A small group of scientists and clinicians are trying to build awareness of this cancer, and how to diagnose and treat it. While this rare and aggressive hematologic malignancy hasnt had a standardized therapeutic approach in the past, that is also changing. The FDA approved tagraxofusp (Elzonris, Stemline) in 2019 as the first medication indicated to treat BPDCN, according to a drug review published June 24, 2020 in the journal Expert Review of Anticancer Therapy.1
Progress aside, theres still a long way to go in creating awareness among dermatologists, primary care physicians, emergency department providers and pediatricians all of whom are most likely to encounter people with possible BPDCN, says Naveen Pemmaraju,M.D., associate professor in the department of leukemia at The University of Texas MD Anderson Cancer Center. Dr. Pemmaraju is senior author on the recent Expert Review of Anticancer Therapy paper, and he led the pivotal study published in April 2019 in the New England Journal of Medicine that led to the FDAs approval of tagraxofusp for the treatment of BPDCN.2
Having the clinical trial is great, but now that the drug is commercially available there was a call for what are the practical considerations for Tagraxofusp in BPDCN? Dr. Pemmaraju says. Thats why we wrote this paper.
Dermatologists, take noteDr. Pemmaraju points to three stand-out practical considerations for providers.
One is that most people in medicine have yet to even hear about the disease, he says. When people on the frontlines see skin lesions, the only way theyre going to know whether its BPDCN is with a biopsy. Tissue is the issue, as they say. If something looks irregular, abnormal and if its clinically indicated to do a biopsy, do a biopsy. A lot of these patients get mistaken for other diseases, infections, other cancers. So, I think the biopsy is important.
The need for expert review goes along with the first practical consideration.
CD123 (interleukin-3 receptor alpha unit) is overexpressed in 100% of BPDCN patients. But thats not the only way to differentiate this cancer on biopsy. CD123, CD4, CD56 as the golden triad of BPDCN, according to Dr. Pemmaraju.
Now we know there are other markers, TCL1, TCF 4, CD303 and others. When you put together these three to six markers, it helps you distinguish BPDCN, which is extraordinarily rare compared to the more common cancers like acute myeloid leukemia (AML), cutis, cutaneous t-cell lymphoma. Obviously, this differentiates completely from skin infections and all these other noncancer entities, Dr. Pemmaraju says.
The second practical consideration is that, although rare diseases commonly have limited-to-no treatment options, a therapy for BPDCN exists and more are being investigated. Studies suggest that BPDCN incidence is as low as 0.44% of all hematologic cancers and 0.7% of cutaneous lymphomas, according to the paper. Despite the low numbers, BPDCN treatment is available with tagraxofusp. Researchers are also conducting multiple clinical trials for BPDCN drugs in development, including the B-cell lymphoma-2 (BCL-2) inhibitor, venetoclax, and a chimeric antigen receptor (CAR) T-cell therapy.
The third consideration: The significance of dermatologists and dermatopathologists roles in BPDCN diagnosis.
As we educate our fellow hematologists/oncologists, we must continue to educate dermatologists, many of whom are going to see this disease for the very first time in the clinic before anybody else. I believe BPDCN will soon appear on dermatology and derm-path board exams, Dr. Pemmaraju says.
BPDCN skin lesions usually present as dark and purplish and can be maculopapular.
I would say clinically there is a somewhat distinct look, although I would add the caveat that these lesions act heterogeneously, he says.
Focusing on TagraxofuspResearchers reported that tagraxofusp, aCD123-directed cytotox, demonstrated efficacy in BPDCN patients along with an overall manageable safety profile, according to the NEJM study.
A historically deadly disease, BPDCNs median overall survival after chemotherapy had been 8 to 14 months, according to Dr. Pemmaraju. Hematopoietic stem cell transplant might prolong survival in patients healthy enough to have the stem cell transplant.
Among the 29 previously untreated patients, the survival rates of 59% at 18 months and 52% at 24 months rates that were influenced by the number of patients who went into remission after tagraxofusp therapy and could thus undergo hematopoietic stem-cell transplantation represent an improvement over rates in historically published data. We also observed a 67% overall response rate among previously treated patients. Notably, we report a meaningful survival with tagraxofusp among patients with relapsed or refractory disease (median overall survival, 8.5 months), Dr. Pemmaraju and colleagues report in the NEJM.
