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Rare Cancers: The Time to Act is Now

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Rare cancers are those affecting very small numbers and up to now have been poorly diagnosed, researched, funded, and treated, resulting in very high death rates even as morbidity for common cancers decline.

Diagnostic breakthroughs like genome sequencing make earlier stage diagnosis possible and breakthroughs in personalized treatment, including cell and gene therapies, provide new hope, including potential cures.

CCSN welcomed our host panelist Durhane Wong-Rieger, President & CEO of Canadian Organization for Rare Disorders and Chair of Rare Disease International. Durhane was joined by Lisa Machado, Founder and Chair of the CML Network for this engaging and educational webinar on the unique issues presented in rare cancers.

The webinar was followed by a question & answer session.

Published in: Healthcare
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Rare Cancers: The Time to Act is Now

  1. 1. Rare Cancers: The Time to Act is Now Durhane Wong-Rieger & Lisa Machado
  2. 2. A rare cancer diagnosis changes everything. living RARE lisa@cmlnetwork.ca cmlnetwork.ca @cdncmlnetwork
  3. 3. Life and cancer.
  4. 4. What’s so unique about rare cancers? Lack of information & education Remote and not-so-remote areas underserved Limited reimbursement Lack of public awareness Few or no support organizations
  5. 5. The reality of advocacy in the world of rare cancers. Patient Support Education, peer connection, navigation Health Policy Influence government/policy-makers Research Collaborate with industry, clinicians Local Own backyard issues National Partner with organizations to share information, support, promote Global Members of international like-minded bodies
  6. 6. Thank you lisa@cmlnetwork.ca cmlnetwork.ca @cdncmlnetwork
  7. 7. Rare Cancers: The Time to Act is Now Durhane Wong-Rieger, PhD Canadian Organization for Rare DIsorders
  8. 8. Key Points • Rare Cancers “not so rare” • Genomic-defined cancer subtypes leading to targeted therapies • Effective therapies for rare cancers based on genetic defect • Canada’s Orphan Drug Regulatory Framework to support R&D and access to rare disease drugs • Precision therapies: immunotherapies, CA • Lifecycle approach– from clinical trials to real-world use • Managed Entry Programs—Right drug, right patient, right time, right price
  9. 9. Common vs Rare (Less Common) Cancers • 206,600 new cases of all cancer in Canada in 2017 • Est. 80,800 deaths from all cancer in Canada in 2017 • Est. 62,100 new cases of Rare/Less Common cancers • Est. 35,500 deaths from rare cancers • Incidence RLS:All cancers = 1:3.32 • Deaths RLS:All cancers = 1:2.7 • % deaths to new cases All cancers = 39.1% • % deaths to new cases rare cancers = 57%
  10. 10. Genetic Testing Improves Breast Cancer Odds • Gene mutations BRCA1 & BRCA2 increase risk breast & ovarian cancers – 5 -10% breast & 15% ovarian cancer = screening & prevention • HER2-positive accounts for 20% breast cancers – Targeted treatments: Herceptin, Perjea, Tykerb, Nerlynx • Scanning whole genome (DNA testing) can identify frequent and rare genomic cancer alterations and target appropriate drugs – ID genetic aberrations advanced breast cancer; match therapies • Whole-genome analysis of 423 patients ID genetic alterations in 2/3 – 46% had targetable genomic alteration; 39% had rare alteration – 28% with targetable alterations matched with treatments already in clinical trials
  11. 11. Lung Cancer: Genetic Typing Improves Odds • 2nd Most Common: – 28,600 new cases and 21,100 deaths in 2017 – 14% of all new cancer cases and 26% of all cancer deaths • About 4/5 = non-small-cell lung cancer (NSCLC) – Treatment = surgery, chemotherapy; survival < 70% at 5 years • About 4% NSCLC have genetic ALK-mutation (kinase activity) – Patients generally younger and nonsmokers – 900-1,000 ALK+ Canadians per year • 1st ALK-targeted therapy: Xalkori (Crizotinib) inhibits ALK activity – Response rate (tumour stabilized or shrinkage) = 67-90% – 3 year progression-free survival = 63% (one LT study) – 3 year overall survival = 72% (one LT study)
  12. 12. Definitions • “Less common cancers” = those with an incidence of between 6 and 12 (inclusive) per 100,000 persons per year • “Rare cancers” = those with an incidence of < 6 per 100,000 persons per year • Types of rare cancers = total of 186 cancer types defined as rare • “Ultra-rare cancers” = those with an incidence of equal to or less than 2 per 100,000 persons per year
  13. 13. Drug Information Association www.diahome.org 15
  14. 14. Rare Cancers Not So Rare • RARECARE definition (incidence <6/100,000/year) – Annual incidence rate of all rare cancers (in Europe) was about 108 per 100,000 – About 186 known rare cancers (in Europe) – 22% of all cancer diagnoses (new cases) – 24% of all cancer prevalence (total cases) • Five-year relative survival was on average worse for rare cancers (47%) than common cancers (65%)
  15. 15. Drug Information Association www.diahome.org 17
