunity to attend the ASH(American  Society of Hematology) annual conference this year in Orlando from 12/6 to 12/10, and came away with the feeling the future of myeloma treatment is excellent!  These are my top line takeaways.

– Myeloma has a tight knit   community of researchers, myeloma specialists, patient advocates, drug companies, and myeloma organizations like the IMF, MMRF, LLS, and Myeloma Crowd.   They use Twitter and Facebook as communication tools which provides real time information to the myeloma patient community. There are more than 24 major categories of blood diseases represented at ASH, and  myeloma was frequently listed in the top 10 of ASH twittter online influencers.  Given myeloma is one of the the 24 major diseases, you would expect them to have less one top influencer in the top ten, but they had between 6 to 10 of the top 10.  Just remarkable!

–  Each new class of drug which is approved has resulted in an increase of one year in life expectancy.  We just had one new drug approved, Selinexor.   We also have 7 drugs which have a high probability of approval.  These are the ones with at least two FDA designations like Orphan Drug and Fast Track.  Effectively the ones the FDA finds  fill an unmet need and  have the most potential, kind of like the Teachers Pets of drugs.  CAR T, BiTE’s, ADC’s, AWC, melflufen, multi target CAR T, CLR131, allogeneic CAR T and many more, are all new classes of drugs which bodes well for current and future patients life expectancy.  I estimate a minimum of 6 new classes of drugs approved for myeloma which, if history repeats itself, would take the current NCI, national average life expectancy, for all patients from 6 years to 12 years.  The 15% who consult or see a myeloma specialist which now have a life expectancy of 10+ would likely see to exceed 16+ years.

–  When Daratumumab was approved in RRMM(Relapsed Refractory Myeloma) it had been approved  with an ORR(overall response rate) of 24% and a PFS of  just 1.9 months.  Daratumumab has been moving to early stages of the disease, and in combinations with EVERYTHING!  It seems “Things Go Better With DARA”!  These new combination with Darzalex are exhibiting exceptional resultsl. At this year’s ASH we have new drugs which have shown better initial results in heavily pretreated patients who have also failed Darzalex.  The newly approved Selinexor had an ORR of 26% and twice the PFS at 3.7months.  A drug in clinical trial, GSK2857916 has an ORR of 38.5% in  patients who had previously been treated with daratumumab, and a PFS of 7.9 months  This is 4 times more effective than the Darzalex PFS!  The big question is how will these and all the new classes of  drugs perform when moved to earlier in the disease process.  I think the sky is the limit!

– A big finding for me was a repeated use of the phrase “treatment used as a Bridge”.   At end stage myeloma, patients are often too sick to qualify for clinical trials.  If  treated with one of the end stage drugs like  Panobinistat, Selinexor, or many other combinations the disease may be able to be controlled enough to make it into available clinical trials. 

–  Treatments for early stage myeloma have shown excellent results,  For example high risk smoldering myeloma has a 50% chance of progressing to full blown myeloma each year, however an all oral clinical trial of IRd (Ninlaro, Revlimid, dex) had a 0% chance of progressing in the first 30 months or 2 1/2 years.  Many such trials for high risk smoldering myeloma are in process.  Soon the question will be how do we screen the population to catch myeloma in the precursor stages.  This may reduce or eliminate the devastating end organ damage associated with finding myeloma in the later stages of the disease progression.  The future looks bright!

The House Has Passed A Bill Which Should Help Patients To Get Access To Life Saving Drugs!  BUT!

This is great news for the myeloma patient community.  Cost is important, however the critical feature to the patient is ACCESS TO LIFE SAVING DRUGS!

The Congressional Budget Office estimates that the proposal would reduce Medicare drug costs by $500 billion over 10 years in three major ways:

· It would allow Medicare to directly negotiate prices for up to 250 of the mostly costly drugs that have no competition from generics or biosimilar drugs. Companies that don’t negotiate would be subject to an excise tax of up to 95 percent on gross sales.

·  The US prices of those drugs would be tied to a benchmark index of prices in other developed countries.

·  And, for all drugs, manufacturers would have to rebate back to Medicare any price increases that exceed the rate of inflation.

The most significant element of the bill for drug access is the capping of out of pocket and co pay for  Medicare at $2000 per year. Currently there is no out of pocket maximum limit.  Most all newly diagnosed myeloma patients usually have a treatment regimen including Revlimid, and the copay would be $2600 for the first month.  Under the House plan it would be limited to $2000.  Great news, if is was not for the fact 50% of prescriptions with a co pay over $2000 are abandoned at the pharmacy.  I believe Senate bill allows this to be spread across the entire year.  So what is the BUT?

The BUT is the house passed it on Party Lines, and  President Trump, who supported the idea of negotiating drug prices during his presidential campaign, now strongly opposes the House Democrats’ bill for part D.   The measure will likely die in the Senate, where Majority Leader Mitch McConnell (R-KY) may block it from ever coming to a vote. 