Interestingly, patients skin lesions went away rapidly in the vast majority of people treated with tagraxofusp, according to Dr. Pemmaraju.
Capillary leak syndrome emerged as the most drug-associated serious adverse, manifested by edema, tachycardia, hypoalbuminemia and hypotension.
There also is the issue of drug resistance. Researchers have found, in vivo, that resistance to tagraxofusp in cell lines is associated with decreased DPH1 expression and azacitadine might reverse tagraxofusp resistance.
More research is needed to study how combining tagraxofusp with other targeted therapies may improve outcomes. Research also is needed to study tagraxofusp use in pediatric patients. The FDAs approval is for tagraxofusp is as first-line therapy for BPDCN for adults and children ages 2 years and older. But only a small number of pediatric patients have been treated so far, the authors report.
DisclosureMD Anderson funded the study. Dr. Pemmaraju has received research funding/clinical trials support from Affymetrix, SagerStrong Foundation, Novaris, Stemline, Samus, AbbVie, Cellectis, Daiichi Sankyo, and Plexxikon.
1. Lee SS, Mccue D, Pemmaraju N. Tagraxofusp as treatment for patients with blastic plasmacytoid dendritic cell neoplasm. Expert Rev Anticancer Ther. 2020;:1-8.
2. Pemmaraju N, Lane AA, Sweet KL, et al. Tagraxofusp in Blastic Plasmacytoid Dendritic-Cell Neoplasm. N Engl J Med. 2019;380(17):1628-1637.
Go here to read the rest:
- Adipose Tissue-derived Stem Cell Therapy Market: Worldwide Industry to Boost in the Period of 2020-2025 - Express Journal - October 19th, 2020
- Global Stem Cell Therapy Market 2020: Trends, and Opportunity Analysis, Top Manufacturers And Forecast to 2027 - PRnews Leader - October 19th, 2020
- Stem Cell Therapy Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) - Eurowire - October 19th, 2020
- Global Adipose Tissue-derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 -... - October 19th, 2020
- Stem Cell Therapy Market Trends, Opportunities, New Technologies and Developments, Competitor analysis and Market Sizing from 2020 to 2026 - PRnews... - October 19th, 2020
- Global Adipose Derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 - TechnoWeekly - October 19th, 2020
- Notch Therapeutics Strengthens Leadership Team with Appointment of Kamran Alam as CFO and Gregory Block as SVP, Corporate Development - PRNewswire - October 19th, 2020
- Stem Cell Therapy Market To Observe Exponential Growth By 2020-2027 | Verified Market Research - Stock Market Vista - October 19th, 2020
- Stem Cell Therapy Market 2020 growth factors, latest trend and regional analysis of leading players by 2025 - News by Decresearch - October 19th, 2020
- Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Eurowire - October 19th, 2020
- Autologous Cell Therapy Market is Anticipated to Expand at a CAGR of 18.1% from 2019 to 2027 - Eurowire - October 19th, 2020
- Cell Therapy Market Trend,COVID-19 Impact,Current Industry Figures With Demand By Countries And Future Growth - PRnews Leader - October 19th, 2020
- Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration - BioSpace - October 19th, 2020
- Cell Therapy Manufacturing Market to be Worth USD 8 billion by 2030, predicts Roots Analysis - The Think Curiouser - October 19th, 2020
- Novellus Therapeutics Exclusively Licenses Induced Mesenchymal Stem Cells (iMSCs) to NoveCite for COVID-19 Related Acute Respiratory Distress Syndrome... - October 19th, 2020
- UofL-born company secures an additional $115 million to advance cell therapy - uoflnews.com - October 19th, 2020
- Global and United States Cancer Stem Cell Therapy Market Leading Players Expected to Witness the Highest Growth 2025 | AVIVA BioSciences, AdnaGen,... - October 19th, 2020
- Can Stem Cell Therapy Repair Damaged Knees? - September 2nd, 2020
- Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire - September 2nd, 2020
- COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle - BioPharma-Reporter.com - September 2nd, 2020
