Making great drugs – only half the battle?

This month’s “The View from Here” deals with the field of oncology; great advances have been made, especially with respect to immuno oncology. Brilliant new drugs are becoming available, it would seem, every day. But is that everything? Do we lose sight of the patient?

I hope that you’ll indulge me, for although I will be covering cancer in this Editorial, I’m going to change tack slightly to look at a cause that I’ve been passionately involved with. I’ll endeavour to explain below and perhaps then you’ll realize why there is a photo of a significantly more talented (and might I say younger and better looking) musician than I featured in this article. Below, she outlines her personal route to recovery. Please read this and feel inspired and hopefully, inspired to become a stem cell donor.

Immuno oncology has to be one of the great success stories of recent drug discovery and in particular the development of monoclonal antibody therapies, such as nivolumab and ipilimumab, targeting the Programmed Death 1 (PD-1) pathway. This approach has great promise as a monotherapy or an adjunct for the treatment of cancers and several monoclonal antibodies of this class are currently licensed for the treatment of metastatic melanoma. Many cancers avoid destruction by the immune system through the overexpression of the cognate ligands for the PD-1 pathway, namely PD-L1 and PD-L2 (for more information, see the excellent review from Troels H. Borch, Marco Donia, Mads H. Andersen and Inge M. Svane, that is the associated download with this story), as a result, agents interfering with the PD-1 pathway and its ligands can unmask the cancer cell for destruction by the native immune system. The approach shows great promise and the list of cancers that might be treated by this route grows daily.

One of those cancers that might be able to be treated by these monoclonal antibodies is non-Hodgkin’s Lymphoma  and, as you may or may not be aware, is a subject close to my heart. Although these drugs can be  effective as monotherapies, it is becoming clear that to achieve maximum benefit for the patient, a stem cell transplant is frequently essential.

Let me relay the words of the celebrated trombonist, Carol Jarvis as she outlines her personal struggle with a related blood dyscrasia, Hodgkin’s Lymphoma (see below). This is the sort of story that should energize you to get out of bed in the morning and get to the bench with a renewed sense of purpose. I quote now from Robin Ganellin (interview included as a download to this article) to this end, who stated: “That is a hell of a rewarding feeling when you have made something that has become a medicine and people turn round and thank you for doing it. When you talk about professional reward, the people aspect is really something.” Before I get on to this however, I’d like to let you know about another talented young trombonist, Stephen Sykes, who has also had the misfortune to have contracted non-Hodgkins Lymphoma. I’m sure Carol will be pleased in years to come to have some competition from him on the trombone front. Through a massive effort from his family and the brass-playing community of the World, the money for his treatment (as nivolumabat at the time was not licensed in the UK for non-Hodgkin’s Lymphoma and as such was non-reimbursable) through what has come to be known as the Acrobat Challenge . So successful was this initiative, that $150,000 (or thereabouts) was raised in a little over a month. Stephen has now had a stem cell transplant and is on the long road to recovery, I hope that you all wish him well. Please consider registering and please share this post as widely as you possibly can. Every registration can help someone, even if it is not Stephen.

Carol’s story

Six years ago today (7th July) I was in a taxi in Paris, having just landed back to rejoin a tour after a day off. I had received 4 doses of a brand new drug a few weeks before, and this drug was my last possible chance of survival, and the last option my specialists could find to try and stop my cancer, after every other line of treatment (countless different courses of chemotherapy, intensive chemotherapy, radiotherapy, a stem cell transplant, and other clinical trial drugs and experimental therapies) had continuously and repeatedly failed for the past seven years.

Six years ago, I had flown home on my day off, for a scan to see if this new treatment was working or not. Less than 12 hours after my scan, I had landed back in Paris and I received a phone call from one of my specialists; “Carol, you’re in a metabolic remission. There is no sign of cancer”. For the past 5 years that same specialist had repeatedly told me, every time I saw him, to get my head around the fact that I was going to die, because I wasn't responding to any treatment. So to get a call from him telling me the news that I was in remission for the first time in 7 years, was mind-blowing. That miracle drug had been in the production stages for the past 15 years and 3 years ago it was FDA approved; the first drug to be FDA approved for Hodgkin’s Lymphoma in over 40 years.
A donor bone marrow transplant was the huge decision I was faced with then; the idea behind it would be to secure my remission. Without the transplant I would live approximately 3 years, but the procedure held a 30% survival rate. My specialists gave me a few weeks to decide, and I was amazed that I seriously considered both avenues and could have easily chosen to just live for 3 years.
But 2 donor matches were found out of 11 million people (one was pregnant so she couldn't help, but thankfully a German man said yes), and I decided to give it my best shot, and signed on the dotted line.

I had two 'goodbye' parties, in case I didn't make it. I got my Will written, and set up a Caring Bridge blog where my mum was an admin and could ultimately announce my death via there, and not have to go through the pain of continuously telling people the bad news.

I still can't get my head around all the statistics that were stacked up against me, and how ridiculously lucky I have been to survive all of that. It still completely baffles me.

In the past couple of years, I've been campaigning to help keep that FDA approved drug in use in the U.K. Obviously these drugs cost a lot of money, and it was going to be removed from the cancer drugs fund because of its expense. I even spoke at the Houses of Parliament to MPs about government policies, to help change industry practise, bring about improvements for those affected by cancer, and to try and save this drug. And I'm very pleased to say that this drug is now safe, and will continue to be in use on the NHS.

As to my health ever since recovering from my bone marrow transplant; I'm still in a complete remission and my health has never been so good.

I can't thank you all enough for being there for me, throughout the ups and downs of this crazy journey of mine. Thank you for laughing and joking with me, at the ridiculousness of it all - without that I would've really struggled.

So, today is remission anniversary, the day I began to take my life back. So today, at some point, I urge you all to stop what you're doing and just look around you. Breathe. Soak up what you're looking at, what you hear, what you touch, what you smell, what you feel. And allow yourself to simply be amazed at being alive and being able to experience and be inspired. Even when things get too much, work piles on top of you, people treat you badly - you've still got the gift of life. Treasure it. Xxx

So why am I making a big deal of this? Well, as you can see, all the biologists, molecular biologists, chemists, pharmacologists have done the hard part and made some of the best drugs around. But, and there is always a but, to ensure that patients like Carol have the opportunity to go on entertaining the world (or for Joe Public just to get back to a normal life) we need to finish the job. In this case, finishing the job means getting stem cell donors. So I’m appealing to you to consider being swabbed to see whether you could be a stem cell donor. It’s the simple part. But the simple part, as with so many things, may turn out to be the most important.

In the UK you just need to contact DKMS and register. They’ll send you all that you need – it is just cheek swabs and takes 5 minutes. For people like Stephen and Carol, it could be the most important 5 minutes that you’ll ever spend.

Although I’m giving the UK address here, DKMS is a world-wide organization and below are other links that may be more appropriate for you in different parts of the world. I’ve also added a fact sheet as a download so that you can see just how important this work is.

DKMS Germany

DKMS Poland


DKMS Chile

Every life is important and precious, all will bring joy into someone else’s life, some will impact more than others but every registration to the stem cell database gives someone else the chance of a long, fulfilling life. Just 5 minutes of your time.

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