The Senate has a bill which the president will likely back BUT is absent one of his initial major objectives to negotiate Medicare, and Medicaid oral drug prices. This bill well likely be rejected if voted on in the House.   The concern in the Senate has to do with the thought this would limit new drug development.   Private insurance companies negotiate prices now, as do all single payer programs throughout the industrialized nations of the world.  It is also the talking point all the drug lobbyists are spinning!   It is known that 8 in 10 American’s feel Medicare should be able to negotiate drug prices.   BUT the measure does not address the problem copays are always computed as a percentage of the list price and should include provisions to make copays a percentage of the negotiated price. Under the existing system if the cost of a drug is negotiated down by 50% the copay would remain the same.  Our entire economy is based on negotiating price and trying to get a better deal by leveraging buying power or “The Art Of The Deal”.  The USA has 4.3% of the world’s population, but is estimated to be 45% of the world’s total prescription drug spending by Reuters in 2021.  As President Trump has noted the world gets a free ride.  Like China’s predatory trade practices, this must STOP!


This is a very complex problem which requires far more of a discussion than I can discuss here. I plan on providing a multi part You Tube broadcast which I will title “The BS Report”. 

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

Multiple Myeloma SPECIALIST List RESOURCES, Why is it Important to see a Specialist  By: Dana Holmes

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I can offer my opinion from a Smoldering Myeloma patient’s perspective as it relates to a Multiple Myeloma Specialist and what/why we recommend in our Smoldering Myeloma Support Group. There are “tiers” of specialists.

An MM specialist is a hematologist and likely also an oncologist who ONLY treats patients with MM. They DO NOT treat any other blood cancers/disorders (Leukemia/Lymphoma) or solid tumor cancers. The top tiered MM specialists are the ones who are conducting and publishing the research and are the Principal Investigators of the clinical trials offered to the MM community.

They are also the doctors who are presenting this data/research at their “industry” seminars and conferences, such as ASH (American Society of Hematology) and ASCO (American Society of Clinical Oncology), European Hematology Association (EHA) etc. Most are associated with large academic/university centers and cancer centers. Many are the Directors or Chiefs of their Departments and are members of the International Myeloma Working Group. IMWG members are the “governing” body of the International Myeloma Foundation. They are a group of over 200 top MM specialists from around the world…they convene annually to develop consensus guidelines which are disseminated to local hematology practices.

A general hematologist will treat ALL BLOOD disorders, including MM. A general oncologist will treat all cancers. Some of these doctors can be both hematologists and oncologists as well.

The overall outcomes for patients who are under the care of MM specialists are greater than for those who remain under the care of local community hematologists and oncologists. We highly recommend seeking at least a consult with an MM specialist and finding a local hematologist and/or oncologist who would be willing to partner with them to cover/monitor your care should you not be able to continue under the direct care of the MM specialist.

A recent study by the Mayo Clinic supported specialized care is the way to go. Read about this study in the Myeloma Crowd http://www.myelomacrowd.org/how-to-live-longer-with-multiple-myeloma/ as well as the formal study published in The Journal of Clinical Oncology: Association Between Treatment Facility Volume and Mortality of Patients With Multiple Myeloma http://ascopubs.org/doi/full/10.1200/JCO.2016.68.3805

If you want to determine if your particular doctor is conducting/publishing research, use this link. https://www.ncbi.nlm.nih.gov/pubmed/?term=Angela+Dispenzieri

Enter your doctors name along with “and Myeloma” in the search tool bar. This will bring up any research he/she has published and which has been peer reviewed.

This will work for any disease. If you want to check your physician’s research interest for MM or SMM (as an example) just link his/her name with that disease. You can try to use the term “and MGUS”, but you likely will not find much, as MGUS is part of a disease spectrum. Searching by the highest degree of a disease spectrum will likely yield search results.

If you don’t see anything listed, it is likely he/she is not actively engaged in research for this particular disease.

Do a second search and just enter the physican’s name. His/her research will be listed, and it will provide insight into their clinical interests.

Does this mean he/she is not a “good/great” doctor? No it does not mean that at all, but it infers your disease is not their primary focus. If they are not the researchers, at the very least, you want someone who attends their “industry” seminars and conferences to insure he/she is up to date on all of the emerging information. How do determine this? You ask them. Ask them what conferences they attend? You have every right to be an informed “consumer” of your healthcare. We all deserve the very best care. Many of us travel to get this level of care, as these types of top specialists are not found in great numbers.

Again, this is my opinion (but it is also opinion of many in the myeloma patient community).

MANY OF OUR GROUP MEMBERS HAVE LISTED THEIR MM SPECIALISTS IN THE COMMENT SECTION OF THE GROUP MEDICAL DISCLAIMER DOCUMENT …click on this link to review those comments https://www.facebook.com/notes/smoldering-multiple-myeloma-aka-smm/medical-disclaimer-for-smoldering-multiple-myeloma-aka-smm-facebook-group-est-12/1430234153886158

Our Member’s Multiple Myeloma Specialists Listing
https://www.facebook.com/notes/140081448349479/Our%20Member’s%20Multiple%20Myeloma%20Specialists./1784708618438708/

Why Should Myeloma Patients Visit a Specialty Center? – See more at: http://www.patientpower.info/video/why-should-myeloma-patients-visit-a-specialty-center#sthash.JgqNvYbc.dpuf

Multiple Myeloma Specialists and Hospitals or Treatment Centers ** http://myelomasurvival.com/myeloma-specialists-listing.html

** Please note.. Both of the lists provided in this link from Gary Petersen may contain the name of the Director or Chief of the Myeloma Specialty group only…it may not capture all of the specialists within that group…if you don’t find the name of “your” specialist on either of these lists, just try to determine if his/her Director or Chief is listed…this will assure you they are Myeloma/MGUS specialists and associated with the Director or Chief who is listed.

