SCD Community Forum II Webinar May 9th, 2016 7pm EST

In Prov 29:18 we are reminded that, "Where there is no vision, the people perish..."  I can't think of a more appropriate precept relevant for the SCD Community than this. I'm talking to those affected directly and indirectly by SCD. Why? because, we're already aware of SCD. We're dealing with its effects on a daily basis. Yet again scripture reminds us that even in this state we can look for help; Psalm 121:1 states (in my paraphrased version) "...from whence cometh my help? My help cometh from the Lord!

So what does this inspirational scripture have to do with the webinar alluded to in the title?

Well, the SCD Community Forum is a series of events involving the stakeholders of sickle cell disease: Patients, Caregivers, Advocates, Community Based Organizations (CBOs) and importantly the Faith Based Organizations (FBOs). On May 9th at 7pm EST a Webinar will be held to provide current information from a panel of experts on various topics as well as take live online questions.

The FBO role in the SCD community is foundational. Their mission includes outreach to those afflicted by malady and mistrust. Yet often, the needs of SCD patients and caregivers can be overwhelming. So this Forum series includes a special charge to the FBO to be more effective to their SCD community, particularly beyond the walls of the institution.  Go to the link above    (or copy and paste it into your web browser)  to register for this free webinar and please share with other members of our extended community.

These Last Few Weeks

 I can't believe how time has flown by these last few weeks. In a nutshell, I've been travelling, a lot:

1.) WORLD Symposium 2/27 - 3/5 in San Diego a meeting of industry and researchers of a group of rare diseases known as lysosomal storage disorders;

2.) Mast Therapeutics Headquarters 3/3 just North of San Diego. Met with Company senior leadership. Mast is awaiting topline results from their Phase III trial of vepoloxamer. This was the largest trial ever conducted in SCD patients with VOC (vaso occlusive crises); 

3.) Emmaus Pharmaceuticals Headquarters 3/4 Torrance, CA. Met with Company senior leadership. Emmaus is awaiting approval of their pharmaceutical grade L-glutamine for the management of patients with SCD;

4.) Sickle Cell Community Consortium 3/15 - 3/19 Atlanta, GA. This was the inaugural meeting of the SCCC a national umbrella organization on behalf of local CBOs from 17+ States and Canada that advocate for the SCD Community. I serve as Director of Development;

5.) PCORNET Trustworthiness Meeting 3/28 - 3/29 Washington D.C.  This was a meeting of researchers and advocates in the area of comparative effectiveness. Networked and interacted with parties in position to conduct, host, or advise on the psychosocial research of the SCD community;

6.) 8th Annual Maryland Legislative SCD Awareness Event 4/5 Annapolis, MD. This meeting, coordinated by 5 MD area based SCD Community Based Organizations, updated the Maryland Senators and Delegates about the progress and concerns of the SCD community. Of note Senator Nathan-Pulliam, RN received a favorable vote for her legislation, SB 459. This will study the Cost Benefit of an Adult Sickle Cell Infusion Clinic. Recommendation due to the Governor by 12/1/16.

The Next New SCD Treatment; Quite Possibly

I was encouraged to learn of a milestone achievement today. Mast Therapeutics of San Diego  announced it has completed enrollment in its Phase III SCD EPIC trial. This trial is studying a new drug known as MST-188 or vepoloxamer in SCD patients experiencing pain crisis. By completing enrollment the company is one step closer to filing a new drug application. And because the FDA previously designated the drug candidate as an orphan designation, that places the application in position for not only accelerated approval but special marketing exclusivity. The next step is for the company to analyze the data and the expected timetable for top line results is sometime during the second quarter of 2016. Stay tuned for the top line results announcement.

What Does a Patient Advocate Do For SCD?

I'm often asked , what is Patient Advocacy and how does it work for sickle cell disease patients. Allow me to share some of the work done behind the scenes to provide an example of SCD patient advocacy on a national level. Following are excerpts from a letter I wrote to the then FDA Commissioner, Margaret Hamburg about how the bio/pharmaceuticals companies could be encouraged to develop SCD drugs with something known as a priority review voucher:

"Dear Commissioner Hamburg,  As a parent of a young adult son with SCD and a former biotech industry marketer of orphan drugs, I have a unique perspective about the relevance of a Rare Pediatric Diseases (RPDs) designation for SCD (ref. docket number FDA-2014-D-1461). Primarily, the Priority Review Voucher (PRV) for RPDs provides a powerful stimulus for seeking  more and better SCD treatments.

In 2012 Congress enacted the Creating Hope Act backed by a bipartisan coalition of lawmakers and championed by Congressman Butterfield (Dem - NC). This law encouraged the bio/pharmaceutical industry to develop therapies for rare pediatric diseases.  Known as the Rare Pediatric Disease Priority Review Voucher or RPV, this provides industry with a unique tool, the right to receive an advance to the front of the line for review of a drug candidate application if the disease in question was considered to be rare and affecting the pediatric population.                                                                                                                                                                                                                                                                                                                   New SCD Therapies Needed
Clearly, there is an urgent need for new approaches and new therapies. After all, the medical literature reports an understanding of the underlying problem in SCD that goes back over 100 years.  However, the bio/pharmaceutical industry lacks incentives for developing treatments. So that should there be any drug R&D, the industry will settle for the lowest standards in their clinical development programs. There may be drugs approved but they will be limited to trading off a day or so in the hospital on opioids versus interrupting or reversing the underlying pathology and other dimensions of SCD captured in patient reported outcomes.                                                                                                                                                    

The image of acute painful vaso-occlusive crises (VOCs) in SCD is tragic but just the tip of the iceberg. Below the surface the full burden of disease is more complex. Because the end organ damage from the ischemia of compromised blood flow and oxygenation is the real pathology of concern. The processes involved in preventing damage to tissues and organs and restoring function should be the focus of future treatments along with widely available cures. After all would it be appropriate to manage a heart attack or stroke with IVs and pain meds? Yet that, in effect, is how SCD VOC crises are managed. Crisis management has its place but the focus needs to be on innovation.                               

New Treatments, Better Treatments for SCD

The New Research on SCD
There is a renaissance of drug development in SCD. There are proposed mechanisms of action that are novel. They improve the dynamics of blood flow, interrupt the clot forming reactions of VOC, reduce the body’s insult from VOC’s, provide alternative oxygen transport and other approaches like gene therapy to correct the genetic defect. These go beyond current hydration and opioid pain management. Collectively, these and other successful drug candidates may do for SCD what “cocktail” therapy has done for cancer and HIV/AIDS.  Stay tuned to this Blog for ongoing updates on the development progress of this new category of treatments for SCD.