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 world’s 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 (Chron’s disease). It becomes the world’s first commercial approved “stem cell product”, derived from adipose tissue.
Taking in account these new approvals, South Korea has become the world’s 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 Dendreon’s 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. I’d 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 it’s 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, I’d 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, we’re expecting much better success rate for the progression and approval of cell therapy trials compare to conventional drugs and biologics.
10. More cord blood products licensed in US
Since the first approval of licensed cord blood product (HemaCord) and FDA mandate for cord blood banks, two more products got BLA this year. First is a cord blood product of Clinimmune Labs, University of Colorado Cord Blood Bank. The second product is Ducord (Duke University, Carolinas Cord Blood Bank). These cord blood products are considered as “stem cell products” and could be commercially distributed for homologous (hematological) applications. So, despite high cost and some other hurdles, US cord blood banks are moving to that direction.
Disclosure: I’m involved in CAR T-cell clinical trials in University of Pennsylvania. I don’t have any financial conflicts of interest and I don’t own stocks of any companies, mentioned in this post.