Multiple Myeloma 2017 Update – Why Do People Beat the Average Myeloma Life Expectancy Prognosis? Or How To Improve Your Multiple Myeloma Survival Rate!
http://myelomasurvival.com/myeloma-blog/multiple-myeloma-2017-update-why-do-people-beat-the-average-myeloma-life-expectancy-prognosis-or-how-to-improve-your-multiple-myeloma-survival-rate

Listen to the Myeloma Crowd radio interviews with many of the top myeloma specialists in the world (they provide transcripts if you prefer to read) >>> http://www.myelomacrowd.org/myeloma-crowd-radio/

Multiple Myeloma Research Foundation (MMRF) / Multiple Myeloma Research Consortium Member Institutions
http://themmrc.org/about/member-institutions/

Multiple Myeloma Research Foundation (MMRF) list of Myeloma specialists by state http://www.themmrf.org/living-with-multiple-myeloma/find-a-treatment-center/

The International Myeloma Foundation (IMF) / International Myeloma Working Group (IMWG) Members (Please note, although most members listed are hematologist/oncologists, some may be Radiologists or Metabolic Bone Disorder Specialists, as an example. Best to search their names to find their specialty ) (Also, please note, the IMF has not updated this list, some of these specialists have transferred to other Myeloma centers). http://imwg.myeloma.org/imwg-members/

A video from Dr. Brian Durie, Chairman of the International Myeloma Foundation (IMF) with advice how to locate a Myeloma specialist. https://www.youtube.com/watch?time_continue=12&v=weBGoq7YkYQ

Here is another resource to locate MM specialists (it has some outdated info it seems, doesn’t seem to have captured some moves/relocations, so be sure to double check the names you may find and want to consult with against some of the other lists or just by googling the specialists name to see where they are practicing now.) http://www.expertscape.com/ex/multiple+myeloma

Who’s Who @ the Mayo Clinic : List of MM Specialists http://msmart.org/

Here is a excerpt from a Myeloma Beacon Discussion (April 3, 2014) http://www.myelomabeacon.com/forum/centers-of-excellence-t3088-10.html and a link to Bone Marrow/Stem Cell Transplant Centers in the U.S.

Re: Multiple myeloma centers of excellence by

Dr. Edward Libby on Thu Apr 03, 2014 1:31 pm

Hello from gray (but warming up) Seattle,The following link is to US transplant centers that perform allogeneic stem cell transplants. Even though allo transplantation is not the standard transplant option for multiple myeloma I am posting this link because all allo transplant centers perform auto transplants as well. In general at these centers two out of three transplants are autologous transplants and the majority of autologous stem cell transplants are for multiple myeloma. Centers that perform both autologous and allogeneic transplant are going to be larger and busier. With a larger center that does more transplants there is generally going to be more experience andexpertise. http://bethematch.org/For-Patients-and-Families/Getting-a-transplant/Choosing-a-transplant-center/U-S–transplant-centers/

Types of Bone Marrow Transplants http://www.hopkinsmedicine.org/kimmel_cancer_center/centers/bone_marrow_transplant/types_transplants.html

Here is a good search tool to use to find the research interests of your MM specialists (type their name in the toolbar)
http://www.ncbi.nlm.nih.gov/pubmed/?term=ola+landgren


If History Repeats Itself, The State of Myeloma New Drug Development Is STRONG! The Myeloma Miracle – Part 3

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In the last 16 years multiple myeloma has had 10 drugs approved. All of these drugs have had two things in common, they all were awarded an FDA Orphan Drug(OD) designation, and secondly they all had one of the additional FDA designations of Fast Track, Breakthrough Therapy, Accelerated Approval, or Priority ReviewL.  NO new drug has ever been approved for myeloma in 16 years without at least two of these FDA drug approvals.  To be an orphan drug it must first affect no more than 200,000 patients in the US. The FDA’s Office of Orphan Products Development is tasked to evaluate scientific and clinical data from companies to identify which therapies can improve the lives of people suffering from rare diseases. Orphan status is given to drugs and biologics defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders”. The other destinations are designed to speed the availability of drugs that treat serious diseases, especially when the drugs are the first available treatment or if the drug has advantages over existing treatments. The Food and Drug Administration has developed four distinct and successful approaches to making such drugs available as rapidly as possible:

  • Priority Review (PR)
  • Breakthrough Therapy (BT)
  • Accelerated Approval (AA)
  • Fast Track (FT)

A more in depth description of these additional designations and the advantages of getting them is available if you CLICK HERE. 

