If you haven’t seen it yet, watch this YouTube video which has gone viral among the multiple myeloma patient community:
No question that it is an exciting presentation! But everyone should probably take a step back and calm down a bit.
Why? How could I be critical of such a hopeful video?
Well, critical is too strong. I wouldn’t even say “skeptical.” But like most things in life, there may be more layers of grey here to peal away than I would like to see.
If you read my headline, I’m already mixing my metaphors. Sorry about that! Here are a few things to think about before you start forwarding links to this presentation to all of your friends and family, screaming “CURE!”:
1) Jay Bradner is soliciting funds. Doesn’t mean the concept won’t work. It just means that it is his job to make it all sound good–and he does that convincingly. If down-to-earth, magnificent public speaking skills can fight cancer, we’re in great shape!
2) Multiple myeloma has got to be one of the easiest cancers to slow in the lab. Two or three sub-clinical trials which exhibit “promising anti-myeloma activity” cross my desk every month. And even though a number of these also work a bit in humans, rarely do they meet FDA requirements to either show an amount of improvement greater than drugs currently approved for use against multiple myeloma and/or a significantly improved safety profile.
3) If this is such a good idea, why aren’t the drug companies beating their door down to get a hold of this magic molecule? After all, even if you believe in conspiracy theories and consider pharmaceutical companies to be intrinsically evil, that very quality should mean someone has stolen this idea and is working to further development as you read this–and maybe they are!
I’m not a scientist. I’m not a physician. My Masters of Science degree (MS) is in counseling and teaching at-risk kids, not in chemistry or the biological sciences. But I read a lot of myeloma related research. I interview and speak to multiple myeloma experts regularly. Everything on this video is probably new news for most myeloma docs.
Why? Assuming that it works, this type of pre-clinical research can take up to a decade or more to become available to patients who aren’t actively participating in a clinical trial.
Our doctors are interested in compounds that can help their patients now–or in the near future.
So if this type of video helps inspire you and fills you with hope–GREAT! But I would like to stress that there are already a number of documented, ongoing clinical trials which help me feel that way.
One way or another, there is a lot to be excited about in the world of myeloma research. I hope that Mr. Bradner’s “magic molecule” turns out to be the elusive cure we all are praying for. But if it falls short–or doesn’t pan-out at all–that’s OK! As I write this, there are a dozen new compounds being tested in clinical trials which already show proven anti-myeloma activity.
THERE IS HOPE!
Feel good and keep smiling! Pat
October 31st, 2011 at 11:57 am
I am like evey other person who or whose loved ones are affected by this disease and who are excited by the new discovery by Dr. Bradner. Being told there is currently no cure, we have all already learned to be guardedly optimistic to all news at all times. But this is quite an unnecessary in-your-face shout not to hold your hope high! Two things are clear. One is this is a new discovery that COULD yield promising results. Two is we are already short on hope which we desperately need to power us through this life’s formidable hurdle.
October 31st, 2011 at 12:23 pm
Thanks for commenting, Ben. I knew I was being a bit hard on this clip when I wrote the review. But please note what I wrote. I have highlighted a few key points you may have missed in BOLD:
“So if this type of video helps inspire you and fills you with hope–GREAT! But there are already a number of documented, ongoing clinical trials which help me feel that way.
One way or another, there is a lot to be excited about in the world of myeloma research. I hope that Mr. Bradner’s “magic molecule” turns out to be the elusive cure we all are praying for. But if it falls short–or doesn’t pan-out at all–that’s OK! As I write this, there are a dozen new compounds being tested in clinical trials which already show proven anti-myeloma activity.
THERE IS HOPE!”
Did you read the article? That’s a lot of “hope” I highlighted! I’m sorry you feel so discouraged. If you read my blog regularly, you know I understand! Would you like to share what isn’t going well? Good luck!
October 31st, 2011 at 12:48 pm
Both my wife and I read your blogs and columns regularly and know that you are always positive. That’s why I didn’t quite expect to see the not-so-upbeat article this morning. I did see all the comments in bold and am glad that no matter how I felt, in our heart of hearts we all agree that many hopes are on the horizon.
