As usual, at the end of the year, I overview the most interesting and important events in cell therapy and Regen industry. The list of top 10 events reflects solely my opinion.
1. Debate on regulation of adult stem cells as drugs I think, it was the most important and interesting debate in 2012. In July, the lawsuit of Regenerative Sciences versus FDA got a new turn US Federal court ruled in favor of FDA regulation cultured adult stem cells as drugs. This ruling and ongoing debate has far-reaching implications for cell therapy industry as a whole. Based on court decision, FDA has a power to regulate stem cell clinics, which attempt to offer autologous adult stem cells (AASC) as a medical procedure on the US market. Yet another notable provocative event was an inspection of CellTex facility with following warning letter. FDA consistently and persistently says that almost all of AASC should be regulated as drugs or any other biologics, means via clinical trials (IND) and BLA (Biologics License Application). Federal court ruling and CellTex inspection triggers a lot of good and productive debates among cell therapy industry professionals, patient advocates, academics and physicians. But, it also polarized community on supporters/ non-supporters and industry on compliant/ non-compliant. The latest twist in the debate was a petition to White House Stop FDA over reach into regulating our own stem cells. We will watch these ongoing debates in the next year with a great interest!
2. Two new stem cell drugs approved on Korean market South Korean FDA (KFDA) granted market approval for 2 new stem cell drugs Cartistem (Medipost) and Cupistem (Anterogen). Cartistem is allogeneic umbilical cord-derived mesenchymal stromal cell-based product for degenerative arthritis. It becomes the worlds first approved allogeneic stem cell product and the first commercial stem cell product, derived from umbilical cord. Cupistem is autologous adipose tissue-derived mesenchymal stromal cell-based product for anal fistula (Chrons disease). It becomes the worlds first commercial approved stem cell product, derived from adipose tissue. Taking in account these new approvals, South Korea has become the worlds leader in commercialization of stem cell drugs with 3 products available on the market.
3. Cell therapy industry continues to rise in 2012 Cell therapy industry remains healthy and continues its growth. The continuing rise of industry was described in the recent interim analysis by Chris Mason. The conclusions from this analysis were based on summation of the following measures: (1) positive external influences (regulation), (2) increased commercial activity of federal agencies (NIH, CIRM), (3) stock market and clinical trials activity. If we look at public companies, interim analysis by Lee Buckler demonstrates that Cell Therapy Portfolio significantly outperform other major industry indices (Dow Jones, Nasdaq). Also, it was very very good year for funding (financing) of companies. More than $1B funds were raised and brought to the sector by companies. The expected 2012 revenue of this sector of industry is estimated as $0.9B. Please note, that we still have to wait for updated final industry reports for 2012.
4. BioPharma steps up to commercialize cellular immunotherapy Big biopharmaceutical company Novartis announced the investment in cellular immunotherapy, developed at the University of Pennsylvania for some forms of leukemia. Novartis is planning to obtain a license for manufacturing and worldwide distribution of cell product, based on Chimeric Antigen Receptor (CAR)-modified T-cell technology. This indicates that a Big Pharma company now recognizes the commercial potential of this highly personalized technology and is willing to invest to bring it to the market. As the part of commitment to cell therapy development, Novartis also recently agreed to purchase the Dendreons New Jersey manufacturing plant.
5. 2012 the year of lawsuits Unfortunately, this year we saw so many cell therapy-related lawsuits, that we have never seen before. Id mention only major cases. For the first time, the patient-initiated lawsuit against stem cell therapeutic company (RNL Bio) went public. Very recently we observed another weird case, where stem cell companies sued each other. Companies were suing regulators Cytori against FDA. Finally, stem cell company was threatening academics by strategic lawsuit against public participation (SLAPP phenomenon). As a consequence of many lawsuits in the field, a new specialization (for fraudulent stem cell therapy) of trial lawyers arose.
6. FDA CBER approved the first allogeneic cell-based product on the US market Organagenesis was granted BLA for tissue engineered product GINTUITTM (allogeneic cultured keratinocytes and fibroblasts in bovine collagen). This is the first allogeneic cell-based product, approved by CBER branch of FDA and the first tissue engineered product on US dental market. The product will be used for oral soft tissue regeneration. It took 7 years from its clinical development to commercialization. As far as I know, there is no official information about the market price and reimbursement.