Those drugs  approved for use in mutiple myeloma in the last 16 years and the date of FDA approval for clinical use is as follows:

Velcade       2003            Pomalyst      2013           Ninlaro          2015                           

Revlimid      2006            Faradak       2015           Darzalex       2015                 

Doxil            2007            Empliciti       2015          Selinexor      2019 

Kyprolis       2012     
                                 
How can history repeat itself?  First if we find what drugs which have at least  two FDA designations but are not as yet approved by the FDA for clinical use,  this just may be a very good list to see what the next FDA approvals likely will be.  I count 7 drugs which have an orphan drug and one other FDA designation.  For all of the drugs above which have been approved the average time from orphan drug designation to FDA clinical approval is 4 years, as compared to an average of 8 years without these two designations.  A remarkable feat of twice as fast to market.  Those 7 new drugs are with dual FDA designations are:

Drug                             Company                 Designations      Description

AMG420                        Amgen                        OD&FT          (BCMA) Bispecific T-Cell Engager
                                                                                                (BiTE®) Antibody Construct
CLR131                         Celectar                      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

Many more drugs have an OD designation, but as yet have not received the second designation. So additional likely candidates could be added if and when they receive an additional designation by the FDA.  This is remarkable when you consider the average orphan disease has less than one approved drug for each of the 6000 orphan diseases!  If history repeats itself, I would argue we have a high probability of having 7 new drugs approved for the treatment of myeloma within 4 years or a rate almost 3 times that of the historic rate of myeloma drug development.  This would be absolutely miraculous!  I must thank the FDA with government support for putting in place the Orphan Drug Act, but again there is a synergistic magical sauce in the myeloma care community which has made myeloma a template for rare disease drug development.  All the players in this community which includes the researchers, academics, myeloma specialists, patients, caregivers, patient advocates, large and small drug companies, supportive care teams, NCI, FDA, IMF, MMRF, LLS, Myeloma Crowd, and all those I may have left out, work in a talented cooperative team environment!   

In this group of drugs are 6 new drug classes, and for each new class of drugs approved by the FDA we have had a 1 year increase in life expectancy.  If history repeats itself, 6 new drugs would mean 6 more years of life expectancy for all myeloma patients, and 6 more steps towards THE CURE.

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1


The Multiple Myeloma Miracle (Part 2) –¬† Does The FDA Have A Crystal Ball?

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A recent study for drug development shows that only 1 in 10 drugs which enters into a clinical trial is approved by the FDA!  The article sates:

Washington, D.C. (January 9, 2014) – Today, Nature Biotechnology published a peer-reviewed paper co-authored by the Biotechnology Industry Organization (BIO) Industry Analysis and BioMedTracker (BMT) highlighting results of a study showing that the overall success rate for drugs moving through clinical trials to FDA approval from late 2003 to the end of 2011 is near one in 10. Previous reports, taken from earlier years, showed the rate of drug approvals is one in five to one in six.

Before Congress enacted the ODA (Orphan Disease Act) in 1983, only 38 drugs were approved in the USA specifically to treat orphan diseases.  Which is just less than 6 drugs for every 1000 rare diseases.  Now that is just appalling!  But since this act was put in place another 503 drugs were approved for orphan drugs or 8 per 100 rare diseases, more than 10 times as many drugs.  To become an approved orphan drug, a drug company must first apply for a “orphan drug designation”, if they are approved for a designation, they receive many benefits, but must still go through the rigorous FDA approval process.

If we look at the most recent data and compare new orphan drug approvals by the FDA compared to the number of recent orphan drug designations, their success rate is 1 in 10, or very close to that for all cancers.  This is a little different measure than the clinical trial numbers for all cancers because the designations are based on a FDA review of an application presented to the FDA by the drug companies, and many may not be in clinical trial.  They are most likely not in clinical trial because the FDA provides a 50% tax break on all clinical trial costs, so why spend on clinical trials until you qualify for the rebate.  The number of designations are therefore probably much greater than the orphan drugs on clinical trial.  The likelihood is the actual approvals for drugs given an orphan drug designation that reaches clinical trial is much greater than are the ones based on the number designated as orphan drugs.  Hope I did not lose you because I did not have access to the number of orphan drugs on clinical trial which made it to FDA approval, so I use the designations as a surrogate.   What is absolutely remarkable is drugs now designated as orphan drugs have the same probability of approval as do all drugs in clinical trial.

How does the FDA come into play on these outcomes?  What about this crystal ball? And how does the Myeloma Miracle fare in this type of comparison?   The FDA requires an application process for a drug to be approved.  The documentation is then reviewed by the FDA.  When reviewing a request for orphan drug designation, FDA considers the mechanism of action of the drug to determine what distinct disease or condition the drug is intended to treat, diagnose or prevent. Whether a given medical condition constitutes a distinct disease or condition for the purpose of orphan-drug designation depends on a number of factors, assessed cumulatively, including: Pathogenesis of the disease or condition; course of the disease or condition; prognosis of the disease or condition; and resistance to treatment. These factors are analyzed in the context of the specific drug for which designation is requested.  If the FDA approved the designation, they believe the drug to be a good candidate for the intended orphan disease, provide financial incentive, fast track the approval, and help the approval process with incite into what is required to obtain FDA approval.  The FDA becomes an invaluable asset. 

