[HN Gopher] Trial results for new lung cancer drug are 'off the ...
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       Trial results for new lung cancer drug are 'off the charts'
        
       Author : charlieirish
       Score  : 113 points
       Date   : 2024-05-31 16:21 UTC (6 hours ago)
        
 (HTM) web link (www.theguardian.com)
 (TXT) w3m dump (www.theguardian.com)
        
       | noncoml wrote:
       | Lung cancer is not the death penalty that it use to be if you are
       | "lucky" to have one of the mutations that this, and other similar
       | drugs, target.
        
         | admissionsguy wrote:
         | According to a (very) quick google search:
         | 
         | - the drug is for "ALK positive Non-small cell lung cancer
         | (NSCLC)"
         | 
         | - NSCLC is 85% of all lung cancers, of which 3% to 5% are ALK+.
         | 
         | so it would appear to apply to at most 4% of all lung cancers.
         | Are the numbers correct? Even so, huge deal for tens of
         | thousands of people each year.
        
           | odyssey7 wrote:
           | "Lorlatinib and crizotinib are both ALK tyrosine kinase
           | inhibitors (TKIs). ALK TKIs are targeted treatments that bind
           | to the ALK protein found in ALK-positive non-small cell lung
           | cancer and stop the growth of tumour cells."
           | 
           | Great, so this is a targeted approach that works 60% of the
           | time (in the case of Lorlatinib) in one highly specific area
           | of the highly diverse space of possible cancers, many of
           | which have never been observed before and for which you could
           | never enroll enough patients to have a robust clinical trial.
           | 
           | How can this approach be generalized and scaled, meaning that
           | for a given patient with a given cancer, you can run an
           | algorithm and synthesize molecules that will work?
           | 
           | In CS the paradigm involves knowing how to solve an unseen
           | problem instance, not just remembering solutions to specific,
           | well-studied problem instances.
        
             | admissionsguy wrote:
             | > meaning that for a given patient with a given cancer, you
             | can run an algorithm and synthesise molecules that will
             | work?
             | 
             | The paradigm is called rational drug design (+ personalised
             | medicine) and its scope is quite limited at the current
             | level of technology. To achieve what you describe, we need
             | to solve all of the following:
             | 
             | - a way to recognise the molecular mechanism of a
             | particular cancer and identify potential drug targets
             | 
             | - a way to characterise the targets - in the previous step
             | you identified a protein, now you need to know its shape
             | (X-ray crystallography (potentially years), AlphaFold). We
             | need to know how the target should be modified to disrupt
             | the disease.
             | 
             | - a way to design a molecule that binds the target
             | specifically and in the desired way
             | 
             | - a way to synthesise the molecule quickly & efficiently
             | 
             | - a way to predict the molecule's interactions at all
             | stages of metabolism, taking into account the patient's
             | individual phenotype (different people have significant
             | variations in drug-digesting enzymes)
             | 
             | Significant (decades at the current rate) progress in all
             | of these areas is needed before this sort personalised
             | medicine has a chance of becoming feasible.
        
               | jghn wrote:
               | Also the question of how to handle clinical trials when
               | the treatment is by design n=1. This world is getting
               | less murky but as far as I know isn't yet 100%
               | straightforward.
        
               | odyssey7 wrote:
               | Thanks, this is just the sort of information I was hoping
               | for.
        
           | noncoml wrote:
           | There are similar drugs(eg osimertinib) for EGFR+ cancers,
           | which are estimated to be 10-33% of NSCLC.
        
           | hooverd wrote:
           | 4% here, 4% there. Maybe you don't have a generalized
           | treatment, but treatments for different subtypes add up.
        
           | maherbeg wrote:
           | True, but each advance adds up. Just look at a history of
           | curing hodgkin's lymphoma which omits a lot of detail still h
           | ttps://www.hematology.org/about/history/50-years/milestones..
           | .
        
       | hnpolicestate wrote:
       | Still no progress with small cell lung cancer?
       | 
       | It killed my 43 year old aunt in less than two months back in
       | 2002. Smoker though.
        
         | dannykwells wrote:
         | Durva is starting to show some benefit here.
         | 
         | https://www.cancernetwork.com/view/durvalumab-significantly-...
        