My wife was diagnosed with SMM through a routine physical two months ago. You can imagine the firestorms that have gone through her and the family. Added to the mix is a scheduled MRI in a few days to see whether there has been any bone involvement. With this sudden change, life has been very hard but we are coping.
October 31st, 2011 at 12:55 pm
Hi Pat, I follow your blog and your Myeloma Beacon columns and I love your positive, upbeat nature in the face of this stinking myeloma. I was dx. 8/11.
I found out about the video from Margaret’s Corner blog and she has diligently broken down the research for her readers with intelligence, grace and humor. I hope Dr. Bradner’s JQ1 is the real deal as well as Dr. Carl June’s landmark gene therapy trials which are now being started at UPenn for MM. The thing that Bradner did that is remarkable is that he went public with the discovery and gave 70 research facilities the “recipe”. That act belies his being a salesman to raise money. He partnered already with Tensha Therapeutics and they are fully financed already with $15million for developing JQ1. I think Bradner’s actions are simply remarkable, they are fully open and defy logic in a good way in our overly commercial world. Resreachers at Notre Dame have just released their own supportive findings of JQ1. I guess Bradner sent them the recipe! Terry From NJ
October 31st, 2011 at 1:00 pm
Pat,
I thought your article was “spot on”. I did not find it negative at all. I think this spread so quickly because Dr. Bradner is such a great speaker. Like you, my academic background is not in Science. This was presented in a way us “non-science” types can understand.
From my perspective it is most refreshing to hear about a Researcher that is trying to go after Cancer from a new perspective. What passes for “cutting edge” in MM therapy today are things like Tandem Autos using substances like Melphalan. Anything we can do to get away from the routine use of Mustard Gas derivatives for “therapy” is indeed great news.
Mark
October 31st, 2011 at 1:32 pm
I may not understand the giving away of the JQ2 exactly. But, I am hoping that since it is being put out in the public for all to test and develop, if it is real it will not get bought up and kept off the shelves. If it could do what he says, it would be like discovering penicillin. Healthcare costs could drop a lot, and hopefully we would have too many cancer centers.
Odd of it being that magic bullet, 10%, 20%…or even less.
Seems too good to be true, it today’s world of everyone chasing the buck, why would you give it away if it was so good? Should be investors lining up at the doors.
October 31st, 2011 at 1:44 pm
Dear Ben-
I’m so, so sorry that you and your wife are being forced to become a part of our dark, crazy world. Has she seen and been briefed on her genetic testing results yet? I understand this a lot to adjust too. But an otherwise healthy person that is diagnosed this year can expect to live a decade or more. And with all of the new drugs in the pipeline, all bets are off! A majority of newly diagnosed patients will most likely die from something else. So yes, it’s hard. Sort of like being diagnosed with advanced diabetes. So as long as you have good insurance and can work together to help keep your wife’s body in tip top shape, there is much to be thankful for. I’m sorry if my “change of pace” threw you… I hope you can continue to rely on me for accurate multiple myeloma news from a patient’s perspective…
October 31st, 2011 at 1:45 pm
I agree, Mark. With everything you wrote…
October 31st, 2011 at 1:51 pm
Thanks for the update and clarification, Terry! I was only responding to what I saw–and he asked for funding on the stage–not that there’s anything wrong with that! Let’s remember I’m on his side. Maybe it’s time to write an article about cancer cure conspiracy theories, because sometimes I think there is some truth there…
October 31st, 2011 at 6:45 pm
Hi, Pat:
Thank you for your encouragement. Yes, it was not unlike a plunge into a dark place. But the camaraderie evident in places like the Beacon has helped a lot. My wife’s FISH was negative and Cytogenetics was normal. But we didn’t have the GEP done and are worried about what it might say. It’s now one day at a time.
October 31st, 2011 at 10:23 pm
Hi Pat,
I think you might be looking to quell false hopes regarding this work. While that may be prudent for skeptics, I think the myeloma community sees the promise differently. With an incurable disease, we can use a few hand holds.