For myeloma the FDA seems to have a BIG SHINY and almost flawless crystal ball!  The process of approval for drugs from designation as an orphan drug to FDA approval is 5 years, so if I look back at 2010 through 2014 designations I should be able to capture most of the approvals from 2015 through 2018.  When I do this there were 21 designations for Myeloma and 9 approvals.  This would be a 42% chance of an orphan drug designation for myeloma being approved by the FDA versus 10% for all drugs and for the recent experience for all orphan drugs.   Four times more likely to approved than for any other drug! 

What are the take a ways from this posting?

1) The FDA orphan drug designation process provides a significant improvement in the approval of all orphan drugs!   From 38 total orphan drugs to 548 total drugs.

2) Even with this improvement there is still just 548 orphan drugs for the 6000 orphan diseases.  Less than 1 in 10 orphan drugs has an orphan disease specific drug.  It means we must have many more orphan drug designation applications  because 1 in 10 will get approved! More applications means more approved drugs.

3) The Myeloma Miracle continues with a total of 19 approved drugs!  CLICK HERE.
To have 19 total drugs, myeloma must not only have 4 times advantage in approvals, but must have many more orphan drug designation applications entered. I become more astounded by the progress made by the Myeloma Treatment Community (researchers, myeloma specialists, patient advocates, drug companies large and small, caregivers, patients, FDA, NCI, IMF, MMRF, and Myeloma Crowd)

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1


The Myeloma Miracle –¬† A Rare Success For a Rare Disease

In the United States the NIH (National Institute of Health) defines a rare or orphan disease as one which has fewer than 200,000 people. There is an estimated 6000 to 7000 rare diseases.  I recently read a report about the success of the 1983 Rare Disease Act.   It has been instrumental in bringing new drugs to market for Orphan Diseases.  Prior to the law there had been limited to no success, and that is why this act was put in place to provide financial incentives for drug companies to pursue orphan drugs.  Their success is obvious in the following graph.
Approvals of new orphan drugs went up from near zero each year to an average of 25 each year for the last 10 years.  So in the last 10 years there have been around 253 new drugs approved for rare diseases in the US.  This is exceptional progress and improvement, and can be explained by the financial  incentives and the reduced time for approval of the rare disease designation by the FDA.  But if you use the lower estimate of 6000 rare cancers, the average new drug approval per rare cancer is .04 new drugs per rare cancer.  Yes, that is just 4 new drugs for every 100 rare cancers.  As you know each new drug has improved myeloma life expectancy, and in the last 10 years we have had 7 new drugs approved for myeloma!  Myeloma has had 175 times the average drug approvals for rare diseases in the last 10 years. 

In addition, we know that every new drug provides a longer life expectancy.  In the last 10 years the National Cancer Institutes SEER data base has shown life expectancy for myeloma going up from 3 years to now 6 years or a 100% improvement.  Miracle, or the secret sauce, of a dedicated and oh so talented group of researchers, myeloma specialists, myeloma organizations(IMF, MMRF, Myeloma Crowd, LLS), FDA, NCI, advocates, care givers, drug companies big and small, and care teams.  I wish someone could bottle this secret sauce and make it available to the other 5999 rare diseases!  We have been blessed with an abundance in new drug development. 

One thing for certain is that if the life expectancy goes up by 100%, so does the cost to keep this chronic disease in check and  the need for the next new drug until a cure is developed.   Will we get the next new drug if there is little if any financial incentive for the drug manufactures?  Based on the results of the Orphan Drug Act, incentives provide improved drug supply! 

For now let’s just celebrate our Myeloma Miracle, and hope and pray it continues until we find a cure, and quickly!

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

Manipulation of the Stocks of Micro Cap Biotech Companies is KILLING Cancer Patients!

PictureCapital Vultures Attacking Children, the Sick, and Frail! Just a MONEY Game to them! But LIFE OR DEATH to us!

Why do I care about this subject? I am a multiple myeloma patient, and have been kept alive through the development of innovation new drugs for the treatment of myeloma. Multiple Myeloma is an incurable cancer of the blood and has an average life expectancy of just 5 years. It is also a rare disease often called an orphan disease. Most of the innovations for this disease has their genesis from small bio tech companies (baby or micro cap biotech). I became interested in this issue because I follow all news on new myeloma drugs and the companies which are developing them. I also invested in some of them, and watched them closely. What I notice was even though a company put out a press release on a clinical trial showing good results, the stock price would go up, but then it will go back down lower than it started. Often in the same day. I am an engineer and have an MBA, so this made NO SENSE to me.  Therefore I decided to do a little digging and uncovered some unnerving and frightening findings!

