Last week, The Dish posted a blog titled “Fifteen Cell Therapies/Stem Cell Therapies in Phase III Clinical Trials,” where we gave an overview of five stem cell therapies currently in Phase III clinical trials, the diseases states they are treating, the technologies they employ, and the companies who are working to bring these products to market. In Part II we will cover five more therapies and next week the final five in Part III. These therapies were identified in a report from the Pharmaceutical Research and Manufacturers of America’s titled, “Medicines in Development – Biologics, 2013 Report.” In the report, 69 cell therapies were identified as having clinical trials under review with the Food and Drug Administration (FDA), including 15 in Phase III clinical trials. Of the therapies listed, nine therapeutic categories were represented and include: cardiovascular disease, skin diseases, cancer and related conditions, digestive disorders, transplantation, genetic disorders, musculoskeletal disorders, eye conditions, and other.

ELAD –Cell Therapy (Orphan Drug)

Sponsor – Vital Therapies

Liver Failure

Vital therapies has created ELAD, a cell based bioartificial liver that has been granted Orphan Drug Designation. The product has completed a Phase II/III clinical study and according to the Vital Therapies website is “in the process of finalizing a pivotal clinical trial program designed to support ELAD’s potential approval in the United States, Europe, Australia and New Zealand. This program incorporates two randomized, controlled trials in severe acute alcoholic hepatitis and one randomized, controlled trial in fulminant hepatic failure. We expect to initiate this clinical program in Q1 2013.”

ELAD is made up of a cartridge, which contains proprietary C3A human hepatocyte cells licensed from Wistar Institute and further developed by Baylor College. The cells are grown in specialized cartridges that serve as an artificial liver at the patient’s bedside. Blood is pumped from the patient through the system and the cells are able to perform most of the metabolic functions of a normal liver.

According to the Vital Therapies website, in the United States there were about 6,000 liver transplants performed in 2006, but 16,000 patients were on the waiting list. Each year only about one-third of people who need a liver transplant will receive one. The goal of ELAD is to provide liver support for up to 30 days hopefully allowing patients time to find a suitable liver transplant or give their own liver time to recover to a healthy state.

HP802 (allogenic human skin replacement)–Cell Therapy

Sponsor – Healthpoint Biotherapeutics (recently acquired by Smith & Nephew)

Leg Ulcers

Healthpoint Biotherapeutics’ HP802 is currently enrolling a Phase III study for treatment of venous leg ulcers. According to Healthpoint Biotherapeutics’ website, “the product is an allogeneic living human cell suspension that consists of two components that are sprayed sequentially on the wound bed at the time of treatment: a fibrinogen solution and a cell preparation containing a mixture of growth arrested, living, allogeneic epidermal keratinocytes and dermal fibroblasts”. The product is believed to release growth factors and cytokines into the wound, which promote wound healing in the patient’s cells.

Venous leg ulcers are caused by poor circulation in the legs caused by damaged veins. Typical risk factors for venous leg ulcers are peripheral arterial disease, obesity, diabetes, immobility, deep vein thrombosis, and serious leg injury.

StemEx (carlecortemcel-L) – Stem Cell Therapy (Orphan Drug) (Fast Track)

Sponsor – Gamida Cell and Teva Pharmaceutical

Hematological Malignancies

StemEx is a joint venture between Gamida Cell and Teva Pharmaceutical. StemEx is currently in a Phase III clinical trial for treatment of hematological malignancies. The product would serve as a replacement for a bone marrow transplant and has been granted both Orphan Drug Designation and Fast Track Status with FDA. Patients that suffer from hematological malignancies, such as leukemia and lymphoma, almost always require bone marrow transplants and many patients will not be able to receive one because they will be unable to find a donor that is a suitable match. According to Gamida Cell’s website “StemEx is a graft of stem/progenitor derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit.” If approved, this product would be able to provide a suitable replacement for bone marrow transplant using donor bone marrow and would greatly increase the number of hematological malignancies patients that could receive treatment.

LaViv – Fibroblast Cell Therapy

Sponsor – Fibrocell Science

Scars

LAVIV for treatment of scaring has completed a Phase II/III clinical study. LAVIV is already approved by the FDA for treatment of moderate to severe nasolabial fold wrinkles in adults. In this autologous therapy, skin is collected from behind the patient’s ear then the sample is expanded to hundreds of millions of fibroblast cells prior to treatment. Fibrocell Science states the expansion time plus quality control takes 90 days. Cells are then injected back into the patient at the site of the scar or wrinkle.

GSK 2696273 – Stem Cell Therapy

Sponsor – GlaxoSmithKline

Adenosine Deaminase Severe Combined Immune Deficiency

GlaxoSmithKline’s 2696273, although listed as a stem Cell Therapy, uses an ADA gene transfer into hematopoietic stem/progenitor cells for the treatment of Adenosine Deaminase Severe Combined Immune Deficiency. According to the clinical trials.gov listing for their Phase II study, “The drug product studied in this protocol consists of autologous CD34+ hematopoietic stem/progenitor cells engineered ex vivo with a retroviral vector encoding the therapeutic gene ADA. The engineered CD34+ cells are infused following a nonmyeloablative conditioning with busulfan to make space in the bone marrow.”

Adenosine Deminase Severe Combined Immune Deficiency (ADSCID) is a genetic disorder that is inherited. Children with (ADSCID) have a damaged immune system that does not provide immune protection from common challenges such as bacteria, fungi and viruses. These infections can be life threatening to children with this disorder and as a result, they typically only live a year or two.

I hope that this review has been useful in identifying cell therapies that are in late stage trials. I will post part III of this blog next week with the remaining 5 therapies covered. In the meantime, which of these therapies do you think is the most promising? Which would be the most significant?