Brief Answer:
Research for Bulbar Palsy

Detailed Answer:
Good evening. I am an Adult Neurologist from the XXXXXXX area of the world. I've read the responses given to you for your wife's medical condition and understand you are satisfied with Dr. Ulabidin's suggestions and recommendations.

Your current query is one to uncover where clinical research is being done on Bulbar Palsy. Is your wife interested in being recruited into such research? What has your doctor from UCSF said about this to you? And what has your investigation turned up so you are no doubt doing your best to discover available options. I understand you live in Rural Nevada....To an Midwest, Great Lakes Region resident who's actually never been outside the airport in Las Vegas, rural Nevada means, DESERT. LOL......

Are you familiar with the following website and organization: National Center for Advancing Translational Services? It's where patients can actually look to matchup their conditions with active research going on in the U.S. which is known to the NIH (National Institute of Health) and administered through the ORDS (Office of Rare Diseases Research).

https://rarediseases.info.nih.gov/gard/10928/progressive-bulbar-palsy/resources/6


Now, if you are unable to specifically match your wife to a particular study then, look at the following information and multiple places to inquire about ongoing research trials which may be interested in enrolling your wife (I believe your wife is receiving stem cell therapy?). Keep in mind that I've also included some sites that are dedicated to ALS research. I'm assuming that you are interested in ALL reasonable possibilities of getting help for your wife although it is clearly controversial as to whether or not true autoimmune PBP is either a form of or related to ALS. Also, I've included sites having to do with Muscular Dystrophy, Spinal Muscular Atrophy, and Post Polio research. You never know which group (even if in an unrelated disease entity) may be doing trials that overlap with clinical features your wife may have. Therefore, getting the research inclusion and exclusion criteria may be worthwhile.

I include all the sites for completeness:

Research options fall largely into three categories: drugs, gene therapy, and stem cells.

Clinical trials are testing whether different drugs or interventions are safe and effective in slowing the progression of MNDs in patient volunteers.

1. The antibiotic ceftriaxone has been shown to protect nerves by reducing glutamate toxicity—believed by many scientists to play a critical role in the development of ALS—in a mouse model of the disease. One study found that cellular ability to manage glutamate can alter the course of ALS. The drug is currently being tested in a NINDS-sponsored multi-center human clinical trial.

2. The novel compound dexpramipexole has shown neuroprotective properties in multiple preclinical studies of ALS, and may work by increasing the efficiency of mitochondria—the energy-producing portion of the body’s cells. Mitochondria in the motor neurons undergo significant stress in ALS patients. The compound is currently being tested in an industry-sponsored multi-center clinical trial.


3. Several early-stage clinical trials are testing the safety and feasibility of novel treatment strategies for ALS. These include cell-based approaches such as the transplantation of neural precursor cells into the spinal cord of ALS patients, and the infusion of so-called “anti-sense” compounds into the fluid that surrounds the spinal cord and brain to block production of toxic SOD1 protein in ALS patients who carry SOD1 mutations.

4. Cellular and molecular studies seek to understand the mechanisms that trigger motor neurons to degenerate. Examples include the following:

For more information on neurological disorders or research programs funded by the National Institute of Neurological Disorders and Stroke, contact the Institute's Brain Resources and Information Network (BRAIN) at:

BRAIN
P.O. Box 5801
Bethesda, MD 20824
(800) 352-9424
http://www.ninds.nih.gov

Information also is available from the following organizations:
ALS Association
1275 K Street, N.W.
Suite 250 XXXXXXX DC 20005
YYYY@YYYY
http://www.alsa.org External link
Tel: 202-407-8580
Fax: 202-289-6801

Muscular Dystrophy Association
National Office - 222 S. XXXXXXX Plaza
Suite 1500 XXXXXXX IL 60606
YYYY@YYYY
http://www.mda.org External link
Tel: 800-572-1717
Fax: 520-529-5300

ALS Therapy Development Institute
300 Technology Square
Suite 400 XXXXXXX MA 02139
YYYY@YYYY
http://www.als.net External link
Tel: 617-441-7200
Fax: 617-441-7299
XXXXXXX XXXXXXX ALS Foundation
5550 W. Touhy Avenue
Suite 302
Skokie, IL 60077-3254
YYYY@YYYY
http://www.lesturnerals.org External link
Tel: 888-ALS-1107 847-679-3311
Fax: 847-679-9109

Project ALS
3960 Broadway
Suite 420
New York, NY 10032
YYYY@YYYY
http://www.projectals.org External link
Tel: 212-420-7382 800-603-0270
Fax: 212-420-7387

Spastic Paraplegia Foundation
P.O. Box 1208
Fortson, GA 31808
YYYY@YYYY
http://www.sp-foundation.org External link
Tel: 1-877-SPF-GIVE (1-877-773-4483)
Fax: 877-SPF-GIVE

Cure SMA
925 Busse Road
Elk Grove Village, IL 60007
YYYY@YYYY
http://www.curesma.org External link
Tel: 800-886-1762
Fax: 847-367-7623

Fight SMA
1321 Duke Street
Suite 304 XXXXXXX VA 22134
YYYY@YYYY
http://www.fightsma.org External link
Tel: 703-299-1144

Kennedy's Disease Association
P.O. Box 1105
Coarsegold, CA 93614-1105
YYYY@YYYY
http://www.kennedysdisease.org External link
Tel: 559-658-5950

Spinal Muscular Atrophy Foundation
888 Seventh Avenue
Suite 400
New York, NY 10019
YYYY@YYYY
http://www.smafoundation.org External link
Tel: 877-FUND-SMA (877-386-3762) 646-253-7100
Fax: 212-247-3079

Post-Polio Health International
4207 Lindell Blvd.
#110
St. XXXXXXX MO 63108-2930
YYYY@YYYY
http://www.post-polio.org External link
Tel: 314-534-0475
Fax: 314-534-5070


I hope this answer satisfactorily addresses your question. If so, may I ask your favor of a HIGH STAR RATING with written feedback?

Also, if there are no other questions or comments, I'd appreciate it greatly if you would CLOSE THE QUERY on your end so this question can be transacted and archived for further reference by colleagues as necessary.

Please direct more comments and questions to me in the future at:

bit.ly/drdariushsaghafi and I would be honored to answer you and continue this discussion.

All the best.

The query has required a total of 55 minutes of physician specific time to read, research, and compile a return envoy to the patient.

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