How on earth can the manipulation of stock impact the lives of cancer patients? If you look at  how new drugs are developed, you will find many begin in academic teaching hospitals or through the support of NIH(National Institute of Health) grants to universities and start up bio techs. You can look on line and you will find different estimates from 42% to 100% of new drugs developed. CLICK HERE or CLICK HERE.  The NCI(National Cancer Institutes) usually provides funding to those bio-techs which really show great potential, and also for those which focus on rare cancers like Triple Negative Breast Cancer, Rare Pediatric Cancers, High Risk and End Stage Myeloma, Mesothelioma, etc. The NCI has a focus on Rare Cancers because generally rare cancers do not have the numbers like Lung or Breast Cancer to interest the Large Biotech Companies. Twenty percent of all cancer patients have rare cancers, and have been under served. This shows up as a 5 year rate of death almost twice that of the most common cancers. However in Pediatric Cancers 72%, not 20%, of all cancers are rare cancers. CLICK HERE.

If these are rare diseases and do not represent a large number of patients then why should we worry about them? Well, each of these cancers may in itself be small in number, but together they represent 20% of all cancers or 350,890 rare cases each year in the USA. So if the baby bio tech are forced into bankruptcy,the drugs to treat these diseases will never make it to market. Therefore, the process for most new drug development collapses for rare, and for that matter all cancers!

I have explained the process of how and why these Capital Vultures raid these small vulnerable micro bio techs through the use of predatory attacks (short sales) of the baby biotech stocks. This drives down the price of the stock to near worthless levels forcing these companies into bankruptcy. You can read this explanation if you CLICK HERE and CLICK HERE. Without these companies we will have fewer and fewer new drugs and treatments, and more cancer patients WILL DIE.

I will give you just one of hundreds of examples. SELLAS Life Sciences (SLS) is a small biotech company who has had its capital base destroyed by these attacks. In just one year the stock has lost 92% of its value through these unlawful attacks. So to fund their clinical trials they must now issue 11 times more stock to obtain the same funds by selling stock. Maybe this is a bad company, and they deserve this valuation? This may be true if this company has not developed new drugs now in clinical trial which show excellent results in the undeserved cancers, Triple Negative Breast Cancer, Mesothelioma, and High Risk Myeloma. 

The knowledge of this predatory activity is widespread, and has been debated for 20 years, but because it has only talked about the loss of capital it falls on deaf ears, or is justified as improving market liquidity. It does increase liquidity by stealing funds invested in these companies by NIH, retirement investments, and investors and poring it into the accounts of the Capital Vultures and out of the biotech companies. A complete though long explanation of this activity can be read if you CLICK HERE.

So much has been written about this, I am sure this will fall on deaf ears as well. We can only do our best to let people understand this is not just money. Money, like material things can be replaced, but when you are looking for the next line of treatment for your cancer and it does not exist because of greedy, heartless, criminal, morally corrupt, and probably rich Capital Vultures, these next new last chances for life drugs will not exist. Please help to give this the attention it demands of your government representatives, or you can do what I have done is to send twitter requests to some of the people who should be able to make a difference with a link to this blog post. Some twitter addresses of influential people are: @maziehirono @TNB @JoeBiden @realDonaldTrump @RepJoeKennedy  @SecAzar @SEC_Enforcement @SenateGOP @TheJusticeDept @FDACommissioner @FLOTUS  @SenSanders @NIHFunding @HHSGov @RepCummings @BCAction @SpeakerPelosi @SenWarren @JusticeATR @NIH  @DavidP4AD @Myeloma_Doc @USATODAYhealth @NYTHealth @SeemaCMS @mtmdphd @cure_talk @Myelomaauthor @US_FDA @FDAOncology @SagarLonialMD @Rfonsi1 @myelomadoc @VincentRK @TargetedOnc @AmericanCancer


I live in Florida, and sea turtles are endangered. They are endangered because they have been slaughtered for their eggs, meat, skin, and shells, and predators have found the nests as easy pickings. This has made sea turtles a protected species. In addition, the nests are found and fences are installed around them to prevent the nests from being raided by predators, and signs posted for humans stating messing with these nests is a crime! If we can protect turtles before they become extinct, why can’t we protect micro cap biotech companies from the Capital Vulture predators, so endangered baby bio-techs do not go extinct. How many LIVES CAN BE SAVED?

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

A Myeloma Patient’s Guide To The Best Of ASCO 2019 By Dr. Joseph Mikhael

If you are like most of the myeloma patient community, you will not be attending  the large conferences where the best myeloma specialists in the world congregate and discuss the current and future treatment options.  So how do we find out about the best and most promising current and future treatments presented at these meeting?  One way is to follow the action at the conferences through myeloma focused twitter, Facebook, and other online options.   However, I have found that some people have a better perspective than most, and they are the myeloma specialists who attend these meetings.  

