NeuralStem and the ‘Right-To Try’

Colorado has recently passed ‘Right-to-Try’ (RTT) legislation permitting companies to offer experimental treatments for fatal diseases that have not been FDA approved—and the requirement of payment is permitted if the Company so chooses. The criterion is that they have reached Phase II testing, the law based largely on a framework developed by the libertarian Goldwater Institute. The first CNS company to embrace this concept is, not surprisingly, NeuralStem, whose CEO, Richard  Garr, is head of the Goldwater Institute’s Advisory Council on this topic, and who undoubtedly played a role in the RTT legislation design. Garr very quickly (on June 5) told BioCentury that NeuralStem would begin training Colorado neurosurgeons to administer NeuralStem’s stem cells (NSI-566), which are implanted in the spinal cord; that no permission would be sought from the FDA; and the decision of whether to charge for the treatment had not yet been made.
By the next day, Garr had published a comment on his blog that backpedaled somewhat, quoting an earlier statement by FDA Commissioner Margaret Hamburg that expressed support for making treatments available to patients with life-threatening illness, and stating that NeuralStem would not make a treatment available under RTT if the FDA told them not to. We suspect that his BOD, and perhaps NeuralStem’s legal counsel, may have suggested that his initial stance might have come across as unwisely cavalier, given that federal oversight trumps state control of such issues, as Massachusetts Governor Deval Patrick learned when he tried to outlaw Zohydro in March.

NIR has a profoundly ambivalent view of this issue. On the one hand, patients with ALS have but a few years of life expectancy, and the gradual loss of basic motor functions–including respiration–does not imbue those years with much of what is generally considered to be ‘quality-of-life’ (this is not to diminish the value of what their lives can still include). To put it another way, longterm safety issues would be a good problem to have. Such treatment is an option we ourselves would want to have available, our choice to make.

Having said that, there is another side to the argument. On June 15, BioCentury produced a television segment on this issue, NeuralStem’s Garr did not appear–we have no doubt he was asked–which speaks to some newly adopted caution. However, Lucy Walker, a spokesperson for the Goldwater Institute, said in reference to NSI-566: “That drug has an 85% efficacy rate in trials.” Which nicely illustrates the slippery slope on accuracy in this scenario:  NSI-566 is a cell therapy, not a drug, and while NeuralStem has aggressively highlighted interim and sometimes anecdotal clinical status reports for NSI-566 trial subjects, NSI-566 by no means has established a “85% efficacy rate”. But any ALS patient with access to air travel could be forgiven for seeing this as an opportunity to be seized  whatever the cost, if indeed NeuralStem decides to charge for this technically challenging therapy.

Given that NeuralStem has already decided to sponsor clinical trials of its cell therapy in China and Mexico that are not FDA-vetted, they are already–in our view–walking a tightrope with the FDA. Getting out in front of the RTT movement risks NeuralStem being cast as a Rebel Without A Regulator, which may be why one cannot find anything on the NeuralStem website or Garr’s blog that repeats his more brash stance of June 5.

We are reminded of the fetal stem cell era in the treatment of Parkinson’s, when a few US neurosurgeons, operating under their Physician INDs, carried out cell implantations on patients who were able to pay the $50,000 and up rate. In the long run, some of these patients had a serious worsening of their dyskinesias. One can argue that ALS’s time course is so much shorter (and NSI-566 far better vetted) that this is a risk that patients should be able to take if they so wish, but this is a reminder that there are ethical, clinical, and regulatory downsides to this endeavor. We would hate to see the FDA back track on Hamburg’s commendable shift towards espousing easier patient access if they perceive  any mercenary exploitation (we are not claiming that NeuralStem is doing so–but they could easily be seen as pushing the envelope) in response to the loosening of controls at the federal or state level.

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