Geron Ends Stem Cell Programs in November: Big hope for a spinal cord injury trial, big loss for a field — the most discussed news of the year. Despite the company’s official comment citing a “purely business decision,” many professionals think that a “lack of impressive preliminary results” also played a role. The company is now seeking a partner to take over that trial. The effect on the cell therapy industry remains to be seen — but for some developers, scientists, and patients it’s a big loss. We will remember Geron as a pioneer in clinical translation of embryonic stem cell therapy, a prototype developer and trailblazer in the regulatory path.
First Patients Treated with Embryonic Stem Cell Products: Despite Geron’s discontinuation, five spinal cord injury patients were treated. And as far as we know, none of them developed complications or adverse events related to their treatment. But we don’t have any information about functional improvement in disease course. Another company — Advanced Cell Technology (ACT) — continues to enroll and treat patients with eye diseases in two US phase 1–2 trials and one UK trial. We don’t have preliminary results or information about the number of patients treated so far, but we expect it soon.
World’s First Stem Cell Therapeutic Approval for Nonhomologous Use: In July, South Korean company FCB PharmiCell got an approval by the Korean FDA (KFDA) for commercial sales of HeartiCellgram, the world’s first stem cell product for nonhomologous use (post acute myocardial infarction). This is an autologous bone marrow–derived product enriched for mesenchymal stem cells. According to The Scientist, “there has been concern over a lack of independent validation of the treatment, Reuters reports, as FCB-Pharmicell’s new product is yet to be vetted by a peer-review publication.” In cost/benefit terms, patients will pay ∼US$19,000 for a 6% improvement in their left ventricular ejection fraction.
The “Dendreon Effect”: After the remarkable approval of the first immunotherapeutic cellular cancer vaccine on the market, the whole field was watching Dendreon’s performance. Good news came first: The expensive Provenge cell therapy drug gets reimbursement approval from Medicare! Later, the company announced that earnings missed its forecasts and sales were slowing. Dendreon’s shares fell dramatically; some investors lost their trust and confidence in the company. Seeking Alpha reported that “This ‘Dendreon Effect’ pulled down the share price of several companies developing cancer vaccines.”
FDA Approves First Personalized Aesthetic Cell Therapy: In June, Fibrocell Science announced FDA approval for laVív azficel-T, the first personalized aesthetic cell therapy to be marketed in the United States. This autologous cell product (made from a patient’s skin fibroblasts) will be commercially used in adult patients with facial wrinkles. The cost for a course could vary ∼US$1,600–3,300. We can’t wait the first sales results and postmarketing analysis. RIP, Botox?
New Gene Therapy Drug Approved: In December, Russian company Human Stem Cell Institute announced approval of its gene therapy drug for treatment of peripheral arterial disease: the first gene product approved outside China. Neovasculogen (VEGF) stimulates angiogenesis and could be considered the first gene drug in regenerative medicine. I interviewed CEO Artur Isaev for the Stem Cell Assays blog on 19 December 2011: http://hematopoiesis.info/2011/12/19/gene-therapy-drug-approved-russian-market-interview-artur-isaev.
Biggest Business Deal in Regenerative Medicine History: In May, biopharmaceutical giant Shire acquired Advanced BioHealing for US$750 million in cash. This was clear proof that regenerative medicine investors can get a big cash-on-cash return. The deal broke a long-persistent venture capitalist dogma that “healthcare returns just can’t match tech returns.“ We can!
First Product Approved for Cell Tracking in Clinical Trials: In June, Celsense announced FDA approval for the first use of its cell-tracking imaging product in clinical trials. Now we can track cells after transplantation and gain more knowledge about the mechanisms of their therapeutic action.
Dubious Stem Cell Clinic Closed: In May, authorities forced the German XCell Center to close. It was the biggest “stem cell clinic” in the Western world and a famous “stem cell tourism” center. This case shows that authorities can deal with unproven “stem cell treatments.” Such “stem cell tourism” is big business and should be regulated.
Online Professional Community Is Growing Strong: The online cell therapy community grew tremendously in 2011. I’m discussing news with professionals every day using LinkedIn and Twitter. The Cell Therapy Industry group on LinkedIn has >2,300 members and continues to grow. Some people worth following on Twitter include @celltherapy, @david_brindley, @Prof_ChrisMason, @natashadavie, @BLLPHD, and @pknoepfler. There’s also the Cell Therapy Bioprocessing Tools and Reagents Database (ISCT pass-log required) and Cell Therapy Trials tracking on Twitter.2011 was very interesting and remarkable year full of exciting news and events. I expect even more in 2012.
Editor’s Note: 2012 Starts with a Bang
Later this year, I’ll provide a more in-depth update in the form of a special report, but here’s a preview: Dendreon posted surprisingly positive results for 2011′s fourth quarter. And in January, South Korea approved the world’s first allogeneic stem cell product. Cartistem therapy for regenerating knee cartilage using stem cells was developed from umbilical cord blood by Seoul-based Medipost, which is in now licensing talks with two top drugmakers. US trials are expected to begin in 2015.
—Cheryl Scott, senior technical editor
Alexey Bersenev, MD, PhD, is a resource technologist in the clinical cell and vaccine production facility at Children’s Hospital of Philadelphia, 34th Street and Civic Center Boulevard, Philadelphia, PA 19104; firstname.lastname@example.org; www.chop.edu. His research focus is on normal and malignant hematopoiesis. He is also founder of the Stem Cell Assays blog (http://stemcellassays.com), where this list was originally posted.