So recently, Dr. Joseph Mikhael offered to provide a myeloma specialist’s view on the best of the best for ASCO(American Society of Clinical Oncology) 2019.  Dr. Michael is not just any myeloma specialist, but is the Chief Medical Officer of the International Myeloma Foundation. He is tasked  to advance the mission of the IMF in research, education, advocacy and patient care to improve lives of patients with multiple myeloma..  The IMF is the oldest and highly respected patient focused myeloma support organization. Therefore Dr. Mikhael provides an invaluable perspective and has an outstanding perspective for the CURE TALKS myeloma broadcasts.  The Broadcast is scheduled for 6/5/19 at 4PM EST.  You can sign up for this broadcast and enter questions for Dr. Mikhael if you CLICK HERE!  

Dr. Mikhael has provided a summary for ASH(American Society of Hemotology) 2018 which was just 6 months ago. ASH and ASCO are two of the largest conferences where myeloma has a significant representation. I have provided a 4 minute snip-it of his ASH experience, and you can expect a full hour of his expert opinion for ASCO19, and have the opportunity to ask your questions for the last 15 minutes.  

Another myeloma specialist,  Dr.  Michael  Thompson of Aurora Heath Care, Milwaukee provided an excellent summary of the best of the best abstracts of ASH 2018 last December.  Prior to ASCO 2019 Dr. Thompson  has provided a similar summary.  If you would like to do some pre CURE TALKS research I am providing his excellent summary below.

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If you might notice, even the experts do not have a unanimous consensus on any one abstract.  So it will be very interesting to find out what the IMF Chief Medical Officer feels to be the most significant ASCO findings. Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

Myeloma Life Expectancy – The Good News And The NEW PROJECTION!

In April the National Cancer Institute Published the SEER Cancer Statistics, and it showed myeloma life expectancy had improved from 5.5 years to 6 years.  For a the last few years I have been projecting the change in myeloma life expectancy.  The SEER data for myeloma patients can be found if you CLICK HERE.  My estimate for last few years have been spot on.  If you want to look at the history of these projections you can CLICK1, and CLICK2.

Below is a look at the history of the survival for myeloma in the USA and my prediction for the next 1 year improvement. 

year                                  survival milestones        years between milestones

1975                          2 years                               –

1998                          3 years                             23

2004                          4 years                               6

2008                          5 years                               4
 

2010                          6 years                               2

2014(projection)        7 years                               4

The rate of change for myeloma improvement looks to have once again plateaued for a few years.  The 5 year survival has remained nearly the same for the last 4 years which does not bode well for a big move upward during those years.  To really impact survival we must slow the high death rate in the first three years after diagnosis.  With early testing with the Promise Study, the work with iStopMM, and early treatment for high risk smoldering myeloma  slowing early death may be achievable. 

Another part of this is because a number of new drugs were approved in 2015, and the improvement for these drugs will not be represented in most of the current data published by the National Cancer Institute.  But still if 35% of patients die in the first 3 years there is just 15% more to die to be at the median.  Early detection is the key to significant improvements becoming a reality. 

It is still a mystery to me why all the data provided by myeloma specialists show a 3 year survival in the 85 to 95% range and the SEER data for 3 years is just 65 to 66%.  Which would mean you are 3.5 times more likely to die at the average SEER facility vs. at one of the centers with a myeloma specialist. 

In addition, the likelihood of any improvement next year will be limited by a small backwards move in the 5 year survival as noted int the following graph.

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1

Help To Give End Stage Myeloma Patients A New Last Chance!  HELP, We Need Everybody’s  HELP!

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Cindy Chmielewski (or on twitter @MyelomaTeacher) does not give up easily.  She is a Myeloma Patient Advocate and works tirelessly to provide assistance to the myeloma patient community.  She was at the FDA advisory meeting which was to provide a nonbinding recommendation on the approval of Selinexor for end stage myeloma patients who have exhausted all available drugs.  She and all myeloma patients, advocates,and myeloma specialists had recommended the FDA give these patients the opportunity to have access to the drug.  I believe the draft document which was reviewed was quite negative, but all of the questions raised were answered by the myeloma patients, myeloma specialists, and advocates to their satisfaction.  Selinexor has been given positive feedback by myeloma specialists almost universally, so the fact the approval was delayed for at least 2 years was heartbreaking. One interesting comment on twitter by the Patient Advocate Yelak Biru stated, “there is precedence where the FDA has approved Panobinistat despite negative vote from the independent ODAC committee. There may be a potential for them to do that as these cohort of patients really need an option.”  Cindy wrote the following request, and I will write the FDA, and pass it on to all of you to do the same.  The panel was influenced because the drug can be used on a compassionate use basis, but the myeloma specialists made clear the 85% of patients being treated at community hematologists would not recieve Selinexor due to the lack of available necessary resources at  community practices.   Together we can SAVE LIFE! 

Cindy <lawcynthia@aol.com>
To:Myeloma community
Mar 6 at 10:02 AM

Hello My Friends,

Many of you were also disappointed in the outcome of the ODAC meeting on Selinexor.  There is still time to voice your concern.

Below are the email and phone contact details for Ann Farrell and Nicole Gormley, who are leaders at FDA responsible for Hematology/Oncology drug approvals at FDA, should you or other advocates decide you would like to reach out. Feel free to share this with others.

