Hagop KantarjianLeukemia research and cancer drug policy

Unmet needs in CML: increasing treatment-free remission & accessibility to TKIs

8 Sept 2023 · 1 min

Watch on YouTube

1:22

In this commentary, Dr. Hagop Kantarjian outlines two major unmet needs in chronic myeloid leukemia (CML). First, while near‑normal overall survival is now achievable, only about 40% of patients achieve treatment‑free remission (TFR) with existing therapies. He argues that expanding TFR to 60‑70% will require new agents, highlighting oral decitabine (a hypomethylating drug) and ropegylated interferon (a newer, less toxic interferon formulation) as promising research avenues. Second, he stresses the necessity of making second‑ and third‑generation tyrosine‑kinase inhibitors (TKIs) affordable and universally available, noting that high costs—especially in the United States—prevent full patient access. The overall message is a call for both therapeutic innovation to raise TFR rates and policy/price interventions to ensure all CML patients can receive optimal TKI therapy.

Chapters

So for CML, survival being close to normal is one endpoint that has been met. The second important endpoint is to stop therapy in as many patients as we can, which is referred to as treatment-free remission. Today, we probably can achieve that with the current tools in 40% of the patients. So what we need is an additional form of therapy that increases the treatment-free remission to about 60 or 70%. We're exploring a couple of options like the oral decitabine, which is a hypomethylating agent, also reverting back to the ropegylated interferon, which is an effective interferon formulation, more effective than the historical interferons, but also much less toxic. So these are 2 venues of research to increase the potential rate of treatment-free remission. The third aim would be to make TKIs affordable and available to 100% of the patients with CML. And that's not the case today because of the high cost of the second and third generation TKIs, at least in the United States.