NeoStem
420 Lexington Avenue
Suite 350
New York, New York 10170
Telephone: 1.212.584.4180

Target: Malignant Melanoma

About Melanoma

Melanoma, which originates in pigment-producing cells known as melanocytes, is the most lethal form of skin cancer. Melanoma is often caused by unrepaired DNA damage to skin cells from UV radiation. Patients who have progressed to stage IV melanoma have a cancer that has metastasized, or spread to distant sites in the body which may include the lymph nodes, lungs, liver, or brain. As a result, advanced melanoma is exceedingly difficult to treat, with a 5-year survival rate of approximately 15%1. There are 120,000 new cases per year in the U.S.2 and melanoma kills an estimated 8,790 in the U.S. annually3.

Treatments for stage IV melanoma are typically directed at slowing the growth of the cancer and prolonging survival. Current treatment options include radiation, chemotherapy, surgical resection, immunotherapy, or a combinatory approach. Unfortunately, most current approaches provide only temporary relief, with the metastatic cancer returning 3 to 6 months after treatment. Administering certain anti-melanoma drugs at higher doses appears to be more effective, but often results in more severe side effects.

In recent years immunotherapy and targeted drugs have emerged as promising approaches to treating advanced cancer. NeoStem’s lead product candidate, DC/TC, targets malignant melanoma

NeoStem’s Phase 3 Intus Study of DC/TC*                      

NeoStem has received U.S. FDA approval to commence a Phase 3 trial assessing the efficacy of its patient specific cancer immunotherapy DC/TC (irradiated autologous in vitro proliferating melanoma cell line loaded onto an autologous dendritic cell combined with granulocyte macrophage colony-stimulating factor {GM-CSF}) in patients with late stage metastatic melanoma. The study has Special Protocol Assessment (SPA) and Fast Track designation, and the therapy has been awarded Orphan Drug designation. For additional details, please visit the trial’s listing on clinicaltrials.gov.

(Notice: Patient recruitment for this trial has not yet begun.)

The study is expected to be initiated later in 2014.

About DC/TC

DC/TC is an autologous initiating (stem) cell immune based therapy intended to eliminate the tumor cells capable of causing disease recurrence and is initially directed at patients with metastatic melanoma. NeoStem’s immunotherapeutic approach uses the patient’s isolated and purified tumor stem cells to train the immune system (Killer T-cells, CD8+) to identify and eliminate cancer stem cells, the root cause of tumor formation and the key drivers of tumor escape, tumor genesis, self-renewal and recurrence of cancer. We believe this platform technology is applicable for a broad spectrum of solid tumor cancers.

Phase 2 Results

In a Phase 2 randomized clinical trial of DC/TC, patients showed a significant improvement in two year overall survival from 31% in controls to 72% in treated patients (p=0.007) with advanced melanoma (Stage IV and recurrent Stage III). The toxicity profile was favorable with no grade IV and only one grade III (allergic reaction) event in the study. View trial results.

Facts about DC/TC

  • DC/TC is intended to be given after surgery, radiation, chemotherapy, targeted therapies, or other immunotherapies that decrease the total cancer volume, but do not eliminate the cancer initiating (stem) cells or progenitor cells.  The treatment can also be given prior to other treatments.
  • Unlike approved and many experimental dendritic cell therapies which are simply a means of presenting to the patient’s immune system the broad array of antigens associated with the cancer stem cells, NeoStem’s therapy is designed to specifically expose the immune targets of the cancer initiating (stem) cells that make cancer lethal. This focuses the immune attack on the specific elements of the tumor that enable it to grow and metastasize, which is not the case with currently available approaches.
  • In the candidate’s randomized Phase 2 trial, survival was better in patients whose immune system was exposed to antigens presented by the exogenously expanded dendritic cells, rather than just by injecting the irradiated cancer cells themselves.
  • Patients may be enrolled in the study regardless of prior therapy, they are not restricted in terms of therapy at the time they would be receiving DC/TC and there is no subsequent restriction after they have completed that therapy. The key entry criterion is that the patient must have had a tumor resected from which a stem cell has been able to establish the self-renewing cell line.
Summary of Intus Study
Target Patients with metastic melanoma (Stage IV or recurrent Stage III)
Location and Number
of Subjects
United States and Europe, multicenter, 250 patients**
Design Double blind, placebo controlled, randomized (2:1)
Endpoint Overall Survival
Treatment Group DC/TC (autologous dendritic cells pulsed with irradiated tumor cells in GM-CSF)
Control group Autologous mononuclear cells (MC) in GM-CSF
Special Protocol assessment (SPA) Indicates FDA is in agreement with the design, clinical endpoints and planned clinical analysis of this Phase 3 trial and could serve as the basis   for a Biologics License Application

* NeoStem has submitted a United States Adopted Names Council application for the Company’s DC/TC product for metastatic melanoma to use the generic name Melapuldencel-T

** Company may overenroll by 10%

  1. AJCC Cancer Staging 2010 (based on 17 academic centers)
  2. American Cancer Society
  3. Skin Cancer Foundation

 

NeoStem - Cell Therapy Development - Clinical