Monthly Archives: August 2021

The necessity for compassionate use/expanded access

My participation in this, my sixth phase I clinical trial, would not be possible without the concept of compassionate use/expanded access.

That is because I did not qualify–was precluded–from the actual trial. The reason? Because I’ve had too many previous therapies–specifically ALK inhibitors. Three to date. Well, until yesterday 🙂 Now four.

Fortunately, there was a back door. And I had a crew of people knocking on it for me. Colin Barton, who works so much magic behind the scenes at ALKPositive as chair of the Medical Committee. I talk a lot about goddesses; this man is a god of good. An ALK+ patient himself, he uses his smarts, personal resources and the fervor of a man on a mission to advocate for the other members of this club. He has initiated conversation with the decision makers at various pharmaceutical companies. I shared with him that at first I thought this might be a fool’s errand, but Colin is a visionary and he saw something I did not. And he has assembled an amazing team of fellow patients who work tirelessly to ferret out potential treatments that could benefit the ALK community.

My oncologist Dr. Jess Lin was also relentless on my behalf–contacting the sponsor almost daily. Jess presents as sweet and mild mannered, but she can be ferocious when it comes to her patients.

Of course, this was a time sensitive matter and some of my friends (talking about you Rob Densen) were not satisfied with the pace as I waited for drug. Where I was hesitant to reach out he was not and I am grateful.

So there we have it. Today I took my second tablet and then lay down for an hour so as to better visualize the cancer exploding like a mouthful of Pop Rocks. And, to continue with that metaphor, my mind continues to be blown by what a long, strange trip the last two months have been. I spent a whole lot of time thinking about dying all while hoping it wasn’t true.

Not natural bedfellows–these two thoughts. At least if you are hoping for a good nights sleep.

Therefore I am happy to report that I am still feeling good/better and that my dreams last night were sweet.



A fourth generation ALK inhibitor provided to me by Turning Point Therapeutics through compassionate use.

At 1:30 pm today I took the first dose. Before handing me the tiny tablet (it is one mg), Dr. Lin and my nurse Heather performed a little ritual—a blessing of sorts. And then, down the hatch.

By Dr. Lin’s estimate I am the third person in the world to take this drug (it is in clinical trial in Australia) and therefore it is impossible tp predict side effects.

Truthfully, I am far more interested in efficacy. And the thing is, if this experimental therapeutic is going to be effective for me, I may know very soon. Likewise if there is no response.

After dosing I hung out at the hospital for an additional two hours of observation. I squeezed in my second nap of the day (I had been there since 7:30 am) and when I awakened I just had this sense that something was going on in my body. Something good.

So light some candles. Cross some fingers. Have a chat with the universe on my behalf.



What a busy seven days it has been. Melinda and Sally, Jemesii, Peter and his girlfriend Caroline and my brother Bink and sister John have all visited over the past week. It has been great fun, much has been accomplished and I am exhausted.

However, I also have had more baseline energy. A function of restarting TNO155? Perhaps.

This morning I was scheduled for installation of a PleurX-cath–financial assistance approved. My breathing has also been some improved and I went to the appointment hopeful that I should be turned away again–but this time for lack of necessity.

And indeed a Pleur-x was deemed unnecessary at this junture. Instead I had another thoracentesis which yielded 150 ml of serosanguinous fluid.

Last night I learned that TPX-0131 would be at MGH no later than early next week. I know my oncologist has been working tirelessly on my behalf to forestall more delays, and my friend Rob Densen and his pals at Senator Markey’s office got involved as well. Moral of the story? Choose your oncologist’s, your friends, and their friends carefully.

To put a dot on that i, I shall take my final dose of TNO155 tonight—and my final dose of lorlatinib on Sunday as I begin washout. And then, next Thursday, my lead in dose of TPX-0131.

There have been moments (hours, days) in the preceding weeks where I felt I was down for the count. I don’t feel that way today. Raggedy and realistic about the fight that lies ahead, but confident that I’m going to get a shot at it.



And (allow me some dark humor please) the suspense is killing me.

Unfortunately hurry up and wait can play an overarching role in the management of cancer. There are even rooms for this–Waiting Rooms. And I have spent far too much of my precious time in such.

At the moment my waiting is of a different sort. The expectation was that I should be starting on TPX-0131 this week. However, at my appointment last Thursday, I learned that lead in dose has been pushed back by another week. FDA and the IRB have both signed off but this holdup is in manufacturing and labelling. Whatever that means. At this stage, I am nervous that this won’t be the last delay and–between you and me–it’s getting more and more difficult to hold my breath in the interim.

What did happen last Thursday is that I started back on the SHP2 inhibitor TNO155.

Per my oncologist’s advice, I shall not be getting a pleurodesis, but rather a (déjà vu!) pleur-x catheter. Financial assistance has been applied for and the pulmonologist’s team spoken to. That will take place Thursday morning, after which a visiting nurse shall make two visits both to drain the catheter but also to teach someone else how to do so–it is not possible to do alone.

And then, hopefully, a week later I shall be taking a fourth generation ALK inhibitor.

It is early days–I shall be one of the first–but this is the best of my current options. One on which I am pinning a whole lot of hope.

In the meantime, I am laying low. Continuing to tie up ends with my committee of personal assistants (bless each of you) and also spending time with my two kids who live in Boston. Today we went through family photos and despite their trepidation (what all this preparation represents), had a wonderful day.

And I, of course, hope that there shall be many more.

Wonderful days.