7. US manufactured stem cell drug approved in Canada and New Zealand Prochymal (Osiris) allogeneic bone marrow-derived mesenchymal stromal cells for acute pediatric GVHD (Graft-versus-Host Disease) got approved in Canada and New Zealand. Osiris is seeking market opportunities for Prochymal in other countries.
8. FDA to regulate marketing claims This year, US FDA issued two warning letters, where called to cease marketing claims, related to regenerative medicine and stem cells. The first letter addressed marketing claims by Lancome according its cosmetic products. The claim was about the cream, which can improve the condition around the stem cells and stimulate cell regeneration. In the second case, the agency warns a firm, which makes and sells fat processing device, about illegal stem cell marketing claims. I think its very good move! False marketing claims about stem cell magic and regeneration magic could unreasonably inflate public expectations and threaten the legitimacy of compliant industry.
9. Industry-sponsored cell therapy clinical trials progress very well We saw a very good progress in clinical trials space in 2012. We can conclude that, based on number of trials, which have moved from early to late stages, analysis of published or reported results and number of newly registered trials. From the early stage companies with a good progress in 2012, Id indicate Advanced Cell Technology, NeuralStem and StemCells Inc. There are about 50 late stage (2/3 or 3 or pivotal) ongoing clinical trials, sponsored by ~40 companies. There were no major setbacks or significant number of failures in industry-sponsored clinical trials in 2012. As for potential market approvals in 2013, we should watch Sanofi with MACI in Europe, Pharmicell with product Livercellgram in Korea, GamidaCell with product StemEx in Israel, cancer vaccine by NovaRx in US and some others. Overall, were expecting much better success rate for the progression and approval of cell therapy trials compare to conventional drugs and biologics.
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Regenerative medicine and cell therapy industry in 2012 | Stem Cell ...
- ACT - Stem Cell Trial for AMD and Stargardt - Phase 1 - September 15th, 2011 [September 15th, 2011]
- Excerpt from: "The Urban Wire" Internet Radio Podcast: FDA Oks embryonic stem cell trials for humans - September 15th, 2011 [September 15th, 2011]
- Advanced Cell Technology (ACT) AMD stem cell clinical phase - September 15th, 2011 [September 15th, 2011]
- Stem Cells Retina and cornea regeneration ACT Dr lanza - Advanced Cell Technology - September 15th, 2011 [September 15th, 2011]
- Spinal Patient Receives Stem Cells in First Experimental Treatment - September 15th, 2011 [September 15th, 2011]
- Bloomberg: Geron, StemCells Rise 4% - September 15th, 2011 [September 15th, 2011]
- Bringing Stem Cell Cures to the Clinic: UC Davis GMP Facility - September 15th, 2011 [September 15th, 2011]
- Researchers in California Show Results for Spinal Cord Injuries Using Embryonic Stem Cells - September 15th, 2011 [September 15th, 2011]
- Bill Caldwell, Chairman - September 20th, 2011 [September 20th, 2011]
- Advanced Cell Technology OneMedForum 2011 - September 20th, 2011 [September 20th, 2011]
- ACT edmund vision stem cells program Advanced Cell Technology - September 20th, 2011 [September 20th, 2011]
- Texans for Stem Cell Research Learn for Life: KXAN Coverage Aired August 16th, 2011 - September 20th, 2011 [September 20th, 2011]
- ACT - Dr Lanza - Hermangioblast program - Advanced cell technology - September 23rd, 2011 [September 23rd, 2011]
- Anticipating the Next Decade of the Genome - Francis Collins, Director of NIH - September 24th, 2011 [September 24th, 2011]