In a recent entry to Margaret Graziano on her blog, Dr. Bradner indicated a tailored molecule might see clinical trials within two years. While there are certainly no guarantees, this sort of targeted genome approach at halting myeloma seems intuitively correct to me. It is very true that it is possible that it could be years before such therapies might be widely available. However, with good results, a race could be on, as was the case with Bortezomib where a complete response in an early clinical trial in 1999 resulted rapid progress towards FDA approval of Velcade in 2003.
I see Bradner’s appeal as less calculated; I think he is really on a crusade to find better treatments and I applaud that.
Cup half full,
Danny
P.S. You already surfaced Bradner’s work on JQ1 on your blog on 5 September…
http://multiplemyelomablog.com/2011/09/researchers-hope-shutting-down-myc-may-be-key-to-defeating-multiple-myeloma.html
October 31st, 2011 at 11:16 pm
Sounds like she is in pretty good shape, relatively speaking of course! No real bone or kidney damage, newly diagnosed… Hard to see it now, but from where I’m sitting, I would say her future looks so bright, she needs to wear shades!
October 31st, 2011 at 11:21 pm
I have already done something on him. That was sort of my point. To me, his work is no more or less promising than the half dozen others I read about each month. I respect your opinion, Danny. If you believe this is different, that’s great! I want to remind everyone that I’m not rooting against this guy!
November 1st, 2011 at 10:44 am
Hi, Pat and Terry:
Back in September we were excited to hear the news about Dr. June’s and his teams’ experiment with T-cell therapy on Leukemia. Different from conceptual approaches like the JQ1, this has been proven to be successful with another blood disease with clinical successes on three late-stage parients. Hence Terry’s mentioning of the start of its application to MM really caught our eyes. Do you have any specific places where I can get more information? If not, can any of you talk more about it here?
By the way, Terry, my wife was also diagnosed in August. Just as you, we live in central New Jersey. Good luck to you!
November 1st, 2011 at 11:42 am
Ben S.
The T-cell Therapy developed at UPENN perfectly illustrates Pat’s point about why patients should not get to too excited when a new theory is developed. I can find references of researchers attempting to use T-cell therapy back to the mid-80′s. That idea is a rather old one.
That is an approach that is very similar to NK (Natural Killer) cell therapy. NK Cells are a small group of T-cells. It took at least 30 years for Researchers to get the approach of T-cell therapy to a point where it may be successful. The Beacon ran an article about this and note one of the problems if applied to MM patients – it may destroy all Plasma Cells.
http://www.myelomabeacon.com/news/2011/08/12/gene-therapy-advance-in-leukemia-suggests-new-treatment-options-for-multiple-myeloma/
One of the big problems MM patients have is that the Immune System does not identify the Cancer as foreign and something to kill. There is evidence that the “healthy” Immune Cells attempt to keep the Myeloma cells alive and growing. That is why Allogeneic Transplants is a potentially curative procedure for Myeloma.
Best of luck to you,
Mark
November 1st, 2011 at 12:23 pm
Thanks, Mark. I went back to read the linked article again. While the idea may date back to 30 years ago, the article did articulate that the approach taken by UPenn this time was entirely different. It was customized to allow the T-cells to target the B-cells. The article also mentioned that, even if the T-cells, when modified to fight MM, are not able to distinguish between myeloma and normal plasma cells, it is possible for a person to live without plasma cells. Even if the latter means the patient may have to get plasma cell infusions periodically, it surely beats living with the myeloma cells. The other differences from previous experiments that stand out, at least from the news reports, are that the T-cells were able to multiply rapidly for sustained attacks and were able to stay behind in small numbers to continue the job. But most important of all is it has had a clean-cut clinical success. To me, it spoke the loudest.
November 1st, 2011 at 2:23 pm
Ben S,
I think you may be missing my point. I was comparing the fact that it took 30 years plus to figure out a way that MAY make T-Cell therapy work. It could take a similar amount of time to find a way to get the JQ1 theory to work. I hope they can figure out how to implement JQ1 theory a whole lot quicker than it took them to potentially figure out how to make T-Cell theapy work.
