Posted on June 25th, 2014
by Douglas Losordo
Even if you have never had cancer you probably know that treatment for cancer is rough. Nausea, plummeting blood counts, risk of life threatening infection, hair loss ….the list goes on. In fact, the toxicity of cancer treatments is so common that among cancer doctors it’s more or less expected. I’ll come back to that a little later.
But it turns out that that might not have to be the case.
A year or two before I joined NeoStem I was at a meeting on “Cell Therapy” and I saw a presentation of data from a randomized, blinded Phase 2 investigational study of a cell based treatment for metastatic melanoma. Melanoma is the most lethal form of skin cancer and once it reaches the stage of being recurrent or metastatic it is almost uniformly fatal, with only a small percentage of people surviving. The presentation described a new therapy in which each patient’s tumor was used to train the patient’s own immune system to attack the tumor. The data from the clinical study showed that the treated patients had more than a doubling of their two year survival, from 31% for controls to 72% for those who received the novel therapy.
At the recent American Society for Clinical Oncology meeting in Chicago, a pooled analysis of data from three trials for this melanoma cell therapy was presented (http://www.neostem.com/media/press-releases/news-item/neostem-presents-asco/). There has been over 20 years of experience investigating this therapy at this point. The toxicity data demonstrated no Grade IV (life threatening toxicity) and only one Grade III (allergic reaction attributed to the GM-CSF1) event in the pooled data. No significant adverse effects were reported regarding hematopoietic cells or renal function, hepatic function, or patient performance status. In addition the pooled survival data were also encouraging: For all patients 5 year survival with the treatment was 33%, which doesn’t sound great until you compare it to the 20% survival for control subjects. Furthermore the survival advantage was evident for all subgroups of patients. I hasten to point out that this observational evidence is no substitute for a properly conducted randomized controlled trial, but one can’t help but be a little curious about this novel approach
So wait a minute. Better survival, with less than traditional cancer-therapy levels of toxicity? Why haven’t you heard about this yet? Maybe this goes back to the “expectation” of toxicity for potent cancer therapies. This treatment is clearly not part of that paradigm.
NeoStem is here to change paradigms. During my first week at NeoStem, I started making phone calls to see what was happening with this technology, because I knew it had not yet advanced to a Phase 3 study. It just so happened that the trial was approved, but there was not yet funding for the work. So, simply put, and taking a page out of the old razor commercial, we liked the technology so much, we bought the company. We are delighted that the inventor of this therapy, Dr. Robert Dillman, is now the Vice Present of Oncology for NeoStem Oncology following the acquisition of California Stem Cell, now NeoStem Oncology. Dr. Dillman has championed this paradigm-changing approach to cancer therapy for over 20 yrs. In 2011 he formed a partnership with Dr. Hans Keirstead and the team at California Stem Cell to refine the manufacturing of the melanoma cell therapy. Together we are now poised to launch a randomized, controlled Phase 3 pivotal clinical trial of this therapy in 2014 which, if positive, could result in the approval of this treatment for advanced melanoma.
And this strategy isn’t limited to melanoma – it could be logically applied to many tumor types based on the stimulation of an aggressive response of each patient’s own immune system against the patient-specific tumor.
Can the oncology community come to accept a cancer therapy that isn’t toxic?? I’m pretty sure the patients can.
Read other blog posts by Dr. Douglas Losordo:
Forward Looking Statements
This blog contains “forward-looking” statements within the meaning of the Private Securities Litigation Reform Act of 1995, as well as historical information. Such forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements, or industry results, to be materially different from anticipated results, performance or achievements expressed or implied by such forward-looking statements. When used in this blog, statements that are not statements of current or historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, the words “plan,” “intend,” “may,” “will,” “expect,” “believe,” “could,” “anticipate,” “estimate,” or “continue” or similar expressions or other variations or comparable terminology are intended to identify such forward-looking statements, although some forward-looking statements are expressed differently. We remind readers that forward-looking statements are merely predictions and therefore inherently subject to uncertainties and other factors and involve known and unknown risks that could cause the actual results, performance, levels of activity or our achievements or industry results, to be materially different from any future results, performance levels of activity or our achievements or industry results expressed or implied by such forward-looking statements. Such forward looking statements appear in this blog. Factors that could cause our actual results to differ materially from anticipated results expressed or implied by forward-looking statements include, among others:
• our ability to obtain sufficient capital or strategic business arrangements to fund our operations and expansion plans, including meeting our financial obligations under various licensing and other strategic arrangements, the funding of our clinical trials for product candidates in our development programs for our Targeted Cancer Immunotherapy Program, our Ischemic Repair Program and our Immune Modulation Program, and the commercialization of the relevant technology;
• our ability to build and maintain the management and human resources infrastructure necessary to support the growth of our business;
• our ability to integrate our acquired businesses successfully and grow such acquired businesses as anticipated, including expanding our PCT business internationally;
• whether a large global market is established for our cellular-based products and services and our ability to capture a meaningful share of this market;
• scientific and medical developments beyond our control;
• our ability to obtain and maintain, as applicable, appropriate governmental licenses, accreditations or certifications or comply with healthcare laws and regulations or any other adverse effect or limitations caused by government regulation of our business;
• whether any of our current or future patent applications result in issued patents, the scope of those
patents and our ability to obtain and maintain other rights to technology required or desirable for the conduct of our business; our ability to commercialize products without infringing the claims of third party patents;
• whether any potential strategic or financial benefits of various licensing agreements will be realized;
• the results of our development activities, especially:
• the results of our planned Intus Phase 3 clinical trial of NBS20 being developed to treat metastatic melanoma;
• the results of our PreSERVE Phase 2 clinical trial of NBS10 being developed to treat acute myocardial infarction for which we released initial data on November 17, 2014 and for which all 6 and 12 month data has been collected; however it is subject to ongoing analysis, and currently reported results, although promising, are preliminary and there can be no assurance that further analysis may not reveal negative, or less promising, results;
• our ability to complete our other planned clinical trials (or initiate other trials) in accordance with our estimated timelines due to delays associated with enrolling patients due to the novelty of the treatment, the size of the patient population and the need of patients to meet the inclusion criteria of the trial or otherwise; and
• the other factors discussed in “Risk Factors” in our Form 10-K filed with the Securities and Exchange Commission (“the SEC”) on March 2, 2015, and elsewhere in the Annual Report on Form 10-K.
The factors discussed herein, including those risks described in Item 1A. “Risk Factors” in the Company's Annual Report on Form 10-K filed with the SEC on March 2, 2015 and in the Company's other periodic filings with the Securities and Exchange Commission (the “SEC”) which are available for review at www.sec.gov under “Search for Company Filings” could cause actual results and developments to be materially different from those expressed or implied by such statements. All forward-looking statements attributable to us are expressly qualified in their entirety by these and other factors. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by law, the Company undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.