Contact Information

Richard Pazdur, MD
Director, Oncology Center of Excellence
Acting Director Office of Hematology Oncology Products
301-796-2340
Richard.pazdur@fda.hhs.gov

Ann Farrell, MD
Director, Division Hematology Oncology Drug Products
301-796-1352
Ann.farrell@fda.hhs.gov

Nicole Gormley
Cross-Disciplinary Team Leader and Supervisory Physician
240-402-0210
Nicole.gormley@fda.hhs.gov

Thanks,

Cindy

Thank You Cindy and Yelak.  Selinexor is a new class of drugs, and each new class of drugs has increased myeloma life expectancy by 20 to 25% from 3 years to nearly 6 years.   


Cancer Patients Beg For Help But The Government, Drug Companies, PBM’s, and Insurance Companies Point Fingers While Patients DIE!

Only one copay program out of 7 is now open for myeloma and we are just through Feb.  This is the real domino theory.  Once one falls, it puts pressure on those that are open resulting in all of them closing in short order.  Two years ago I wrote  a blog post headlined Can You Hear It Now? The Jerry-rigged #Myeloma Safety Net Is Getting Shredded! when in March the co pay programs were running out of money.  It got progressively worse for months.  I   had once thought this issue only applied to multiple myeloma with only 30,000 new patients each year. Myeloma is considered an orphan cancer which has gotten much less  focus  than other cancers like lung or breast cancer,  which have 10 times more newly diagnosed than myeloma.   However it is plain to see it affects most of the  1,735,000 cancer patients newly diagnosed each year. PANF(Patient Assistance Network Foundation) is one of the largest of the copay programs and provides assistance for 69 different diseases, but currently 51 are closed or out of funds.  That is 74% of the total and it is just 2 months into the year.  People depend on these programs and they have proved to be unreliable.  Who would accept a 25% service level.  No one!  A short primer on how we got here!

–  Medicare provided drug coverage for Medicare patients in 2006 – Good
– But to get it signed into law Drug Companies got a guarantee the price was a nonnegotiable list price – very bad
– Whenever foreign countries or PBM’s for private insurance push net prices down (they can negotiate for lower prices) drug companies increased list price -very bad
– PBM’s wanted higher rebates from drug companies because the drug companies list prices went up –  very bad
– Medicare would not allow co pay for Medicare patients managed by the drug companies to get more generics used – misguided and deadly
– Insurance companies got lower net prices, but if the list was $100 and the net $50 they charge the copay % at list –  very shady
– Prescription prices have increased over 100%(more for cancer drugs) since 2006 while inflation was less than 25%. –  co pays went up more than 75% –  very bad
– Drug companies funded third party co pay programs like LLS, PANF, PAF, etc to help patient afford their drugs – good for patients
– In 2017 drug companies underfunded the third party co pay programs,  patients did not get drugs – patients died. 

It got better in 2018, but we are now in what I will call “The Circle Of Death” because players(PBM’s,Drug Co.,Govt, Insurance Co) game the system and patients lose.  PANF has completed a survey of patients receiving co pay for the diseases they cover.  Co Pay is critical to the health and well being of the patient. You can read it if you CLICK HERE. They note  half of all patients skip doses prior to obtaining copay assistance, and with the high cost of cancer drugs it  is most likely far greater.  During cancer treatment this will reduce efficacy.  The players are killing there customers and their cash flow!

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It does seem to be a SNAFU!  We need to change SNAFU to SWAP(Situation Wonderful All Patients).  So what can be done to correct this SNAFU in the short and long term?

Short Term Solution( Fix the present system)

– Drug Companies MUST increase their contributions to the third party co pay assistance programs.  All  available myeloma  drugs will be used in the course of myeloma and also for most late stage cancers, so ALL drug companies must BUCK UP!
– The government should allow drug company directed co pay programs if there is no generic or bio-similar available.  This should achieve the same generic drug use but simplify the current convoluted process. 

Long Term Solution(The permanent fix)

– You have heard a lot of the best ideas, let Medicare Negotiate Prices, PBM’s negotiate a best price without rebates, have transparent pricing for drugs and healthcare services, provide a pricing review board for all patented drugs, have a maximum monthly total co pay limit of $100 to $150, etc.  A very comprehensive review of what needs to be accomplished in the area of drug pricing is outlined in a document titled Addressing Out Of Control Prescription Drug Prices, Federal and State Strategies. To View CLICK HERE.

Until this safety net is repaired, patients will fall through the gaping holes in the net. The system had worked for those who found these assistance programs, but now these patients are at risk as well.  I found out about the LLS program at an IMF support group meeting, and I, like most  myeloma patients, had to stumble onto the programs.  Cancer patients should be counseled on available assistance by the care team.  At least a brochure headlined “Assistance for the High Cost of Care and Drugs”.  So it is time for the talking to stop and for Government to TAKE ACTION!  I have likened this withholding of medication from the poor, disabled, and seniors as unintentional de facto GENOCIDE.  With the recent congressional meetings and the testimony of families who have lost loved ones because of this SNAFU, it can no longer be considered unintentional!

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1