Pat’s Place: Clinical Trials Need To Include All Of Us
Just when you think you’ve got this myeloma thing figured out, there’s so much to learn all over again.
Looking ahead, most of us have an idea about what we’d do when we start running out of FDA-approved drugs to help us: join a clinical trial. And there are literally hundreds of them for multiple myeloma patients.
So no worries, right? Simply pick one from column A, B, or C, and away we go. Hopefully, our doctor can help us find one that is likely to work for us and recruiting patients close to home. After all, that’s what clinical trials are designed to do; help us live longer.
So why should I be surprised that sometimes it isn’t as easy as it sounds?
Have you had an allogeneic (donor) stem cell transplant? Sorry, most trials won’t let you in. Others exclude patients that have had two or more autologous stem cell transplants.
Are you a nonsecretor like me? Very few trials make allowances for us. Too tough to measure and standardize results, I guess.
Trouble is, the number of nonsecretors is increasing. It turns out that many late-stage myeloma patients become nonsecretors over time. Apparently, older myeloma cells tend to secrete less measurable protein.
Ironic, isn’t it? More and more patients are being excluded from trials at a time when there is a shortage of participants; researchers are begging for more. And, by excluding patients like these, many trials are inaccessible to those of us who need them most.
There are a few, outside-the-box, long-shot trials that allow one or more of the above categories to join. But, if you guessed they tend to be the ones least likely to help us most, you’re right.
I did learn about a new trial that may allow nonsecretors from a doctor at the Mayo Clinic in Jacksonville the other day. But it is only a Phase 1 trial, meaning the primary goal is to determine the safest and most effective dosing, not necessarily keep patients alive.
Get me in a Phase 2 or 3 trial, featuring one of the new immunotherapy drugs! But so far, I’m out of luck. I have been considering a modified allo trial, using engineered T-cells. But I’m concerned about the time commitment needed to do an allogeneic trial (three or four months away from home), as well as being limited from accessing more trials in the future. Also, it’s risky. The approach may minimize graft-versus-host disease (GVHD), which should make the transplant safer. But many myeloma experts believe there needs to be some graft-versus-tumor effect (which goes hand in hand with GVHD) in order for an allo to control multiple myeloma.
Which brings me to the million dollar question: Assuming a patient can get in to a trial, how will we know it’s the right trial for us?
Not having enough choices isn’t good. But too many options can be problematic, too. If you are fortunate to qualify for several different trials, how do you know which one may work best and help you most? Patients need better direction as to which trial to pick. Select the wrong trial near the end, and you may not get another shot.
May I make a few suggestions?
First, include all myeloma patients in clinical trials. It seems silly to exclude any late-stage patient from a trial. Researchers, drug companies, and patients need all the help they can get!
Second, our doctors need to be more knowledgeable about which trials may help us most. It shouldn’t be as difficult as it seems. If we’ve had a good (or not so good) experience with one or more drugs from a particular class, start there. I managed to locate several different clinical trials that seemed like good fits. For example, one used an FDA-approved drug that I hadn’t tried yet as part of the control arm, combined with an experimental immunotherapy drug I’d heard good things about in the experimental arm. Either way, I win! Unfortunately, that’s before I learned I didn’t qualify for this and so many other trials because of the nonsecretor thing.
Third, trial-related expenses should be covered for trial participants. I had been led to believe that my medical expenses would be covered after I enrolled in a clinical trial. Not always true! Remember the modified allo trial I referenced earlier? Researchers expect the patient to pay for the transplant – that’s the most expensive part. The trial will pay for modifying the donor T-cells to be injected back into the patient, but not the transplant itself.
We all understand that our healthcare system isn’t perfect. Unfortunately, neither is the way clinical trials are designed and enroll patients. Here’s hoping that more trials will begin accepting a wider variety of patients, providing better direction for when and why a patient should join, and paying for their trial-related expenses.
Clinical trials serve a dual purpose: to help bring new and more effective drugs to market, and to help save patients’ lives. Here’s hoping that researchers don’t get so caught up in selection criteria and the numbers that they forget about the life-saving part.