         | adamredwoods wrote:
         | My condolences, SCLC is rough. Sadly, it is different than
         | large-cell, and the mutations are different.
         | 
         | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8038650/
        
       | zzzeek wrote:
       | Why do all the new cancer drugs coming out end in the letter "B"?
        
         | abracadaniel wrote:
         | I think the *mab ones are monoclonal antibodies, and the *nib
         | ones are inhibitors. I don't think it's a hard rule though.
        
         | andyjohnson0 wrote:
         | Its a side-effect of the naming convention for generic drug
         | naming [1]. Small molecule inhibitors end with "ib". Monoclonal
         | antibodies end with "mab". Etc.
         | 
         | [1] https://en.wikipedia.org/wiki/Drug_nomenclature
        
       | adamredwoods wrote:
       | Tyrosine kinase, when mutated, causes uncontrolled cell growth.
       | 
       | TKIs: Crizotinib (1st gen), alectinib.
       | 
       | >> Lorlatinib is a third-generation, highly potent, macrocyclic
       | ALK/ROS1 TKI that competitively binds to the adenosine
       | triphosphate-binding pocket, blocking ALK-dependent oncogenic
       | signaling. The advantage of Lorlatinib is high penetration of the
       | blood-brain barrier by decreasing p-glycoprotein-1-mediated
       | efflux. Besides, it has broad-spectrum activity against most
       | known resistance mutations that develop during treatment with
       | first and second-generation ALK TKIs, including ALK G1202R
       | mutation. The introduction of Lorlatinib to salvage these
       | patients has shown the potential to add life. This has been shown
       | in a global phase II study and other real-world studies, however,
       | data is scant from LMIC. The most common toxicities were
       | peripheral edema (9-48%), hyperlipidemia (47-94%), weight gain
       | (3-25%), peripheral neuropathy (30%), fatigue (15-30%) and
       | cognitive effect (6-18%) in earlier studies. The treatment
       | discontinuation rate varied from 3-14% due to toxicity.
       | 
       | https://www.nature.com/articles/s44276-024-00055-9
       | 
       | https://en.wikipedia.org/wiki/Lorlatinib
       | 
       | https://en.wikipedia.org/wiki/Tyrosine_kinase
        
         | JumpCrisscross wrote:
         | > _advantage of Lorlatinib is high penetration of the blood-
         | brain barrier_
         | 
         | Why is this essential to fighting lung cancer?
        
       | dannykwells wrote:
       | Note that this drug is not new - it is fully approved for ALK+
       | NSCLC and has been since 2015.
       | 
       | It's not clear why these data are just coming out now or what is
       | different from the original trials run in this setting.
       | 
       | https://en.wikipedia.org/wiki/Lorlatinib
        
         | westcort wrote:
         | Not mentioned in the article is the fact that lorlatinib,
         | original studied in the phase 3 CROWN trial, was used in
         | frontline management of advanced ALK-mutated non-small cell
         | lung cancer (NSCLC) [0]. Five-year data indicate a significant
         | progression-free survival benefit, with 60% of patients free of
         | progression. Even after 60.2 months of follow-up, the median
         | progression-free survival has not yet been reached [1]. It is
         | the durability of progression-free survival that are most
         | impressive and surprising here. That said, only 4% to 8% of
         | patients with NSCLC will have ALK mutations/rearrangements, so
         | while amazing, this finding is not applicable to all patients
         | [2].
         | 
         | [0] https://www.nejm.org/doi/full/10.1056/NEJMoa2027187
         | 
         | [1] https://ascopubs.org/doi/10.1200/JCO.24.00581
         | 
         | [2] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7597761/
        
       | benmarten wrote:
       | RCT? Doesn't look like it...
        
         | ilya_m wrote:
         | It is:
         | 
         | > In the phase 3 trial, 296 patients with advanced forms of
         | non-small cell lung cancer were randomly assigned to receive
         | either lorlatinib (149 patients) or crizotinib (147 patients,
         | of whom 142 ultimately received treatment).
        
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       (page generated 2024-05-31 23:01 UTC)