DISEASE-A-WEEK CHALLENGE: California vs. Chronic Disease

By Don C. Reed

Did you know October 14th is Stem Cell Awareness Day?

It is no accident that I requested that date to be the official publication day for my new book, STEM CELL BATTLES: Proposition 71 and Beyond. https://www.worldscientific.com/worldscibooks/10.1142/9255.html.

I wrote the book so people could know the accomplishments (and the tremendous struggles) of the California stem cell program, which began as the citizens’ initiative, Proposition 71, the Stem Cells for Research and Cures Initiative.

Every Wednesday between now and then, (starting May 27), I will produce a “Disease-a-week” summary of the amazing work the program is doing on one “incurable” illness or injury.

Below is a preview: a list of 21 diseases and disabilities that will be featured: samples of the amazing California stem cell program in action.

If one of these conditions affects someone you love, mark the date on your calendar, and check back for the article at this website, or my personal weblog, WWW.STEMCELLBATTLES.COM

Quotes are drawn from CIRM’s public website: www.cirm.ca.gov. Words in parentheses (like these) are mine. Please note: I have no official connection with the California stem cell program, and am neither doctor nor scientist: just a father of a paralyzed young man, Roman Reed, whose courage inspires me every day. We are determined there will be cure, but it will only happen if the field advances.

Want to help fight chronic disease? Forward this to your social networks: Twitter, Facebook, GooglePlus– , and especially any patient or disease organizations to which you may belong. Thanks!!

May 27: ALS (Lou Gehrig’s Disease) “…Some projects (seek to) understand the origin of the disease and what causes the (the death of) motor neurons … Cells (astrocytes) secrete a chemical that damages the neurons. (CIRM-funded scientists) learned how to take… cells and turn them into motor neurons (for replacement) and the astrocytes (which may be part of the problem)…”

June 3: Alzheimer’s Disease: “Using cells from Alzheimer’s patients …researchers are creating…models of the disease in a lab dish. They can then test for drugs… that eliminate symptoms of the disease…some groups are trying to (turn) embryonic stem cells into a cell type that can… replace cells destroyed…. Stem cells (may) deliver factors (to) protect brain cells…(or, like tiny brooms) clear out… protein that builds up and clogs neurons”.

June  10:  Arterial Limb Disease: “Hardening of the arteries…may result in amputation due to wounds that refuse to heal because of poor blood circulation…Researchers (hope) to coax the patients’ bodies into growing new blood vessels…

June  17: Arthritis: “ a disabling condition that afflicts 6 million Californians, costing nearly $32 billion annually for health care and lost wages… Stem cell therapies (may be used) for cartilage repair… A patient’s own cells…(may grow new) cartilage and prevent arthritis progression…”

June 24: Autism: “…CIRM (is) investigating how the brain matures… studying stem cells as they… change into different types of nerves… to learn how diseases such as autism form, and develop future therapies.  Several CIRM-funded projects (use) stem cells derived from people with autism… Scientists can expose those cells to drugs and see which one alleviates symptoms….”

July 1: Blindness: “The leading cause of blindness…is Age-related Macular Degeneration (AMD)…In AMD the layer of cells that support photoreceptors  (light-sensing cells) is destroyed…. CIRM-funded teams are (trying to replace) these support cells….”

July 8: Segmental bone fracture: “… a complex medical condition (causing) great suffering to patients, long-term hospitalization, repeated surgeries, loss of working days, and considerable costs to the health system. …(One) approach for treatment might utilize ultrasound to deliver a bone-forming gene to stem cells. …(and) trigger the stem cells to (grow) bone…”

July 15: Cancer: Brain Tumor:  “…malignant brain tumors (are) hard to treat because…(they) spread throughout the brain… (However) certain types of stem cells migrate toward these tumor cells… CRM scientists are trying to engineer these stem cells to produce cancer-killing molecules. …These would seek out cancer cells and deliver therapy where needed…”

July 22: Cancer: Melanoma: …When (melanoma) spreads… it becomes highly resistant to most current therapies… (A healthy body has) specialized cells in our immune system…(which) seek out and destroy cancer cells. But when melanoma (spreads)  there are not enough of these cells. Since the cells use a protein called the T-Cell Receptor (TCR) to seek out …cancers…  (Adding) TCR genes to a group of immune cells (may) boost their cancer-fighting abilities…”

July 29:  Cancer: Solid Tumor: “Solid tumors include cancers of the brain, ovary, breast, colon and other tissues…. (growing from) cancer stem cells. …Chemotherapy kills (most) of the tumor, but leaves behind the cancer stem cells that can…form a new tumor. Stem cell scientists are studying (these) cancer stem cells…. If cancer stem cells (from different parts of the body) share characteristics that allow them to be destroyed by the same drug, then a single new drug could significantly improve cancer treatment for a range of different cancer types….”