Feel good and keep smiling!
Pat Killingsworth is a multiple myeloma patient and columnist at The Myeloma Beacon. You can view a list of all his columns here.
If you are interested in writing a regular column for The Myeloma Beacon, please contact the Beacon team at .
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I think I understand why they do it...keeping the known factors (patients) as homogenous as possible. Also, a drug that kills too many patients gets bad PR and that kills the drug company. So they probably want the cream of the crop of eligible MM patients.
Seems there should be a way of signing along a dotted line where you understand the risks and accept the consequences. Also, this information would not become part of an official trial where it could hurt a drugs chances of being approved.
Sorry you're having to navigate these waters that most of us will have to also navigate someday.
Take care Pat. And just so you know, I am currently smiling and I feel pretty good--ha.
Pat, I'm with you. I'm also a late stage myeloma patient who is not eligible for most clinical trials (low counts due to 4 & 5 drug combo cocktails while trying to find trials/ have to show disease progression but if I do that I won't make it to the trial). If I read one more time about how more patients need to sign up for trials I'm going to scream. I'm healthy in all other ways except myeloma. This does not make sense to me.
Hi Pat,
This following item from your article caught my eye. "The approach may minimize graft-versus-host disease (GVHD), which should make the transplant safer. But many myeloma experts believe there needs to be some graft-versus-tumor effect (which goes hand in hand with GVHD) in order for an allo to control multiple myeloma."
I spoke with a young lady volunteering at the infusion center I go to. I asked her why she was volunteering and she said that she was a cancer survivor, leukemia. She had an allogeneic stem cell transplant; exact match from her sister. She also mentioned she did not have any GVHD and that worried her doctors for the reason you mentioned, but still she's now free of the cancer. So maybe the concerns doctors have about no GVHD, no cure isn't the last word.
All the best, Steve
Yes, Stan, I understand why researchers do it this way; you're right. But read Nancy's comment. Spot on. I'm in a similar situation. Never said making it better would be easy...
Steve's right, too. GVHD may not be necessary. Or maybe it is for some and not others?
Excellent article that echoes a comment my oncologist made a few appointments backs. He said it used to be that the life expectancy for myeloma patients was low for most--so he is not convinced the oncology world has a real understanding of what myeloma looks like 7 years, 10 years, 15 years out. "We rarely had to deal with that scenario." And as you point out, now that they have to deal with this issue, the clinical trials world is not prepared. I understand homogenous groups and the need for that in trials, but you'd think one could find enough patients in the late stages that appropriate trials could be conducted.
Pat, the timeliness of this article for me is perfect. Thank you!
April, glad you brought that up. I'm sure this is one of the points he was trying to make: the drugs we're using were never designed to be used for five years at a time! FDA safety testing for a lot shorter time. Still, what choice do we have? Even more choices! Bring on elotuzumab, ixazomib, daratumumab...
I am caregiver to my wife (IgG, 17p deletion, diagnosed 6 years ago).
None of the previous six courses of treatment was effective for more than six months, including a SCT, RVD, and Pom. She has been on a Phase 2/3 trial of Darma now for 14 months. The most effective treatment to date and seems to be approaching VGPR. Fingers crossed.
I recently completed a five week Coursera online course - Designing and Interpreting Clinical Trials - from Johns Hopkins. It's online and free.
I highly recommend it for better understanding the complexities that Researchers face when designing trials. Once armed with this knowledge, you may be able to identify more suitable trials for you and you will absolutely understand the parameters that restrain researchers.
My husband has high risk late stage myeloma. He was told repeatedly that he did not qualify for clinical trials because of his 20% kidney function. He was put on panobinostat a month ago. His light chain number was in the 300 range at the time and shot up to 26,435 in two weeks. Needless to say, we wished he never took the drug and that his doctor had tried Pomalyst, Kyprolis and dex instead, as he had told us that he would before panobinostat became available. Now he's out of options and told to look for hospice care.