Back to the T-Cell therapy research from UPenn. All it has done is put 2 out of 3 patients in CR at 1 year. While you say that this has a “clean-cut clinical success”, the Lead author of the study was not as positive.
“Only wider testing, wrote lead author Walter J. Urba of the Chiles Research Institute in Oregon, will show whether the breakthrough is “an authentic advance” toward a novel medicine, or yet another “lead that runs into a barrier.”"
http://articles.philly.com/2011-08-10/news/29872354_1_gene-therapy-jesse-gelsinger-leukemia-cells
Here is a quote from Dr. June:
“It worked great. We were surprised it worked as well as it did,” said Dr. Carl June, a gene therapy expert at the University of Pennsylvania. “We’re just a year out now. We need to find out how long these remissions last.”
http://remissionman.com/2011/08/11/amazing-therapy-wipes-out-leukemia-in-penn-study/
I am VERY excited about this research. I cannot believe how brilliant these Scientists are. It is difficult to get great theories to work in Humans. Not having Plasma cells is a very significant side effect. Much success to you and your Wife in your war with Myeloma!
Mark
November 1st, 2011 at 3:18 pm
I’m loving this! How interesting. I hope my readers take the time to read these insightful comments. Thanks, guys!
November 5th, 2011 at 11:08 pm
Sorry to seem stupid, but what exactly is JQ1? I think I need an idiots guide. What do the letters stand for?
November 5th, 2011 at 11:53 pm
I think that you are being way too hard on yourself, Monika. JQ1 is a small molecule inhibitor. At this point, researchers involved with the project are so optimistic about this development you might as well call it a “magic molecule.” I’m not sure I understand the details. I just hope it works! Pat
November 6th, 2011 at 2:27 pm
Would’ve understood better if I’d watched the video first. Sounds interesting. I’m based in the UK and going to see my consultant this week, so will chat to him about it. I’ve got Bence Jones MM, and was diagnosed 6 years ago. I had a STC following induction with ThalDex and achieved complete remission which lasted four and a half years. Have been on Velcade and Dexamethasone for about a year and am now considering the next strategy. Bence Jones MM is measured in light chains rather than PP and my light chains have been reducing slowly but steadily while on this treatment. I had a bone marrow biopsy recently and have found out that I still have 60% myeloma involvement in my marrow! That’s higher than it was before I started my treatment. Doesn’t make any sense.
November 6th, 2011 at 4:21 pm
Myeloma is relentless! Sounds like it’s time to add another “assisting drug” to the mix–something like Doxil and/or vorinostat, panobinostat or even cytoxan. Still lots of options, although I believe fewer in the UK. Best of luck!
November 7th, 2011 at 2:48 am
Thanks Pat. Had Cytoxin at the beginning of my treatment. Very effective. The drop in my light chains was meteoric, unfortunately the rise when I stopped taking it was also meteoric. The hospital I attend is the Royal Marsden and my consultant is Prof. Gareth Morgan, There isn’t anything I can’t have access to. Us Brits moan about everything and create our own bad publicity, but in fact we’re very lucky not to have to worry about whether or not our insurance will cover certain treatments. The Dana Farber institute does seem to be at the cutting edge of research, however, and it’s always a good idea to view all the options. Hope JQ1 is as good as it sounds. Thanks for your advice
November 7th, 2011 at 8:13 am
Glad you are being well taken care of, Monika. Cytoxin works surprisingly well for a lot of patients… It should be given a bit more respect. Never hear much about it because it has been around for so long.
November 7th, 2011 at 11:13 am
What is unique about this researcher’s findings is his approach to further developing it. At least that was what I took away from it as being somewhat revolutionary in disease research. Open Source. Giving it away in the hopes that others would be interested in seeing what they could do with it and thus find a solution sooner.
November 7th, 2011 at 11:12 pm
I’m glad researchers are starting to be more open and share data… Even Dr.Bradner admits drug resistance is the toughest hurdle to ever finding a cure. We are going to need all the help we can get!