- New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem... - September 2nd, 2020
- Optimized Freezing Solutions for Clinical Application of Cell Therapy Products - Technology Networks - September 2nd, 2020
- Global Cord Blood Banking Market 2020 with Analysis of 44 Industry Players - PRNewswire - September 2nd, 2020
- Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 - PRNewswire - September 2nd, 2020
- New Comprehensive Report on Stem Cell Therapy Market to Witness an Outstanding Growth during 2020 2025 with Top Players Like Chiesi Pharmaceuticals,... - September 2nd, 2020
- CAR T-Cell Optimization Starts in Production, Extends to Therapy - Genetic Engineering & Biotechnology News - September 2nd, 2020
- Ruxolitinib May Be Another Option for Children With Steroid Refractory aGVHD - AJMC.com Managed Markets Network - September 2nd, 2020
- Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market - The News Brok - September 2nd, 2020
- Oldest male polar bear on record humanely euthanized at Point Defiance Zoo - MyNorthwest.com - September 2nd, 2020
- FDA Approves First Maintenance Therapy for AML - Medscape - September 2nd, 2020
- Elixirgen Therapeutics planning to begin Phase I/II Clinical Trials of its COVID-19 Vaccine Candidate EXG-5003 at Fujita Health University -... - September 2nd, 2020
- Hill Highlights the Potential of Selinexor as a Less Intensive Option for DLBCL - OncLive - September 2nd, 2020
- Global Rheumatoid Arthritis Stem Cell Therapy Market Dynamics, Forecast, Analysis and Supply Demand 2018 to 2028 - Scientect - September 2nd, 2020
- Growing at an annualized rate of over 20%, the cell therapy manufacturing market is estimated to reach close to USD 10 Billion by 2030, claims Roots... - September 2nd, 2020
- Strategic Analysis to Understand the Competitive Outlook of Cell Therapy Manufacturing Market - The News Brok - September 2nd, 2020
- In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Canine Stem Cell Therapy Coronavirus Impact Editon of Key Players VETSTEM... - August 10th, 2020
- Tevogen Bio Announces Partnership With Preeminent Scientist Professor Neal Flomenberg, MD, to Investigate Proprietary T-Cell Therapy for Treatment of... - August 10th, 2020
- Marker Therapeutics Reports Second Quarter 2020 Operating and Financial Results - PRNewswire - August 10th, 2020
- University of Minnesota expands clinical investigation of engineered iPSC-derived natural killer cells, opening U.S. clinical trial for the treatment... - August 10th, 2020
- Clinical study using mesenchymal stem cells for the treatment of patients with severe COVID-19 - DocWire News - August 10th, 2020
- UPDATED: After a 4-year sojourn, struggling microbiome pioneer Seres claims a breakout PhIII comeback. And shares respond in frenzied spike -... - August 10th, 2020
- New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19 - PharmiWeb.com - August 10th, 2020
- Covid-19 roundup: CureVac beefs up its unicorn IPO dreams as billionaire owner takes this Covid-19 mRNA player on a forced march to Nasdaq; Kodak's... - August 10th, 2020
- Coronavirus Drug and Treatment Tracker - The New York Times - August 10th, 2020
- Nearly 70K donated so far towards Wistaston boy's 120K lifesaving treatment - Cheshire Live - August 10th, 2020
- Avacta extends partnership with Daewoong Pharmaceutical and AffyXell Therapeutics for Covid-19 stem cell treatments - Cambridge Independent - August 9th, 2020
- Three Years After Stem Cell Trial for Heart Failure was Abandoned New Evidence Shows it Works - Diagnostic and Interventional Cardiology - August 9th, 2020
- Stem Cell And Platelet Rich Plasma (PRP) Alopecia Therapies Market 2020 Size by Product Analysis, Application, End-Users, Regional Outlook,... - August 9th, 2020
- IMAC Holdings Receives FDA Authorization to Initiate Clinical Study of Its Umbilical Cord-Derived Allogenic Mesenchymal Stem Cells for the Treatment... - August 9th, 2020
- Strathmore man with MS calls for more Canadian treatment alternatives - CTV Toronto - August 9th, 2020
