Yesterday Linnea Olson set the stage to find her best next treatment with a description of her medical history and available data.
To kick the meeting off, Linnea diligently presented her history (which took about 15 minutes), then the crowd of two dozen participants asked useful clarifying questions and discussed possible therapies (another 15 minutes), and then (not recorded) we had a fascinating discussion among the participants.
As you may know, Linnea has been on five clinical trials, and is facing a difficult decision.
While she has learned to live with her disease as a manageable, chronic condition, she was just booted from her fifth phase 1 clinical trial (a MEK inhibitor combined with an ALK+ inhibitor, lorlatinib) due to retinopathy (eye damage). She is staying on an ALK inhibitor, whose side effects she tolerates, and then will rescan and also explore the possibility of enrollment in another phase 1 trial–this one a combo of lorlatinib and a SHP2 inhibitor. Her concern is that SHP2 is on the same pathway as MEK and has the potential for eye issues as well. She would love to have other possibilities for treatment. At 16 years, she is very far out on the skinny branches when it comes to options.
The recording has much more detail that Linnea puts on a timeline.
In the discussion which followed Linnea’s medical history presentation, the crowd discussed dosing, tissue access, and two possible treatment options: ERK inhibitors and a fourth generation ALK inhibitor.Kimary Kulig asked about dosing as an option (to minimize the negative side effects) and compassionate use. Grace Cordovano asked about Linnea’s biopsy history and access to tissue for analysis. Linnea said that her tissue was used for research, and she didn’t have access to it. Kimary Kulig suggested Linnea could withdraw her consent. The amount of excess tissue was small since it was a needle biopsy. Tim Stuhlmiller offered suggestions for possible drugs that target the MEK pathway, with the proviso that the eye issues side effects may be an issue with all of the options targeting that pathway. He also introduced the option of ERK inhibitors, which are the next drugs down the MEK pathway. xCures (where Tim works) is running an expanded access basket trial for ulixertinib from BioMed Valley Discoveries to access this drug and novel combinations. Emily Venanzi introduced another option: a fourth generation ALK inhibitor, TPX-0131, that the ALK+ community has been working on with Turning Point and which is in a phase 1 trial in Australia. Willy Hoos added that you can get compassionate access if efficacy has been proven, even as the company is working through the dosing experiments.
To me, the highlight of the informal discussion which followed was when Lars Engstrom, a researcher at pharmaceutical companies where he has been developing the drugs that Linnea has been using, gave an emotional testimonial of how he thinks of Linnea when he does his work. It was also interesting to hear from Emily Venanzi about her ALK+ patient group, founded on Facebook 5 years ago, which has grown to gather patients’ data and fund research.
Next week Linnea should have results from the Foundation Medicine and Lucence liquid biopsy tests to report. She had her blood draw on Wednesday.