  16. 16. Most “Common” Rare Cancers • Uterine cervix and thyroid carcinoma are rare according to the incidence (RARECARE) criterion and ‘common’ according to the prevalence criterion. • Six cancers are common according to the incidence criterion and rare according to the prevalence criterion: – Stomach adenocarcinoma, – Pancreatic adenocarcinoma, – Lung adenocarcinoma, – Lung squamous cell carcinoma, – Poorly differentiated endocrine carcinomas of lung and – Group othernon-Hodgkin mature B cell lymphomas.
  17. 17. Treating Myelofibrosis: Rare Leukemia • Leukemia fairly common – 5,800 Canadians will be diagnosed with leukemia. – 2,600 Canadians will die from leukemia • Myelofbrosis = 1.5/100,000 – Median survival of 3 years – 50% have genetic defect: JAK2 inhibitor • Ruxolitinib (Jakafi) (approved in 2011) addresses JAK2 – Early results = 52% reduction in risk of death – Overall survival increased (81% compared to 61%)
  18. 18. Genetic Testing vs. Genomic Testing Genetics • Study of inherited traits passed from one generation another • >> risk for getting cancer • Ex: Test for BRCA1/BRCA2 to predict risk for breast or ovarian cancer • HER2-positive accounts for 20% breast cancers – Targeted treatments: Herceptin, Perjea, Tykerb, Nerlynx Genomics • Study of activity of genes influencing tumour growth and spread • >> choice of course of care • Oncotype DX test to predict aggressiveness of tumor ad benefit from chemotherapy. • Whole-genome analysis identified variations not ID in genetic testing – 46% targetable and 39% had rare alteration – 28% matched with treatments already in clinical trials
  19. 19. Standard Chemotherapy vs. Targeted Therapies Standard Chemotherapy • Act on all rapidly dividing normal and cancerous cells • Identified because they kill cells • Are cytotoxic (that is, they kill tumor cells) Targeted Therapies • .Act on specific molecular targets associated with cancer • Chosen or designed to interact with target cells • Are often cytostatic (that is, they block tumor cell proliferation
  20. 20. Types of Targeted Therapie • Hormone therapies prevent body from producing hormones or interfering with hormones needed for certain tumours • Signal transduction inhibitors interfere with inappropriate signaling that allows malignant cells to divide continuously • Gene expression modulators modify function of proteins that play a role in controlling gene expression • Apoptosis inducers cause cancer cells to undergo controlled cell death • Angiogenesis inhibitors block growth of new blood vessels to tumors, blocking oxygen and nutrients necessary for tumour growth • Immunotherapies trigger immune system to destroy cancer cells • Monoclonal antibodies deliver toxic molecule, e.g. radioactive substance or poisonous chemical, ultimately killing cancer cell
  21. 21. Precision Medicine for Cancer • Precision Medicine – Matched to individual genetic variations – Patients’ tumors have genetic changes that cause cancer to grow and spread. – Changes that occur in one person’s cancer – Same cancer-causing changes may be found in different types of cancer. • To find which genetic changes in individual’s cancer, – Collect blood sample and use DNA sequencing, genomic testing, molecular profiling, or tumor profiling – Find right drugs for the genetic variants
  22. 22. Tumor cells can evade destruction by cytotoxic T cells
  23. 23. CAR-T-Cell Therapy • CAR T-cell therapy = form of immunotherapy that uses specially altered T cells — a part of the immune system — to fight cancer. • CAR-T process – Sample of a patient's T cells are collected from blood – T-cells are modified to produce special structures called chimeric antigen receptors (CARs) on their surface – CAR T cells are reinfused into the patient – The new receptors enable them to latch onto a specific antigen on the patient's tumor cells and kill them
  24. 24. BITE Technology • T cells target tumour cells, suppressing tumour growth • However, tumor cells have different ways of evading destruction by cytotoxic T cells • Bispecific T Cell Engager (BiTE®) antibodies can bridge cytoxic T-cells to (cancer) cells with specific cell surface antigens • Result is release of protein-destroying substances against target tumours cancer cell(s) • BiTE® technology can target different tumors throughout the body (non-site specific cancers)
  25. 25. Opportunities & Challenges • Opportunities – Genetic testing – Genomic testing/Next generation sequencing – Targeted therapies (hormone, immunotherapies, vaccines) – Non-site specific therapies – Durable and curable cell and gene therapies • Challenges – Traditional clinical trials – Regulatory approvals (concordance, capacity, collaboration) – Valuation and long-term expectations – Financing (investment) for access
  26. 26. Thank You! Durhane Wong-Rieger Canadian Organization for Rare Disorders www.raredisorders.ca 416-969-7435 durhane@sympatico.ca 32
  27. 27. Canadian Cancer Survivor Network Contact Info 1750 Courtwood Crescent, Suite 210 Ottawa, ON K2C 2B5 Telephone / Téléphone : 613-898-1871 E-mail: jmanthorne@survivornet.ca or info@survivornet.ca Website: www.survivornet.ca Twitter: @survivornetca Facebook: www.facebook.com/CanadianSurvivorNet Instagram: @survivornet_ca Pinterest: http://pinterest.com/survivornetwork/

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