August 5: Deafness: “…Approximately 36 million American adults have hearing loss. (A) common cause of deafness occurs when the cells that detect sound in the inner ear… lose their function.  These cells contain… hair-like structures that turn sound into electrical signals (which) the brain interprets as sound. If the hairs are damaged … they no longer transmit sounds to the brain. Researchers… coax stem cells to form these hair-like structures in the lab…. Hair cells (made) from stem cells could replace the damaged cells and restore hearing…”

August 12:  Diabetes:  “As many as 3 million people in U.S. have Type 1 diabetes… a disorder where the body’s immune system destroys cells in the pancreas that make insulin.  …Without insulin…sugar builds up in the bloodstream (and) can damage the kidneys, blood vessels and retina. … replacement insulin-producing cells (can be) derived from human embryonic stem cells. (which may be able to regulate blood sugar.  CIRM-funded teams have placed cells in a device (put)  under the skin (to) shield the cells from the immune system…”.

August 19: Epidermolysis Bullosa: “(EB is) a devastating  skin disease in which the protein that normally anchors the skin to the underlying tissue is absent. …The slightest friction can rub skin off. Children with the most severe form of the disease develop disfiguring scar tissue and often die as young adults…   The goal of a stem cell therapy is to replace the protein that is missing…(One approach) is to genetically engineer a patient’s own skin cells to contain the protein…”

August 26:  HIV/AIDS: “HIV…is a virus that infects cells of the immune system, undermining the body’s ability to fight disease…(causing) susceptibility to infections, cancers and other diseases, and eventually… death. … A gene called CCR5, ( lets) HIV enter cells. Scientists hope to create   CCR5 mutations. (These) genetically altered cells would then repopulate the person’s blood system with one that lacks CCR5…(thereby preventing HIV from getting in.)”

September 2: Huntington’s Disease: “…The disease is uniformly fatal, usually in 10-20 years. HD results from a mutation to one gene…(which) produces a protein that is toxic to nerve cells. California has funded several projects that probe into both the nature of the mutation and ways to prevent or repair the damage from (it)….  One team proposes to use embryonic stem cells, to mature them into early forms of  nerve cells and genetically modify them to deliver a compound that could protect the patient… from the toxic protein.  The other team plans to use mesenchymal stem cells to deliver a genetic fragment called iRNA to the nerves and shut down the faulty gene…”

September 9:  Multiple Sclerosis: “…occurs when body’s immune system attacks the (myelin) cells that protect neurons… As people lose myelin…, they start feeling weak…. Over  time…paralysis. About 400,000 people are living with MS in US.  One approach may be to repopulate the person’s blood system with  immune cells that won’t attack the myelin. (Or to)… find drugs that could stimulate the body’s own stem cells to replace the damaged myelin.”

September 16: Osteoporosis: “(This bone-thinning condition causes nearly) 2 million women…to develop bone fractures each year…  (Good) bone-forming stem cells decrease tenfold between ages of 30-80, (and those remaining) are less effective at repairing bone. CIRM (seeks to) increase number of these stem cells or improve their effectiveness to keep bones healthier longer. One team is testing bone-forming stem cells in animal models and revving up those cells with a hormone… Another group has isolated (from fat tissue)…a stem cell…capable of forming bone…”

September 23: Parkinson’s Disease(PD):  … (PD) occurs when (nerve cells) stop functioning in the part of the brain that controls movement.  (In a healthy body) these nerve cells send signals using a molecule called dopamine… CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, …then coaxed those cells to become the type of neuron affected by PD. Now (scientists can) expose those cells to different drugs to find which ones eliminate disease….”

September 30:  Respiratory Disorders: “Severe blockage of major airway (trachea) is rare but… (can cause) death.  …Physicians currently treat these blockages through surgery or by using a stent to hold the airway open.  Neither approach is very good…. UC Davis team uses a trachea from a cadaver as a scaffold, (first) removing the soft tissue cells and then seeding the…scaffold with two types of stem cells from the patient.  The (resulting) construct is grown in a bioreactor until ready for transplant…the team has already used the procedure through the European compassionate use exemption in five dying patients, saving three of their lives…”

Otober 7:  Sickle Cell Disease: “…is a genetic disorder that causes red blood cells to assume a sickle shape, clogging blood vessels with episodes of excruciating pain, and progressive organ damage. …Researchers intend to remove bone marrow from the patient and fix the genetic defect in the blood-forming stem cells. These cells can be (put back) into the patient (for) a healthy blood system.

October 14: Spinal Cord Injury: “California’s stem cell agency retains many grants for research to move potential… therapy forward…(One is the former Geron embryonic stem cell trial, now moving forward under Asterias Biotherapeutics.) Much work tries to determine which type of nerve cell is the best to transplant… Other research is to see if transplanted cells become part of the existing nerve system…  One obstacle …is the scar at the site of injury (which may) block the growth of transplanted cells. …Synthetic scaffolds…may help the cells bridge the scar…”

* Information on the author’s forthcoming book: STEM CELL BATTLES: Proposition 71 and Beyond: How Ordinary People Can Fight Back Against the Crushing Burden of Chronic Disease (With a Posthumous Forward by Christopher Reeve) can be found at:


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