- New Epilepsy Treatment Under Study For Seizures - Part 1 - September 24th, 2011 [September 24th, 2011]
- A Thank You For Trevor Raborn - September 24th, 2011 [September 24th, 2011]
- CBLI Breaking News - September 24th, 2011 [September 24th, 2011]
- What is Cord Blood Banking? The Medical Potential of Newborn Stem Cells - September 24th, 2011 [September 24th, 2011]
- Stem cell medical breakthrough? - September 24th, 2011 [September 24th, 2011]
- NIST Colloquium Series: Next Generation Neural Implants - September 24th, 2011 [September 24th, 2011]
- Alan Lewis: Embryonic Stem Cell Therapies for diabetes - September 24th, 2011 [September 24th, 2011]
- 2011 Predictions for Hair Transplant Developments | Arocha Hair Restoration - September 24th, 2011 [September 24th, 2011]
- Healthbeat - Stem Cell Research - September 24th, 2011 [September 24th, 2011]
- Living With ALS - September 24th, 2011 [September 24th, 2011]
- A Revolutionary New Potential Treatment for Glioblastoma... - September 24th, 2011 [September 24th, 2011]
- Lyme Disease: Challenges and Innovations - September 24th, 2011 [September 24th, 2011]
- 2010: Cord blood saving lives [CNN: 5-30-2011] - September 24th, 2011 [September 24th, 2011]
- Biotechnology, Diagnostics, and Genomics: Panel Discussion - September 24th, 2011 [September 24th, 2011]
- Pharmacogenomics - September 24th, 2011 [September 24th, 2011]
- Brickheads - Osteobots Practice Skit.mpg - September 24th, 2011 [September 24th, 2011]
- A veteran lives with ALS - September 26th, 2011 [September 26th, 2011]
- Healthy Body = Functional Body. Hollywood Look Side Effect: Healthy Function - September 26th, 2011 [September 26th, 2011]
- 2009 Flu Summit: Opening Remarks - September 26th, 2011 [September 26th, 2011]
- Cord Blood Breakthrough - September 28th, 2011 [September 28th, 2011]
- SENS5 - Leucocyte transfusions as a novel immunotherapy for cancer patients - October 2nd, 2011 [October 2nd, 2011]
- Lymphoma Drug SGN-35 - The Nebraska Medical Center - October 2nd, 2011 [October 2nd, 2011]
- ACell Hair Restoration with Dr. Gary Hitzig and Dr. Amiya Prasad - Video - October 14th, 2011 [October 14th, 2011]
- Hans Keirstead: Developing therapies based on embryonic stem cells - Video - October 14th, 2011 [October 14th, 2011]
- NJN News Healthwatch Report - Cornea Treatment Study - Video - October 20th, 2011 [October 20th, 2011]
- HIV/AIDS may be prevented by new lubricant - Video - October 22nd, 2011 [October 22nd, 2011]
- MFIII Switzerland PE Sheep Placenta Glandular therapy Study - Client Interviews - Video - October 22nd, 2011 [October 22nd, 2011]
- Love Diabetes: BCG and Noncompliant Patients - Video - October 25th, 2011 [October 25th, 2011]
- Dr. Alimorad Farshchian statement on embryonic stem cells - Video - October 27th, 2011 [October 27th, 2011]
- StemCellTV Daily Report-October 29, 2011 - Video - October 30th, 2011 [October 30th, 2011]
- Stephen McKenna MD - 8 - Video - October 31st, 2011 [October 31st, 2011]
- ALS Stem Cell Trial at Emory - Video - November 22nd, 2011 [November 22nd, 2011]
- Target Product Profile: Ellen Feigal - 2011 CIRM Grantee Meeting - Video - December 6th, 2011 [December 6th, 2011]
- Monoclonal Antibodies: Stanford Academia-Industry Collaboration - Video - December 8th, 2011 [December 8th, 2011]
- Parkinson's Disease Guidelines (Part 2) - Video - December 9th, 2011 [December 9th, 2011]
- Macular Degeneration Drugs - Video - December 13th, 2011 [December 13th, 2011]
- BrainStorm Cell Therapeutics (OTCBB:BCLI) Has Potentially Developed A Treatment For ALS - January 31st, 2012 [January 31st, 2012]
- StemCells, Inc. Receives FDA Authorization for Age-Related Macular Degeneration Clinical Trial - February 3rd, 2012 [February 3rd, 2012]
