FDA & CLINICAL TRIALS

Understanding

the FDA process for Regenerative Medicine
and especially, the highly anticipated 
induced pluripotent (iPS) cell-based 
replacement therapy for Parkinson's disease.

Updated: 03/20/2020

The FDA approved clinical trial process cannot begin until the research for Drs. Loring's and Bratt-Leai's  autologous dopamine neuron replacement therapy is completed. It is difficult to predict with any certainty when the clinical trials will begin. In the past we have shared a estimated timeline based on the information we had at the time. The timeline has continually been adjusted due to various reasons. We realize this had been frustrating. Please understand that it is not the FDA, scientists nor Summit that is the  "bad guy" in this scenario. for those with Parkinson's disease it is time that is the foe. Everyone involved with the research project has and continues to work with diligence for its successful availability to all who desire it safely.


With that in mind, below is some information to help you understand the FDA's and its efforts for a safe and dependable therapy...

                            For REFERENCE: The typical timeline for a pharmaceutical drug to be publicly accessible.

                            The process for a pharmaceutical drug to receive FDA approval to begin clinical trials

                            typically takes 12+ years at an average cost of US$350 million. Once FDA approval is

                            received, the “investigative drug” then begins three phases of clinical trials:

                                         Phase I - Establishing a drug’s safety and profile (approximately 1 year).

                                         Phase II – Assess a drug’s effectiveness (approximately 2 years).

                                         Phase III – Determining a drug’s effectiveness and identify adverse reactions

                                                           (about 3 years).

                            Once Phases I,II, & III are completed, a 100,000+ page application is submitted for

                            processing which usually takes an additional 2.5+ years for the drug to receive final FDA

                            approval for physicians to prescribe.

 

 

However, the FDA is working to meet the unmet needs of diseases with no cures. Parkinson’s disease is a disease with unmet medical needs. Although the iPS cell-based therapy has not received its classification as of this date, the FDA has developed a new policies to address the pace at which scientific discoveries are being made today.

Aspen Neuroscience is currently working with a newer FDA division called: “Center for Biologics Evaluation and Research (CBER)”. CBER is working with the Regenerative Medicine field to develop new policies to “expedite” stem cell-based therapies to meet patient’s needs and to meet the challenging pace of discoveries in science present. 

                            Fast Track Designation: to facilitate the development and expedite review of drugs and

                            biologics intended to treat serious or life-threatening conditions and that demonstrate the

                            potential to address unmet medical needs. 

 

 

 

                            The 21st Century Cures Act: modifies the FDA Approval process. The development

                            and bring new innovations and advances to patients who need them faster and more

                            efficiently.

 

 

                            RMAT designation: Regenerative Medicine Advanced Therapy (RMAT): a drug is eligible

                            for regenerative medicine advanced therapy (RMAT) designation if:

                                      1. The drug is a regenerative medicine therapy, which is defined as a cell therapy,

                                           therapeutic tissue engineering product, human cell and tissue product, or any

                                           combination product using such therapies or products meeting FDA criteria.

                                       2. The drug is intended to treat, modify, reverse, or cure a serious or life-threatening

                                           disease or condition; and 

                                       3. Preliminary clinical evidence indicates that the drug has the potential to address

                                           unmet medical needs for such disease or condition.

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