- Wave of New Therapies Improve Outcomes for Patients with Multiple Myeloma - Dana-Farber Cancer Institute - August 9th, 2020
- Adipose Derived Stem Cell Therapy Market: Technological Advancement & Growth Analysis with Forecast to 2026 - Chelanpress - August 9th, 2020
- ASCO Expert on the Possible Benchmark Analysis of Autologous Transplantation in B-Cell Lymphomas - Cancer Network - August 9th, 2020
- Cell Therapy Market: Study Navigating the Future Growth Outlook | Osiris Therapeutics, NuVasive, Vericel Corporation - Chelanpress - August 9th, 2020
- Global Stem Cell Therapy Market 2020: Growth, Demand, Service, Types, Applications, Key Players and Industry Forecast till 2025 - Chelanpress - August 9th, 2020
- Personalized Cell Therapies Market to Show Incredible Growth by 2025 - Owned - August 9th, 2020
- Nerve Repair and Regeneration Market worth $9.7 billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire - August 9th, 2020
- High-throughput 3D screening for differentiation of hPSC-derived cell therapy candidates - Science Advances - August 9th, 2020
- Immatics Extends Cell Therapy Manufacturing Collaboration with UTHealth - GlobeNewswire - August 9th, 2020
- Contract Manufacturing Services market for stem cells is expected to be worth over USD 2.5 billion by 2030, claims Roots Analysis - Market Research... - August 9th, 2020
- In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Cancer Stem Cell Market Report 2020 Key Players Thermo Fisher Scientific Inc.,... - August 9th, 2020
- Tafasitamab Combination Approved for Adults With R/R DLBCL - AJMC.com Managed Markets Network - August 8th, 2020
- Celularity announces the activation of first California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19 - PRNewswire - August 8th, 2020
- Trending News on Targeted Oncology, Week of August 7, 2020 - Targeted Oncology - August 8th, 2020
- Find Out Why Bioreactor Market is thriving worldwide by top key players like mergency Response USA, Buddi US, LLC, Connect America - Owned - August 8th, 2020
- BCLI: Phase 3 ALS Data Expected by the End of November 2020 - Zacks Small Cap Research - August 8th, 2020
- New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19 - GlobeNewswire - August 8th, 2020
- Immatics Extends Cell Therapy Manufacturing Collaboration with UTHealth - marketscreener.com - August 8th, 2020
- Impact of COVID-19 Outbreak on Canine Stem Cell Therapy Market Expected to Secure Notable Revenue Share During 2020-2026 - Research Newspaper - August 8th, 2020
- Calidi Biotherapeutics in San Diego receives FDA Approval for COVID-19 Treatment clinical trial - - KUSI - August 7th, 2020
- Edited Transcript of BLCM.OQ earnings conference call or presentation 6-Aug-20 9:00pm GMT - Yahoo Finance - August 7th, 2020
- NICE Amends Guidance on Haematopoietic Stem Cell Transplantation During the Pandemic - Medscape - August 6th, 2020
- AlloVir raises $276M IPO to run broad cell therapy program - FierceBiotech - August 6th, 2020
- Identification and Treatment of Tuberculosis in Pediatric Recipients o | IDR - Dove Medical Press - August 6th, 2020
- Unproven 'stem cell' therapies for COVID-19 pose harm to public, says UB expert - UB Now: News and views for UB faculty and staff - University at... - August 4th, 2020
- Future Prospects of Animal Stem Cell Therapy Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | Medivet Biologics LLC,... - August 4th, 2020
- Placental Stem Cell Collection and Storage Market 2020 Segmented by Major Market Players, Types, Applications and Countries Forecast to 2026 - Market... - August 4th, 2020
- Cytovia Therapeutics Acquires Worldwide Rights to CytoImmune Therapeutics' First-In-Class EGFR Dual-targeting CAR for NK Cell Treatment of... - August 4th, 2020
- UPDATE - Cytovia Therapeutics Acquires Worldwide Rights to CytoImmune Therapeutics' First-In-Class EGFR Dual-targeting CAR for NK Cell Treatment of... - August 4th, 2020
- MediVet Biologics Rebrands as Ardent Animal Health - PR Web - August 4th, 2020