- Stemedica Cell Technologies - Documentary - Video - February 4th, 2012 [February 4th, 2012]
- (GMCR, STEM, DSCO) CRWENewswire.com Stocks In Action - Video - February 7th, 2012 [February 7th, 2012]
- Bioheart Acquires Exclusive Rights to Ageless Regenerative Institute's Adipose Cell Technology - February 14th, 2012 [February 14th, 2012]
- Neuralstem Announces Closing of $5.2-Million Registered Direct Offering - February 14th, 2012 [February 14th, 2012]
- Scientists Repair Heart Attack Damage Using Patient's Own Stem Cells To Regrow Healthy Heart Muscle - February 14th, 2012 [February 14th, 2012]
- Stem Cells Regrow Healthy Heart Muscle In Heart Attack Patients - February 15th, 2012 [February 15th, 2012]
- Groundbreaking Clinical Trials Study Cord Blood Stem Cells to Help Treat Brain Injury and Hearing Loss - February 17th, 2012 [February 17th, 2012]
- Novelos Therapeutics Announces Enrollment of First Patient in Lung Cancer Trial With I-124-CLR1404 (Light) Cancer ... - February 22nd, 2012 [February 22nd, 2012]
- IntelliCell BioScience Inc. Procedure Enables Norwegian Star Basketball Player to Fully Recover from Patella ... - February 22nd, 2012 [February 22nd, 2012]
- The Lancet Publishes Results from Gentium's Phase III Defibrotide Trial for the Prevention of VOD in Paediatric Patients - February 27th, 2012 [February 27th, 2012]
- Bioheart Issues Shareholder Letter Announcing Progress and New Initiatives - March 1st, 2012 [March 1st, 2012]
- Bioheart Issues Shareholder Letter Announcing Progress - March 1st, 2012 [March 1st, 2012]
- Novelos Therapeutics and Academic Collaborators Have Three Abstracts Accepted for Presentation at 2012 AACR Annual ... - March 1st, 2012 [March 1st, 2012]
- Marshall Edwards Submits Investigational New Drug Application for Oncology Drug Candidate ME-344 - March 6th, 2012 [March 6th, 2012]
- Will StemCells Walk The Talk? - March 7th, 2012 [March 7th, 2012]
- Internationally Recognized Leukemia Physician and Researcher to Lead Sylvester Comprehensive Cancer Center - March 7th, 2012 [March 7th, 2012]
- Criminal investigation after patient dies at office of doctor linked to stem cell - March 9th, 2012 [March 9th, 2012]
- Novelos Therapeutics Announces Enrollment of First Patient in Brain Cancer Trial with I-124-CLR1404 (LIGHT) Cancer ... - March 15th, 2012 [March 15th, 2012]
- Vitro Biopharma Receives Approval for Presentation to the International Society for Cellular Therapy - March 20th, 2012 [March 20th, 2012]
- Looking for Innovative Small-Cap Biotech Companies: George Zavoico - March 26th, 2012 [March 26th, 2012]
- Seattle Genetics Announces Pivotal ADCETRIS™ (Brentuximab Vedotin) Hodgkin Lymphoma Study Published in Journal of ... - March 27th, 2012 [March 27th, 2012]
- Gentium Reports Fourth Quarter and Year End 2011 Financial Results - March 28th, 2012 [March 28th, 2012]
- Basketball’s influence on stem cell treatments in sports medicine - March 30th, 2012 [March 30th, 2012]
- Cardium Announces U.S. Market Introduction of Excellagen™ - March 31st, 2012 [March 31st, 2012]
- Optimer Pharmaceuticals, Inc. Announces Presentation at ECCMID of Post-Hoc Subgroup Analysis of DIFICID in Adult ... - March 31st, 2012 [March 31st, 2012]
- New Data Demonstrates DIFICLIR™ May Offer Benefits for Cancer Patients, who are at High Risk of Clostridium Difficile ... - April 1st, 2012 [April 1st, 2012]
- SynGen Inc. Receives $5 million Equity Investment from Bay City Capital, LLC - April 19th, 2012 [April 19th, 2012]
- Gene Ray, Founder of $2.6 Billion Titan Corp, Invests in Medistem's ERC Technology and Joins Advisory Board - April 26th, 2012 [April 26th, 2012]