The Life Sciences Report: Josh Levine Names Three Biotech Game-Changers
TLSR: Josh, what's your next idea?
JL: The third would be Neuralstem Inc. (CUR:NYSE.MKT). It also has a very compelling technology platform. The company is in the stem cells space, but unlike other stem cell companies it has a unique approach in a number of ways. Most importantly, Neuralstem has created an entire bank of proprietary neural stem cells by isolating stem cells from the brains and spinal cords of aborted fetuses. Based on its successful phase 1 trial for amyotrophic lateral sclerosis (ALS) the company's hope is that cells injected into the spinal cords of patients will provide support, perhaps by releasing growth factors, and prevent motor neurons from dying.
TLSR: These are adult stem cells of embryonic origin, but do they maintain their immune privilege as they do during a pregnancy? In other words, I'm wondering if these patients may have to go on immunosuppressants when they receive these cell transplants.
JL: It's a good question. In fact, with regard to Neuralstem's phase 1 trial for ALS, researchers discovered that patients could not tolerate immunosuppressants prescribed after surgery and most of them had to drop or reduce the dosage of at least one of the two anti-rejection medicines.
"The most important part of IP is what's inside the heads of the people who work for the company."Interestingly, by using a process of "DNA fingerprinting," researchers found that among the five patients who had discontinued all suppression meds for 57 to 638 days prior to death, there was no correlation of DNA content to survival period without immune-suppression drugs. Neuralstem reports that these data suggest that long-term immunosuppression of patients is not required for long-term survival of its cells, which may lead to only transient use of these drugs in future ALS trials.
TLSR: One of the 15 ALS patients from this phase 1 trial has talked to media because he is doing quite well. Even though this trial is a safety-and-dose study, and not powered to determine efficacy, we may be seeing something interesting happen here.
JL: That is true. The patient has talked to the press; he is the only one who has. He's had quite stellar results. The therapy not only halted his ALS, but his symptoms seem to have reversed in a disease that does not reverse. Now, this is just one patient. There's no way to know what's going to happen beyond that. But this patient has really stunned researchers and others looking at the company's technology. It's certainly promising, and great for this patient and his family.
TLSR: I realize that Neuralstem didn't take this story to the media, but the company has put it on its website as part of the news flow. Do you think this could be attracting an unsophisticated retail investorship in the company?
JL: There's always the possibility, but I don't think we have seen that happening. And remember, the company is not out promoting this. In fact, I don't know if you saw CEO Richard Garr's Dec. 4 blog post, in which he addressed this. He stated that this is only a single patient in a longer-term phase 1 trial. He talked about the FDA protocols because of the novelty of Neuralstem's approach, noted that this is only interim data, and said that though something like this can happen, people shouldn't expect this to be the norm.
Garr also pointed out—and I think very importantly—that ALS is a one-direction disease. It is a downward spiral to a terrible death. The other point he made is that people shouldn't expect Neuralstem's therapy to be a cure. If it can halt ALS in its tracks or slow it down, it would be an enormous success.