Cell therapy highlights from #ASH14

by Alexey Bersenev on December 8, 2014 · 0 comments

in Uncategorized

Annual 56th meeting of American Society for Hematology (ASH) is about to finish in San Francisco. I was following conference via twitter and I was amazed by stream of tweets – very good tweets, high value tweets, unpublished data tweets, 1 tweet per second! There was no “scientific tweets”, but almost all were “clinical”. Here, I’m going share some fresh exciting information from ASH 2014.


1. CAR T-cell therapies
Using gene-modified T-cells is one of the most popular topic this year at ASH. Few companies and academic institutions reported results of Phase 1-2 trials, assessing CAR T-cells in hematological malignancies. Efficacy – 80-90% in “no options” patients in some leukemias (ALL). I summarized tweets about CAR T-cell therapy in one archive.

2. “Preliminary success” in gene therapy of beta-thalassemia
Blubird Bio reported “transfusion-free” outcome in 4 patients from 3 to 12 months. Even though, it’s too early to conclude, but transfusion independence in some patients with normal level of Hb is a big achievement. Read more here.

3. TILs in metastatic melanoma
Lion Biotechnologies reported preliminary results of Phase 2 trial, assessing autologous tumor infiltrating lymphocytes (TILs) in stage 4 metastatic melanoma. In 101-patients randomized trial, efficacy was 54% even after failing other “high profile” experimental treatments (ipilimumab and anti-PD1 therapy). 13 patients became melanoma-free for more than 2 years (reported observation time).

4. MSCs for GVHD
I’ve picked 2 studies, assessing mesenchymal stromal cells in GVHD. The first, 77-patients Russian trial, assessing allo- matched MSC for GVHD prophylaxis. MSC decreased twice of acute GVHD incidence and increased 5-year survival. The second study, by Japanese company JCR Pharmaceuticals, assessing allo- MSC in steroid-resistant acute GVHD. The candidate product is Prochymal (commercialized as JR-031), inherited from Osiris and pushing to Japanese market by Mesoblast via JCR. After 8 infusions, about half of patients had complete response at time point 6 months. But only 60% survived longer than 6 months after first infusion. All patients had adverse events (relation to MSC is not proven). Unfortunately, there was no placebo group and total number of patients was only 25.

5. New “mild” conditioning in stem cell transplant
Toxicity of conditioning before stem cell transplant is a huge problem and one of the major causes of mortality. “reduced intensity” preparative regimens have been tested in trials, but many physicians still suggest myeloablation and radiation for durable engraftment of donor cells. Very interesting study from Dana-Faber Cancer Institute, suggests that immunosuppression only could be used for successful engraftment of donor stem cell in patients with dyskeratosis congenita. Only first 4 cases were reported, but it looks promising. Such “mild” regimens could be potentially applied in hematopoietic cells transplantation for autoimmune (lupus, systemic sclerosis) and neurological conditions (multiple sclerosis). Also read here.

6. New agent for enhancement of cord blood cells
Fate Therapeutics continues to play with cord blood cells enhancers. New compound FT4145 is able to boost 60-fold expression of homing receptor CXCR4 on CD34+ cells. Company reports preclinical data for FT4145. Also, company reported changes in manufacturing process in Phase 2 of ProHema cord blood enhancement.

7. StemRegenin-1 to expand cord blood cells
John Wagner from U of Minnesota reported preliminary results of cord blood expansion by compound HSC835 (commercialized by Novartis). Results are impressive! Great expansion of CD34+ cells ex vivo, durable engraftment from manipulated unit up to 2 years, shortening neutrophil engraftemt to 11 days. Wagner proposing it as a fix for collection inefficiencies: “units with cell counts <1 billion nucleated cells should no longer be discarded". Trial with one manipulated unit is coming.

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